Recently, the FDA issued Collecting Comprehensive and Representative Input, the first of four patient-focused drug development (PFDD) guidance documents. As noted in a statement from FDA Commissioner Scott Gottlieb, M.D., “Our work demands that we must continue to reflect on how we can make the science of drug development and review more modern and more patient-centered, so that approved products impact the metrics that real-world patients and families value most.”

The guidance provides some important considerations and guardrails for ensuring patient experience data provides meaningful value in product development and regulatory decision-making. It covers:

  • General considerations for collecting patient experience data and sampling types (e.g., determining the study objective, target population and approach)
  • Methods for collecting and analyzing patient experience data – including a review of qualitative, quantitative and mixed-methods research
  • Data collection/management operationalization and standardization (e.g., identifying patients/sites, adhering to relevant laws/policies and FDA-supported data and monitoring guidance)

The key takeaway is that while patient experience data can take many forms, it must be comprehensive, representative and methodologically sound – with the same level of rigor as any other data submitted to the Agency. Exactly what this looks like is going to depend on the goal of the research, the disease state and what matters most to patients. The FDA is committed to working with patient stakeholders and industry to make this a reality and made it crystal clear that sponsors should involve them early and secure feedback on research design and regulatory requirements.

The remaining draft guidance documents to be issued the next three years in a stepwise fashion will take a deeper dive into other aspects of PFDD, including more guidance on methods for eliciting patient feedback and the creation of clinical outcome assessments. These documents are part of the FDA’s PFDD efforts in accordance with the 21st Century Cures Act and The Food and Drug Administration Reauthorization Act of 2017 Title I.

This guidance represents the FDA’s response to rising patient expectations and a commitment to validate the perspectives of those who need new medicines most. Now it’s on industry, patient advocacy organizations and researchers to heed the call.

About the Author:

Stephanie Budnick has more than 12 years of experience in healthcare communications. She has supported numerous U.S. and global clients across a number of therapeutic areas. Her work has included the creation of strategic partnerships, data/regulatory communications, crisis and issues management, social/digital media engagement, national public awareness campaigns and advocacy programs. This includes award-winning unbranded public awareness campaigns for alcoholism/addiction, Alzheimer’s disease, diabetes and mental health. Stephanie graduated with a B.S. in Biology from the University of Virginia.

Keri McDonough is a communications and advocacy relations strategist with experience in a range of therapeutic areas, including multiple sclerosis, mental health, vaccines and oncology. She thrives on problem-solving, providing counsel on complex, ever-changing marketplace dynamics and connecting stakeholders to achieve shared goals. Partnering with clients and her teams, she develops and delivers compelling purpose-driven and award-winning programming. Over her 20-year career, Keri has cultivated an expertise in patient communications and advocacy relations with an emphasis on the power of patient perspectives to educate, motivate and activate audiences.