FDA attempts to promote innovation through guidance and flexibility

June 26, 2011

Although the regulatory environment in the U.S. has been criticized for stifling pharmaceutical innovation and investment, several targeted Food and Drug Administration (FDA) activities have been designed to support innovation, said Douglas Throckmorton, MD, Deputy Director for Regulator Programs, Center for Drug Evaluation and Research at the FDA.

Although the regulatory environment in the U.S. has been criticized for stifling pharmaceutical innovation and investment, several targeted Food and Drug Administration (FDA) activities have been designed to support innovation, said Douglas Throckmorton, MD, Deputy Director for Regulator Programs, Center for Drug Evaluation and Research at the FDA.
         One focus of the FDA in fostering innovation has been issuance of guidance to improve clinical trial design and conduct. In 2011, the FDA drafted guidance on adaptive clinical trial design, in which a planned clinical trial design can be modified, as well as hypotheses following an interim analysis of data. The intended effect is to increase the likelihood of successfully meeting a study objective or improve the understanding of a drug’s treatment effect. 
         Throckmorton said that refining clinical trial populations to include only those patients most likely to benefit from an intervention would yield greater efficiency of clinical trials; “otherwise, it’s a waste of resources.”
         He noted that the FDA supported the expanded use of cardiac stents in diabetes through innovative indications for data collection, such as a performance goal derived from previous studies and literature on bare-metal stents.
         In addition, the FDA has partnered with Duke University in founding a public-private partnership (Clinical Trials Transformation Initiative) focusing on improving clinical trials; 60 members from government, industry, academia, patient and consumer representatives, clinical investigators, professional societies, and clinical research organizations comprise the initiative.
         Cardiac safety of diabetes drugs has received renewed emphasis with the recent unearthing of cardiac risk associated with rosiglitazone and COX-2-specific nonsteroidal anti-inflammatories. The FDA has since assessed the cardiovascular safety of medications more fully than in the past, said Throckmorton, including the requirement for a risk ratio less than 1.3 (for cardiovascular events) for approval without the need for postmarketing trials, and approval contingent on postmarketing trials if the risk ratio is greater than 1.3 but less than 1.8.
         Since the emphasis on cardiovascular safety, there has been no decline in the number of commercial investigational new drug applications (INDs) submitted to the FDA for treatments for diabetes, he said. Seventy INDs for diabetes drugs were submitted after the cardiovascular guidance compared with 63 before the guidance.