Elevating and Improving Patient-Focused Drug Development


By Jason Harris, NHC Associate Director, Policy and Programs

The driving force behind all of our work at the National Health Council (NHC) is meaningfully engaging patients in every aspect of health care and research. With that in mind, the NHC has actively participated in elevating and improving patient-focused drug development (PFDD). This is an important program for the NHC and our members. To help accomplish PFDD, the FDA will prepare four guidance documents in the next few years.

In September, the NHC submitted comments on the Food and Drug Administration’s (FDA’s) first draft guidance on Patient-Focused Drug Development: Collecting Comprehensive and Representative Input.  Overall, the NHC commended the FDA for its efforts and timely actions to encourage and guide meaningful patient engagement, saying that, “The FDA’s first draft guidance on [PFDD] is an important milestone in this area. … (And) is the first of a series of guidance documents the FDA will release to address the collection and submission of patient experience data. … (T)hese guidances must evolve to accommodate new research, evidence, and learnings in the science of patient engagement. As such, the NHC is committed to working with the FDA to ensure this and future guidances support our shared goal to strengthen patient engagement in the research, development, and regulation of medicines.”

In less than one week, on Oct. 15-16, the FDA is hosting a two-day public workshop to convene a discussion on the next two guidance documents:

  • methodological approaches that may be used to develop and identify what is most important to patients and caregivers with respect to burden of disease, burden of treatment, and the benefits and risks in the management of the patient’s disease; and
  • best practices for selecting, developing, or modifying fit-for-purpose Clinical Outcome Assessments (COAs) to measure the patient experience in clinical trials.

This meeting is a crucial next step in the FDA’s commitment to meaningfully engage patients in drug development, from the pre-clinical stage all the way through post-marketing. The NHC will participate in the October workshop, report back to members on the discussion during the next Medical Innovation Action Team meeting, and convene a multi-stakeholder working group to strategize how the NHC can be most effective in submitting our comments in December.

For more information about our work in patient engagement, click here.