Clinical trials are due for a big change up as the US Food and Drug Administration (FDA) looks to alter a major part of the testing process. In June 2019, FDA created a draft guidance with the goal of diversifying clinical trial populations. In the guidance, FDA encourages drug makers to use a diverse group of participants that more accurately portray the populations that will ultimately utilize the drug in the real world.
RAPS reports that FDA’s new draft guidance “recommends approaches that sponsors of clinical trials to support a new drug application [NDA] or a biologics license application [BLA] can take to broaden eligibility criteria, when scientifically and clinically appropriate, and increase enrollment of underrepresented populations in their clinical trials.”¹ Underrepresented or underserved populations include women, the elderly, and minorities.
Recommendations for inclusion and exclusion criteria
Inclusion and exclusion criteria are important for patient protection and improving data interpretability. However, FDA states that some patients are “excluded from trials without strong clinical or scientific justification.”¹ FDA is concerned that removing patients like pregnant women, the elderly, children, or patients with comorbidities or who are taking other medicine, without justification, could result in pharmaceutical companies overlooking important safety issues and results that do not reflect the general population.
The guidance document itself consists of 18 pages of recommendations on how to correct the issues surrounding undiversified trials. In order to try and maximize potential participants, FDA wants pharma companies to look over the criteria being used to eliminate certain people and change or eliminate such criteria if they are not required for the success of the trial or the safety of the subject. The document also discusses the topic of expanded access to increase patient access to investigational drugs.
Recommendations for rare disease trials
When it comes to rare diseases, FDA states that “because rare diseases often affect small, geographically dispersed patient populations with disease-related travel limitations, special efforts may be necessary to enroll and retain these participants to ensure that a broad spectrum of the patient population is represented,”¹ They go on to suggest working with patient advocate groups on the process of the trials in order to ensure the trials are not discouraging subjects from participating.
FDA describes several additional approaches to increasing patient diversity within the guidance, including expanded access, re-enrolling patients from earlier studies in later phases, and utilizing an open-label study after early-phase studies. If your team is in the process of planning a clinical trial for your drug, device, or diagnostic, Pearl Pathways can help you through every step of the process, from study design to enrolling the last patient and closing out the study. To learn more, read about our CRO services.