CBER Wants to Ensure Patient Voice Remains Central to Gene Therapy Development

The FDA’s Center for Biologics Evaluation and Research (CBER) hosted a public patient listening meeting in September on Patient and Care Partner Perspectives on Safety Considerations for Approved Gene Therapy Treatments for Rare Diseases. During this meeting there were over 35 patient and advocate representatives who had four minutes each to share their experiences, questions, concerns, and hopes for these transformative treatments. This event underscored CBER’s commitment to ensuring the patient voice remains central to the development of gene therapies. 

Key themes that emerged from the discussion offer a glimpse into the complexities of living with a rare disease and the unique factors influencing treatment decision-making. 

Risk, Safety, and Uncertainty 

• Expressed concerns about immune responses and side effects, particularly on unknown long-term impacts. The need for clear communication about these risks was repeatedly stressed.   

• Different types of gene therapy (e.g., gene editing vs. gene silencing) come with distinct considerations, and patients are often torn between pursuing early treatment versus waiting for additional safety data.  

• Need to prioritize setting realistic expectations, understanding that while gene therapies might slow or stop disease progression, the potential to be curative is still unknown. 

Informed Decision Making, Transparency, and Support 

• Emphasized the need for centralized, easy-to-understand information on therapy risks, benefits, side effects, and quality-of-life impacts from reliable sources like the FDA.  

• With more than one treatment often available, patients seek comparative information to make well-rounded choices, including details on trial designs, long-term follow-up protocols, and potential impacts on future treatment eligibility.  

• There is a need for educational resources, mental health support, and peer networks to help with decision-making and coping with the emotional toll of gene therapy. 

Quality of Life and Outcomes 

• For many, quality of life takes priority over length of life, and highlighted the importance on therapies that enhance daily functioning, reduce treatment burden, and address symptom specific needs.  

• Additionally, some patients may need more than one type of therapy, thus a call for flexible treatment protocols that allow combination therapies and additional options if needed. 

• The CART-recipient community is pushing to move CAR T therapies to the first line of treatments (most are being used after many other treatments fail) and to reconsider the black box label. Both have become barriers to patients achieving better outcomes in a timely manner. 

Practical, Financial, and Logistical Barriers 

• High costs and challenges with insurance coverage create significant barriers, affecting the accessibility and feasibility of gene therapies for many families. There were many logistical hurdles brought up such as treatment frequency, travel for follow-up care, and the complexity of therapy administration. 

• Patient advocacy groups stressed the need for remote participation and reimbursement of travel costs to be able to maintain long term follow up. 

Inclusivity and Equity in Clinical Trials 

• It is important to have inclusive trial designs that account for demographic diversity and genetic variations, to better ensure that therapies are safe and effective across populations. Decentralizing trials could lessen the travel burden, make participation easier for patients, and promote equitable access to gene therapies. 

Patients, caregivers, and advocates bring essential insights to deepen the understanding of the treatment journey and ways the drug development process can be improved to best serve them. CBER’s ongoing promise to be open, inclusive, and patient-centered shows hope to reshape the future of gene therapy, making it more accessible, understandable, and supportive for those affected by rare diseases. We hope through these increased collaborations and discussions, steps will be taken towards prioritizing what is most important to patients and families.   

Join FDA CBER for session 2 of this series focused on “Patient and Care Perspectives on Early Enrollment into Gene Therapy clinical Trials for Rare Diseases” on December 4.  

For more timely education and engaging discussions on cell and gene therapies, register for ASGCTs upcoming summit, Empowering Patients 2025 and subscribe to the bi-monthly newsletter, The Patient Press.

Allison Bradbury, PhD, Lauren Beretich, PhD, MS, CGC, and Yulia Grishchuk, PhD are members of ASGCT Patient Outreach Committee.