This guidance explains how, during its evaluation of a drug development program, FDA may consider whether to rescind a breakthrough therapy designation (BTD). This guidance is consistent with, and supplements, the information on BTD contained in the guidance for industry Expedited Programs for Serious Conditions––Drugs and Biologics (May 2014) and other BTD policies and procedures of the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER).
Section 506(a) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 356(a)) provides for the granting of BTD “if the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.” The BTD program is intended to facilitate and expedite the development of those drugs that receive designation and involves a resource commitment from FDA to provide early and frequent advice, conduct multidisciplinary meetings involving senior managers, and when appropriate, expedite the review of resultant marketing applications. Thus, it is important that available evidence continues to fulfill the standards for BTD.
Breakthrough therapy designation applies to a drug (either alone or in combination with other drugs) and the specific use for which it is being studied. The information supporting the granting of BTD for a particular drug may change over time. Some drugs that appear promising in early development may not be shown to be safe or effective in later trials, or the magnitude of a treatment effect suggested by early development may not be observed in later stages of development. Accordingly, given the resource-intensive nature of the BTD program, and in keeping with the Agency’s authority to grant BTD only to drugs that meet the legal criteria, FDA periodically assesses whether designated products continue to meet the criteria for BTD. If the designation is no longer supported by subsequent data, FDA may rescind the designation.
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