France
Casgevy, the world’s first CRISPR gene editing therapy, is the second drug to be accepted onto a managed access scheme via England’s Innovative Medicines Fund, offering a new treatment for patients with transfusion-dependent beta thalassemia.
England’s NICE has “misclassified” HER2-low metastatic breast cancer as a medium severity disease, say AstraZeneca and Daiichi Sankyo, the co-developers of Enhertu. NICE said it would consider a rapid re-appraisal if the companies offered a “fair price” for the drug.
Scotland’s health technology assessment body has agreed to reimburse Chiesi’s Elfabrio for Fabry disease, mirroring the decision from several other European countries – however, France turned down the rare disease drug, while Germany questioned its benefit.
The Haute Autorité de Santé says that the risk of granting early access may only be justifiable if uncertainties over the clinical benefit of a drug can be addressed within a “reasonable” timeframe.
J&J says it has “exhausted all current viable avenues” to get its antidepressant nasal spray Spravato reimbursed on England’s National Health Service, after NICE decided against re-appraising the drug following numerous funding rejections.
The CNIL plans to update its data protection methodologies based mostly on the results of a new consultation and the outcome of a pilot in which companies are asked to identify the challenges they have faced when designing trials with decentralized elements.
A study from the health technology assessment department of France’s national health agency, HAS, has identified which pharmaceutical products are most likely to receive a request for post-market studies. The agency hopes this information will aid in companies’ forward planning.
Adult immunization programs can save “billions” for society, but their value is underestimated because of challenges around measuring broader value evidence and a lack of incentives for companies to collect this data, says the Office of Health Economics.
Santhera Pharmaceuticals did not provide enough evidence to demonstrate that its Duchenne muscular dystrophy drug Agamree was a cost-effective use of resources, according to draft guidance from England’s health technology assessment body, NICE.
The health technology assessment institute, NICE, is not yet ready to recommend Casgevy for sickle cell disease and says it wants more data. Meanwhile, an access agreement relating to the treatment’s use for transfusion-dependent β-thalassemia is making progress in England, as are reimbursement talks for SCD in other European countries.
The bill is the latest in a series of actions intended to address the continuing problem of medicine shortages.
The key themes of France’s latest initiative on shortages are repatriating the production of certain essential medicines and their active substances, earlier detection of potential supply problems, and more effective and transparent communications with all actors in the supply chain.
The new committee, which will initially be in place for a year, will advise the French drug regulator when it is preparing its opinions on substitution in specific biosimilar product groups.
Sanofi says it will engage further with NICE after the health technology assessment body rejected Sarclisa for previously-treated multiple myeloma, and that it is “hopeful a way forward can be found.”
All product classes are affected by supply chain issues, with drugs for the cardiovascular and nervous systems, anti-infectives and anticancer drugs particularly at risk, says the French regulator.
Pfizer’s Talzenna is soon to be made available to National Health Service patients in England and Wales with HER2-negative advanced breast cancer with inherited BRCA mutations, after the company agreed to provide the PARP inhibitor at a larger discount than it originally offered.
The French regulator says “additional investigations” are being conducted into a fatal case of Lyell syndrome and other reports of serious cutaneous side-effects, with a view to taking “appropriate measures.”
A new expert body is examining the use of semaglutide and other GLP-1 receptor agonists, focusing on safety risks and ongoing supply issues.
CSL’s hemophilia B gene therapy Hemgenix is the first medicine to be granted reimbursement through France’s experimental “direct access” program, several months after England’s HTA body dubbed the one-time product too expensive for national funding.
The product information for all pseudoephedrine-containing medicines in the EU will need to be updated, the European Medicines Agency said today.