By Kirsten Axelsen

I recently moderated a discussion among people who have
spent their careers working in biotech where we discussed methods to achieve
more accurate representation of all racial and ethnic groups in pivotal
clinical trials for new drugs in the United States. Despite decades of federal
guidance, investment, commitment, and effort, most drugs that are approved in
the US do not have Blacks or Hispanics enrolled in their pivotal clinical
studies in the same proportion as they are represented in the population of the
US or in the same proportion as those that suffer from the disease in question.
We discussed exemplars of the way forward and opportunities to champion health
equity through equitable clinical trials.

The panel hosted by Academy Health as a part of their
Datapalooza conference consisted of Amrit Ray, Van Crocker, Sarah Thompson, and
Dave Bernstein. Collectively there are roughly 100 years of experience working
in biotech, clinical trial management, real world evidence, regulation, and
research grants between us. We considered the fundamental issues related to
clinical trial design, including ensuring accurate inclusion as a priority
among sponsors, developing inclusion and exclusion criteria that increase the
accuracy of representation, and ensuring that the biases of the people
enrolling participants in clinical trials are well understood and managed. Additionally,
we discussed how to mitigate the assumption that people are often not asked to
join a trial because there is a belief that they can’t participate or can’t
afford to participate through data-driven decisions.

When we all started working in the biotech industry 15 or 20
years ago, the science, technology, and regulation hadn’t evolved to where it
is today, limiting potential paths forward. We discussed multiple practices and/or
policy solutions using contemporary resources to rectify the inequitable
representation in clinical trials. One was deploying the wealth of electronic
health data collected when dispensing medicines in pharmacies, processing
insurance claims, or managing treatment in the physician’s office or hospital
to better recruit for clinical trials. While there are established techniques
to use this information without compromising privacy, companies that own the
data are often uncomfortable with the premise of using it for trial
recruitment.

Another relatively new option is expanding the use of remote-enabled
trials as a result of the COVID-19 pandemic. While there were home devices
pre-pandemic, we could not have envisioned that a person could do their own
nasal swab at home with a videoconference. The pandemic enabled a change in
trial protocols that can endure and allows a wider range of people to participate.

Then we considered that regulatory incentives that have been
offered in pediatric studies, rare diseases, or conditions of high unmet need
could also be considered and deployed to increase diversity in clinical study.
There is now sufficient evidence to show that they work and have significantly
changed the focus of clinical study.

A clear thread ran among all the themes that evidence and
epidemiology should drive decision-making, not centuries of bias and socioeconomic
and educational inequities that too often have a role in who gets included and
excluded in clinical study. A lot is being done in both the for-profit and
non-profit sectors, including grantmaking and investment in new partnerships
and methods of patient engagement. But to get evidence and data to drive the
decision-making, it appears that guidance and goals alone are not making a
large enough impact.

Doing things differently—whether it means relaxing exclusion
criteria, using electronic health records or other emerging data sources to
recruit, or investing in different methods and community settings for
recruitment—has associated costs and risks. Typically, when society wants an
organization to change behavior, some sort of incentive or protection is offered
through regulation, tax incentives, or other means.

Policymakers should look to the tools we have highlighted to encourage more representative and therefore improved science and clinical study. It is crucial to consider how to deploy these resources to remedy this persistent issue in drug development. They should aim to assist all stakeholders (sponsors, clinicians, and others) in understanding the risks and opportunities of more active innovation so that the drugs that are being approved are studied in the populations that will be taking them.

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