Testimony of Dr. Diana Zuckerman, NCHR, at the FDA MDUFA Meeting

September 30, 2021

Thank you for the opportunity to speak today about the importance of diversity and inclusion. I am Dr. Diana Zuckerman, president of the National Center for Health Research, which is a public health think tank.

As you all know, only the highest risk medical devices are required to submit clinical trial data on safety and effectiveness to the FDA.  De Novo and PMA usually require safety and effectiveness data, but diversity is often lacking.

Appropriations and device user fees have been inadequate to support excellent pre-market review or post-market surveillance.  Subgroup analyses is often lacking or poorly conducted, even when diversity is adequate

We recently published a study in a major journal that examined demographic profiles and subgroup analysis in 22 high-risk medical devices reviewed by the FDA Advisory Panels during 4 recent years. I will describe just a few of the findings:

  • The number of nonwhite patients in pivotal trials ranged from 4 (3%) to 6,788 (17%). Most were on the smaller range, with very few over 100.
  • Of 19 treatment devices, 7 had subgroup analysis for race for effectiveness and only 3 for safety.

Because the number of non-white patients was usually small, even when subgroup analyses were conducted and demographic differences were found, the conclusions were often discredited by FDA, blamed on chance differences due to small sample size.

And, even when subgroup analyses were conducted with significant differences found, the results were often not included on the labeling.

In the last year, our staff have spoken during the public comment period of numerous FDA Advisory panel meetings, basing our remarks on the data provided to the FDA about specific devices or types of devices.  Our findings were similar.

At a Medical Device Panel meeting about the classification of 4 devices in September 2020, we concluded “There is no information about whether patients of color were included, patients over 65, or other vulnerable groups.”

At an April 2021 panel meeting about the OCS Heart System device for preserving hearts for transplantation, we reported that “Around 80% of the donor recipients in all 3 studies were male.” Since the studies were relatively small, the 20% who were women is not large enough to conclude “whether the results would be better or worse among women.”

I want to emphasize that as a patient, I don’t really care if a device is better for men than women, or better for certain demographic groups than for others.  What I care about, and what most patients care about, is whether the benefits outweigh the risks for patients like me, and whether that balance of benefit to risks is better or worse than other alternative treatments.  That’s why subgroup analyses should look at specific demographic groups, and determine if the benefits outweigh the risks for that group.

The important question today is how can MDUFA improve diversity and inclusion?

  • User fees should provide support for FDA staff to work with sponsors to require diversity AND subgroup analyses and include subgroup results on labels and widely available.
  • User fees should provide support for FDA staff to work with patients to create Patient Booklets, Informed Consent Checklists, etc. that include information about which types of patients were studied and how results varied.
  • Most important, there should be a cost to companies who do not include a diversity of patients in their studies. FDA should ensure that approved indications reflect only the types of patients that were actually studied, not “all adults of all ages.”
  • User fees should help support comprehensive post-market databases and surveillance that highlight information from subgroup analyses
  • User fees should help support FDA “Dear Doctor” letters, warnings to patients about lack of data, and other crucial information that should be provided directly to patients and their physicians.