Rare Daily Staff

It was not long ago that the biopharmaceutical industry complained about the slow pace at which regulators moved to modernize regulatory science, but last week U.S. Food and Drug Administration Commissioner Scott Gottlieb turned the tables on industry to decry the slow pace with which industry is adopting innovative trial designs.

In a statement issued by the agency last week, Gottlieb noted that the FDA has worked to identify innovative trial designs, evaluate the role of decentralized clinical trials and mobile technologies, and help validate novel endpoints that can enable trials to generate reliable evidence needed to assess product safety and efficacy more efficiently. But, he said, there’s been a “continued reluctance” among sponsors and clinical research organization to adopt innovative approaches.

“In some cases, the business model adopted by the clinical trial establishment just isn’t compatible with the kind of positive but disruptive changes that certain innovations can enable,” he said. “We appreciate that scientific and technical complexity is a real and ongoing challenge, but industry and academia also need to invest in and leverage these approaches and develop new incentives that reward collaboration and data sharing across the clinical research enterprise.” 

Gottlieb said modernizing clinical trails is an agency-wide priority as precision medicine is making possible earlier regulatory decisions about the risks and benefits of new medical products as a result of advances in genomics and the use of biomarkers. While he acknowledged that precision medicine is posing challenges to the entire medical research ecosystem, he said the agency is committed to developing regulatory frameworks that are appropriate for precision technologies that support emerging therapies.

“But these opportunities can be delayed or stymied by a clinical research enterprise that is often extraordinarily complex and expensive,” said Gottlieb. “Efforts to streamline medical product development based on advancing science can be frustrated by legacy business models that discourage collaboration and data sharing, and the adoption of disruptive technologies that make clinical research more effective.”

Gottlieb pointed to several efforts the agency has undertaken to encourage the use of innovative trial designs. For instance, he pointed to the Clinical Trial Transformation Initiative, which was established to explore the use of decentralized clinical trial and the use of digital health technologies. He also pointed to the FDA’s establishment of master protocol  trial designs that can be used to simultaneously evaluate multiple drugs with a single common control.

The agency released several new guidances with the statement. This included a draft guidance for the use of so-called “enrichment strategies,” which use clinical laboratory tests, genomic, or proteomic factors that target a groups of patient for the use of a precision medicine. It also released a draft guidance on risk-based approaches to monitoring clinical studies. And, the agency released a final guidance for industry on nonclinical development of pharmaceuticals for severely debilitating or life-threatening hematologic disorders.

“Without a more agile clinical research enterprise capable of testing more therapies or combinations of therapies against an expanding array of targets more efficiently and at lower total cost,” Gottlieb said, “important therapeutic opportunities may be delayed or discarded because we can’t afford to run trials needed to validate them.”  


Photo: FDA Commissioner Scott Gottlieb

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