Patient engagement is important in all drug development, but it becomes especially critical and fundamentally different for gene therapies (GTx). Unlike many conventional treatments, gene therapies often involve a one-time, permanent intervention, limited patient populations, high uncertainty, long-term follow-up, and unique ethical and practical considerations.

Fueled by a global biopharmaceutical client’s desire to better understand what makes patient engagement in GTx distinct, VOZ conducted research and interviews with patient organizations and patient engagement leaders in biopharma companies with GTx drugs in development to inform the company’s overarching gene therapy patient engagement strategy.

Key learnings include:

Many gene therapies target rare genetic disorders, where small patient populations and limited natural history data make patient community input especially critical. Unlike more prevalent conditions, where researchers can rely on more extensive epidemiologic and clinical data, rare disease research depends heavily on input from patients and care partners. As experts in their lived experience, they can help define disease burden, identify meaningful outcomes, and inform patient-centric trial design.

Benefit-risk decisions are fundamentally different for GTx than for traditional drugs. GTx are often administered as a one-time intervention, designed to provide long-term or “curative” effects, but cannot be stopped or adjusted over time. As a result, understanding patient perspectives on acceptable risk is especially important.

Patient follow-up requirements are often more extensive, requiring long-term commitment from patients and caregivers and creating a unique, ongoing relationship with the care team. Ensuring they understand this level of involvement before pursuing gene therapies is essential for long-term success.

Caregivers play an especially important role, as GTx frequently target pediatric conditions, neurodevelopmental disorders, and severe disabilities. Incorporating caregiver perspectives in development decision-making can meaningfully shape outcomes in many conditions targeted by GTx.

Ethical considerations are more complex, given the uncertainty around long-term effects, the scientific complexity that can complicate informed consent, the potential impact on future generations, expectations of a “cure,” equity of access, and more. These considerations make patient and caregiver engagement especially important in GTx development, as patients and families help define ethical boundaries for innovation and educational needs.

Patient organizations are research partners helping to close critical gaps in science and knowledge by funding natural history studies, creating disease registries, identifying patients for trials, and educating patients about the many complexities of gene and cell therapy development.  Engaging patient organizations as strategic partners can be of significant mutual benefit, yet it often requires funding, capacity, and ways of working that may be new for some companies.

At VOZ, we have a keen understanding of the power of early and sustained patient engagement that allows for the alignment of GTx innovation with patient needs. Given the unique scientific, ethical, and practical considerations of GTx, this understanding is essential to ensure that the transformative promise of these therapies translates into outcomes that matter most to patients and families.

– Sharon Dion, Senior Vice President, Client Services at VOZ Advisors