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13165	ASPIRE Design Challenge 4: Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose	Summary of NCATS ASPIRE Design ChallengesSummary of NCATS ASPIRE Design Challenge 4: Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and OverdoseHow to EnterDates and DeadlinesThe IC’s Statutory Authority to Conduct the ChallengeSubject of the Challenge CompetitionConcurrent Companion NCATS ASPIRE Design ChallengesRegistration Process for InnovatorsThe PrizeEvaluation and Winner SelectionBasis upon Which Submissions Will Be EvaluatedSummary of NCATS ASPIRE Design ChallengesThe National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), is inviting novel design solutions for A Specialized Platform for Innovative Research Exploration (NCATS ASPIRE) Design Challenges as part of the NCATS ASPIRE Program. The goal of the NCATS ASPIRE Design Challenges is to reward and spur innovative and catalytic approaches toward solving the opioid crisis through development of (1) novel chemistries, (2) data mining and analysis tools and technologies, and (3) biological assays that will revolutionize discovery, development and preclinical testing of next-generation, safer and non-addictive analgesics to treat pain, as well as new treatments for opioid use disorder (OUD) and overdose. The first phase of these prize competitions is implemented through a suite of concurrent companion Design Challenges that comprises separate Challenges for each of four areas — chemistry database, electronic laboratory knowledge portal for synthetic chemistry, algorithms and biological assays — and an additional Challenge for a combined solution to at least two Challenge areas. At this stage, innovators are expected to submit designs, not final products or prototypes.NCATS envisions following these Design Challenges with a follow-on but distinct final Reduction-to-Practice Challenge, which will aim to invoke further scientific and technological development of the model system. Winners of the Design Challenges will be invited to present their designs so that, in the envisioned follow-up Reduction-to-Practice Challenge, an open competition, teams will be able to form multidisciplinary collaborations to advance and integrate the most feasible and promising approaches to the multiple Challenges into a single integrative platform. Innovators will be invited to demonstrate final solutions.The NCATS ASPIRE Design Challenges are part of NIH’s Helping to End Addiction Long-term (HEAL) initiative to speed scientific solutions to the national opioid public health crisis. The NIH HEAL Initiative will bolster research across NIH to (1) improve treatment for opioid misuse and addiction and (2) enhance pain management. More information about the HEAL Initiative is available at https://www.nih.gov/research-training/medical-research-initiatives/heal-initiative.NCATS refers to participants in the NCATS ASPIRE Design Challenges as “innovators,” because all solutions will require highly innovative approaches to achieve success. Innovators should clearly state how and why the proposed solution would provide significant advances over currently available tools. Innovators may choose to compete in one or more individual Challenges to address a single area (Challenges 1-4) or propose a combined solution for at least two Challenge areas (Challenge 5).Back to topSummary of NCATS ASPIRE Design Challenge 4: Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose Challenge 4 aims to address the need for novel, physiologically relevant biological assays that can accurately replicate the safety profile and effectiveness of existing drugs to treat addiction and/or overdose and that can be reliably used in predictive assessments of new analgesics or drugs to treat addiction and/or overdose and/or be able to anticipate the degree of addictiveness of an analgesic prior to clinical testing. This Challenge requires submission of only a detailed description of the design of the assay(s), not the final working assay(s).Evaluation criteria that reviewers will be asked to address are specified below.Back to topDates and DeadlinesSolutions must be submitted to Challenge.gov by NOON Eastern Time on May 31, 2019. The Challenge begins: December 31, 2018 Submission period: December 31, 2018-May 31, 2019Judging period: June 17, 2019-August 2, 2019Winners announced: August 2019For further information send an email to NCATSASPIREChallenge@mail.nih.gov.Back to topThe IC’s Statutory Authority to Conduct the ChallengeThe general purpose of NCATS is to coordinate and develop resources that leverage basic research in support of translational science and to develop partnerships and work cooperatively to foster synergy in ways that do not create duplication, redundancy and competition with industry activities (42 USC 287(a)). In order to fulfill its mission, NCATS supports projects that will transform the translational process so that new treatments and cures for diseases can be delivered to patients faster by understanding the translational process in order to create a basis for more science-driven, predictive and effective intervention development for the prevention and treatment of all diseases. NCATS is also conducting this Challenge under the America Creating Opportunities to Meaningfully Promote Excellence in Technology, Education, and Science (COMPETES) Reauthorization Act of 2010, 15 U.S.C. 3719. In line with these authorities, this Challenge(s) will lead to innovative designs for developing technology to revolutionize discovery, development and preclinical testing of new and safer treatments of pain, opioid use disorder (OUD), and overdose; the result will be generalizable tools that will be widely available to fill longstanding gaps that have impeded the marriage of basic and translational sciences.Back to topSubject of the Challenge CompetitionChallenge 4. Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose. This Challenge aims to reward and spur innovative solutions to the development of a novel physiologically relevant biological assay to advance preclinical discovery and development of non-addictive treatments for pain, drug addiction or overdoses. This Challenge requires submission of only a detailed description of the design of the assay(s), not the final working assay(s). Current in vitro assays and in vivo models to study pain and addiction and test potential treatments are very limited. Pharmacological probe, lead and drug development have traditionally utilized canonical cell lines (such as CHO or HEK293) that heterologously express the molecular target of interest, and studies that are performed in animal models often do not fully recapitulate human physiology and may identify candidate compounds without effects in more human physiology-relevant systems. Therefore, NCATS is inviting innovators to propose and develop novel “disease-in-a-dish” assays of pain perception, addiction or overdose that advance the understanding of different types of pain, identify differences in inter- and intra-individual pain and the associated risk of developing chronic pain and/or addiction and make possible the development of improved, non-addictive drugs to treat these conditions. 2D and 3D human disease-relevant screening platforms may include but are not limited to normal and diseased human iPSC-derived sensory/pain neurons (such as peripheral, dorsal root ganglia [DRG], spinal cord and thalamus); neurons relevant to reward pathways; and other tissues important for pain, addiction and overdose, including blood-brain barrier (BBB). The assays will be adjudicated on their physiological relevance, robustness and reproducibility, and their future potential to be amended to automation and scalability for medium to high-throughput screening assays. Initially, the assay can be designed for low-throughput screening; it is expected that even at low throughput, multiple technical replicates will be compared in the same experiment. Eventually, the assay should be amenable to automation and scalable to medium- (96-well plate) to high-throughput screening (using, for example, high-content imaging, drug validation/toxicity, functional genomic screening).The innovators are encouraged to consult NCATS’ Assay Guidance Manual to learn more about appropriate designing of a drug screening assay.For the envisioned Reduction-to-Practice phase, the innovators will be expected to demonstrate the viability rate of cells in 2D or 3D models, at a particular throughput (90% viable), and provide data indicating how the functional morphological features are maintained in the system over time.Back to topConcurrent Companion NCATS ASPIRE Design ChallengesNCATS has recently explored the development of A Specialized Platform for Innovative Research Exploration (ASPIRE) to aid in the discovery and development of novel and effective treatments while at the same time making the process faster and more cost-effective. The NCATS ASPIRE Program aims to develop and integrate automated synthetic chemistry, biological screening and artificial intelligence approaches in order to significantly advance our understanding of the relationship between chemical and biological space and enable further access into biologically relevant chemical space. The platform will utilize currently available knowledge to develop innovative algorithms and predict and synthetize novel structures capable of interacting with specific targets; enable small-scale synthesis of the predicted molecules; and incorporate in-line, rapid biological testing of the molecules. Any new data obtained through this process would then be fed back into the system to further improve design, synthesis and biological characteristics of molecules.Over 25 million people in the United States experience pain every day (2012 National Health Interview Survey data) and need safe, addiction-free treatments to alleviate their suffering. This clinical demand is of tremendous importance given that overprescribing of opioids for managing acute and chronic pain has fueled the current epidemic of opioid use disorder and overdose deaths, and the effectiveness of opioids for long-term pain management is being questioned. Safe, effective and non-addictive drugs (small molecules and biologics) to treat pain, mitigate addiction and reverse overdose are key to addressing the opioid crisis. Given failures and limitations of previous drug development efforts, drugs that recognize novel targets, have novel structures and can be identified in human-based, physiologically relevant in vitro systems are needed. To advance the NCATS ASPIRE Program and reward and spur innovative solutions to the development of new drugs for pain, addiction and overdose, NCATS is issuing this Challenge and concurrent companion Challenges to highly collaborative innovators interested in designing novel approaches that would lead to efficacious and non-addictive pain treatments and/or novel treatments for addiction and overdose.The ultimate goal of the NCATS ASPIRE Program is development of a platform that a wide spectrum of scientists can use to advance their translational science relevant to development and preclinical testing of new and safer treatments of pain, opioid use disorder (OUD) and overdose. Furthermore, it is essential that the approaches described and proposed here are applicable to any translational problem.Challenge 1: Integrated Chemistry Database for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of an open-source, controlled-access database that incorporates all currently available chemical, biological and clinical data of known opioid- and non-opioid-based analgesics, drugs of abuse and drugs used to treat drug abuse.Challenge 2: Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of a next-generation open-source electronic lab notebook (eLN) that collects, organizes and analyzes data relevant to the chemical synthesis and analyses of known opioid- and non-opioid-based analgesics, drugs of abuse and molecules used to treat drug abuse into an electronic laboratory knowledge portal for synthetic chemistry (electronic synthetic chemistry portal; eSCP).Challenge 3: Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of open source, advanced machine learning algorithms that would facilitate the discovery of novel, efficacious and non-addictive analgesics and/or treatments for drug abuse by utilizing the data collected in open source databases (Challenge area 1), eSCPs (Challenge area 2) and biological assays (Challenge area 4).Challenge 5: Integrated Solution for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs the design of innovative, comprehensive solutions to the opioid crisis through innovative approaches that integrate solutions to at least two Challenge areas (Challenges 1-4: Integrated Chemistry Database, Electronic Synthetic Chemistry Portal, Predictive Algorithms and Biological Assays, respectively) into a single platform.Note: Each component of Challenge 5 (above) is also available as an individual Challenge at Challenge.gov.Back to topRegistration Process for InnovatorsInnovators may access the registration and submission platform in one of the following ways:Access www.challenge.gov and search for “NCATS ASPIRE Design Challenge”Back to topThe PrizeAmount of the Prize; Award-Approving Official. The total prize purse is $500,000. Up to five (5) winners will be selected. NIH reserves the right to cancel, suspend and/or modify this Challenge at any time through amendment to this notice. In addition, NIH reserves the right to not award any prizes if no solutions are deemed worthy. The Award Approving Official will be Christopher P. Austin, M.D., Director of the National Center for Advancing Translational Sciences (NCATS).Payment of the Prize. Prizes