Monday, July 15, 2024

Research and Development, FDA’s Platform Designation Program Targets R&D Challenges

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Research and development will benefit from a new FDA program designed to streamline approval pathways for multiple drugs developed on the same platform. This initiative aims to create development, manufacturing, and review process efficiencies, which could significantly benefit biopharma companies with technology platforms.

The FDA recently issued draft guidance for the Platform Technology Designation Program for Drug Development. This program is designed to identify and incorporate platform technologies that can bring “significant efficiencies” in the drug application process. The FDA will assess whether the platform technology meets the eligibility requirements and determine its potential for improving the application process.

Research and Development: FDA Defines “Platform Technology” for Streamlined Drug Approvals

The FDA’s definition of “platform technology” may differ from the industry’s. The agency describes it as a “well-understood and reproducible technology,” which could include a nucleic acid sequence, molecular structure, mechanism of action, delivery method, vector, or a combination of these technologies.

Under this program, biotech and pharma companies with a designated technology platform will receive advisory support from the FDA during the development process. The program will also prioritize interactions for products deemed to have the most significant public health benefit or impact.

The program aims to create more efficiencies in drug development, which is a welcome change for an industry facing rising costs. According to a report from Deloitte, the average cost of developing a new drug for the top 20 global pharma companies rose to $2.3 billion in 2022, primarily due to longer development timelines and expensive clinical trials.

AI and automation have played a significant role in accelerating drug development, with PwC predicting a potential 60% to 70% reduction in process timelines. In 2021, more than 100 drug and biologic application submissions included AI or machine learning (ML) components. The FDA is currently working on guidelines to regulate AI in drug development, which is expected to be issued in the future.

Research and Development

Research and Development: FDA Formalizes Pathways for Platform Technologies, Highlighting Benefits for Gene Editing

The new guidance formalizes existing pathways for drugs developed using platform technologies, which are already widely used. The FDA has previously highlighted the benefits of a formal platform designation program, particularly for gene editing technologies like CRISPR. Phillip Kurs, regulatory counsel for the FDA’s Center for Biologics Evaluation and Research, emphasized the timeliness of this program, noting the opportunities in gene editing where minor changes can be made without altering the overall platform.

The program allows companies with an approved new drug application, abbreviated new drug application, or biologics license application to request a platform designation for future applications. Companies can request a pre-submission meeting to discuss their planned platform technology designation request. The guidance suggests making the request at the same time as or following an IND application.

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The guidance was published in the Federal Register, with a comment period open until July 29. The Biotechnology Innovation Organization (BIO) has requested a 30-day extension to allow stakeholders more time to provide feedback.

The FDA’s Platform Technology Designation Program for Drug Development represents a significant shift in the regulatory landscape, aimed at enhancing efficiency and reducing costs in the drug development process. By leveraging well-understood and reproducible technologies, this program promises to expedite the approval of multiple drugs developed on the same platform, benefiting both the industry and public health.

As biopharma companies navigate this new program, the FDA’s guidance and support will be crucial in optimizing development strategies and ensuring successful applications. This initiative underscores the FDA’s commitment to fostering innovation while maintaining rigorous standards for safety and efficacy.

The approval of Iqirvo (elafibranor) for the treatment of primary biliary cholangitis (PBC) exemplifies the potential impact of such streamlined processes. The new treatment option for PBC, based on data from the phase 3 ELATIVE trial, provides a much-needed solution for patients with this chronic liver disease. As more platform technologies are recognized and incorporated into the FDA’s program, the future of drug development looks poised for greater efficiency and innovation.

Resource: Food and Drug Administration, May 24, 2024

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