Translating a basic discovery to a demonstrated improvement in public health requires a translational research team of scientists, clinicians, research participants and other stakeholders having a wide range of expertise and perspectives of the scientific and operational roadblocks. Progressing through the phases of the translational science spectrum requires the creation of productive and mutually beneficial collaborations that depend not only on individual excellence, but on teamwork, coordination, cooperation and communication. Partnerships and collaborations across individuals and organizations are essential because the expertise, capabilities and viewpoints required for successful translation tend to reside in different groups with distinct missions. For example, developing a potential therapy to the point of regulatory approval can require expertise in molecular biology, medicinal chemistry, compound synthesis and formulation, pharmacology and toxicology, technology transfer, clinical science, regulatory science, integrating patient perspectives, and entrepreneurship, among others. However, incentive structures and professional and cultural barriers can make teamwork difficult to navigate. For this reason, NCATS places high value on innovation in team science and partnership development, and designs and tests novel partnership structures that cut across traditionally siloed scientific disciplines, organizations and sectors. Successful approaches are shared for all in the biomedical research community to use to improve their own translational efforts. Additionally, NCATS recognizes that translation is a global effort and therefore engages in partnerships and collaborations with international entities in multiple sectors.
Objective 2-1: Engage patients, community members and nonprofit organizations meaningfully in translational science, and develop and broadly disseminate best practices for patient-focused research.
Engaging patients, community members and nonprofit organizations in all aspects of translational research is important to advancing translation. As the eventual recipients of these research endeavors, patient participation as members of the research team provides insight and meaning to the creation, testing and clinical implementation of new medical interventions. Engagement of patients and their communities throughout the lifecycle of a translational research project ensures the outcomes are relevant to and directly address patient needs and will be more readily adopted by the community. NCATS views the science of patient and community engagement as a key area for exploration and innovation and will drive research efforts to develop, demonstrate and broadly disseminate best practices for patient-focused research.
- Identify and disseminate fundamental principles of successful patient and community engagement.
- Develop educational and training resources for diverse audiences on the best practices for patient and community engagement.
- Identify the factors that facilitate patient-centered activities and develop tools to reduce resistance or remove barriers to patient engagement.
- Ensure that all research activities at NCATS appropriately include patient and community members in efforts that improve research design, implementation and dissemination.
Objective 2-2: Share resources and expertise across the federal government through collaborative research, particularly with other NIH Institutes and Centers.
NCATS serves as a partner, convener and expert on translational science and shares its expertise and resources with colleagues at other NIH Institutes and Centers (ICs) and across the federal government. NCATS enables our colleagues to move their translational efforts to a higher level by matching their domains of expertise with NCATS’ translational science expertise and technologies to advance research more efficiently and effectively. NCATS also attempts to bridge translational pitfalls by identifying and collaborating with other government agencies to enable the research to progress, even after NCATS’ specific role in the effort is complete. Partnering with researchers across NIH who have knowledge of particular disease areas is essential to demonstrating the utility of translational technologies and strategies developed by NCATS. The same is true with other government agencies with complementary roles in the translational ecosystem. For example, partnerships with the Food and Drug Administration (FDA) are critical to ensuring innovations and interventions are developed in close consultation with the regulators who will ultimately evaluate them. NCATS is committed to working with all of its federal partners to collaboratively advance translational science.
- Partner with NIH ICs to identify common roadblocks in translation that apply across diseases so that NCATS can focus its science in areas of greatest need.
- Partner with NIH ICs to support translational research to accelerate intervention development by combining translational science expertise with disease expertise.
- Create and share template agreements that facilitate the initiation of new collaborations among various stakeholders with government agencies.
- Collectively develop the scientific tools, standards and approaches that support efficient translation of research for regulatory assessment.
- Serve as a convener for issues of common interest across the federal government with regard to translational research and translational science.
- Provide interagency leadership to coordinate rare diseases research.
- Coordinate with the FDA and other federal agencies to advance areas of the translational landscape that are rapidly evolving, such as adaptive/novel clinical trial designs, and patient and community engagement strategies.
Objective 2-3: Form innovative collaborations with scientists at academic and other research institutions to translate early-stage discoveries into novel medical interventions.
Investigators at research institutions in the U.S. and around the world make discoveries in basic, clinical and public health research that can serve as the foundation for translational research to improve public health. Academic medical research institutions have extensive capabilities and capacity to carry out clinical, patient care, and public and community health research. NCATS teams with these investigators to collaboratively “de-risk” promising pre-clinical interventions by applying expertise and resources to efficiently generate necessary evidence and data for safety and efficacy assessment. When successful, these collaborative efforts provide evidence to attract external partners for further development and dissemination. Importantly, each collaborative project has a dual mission: first, to de-risk and advance a project to the next phase of translation; and second, to understand the scientific and operational underpinnings of successful approaches and apply them to future projects for more efficient translation.
- Build novel types of collaborations with researchers to combine their deep knowledge of disease biology and chemistry with the expertise of NCATS researchers in pre-clinical development, project management, clinical research and regulatory science to advance the development of promising therapeutic candidates.
- Design innovative collaborative projects to advance clinical translational science in all facets from first-in-human intervention studies to community health.
- Form and participate in international partnerships with groups and organizations around the world as appropriate to leverage complementary capacities and accelerate progress.
Objective 2-4: Devise new collaborative structures with private-sector partners to advance translational technologies for patient benefit.
NCATS’ mission includes devising new collaborative partnerships with private-sector entities in order to increase the efficiency and effectiveness of translation. The Center proactively seeks the necessary partners to ensure that the programs to which it commits resources have the greatest chance of success in reaching patients. Such projects in the later stages of therapeutic development generally require collaboration with a biopharmaceutical company with the expertise and financial and technical resources to achieve the ultimate goal of patient benefit. NCATS also supports small businesses and entrepreneurship programs as an integral part of advancing translational science and developing new translational technologies.
- Partner with colleagues in the pharmaceutical, biotechnology and venture capital communities to identify and address translational science roadblocks in their areas of expertise.
- Identify common priorities and projects with industry partners that would leverage complementary efforts to achieve translational goals more efficiently, and avoid duplication.
- Promote collaborative efforts between biopharmaceutical companies and the biomedical research community to advance therapeutics development, particularly in the areas of rare diseases and drug repurposing.
- Assist small businesses in developing and commercializing translational technologies and products, such as innovative tools and platforms that would support the creation of interventions or disease diagnostics.
- Partner with the private sector to provide exchange programs and experiential learning opportunities for translational scientists.