Once a promising investigational therapy or medical device is pioneered in a laboratory setting, it moves through the clinical trial process. 

At the conclusion of the clinical trial process, the trial sponsor creates a robust, detailed data package called a New Drug Application (or NDA), and submits it to regulatory authorities for review. The NDA contains data and evidence to support the company’s claims about their new therapy’s efficacy and safety profile.

In the U.S., the Food & Drug Administration (FDA), through its Center for Drug Evaluation and Research (CDER), is responsible for reviewing these submissions from drug developers and granting regulatory approval to new drugs and medical devices. A team of CDER physicians, statisticians, chemists, pharmacologists, and other scientists reviews the sponsor’s NDA data and proposed labeling instructions. 

The shared goals of both trial sponsors and regulators are to ensure the drug or device under investigation is safe and effective and that physicians and patients have access to relevant information they need to prescribe and use the medication safely, and to be aware of known safety risks or potential side effects or adverse events that have arisen during the clinical trial process. 

The typical FDA approval process takes place within a structured framework that includes:

Analysis: FDA reviewers analyze the current treatment landscape to understand the broader context surrounding the NDA. This helps FDA to evaluate potential risk/benefit tradeoffs associated with the new therapy.

Assessment of benefits and risks from clinical data: FDA reviewers evaluate the NDA and take into account any uncertainties that may arise from imperfect or incomplete data.

Strategies for managing risks: Given that all medications have potential risks for certain patients, efforts are made to develop risk management strategies (including detailed instructions and warnings provided in the FDA approved drug label).

Part of the NDA approval process may involve an inspection by the FDA of one or more of the clinical trial sites, sponsors or other partners to identify issues in the conduct of the study related to data accuracy, reliability, or regulatory compliance that may affect the assessment of the clinical data. 

Under certain circumstances, the approval process for a new drug can be expedited. The FDA’s Accelerated Approval mechanism can be applied to promising new therapies that treat a serious or life-threatening condition. 

In the U.S., the FDA also offers several other specific programs that would allow for faster review and a shorter pathway to commercialization. These include the following:

Fast Track: This designation provides a route whereby drug developers can seek to advance the FDA review of new drugs that will treat serious conditions or fill an unmet medical need, based on the ability to demonstrate that the drug provides a promising therapeutic impact during initial preclinical (animal) and early human trial data.

Breakthrough Therapy: This designation provides another route to expedite the FDA review process for promising new therapies that are intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over an available therapy.

Priority Review: Using this mechanism, the FDA directs its staff and resources to review and render its decision on a new drug application within six months, compared to the customary 10 months under standard review. It is usually reserved for use for medications aimed at serious medical conditions or those with an unmet medical need.

This blog is intended to be informational in nature. The information and other content provided in this blog, or in any linked materials, are not intended and should not be construed as medical advice, nor is the information a substitute for professional medical expertise or treatment.

If you have any questions or concerns, please talk to your doctor. Never disregard professional medical advice or delay in seeking it because of something you have read on this blog or in any linked materials. If you think you may have a medical emergency, call your doctor or emergency services immediately.

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