Strategy Statement
FDA Commissioner Scott Gottlieb, M.D., said that it is “an agency-wide priority” to develop new strategies to modernize clinical trials that advance precision medicine, patient protections and more efficient product development. In a statement released March 14, Gottlieb said that the FDA wants to modernize its approach to take advantage of advances in precision medicine and develop a regulatory framework to test product safety and efficacy as efficiently as possible. The objective is to give patients earlier access to important new therapeutic options while maintaining their safety.
However, Gottlieb said that attempts to streamline medical product development based on advancing science is difficult because of established models “that discourage collaboration and data sharing” and new technologies “that make clinical research more effective.” He cautioned that unless clinical research tests more therapies or combinations of therapies against an array of targets more efficiently and inexpensively, important therapeutic opportunities could be delayed or lost.
The FDA is working with such organizations as the Clinical Trial Transformation Initiative, 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.” As a result, the FDA has come up with master protocol trial designs that can “evaluate, in parallel, different drugs compared to their respective controls or to a single common control.” Because these trials can be updated easily, the infrastructure can last for decades, reducing administrative costs and time.
Gottlieb said that new research paradigms are necessary for breaking down barriers between real world data and clinical research, enabling rapid evidence sharing that improves both domains across a learning health care system. More trials can use data from electronic health records and adopt electronic informed consent, thus enabling enrollment of more patients in clinical trials closer to where they live and work, reducing barriers to clinical trial participation and accelerating researchers’ ability to ask and answer important questions.
The FDA released guidance for sponsors for incorporating patients with challenging health conditions into oncology clinical trials — patients with brain metastases or previous malignancies, patients with organ dysfunctions and adolescent and pediatric patients. It also released guidance for industry on strategies to support the development of precision medicines and guidance on risk-based monitoring using computerized systems for effective oversight. Enrichment Strategies for Clinical Trials to Support Determinations of Effectiveness of Human Drugs and Biological Products and A Risk Based Approach to Monitoring of Clinical Investigations: Questions and Answers Guidance for Industry can bring about efficient development of novel innovations and provide evidence to determine product safety and efficacy.
Another guidance for industry, Severely Debilitating or Life Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals (SDLTHDs), is designed to streamline the development of pharmaceuticals for treating SDLTHDs while protecting patients’ safety and avoiding unnecessary use of animals while conducting trials. It addresses the appropriate use of non-clinical tests and applies to drugs used both to treat the active disease and to prevent the recurrence of a life-threatening or debilitating event.
Gottlieb said that enrichment is also a key strategy in the development of precision medicine, targeting treatments at groups of patients based on clinical laboratory tests, genomic or proteomic factors. He concluded that risk-based monitoring, as a component of a sponsor’s overarching quality risk-management systems and trial-specific quality by design programs, provides more efficient oversight of trials while protecting human subjects and assuring data integrity.
