Getting effective treatments for diseases and conditions to people as quickly as possible while guaranteeing their safety and efficacy, as well as protecting the safety of trial subjects, has always been a delicate balancing act for the U.S. Food and Drug Administration, the federal agency responsible for the safety of pharmaceuticals and medical devices, among other things.
That act has become more complex with the exponential jumps in medical research, particularly those related to the human genome, says FDA Commissioner Scott Gottlieb. With the increasing prevalence of “precision medicine,” which targets a specific vulnerable group on the basis of current disease, genetic predisposition or the like, researchers have the opportunity to test their products on the populations most likely to need, and to respond, to them.
However, according to Gottlieb, protocols for clinical trials — up to now the main vehicle for testing medical innovations — have not kept pace.
Thus, “modernizing clinical trials is an agency-wide priority,” Gottlieb said in a statement released March 14. Using means other than clinical trials in some instances is also a valid modernization, he said.
Gottieb noted there is considerable resistance to innovation among many in the research community who are wedded to old protocols, but the FDA nevertheless recently issued guidance to those seeking to streamline the process: Strategies for Clinical Trials to Support Demonstration of Effectiveness of Human Drugs and Biological Products; Guidance for Industry; Availability and a Risk Based Approach to Monitoring of Clinical Investigations: Questions and Answers Guidance for Industry and Severely Debilitating or Life Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals. It invites public, industry and scientific comment on each statement.
One aim of the FDA, according to Gottlieb’s statement, is to bring more “real world data” into clinical research. For example, relying on precision medicine to target real-life patients who are at high risk of, or already, suffering from specific conditions rather than testing on blanket populations would increase knowledge of the “real world” efficacy of a treatment while also streamlining the time and cost of investigative studies, and “The same information used to select patients based on their likelihood of responding positively to a drug in a trial can be used to guide real world care,” according to Gottlieb in the statement.
In his statement, Gottlieb emphasized that while the agency believes it should be a priority to develop strategies for getting the right treatments to the right patients as efficiently as possible, the FDA remains committed to protecting the safety of humans who participate in the trials and to limit, as much as possible, the use of animals.
