The House Appropriations Agriculture, Rural Development, and FDA Subcommittee advanced the FDA’s fiscal year 2025 funding bill, beginning a likely long and difficult budget cycle. 

Seven rare disease programs make up the first round of participants in the US FDA’s ‘Operation Warp Speed’-style program. Sponsors will benefit from ‘rapid, ad hoc’ communication with the agency.

Gene therapy for wet age-related macular degeneration is one of two later-stage gene therapies flagged in CVS Caremark’s latest pipeline report that are outliers because they target conditions afflicting millions of patients and could dramatically increase Part B spending.

US suitability petition submissions see an uptick against the backdrop of GDUFA III new goals for the FDA's response to such petitions. The Pink Sheet tracks some of the activity as applicants seek to tap market opportunities without the need for new clinical data.

Regulators from the US, Canada, European Union, Switzerland and Japan talk about the benefits of global collaboration, and reasons why they might reach different decisions on the same drug, at the American Society of Clinical Oncology annual meeting.

EMA charges participants to receive scientific advice through the fledgling program, unlike the US FDA, which may not fit the budgets of some complex generic sponsors. At the same time, sponsors also may simply not be aware the program exists yet.

Acknowledging that it may be a barrier to some extent, EMA’s senior scientific specialist of clinical pharmacology Kevin Blake notes that EU reference medicinal products are a legal requirement for bioequivalence studies and cannot be circumvented.

While EU preparations are underway to introduce strict legal requirements for all AI systems, the UK has doubled down on its flexible, non-regulatory framework. In this second of a two-part article, a lawyer explains the pros and cons of each for pharma and medtech firms.

J&J says it has “exhausted all current viable avenues” to get its antidepressant nasal spray Spravato reimbursed on England’s National Health Service, after NICE decided against re-appraising the drug following numerous funding rejections.

The House Appropriations Agriculture, Rural Development, and FDA Subcommittee advanced the FDA’s fiscal year 2025 funding bill, beginning a likely long and difficult budget cycle. 

New regulation on  substances of human origin (SoHO) will help improve Europe’s “strategic autonomy” and improve access to such substances, say industry representative.

Updated clinical trial reporting rules and stronger participant protections are among the several changes to research in Switzerland that will apply from November, while new regulations on clinical trial transparency will apply from March 2025.

The advisory committee also hails the treatment-to-amyloid clearance approach used in the Phase III trial as innovative, but questions how it should apply in clinical practice.

The notice of opportunity for hearing on the US FDA’s proposal not to approve an insomnia sNDA for Vanda’s Hetlioz opens another front in the company’s aggressive campaign against complete response letters and generic competition to the sleep disorder drug.

In an ‘ask me anything’ Q&A on Reddit, Sarah Yim, director of US FDA’s Office of Therapeutic Biologics and Biosimilars, offered some insights on the US biologics naming convention, biosimilar interchangeability, and the possibility of future competition on cell and gene therapies.

Stay up to date on regulatory guidelines from around the world with the Pink Sheet's Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.

The European Parliament could have helped to stimulate R&D into new drugs for rare diseases if it had not stuck so closely to the European Commission’s original proposals on orphan exclusivity periods, say Baker McKenzie lawyers Els Janssens, Julia Gillert, Magda Tovar and Olha Sviatenka.

As of today, drug developers can apply to take part in two pilot programs that will involve regulators across the EU strengthening their coordination when it comes to providing companies with advice on their applications for clinical trials and marketing authorization.

In an interview with the Pink Sheet, Troy Tazbaz, director of the US FDA's Digital Health Center of Excellence, says the agency lacks authority to regulate assurance labs, which would be used to help AI developers ensure their models are working correctly before submitting them for approval.

Regulators from the US, Canada, European Union, Switzerland and Japan talk about the benefits of global collaboration, and reasons why they might reach different decisions on the same drug, at the American Society of Clinical Oncology annual meeting.