FDA and novel therapeutics; MHRA shortens marketing authorization process
Improving access to innovative new medicines is a priority for health authorities globally. In the US, the FDA has been increasingly focused on novel therapeutics. Meanwhile, the UK’s MHRA has been taking its own steps to improve marketing authorization processes and tackle some of the challenges faced since the country’s exit from the EU. Efforts from regulators to support faster access to medicines reflect continued advances in cell and gene therapies as innovators seek ways to tackle limitations with existing and emerging technologies.
FDA takes a lead with its approach to supporting novel therapies
Over the past few years, the FDA has been striving to support the development and ultimately approval of novel therapeutics. The agency established an advisory committee to evaluate genetic metabolic disease treatments in December 2023 and that same month also issued final guidance aimed at helping drug and biologics developers to conduct efficient and successful drug development programs for rare diseases. Last year, the agency also granted approval to 72 novel agents. Learn more about what the FDA is doing to support innovation.
UK seeks to improve new medicine access with new regulatory developments
The UK’s MHRA is taking steps to ensure timely access for UK patients to both existing and innovative new medicines. From the start of 2024, the International Recognition Procedure will take into account the expertise and decision-making of trusted regulatory partners for marketing authorizations and product lifecycle activities, while the Windsor Framework, in effect from January 2025, seeks to restore the smooth flow of trade within the UK internal market. Learn more about these new procedures.
Gene Therapy Trends in 2024 and Beyond
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Cell and gene therapies bring huge promise to patients but there are issues with these exciting products. Among the current limitations faced are safety, immunogenicity issues, targeting challenges, dosing issues, or regulatory and payer barriers. Researchers are gaining a greater understanding of the obstacles faced and are assessing potential new solutions to these challenges.
Learn more about trends with cell and gene therapies in the year ahead.
Event: DIA RSIDM Forum session on the CTR and submissions in the CTIS
During the DIA RSIDM Forum, PharmaLex’s Cary Smithson will chair a session on sponsor approaches to comply with EU CTR, the importance of managing business process changes, and the challenges they dealt with along the way. Join Cary and speakers from industry for this insightful session, titled Complying with EU CTR, Managing Business Change and Submissions in CTIS to be held in North Bethesda, Maryland, on 14 February at 8.30am EST.