Ask the Expert: Dr. Rachel Witten

What recent developments at the FDA do you believe will most impact the gene therapy industry?

The FDA’s Office of Therapeutic Products (OTP) is making strategic moves in the gene therapy industry to keep pace with advancements, support innovation, and streamline regulatory processes. With nearly 1000 clinical trials in North America for cell and gene therapies (CGT), the FDA expects to approve 10 to 20 CGT products annually by 2025. At RMC, we are experiencing more open and flexible interactions with OTP, supporting these strategic objectives. Other notable OTP initiatives include CoGenT, an effort to heighten collaboration and harmonization with global regulatory partners for concurrent reviews of gene therapy applications, inspired by the OCE’s Project Orbis, and START, aimed at accelerating the development of rare disease therapeutics by offering communication support to address clinical development issues early on.

What is one piece of advice you would give to a sponsor manufacturing a gene therapy at an early stage (Pre-IND) of development?

Engaging openly and early with the FDA (via INTERACT or pre-IND meetings) can offer valuable guidance and feedback regarding manufacturing and testing plans. These interactions can help clarify regulatory expectations and provide insights into the most critical aspects of the product’s development that need attention. I recommend that sponsors thoroughly justify their plans, including the various elements of their clinical design and development, to facilitate smoother reviews and expedite clinical trials and eventual marketing authorization.

In your opinion, what challenges do sponsors currently face, and how do you foresee this evolving based on your experience?

Sponsors of clinical trials for rare diseases face numerous challenges that can impede the development and commercialization of new treatments. Regulatory pathways are often less straightforward due to the unique nature of each condition and the limited patient populations available. The rarity of these diseases means that finding enough participants for clinical trials is difficult, often necessitating the expansion of trials across broader geographical areas, leading to prolonged study timelines and increased costs. Additionally, securing funding for research and development is particularly challenging for rare disease treatments due to the lower potential return on investment compared to treatments for more common conditions, making it difficult to attract traditional investment sources. These challenges will likely evolve as the regulatory environment adapts to new and emerging technologies, which we are starting to see.

What are you most excited about in your role at RMC?

Working as a clinical consultant at RMC places me at the forefront of new medical therapies, giving me the opportunity to see innovative treatments and drugs come to life. This role is incredibly rewarding, especially when the clinical trials I consult on could lead to groundbreaking treatments for rare or hard-to-treat diseases. Clinical trials present numerous challenges—from design issues to regulatory compliance and patient recruitment to data management—and I am tasked with navigating these complexities through innovative thinking and strategic planning. Ultimately, the most fulfilling aspect of my job is the knowledge that my work contributes directly to improving patient outcomes and advancing public health. Each clinical trial I help design and execute has the potential to develop new therapies that not only improve but also save lives.