While many gene therapy clinical trials appear to be on the cusp of generating some game-changing healthcare innovations, patients are on a steep learning curve navigating the new landscape, says Dr. Meagan Vaughn, PhD, RAC, a research scientist with Rho. “The pace of development is exponential right now in terms of how fast things are moving, and how many different companies are developing these products,” she notes.
It’s a brave new world where patients will be under pressure to learn more and face the consequences of participating in clinical trials with a much longer time arc and a narrower margin for indecision, Vaughn says. “There’s a level of education that’s needed to make sure that these patients are truly informed, because often with gene therapy, once you get a dose, you can never get another dose, because then you’re going to have antibodies that will prevent you from” further dosing, she adds.
The stakes are high on many levels. “The decision to participate in a trial might be your only shot ever,” Vaughn explains. For example, she says, “If you’re participating in a trial that’s first-in-human, and they’re starting with a lower dose, and it’s not an effective dose, that might be” your first and only chance to participate. In essence, you’ve just been eliminated from assisting with subsequent trials testing higher dosage or other protocol variations.
Understanding Regulatory Guidance for Gene Therapy Development
It has been a big year for gene therapy. In the past 12 months, three gene therapy products received approval from FDA. In July 2018, FDA Commissioner Scott Gottlieb released a statement on the FDA’s efforts and commitment to advance gene therapy. The statement included the announcement of six new or revised guidance documents related to the development of gene therapy products. In this ACRP 2019 presentation, Dr. Vaughn will review Commissioner Gottlieb’s statement, provide an overview of the six guidance documents, and discuss the implications for clinical development of new gene therapy products.
Among patients, “there’s not a lot of experience yet, and not a lot of confidence yet, there’s a lot of hope; but there’s not a lot of knowledge from the patient perspective about what these therapies are, and what they can do,” Vaughn says.
Patients have questions about many issues, including the potential long-term effects of gene therapy trials. “I don’t think a lot of patients really understand that there are multiple different kinds [of these therapies], and that they act in different ways, and that they have different ways of modifying your genes or providing therapy that will then potentially have an impact on their lives.”
Author: Michael Causey