FDA issues new guidance for Breakthrough Therapy designation
Published: Jun 24, 2022
By Alex Keown
Breakthrough Therapy designation is an important tool that expedites the development and review of a drug candidate that is believed to provide a substantial improvement to patients over available options.
The FDA noted some drug candidates that receive the Breakthrough Therapy designation during the early stages of development may not pan out during later-stage clinical trials. If the data no longer supports the designation, the FDA can rescind it. Not only can the designation be rescinded if clinical data no longer supports the necessary safety and efficacy, but the FDA also noted that it can revoke the designation if another drug is approved for that same unmet need for which the original drug received the designation.
An additional reason to revoke a Breakthrough Therapy designation is if the company that is developing the drug candidate no longer pursues the development of the drug for the indication that sparked the need for the designation.
“In assessing whether the criteria for BTD continue to be met, FDA typically gives greater weight to trials that are conducted in larger populations, use a well-understood and widely accepted, well-constructed clinical endpoint, and incorporate certain design features (e.g., randomization, blinding). Thus, the quality of evidence available may impact FDA’s decision-making,” the agency wrote in its six-page draft document.
The FDA also noted that it maintains the authority to not rescind the designation, even if clinical results appear to not support the award. The regulatory agency said if there are no “significant issues with the conduct and design of a subsequent study, the subsequent study may be given less weight in assessing whether the criteria for BTD are still met,” meaning the decision will be made on a case-by-case basis.
In addition to its guidance on Breakthrough Therapy designation, the FDA also released an action plan for rare, neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). The new action plan is a five-year strategy aimed at improving the lives of people facing these often deadly diseases through the advancement of new treatments and medical devices.
In its announcement, the FDA said the plan focuses on the promotion of innovation for these neurodegenerative diseases through targeted activities, which include the establishment of the FDA Rare Neurodegenerative Diseases Task Force, as well as developing disease-specific strategies and bolstering public-private partnerships for rare neurodegenerative diseases.
“The action plan is a blueprint for how the agency will move forward in aggressively tackling challenges in drug development for rare neurodegenerative diseases, including ALS, in order to improve patients’ health. Specific actions include regulatory science initiatives, enhancements to existing programs and new policy initiatives,” the FDA said in its announcement.
With ALS, patients and patient advocates have been fighting for an improved regulatory process that will bring those experimental treatment options to them at a quicker pace due to the rapid deterioration the disease causes.
With the new action plan, the FDA said part of the focus is ALS-specific which provides a “forward-leaning framework” to assess the disease priorities. Amidst that framework is a plan that will facilitate patient access to new drugs when possible and also improve participation in clinical trials through decentralized trial structures. The FDA said it also wants to enhance the trial infrastructure and agility in order to “enable early selection of promising therapeutic candidates for further development, optimize clinical trial design, improve access to the trials, streamline clinical trial operations, and reduce the time and cost of drug development.”