Diversity and Inclusion in Clinical Trials for Rare Diseases: Together We Can Achieve the Unachievable

Session Description

On April 13, FDA Commissioner Robert Califf, M.D., announced in a news release. “Going forward, achieving greater diversity will be a key focus throughout the FDA to facilitate the development of better treatments and better ways to manage diseases that often disproportionately impact underserved and marginalized communities.” The agency issued a new guidance recommending applicants to develop and submit a “Race and Ethnicity Diversity Plan” of clinical trials-participants (e.g., diverse race and ethnicity and other underrepresented populations defined by demographics such as sex, gender identity, age, socioeconomic status, disability, pregnancy and lactation status, and co-morbidity).

While the benefit of this initiative is obvious: it helps provide evidence that the study drug will be safe and effective in the wider target patient pool, the situation for rare diseases is rather absurd. Because of the small sample size of patients with specific rare disease, more inclusive study designs should, at least theoretically speaking, results in achieving enrollment success. However, logistically it can be very hard to find and connect these disparate minorities patients with investigators participating in a clinical trial. These minority populations are often underrepresented in genome wide associate studies. Since 80% of rare disease are genetic in nature, these patients are not even diagnosed. Even if diagnosis is made, several significant barriers (e.g., geographical, financial, study design and infrastructure) to participate in clinical trials are often prohibitive resulting in health disparities among underrepresented populations.

This session aims at raising the awareness to utilize clinical pharmacology concepts and tools to address design barriers and enable greater inclusion and retention of minorities with rare diseases in clinical trials. Quantitative and evidence-based approaches to help broadening the inclusion criteria and labeled indications are highlighted. We aspire those minorities with rare disease across the globe will be able to get equal access to treatment.

Learning Objectives

1. Understand from patients and patient advocate perspectives, the challenges that minority patients with rare diseases face.

2. Explain the regulatory considerations and industry perspectives as well as the role of clinical pharmacologist in designing studies and analyzing the data to facilitate equal access to therapies.

Session Schedule

9:30 AM - 9:38 AM ET
Introduction
Mariam Ahmed, PhD and Noha Rayad, PhD

9:38 AM - 9:45 AM ET
Patient's Voice
Michelle Lorenz

9:45 AM - 9:55 AM ET
Innovative Approaches to Reach Out to Minorities With Rare Diseases
Clare Grace, PhD

9:55 AM - 10:05 AM ET
Increasing Diversity in Clinical Research: Steps Towards Precision Health and Reducing Health Disparities
Youssef M. Roman, PharmD, PhD

10:05 AM - 10:15 AM ET
Regulatory Perspectives on Diversity and Inclusion in Clinical Trials
Michelle Campbell, PhD

10:15 AM - 10:30 AM ET
Q&A and Closing Remarks