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During a meeting to commemorate Rare Disease Day on Monday, officials from the US Food and Drug Administration (FDA) highlighted the importance of collaboration among stakeholders, while FDA Commissioner Marty Makary touted recent FDA initiatives to speed the development of new drugs for rare diseases.

The European Medicines Agency (EMA) has announced that its pilot program for the stepwise pediatric investigation plan (sPIP) has successfully supported timely pediatric development while maintaining scientific rigor. This program will be incorporated into the existing pediatric regulatory landscape.

The Medicines and Medical Devices Safety Authority (Medsafe) is holding a consultation on plans to extend the abbreviated procedure for new medicine applications and certain change notifications.

The European Medicines Agency (EMA) has published a revised guideline on the development of drugs aimed at treating peripheral arterial occlusive disease (PAOD) in the lower extremities. It will take effect on 30 June 2026.

The US Food and Drug Administration's (FDA) Center for Drug Evaluation and Research (CDER) approved 46 novel drugs in 2025, representing a slight decrease from the novel approvals in recent years. More than half of these approvals were for rare disease indications, with 25 drugs with orphan designation approved last year.

The US Food and Drug Administration (FDA) has announced it will take a flexible approach to reviewing all new cell and gene therapy (CGTs) biologics license applications (BLAs) to expedite the development of these products, rather than applying its flexibilities on a case-by-case basis.

Pharmaceutical industry groups are asking the US Food and Drug Administration (FDA) to revise its draft guidance on postapproval methods to capture safety and efficacy data on cell and gene therapies (CGT) to include a broader registry system that can collect postapproval data on these products. This data should be gathered by a variety of public and private organizations, not just clinical professional societies that currently collect postapproval data.

The International Council for Harmonisation's (ICH) Cell and Gene Therapy Discussion Group (CGTDG) recently released a discussion paper recommending a more tailored approach in its guidelines for cell and gene therapy products. ICH said the updated approach would better reflect the unique characteristics of these products in comparison to traditional drugs.

TGA has adopted about 370 international scientific guidelines as part of Australia's policy of aligning with comparable overseas regulatory counterparts wherever possible. While most of the guidelines in the latest batch would replace those already adopted, two European guidelines are new.

Commenters responding to the US Food and Drug Administration's (FDA) draft guidance on the development of regenerative medicine therapies for serious or life-threatening diseases want more details from the agency on which products qualify for regenerative medicine advanced therapy (RMAT) designation.

US Food and Drug Administration (FDA) officials are still 'working out the details” of the plausible mechanism pathway being proposed by FDA Commissioner Marty Makary, said Teresa Buracchio, director of the Office of Neuroscience within the Center for Drug Evaluation and Research (CDER), during a Duke Margolis Institute for Health Policy workshop on individualized therapies on Thursday.

Two of the US Food and Drug Administration's (FDA) top regulators proposed a new approach for approving personalized medicines under a so-called 'plausible mechanism” pathway. The pathway would enable the agency to approve individualized gene therapies and other bespoke medicines in cases where randomized trials are not feasible, and the biological cause of the disease is known.

WASHINGTON – Denise Gavin, the director of the Office of Gene Therapy at the Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA), emphasized that clinical teams and chemistry, manufacturing, and controls (CMC) staff need to work closely to accelerate the development of cell and gene therapy (CGT) products.

The US Food and Drug Administration (FDA) considers several factors when evaluating whether registry data are fit for purpose for regulatory decision-making, according to Pallavi Mishra-Kalyani, deputy director of the Division of Biometrics at the Center for Drug Evaluation and Research (CDER). These factors include whether the data are relevant and reliable.

Pharmaceutical companies in the US are requesting additional information on how to conduct stability testing for both new and existing drugs in response to the International Council for Harmonisation's (ICH) recent Q1 guideline. They have raised questions regarding the scope of the guideline and the appropriate timing for stability testing of drugs in development.

This is what six regulatory affairs professionals learned from their experience at the RAPS Kellogg Executive Development Program.