A Breakdown of Food and Drug Administration Decisions


Here’s a resource for what FDA decisions mean – and how they affect patients.

The Food and Drug Administration (FDA)’s approval process is often a lengthy one. While a drug or treatment regimen is on its way to approval, it may receive certain designations to assist this process.

Understanding FDA lingo can be confusing at times. So, here’s a cheat sheet for the next time you stumble on breaking news about a new therapy or FDA action.

  • Investigational New Drug (IND) Application. After a drug sponsor develops a new drug compound and seeks to have it approved by the FDA, they will submit an IND application to develop a plan for testing the drug on humans. This occurs in the preclinical setting of drug development and includes information about the drug’s composition and how it is made. For a drug to move past the IND setting, the FDA must determine that it will not place the patients on the trial at an unreasonable risk for harm.

  • New Drug Application (NDA). When drug sponsors submit an NDA to the FDA, they are formally asking them to approve the drug to be marketed in the US, only after clinical trials have concluded. An NDA contains all clinical trial data, as well as manufacturing, labeling, and biological and chemical information to determine the drug’s appropriate use. The FDA’s Center for Drug Evaluation and Research (CDER) then uses this information to determine whether or not the drug is sufficiently safe and effective for approval.

  • Biologics License Application (BLA). Drug manufacturers submit BLAs to the FDA as a means of asking the agency if they can introduce or deliver for introduction a therapy across state commerce. It is generally submitted after an Investigational New Drug (IND) application and following the appropriate studies have been conducted. BLAs must contain application information, product/manufacturing information, preclinical studies, clinical studies and labeling. If an agent is already FDA-approved in one indication, the manufacturer can submit a supplemental biologics license application for it to be used in another setting.

  • Review Designation. This establishes the timeline, milestones and a goal date by which an application is reviewed under Prescription Drug User Fee Act (PDUFA) performance goals. The review designation can be either standard or priority.

  • Standard Review. All nonpriority applications are considered standard applications. Under Standard Review, the FDA takes 10 months to decide if they are going to approve or not approve a therapy in a certain indication.

  • Priority Review. A priority review essentially speeds up the process by which a drug is assessed by the FDA. With a priority review, the review process is shortened to six months. These are given to drugs that are particularly promising in clinical trials and/or meet an unmet need. A drug must show evidence of increased effectiveness in the treatment, prevention or diagnosis of a condition; eliminate a treatmentlimiting drug reaction; document enhancement of patient compliance that is expected to lead to an improvement in serious outcomes; or show evidence of safety and effectiveness in a new subpopulation to be considered for priority review.

  • Breakthrough Therapy Designation. This expedites the development and review of therapies that can be used to treat serious or lifethreatening conditions. In order to be granted a breakthrough therapy designation, there must be preliminary clinical evidence showing that the agent is better than available therapies in at least one clinically significant endpoint. Once a drug gets a breakthrough therapy designation, it is given all the program features of a fast track designation.

  • Fast Track Designation. Fast track designations can be based on clinical or nonclinical data, and they speed up the development and review of drugs to treat serious conditions. This is based on whether the drug will have an impact on factors like survival, dayto-day functioning or the likelihood that the condition, if left untreated, will progress from a less severe condition to a more serious one. After a drug is given a fast track designation, the drug manufacturer will have more frequent interactions with the FDA and may be given a rolling review.

  • Accelerated Approval. A drug can be granted an accelerated approval based on a “surrogate endpoint,” which will likely predict clinical benefit. These are often granted in serious diseases that fill an unmet medical need. Of note, after a drug receives an accelerated approval and goes to market, drug companies are still required to conduct studies to confirm the anticipated clinical benefit. If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug; if the trial does not, then the agency could remove the drug from the market.

  • Prescription Drug User Fee Act (PDUFA). The PDUFA was passed by Congress more than two decades ago. With this, drug companies agree to pay fees that boost FDA resources and can help speed up the drug approval process, and the FDA agrees to time frames for its review of new drug applications.
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