Biopharma companies as well as policymakers know the significance of assessing medical and drug products across a broad range of racial and ethnic universes so as to fully comprehend the medical effects on patients. This has indeed pushed the support for studies to make sure that patients enrol in proportion to the burden and prevalence of the disease across relevant subgroups, however, achieving such a patient representation in clinical trials which is equitable has been a challenge.
After decades of FDA rolling out guidance docs and coming up with programs so as to encourage the sponsors to look for a broader representation in clinical trials, the policymakers have gone on to add a stick to the process, which means that research sponsors will not have to take care of this issue by giving Diversity Action Plans- DAPs to the agency in case they are proposing or seeking approval for phase III studies. This provision has been included in the Food and Drug Omnibus Reform Act, which was approved by Congress last December. This was a part of the consolidated appropriations act that made way for federal spending across 2023.
Among the many FDORA provisions which include designing to modernise FDAs accelerated approval program, push FDA inspections and also broaden the oversight of cosmetics, the roll out requires the DAPs. It is well to be noted that the FDA has an entire year to issue any guidance on this program, and there can be some waivers and exceptions as well. Apparently, it has been decided that the FDA is going to hold a public workshop on this programme and also issue some additional guidance on certain topics, as well as submit its annual report to Congress once the programme begins.
The need to put forth DAPs is supported by studies and analyses that show inadequate representation of patients across many clinical trials. A review by one of the leading pharmaceutical drug manufacturers’ last 17 years clinical trials gives out an uneven picture of various minority and ethnic group enrolment and how it has had an impact on the treatments and varied drug areas.
The results of 495 clinical trials that involved US participants were mixed. Apparently, there were more black or African American patients who took part in the HIV drug study as compared to the group’s high prevalence in that disease population. In fact, very few Asian patients became a part of the study on coronary heart disease which was incidentally slightly more than Hispanic. As a matter of fact, all the minority groups were underrepresented in the trials related to vaccines.
An analysis that was done by the Government Accountability Office in December last year concluded that certain population groups were underrepresented across various clinical trials despite years of efforts that were put in to enhance research diversity. This report stressed cancer clinical trials and gave consistently low numbers in terms of the range of patients from varied racial and ethnic groups.
The report also put forth the practices which were developed at cancer centres that further helped in the advancement of diverse enrolment across the research. This included assigning the community ambassadors to enhance the awareness of clinical trials in local areas, improving training and increasing the diversity of the workforce across centres to better analyse and roll out clinical trial opportunities, and providing logistical and financial support to overcome the barriers to participation from patients across research programs.
Last year FDA had issued a draft guidance in terms of coming up with race and ethnicity diversity plans in order to enhance the research enrolment of apt number of participants from underrepresented populations and that the advisory may very well provide the base for moving forward. The guidance also lays out the value of the submission of diversity plans as part of the initial investigation process and subsequently into market applications, noting the challenges as well as successes.
The sponsors have already queried the agency on specifics in order to apply diversity goals across the global development programmes so as to assess appropriate enrolment from underrepresented populations. There is another challenge that states that, if at all, the diversity goals have to apply across each individual study or the entire development program. The sponsors are particularly wanting to know more about the dealings of the FDA with research programmes that do not meet the set patient enrolment goals.
The expert panel has proposed that the FDA give away added incentives for the sponsors so as to meet the diversity goals in terms of fast-track eligibility, tax credits, extended market exclusivity, and a reduction in fees, which is similar to the incentives that are provided to firms involved in manufacturing orphan drugs.
The researchers, besides this, could get more leeway so as to reimburse the minority study participants and also cover expenses when it comes to transportation, lost wages, and dependent care.