awarded under this competition will be paid by electronic funds transfer and may be subject to federal income taxes. HHS/NIH will comply with the Internal Revenue Service withholding and reporting requirements, where applicable.Matching Requirement. A for-profit private entity solver (innovator) receiving a prize under this Challenge must match funds or provide documented in-kind contributions at a rate of not less than 50% of the total federally awarded amount, as stipulated by Public Law 115-141, the Consolidated Appropriations Act of 2018. Such a winner(s) will be required to demonstrate that matching funds and/or in-kind contributions were committed to achieve the winning solution. Such a winner(s) must identify the source and amount of funds used to meet the matching requirement or describe how the value for in-kind contributions was determined.Back to topEvaluation and Winner SelectionBasis upon Which Winners Will Be Selected. A panel of federal and non-federal reviewers, with expertise directly relevant to the Challenge, will evaluate the solutions based on feasibility and ability to achieve the criteria listed below. The solutions and evaluation statements from the technical panel will then be reviewed by federal employees serving as judges, who will select the Challenge winners, subject to the final decision by the Award Approving Official. The NCATS will provide feedback from the technical experts and judges to the winners and non-winners on their respective submissions.The points assigned to each set of evaluation criteria are guidelines from NCATS to suggest which scientific milestones are of emphasis and interest to the Center. All winners are highly encouraged to participate in future NCATS ASPIRE Reduction-to-Practice Challenges that NCATS is planning. Only complete submissions will be reviewed.Submission Requirements and TemplateInstructions for submission: Please format the proposal using the Submission Template and submit it to Challenge.gov as a PDF. Brief instructions on the submission process can be found below. Detailed instructions are provided in the submission template.Back to topBasis upon Which Submissions Will Be EvaluatedChallenge 4. Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose. This Challenge aims to address the need for novel, physiologically relevant biological assays that accurately replicate the safety profile and effectiveness of existing drugs and that can be reliably used in predictive risk assessments of new analgesics or drugs to treat addiction and/or overdose to molecular signature(s) and/or be able to anticipate the degree of addictiveness of an analgesic prior to clinical testing. This Challenge requires submission of only a detailed description of the design of the assay(s), not the final working assay(s).The goal of this Challenge is to reward and spur innovative solutions to the development of a novel physiologically relevant biological assay to advance preclinical discovery and development of non-addictive treatments for pain, drug addiction or overdoses. Current in vitro assays and in vivo models to study pain and addiction and test potential treatments are very limited. Pharmacological probe, lead and drug development have traditionally utilized canonical cell lines (such as CHO or HEK293) that heterologously express the molecular target of interest, and studies that are performed in animal models often do not fully recapitulate human physiology and may identify candidate compounds without effects in more human physiology-relevant systems. Therefore, NCATS is inviting innovators to propose and develop novel “disease-in-a-dish” assays of pain perception, addiction or overdose that advance the understanding of different types of pain, identify differences in inter- and intra-individual pain and the associated risk of developing chronic pain and/or addiction and make possible the development of improved, non-addictive drugs to treat these conditions. 2D and 3D human disease-relevant screening platforms may include but are not limited to normal and diseased human iPSC-derived sensory/pain neurons (such as peripheral, dorsal root ganglia [DRG], spinal cord, thalamus); neurons relevant to reward pathways; and other tissues important for pain, addiction and overdose, including blood-brain barrier (BBB). The assays will be adjudicated on their physiological relevance, robustness and reproducibility, and their future potential to be amended to automation and scalability for medium to high-throughput screening assays.Initially, the assay can be designed for low-throughput screening; it is expected that even at low throughput, multiple technical replicates will be compared in the same experiment. Eventually, the assay should be amenable to automation and scalable to medium- (96-well plate) to high-throughput screening (using, for example, high-content imaging, drug validation/toxicity, functional genomic screening).The innovators are encouraged to consult NCATS’ Assay Guidance Manual to learn more about appropriate designing of a drug screening assay.For the envisioned Reduction-to-Practice phase, the innovators will be expected to demonstrate the viability rate of cells in 2D or 3D models, at a particular throughput (90% viable), and provide data indicating how the functional morphological features are maintained in the system over time.Judging criteria:Evaluation Criterion 1: Impact and Innovation (20 points)To what degree is the proposed assay creative, original and biologically relevant?To what extent is the assay design feasible? Does it have a high likelihood of success?To what extent is the proposed solution innovative, and how high is its potential to significantly improve the state of science?To what extent will the proposed assay accelerate discovery, development and preclinical testing of new and safer treatments of pain and/or opioid use disorder (OUD) and overdose?To what extent are novel concepts, approaches, methodologies and technologies proposed or existing approaches applied to the design in a novel way?To what extent does the proposed assay incorporate cross-disciplinary techniques (e.g., cell biology, imaging, electronics, engineering, etc.)?Is the proof-of-concept data using currently available pain drugs, drugs of addiction and/or addiction/overdose treatments proposed?How well will the assay(s) demonstrate biological and physiological relevance to a specific application (pain, addiction or OUD)?Is a relevant proof-of-concept study of the innovators’ choice proposed for the subsequent Reduction-to-Practice stage?Evaluation Criterion 2: Model System and Assay Design (20 points)How physiologically relevant is the proposed model system that is being assayed?To what extent is the assay designed and amenable for validation taking into consideration the target being studied using appropriate drugs/compounds and controls?Are the appropriate cell types co-cultured in the model system at physiological ratios? What is the origin of the cells, and how relevant are the cell types to the model system?What is the assay’s readout (e.g., biochemical, fluorometric, phenotypic), and is this sufficient to maximize the value of the assay?How long will it take to perform the assay in full? Is the timing of the assay appropriate? (What is an assay’s development cycle time?)How many technical replicates will the assay support?Can the assay be multiplexed?Are secondary/specificity/selectivity assays proposed to confirm the initial data?What are instrumental, computational and data storage requirements?Evaluation Criterion 3: Assay Robustness, Reproducibility and Scalability (10 points)How well does the proposal describe plans to assess inter- and intra-laboratory utility, transferability and reproducibility?How well are the protocols described? Are the protocols sufficiently clear and detailed to facilitate inter- and intra-laboratory utility and reproducibility?For the Reduction-to-Practice phase, how would an assay’s robustness and reproducibility be demonstrated?Is the assay designed to minimize bias — for example, are the cells seeded, differentiated, cultured manually?Are appropriate experiments proposed that will address the stability of the assay components and reagents?To what extent is the data collection and analysis automated?To what degree could the assay be integrated with other Challenge(s) in a subsequent Reduction-to-Practice phase?Back to top			ASPIRE DESIGN CHALLENGE 4:			ASPIRE DESIGN CHALLENGE 4:
13168	ASPIRE Design Challenge 3: Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose	Summary of NCATS ASPIRE Design ChallengesSummary of NCATS ASPIRE Challenge 3: Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and OverdoseHow to EnterDates and DeadlinesThe IC’s Statutory Authority to Conduct the ChallengeSubject of the Challenge CompetitionConcurrent Companion NCATS ASPIRE Design ChallengesRegistration Process for InnovatorsThe PrizeEvaluation and Winner SelectionBasis upon Which Submissions Will Be EvaluatedSummary of NCATS ASPIRE Design ChallengesThe National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), is inviting novel design solutions for A Specialized Platform for Innovative Research Exploration (NCATS ASPIRE) Design Challenges as part of the NCATS ASPIRE Program. The goal of the NCATS ASPIRE Design Challenges is to reward and spur innovative and catalytic approaches toward solving the opioid crisis through development of (1) novel chemistries, (2) data mining and analysis tools and technologies, and (3) biological assays that will revolutionize discovery, development and preclinical testing of next-generation, safer and non-addictive analgesics to treat pain, as well as new treatments for opioid use disorder (OUD) and overdose. The first phase of these prize competitions is implemented through a suite of concurrent companion Design Challenges that comprises separate Challenges for each of four areas — chemistry database, electronic laboratory knowledge portal for synthetic chemistry, algorithms and biological assays — and an additional Challenge for a combined solution to at least two Challenge areas. At this stage, innovators are expected to submit designs, not final products or prototypes.NCATS envisions following these Design Challenges with a follow-on but distinct final Reduction-to-Practice Challenge, which will aim to invoke further scientific and technological development of the model system. Winners of the Design Challenges will be invited to present their designs so that, in the envisioned follow-up Reduction-to-Practice Challenge, an open competition, teams will be able to form multidisciplinary collaborations to advance and integrate the most feasible and promising approaches to the multiple Challenges into a single integrative platform. Innovators will be invited to demonstrate final solutions.The NCATS ASPIRE Design Challenges are part of NIH’s Helping to End Addiction Long-term (HEAL) initiative to speed scientific solutions to the national opioid public health crisis. The NIH HEAL Initiative will bolster research across NIH to (1) improve treatment for opioid misuse and addiction and (2) enhance pain management. More information about the HEAL Initiative is available at https://www.nih.gov/research-training/medical-research-initiatives/heal-initiative.NCATS refers to participants in the NCATS ASPIRE Design Challenges as “innovators,” because all solutions will require highly innovative approaches to achieve success. Innovators should clearly state how and why the proposed solution would provide significant advances over currently available tools. Innovators may choose to compete in one or more individual Challenges to address a single area (Challenges 1-4) or propose a combined solution for at least two Challenge areas (Challenge 5).Back to topSummary of NCATS ASPIRE Challenge 3: Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and OverdoseChallenge 3 aims to address the need for open source, advanced machine learning algorithms that would facilitate the discovery of novel, efficacious and non-addictive analgesics and/or treatments for drug abuse. This Challenge requires submission of only a detailed description of the design of the algorithms, not the final working versions. While initially the innovators can use their own training sets to demonstrate functionality of an algorithm, in the envisioned follow-on Reduction-to-Practice stage, the algorithm will be expected to incorporate and analyze data generated from Challenges 1, 2 and 4.Evaluation criteria that reviewers will be asked to address are specified below.Back to topDates and DeadlinesSolutions must be submitted to Challenge.gov by NOON Eastern Time on May 31, 2019. The Challenge begins: December 31, 2018 Submission period: December 31, 2018-May 31, 2019Judging period: June 17, 2019-August 2, 2019Winners announced: August 2019For further information send an email to NCATSASPIREChallenge@mail.nih.govBack to topThe IC’s Statutory Authority to Conduct the Challenge The general purpose of NCATS is to coordinate and develop resources that leverage basic research in support of translational science and to develop partnerships and work cooperatively to foster synergy in ways that do not create duplication, redundancy and competition with industry activities (42 USC 287(a)). In order to fulfill its mission, NCATS supports projects that will transform the translational process so that new treatments and cures for diseases can be delivered to patients faster by understanding the translational process in order to create a basis for more science-driven, predictive and effective intervention development for the prevention and treatment of all diseases. NCATS is also conducting this Challenge under the America Creating Opportunities to Meaningfully Promote Excellence in Technology, Education, and Science (COMPETES) Reauthorization Act of 2010, 15 U.S.C. 3719. In line with these authorities, this Challenge(s) will lead to innovative designs for developing technology to revolutionize discovery, development and preclinical testing of new and safer treatments of pain, opioid use disorder (OUD), and overdose; the result will be generalizable tools that will be widely available to fill longstanding gaps that have impeded the marriage of basic and translational sciences.Back to topSubject of the Challenge CompetitionChallenge 3. Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose. This Challenge aims to reward and spur innovative solutions to the development of machine learning algorithms that would aid the discovery of novel analgesics and/or treatments for opioid addiction and overdoses. For example, an algorithm can be developed to identify side groups and/or include ratio of bond types or atom types in a molecule in order to identify signatures that are less likely to trigger addiction. This Challenge requires submission of only a detailed description of the design of the algorithms, not the final working versions. The ultimate goal is to utilize the data from Challenge 1 and Challenge 2 during the envisioned Reduction-to-Practice Challenge to provide proof-of-concept and design, synthesize, optimize and test novel compounds that are less likely to trigger addiction or are able to treat addiction/overdose. Optimized novel small-molecule leads would be tested for their bioactivity using physiologically relevant models developed in Challenge 4 in order to provide proof-of-concept for therapeutic hypotheses to treat pain, addiction and overdose.Back to topConcurrent Companion NCATS ASPIRE Design ChallengesNCATS has recently explored the development of A Specialized Platform for Innovative Research Exploration (ASPIRE) to aid in the discovery and development of novel and effective treatments while at the same time making the process faster and more cost-effective. The NCATS ASPIRE Program aims to develop and integrate automated synthetic chemistry, biological screening and artificial intelligence approaches in order to significantly advance our understanding of the relationship between chemical and biological space and enable further access into biologically relevant chemical space. The platform will utilize currently available knowledge to develop innovative algorithms and predict and synthetize novel structures capable of interacting with specific targets; enable small-scale synthesis of the predicted molecules; and incorporate in-line, rapid biological testing of the molecules. Any new data obtained through this process would then be fed back into the system to further improve design, synthesis and biological characteristics of molecules.Over 25 million people in the United States experience pain every day (2012 National Health Interview Survey data) and need safe, addiction-free treatments to alleviate their suffering. This clinical demand is of tremendous importance given that overprescribing of opioids for managing acute and chronic pain has fueled the current epidemic of opioid use disorder and overdose deaths, and the effectiveness of opioids for long-term pain management is being questioned. Safe, effective and non-addictive drugs (small molecules and biologics) to treat pain, mitigate addiction and reverse overdose are key to addressing the opioid crisis. Given failures and limitations of previous drug development efforts, drugs that recognize novel targets, have novel structures and can be identified in human-based, physiologically relevant in vitro systems are needed. To advance the NCATS ASPIRE Program and reward and spur innovative solutions to the development of new drugs for pain, addiction and overdose, NCATS is issuing this Challenge and concurrent companion Challenges to highly collaborative innovators interested in designing novel approaches that would lead to efficacious and non-addictive pain treatments and/or novel treatments for addiction and overdose.The ultimate goal of the NCATS ASPIRE Program is development of a platform that a wide spectrum of scientists can use to advance their translational science relevant to development and preclinical testing of new and safer treatments of pain, opioid use disorder (OUD) and overdose. Furthermore, it is essential that the approaches described and proposed here are applicable to any translational problem.Challenge 1: Integrated Chemistry Database for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of an open-source, controlled-access database that incorporates all currently available chemical, biological and clinical data of known opioid- and non-opioid-based analgesics, drugs of abuse and drugs used to treat drug abuse.Challenge 2: Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of a next-generation open-source electronic lab notebook (eLN) that collects, organizes and analyzes data relevant to the chemical synthesis and analyses of known opioid- and non-opioid-based analgesics, drugs of abuse and molecules used to treat drug abuse into an electronic laboratory knowledge portal for synthetic chemistry (electronic synthetic chemistry portal; eSCP).Challenge 4: Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of novel, physiologically relevant biological assays that accurately replicate the safety profile and effectiveness of existing drugs to treat addiction and/or overdose and that can be reliably used in predictive risk assessments of new analgesics or drugs to treat addiction and/or overdose and/or be able to anticipate the degree of addictiveness of an analgesic prior to clinical testing.Challenge 5: Integrated Solution for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs the design of innovative, comprehensive solutions to the opioid crisis through innovative approaches that integrate solutions to at least two Challenge areas (Challenges 1-4: Integrated Chemistry Database, Electronic Synthetic Chemistry Portal, Predictive Algorithms and Biological Assays, respectively) into a single platform.Note: Each component of Challenge 5 (above) is also available as an individual Challenge at Challenge.gov.Back to topRegistration Process for InnovatorsInnovators may access the registration and submission platform in one of the following ways:Access www.challenge.gov and search for “NCATS ASPIRE Design Challenge”Back to topThe PrizeAmount of the Prize; Award-Approving Official. The total prize purse is $500,000. Up to five (5) winners will be selected. NIH reserves the right to cancel, suspend and/or modify this Challenge at any time through amendment to this notice. In addition, NIH reserves the right to not award any prizes if no solutions are deemed worthy. The Award Approving Official will be Christopher P. Austin, M.D., Director of the National Center for Advancing Translational Sciences (NCATS).Payment of the Prize. Prizes awarded under this competition will be paid by electronic funds transfer and may be subject to federal income taxes. HHS/NIH will comply with the Internal Revenue Service withholding and reporting requirements, where applicable.Matching Requirement. A for-profit private entity solver (innovator) receiving a prize under this Challenge must match funds or provide documented in-kind contributions at a rate of not less than 50% of the total federally awarded amount, as stipulated by Public Law 115-141, the Consolidated Appropriations Act of 2018. Such a winner(s) will be required to demonstrate that matching funds and/or in-kind contributions were committed to achieve the winning solution. Such a winner(s) must identify the source and amount of funds used to meet the matching requirement or describe how the value for in-kind contributions was determined.Back to topEvaluation and Winner SelectionBasis upon Which Winners Will Be Selected. A panel of federal and non-federal reviewers, with expertise directly relevant to the Challenge, will evaluate the solutions based on feasibility and ability to achieve the criteria listed below. The solutions and evaluation statements from the technical panel will then be reviewed by federal employees serving as judges, who will select the Challenge winners, subject to the final decision by the Award Approving Official. The NCATS will provide feedback from the technical experts and judges to the winners and non-winners on their respective submissions.The points assigned to each set of evaluation criteria are guidelines from NCATS to suggest which scientific milestones are of emphasis and interest to the Center. All winners are highly encouraged to participate in future NCATS ASPIRE Reduction-to-Practice Challenges that NCATS is planning. Only complete submissions will be reviewed.Submission Requirements and TemplateInstructions for submission: Please format the proposal using the Submission Template and submit it to Challenge.gov as a PDF. Brief instructions on the submission process can be found below. Detailed instructions are provided in the submission template.Back to topBasis upon Which Submissions Will Be EvaluatedChallenge 3. Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose. This Challenge aims to reward and spur innovative solutions to the development machine learning algorithms that would aid the discovery of novel analgesics and/or treatments for opioid addiction and overdoses. For example, an algorithm can be developed to identify side groups and/or include ratio of bond types or atom types in a molecule in order to identify signatures that are less likely to trigger addiction. The ultimate goal is to utilize the data from Challenge 1 and Challenge 2 during the envisioned Reduction-to-Practice Challenge to provide proof-of-concept and design, synthesize, optimize and test novel compounds that are less likely to trigger addiction or are able to treat addiction/overdose. Optimized novel small-molecule leads would be tested for their bioactivity using physiologically relevant models developed in Challenge 4 in order to provide proof-of-concept for therapeutic hypotheses to treat pain, addiction and overdose.Judging criteria:Evaluation Criterion 1: Impact and Innovation (20 points)Did the team identify potential roadblocks and suggest additional expertise that would be utilized to facilitate resolution of roadblocks to implementation?Given that innovation is considered using a groundbreaking or paradigm-shifting approach or using existing approaches in an innovative way, to what degree is the proposed design innovative, creative and original?To what extent is the proposed approach feasible, and how high is its likelihood to succeed?Has the innovator or team of innovators demonstrated that appropriate expertise was utilized during development of the design?Have the innovators established their own initial training datasets that can be used to demonstrate the algorithms’ functionality on the set?Does the training data set include structurally diverse compounds?To what extent have the innovators demonstrated why/how the proposed solution can outperform existing algorithms?How well has the proposed solution demonstrated its ability to provide critical information necessary for the development of novel treatments and therapies for pain, drug addiction and/or overdose?Evaluation Criterion 2: Algorithm Design Functionality and Implementation (20 points)How well are the algorithms documented for completing a task on a training set and making predictions on a new set of compounds? (Are the steps precisely stated)?To what extent are the algorithms implemented with open source codes/packages or commercial software packages?Have the innovators designed a web portal or tool/platform that is accessible to research community to run the proposed algorithms and generate predictions on their compounds of interest?How well have the innovators explained how their algorithms meet the criteria of precision, uniqueness, finiteness, definiteness, input, output and effectiveness?How well can the algorithms identify structural and/or functional similarities in a diverse set of molecules?How well can the algorithms identify specific signatures that may be associated with addiction?How well can the algorithms compare the mechanism of action between multiple drugs and identify structural/functional similarities between them?How successful are the algorithms in identifying structurally diverse drugs with similar pharmacological effect?To what degree can the algorithms provide additional context for drug activity that can be used to identify or anticipate off-target effects?Evaluation Criterion 3: Record of Innovation (10 points)How well have the innovators demonstrated experience with creating solutions that:Connect across multiple computational modeling and simulation tools and frameworks;Evaluate and adjust models based on new data, as the data becomes available; andEvaluate and adjust models based on the successes or failures of predicting phenomena observed in humans and, where biologically justified, in animals?Back to top			ASPIRE DESIGN CHALLENGE 3:			ASPIRE DESIGN CHALLENGE 3:
