Hemgenix first drug to clear France’s ‘direct access’ route

CSL Behring’s haemophilia B gene therapy Hemgenix has become the first drug to be authorised for use under France’s recently-introduced ‘direct access’ programme, which aims to shorten the time between the registration of innovative treatments and their availability to patients.

The route – which is being piloted for two years – allows drugs deemed to be innovative and with a major medical benefit by the Haute Autorité de Santé (HAS) to be made eligible for reimbursement by French social security without the usual pricing and sometimes lengthy negotiations with the Economic Committee for Health Products (CEPS).

It allows treatments to be fully reimbursed by the health insurance system for up to a year and is targeted at drug types that are not covered by France’s AAP early access scheme, which came into force in 2021 but only covers drugs that are “presumed to be innovative, including with regard to a potential clinically-relevant comparator.”

For new medicines like cell and gene therapies (CGT) there is often nothing to compare with, a limitation that the direct access programme was designed to address and was promised by President Emmanuel Macron in 2021.

Hemgenix (etranacogene dezaparvovec) was approved by the European Commission in February as the first-ever gene therapy for the bleeding disorder, getting a green light for use in patients with severe and moderately severe symptoms and no history of Factor IX (FIX) inhibitors – antibodies that can develop in patients treated with standard FIX replacement therapies.

At the moment, haemophilia B patients require lifelong treatment with intravenous FIX inhibitors, which can have a significant impact on quality of life and wellbeing, according to CSL Behring. As a one-shot treatment, Hemgenix raises the prospect of freeing patients from the burden of frequent infusions to control bleeding symptoms.

The company said it submitted a request for direct access to the Ministry of Health in early October, adding that being accepted “paves the way for other European countries to implement innovative access solutions.”

“It is hugely encouraging to see new pathways for access being explored, enabling eligible patients to receive gene therapy that could have a potentially transformative impact for them and their loved ones,” commented Dr Lutz Bonacker, CSL Behring’s general manager for Europe.

“This approval is an example of how effective collaboration and innovative thinking can deliver ground-breaking access solutions for patients across Europe, where there is still significant unmet patient need in haemophilia B,” he added.

At the moment, EU regulations say that member states should provide access to new therapies within 180 days of marketing approval, which has proved to be difficult with France’s conventional negotiation system.

It’s worth noting that to prevent companies from exploiting the system and pricing their therapies very high to start with, the direct access scheme includes a clawback clause once CEPS negotiations have been completed.

There has been criticism that the scheme does not go as far as Germany’s system, which was held up by Macron as a model to follow and allows manufacturers to launch a drug as soon as it receives a marketing authorisation without the initial appraisal demanded in France. Germany’s system is also applicable to all new drugs, not just those rated as offering major medical benefits.