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Regulatory Affairs
Published in John M. Centanni, Michael J. Roy, Biotechnology Operations, 2016
John M. Centanni, Michael J. Roy
Breakthrough Therapy: A breakthrough therapy designation is granted to products that are intended to treat serious condition and have preliminary clinical evidence demonstrating substantial improvement over the current standard of care therapies. Request for consideration of this product designation occurs during clinical development under an active IND. Benefits of this designation include intensive development guidance, organizational commitment to involve senior managers and experienced reviewers, Rolling Review, and other actions that would expedite the review process. If the FDA determines the product does not meet the breakthrough criteria, they will provide a nondesignation letter to the sponsor stating this status was not granted and explain the reasons for the decision.
Commissioning, Qualification, and Validation
Published in Terry Jacobs, Andrew A. Signore, Good Design Practices for GMP Pharmaceutical Facilities, 2016
On July 9, 2012, the FDA’s Safety and Innovation Act (FDASIA) was signed into law. One of the goals of FDASIA is to promote innovation to speed patient access to new products. Under FDASIA, new drugs may be given special designations to expedite development and agency review. This includes granting fast-track, breakthrough therapy, accelerated approval, and priority review designations. The goal of these designations is to expedite the development and review of new drugs with preliminary evidence indicating the drug may offer a substantial improvement over other available treatments for serious or life-threatening diseases, and especially for those that offer treatments where no other therapies are available. The ultimate goal is to bring these therapies to patients as soon as it can be concluded that the benefits justify the risks [15, 16]. This has led to earlier patient access to new, often lifesaving therapies.
Biologics regulation, second-to-market competition, and the use of blockchain technology: an opportunity for the FDA to support responsible biotechnology innovation
Published in Journal of Responsible Innovation, 2020
In 2012, the U.S. Congress passed the Food and Drug Administration Innovations Act (FDASIA). Under Section 901 of the FDASIA (which amends the Federal Food, Drug, and Cosmetics Act), the FDA is authorized to base Accelerated Approval Review for drugs that address serious conditions that fill an unmet medical need on whether the drug has an effect on a surrogate or an intermediate clinical endpoint or marker. The Breakthrough Therapy Review, also implemented under the FDASIA, is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and provides preliminary critical evidence which indicates that the drug may demonstrate substantial improvement over an available therapy on a clinically significant endpoint(s).