Recommendations designed to address the underrepresentation of Black patients in clinical trials for multiple myeloma were recently released. Details about the initiative, published by Gormley et al in Blood Cancer Discovery, form a road map for designing multiple myeloma clinical trials to eliminate racial bias by including more Black patients, as well as for gathering real-world data from health records about the effects of drugs in this population.
Through a joint workshop initiated by the U.S. Food and Drug Administration (FDA) and the American Association for Cancer Research (AACR), a cohort of researchers, physicians, patients, statisticians, representatives from the pharmaceutical industry, and regulators reviewed existing issues with the goal of improving effectiveness of multiple myeloma therapies in Black patients.
Multiple Myeloma in Black Patients
Multiple myeloma is diagnosed in about 30,000 people in the United States annually, resulting in more than 12,500 deaths. Black patients are more than twice as likely as White patients to be diagnosed with multiple myeloma—15.9 vs 7.5 cases per 100,000 population. They are also more likely to die from the disease—5.6 vs 2.4 myeloma deaths per 100,000 for Black vs White patients.
An array of new treatments in recent years have generally improved outcomes for patients with myeloma. However, data from large multiple myeloma clinical trials shows a decrease in enrollment of Black patients by 3.5% over a recent 10-year period. Even more critically, most racial and ethnic minority patients participated in trials that did not involve novel agents.
Kenneth C. Anderson, MD
“There hasn’t been as much progress [for Black patients] as there has been in other groups,” said corresponding study author Kenneth C. Anderson, MD, Program Director of the Lebow Institute for Myeloma Therapeutics and the Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute. “The number of [Black patients] enrolled in clinical trials of novel agents or treatments of multiple myeloma has been tragically low. When they have enrolled, their outcome to treatment with novel therapies has been the same or even better than other patients,” he said.
Black patients comprise 20% of people diagnosed with myeloma, but between 2003 and 2017, only 4.5% of patients in new drug and biologic license applications for myeloma were Black. This disparity has raised concerns that the findings of therapeutic clinical trials may not be entirely valid for Black patients, due to underlying genetic and biologic differences that have been discovered between Black and White patients with multiple myeloma.
Recommendations
The new recommendations emerged from a February 2020 FDA-AACR workshop in Washington, DC. The group recommended a number of changes to the design of clinical trials of drugs for which manufacturers are seeking approval. They included:
- Broadening eligibility criteria whenever possible. For example, study criteria that reject patients with conditions like high blood pressure and kidney disease may disproportionately exclude Blacks. Including such patients may allow researchers to collect more data in racial and ethnic subpopulations.
- Requiring trial sponsors to complete a diversity study plan that sets targets for enrolling diverse participants.
- Appointing a diversity officer to assist with trial design and recruitment. Trial design should encompass disease subtypes and features most commonly seen in Black patients. Patients and patient advocates involved in the workshop strongly supported the recommendation of a diversity officer “to define strategies that support [Black] participation in clinical trials.” The presence of a diversity officer “will hold researchers and industry accountable to conduct more inclusive and patient-centric trials,” the recommendations stressed.
Other recommendations concerned gathering clinical trial data in the postapproval period. Studies conducted after the drug has entered clinical use could identify differences among racial and ethnic subpopulations with regard to safety and efficacy. The recommendations also sought to increase diversity by directing stakeholders to devise strategies to overcome clinical, social, and socioeconomic impediments to trial access.
Dr. Anderson emphasized the importance of patients’ participation in the workshop that formulated the recommendations.
“Our patients are truly the inspiration and heroes of this collaborative effort to eliminate the glaring issue of racial disparities in clinical trials,” he said. “If we can make clinical trials more inclusive and representative of real-world patients, we may not only enhance participation of [Black] patients, but also provide a paradigm for new drug development more broadly.”
Disclosure: For full disclosures of the study authors, visit bloodcancerdiscov.aacrjournals.org.
