Gaijin Panty

Last week, the seventh annual Patient and Health Advocacy Summit brought  hundreds of patient advocates, industry experts and policymakers together to engage on important issues facing the patient community.

The summit concluded with a session on patient-focused drug development (PFDD) with distinguished panelists representing diverse perspectives: Paul Hastings, President and CEO of Nkarta Therapeutics; Laurie Burke, MPH, Founder of LORA Group; Cartier Esham, PhD, EVP of Emerging Companies Section and SVP of Science and Regulatory at BIO; Annie Kennedy, SVP of Legislation and Public Policy at Parent Project Muscular Dystrophy; and Pujita Vaidya, MPH, Acting Director of Decision Support and Analysis Team at the Office of Program and Strategic Analysis of the Food and Drug Administration.

In the new PFDD era, patients are helping regulators and developers understand diseases more holistically, so we can expect the drug development process to increasingly focus on aspects that patients value in their treatment.  This new level of involvement provides exciting opportunities for patient and health advocates to have a voice throughout the regulatory development of new treatments.

In recent years, FDA has incorporated PFDD into its processes thanks to regulatory milestones such as the Prescription Drug User Fee Agreement V, the 21^st Century Cures Act and the evolution of criteria for FDA’s clinical outcomes assessments (COA).  These developments have allowed FDA to incorporate patient perspectives into its regulatory approval process by integrating patient input, designing studies with the patient in mind, integrating patient-reported outcomes and better communicating information to patients and providers.

PFDD has led the FDA to progressively use patient data to better understand the disease or condition, conceptualize the clinical benefit and modify the outcomes measure.  Most prominently, this includes real-world evidence (RWE) and real-world data (RWD), both of which harness patient data that supports healthcare decision-making, namely coverage decisions, clinical practice use, and innovative clinical trial designs.

As Dr. Esham described, PFDD has brought about a monumental shift in drug development because, perhaps we didn’t know what mattered most to patients before PFDD came to fruition.  When we listen to the patients, we learn things that can help inform the drug development process.  This feedback loop benefits from involving everyone in the ecosystem, from drug developers to payers to patients.  The next step is to build on these efforts by working together in the patient community to help define what it really means for the patient to have a positive experience.

The panel illustrated many opportunities where the patient community can have an impact on drug development.  Patient groups can meet directly with developers to provide information on their disease or condition.  At FDA, patients can now participate in COA development; inform understanding of a disease or condition; participate in development of best practice standards; and participate in generation of RWE and RWD to support new uses of approved drugs.  There are also open agency forums where the patient experience can be shared.  As Kennedy put it, “FDA has put us smack dab in the middle of [its] work.”

As the two-day summit came to a close, Dr. Julie Gerberding, EVP for Communications, Global Policy and Population Health & Chief Patient Officer at Merck & Co., provided closing remarks to the crowd of patient and health advocates.  Her message was one of empowerment and togetherness.  She implored those present to mobilize their most potent asset: a collection of voices that acts as a powerful source of energy.

Gerberding tasked the community to come together with an articulate message so that advocates can capitalize on opportunities to make the patient experience more prominent throughout the drug development process.  Hopefully the summit empowered attendees with the tools and knowledge to do just that.