African Americans account for 20% of multiple myeloma (MM) diagnoses, which is more than twice the incidence rate among Whites, but make up only 4.5% of participants in US-based clinical trials for new MM therapies.1 To address the underrepresentation of African Americans in these studies, which in turn likely hinders their access to the most advanced treatments, a group of clinicians, scientists, and regulatory and industry stakeholders created a set of recommendations for trial design and later stages of drug development.2

“Clinical trials of novel agents for MM have not enrolled numbers of African American patients to the extent that either the safety or efficacy of novel agents can be adequately assessed [in this racial group],” said Ken Anderson, MD, Kraft Family Professor of Medicine at the Dana-Farber Cancer Institute, who co-chaired the workshop in which the recommendations were developed. He said that this issue motivated the workshop, which was convened by the US Food and Drug Administration (FDA) and the American Association for Cancer Research (AACR).

In addition, Dr Anderson noted, African Americans have not benefited as much as patients of other races have from advances in MM treatments, even though their outcomes appear to be just as good as those of other patients when they are involved in clinical trials.3

Continue Reading

The workshop included clinicians from academic medical centers, leaders from the FDA, patient advocates, and representatives from advocacy groups. Importantly, Dr Anderson said, one of the working groups that vetted and endorsed all the recommendations in the article consisted of representatives from pharmaceutical and biotech companies. The workshop participants made separate recommendations for preapproval clinical trials, which are first-in-man studies of new MM treatments, and for postapproval clinical trials, which may be conducted to further investigate approved medications, such as in certain patient populations or in combination with other therapies.

A key recommendation in the preapproval setting is to broaden eligibility criteria to allow patients with MM and comorbidities such as hypertension and kidney disease to participate in the trial. Dr Anderson noted that African American patients often have comorbidities that exclude them from enrollment. More broadly, he added, there was strong support among all workshop participants for the concept that drug sponsors develop a prospective diversity study plan to ensure that trial enrollment is reflective of the population and, in this case, the racial groups affected by the disease. For phase 2 and 3 clinical trials, the drug sponsor should appoint a diversity officer to help design the trial and create recruitment strategies to achieve the goals in the diversity study plan.

Another barrier to African Americans individuals taking part in MM clinical trials is the fact that trials often do not have sites in predominantly African American neighborhoods, said Dr Anderson, who is also a professor at Harvard Medical School. The recommendations suggest steps to overcome this barrier, particularly in the postapproval setting, such as advising trial stakeholders to engage with patient advocacy groups and form partnerships with social groups such as churches, sororities, and fraternities.

The article includes recommendations for the analysis of existing real-world data to address questions that have not or cannot be explored through clinical trials. Real-world data are a “treasured resource” for understanding the efficacy and side effect profile of drugs after approval, Dr Anderson said, adding there has been great progress in developing structures to collect these data, such as electronic medical records and direct-to-patient processes.