Clinical Trials/R&D

The European Commission’s landmark AI Act will introduce strict new rules for AI systems, with the exception of products used solely for scientific research, something that could be good news for pharma, according to industry body EFPIA.

The European Health Data Space is expected to give researchers, industry and public bodies access to large amounts of high-quality health data for the development of new treatments and vaccines. The European Parliament has also approved new rules on Substances of Human Origin.

New draft guidance pushes industry to broaden clinical trial eligibility criteria while offering some protection from fears it could hurt efficacy results.

House appropriations subcommittee chair Andy Harris, R-MD, says the agency ignored several important factors in recommending that marijuana be reclassified as a Schedule III drug; Califf tells ranking member Sanford Bishop, D-GA, that although work is progressing on animal testing alternatives, 'we're a long way right now' from eliminating animal studies before first-in-human trials.

Nicole Verdun said children could participate in gene therapy clinical trials earlier if the necessary controls are in place.

Great Ormond Street Hospital is piloting a new approach to gene therapy delivery in which it will submit a marketing authorization application to the UK regulator for a lentiviral gene therapy used to treat ADA-SCID, a rare disease.

Scientific advice could help companies make up for the lack of involvement in scoping, but slots are in short supply.

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.

China’s public payer perceives innovative drugs in a different way from that of the country’s top drug regulator, a former senior healthcare security official with knowledge of the matter reveals. The posture has unnerved pharma companies, which have been hit by sharp price discounts.

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.

Recently retired US FDA Principal Deputy Commissioner Janet Woodcock will be advising the Haystack Project, with the goal of helping rare disease organizations encourage creativity in drug development programs without jeopardizing regulatory success, Woodcock told the Pink Sheet in an interview.

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.

CDER’s Center for Clinical Trial Innovation (C3TI) will support innovative clinical trial approaches designed to improve the quality and efficiency of drug development and regulatory decision-making. The initiative includes demonstration projects on Bayesian analyses, selective safety data collection and point-of-care trials.

New EU guidance outlines the process through which scientific committees, national competent authorities and other relevant decision-makers can request real-world evidence studies for regulatory purposes.

The US FDA’s Oncologic Drugs Advisory Committee unanimously agreed that accelerated approvals should be enabled by multiple myeloma trials using MRD as a surrogate endpoint.

A new center for personalized medicine in Estonia will see PacBio’s long-read whole genome sequencing technology used to unlock new information about how genetics impact patients’ drug responses, experts from the project tell the Pink Sheet.