BIMO inspections and investigators have declined since 2017, and investigators express frustration to the Government Accountability Office that their inspection classification recommendations are downgraded by the Center for Drug Evaluation and Research.

User fee goal dates in April include four novel agents with breakthrough therapy designations, including Pfizer’s hemophilia B gene therapy and Immunity Bio’s IL-15 superagonist complex for bladder cancer.

New poll from advocates pushing for courts and the states to stay out of FDA’s scientific decisions indicates majority of US voters support FDA drug safety determinations across party lines, though Republican support is more tentative.

US FDA’s drug center reported a net increase of more than 300 employees in FY 2023, while the biologics center saw an overall increase of more than 30.

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.

Flexible thinking and rigorous standards will both be needed to develop psychedelics as drug therapies in order to surmount the many complicating factors, from unique ‘set and setting’ aspects to functional unblinding, speakers at Reagan-Udall Foundation meeting agree.

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.

Sponsors need guidance on ethics requirements and clarity around national regulations to conduct cross-border clinical trials in the EU, a multi-stakeholder forum says.

Japan now allows pseudonymized personal data for medical use under a licensing system for wider use of real-world data. Meanwhile, a national cost-effectiveness assessment scheme has slashed reimbursement prices for Lagevrio and Kerendia, and Alexion’s Voydeya has been added to the reimbursement tariff.

As Merck’s 21-valent vaccine approaches its 17 June user fee goal, the US CDC’s vaccine committee is looking at new adult age-based recommendations and bracing for a full pipeline led by GSK’s 24-valent candidate.

A new bill is intended to address the increase in medicine supply problems in Denmark in recent years.

While the EU Health Technology Assessment Regulation could reduce divergence in reimbursement decisions made across member states, many national-level HTA hurdles and challenges will remain, market access experts from EFPIA say.

The combined format is the new default for briefing advisory committee members, US FDA Oncology Center of Excellence Director Pazdur declared.

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.

Insurance plans put on notice that CMS is concerned about restrictions on drugs with negotiated prices, but it’s not clear what the agency can or will do about it.

As it stands, drugmakers will have just 90 days to prepare their dossiers for EU-wide joint clinical assessments under the new Health Technology Assessment Regulation. Market access experts from EFPIA tell the Pink Sheet that this short deadline could delay patient access to complex medicines, such as innovative cancer drugs.

The generics and biosimilars industry body Medicines for Europe says disparate national stockpiling requirements are not a solution to shortages and can bring “significant risks” for the supply chain and access to medicines.

The rulemaking is supposed to formalize the Patent Trial and Appeal Board process, which has been in an interim status since a Supreme Court decision, giving manufacturers more ability to request oversight of the decisions.