Hemgenix hemophilia B gene therapy reimbursable in Germany
Therapy covered by public healthcare system after deal with CSL Bering
The cost of the one-time hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) will now be covered for eligible patients in Germany through the country’s public healthcare system.
This comes through a reimbursement price agreement between developer CSL Behring and GKV-Spitzenverband, the German National Association of Statutory Health Insurance Funds.
“The agreement on the reimbursement price of Hemgenix is a decisive step for the care of people with [hemophilia] B in Germany,” Christian Wieszner, managing director of CSL Behring Germany, said in a press release. “It enables access to a groundbreaking therapy and [recognizes] the long-term medical and economic benefits of gene therapy.”
Given via an infusion into the bloodstream, Hemgenix aims to provide a working version of the F9 gene to individuals with hemophilia B. People with the disease have mutations in this gene that cause them to be unable to produce sufficient amounts of functional factor IX, known as FIX, a blood clotting protein.
By providing cells with a working F9 gene, the gene therapy provides patients with the tools to continuously produce FIX, thereby preventing the excessive or spontaneous bleeding episodes that characterize the disease. It’s also expected to eliminate or reduce the need for routine preventive therapies often used by patients.
List price in US of hemophilia B gene therapy Hemgenix is about $3.5M
Hemgenix has been approved for hemophilia B in several countries, including the U.S., Canada, the U.K., Switzerland, Australia, Saudi Arabia, Taiwan, South Korea, and Hong Kong. In the European Union, it is conditionally approved for adults with severe or moderately severe hemophilia B without inhibitors, a type of neutralizing antibodies against FIX.
As with other gene therapies, Hemgenix is among the most expensive medications in the world, with a list price in the U.S. of about $3.5 million.
Since the therapy’s regulatory approvals, CSL Behring has been working with stakeholders across Europe and elsewhere to come to reimbursement agreements to ensure patients can access the treatment.
This agreement in Germany follows ones in other European countries, including Denmark, Switzerland, Spain, the U.K., and Austria. Eligible patients in France and Denmark have already been treated with Hemgenix, according to CSL Behring.
The performance-based payment model … is unique in Germany. … Reimbursement is linked to treatment success of the individual patient.
In Germany, Hemgenix will be covered under an outcomes-based model, meaning that reimbursement will be tied to treatment efficacy. While the exact terms were not disclosed, these types of agreements usually mean that if a treatment doesn’t lead to certain clinical outcomes for a patient, its developer may offer refunds or discounts on its price.
This type of model is sometimes used for very expensive treatments, such as gene therapies, to help justify their high cost to insurers and public healthcare systems. This is the first time such a performance-based agreement has been implemented in Germany, per the release.
“The performance-based payment model … is unique in Germany,” said Stefan Neudoerfer, CSL Behring’s chief negotiator in Germany. “Reimbursement is linked to treatment success of the individual patient.”
According to Neudoerfer, the model addresses reimbursement challenges that can arise with gene therapies. That includes the lack of available data on their long-term efficacy, “which is inherent for any one-time therapy,” Neudoerfer said.
Ongoing studies are continuing to evaluate the long-term effectiveness and safety of Hemgenix. Recently presented data from the Phase 3 HOPE-B trial (NCT03569891) showed the gene therapy led to sustained bleed control for up to four years among men with severe or moderately severe hemophilia B.
Hemgenix is currently the only hemophilia B gene therapy being marketed anywhere in the world. Pfizer halted development and commercialization of its own hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt; sold as Durveqtix in Europe) earlier this year, citing limited interest from patients and physicians.
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