13171	ASPIRE Design Challenge 2: Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and Overdose	Summary of NCATS ASPIRE Design ChallengesSummary of NCATS ASPIRE Design Challenge 2: Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and OverdoseHow to EnterDates and DeadlinesThe IC’s Statutory Authority to Conduct the Challenge Subject of the Challenge CompetitionConcurrent Companion NCATS ASPIRE Design ChallengesRegistration Process for InnovatorsThe PrizeEvaluation and Winner SelectionBasis upon Which Submissions Will Be EvaluatedSummary of NCATS ASPIRE Design ChallengesThe National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), is inviting novel design solutions for A Specialized Platform for Innovative Research Exploration (NCATS ASPIRE) Design Challenges as part of the NCATS ASPIRE Program. The goal of the NCATS ASPIRE Design Challenges is to reward and spur innovative and catalytic approaches toward solving the opioid crisis through development of (1) novel chemistries, (2) data mining and analysis tools and technologies, and (3) biological assays that will revolutionize discovery, development and preclinical testing of next-generation, safer and non-addictive analgesics to treat pain, as well as new treatments for opioid use disorder (OUD) and overdose. The first phase of these prize competitions is implemented through a suite of concurrent companion Design Challenges that comprises separate Challenges for each of four areas — chemistry database, electronic laboratory knowledge portal for synthetic chemistry, algorithms and biological assays — and an additional Challenge for a combined solution to at least two Challenge areas. At this stage, innovators are expected to submit designs, not final products or prototypes.NCATS envisions following these Design Challenges with a follow-on but distinct final Reduction-to-Practice Challenge, which will aim to invoke further scientific and technological development of the model system. Winners of the Design Challenges will be invited to present their designs so that, in the envisioned follow-up Reduction-to-Practice Challenge, an open competition, teams will be able to form multidisciplinary collaborations to advance and integrate the most feasible and promising approaches to the multiple Challenges into a single integrative platform. Innovators will be invited to demonstrate final solutions.The NCATS ASPIRE Design Challenges are part of NIH’s Helping to End Addiction Long-term (HEAL) initiative to speed scientific solutions to the national opioid public health crisis. The NIH HEAL Initiative will bolster research across NIH to (1) improve treatment for opioid misuse and addiction and (2) enhance pain management. More information about the HEAL Initiative is available at https://www.nih.gov/research-training/medical-research-initiatives/heal-initiative.NCATS refers to participants in the NCATS ASPIRE Design Challenges as “innovators,” because all solutions will require highly innovative approaches to achieve success. Innovators should clearly state how and why the proposed solution would provide significant advances over currently available tools. Innovators may choose to compete in one or more individual Challenges to address a single area (Challenges 1-4) or propose a combined solution for at least two Challenge areas (Challenge 5).Back to topSummary of NCATS ASPIRE Design Challenge 2: Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and OverdoseChallenge 2 aims to address the need for a next-generation open-source electronic lab notebook (eLN) that collects, organizes and analyzes data relevant to the chemical synthesis and analyses of known opioid- and non-opioid-based analgesics, drugs of abuse and molecules used to treat drug abuse into an electronic laboratory knowledge portal for synthetic chemistry (electronic synthetic chemistry portal; eSCP). This Challenge requires submission of only a detailed description of the design of the eSCP, not the final working portal. The innovators may use currently available open source eLNs and modify them to meet the challenge’s requirements. While the focus of this Challenge is on a specific translational problem relevant to pain treatments and opioid use disorder and overdose, the eSCP should be designed to be generally applicable to any kind of laboratory chemical and biological data collection, recording and analysis of data relevant to any translational Challenge; modular; and well architected for advanced machine learning applications.Evaluation criteria that reviewers will be asked to address are specified below.Back to topDates and DeadlinesSolutions must be submitted to Challenge.gov by NOON Eastern Time on May 31, 2019. The Challenge begins: December 31, 2018 Submission period: December 31, 2018-May 31, 2019Judging period: June 17, 2019-August 2, 2019Winners announced: August 2019For further information send an email to NCATSASPIREChallenge@mail.nih.govBack to topThe IC’s Statutory Authority to Conduct the Challenge The general purpose of NCATS is to coordinate and develop resources that leverage basic research in support of translational science and to develop partnerships and work cooperatively to foster synergy in ways that do not create duplication, redundancy and competition with industry activities (42 USC 287(a)). In order to fulfill its mission, NCATS supports projects that will transform the translational process so that new treatments and cures for diseases can be delivered to patients faster by understanding the translational process in order to create a basis for more science-driven, predictive and effective intervention development for the prevention and treatment of all diseases. NCATS is also conducting this Challenge under the America Creating Opportunities to Meaningfully Promote Excellence in Technology, Education, and Science (COMPETES) Reauthorization Act of 2010, 15 U.S.C. 3719. In line with these authorities, this Challenge(s) will lead to innovative designs for developing technology to revolutionize discovery, development and preclinical testing of new and safer treatments of pain, opioid use disorder (OUD), and overdose; the result will be generalizable tools that will be widely available to fill longstanding gaps that have impeded the marriage of basic and translational sciences.Back to topSubject of the Challenge CompetitionChallenge 2. Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and Overdose. In order to enable further access into the currently unexplored biological space relevant to treatment of pain and/or drug addiction and overdose, this second Challenge aims to reward and spur innovative solutions to the development next-generation, open source electronic laboratory notebook (eLN) that would serve as an electronic synthetic chemistry knowledge portal (eSCP) and allow for real-time molecular design hypothesis generation and unbiased data collection during the synthesis planning, execution and analysis while providing a strong and comprehensive contextual analysis to biological testing data of experimental targets and currently known pain drug comparators. This Challenge requires submission of only a detailed description of the design of the eSCP, not the final working portal. The innovators may use currently available open source eLNs and modify them to meet the Challenge’s requirements. The ultimate goal is to effectively utilize data and outcomes from both positive and negative synthetic chemistries for future discovery and development of novel structures and chemistries of relevance to the treatment of pain and opioid use disorder. The eSCP is expected to be modular or flexible to accommodate additional features if required and to provide an external control mechanism (e.g., API or CLI). All data entries, including chemical structures, should be in a format that can be easily ported to advanced machine learning algorithms and/or downstream analytical applications.As described in A Quick Guide to ELN Regulatory Requirements, the developer of an eSCP system should be aware of and compliant with:21 CFR Part 11, the U.S. Food and Drug Administration (FDA) regulation of electronic signatures and systemsGood Laboratory Practices, particularly the GLP focus on traceability of data changesGood Manufacturing Practices 2, specifically requirements for validationPFSB 040122, the Japanese guidelineAnnex 11, computer guidelines for the EU’s Good Manufacturing PracticesPotentially the guidelines of industry supplemental organizations:International Standards Organization (ISO) 9001Pharmaceutical Inspection Convention Scheme (PIC/S) Good Practices Guidance for Inspectors “Good Practices for Computerized Systems in Regulated ‘GxP’ Environments”“The Application of the Principles of GLP to Computerized Systems” (OECD 1995)Back to topConcurrent Companion NCATS ASPIRE Design ChallengesNCATS has recently explored the development of A Specialized Platform for Innovative Research Exploration (ASPIRE) to aid in the discovery and development of novel and effective treatments while at the same time making the process faster and more cost-effective. The NCATS ASPIRE Program aims to develop and integrate automated synthetic chemistry, biological screening and artificial intelligence approaches in order to significantly advance our understanding of the relationship between chemical and biological space and enable further access into biologically relevant chemical space. The platform will utilize currently available knowledge to develop innovative algorithms and predict and synthetize novel structures capable of interacting with specific targets; enable small-scale synthesis of the predicted molecules; and incorporate in-line, rapid biological testing of the molecules. Any new data obtained through this process would then be fed back into the system to further improve design, synthesis and biological characteristics of molecules.Over 25 million people in the United States experience pain every day (2012 National Health Interview Survey data) and need safe, addiction-free treatments to alleviate their suffering. This clinical demand is of tremendous importance given that overprescribing of opioids for managing acute and chronic pain has fueled the current epidemic of opioid use disorder and overdose deaths, and the effectiveness of opioids for long-term pain management is being questioned. Safe, effective and non-addictive drugs (small molecules and biologics) to treat pain, mitigate addiction and reverse overdose are key to addressing the opioid crisis. Given failures and limitations of previous drug development efforts, drugs that recognize novel targets, have novel structures and can be identified in human-based, physiologically relevant in vitro systems are needed. To advance the NCATS ASPIRE Program and reward and spur innovative solutions to the development of new drugs for pain, addiction and overdose, NCATS is issuing this Challenge and concurrent companion Challenges to highly collaborative innovators interested in designing novel approaches that would lead to efficacious and non-addictive pain treatments and/or novel treatments for addiction and overdose.The ultimate goal of the NCATS ASPIRE Program is development of a platform that a wide spectrum of scientists can use to advance their translational science relevant to development and preclinical testing of new and safer treatments of pain, opioid use disorder (OUD) and overdose. Furthermore, it is essential that the approaches described and proposed here are applicable to any translational problem.Challenge 1: Integrated Chemistry Database for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of an open-source, controlled-access database that incorporates all currently available chemical, biological and clinical data of known opioid- and non-opioid-based analgesics, drugs of abuse and drugs used to treat drug abuse.Challenge 3: Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of open source, advanced machine learning algorithms that would facilitate the discovery of novel, efficacious and non-addictive analgesics and/or treatments for drug abuse by utilizing the data collected in open source databases (Challenge area 1), eSCPs (Challenge area 2) and biological assays (Challenge area 4).Challenge 4: Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs innovative solutions to the design of novel, physiologically relevant biological assays that accurately replicate the safety profile and effectiveness of existing drugs to treat addiction and/or overdose and that can be reliably used in predictive risk assessments of new analgesics or drugs to treat addiction and/or overdose and/or be able to anticipate the degree of addictiveness of an analgesic prior to clinical testing.Challenge 5: Integrated Solution for Translational Innovation in Pain, Opioid Use Disorder and Overdose rewards and spurs the design of innovative, comprehensive solutions to the opioid crisis through innovative approaches that integrate solutions to at least two Challenge areas (Challenges 1-4: Integrated Chemistry Database, Electronic Synthetic Chemistry Portal, Predictive Algorithms and Biological Assays, respectively) into a single platform.Note: Each component of Challenge 5 (above) is also available as an individual Challenge at Challenge.gov.Back to topRegistration Process for InnovatorsInnovators may access the registration and submission platform in one of the following ways:Access www.challenge.gov and search for “NCATS ASPIRE Design Challenge”Back to topThe PrizeAmount of the Prize; Award-Approving Official. The total prize purse is $500,000. Up to five (5) winners will be selected. NIH reserves the right to cancel, suspend and/or modify this Challenge at any time through amendment to this notice. In addition, NIH reserves the right to not award any prizes if no solutions are deemed worthy. The Award Approving Official will be Christopher P. Austin, M.D., Director of the National Center for Advancing Translational Sciences (NCATS).Payment of the Prize. Prizes awarded under this competition will be paid by electronic funds transfer and may be subject to federal income taxes. HHS/NIH will comply with the Internal Revenue Service withholding and reporting requirements, where applicable.Matching Requirement. A for-profit private entity solver (innovator) receiving a prize under this Challenge must match funds or provide documented in-kind contributions at a rate of not less than 50% of the total federally awarded amount, as stipulated by Public Law 115-141, the Consolidated Appropriations Act of 2018. Such a winner(s) will be required to demonstrate that matching funds and/or in-kind contributions were committed to achieve the winning solution. Such a winner(s) must identify the source and amount of funds used to meet the matching requirement or describe how the value for in-kind contributions was determined.Back to topEvaluation and Winner SelectionBasis upon Which Winners Will Be Selected. A panel of federal and non-federal reviewers, with expertise directly relevant to the Challenge, will evaluate the solutions based on feasibility and ability to achieve the criteria listed below. The solutions and evaluation statements from the technical panel will then be reviewed by federal employees serving as judges, who will select the Challenge winners, subject to the final decision by the Award Approving Official. The NCATS will provide feedback from the technical experts and judges to the winners and non-winners on their respective submissions.The points assigned to each set of evaluation criteria are guidelines from NCATS to suggest which scientific milestones are of emphasis and interest to the Center. All winners are highly encouraged to participate in future NCATS ASPIRE Reduction-to-Practice Challenges that NCATS is planning. Only complete submissions will be reviewed.Submission Requirements and TemplateInstructions for submission: Please format the proposal using the Submission Template and submit it to Challenge.gov as a PDF. Brief instructions on the submission process can be found below. Detailed instructions are provided in the submission template.Back to topBasis upon Which Submissions Will Be EvaluatedChallenge 2. Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and Overdose. In order to enable further access into the currently unexplored biological space relevant to treatment of pain and/or drug addiction and overdose, this second Challenge rewards and spurs solutions to the development of next-generation, open source electronic laboratory notebook (eLN) that would serve as an electronic synthetic chemistry knowledge portal (eSCP) and allow for real-time molecular design hypothesis generation and unbiased data collection during the synthesis planning, execution and analysis while providing a strong and comprehensive contextual analysis to biological testing data of experimental targets and currently known pain drug comparators. This Challenge requires submission of only a detailed description of the design of the eSCP, not the final working portal. The innovators may use currently available open source eLNs and modify them to meet the Challenge’s requirements. The ultimate goal is to effectively utilize data and outcomes from both positive and negative synthetic chemistries for future discovery and development of novel structures and chemistries of relevance to the treatment of pain and opioid use disorder. The eSCP is expected to be modular or flexible to accommodate additional features if required and to provide external control mechanism (e.g., API or CLI). All data entries, including chemical structures, should be in a format that can be easily ported to advanced machine learning algorithms and/or downstream analytical applications.Judging criteria:Evaluation Criterion 1: Impact and Innovation (20 points)Did the team identify potential roadblocks and suggest additional expertise it would utilize to facilitate resolution of roadblocks to implementation?To what extent is the proposal innovative — that is, to what extent does it involve a novel eSCP or an approach that significantly upgrades and appropriately modifies an existing eLN?Has the innovator or team of innovators demonstrated that appropriate expertise was utilized during development of the design?Have the innovators consulted potential user laboratories with regard to the kind, depth and breadth of data the users desire to have in an eSCP, data standards and formats?How well have innovators optimized the molecular design capabilities of the platform to advance the highest-quality hypothetical attributes of target molecules and their proposed synthetic execution plan?To what extent will the data collected enable further mechanistic understanding that is essential to control and optimize chemical reactions so that byproducts are reduced, yields are increased and reaction specificities are improved?Is the eSCP designed to utilize high-quality data from both positive and negative experiments for future discovery and development of novel structures and chemistries of relevance to the treatment of pain and opioid use disorder?Have the innovators adequately described how the eSCP’s application will be demonstrated in laboratories in the envisioned follow-up NCATS ASPIRE Reduction-to-Practice Challenges?While the focus of this Challenge is on pain-related drugs, to what extent is the eSCP designed to be adaptable to collect any kind of chemical and biological data relevant to any future translational Challenge?Are all deposited data, including chemical structures, in a format that can be easily accessible to advanced machine learning algorithms and/or applications?Evaluation Criterion 2: Data Collection, Analysis and Integration (20 points)How well does the eSCP parse reaction information to include precise, unambiguous ontological annotation and reaction role descriptors (e.g., solvent, catalyst, other specific additive roles, etc.)?How well is this information organized — for example, is this information organized in a manner to provide comprehensive reaction analytics (similar but not limited to breakdown of reaction types represented; most commonly used reagents and catalysts; time-related patterns associated with reaction development, including reaction networks that summarize typical reaction steps and reaction pathway associations with common intermediates or final targets)?Are reactions atom mapped or sequenced in a manner to allow use in retrosynthetic applications or reaction templating?How does the proposed solution permit the broad-scope assessment of the database around the technology or methodology used (e.g., C-H activation, photochemistry, etc.) or highlight the overreliance on specific transformations (e.g., Suzuki reaction)?How effectively and intuitively are the data collected and presented during the work process?How extensible are the data structures (e.g., is it possible to collect more than one measurement in a set of conditions or parameters)?How rigorously is reaction information parsed and annotated?What chemical representation and data storage standards are used?How precisely and systematically is reaction ontology being captured?What provisions are included for executing reaction analytics (similar but not limited to top reagents used that week with total amount used; rank order listing of reaction types run, by yield or popularity; number of new compounds synthesized/registered; etc.)?How well does the solution provide a customizable analytics dashboard to follow any number of metrics across the entire eSCP (similar but not limited to total reactions that day, total users active, number of compounds submitted to biological assays, etc.)?How is chemical yield information captured?How are reaction entry errors captured and corrected?How well is reagent selection and inventory management integrated?How well is parallel experimentation implemented?What methods exist for importing and exporting chemical information as well as reaction design and execution criteria, and what data formats are supported?How efficiently can experiments be designed, entered and annotated?What provisions exist for applying custom business rules (e.g., requirements for initiating or closing out an experiment)?How well can the eSCP be controlled by or operated from another application (i.e., is a fully documented API or CLI provided)?How well are experimental success and failures tracked?To what extent does the eSCP facilitate validation and reproducibility of experimental data?How are upgrades to the modular eSCP expected to be applied?How well can the eSCP integrate chemical data with those from biological screening assays?What rigor is applied to the capture and storing of data, and what tools are provided to examine the data integrity? (That is, are reagents appearing in the right rows in a consistent fashion? Can the eSCP distinguish or make alerts when solvents, reagents or catalysts are omitted? Is reagent container identification included or captured? Are the data well ordered such that when the same reagent shows up in multiple reactions, it shows up in the same relevant row in the database?)What methodology is present in the eSCP to allow for a plug-and-play template format that can be used to record, extract and report the data?How easy is it to extract the data from the eSCP document and record in database tables with appropriate metadata suitable for mining and analysis with advanced machine learning applications?How well does the eSCP integrate retrosynthetic tools, machine learning and computational chemistry tools in a manner that facilitates future upgrades and additional links to bioassay data and related chemical structure and synthesis information?Evaluation Criterion 3: Accessibility and User-Friendliness (10 points)Is this an open source eSCP solution?How appealing are user interfaces, and how well are they designed for intuitive use?How well would the eSCP facilitate intra- and inter-laboratory connectivity and intra- and inter-laboratory reproducibility?To what degree is the eSCP’s interface user-friendly and in a form that reduces training and increases user acceptance?How well are non-routine documentation options included in the eSCP (e.g., tables, textboxes, formulas, etc.)?Is the eSCP designed to be remotely accessible (e.g., off-site computers or mobile devices)?Does the design of the eSCP include documentation and management of laboratory chemical/sample inventory, equipment management, usage and label printing and barcodes?How well is the problem of multiple terminologies dealt with (e.g., are dictionaries included)?Back to top			DESIGN CHALLENGE 2:			DESIGN CHALLENGE 2:
12754	NCATS, FDA Co-Host Workshop on Gene Therapy for Rare Diseases	Translational Science Highlight NCATS and the Food and Drug Administration are collaborating to identify bottlenecks to gene therapy development and share best practices for advancing gene therapies, a promising approach to treating many different rare diseases. “We are now at the point of translating the potential of gene therapy — which has been around for a while — into a reality for patients,” said NCATS Director Christopher P. Austin, M.D. These new advances in gene therapies have truly been a long time in coming. The first human study that involved gene therapy, a type of treatment to replace or fix a defective gene in a patient, began in 1990 at the NIH Clinical Center. Nearly 30 years later, in 2017, the Food and Drug Administration (FDA) approved the first three gene therapies for use in the U.S. One of those three is for an inherited disease; the others alter a patient’s own cells to fight two forms of cancer. Inspired by this scientific progress, in August 2018, NCATS and the FDA co-hosted a two-day workshop about gene therapy for rare diseases. Many of these diseases are caused by a mutation in a single gene, so gene therapy holds great promise as an effective treatment approach. Led by NCATS’ Office of Rare Diseases Research and the FDA’s Center for Biologics Evaluation and Research (CBER) staff, the workshop brought together representatives from NIH, the FDA, academia, industry and patient organizations to discuss using gene therapy to treat rare diseases. Discussion topics included recent scientific advances; challenges in manufacturing the viruses, called vectors, that are used to deliver gene therapy products; and how to increase the number of available treatments. “We really are having this meeting at the right time,” said FDA CBER Director Peter Marks, M.D., Ph.D. He noted that hundreds of potential gene therapy treatments are now entering the clinic for testing in people. In July 2018, the FDA released draft guidance on several topics related to gene therapy, such as manufacturing and long-term follow-up of people who receive these agents. Advancing the Science NCATS Director Christopher P. Austin, M.D., at the Growing Promise of Gene Therapy Approaches to Rare Diseases Workshop in August 2018. Exciting scientific advances discussed at the meeting included research in animal models and studies showing new advances in humans. Jay Chiorini, Ph.D., of the NIH National Institute of Dental and Craniofacial Research, shared his research on using gene therapy to make salivary glands work better. Most people who have radiation treatment for head and neck cancer temporarily or permanently lose the ability to make saliva. Without it, eating becomes painful, and people may lose their teeth and have other dental problems. Using gene therapy in mini-pigs that were treated with radiation, Chiorini has been able to show some improvement in how the salivary glands worked. This approach has now entered the clinic, and he has so far enrolled 12 people in a study designed to test different gene therapy doses to find out whether the approach is safe in humans. “We are often faced with the fact that mouse studies don’t translate very well to humans,” said Jerry Mendell, M.D., director of the Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio. However, for his research on the rare disease spinal muscular atrophy type 1, tests in mice did a good job predicting how the treatment would work in humans. Children with this disease have weak muscles, and without treatment, they