Diversity, Equity, and Inclusion Within Gene and Cell Therapy: Where Does It Stand?

There is no shortage of data and research that shows that diversity improves innovation, collaboration, and outcomes. However, it’s no secret that the future of funding and organizational support of diversity, equity, and inclusion (DEI) programs is in jeopardy. Entire states have outlawed funding for DEI programs and their overseeing educational institutions. DEI was once an urgent topic of conversation and policy changes from boards down to workers on the ground. Today, many are wondering if DEI programming should still stay within structures and policy. The DEI movement may seem to be losing traction, but its necessity across all functions of society remains strong.

Within drug development, the DEI conversation is being had and is here to stay. In recent years, FDA has published several guidance documents to enhance collection of patient demographics and improve representation in clinical trials. Patient characteristics such as race, ethnicity, sex, and age can influence the response to a therapy. Historically, clinical trials have enrolled participants who were not representative of the larger patient population, which risks missing specific patient population responses.

So how does this apply to cell and gene therapies (CGT) where often rare indications are targeted, but the size of the patient population is small? While the framework provided by FDA is not specifically written just for CGT, the principles are still applicable, and the FDA framework is one that will benefit patients and the disease community most affected by the trials.

The FDA framework has several takeaways, including:

• Assessment of exclusion criteria and whether the eligibility criteria are representative of the population for which the drug is being developed.

• Reduction of clinical trial burden to participants by offering flexibilities in visit times, location of visit, digital health services where applicable, and information on possible reimbursement opportunities for trial participants.

• Best practices for enrollment, engagement, cultural competency, geography, and trial efficiency.

• Early engagement of patient advocacy groups, key opinion leaders, and patients in rare disease.

So how does one interested in starting a clinical trial begin to strategize the DEI portion of the design? It is important to focus on access, stakeholder inclusion, diverse patient population participants, and better plain language of the protocols. The focus on DEI must be innovative and it is critical that all stakeholders are a part of the clinical trial plan.

The discussion on DEI is not limited to clinical trial design. Scientists and developers should consider diversity when developing new therapies, especially in CGT, where products are often developed to treat the underlying genetic cause of a disease. For example, gene therapies utilizing gene editing should assess off-target effects in the context of genetic heterogeneity within the intended population, as further discussed in this FDA workshop. ASGCT's dedication to advancing diversity, equity, and inclusion in gene and cell therapy is unwavering.

As we continue to foster an environment of learning and support, we hope you've been attending our Annual Meeting this week in Baltimore or virtually. The DEI Committee is proud to present a symposium titled "How to Develop Gene and Cell Therapies with a DEI Forward Approach," featuring giants in the field at the forefront of this intersection. Don't miss this chance to gain invaluable insights and resources. Register now to be part of this pivotal discussion and contribute to the future of gene and cell therapy with diversity, equity, and inclusion at its core.

Dr. Fuentes is a member of ASGCT's DEI Committee.

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