Association for Public Policy Analysis & Management

The Food and Drug Administration stands out among US federal regulatory agencies as one of the most congressional supported and empowered. Moreover, the statutory foundations of its regulatory presence date back to the 1930s. Thus, in studying how public agencies respond to rapidly emerging technologies, the FDA is likely to offer insight into a “best-case” scenario. Drawing from quantitative indicators about the FDA’s scientific and technical capacity and from interviews with FDA personnel and stakeholders, this paper shows how the agency is responding to the use of gene therapy technologies in healthcare. The paper, focusing on the FDA’s experience with CRISPR/Cas-9 gene editing technologies, demonstrates that the FDA, though it faces some constraints on its capacity, is able to maintain a visible and consistent presence in the scientific communities developing the technology. The paper also provides evidence that the agency’s earlier experiences with gene therapies provide an adequate foundation for successfully managing the introduction of new treatments incorporating CRIPSR/Cas-9 technologies.