never talk or sit unassisted and have trouble swallowing. If left untreated, most children with spinal muscular atrophy type 1 die by the age of 2. When newborn mice were given gene therapy by day 1, however, they behaved normally and survived into adulthood. Based on those findings, Mendell conducted a trial in human infants up to 6 months old. The results were impressive: Of the 12 children who received a high dose of the vector, all have survived past age 3, and all are active. Four can stand, two can walk, and all but one can sit without support. Addressing the Manufacturing Bottleneck Panelists at the Growing Promise of Gene Therapy Approaches to Rare Diseases Workshop in August 2018. Despite these scientific advances, manufacturing is turning out to be a critical bottleneck for the production of gene therapy. Producing the specialized vectors that deliver the treatment is a slow and complex process, and only a few facilities can currently do the work. Representatives from some of these facilities talked to workshop attendees about their operations and plans for expansion. Manufacturing is also expensive, and academic researchers and private-sector companies currently have to compete for the same limited manufacturing capacity. Attendees emphasized the need to keep manufacturing affordable for everyone, including academic researchers and people with rare diseases. “How do we make sure we have a pipeline for those products that have a commercial future as well as the ones that don’t — those rare diseases with 10 to 12 patients across the country?” asked Jaysson Eicholtz, M.S., director of the manufacturing facility at Nationwide Children’s Hospital. “How do we get to a point where we can support both types?” When researchers are thinking about conducting a human trial for the first time, they should already be thinking about manufacturing, said Denise Gavin, Ph.D., chief of FDA CBER’s Gene Therapy Branch. That does not mean that a product must be ready for marketing as soon as the first human trials start. However, Gavin said, “Researchers may want to discuss their product manufacturing with someone who knows enough about manufacturing to help them think about whether the approach they’re taking will work when it is time to market a product.” Advancing Treatments for Rare Disease Patients FDA CBER Director Peter Marks, M.D., Ph.D.. The challenge of rare diseases is that there are so many. “We at NCATS are big on ‘It’s great that we made success in an individual disease, but what about the other 6,999?’” Austin said. “How do we advance the next one 10-fold better and the one after that another 10-fold better?” Only about 300 of the thousands of identified rare diseases have an FDA-approved treatment. With only a few new treatments approved each year, it would take more than 1,000 years to address all of the known rare diseases. Through a number of programs, including its Rare Diseases Clinical Research Network (RDCRN), NCATS is working to advance research on many rare diseases at once. Together, the RDCRN-supported researchers study more than 200 rare diseases, making discoveries that may apply to multiple disorders. NCATS also addresses rare diseases through a variety of programs and initiatives, including Therapeutics for Rare and Neglected Diseases, the Genetic and Rare Diseases Information Center, the NCATS Toolkit for Patient-Focused Therapy Development and the Rare Diseases Registry Program. One way to attack multiple diseases at once could be to develop a kind of treatment platform that enables any disease’s relevant gene to be plugged in. The virus used by Mendell and many of the other workshop presenters to deliver their gene therapies could be such a technology, said Steven Gray, Ph.D., an associate professor of pediatrics at the University of Texas Southwestern Medical Center in Dallas. “I would say that is a type of platform technology that could probably be applied to dozens if not hundreds of diseases,” Gray said. “In the meantime, the best I can come up with is to treat one disease at a time.” As workshop presenters demonstrated throughout the meeting, gene therapy is at a moment of great potential. The challenge for NCATS, the FDA and other stakeholders is to find ways to keep accelerating progress so that no disease is left behind. Posted October 2018	NCATS and the Food and Drug Administration are collaborating to identify bottlenecks to gene therapy development and share best practices for advancing gene therapies.	/sites/default/files/gene-therapy3_1260x630.jpg	NCATS, FDA Co-Host Workshop on Gene Therapy for Rare Diseases	NCATS and the Food and Drug Administration are collaborating to identify bottlenecks to gene therapy development and share best practices for advancing gene therapies.	/sites/default/files/gene-therapy3_1260x630.jpg	NCATS, FDA Co-Host Workshop on Gene Therapy for Rare Diseases
12682	NIH Awards $86 Million to Improve Genome Editing Techniques	October 9, 2018Somatic Cell Genome Editing support is aimed at improving therapeutic options for both rare and common diseases.Making changes to a patients DNA can have powerful implications for the treatment of disease. To tap into this immense potential, NIH recently awarded 21 Somatic Cell Genome Editing grants — totaling approximately $86 million over the next five years, pending available funds — to support research aimed at improving methods to edit the human genome. These grants are the first to be awarded through the Somatic Cell Genome Editing (SCGE) program, which was launched in January 2018 with NIH Common Fund support. As a trans-NIH endeavor, SCGE is managed by staff from multiple NIH Institutes and Centers, with leadership from NCATS.An illustration depicting a genome editing tool bound to a DNA helix. New genome editing tools can substitute single “letters” in the DNA code without changing the surrounding DNA structure. (XVIVO Photo)A genome is an individual’s complete set of DNA, the chemicals that carry genetic information. Many diseases are caused by DNA changes that can be inherited from parents or can happen during a person’s lifetime. In the past decade, scientists have developed techniques to edit DNA in living cells. The newly awarded grants focus on somatic cells, which are cells in the body other than reproductive — e.g. sperm and egg — cells. One concern about manipulating genetic material has been the possibility of passing on unintended and potentially harmful genetic mutations. But because somatic cells do not pass DNA to the next generation, genome editing changes in somatic cells cannot be inherited. “Human genome editing technologies have opened up many far-reaching possibilities to treat disease,” said NCATS Director Christopher Austin, M.D. “The intent is that these newly funded initiatives will help speed the translation of genome editing to the clinic and to a greater number of patients with what are currently intractable illnesses and conditions.”Researchers continue to make improvements in genome editing techniques and understanding their effects, but significant barriers remain before they will be used widely to treat patients.“One challenge is to deliver genome editing tools to the right cells at the right time,” said P.J. Brooks, Ph.D., program director, NCATS Office of Rare Diseases Research. “With these awards, research teams will collaborate on several projects to improve the delivery of these tools to disease-relevant cells in the lung, muscle, brain and other tissues.”The awards will also support:New and more effective genome editing tools,Methods to evaluate the safety and effectiveness of various genome editing techniques in different human cell types,Specific animal models in which to test genome editing tools and methods,Testing centers to validate findings to ensure research results can be reproduced, andDevelopment of a genome editing toolkit — including lessons learned, and new techniques and tools — to be broadly disseminated to the scientific community.“Common Fund programs can boost a developing area of biomedical research but don’t focus on a specific organ system or disease,” said James M. Anderson, M.D., Ph.D., director of the Division of Program Coordination, Planning, and Strategic Initiatives, which oversees the NIH Common Fund. “If we can improve the safety and effectiveness of genome editing, we have the potential to treat a wide variety of diseases or conditions.”The broad applicability of genome editing to biomedical research is reflected in the diversity of NIH Institutes and Centers involved the SCGE program. In addition to NCATS, the NIH’s National Heart, Lung, and Blood Institute; National Institute of Allergy and Infectious Diseases; National Institute of Neurological Disorders and Stroke; National Institute of Arthritis and Musculoskeletal and Skin Diseases; and Office of Research Infrastructure Programs are managing these awards. The program will award researchers a total of approximately $190 million over six years, pending the availability of funds. For more information, visit https://ncats.nih.gov/research/research-activities/scge and https://commonfund.nih.gov/editing.For a list of the grantees and information about their awards, see: https://commonfund.nih.gov/editing/fundedresearch.To learn more about the SCGE program and its goals, watch an animated video: https://www.youtube.com/watch?v=27o3s3TktHI&t=1s.Hear an audio version of this story here: https://www.youtube.com/watch?v=0HKq35SHcTQ.	The 21 Somatic Cell Genome Editing program grants will support research aimed at improving treatment options for both rare and common diseases.	/sites/default/files/somatic_editor_1260x630.jpg	NIH Awards $86 Million to Improve Genome Editing Techniques	The 21 Somatic Cell Genome Editing program grants will support research aimed at improving treatment options for both rare and common diseases.	/sites/default/files/somatic_editor_1260x630.jpg	NIH Awards $86 Million to Improve Genome Editing Techniques
12637	NCATS Rare Diseases 2018 Detailed Submission Requirements	Solution Due Dates: Noon ET September 30, 2018 - Noon ET October 28, 2018 Please follow the instructions below to submit your entry. Registration Process for Participants To participate in the NCATS Rare Diseases are Not Rare! Challenge, every participant, whether an individual or member of a team, must first register. Participants may access the registration and submission platform at Challenge.gov by searching for “NCATS Rare Diseases are Not Rare!". To submit a solution on Challenge.gov, please paste a brief summary into the “Description” box. When submitting an entry, please note: The contents of submissions will be hidden from all others on Challenge.gov. Only the agency Challenge managers will see this content. All participants submitting entries must agree to the Terms and Conditions. Please review the detailed rules and information and then check the “agree to terms & conditions” box while submitting a solution on Challenge.gov. Use any format you choose, provided it supports the type of submission – be as creative and original as possible. Appropriate types of submissions (communication vehicles) include, but are not limited to, songs, poems, paintings, dramatic readings, mimes, puppets, posters, comics, animations, photos/collages or names. Each entry for this Challenge requires a complete “Submission Package.” The Submission Package includes a cover letter and the communication vehicle. Cover Letter The cover letter must be written in English and observe the page limit (1 page), page dimension (8.5 x 11 inches), font size (11 point or greater), and margin (1 inch) requirements. In the Cover Letter: Describe how your submission provides a solution to the Challenge (i.e.,how your entry addresses the problem). Explain why you selected the type of communication vehicle. Describe the target audience and suggest means of disseminating the entry. Communication Vehicle Follow all instructions at Challenge.gov to submit the material. Care should be given to select and upload the appropriate file types and formats. Note the following: File types are accepted on the Challenge.gov platform: Images: .jpg/.jpeg, .gif, .bmp, .png Microsoft Office: .doc, .docx, .ppt, .pptx, .pps, .ppsx, .xls, .xlsx PDF: .pdf, .ps Open Office: .odt, .odp, .sxw, .sxi, etc. Text: .txt, .rtf Audiovisual: .swf, .flv, .mp4, .avi, .mpeg Videos are limited to 2 minutes duration. Videos can be embedded in submissions via HapYak and YouTube; alternatively, videos can also be submitted by inserting a link to an external video. The maximum file size, indicated on the submission form, is currently set at 100MB. Note: You must not use the logo or official seal of HHS or the logo of NIH or NCATS in the entries and must not claim federal government endorsement.			NCATS Rare Diseases 2018 Detailed Submission Requirements			NCATS Rare Diseases 2018 Detailed Submission Requirements
