A CDER conversation with John J. Whyte, M.D., MPH, Director, Professional Affairs and Stakeholder Engagement, Center for Drug Evaluation and Research, FDA
The FDA relies upon mandated, industry-sponsored clinical trials to make decisions about medical products. In years past, questions about adequate and equal inclusion of women and people in racial minority groups have been raised. Many stakeholder groups want FDA to require drug companies to include certain percentages of demographic subgroups in clinical trials and analyze subgroup data by sex, race and age before a drug is approved. While the FDA has many regulations and policies in place regarding clinical trials, these studies are the responsibility of the manufacturer that is developing the drug.
To address stakeholder concerns, FDA released an “Action Plan to Enhance the Collection and Availability of Demographic Subgroup Data.” One priority stated in the report is to make demographic data from clinical trials more available and transparent. To reach this goal, FDA’s Center for Drug Evaluation and Research is releasing drug trials snapshots that outline the participation of people in clinical trials by sex, race and age. The drug trial snapshots highlight information in an easy-to-read format.
Tell me more about the drug trials snapshots, how will they help consumers?
The information in a drug trials snapshot is taken from the data a manufacturer submits in a new drug application or a therapeutic biologic application. The snapshot includes information on who participated in the study, how the study was designed, the results of the efficacy and safety studies and the differences in side effects and in benefits among sex, race and age groups.
Even though this information has been publically-available in various formats, it has never been gathered in one centralized location. People can actually go to a snapshot on our web site and see the number and types of men and women who have been involved in clinical trials. They can see how many African-Americans, Hispanics or Asians were involved, how many people participated above or below a certain age and other demographic data.
In short, these snapshots allow consumers to actually see that people like them participated in clinical trials for a certain therapy. One wasn't able to easily access this data before. The snapshots are a game changer.
How will a patient or a consumer take this information and translate it to their healthcare decisions?
First, let me say that consumers should look at this information with caution -- this is not meant to replace the drug label, and it's not intended to replace a conversation with their doctor. As the name indicates, this is a snapshot that shows who was involved in the clinical trials. Clinical trial information is only one piece of data used to assess the safety and efficacy of a drug.
So a consumer wouldn’t use this information alone to choose a drug?
No. The snapshots provide information about the participants in the clinical trials conducted for the drug – they are not a comprehensive medical guide. But they do present a great opportunity for consumers to look at the information and use it to talk with to their doctor about the risks and benefits of the drug for them.
Beyond the snapshots will other information be available?
Within the snapshots there will be additional selected links but not too many because we don’t want to overload people with information. Of course, more detailed information about any drug can always be found on our website at Drugs@FDA.
Why is FDA doing this now?
It is a response to two things: the Food and Drug Administration Safety and Innovation Act and the Agency’s overall desire to be transparent.
The Act directed us to issue a report that addresses clinical trial participation and the inclusion of safety and effectiveness data by demographic subgroups. In developing this report, we were able to identify gaps in trial participation and then discuss these gaps with our external stakeholders and find strategies to overcome persistent challenges in enrolling diverse study populations. One gap was the absence of easily accessible information about the demographic profiles of participants in clinical trials. The drug trials snapshots help to address that information gap for consumers and advocacy groups.
But, at its heart, this is an effort to be transparent – to provide information to the public, and actually show the number and participation of men and women, of various races and age groups within the clinical trials. Being able to share more information and facts will help us to facilitate a thorough and robust discussion about clinical trial demographics. Now, anyone can go to the site and see the numbers for themselves in a quick snapshot.
How often will these snapshots be posted?
In our first iteration, we will post drug trials snapshots for six indications of five new molecular entities approved in May and June of 2014. We're very interested in hearing from consumers, health care providers, patient groups, researchers, and the pharmaceutical industry as to whether the way we present this data is helpful to them. This feedback will be used to inform the next generation of snapshots so that we meet our stakeholders’ needs. Starting January 2015, our goal is to provide a snapshot at the time of every new drug approval.
Do you think you'll hear from patients?
I do. I think we'll hear from consumers as well as their advocacy groups. Advocacy groups in particular have really been asking for this data. They've been working to increase the numbers of women and minorities represented in clinical trials and they want to see the information. So we are providing greater transparency through the drug snapshot.
You mentioned women and minority participation in clinical trials. Why is finding diverse participants for clinical trials so difficult?
Participation in clinical trials is a very complex issue. Part of the problem is probably a reflection on medical care in general – there can be quite a disparity, especially when you look at different races and ethnic groups. So if you're part of a group where just getting access to medical care is a problem, odds are that you’re not going to have the opportunity to even be asked to participate in a clinical trial. Studies show that when minorities and non‑minority members are asked to participate in a clinical trial, the participation rate is roughly equivalent.
We have long recognized that there is underrepresentation of women and other minority groups in clinical trials. One way we're trying to respond to that is by working with our federal partners like the Centers for Medicare and Medicaid Services, and working with physician groups, healthcare systems, and with advocacy groups to raise awareness of the need for widespread participation in clinical trials.
What role does FDA play in clinical trials?
Sometimes there's confusion about clinical trials and FDA’s role. Our regulatory standard is whether a drug is safe and effective. Now having said that, we make drug approval decisions based on clinical trial data – data provided by the drug company. There's been the perception that FDA actually executes clinical trials, we don't -- that's an important distinction.
You’re rolling out the first of these snapshots soon. Looking forward, what do you hope to see from this effort down the road?
Earlier I called this a game changer. I say this because the data will be out there for everyone to review and discuss. In the past there were perceptions about participation rates, but very few people actually knew and understood the data -- now everyone will be able to explore this information. Now we can have that robust statistical, yet clinically-meaningful discussion about whether it matters how many men and women we have in a trial.
Folks will look at the data and, in some cases, see 30 percent women or only five percent African-Americans, and that data, in and of itself, will be factual. Statistically, it is possible to determine that a medical product is safe and effective for both men and women in a study with fewer patients in one or another category. But the questions are: what should those numbers be; what type of analysis should be done and when should it be done; is this about drug development or is it about analyzing trial participation in the post-market setting?
These snapshots, I think, will get everyone on the same page. Then hopefully FDA, along with the collaboration of our Federal partners, industry, health care professionals, and patient advocacy groups, can chart a path forward that will really address the issue of sex and race differences in drug development and drug treatment.
The FDA encourages review and feedback on the Drug Trials Snapshots. The FDA will incorporate the feedback into the next iteration of the Snapshots.
- Drug Trials Snapshot
- FR Notice: New Clinical Trials Demographic Data
- FDASIA Section 907: Inclusion of Demographic Subgroups in Clinical Trials
- Drugs@FDA Database
- FDA Basics Webinar - April 27, 2015: Drug Trials Snapshots