First NCATS Day Demonstrates Commitment to Patients Through Smarter Science

To gain more insight about patients’ needs and discuss opportunities to integrate the patient perspective into translational research, NCATS convened the first NCATS Day on June 30, 2017, on the NIH campus in Bethesda, Maryland. More than 150 people attended, including patients, their families and other caregivers, and representatives of more than 75 patient and disease advocacy groups.

With a theme of “Partnering with Patients for Smarter Science,” the all-day event enabled participants to learn more about NCATS and research supported by the Center. It also served as a forum for NCATS staff and researchers to hear directly from patients about their needs, establish new communication channels or strengthen existing ones and identify ways to enhance patient participation in research.

The Power of Partnering with Patients

The process of developing new treatments takes too long and is too costly. The situation, especially for rare diseases, has been bleak.

“Incremental change is not going to do it,” said NCATS Director Christopher P. Austin, M.D., during his opening remarks. “We need a quantum-level change in our efficiency and effectiveness.”

Austin noted that biomedical researchers are passionate about learning about diseases and finding new and better treatments but often fail to ask the people who are supposed to benefit from that work about what they want from the products under development. Although many NCATS programs place a high value on patient engagement, it is not always obvious how patients and their support organizations can be involved in research. This event was designed to begin a dialogue between NCATS and the public about how the patient perspective can be included in translational science.

James Hendrix, Ph.D., director of global science initiatives for the Medical and Scientific Relations Division of the Alzheimer’s Association, thought the time might be right for disruptive research. “The time is coming to be heretical — even crazy — to transform the current paradigm for developing new treatments,” he said. “What seems to be the most logical path from basic science to new treatments may be too slow. The current situation demands approaches that are higher in risk but potentially higher in reward.” Daniel Soñé Photography.

“Too frequently, at the end of the drug development process, the treatment misses the mark in terms of patients’ needs,” Austin said. “At NCATS, we are committed to getting input from the people living with diseases to make sure that research aligns with their priorities.”

Dr. Christopher Austin. Daniel Soñé Photography.

That is why NCATS strives to find ways to involve patients in all stages of research, ranging from preclinical studies using cell lines and animal models to human trials for evaluating investigational drugs and to dissemination and implementation research focused on identifying best practices. The second goal in NCATS’ Strategic Plan embodies this concept: As the eventual recipients of these research endeavors, patients’ participation as members of the research team provides insight and meaning to the creation, testing and clinical implementation of new medical interventions.

About a third of NCATS Advisory Council members are disease advocates, patients or caregivers. In addition, institutions funded through NCATS’ Clinical and Translational Science Awards (CTSA) Program have community advisory boards to help promote patient engagement and provide feedback to researchers. CTSA Program hubs support training on patient and community engagement, and many employ patient engagement specialists. In addition, CTSA Program researchers have access to the Recruitment Innovation Center, which is transforming recruitment methods in order to enroll participants in clinical trials more quickly, thereby overcoming a major barrier in the testing of potential new treatments.

A Promising Example of Collaboration with Advocates

Many different paths are available to patient groups interested in accelerating translational science. For example, advocacy organizations can directly support research and scientists studying the disease of interest. This approach was highlighted in a case study presented during NCATS Day by Michael Iannotti, Ph.D., a postdoctoral fellow in the NCATS Division of Preclinical Innovation, and Jean-Marc Quach, M.B.A., the executive director of The Alpha-1 Project at the Alpha-1 Foundation. They demonstrated how the foundation is working with NCATS to find new treatments for patients who have a defect in an enzyme called alpha-1 antitrypsin, causing serious liver and lung disease. The Alpha-1 Project supports a research fellowship for Iannotti at NCATS, where he designs new assays (tests) and conducts research into promising drug candidates that could help correct the enzyme’s function. 

Michael Iannotti, Ph.D. (left), listened as Jean-Marc Quach, M.B.A. (right), explained, “This partnership is succeeding because it builds on the strengths of the Alpha-1 Foundation and NCATS. NCATS is sharing its technical expertise, technologies and libraries of compounds. The foundation contributes its deep knowledge of patients’ needs and the resources supporting Iannotti’s research. We are assembling tools and materials that bring us several steps closer to finding a cure for alpha-1 antitrypsin disease.” Daniel Soñé Photography.

“Working with a disease advocacy group has given me opportunities to meet and talk with patients and their families,” Iannotti said. “These interactions would normally not be available to me, since my work is performed in a research lab, not in a clinic. Spending time with the patient community has strengthened my commitment to discovering therapies for alpha-1 antitrypsin deficiency.”