12625	Rules for Participating in the Rare Diseases are Not Rare! Challenge	Solvers must be 18 years of age or older and may participate singly or as part of one or more teams. Teams are not limited in the number of members. Each team must designate a captain who must be a U.S. citizen or permanent resident who is responsible for all correspondence regarding this Challenge. Teams may also merge, collaborate, subdivide, or otherwise organize themselves and their members as needed to prepare a solution for this challenge. To be eligible to win a prize under this challenge, an individual or entity— Shall have registered to participate in the Challenge under the rules promulgated by the NIH as published in this Notice; Shall have complied with all the requirements set forth in this Notice; In the case of a private entity, shall be incorporated in and maintain a primary place of business in the United States, and in the case of an individual, whether participating singly or in a group, shall be a citizen or permanent resident of the United States. However, non-U.S. citizens and non-permanent residents can participate as a member of a team that otherwise satisfies the eligibility criteria. Non-U.S. citizens and non-permanent residents are not eligible to win a monetary prize (in whole or in part). Their participation as part of a winning team, if applicable, may be recognized when the results are announced. May not be a Federal entity or federal employee acting within the scope of their employment; May not be an employee of HHS (or any component of HHS) acting in their personal capacity; Who is employed by a federal agency or entity other than HHS (or any component of HHS), should consult with an agency Ethics Official to determine whether the federal ethics rules will limit or prohibit the acceptance of a prize under this challenge; May not be a judge of the challenge, or any other party involved with the design, production, execution, or distribution of the Challenge or the immediate family of such a party (i.e., spouse, parent, step-parent, child, or step-child). Federal grantees may not use Federal funds to develop their Submissions. Federal contractors may not use Federal funds from a contract to develop their Submissions or to fund efforts in support of their Submission. Submissions must not infringe upon any copyright or any other rights of any third party. By participating in this Challenge, each individual (whether competing singly or in a group) and entity agrees to assume any and all risks and waive claims against the Federal government and its related entities (as defined in the COMPETES Act), except in the case of willful misconduct, for any injury, death, damage, or loss of property, revenue, or profits, whether direct, indirect, or consequential, arising from participation in this Challenge, whether the injury, death, damage, or loss arises through negligence or otherwise. Based on the subject matter of the Challenge, the type of work that it will possibly require, as well as an analysis of the likelihood of any claims for death, bodily injury, property damage, or loss potentially resulting from Challenge participation, no individual (whether competing singly or in a group) or entity participating in the Challenge is required to obtain liability insurance or demonstrate financial responsibility in order to participate in this Challenge. By participating in this Challenge, each individual (whether competing singly or in a group) and entity agrees to indemnify the Federal government against third party claims for damages arising from or related to Challenge activities. An individual or entity shall not be deemed ineligible because the individual or entity used Federal facilities or consulted with Federal employees during the Challenge if the facilities and employees are made available to all individuals and entities participating in the Challenge on an equitable basis. By participating in this Challenge, each individual (whether participating singly or in a group) and each entity grants to the NIH an irrevocable, paid-up, royalty-free nonexclusive worldwide license to reproduce, publish, post, link to, share, and display publicly the Submission on the web or elsewhere. Each participant will retain all other intellectual property rights in their Submissions, as applicable. NIH reserves the right, in its sole discretion, to (a) cancel, suspend, or modify the Challenge, and/or (b) not award any prizes if no entries are deemed worthy. Each individual (whether participating singly or in a group) or entity agrees to follow all applicable federal, state, and local laws, regulations, and policies. By participating in this Challenge, each individual (whether participating singly or in a group) warrants that he or she is the sole author or owner of, or has the right to use, any copyrightable works that the Submission comprises, that the works are wholly original with the Solver (or is an improved version of an existing work that the Solver has sufficient rights to use and improve), and that the Submission does not infringe any copyright or any other rights of any third party of which Solver is aware. To receive an award, Solvers will not be required to transfer their intellectual property rights to NCATS, but Solvers must grant to the federal government a nonexclusive license to practice their solutions and use the materials that describe them. This license must grant to the United States government a nonexclusive, nontransferable, irrevocable, paid-up, royalty-free license to practice or have practiced for or on behalf of the United States throughout the world any invention made by the Solvers that covers the Submission. In addition, the license must grant to the federal government and others acting on its behalf, a fully paid, nonexclusive, irrevocable, worldwide license in any copyrightable works that the Submission comprises, including the right to reproduce, prepare derivative works, distribute copies to the public, and perform publicly and display publicly said copyrightable works. To participate in the Challenge, each Solver must warrant that there are no legal obstacles to providing the above-referenced nonexclusive licenses of Solver’s rights to the federal government. Each individual (whether participating singly or in a group) and entity participating in this Challenge must comply with all terms and conditions of these rules, and participation in this Challenge constitutes each such participant’s full and unconditional agreement to abide by these rules. Winning is contingent upon fulfilling all requirements herein. By participating in this Challenge, each individual (whether participating singly or in a group) agrees to allow NCATS to publicly display (e.g., on web sites) solutions.			Rules for Participating in the Rare Diseases are Not Rare! Challenge			Rules for Participating in the Rare Diseases are Not Rare! Challenge
12622	NCATS Rare Diseases Are Not Rare! 2018 Challenge Details	Summary for NCATS Rare Diseases Are Not Rare! Challenge Dates and Deadlines For Further Information Contact The IC's Statutory Authority to Conduct the Challenge Subject of the Challenge Competition Rules for Participating in the Challenge The Prize Evaluation and Winner Selection Submission Requirements and Instructions Challenge Description Summary for NCATS Rare Diseases Are Not Rare! Challenge The National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), is seeking innovative ways to communicate with others to educate people about rare diseases through social media or art. The goal of this Challenge, which is being led by NCATS’ Office of Rare Diseases Research, is threefold: First and foremost, it is to raise awareness for all rare diseases in a collective manner. Second, it is intended to bring attention to the many people with rare diseases; and finally, it is to highlight the need for research and the development of new treatments. You can help us get the word out by competing in our rare diseases prize competition! In addition to cash prizes for 1st, 2nd and 3rd place winners, the 1st place winner(s) will be invited to NIH to present the winning entry at the Rare Disease Day at NIH event (February 2019); all winners and 10 honorable mentions will be posted on the NCATS public website. Evaluation criteria that judges will be asked to address are specified below. Dates and Deadlines Entries must be submitted to Challenge.gov by Noon Eastern time on October 28, 2018. The Challenge begins: September 30, 2018 Submission Period: September 30, 2018 – October 28, 2018 Judging Period: November 12, 2018 – November 26, 2018 Winners Announced: December 2018 For Further Information Contact alice.chen2@nih.gov, M.D., NCATS, NIH, 301-827-2015; or send an email to DRDRI@nih.gov. The IC's Statutory Authority to Conduct the Challenge The general purpose of NCATS is to coordinate and develop resources that leverage basic research in support of translational science and to develop partnerships and work cooperatively to foster synergy in ways that do not create duplication, redundancy or competition with industry activities (42 USC 287(a)). To fulfill its mission, NCATS supports projects that will transform the translational process so that new treatments and cures for diseases can be delivered to patients faster by understanding the translational process and to create a basis for more science-driven, predictive and effective intervention development for the prevention and treatment of all diseases. NCATS supports rare disease patients and their communities by providing translational research funding, tools and other resources that help address their unique Challenges. It is beneficial to all stakeholders for NCATS to optimize the communication tools available to support effective information dissemination and education. NCATS also is conducting this Challenge under the America Creating Opportunities to Meaningfully Promote Excellence in Technology, Education, and Science (COMPETES) Reauthorization Act of 2010, 15 U.S.C. 3719. In line with these authorities, this Challenge will lead to innovative ways to communicate with others and to educate people about rare diseases through social media and/or art. Subject of the Challenge Competition Rare Diseases Are Not Rare! If you know 10 people, chances are you know someone with a rare disease. There are about 7,000 different rare diseases that affect an estimated 30 million Americans. This is more than twice the number of people living with cancer, more than the number of people living with HIV and Alzheimer’s disease combined, and more than the population of Texas. Some difficulties with rare diseases are that they are hard to recognize, are often hidden conditions, and most currently are not being studied via ongoing medical research. We are asking you to help us bring attention to rare diseases so that they can gain more medical research interest, thereby improving the lives of people with rare diseases. Science, especially genetic medicine, has moved forward to the point now where treatments are possible — such as gene therapies, 3-D printing (devices, tissues and organs) and new drugs. Everyone deserves a chance at an effective medical treatment — whether for a common disease or a rare one — so let’s get the word out! Here are a few facts to consider: Most rare diseases are genetic (around 80%) — they are caused by changes to a person’s DNA (mutations) usually present at birth. We all have DNA, and we all have mutations, whether they cause a disease or not depends on where the mutations are and whether they impact our ability to function. Rare diseases also can be due to infections, such as Ebola, or in the U.S., malaria, Chagas’ disease or tuberculosis. Many types of cancer, such as leukemia and lymphoma, and brain tumors are rare diseases. About half of those diagnosed with rare diseases are children. Rare diseases can affect anyone, in any family, anywhere in the world. Sometimes they run in families, but often they occur with no family history. Rare diseases can affect many different organs and disease areas, such as rare lung diseases (e.g., cystic fibrosis), movement (muscular dystrophy), the brain (certain types of autism or cerebral palsy), the blood (sickle cell anemia), and many others. “Cancer” is actually a collection of hundreds of diseases, even though it is often referred to by just one easily recognizable word. Many common diseases are actually collections of several different rare diseases that affect people in a similar way. For example, “breast cancer” is actually a collection of several different cancers, some of which are rare. You can help by competing in our rare disease prize competition. Here’s what we’re asking you to do: Find a way to communicate with others and to educate people about rare diseases through social media or art. Use any communication vehicle you choose; be as creative and original as possible. Here are examples of appropriate communication vehicles: music video song (with or without sheet music) dramatic reading poem painting mime poster comic animation photo/collage new name for “rare diseases” as a whole puppets Each team or individual may submit only one entry. Rules for Participating in the Challenge Solvers must be 18 years of age or older and may participate singly or as part of one or more teams. Teams are not limited in the number of members. Each team must designate a captain who must be a U.S. citizen or permanent resident who is responsible for all correspondence regarding this Challenge. Teams also may merge, collaborate, subdivide or otherwise organize themselves and their members as needed to prepare a solution for this Challenge. To be eligible to win a prize under this Challenge, an individual or entity— Shall have registered to participate in the Challenge under the rules promulgated by the NIH as published in this Notice; Shall have complied with all the requirements set forth in this Notice; In the case of a private entity, shall be incorporated in and maintain a primary place of business in the United States, and in the case of an individual, whether participating singly or in a group, shall be a citizen or permanent resident of the United States. However, non-U.S. citizens and non-permanent residents can participate as a member of a team that otherwise satisfies the eligibility criteria. Non-U.S. citizens and non-permanent residents are not eligible to win a monetary prize (in whole or in part). Their participation as part of a winning team, if applicable, may be recognized when the results are announced. May not be a Federal entity or federal employee acting within the scope