Are We Just Checking the Box on Patient Input in Drug Regulatory Submissions?

There is little doubt that FDA and the medical products industry are on the patient-centered bandwagon.  Everyone appears to agree that the patient voice should be heard throughout the medical product life cycle.  I think we would also agree that considering the patient voice in regulatory decision-making is of particular importance, and to a certain extent, the ultimate goal of most pre-market, patient-centered activities. Given its been several years since FDA was mandated to listen to patients, there seems to be little fanfare about the actual use of patient input data in regulatory submissions or their review.  A recent article published in Therapeutic Innovation & Regulatory Science took the time to lift the lid on what is actually happening with drug submissions that use patient input data, what that data actually is, and whether or not it’s being used. Spoiler alert:  the results are rather disappointing.

A small provision of Section 3001 in the Cures Act requires FDA to provide a brief, public summary of any patient experience data considered in the approval of a drug application. These “public summaries” take the form of a table that is included in the review summary for new drugs once they are approved. The table does not provide a lot of details, but it does show us when and how we’ve, literally, checked the box on using patient experience data in a drug submission.  The results of the authors’ review of these summaries provide empirical evidence for what most of us already suspected.  Most of the “patient experience data” being considered – if any is considered at all – is in the form of patient reported outcomes (PROs) with very few instances of using qualitative experience data, patient preference information, or even Voice of the Patient PFDD meeting reports when they were available. 

The reality is that there is still a lot of talk, but not a lot of commitment from industry or the FDA to tap into the insights of patients when it comes to regulatory review of new drugs. The use of PROs is becoming routine, but I would love to know how many of them actually reflect the issues that are important to patients.  If patients didn’t have input into the development of the PRO, then we are simply having patients tell us about what we think is important to them. While PROs may have the word “patient” in them, they do not provide patient insights, or details about patients’ experience living with a disease. Unfortunately, using PRO data to support a drug regulatory submission allows FDA and the sponsor to check the patient experience box.

I agree with the authors that we need more transparency about how patient experience data is being used and considered in regulatory reviews.  That does not mean we need FDA or sponsors to disclose proprietary data.  That does mean that we need more than a table buried in a summary that doesn’t tell us much of anything about how effective patient input is in the regulatory decision-making process.  As suggested by the authors of the paper, a simple, detailed, and accessible summary about how patient experience data was used in an approval would go a long way.  Knowing how FDA is making use of these data in review deliberations would encourage more sponsors to include the patient voice in their pre-market submissions. Until we can actually implement the patient-focused regulations in a truly patient-centered and meaningful way, we are simply checking the box.

Kieffer, CM. et al. (2020) FDA Reported Use of Patient Experience Data in 2018 Drug Approvals. TIRS.