Tackling dismal diversity rates in oncology clinical trials
The modern, evidence-based healthcare system is built on a foundation of research conducted in clinical trials. From new drug approvals to updated care guidelines, stakeholders in healthcare and the life sciences have put their trust in this “gold standard” approach to innovation, particularly in the study of cancer and rare diseases.
But there’s always been an elephant in the room: for years, providers and pharmaceutical companies have been aware that clinical trials are rarely representative of the real-world populations that experience the diseases under scrutiny.
New research from the Yale School of Medicine, published in BMJ Medicine, highlights the true extent of the problem. Between 2012 and 2017, the FDA approved 59 new therapeutics for cancer based on the data from 64 clinical trials. Twenty-five different sponsors submitted the applications for these therapies.
Only 56 percent of the sponsors had adequate representation of women in their trials. Just six adequately represented seniors, and four included appropriate representation of non-white racial and ethnic groups, including Black, Asian, and Hispanic or Latinx populations.
The data tracks with previous insights published by the FDA, which noted that 76 percent of participants in global clinical trials were white, 11 percent were Asian, and only 7 percent were Black, despite the fact that approximately 60 percent of the global population lives in Asia and only around 11 percent of people in the world identify as white.
The severe imbalance has significant implications for the safety and efficacy of approved therapies. Drugs that are not tested on the people actually taking them could work much differently than they do in the highly controlled trial environment, leading to health risks and poor outcomes for patients, not to mention financial impacts for manufacturers and care providers.
The FDA is very much aware of the problem. In recent years, the agency has released several guidance documents to help clinical trial sponsors and community care providers increase representation in the research environment, focusing on collecting better demographic data, designing more inclusive trials, and making participation less burdensome for participants.
More representation starts with better information
Healthcare provider organizations and clinical trial sponsors face many of the same problems in terms of lackluster data on their patients. Demographic information, particularly around race, ethnicity, gender, and sexual orientation, is often incomplete, making it difficult to identify where disparities exist, let alone address them.
In guidance from 2016, the FDA makes specific recommendations around racial and ethnic data collection, including allowing patients to self-report their information, using structured formatting for demographic questions, and employing appropriately specific, standardized racial and ethnic categories. Relying solely on overly broad categories, such as “Asian,” can mask disparities in targeted sub-populations, since the continent includes dozens of countries as different in racial and ethnic makeup as Japan, India, and Russia.
While the guidance is primarily aimed at researchers, provider groups must play a part in collecting complete and accurate demographic data to ensure that all patients, including those from historically underserved groups, have access to relevant clinical trial opportunities. In addition, provider organizations interested in attracting researchers to their care sites will have a better chance at partnering with sponsors if they can accurately describe their unique populations and actively promote participation in underrepresented communities.
Broadening criteria to reflect the real-world clinical landscape
Clinical trials are typically designed to exclude any factors that might interfere with observing the mechanism of the therapy under scrutiny. Patients are often expected to be more or less healthy aside from the disease being observed, which is beneficial from a scientific sense but unrealistic in the actual world of patient care.
In 2020, the FDA released additional guidance urging clinical trial sponsors to broaden eligibility criteria to include people with certain comorbidities or other medications in appropriate phases of the research process.
The agency also encourages providers and researchers to reconsider a range of de facto exclusion criteria that have little basis in hard science, such as declining to include older adults, people at the extremes of the weight spectrum, and people with HIV.
Extending inclusivity to these groups could have significant positive impacts on key populations, including chronic disease patients whose providers are not comfortable prescribing new medications without applicable safety information.
Providing actionable support for participants with socioeconomic needs
Enrolling diverse patients in clinical trials is hard enough, but ensuring that individuals from challenged communities stay in the trial until completion might be even harder.
Trials require significant investment from participants, especially when conducted at centralized academic medical centers that may be difficult to access from rural regions. Expenses for travel and childcare can add up quickly, and many patients cannot afford to take time off work for frequent appointments.
Reducing burdens for participants should be a joint effort between trial sponsors and provider groups, particularly as trials become more decentralized and begin to involve community health providers at a greater rate.
The FDA recommends that sponsors and clinicians ramp up efforts to provide financial and logistical assistance to participants in need. The agency does not consider reasonable reimbursements for travel or other necessary expenses as “undue influence,” so stakeholders have room to support patients without ethical concerns.
Sponsors should also consider leveraging virtual care technologies to reduce the number of in-person interactions and streamline the participation experience. Home monitors, telehealth visits, and smartphone apps can all make it easier for participants to adhere to their treatment regimens without excessive time and expense.
With more robust data, a different look at exclusion criteria, and more comprehensive support for participants with socioeconomic needs, healthcare providers and clinical trial sponsors can start to make headway on improving diversity and representation in crucial medical research.
Jennifer Bresnick is a journalist and freelance content creator with a decade of experience in the health IT industry. Her work has focused on leveraging innovative technology tools to create value, improve health equity, and achieve the promises of the learning health system. She can be reached at firstname.lastname@example.org.