Clinical Trials
The Avlayah accelerated approval for treatment of the neurological and peripheral manifestations of Hunter syndrome was based on a surrogate biomarker and may help alleviate concerns about FDA flexibility in reviewing rare disease products.
The US FDA’s anti-allergy therapy review divisions are eager to support less burdensome development pathways for potential food allergy treatments, agency officials indicated during a recent “expert panel.”
Experts from Orchard Therapeutics, the UK drug regulator and Fondazione Telethon encouraged companies developing advanced therapies to speak to regulators in all planned launch regions, as expectations can vary significantly.
The threat of Chinese dominance in the prescription drug sector appears to be a strong, unifying theme for legislators heading into the US Food and Drug Administration user fee reauthorization cycle, but two very different sets of policy issues must be addressed.
While regulators are “catching up” to the use of artificial intelligence in clinical development, some skepticism remains around the use of AI to generate synthetic patient data, says Alexion’s Nick France.
The first multinational clinical trial approval under the EU’s 70‑day fast-track pilot pathway is expected by the end of April 2026.
The agency offered to drop annual IND fees for sponsors not conducting Phase I trials in the US, but industry still raised several concerns about fee incentives for onshoring clinical development.
The EU remains a major global player in biopharma but needs to act quickly or risk falling further behind its rivals, according to EFPIA.
A regulatory roadmap and product development tracker, expedited scientific advice, and submission readiness meetings are being integrated into the European Medicines Agency’s decade-old priority medicines scheme.
Denmark’s medicines regulator has published guidance to support the progression of investigational advanced therapy medicinal products into first-in-human trials by clarifying the expectations for preparing investigator’s brochures.
EU data protection bodies back clinical trial harmonization under the draft Biotech Act, but insist on strong safeguards for sensitive health data.
So far this year, four drug candidates have secured a place on the European Medicines Agency’s priority medicines scheme for promising treatments for unmet medical needs.
While both the US and Europe have similar regulatory approaches to advanced therapy manufacturing, it is easier for patients in the US to access “out-of-specification” treatments if they have no other options, a biotech expert says.
France is preparing to roll out a fast‑track pathway that offers the possibility of 14-day approvals for applications of certain early‑phase national trials, adding fresh momentum to the race for clinical research leadership among countries in the EU.
Clinigen's CEO and senior executives outline how the services company navigated Japan’s rigorous regulatory framework to secure approval for Prolacta’s human milk–based fortifier as a prescription biological product.
Parexel’s CEO Peyton Howell and India chief Sanjay Vyas talk to the Pink Sheet about clinical trials amid global turmoil, the slowdown in big pharma decision-making and pivot in some cases to the FSP outsourcing model, evolving DCT definitions and India opportunities.
Draft guidance from the European Medicines Agency aims to ensure clinical trials in the EU can be initiated and continued safely during public health emergencies by introducing regulatory flexibilities.
An EU group of industry experts has issued “pragmatic” best practice recommendations to help sponsors create clear, fully accessible and standardized lay protocol synopses, meeting key transparency obligations of the Clinical Trials Regulation.
The agency requested uniQure test AMT-130 against a control arm in which patients undergo a sham procedure. The treatment may be more akin to other neurodegenerative gene therapies than the Huntington's chorea drugs that were approved based on placebo-controlled trials.
India’s proposal to speed up multicenter research through a single ethics review system is drawing strong interest from the research community.