Cell & Gene Therapies
Sponsors making “big statements” with “no evidence” to back them up is one of the biggest issues faced by the UK drug regulator when it is assessing the regulatory dossiers for advanced therapy medicinal products, a quality assessor from the Medicines and Healthcare products Regulatory Agency says.
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.
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.
Japan's regulatory authorities recommend approval of products including a cell therapy, while setting reimbursement prices of newly approved drugs and cutting some others following cost-effectiveness assessment.
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.
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.
A regulatory sandbox and greater global harmonization will not only improve pathways for Indian cell and gene and other advanced therapies to reach the UK’s National Health Service but also speed up approvals in India
Designation of more regulatory resources by China’s top drug regulator to the cell therapy sector appears key to the recent consecutive release of guidelines related to CAR-T products in the country.
The EU should focus on supporting the development of collaborative hubs that underpin global excellence in translation, manufacturing and commercialization of advanced therapy medicinal products to keep pace with the US and China, a senior figure from Bayer says.
Platform technologies in the EU are set to benefit from greater regulatory flexibility under the upcoming EU pharma legislation overhaul. Industry is urging policymakers to “avoid misalignment” with other global regulators, including in the US.
The overhaul of the EU pharmaceutical package is expected to introduce two new regulatory “concepts” relating to platform technologies. A European Commission expert explains how the changes could impact industry and what the expected benefits are.
EU regulators must strive to balance scientific rigor with “common sense” when it comes to advanced therapy medicinal products, particularly for patients with no other treatment options, Immatics’ chief medical officer tells the Pink Sheet.
Japan has granted the first positive approval recommendations worldwide for two allogenic cell therapies derived from induced pluripotent stem cells, Sumitomo’s raguneprocel for Parkinson’s and Cuorips’ cardiomyocyte patches for heart failure.
Elevidys granted reimbursement in Japan following its conditional approval at almost $2m per patient, a price local regulators note is still lower than levels in the US.
The Brazilian government wants to boost the country’s capabilities to develop radical innovation that leads to new therapies for the national health system.
Changing regulatory standards for cell and gene therapies extend the flexibility with manufacturing while the tabelecleucel complete response letter showed high expectations for rigor in clinical trials.
The EU’s draft Biotech Act promises faster clinical trial approvals and innovation incentives, but the industry warns that intellectual property provisions have limited appeal.
Experts said the impact of the planned approval standard changes are difficult to assess given the potential for flexibility outlined in the JAMA article, as the FDA again eschews guidance and public comment in making new policy.
The agency wants to ensure orphan drug sponsors cannot benefit from fee exemptions while gaining approval of non-orphan indications.
However, the agency updated warnings on myocarditis risk and mitigation for the gene therapy, including more specific information about its severity and timeframe for occurrence in Duchenne muscular dystrophy patients.