Dr. Scott Gottlieb recently spoke to the International Society for Stem Cell Research in Berkeley, California, to address how the Food and Drug Administration (FDA) needs to adapt its approach to pre-market regulations of novel areas of technology -- such as gene therapy -- focusing on measures of risk rather than clinical applications.
Gottlieb is a physician and resident fellow at the American Enterprise Institute (AEI).
Gottlieb said that, based on its typical clinical approaches, the FDA would see “a gene approach to treating cancer as a treatment for cancer that must undergo and pass the same pre-market review process as other cancer therapies -- rather than as a treatment that uses a gene-based approach. As a result, the product-specific issues can get subordinated to the clinical issues.”
In highly novel areas, “it’s the product-specific issues that create the regulatory uncertainty,” he said.
Simply put: if there is uncertainty, more risk assessment is required.
“It’s the product features of these novel technologies, and not necessarily their clinical applications, that create the hypothetical and known risks,” Gottlieb said. “That means the FDA needs to organize more of its regulatory activities around programs centered on areas such as regenerative medicine and gene editing. It needs to more closely adjust its regulatory approach to address the unique nature of the different risks that it’s trying to address.”
Gottlieb further explained that there are three basic kinds of risk the FDA needs to assess: preventable risks, such as proper warning labels for consumers; inherent risks that are intrinsic to a particular product, such as bone marrow loss with chemotherapeutics; and hypothetical risks, which are difficult to measure in clinical trials.
These hypothetical risks require the FDA “to focus on those areas that create the most potential for risk and channel its resources behind those efforts,” Gottlieb said.
Unfortunately, Gottlieb said, the FDA is very set in its current methodology, and it is difficult to change something already set in its ways. Instead of being hyper-specific, he suggested a multidisciplinary approach in which the FDA is not the only organization investigating these products. The National Institutes of Health and the broader academic community of scientists would be beneficial to pre-market analysis.
Gottlieb stressed that this doesn’t mean the novel technologies should be slowed. Instead, the FDA should reshape its regulations “to give these opportunities the proper priority that considers not only their potentially novel risks, but also their great potential.”
“The qualities that imbue these products with uncertainty also give them their transformative potential,” Gottlieb said. “The attributes that give these products hypothetical risk are the reasons why we should prioritize them under a regulatory framework that’s shaped to their potential.”
