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Compliance In Focus
Posted by Ryan Begun on Thu, Nov 5, 2015

Expedited Review: Breakthrough Therapy

In the wake of the recent Ebola pandemic, we realize the importance of pushing drugs and pharmaceuticalsExpedited_Review_Breakthrough_Therapy through the FDA approval process as quickly as possible. From drug discovery to FDA approval, the average drug takes roughly ten years costing $2.6 billion dollars during the process. Delaying the drug from reaching market just one day can cost the Sponsor millions and potentially the lives of patients hindered by the condition the drug is to treat. With the pressing need for cures and treatments, an expedited approval has the potential to be advantageous for all. Over a series of blogs, I will review the four FDA expedited review programs.

Breakthrough therapy is intended to accelerate the development and review process of pharmaceuticals that aim to treat serious diseases. In addition to the requirement of the drug to treat a serious disease, it must also show preliminary clinical evidence indicating that the drug demonstrates substantial improvement on a clinically significant endpoint(s) over available therapies. According to Section 506(a) of the FD&C Act, for designation of a drug as a breakthrough therapy “…if the drug is intended, alone or in combination with one 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 one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.” In the realm of breakthrough therapy, a clinically significant endpoint refers to one that measures an effect on irreversible morbidity or mortality (IMM).

Pharmaceutical companies are to request expedited review for a breakthrough therapy product with or after the Investigational New Drug (IND) application is submitted to the FDA. In most cases, the breakthrough therapy designation request is submitted as an amendment to the IND. The primary goal of this expedited review is to obtain data to support the efficacy of the drug; it is anticipated that the submission is made no later than end-of-phase-2 to ensure the trial is run as efficient as possible. Since supporting clinical data is required, requests for designation is submitted after the pre-Biological License Application (BLA) or New Drug Application (NDA).

Upon receipt of the request, the FDA has 60 calendar days to issue a response. In the case that a designation letter is issued to the Sponsor, the FDA will encourage communication with the company to provide guidance on subsequent development. The Sponsor will be alerted of any need for the drug development program to continuing to meet criteria to be designated as a breakthrough therapy drug. As the drug goes through its development, it will be reevaluated on whether it should keep the breakthrough therapy designation or not.

Compared to the normal review process, a drug is under the designation of a breakthrough therapy has many advantages to help expedite the process to gain FDA approval. Even though the Sponsor will still need to collect adequate data to demonstrate the drug safety and effectiveness, there are many ways to quicken the process. The FDA will provide timely advice and interactive communication in all aspects from study design to data analysis, resulting in more efficient trials. In addition the FDA will involve senior managers and experienced review and regulatory health project managers with the study. The Sponsor will be eligible for rolling admission, allowing complete sections of applications to be submitted instead of submitting the whole application at once.

Speeding up the review process for a drug that treats a serious disease or condition can have a trickle-down effect that results in saving the lives of many. While it is important to get a groundbreaking drug to market as fast as possible, getting accurate and complete data from clinical trials is equally as imperative. What are your thoughts on this expedited process?

Stay tuned for the upcoming blog on the accelerated approval path of expedited review!


Research Coordinator Support blog CTA

 photo credit: huffstutter via photopin (license)

Topics: FDA, Expedited Review


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