Brandy E. Fureman PhD
Vice President, Research & New Therapies

There is a transformation underway in the research ecosystem; the advent of patient engagement in determining the focus, design, and conduct of research studies and dissemination of their results. This means that the roles of patients and caregivers are changing from research “subjects” to research partners. This paradigm shift provides increased leverage and impact of the stakeholder group that has the most at stake in developing new treatments for diseases. The culture change is apparent in several recent pieces of legislation, now law, including the Affordable Care Act's creation of the Patient Centered Outcomes Research Institute (PCORI), the Food and Drug Administration Safety and Innovation Act's section 1137: Patient participation in medical product discussions [1] and in the 21st Century Cures Act's emphasis on putting patient perspectives first [2]. A growing number of non-profit patient voluntary organizations are also endorsing and employing patient engagement strategies in their work to advance research and cures for disease.

Indeed, FasterCures, an action tank that works to speed and improve the medical research system, is leading the charge to develop a new field; the science of patient input. Their goals are to develop rigorous methods to integrate patient perspectives, needs and priorities into studies across the therapy development pipeline [3]; [4]. FasterCures has collected toolkits to enable patient engagement through its Patients Count [5] initiative, and regularly brings together stakeholders from patient non-profit, government, industry and academic sectors to strategize solutions to accelerate progress in medcal research.

In the epilepsy community, we are embracing patient engagement principles in several large consortia and research projects underway. The Rare Epilepsy Network [6], funded by PCORI, is a patient-powered research network that brings together over thirty different organizations representing people living with rare epilepsies and their caregivers. Enrollment in REN is currently at 1250 individuals and growing, and the survey data collected thus far is available by request for researchers interested in analyzing their own questions about one or more of the rare epilepsies [7]. The most recently funded NINDS Center Without Walls (CWOW), the Epilepsy Bioinformatics Study For Antiepileptogenic Therapy (Epibios4rx; Grant identifier U54NS100064) has a Public Engagement Core that will actively involve voluntary organizations with the EpiBioS4Rx CWOW in public outreach to successfully design and complete clinical research studies, including future trials of prevention therapy in epilepsy. Many of the patient-led organizations on the Epilepsy Leadership Council are hosting, or in the process of building, their own research initiatives (registries, biorepositories, priority-setting conferences and grant funding programs) that embed the patient perspective into the research being conducted on their disorder. The Epilepsy Foundation has also taken these principles to heart. Our newest research program, the Epilepsy Innovation Institute, solicits input from people impacted by epilepsy to guide the selection of topics for the institute to tackle.

The emphasis on patient engagement is changing the epilepsy research ecosystem for the better. A culture of connection between patient partners and clinical investigators from academia and industry can ensure that the perspectives and needs of the study population are included in the conceptualization and design of clinical research studies.

References

  1. www.fda.gov/downloads/ForPatients/About/UCM486859.pdf [Accessed April 19, 2017]
  2. https://blogs.fda.gov/fdavoice/index.php/2016/12/21st-century-cures-act-making-progress-on-shared-goals-for-patients/ [Accessed April 19, 2017]
  3. M. Anderson, McCleary K. Kimberly. "On the path to a science of patient input," Sci Transl Med, 8 (336) (2016), Article 336ps11.
  4. M. Anderson, K.K. McCleary. "From passengers to co-pilots: Patient roles expand," Sci Transl Med, 7 (291) (2015), Article 291fs25.
  5. www.fastercures.org/programs/patients-count/ [Accessed April 19, 2017]
  6. https://ren.rti.org/ [Accessed April 19, 2017]
  7. https://ren.rti.org/Researchers/Request-Data

In Epilepsy & Behavior; July 2017; 72: 71.

Reprinted with permission from Epilepsy & Behavior, Elsevier for Editorials.