FDA supports rare disease drug development; Are you ready for the CTR transition? And new EMA fee regulation

Harmonization, transparency, innovation and patient-centricity have been at the forefront of industry and regulatory developments in recent years, and have been top of mind in 2024. Catch up on some of the key regulatory developments from industry, the FDA and the EMA in PharmaLex’s August newsletter.

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FDA’s OTP announces cell and gene site tours to enhance expertise

The FDA’s Office of Therapeutic Products (OTP), a part of the Center for Biologics Evaluation and Research (CBER), has announced its Cellular and Gene Therapies Interactive Site Tours Program . The objective is to allow CBER project managers and reviewers to exchange know-how with their industry counterparts. The center is seeking to help its staff improve their understanding of the biotechnology manufacturing industry and improve their review skills.

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 approval of novel therapeutics. The agency established an advisory committee to evaluate genetic metabolic disease treatments in December 2023 and has issued final guidance aimed at helping drug and biologics developers to conduct efficient and successful drug development programs for rare diseases. Learn more about what the FDA is doing to support innovation.

Have you transitioned to the CTR?

On 30 January 2025, the three-year transitional period from the “old” regulatory framework of the Clinical Trials Directive to the Clinical Trials Regulation and into the Clinical Trial Information System (CTIS) will end. It’s vital that companies build in adequate time for the transition. Learn more about the considerations that should be incorporated into strategic planning for transitioning to the CTR and CTIS.

How an IPD approach paves the way for a well-planned R&D program

Successfully navigating the many different activities involved in clinical research requires coordination, ideally built on an integrated product development (IPD) approach. First, however, it’s advisable to conduct a gap analysis to determine where you are and what needs to be done, and then use this as the basis of a development plan. Learn more about the key steps involved with an IPD approach.