Lessons Learned from Developing a PRO Measure: The Experience of One Patient Advocacy Group

By Silke Schoch, Senior Associate, Programs

On May 14, 2019, Dory Kranz, CEO of the National Alopecia Areata Foundation (NAAF) and National Health Council Board member, described her organization’s experience developing a patient-reported outcome (PRO) measure. The webinar was the seventh in the NHC’s Clinical Outcome Assessment (COA) series.

Dory described how existing alopecia areata patient-specific PRO instruments are not rigorous enough to meet FDA Guidance on PRO measures (PROM), and generic instruments are insufficient to support product labeling claims. A patient-centered, rigorous, and validated measure would ensure harmonization of outcomes used in upcoming clinical trials, which in turn will facilitate more efficient comparative effectiveness research in the future.

Despite the substantial time and cost of developing a PROM, NAAF determined that they should develop and validate a PROM for the alopecia areata community. The NAAF PROM is still under development, but Dory shared a number of lessons learned to date based on their experiences:

Developing a PRO takes significant time and resources:

Set yourself up to do it right or don’t bother.

Two essentials to be successful include:

  • Board of Directors buy-in at beginning of the project; and
  • Contract a clinical outcome assessment (COA) expert-consultant partner that is trusted by FDA and industry.

Leverage opportunities to meet with FDA staff:

  • Build relationships; and
  • Educate on the disease area.

Leverage existing expertise:

  • Professional societies or consortia, in their case, the International Dermatology Outcome Measures;
  • The Critical Path Institute (C-PATH); and
  • The National Health Council.

Everything takes longer than you can imagine!

Continue good science in the face of bureaucratic delays from all sides.

Watch the webinar here.