NCATS Day participants found the case study enlightening and suggested that future communications include more of these examples presented not only by scientists but also by patients, caregivers and other advocates.

Showcasing NCATS Programs and Initiatives

A series of presentations and poster sessions highlighted a wide range of exciting work taking place at NCATS or with Center support. The idea was to ignite interest in translational research and help patients and advocates identify research programs that might fit with their interests and needs.

For example, Mary Dollear, vice president of the Lupus Society of Illinois, said of NCATS’ Tissue Chip for Drug Screening program, “I am really excited to learn about this technology. People with lupus are often reluctant to participate in clinical trials, because they worry about their disease flaring up. But the tissue chips might be a way to test treatments on tissues from patients to see what might work for them — sort of like a clinical trial for each person.” 

Kathryn Devanny, M.A., M.P.H., director of advocacy for PatientWorthy, said, “I was interested in this event because I wanted to know more about how rare disease patient advocacy groups, both formal and informal, could interact with NCATS’ programs. Advocacy groups are a critical source of patient and disease information, spanning natural history to DNA, and their goals dovetail with those of NCATS. How would such organizations take the first step to working with NCATS? What is the best way for them to engage the scientists, researchers and technology experts at NCATS?” Daniel Soñé Photography. 

The Big Picture

NCATS Day wrapped up with a group discussion focused on the key ideas advanced during earlier breakout sessions. Several themes emerged, including the importance of two-way communication between scientists and patients. The scientific community should discuss research in easily understood language. Efforts to boost science literacy could help communities understand the research process and scientific evidence, as well as roles that they can take on. 

Camille Hart, M.P.H., program manager for community engagement at the CTSA Program hub at the University of Arkansas for Medical Sciences, was on a quest for information about NCATS programs that would be of interest to community partners — both organizations and individuals — back in Arkansas. “Last month, we ran a Patient Scientist Academy to help patients learn about how they can get involved in research as advisors, grant reviewers or study participants,” Hart said. “I am always on the lookout for new ways to engage people in the research process.” Daniel Soñé Photography. 

For example, Jessica Nagro, federal government and health policy manager with the National Psoriasis Foundation, shared an idea: “Our foundation set up a program called Citizen Pscientist that links people with psoriatic disease in the first-ever global online research network dedicated to this condition. The Citizen Pscientists answer questions prepared by researchers, and they can conduct their own analyses and discuss the information.”

Other comments touched on the need for training scientists to engage patients in research in meaningful ways, especially in early-stage studies. Event participants also underscored the importance of recruiting diverse and underserved populations for clinical trials. 

Sue Hargrave of Itching to Help!, LLC, NPC, an advocacy group helping people with chronic urticaria (hives), discussed a poster with Madhu Lal-Nag, Ph.D., lead of the trans-NIH RNAi facility in NCATS’ Division of Preclinical Innovation. Hargrave identified scientific literacy as a patient need and said she was at NCATS Day to “lurk and learn” from Center staff and representatives of other organizations. Communication tools might help patients sort fact from fiction. “Too often, patients focus on ‘treatments’ they read about on the Internet — things that are unproven,” she said. “How can we help people distinguish treatments based on real scientific evidence from anecdotes on social media?” Daniel Soñé Photography. 

Several participants expressed enthusiasm about engaging with NCATS and were interested in “on-ramps” or a “matchmaking” system to find the best fit between patient/disease organizations and the Center. Building on this idea, another suggestion was to create a “decision tree” for moving through translational science, including working with the U.S. Food and Drug Administration and the Centers for Medicare & Medicaid Services.

Several people envisioned opportunities for advocacy groups to come together to develop research platforms and share resources to address collective needs.

Stay Tuned — There’s More to Come

NCATS Day was the first of many events being planned to strengthen ties between the Center and the patient community. On Sept. 8, 2017, NCATS will host rare diseases and other patient community members at its Toolkit for Patient-Focused Therapy Development: Demonstration and Dissemination Meeting. Participants will have an opportunity to learn how the toolkit can help with therapeutic development activities and to provide feedback to expand available content.

“Translational science is a team sport,” Austin said. “We need contributions from all the team members, especially patients, their family members and advocacy organizations. By working together, we can get treatments to patients sooner and ensure that treatments meet patients’ needs.”

And that’s what smarter science is all about.

Posted July 2017