of their employment; May not be an employee of HHS (or any component of HHS) acting in their personal capacity; Who is employed by a federal agency or entity other than HHS (or any component of HHS), should consult with an agency Ethics Official to determine whether the federal ethics rules will limit or prohibit the acceptance of a prize under this Challenge; May not be a judge of the Challenge, or any other party involved with the design, production, execution, or distribution of the Challenge or the immediate family of such a party (i.e., spouse, parent, step-parent, child, or step-child). Federal grantees may not use Federal funds to develop their Submissions. Federal contractors may not use Federal funds from a contract to develop their Submissions or to fund efforts in support of their Submission. Submissions must not infringe upon any copyright or any other rights of any third party. By participating in this Challenge, each individual (whether competing singly or in a group) and entity agrees to assume any and all risks and waive claims against the Federal government and its related entities (as defined in the COMPETES Act), except in the case of willful misconduct, for any injury, death, damage, or loss of property, revenue, or profits, whether direct, indirect, or consequential, arising from participation in this Challenge, whether the injury, death, damage, or loss arises through negligence or otherwise. Based on the subject matter of the Challenge, the type of work that it will possibly require, as well as an analysis of the likelihood of any claims for death, bodily injury, property damage, or loss potentially resulting from Challenge participation, no individual (whether competing singly or in a group) or entity participating in the Challenge is required to obtain liability insurance or demonstrate financial responsibility in order to participate in this Challenge. By participating in this Challenge, each individual (whether competing singly or in a group) and entity agrees to indemnify the Federal government against third party claims for damages arising from or related to Challenge activities. An individual or entity shall not be deemed ineligible because the individual or entity used Federal facilities or consulted with Federal employees during the Challenge if the facilities and employees are made available to all individuals and entities participating in the Challenge on an equitable basis. By participating in this Challenge, each individual (whether participating singly or in a group) and each entity grants to the NIH an irrevocable, paid-up, royalty-free nonexclusive worldwide license to reproduce, publish, post, link to, share, and display publicly the Submission on the web or elsewhere. Each participant will retain all other intellectual property rights in their Submissions, as applicable. NIH reserves the right, in its sole discretion, to (a) cancel, suspend, or modify the Challenge, and/or (b) not award any prizes if no entries are deemed worthy. Each individual (whether participating singly or in a group) or entity agrees to follow all applicable federal, state, and local laws, regulations, and policies. By participating in this Challenge, each individual (whether participating singly or in a group) warrants that he or she is the sole author or owner of, or has the right to use, any copyrightable works that the Submission comprises, that the works are wholly original with the Solver (or is an improved version of an existing work that the Solver has sufficient rights to use and improve), and that the Submission does not infringe any copyright or any other rights of any third party of which Solver is aware. To receive an award, Solvers will not be required to transfer their intellectual property rights to NCATS, but Solvers must grant to the federal government a nonexclusive license to practice their solutions and use the materials that describe them. This license must grant to the United States government a nonexclusive, nontransferable, irrevocable, paid-up, royalty-free license to practice or have practiced for or on behalf of the United States throughout the world any invention made by the Solvers that covers the Submission. In addition, the license must grant to the federal government and others acting on its behalf, a fully paid, nonexclusive, irrevocable, worldwide license in any copyrightable works that the Submission comprises, including the right to reproduce, prepare derivative works, distribute copies to the public, and perform publicly and display publicly said copyrightable works. To participate in the Challenge, each Solver must warrant that there are no legal obstacles to providing the above-referenced nonexclusive licenses of Solver’s rights to the federal government. Each individual (whether participating singly or in a group) and entity participating in this Challenge must comply with all terms and conditions of these rules, and participation in this Challenge constitutes each such participant’s full and unconditional agreement to abide by these rules. Winning is contingent upon fulfilling all requirements herein. By participating in this Challenge, each individual (whether participating singly or in a group) agrees to allow NCATS to publicly display (e.g., on web sites) solutions. The Prize Amount of the Prize; Award Approving Official. The total prize purse is up to $5,000 awarded as follows: First place: $3000 and travel expenses for up to four people to participate in Rare Disease Day at NIH, February 28, 2019 to present the winning entry Second place: $1500 Third place: $500 Honorable mentions (10) will be posted on NCATS’ website The NIH reserves the right to cancel, suspend, and/or modify this Challenge at any time through amendment to this Notice. In addition, the NIH reserves the right to not award any prizes if no entries are deemed worthy. The Award Approving Official will be Christopher P. Austin, M.D., NCATS director. Payment of the Prize. Prizes awarded under this competition will be paid by electronic funds transfer and may be subject to federal income taxes. HHS/NIH will comply with the Internal Revenue Service withholding and reporting requirements, where applicable. Evaluation and Winner Selection Basis upon Which Winner Will Be Selected. A panel of federal and non-federal judges, with expertise directly relevant to this Challenge, will evaluate the entries based on criteria listed below and will select the Challenge winners. NCATS will provide feedback from the judges to the winners and non-winners. The percentages assigned to each set of evaluation criteria are guidelines from NCATS to suggest which features are of emphasis and interest to the Center. Only complete submissions will be reviewed. Submission Requirements and Instructions Instructions for submission: Use any format you choose, provided it supports the type of submission – be as creative and original as possible. Appropriate types of submissions (communication vehicles) include, but are not limited to, songs, poems, paintings, dramatic readings, mimes, puppets, posters, comics, animations, photos/collages or names. Each submission for this Challenge requires a complete “Submission Package.” The Submission Package includes a cover letter and the communication vehicle. Cover Letter The cover letter must be written in English and observe the page limit (1 page), page dimensions (8.5 x 11 inches), font size (11 point or greater), and margins (1 inch). In the Cover Letter: Describe how your submission provides a solution to the Challenge (i.e., how your entry addresses the problem). Explain why you selected the type of communication vehicle. Describe the target audience and suggest means of disseminating the entry. Communication Vehicle Follow the instructions at Challenge.gov to submit the material. Care should be given to select and upload the appropriate file types and formats. Videos are limited to 2 minutes duration. Note: You must not use HHS’s logo or official seal or the logo of NIH or NCATS in the entries and must not claim federal government endorsement. Additional details can be found at: Detailed submission requirements and instructions. Challenge Description Find a way to communicate with others and to educate people about rare diseases through social media or art. For example: Create a music video or animation Write a song (with or without sheet music) Take a photo/create a collage or puppets Create a poem, a poster, a comic, painting or a dramatic reading Come up with a new name for “rare diseases” as a whole Entries will receive up to 5 points for each criterion, for a total of up to 15 points per entry. How creative and original is the entry? To what extent does the entry address rare diseases collectively? How likely is it that the entry could be an effective communication vehicle? Will it appeal to a broad audience? Is it easy to disseminate? Judging criteria: Basis Upon Which Submissions Will Be Evaluated. Note: You must not use HHS’s logo or official seal or the logo of NIH or NCATS in the entries and must not claim federal government endorsement.			NCATS Rare Diseases Are Not Rare! 2018 Challenge Details			NCATS Rare Diseases Are Not Rare! 2018 Challenge Details
12619	NCATS Challenges	This page is for those who are interested in learning more about NCATS' incentive challenges. This resource is intended to be a knowledge repository that includes challenge prize information and submission requirements. See below for specific challenge information. NCATS Rare Diseases Are Not Rare! Challenge Prize Details Detailed Submission Requirements and Instructions			NCATS Challenges			NCATS Challenges
13156	2018 NCATS ASPIRE Design Challenges	Overview of NCATS ASPIRE Design Challenges ×The 2018 NCATS ASPIRE Design Challenges have concluded. See the 2018 Challenge winners. The 2018 NCATS ASPIRE Design Challenges were launched to encourage innovative and catalytic approaches toward solving the opioid crisis by developing “A Specialized Platform for Innovative Research Exploration” (ASPIRE). Developed under the auspices of the Cures Acceleration Network, the NCATS ASPIRE program aims to accelerate the discovery and development of novel and effective treatments, while at the same time making the process faster and more cost-effective. Integrating advancements in computer-aided drug design, automated synthetic chemistry and high-throughput biological screening, the ASPIRE program will provide a new opportunity to break the translational bottlenecks in chemistry and benefit many areas of science and human health. Through the ASPIRE Design Challenges, NCATS is using the ASPIRE platform to support the National Institutes of Health (NIH) Helping to End Addiction Long-termSM Initiative, or NIH HEAL InitiativeSM, a transagency effort focused on improving prevention and treatment strategies for opioid misuse and addiction and enhancing pain management. Launched in April 2018 with funding from Congress, the NIH HEAL Initiative brings new hope for people, families and communities affected by the national opioid public health crisis. The goal of the ASPIRE Design Challenges is to propose innovative and catalytic approaches toward solving the opioid crisis through the development of next-generation addiction-free analgesics with new chemistries, data-mining and analytical tools and technologies, as well as biological assays that will revolutionize discovery, development and preclinical testing of new and safer treatments of pain, opioid use disorder (OUD) and overdose. This prize competition was implemented through a suite of concurrent companion design challenges in four areas: an integrated chemistry database, an electronic synthetic chemistry portal, predictive algorithms and biological assays, as well as an additional challenge for a combined solution for two or more areas. NCATS will select up to five winners for Challenges 1–4 and up to two winners for Challenge 5 to receive cash prizes. NCATS continued the 2018 NCATS ASPIRE Design Challenges with a distinct follow-up, the 2020 Reduction-to-Practice Challenge, which aimed to spur the development of a comprehensive integrated platform for translational innovation in pain, opioid use disorder and overdose. Learn more about the Challenge. These Challenges aim to generate valuable contributions from many stakeholders to inform the Center’s strategic plan, energize the Center’s research efforts, increase public awareness of the opioid crisis and enhance the national effort to reduce the number of opioid overdoses worldwide. Details of the Five Challenges Challenge 1: Integrated Chemistry Database for Translational Innovation in Pain, Opioid Use Disorder and Overdose Challenge 2: Electronic Synthetic Chemistry Portal for Translational Innovation in Pain, Opioid Use Disorder and Overdose Challenge 3: Predictive Algorithms for Translational Innovation in Pain, Opioid Use Disorder and Overdose Challenge 4: Biological Assays for Translational Innovation in Pain, Opioid Use Disorder and Overdose Challenge 5: Integrated Solution for Translational Innovation in Pain, Opioid Use Disorder and Overdose Dates and Deadlines The challenge submission period is now closed. Check back soon to see more information about the winners. The Challenge began: December 31, 2018 Submission period: December 31, 2018-May 31, 2019 Judging period: June 17, 2019-August 2, 2019 (List of Judges) Winners announced: August 2019 View the 2018 NCATS ASPIRE Design Challenge Winners For further information send an email to NCATSASPIREChallenge@mail.nih.gov.		/sites/default/files/ASPIRE-badges_900x900_0.png	2018 NCATS ASPIRE Design Challenges		/sites/default/files/ASPIRE-badges_900x900_1.png	2018 NCATS ASPIRE Design Challenges

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