Clinical Trials
The UK’s Health Research Authority has updated its General Data Protection Regulation “transparency wording template” with more information on how data about research participants would be handled if it is sent abroad.
The EU Clinical Trials Regulation has “so far failed to improve Europe’s competitiveness,” according to a new report commissioned by the pharmaceutical industry body EFPIA.
Oncology staff also said more work is needed before patient registries can be used as an external control in clinical trials, but they strongly encouraged continued investment.
EU regulators have recommended extending a pilot on the use of raw clinical trial data in medicines evaluations after an interim review of the initiative found that using such data improved regulatory decision-making.
New nods for Boehringer Ingelheim’s survodutide, Sagimet’s denifanstat and Mirum’s volixibat bring the FDA’s non-infectious liver disease breakthrough therapy designations to 15, with six designees already approved.
A court order encompassing funding, drug pricing, clinical trials and overall policy implementation aspects is expected to cause a paradigm shift in the treatment of rare diseases in India. Sarepta, Roche and Sanofi are among the companies that have been part of pricing discussions.
Flexibility on the geographic location of clinical trial participants will depend on the underlying reasons the FDA wants diversity in the study, the agency’s Oncology Center of Excellence Director said.
Sponsors can expect to see a copy of final guidance on how to comply with the UK’s new clinical trials regulation in August 2025, while the new framework is set to apply from January 2026.
A US FDA Advisory Committee responded enthusiastically to the potential use of challenge models to enable pivotal trials of novel pertussis vaccines, but most members suggested more refinements are needed before they are ready to use.
Members of the Cardiovascular and Renal Drugs Advisory Committee repeatedly challenged Stealth’s assertion that a new randomized trial in Barth syndrome was not possible, but also said potential functional unblinding and other challenges could compromise a new study.
Slow adoption of alternatives to animal testing in the current decentralized regulatory framework shows the need for a ‘one-stop shop’ at FDA that can provide advice, precedents and qualification programs.
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.
It is time for a much greater focus on pull incentives to ensure viable market conditions for medicines for neglected diseases that are traditionally regarded as unprofitable.
The US FDA is finalizing a guidance intended to encourage the use of decentralized clinical trial techniques. After the COVID-19 pandemic necessitated adoption of the more patient-friendly approaches, the agency is clearly eager to see them become routine.
Having its own priority review vouchers could serve as an important pull incentive for neglected disease R&D in Europe, although the unpredictable nature of the vouchers may deter some investors.
Contract research organizations in India must gear up to comply with new registration requirements coming into effect in April 2025 that aim to enhance the quality and integrity of clinical trials, as well as of any bioavailability and bioequivalence studies conducted by them.
Draft guidance recommendations on setting enrollment goals lead PhRMA and BIO to seek assurances of FDA flexibility absent reliable disease prevalence data.
Industry organizations argued feedback should be expected and object to draft guidance language suggesting it may be optional. The agency also should apply waiver criteria flexibly and address potential consequences if enrollment goals are not met, stakeholders say.
New guidance from the European Medicines Agency explains how in vitro and in vivo models may be used instead of clinical data for the purpose of establishing therapeutic equivalence in a stepwise approach.
Companies dealing with commercial costings or contracts with the National Health Service for early phase clinical trials or trials of advanced therapy medicinal products should soon be able to conclude their negotiations more quickly.