NICE reverses reimbursement recommendation for Hemgenix
Managed access agreement would cover hemophilia B without inhibitors
In a reversal of a previous recommendation, the U.K.’s National Institute for Health and Care Excellence (NICE) has recommended that the National Health Service (NHS), England’s publicly funded health insurance program, should offer reimbursement for Hemgenix (etranacogene dezaparvovec) to eligible adults with hemophilia B.
Reimbursement would come through a managed access agreement under the NHS’ Innovative Medicines Fund (IMF) and cover adults with moderate to severe hemophilia B without inhibitors.
CSL Behring, which markets Hemgenix, said it will work closely with authorized treatment centers to make sure patients have access to the only gene therapy approved for hemophilia B in the U.K. as soon as possible.
“The approval of access to [Hemgenix] in England marks a significant landmark for individuals with hemophilia B, offering a potentially transformative, long-term treatment option capable of improving the daily lives of eligible patients, but also alleviating pressure on the NHS and dedicated healthcare professionals managing hemophilia B,” Amit Nathwani, PhD, a physician and professor of hematology at University College London, said in a company press release. “With the UK’s foundational contributions to hemophilia B gene therapy, this endorsement represents a pivotal step towards realizing this community’s long-awaited access to innovative therapies.”
More data aids in Hemgenix decision reversal
Hemgenix is a gene therapy for hemophilia B that’s designed to provide patients with a healthy version of the F9 gene. Mutations in the gene, which lead to a lack of factor IX (FIX) blood clotting protein, are the cause of the disease’s spontaneous or difficult to control bleeding episodes. The one-time treatment was conditionally approved by U.K. regulators last year based on clinical trial data that showed it was generally safe and capable of lowering annual bleeding rates for men with moderate to severe hemophilia B.
Once a treatment is approved, NICE determines if it should be made eligible for reimbursement to patients under the NHS. The decision involves negotiations with the therapy’s developer to agree upon a price to the English government.
In draft guidance issued last year, NICE indicated that, despite Hemgenix’s clinical benefits, there wasn’t enough long-term data to show that its effects would last such that it couldn’t be determined that the therapy was cost-effective. The committee recommended that it not be covered.
The new decision is backed by additional data from the Phase 3 HOPE-B clinical trial (NCT03569891) CSL Behring has since released that shows a single infusion of the gene therapy continued to lead to significant increases in FIX activity levels that were sustained for up to three years and accompanied by reductions in annualized bleeding rates. Most patients (94%) no longer needed routine infusions of FIX replacement therapy.
Recommending that Hemgenix be covered under the IMF managed access program means the it will be accessible to patients over a limited five-year period while more clinical data from HOPE-B are gathered.
How Hemgenix will be covered
CSL Behring and NICE have agreed the English government will pay for the treatment under an outcomes-based model, which means that payment is contingent on Hemgenix showing the long-term clinical efficacy the developer indicates it should. This offers NHS assurance of the therapy’s value.
Hemgenix’s list price in the U.K. is £2.6 million, or a little under $3.5 million. The agreed-upon price of the gene therapy wasn’t disclosed, but NICE guidelines indicate it’s available to the NHS at a discount.
Hemgenix is the first gene therapy to receive a positive recommendation for managed access through the IMF program and the first advanced therapy medicinal product, that is, a medicine based on genes, tissues or cells, to use this type of outcomes-based payment model, according to CSL Behring.
“We are thrilled that NICE has honored its commitment to a more progressive approach when assessing highly innovative medicines such as [Hemgenix] and this decision affirms the U.K.’s aspiration to be a leader in life sciences,” said Eduardo Cabas, general manager of CSL Behring U.K. and Ireland. “We remain clearly committed to improving the lives of people living with rare genetic bleeding disorders, as demonstrated by this milestone for eligible haemophilia B patients in the U.K. and the NHS.”
Hemgenix is approved in all U.K. countries, but NICE’s recommendation only formally covers England. CSL Behring said it’s working with authorities in Wales, Scotland, and Northern Ireland to ensure patients in there will also have equitable access to Hemgenix.
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