UK Recommends Hemgenix Gene Therapy for Hemophilia B Coverage Under NHS's Innovative Medicines Fund

The UK’s National Institute for Health and Care Excellence (NICE) has reversed its previous recommendation and now suggests that the National Health Service (NHS) should offer reimbursement for Hemgenix (etranacogene dezaparvovec) to eligible adults with hemophilia B. The treatment will come through a managed access agreement under the NHS’ Innovative Medicines Fund (IMF) and cover adults with moderate to severe hemophilia B without inhibitors. The therapy has been approved for the UK through conditionally approved by regulators last year based on clinical trial data that showed overall safety levels and lowered annual bleeding rates in men.

What is Hemgenix Gene Therapy?

Hemgenix is a gene therapy designed to provide patients with a healthy version of the F9 gene. Hemophilia B arises in patients when there is a lack of factor IX (FIX) blood clotting protein due to mutations in the F9 gene. As a result, patients suffer from spontaneous or difficult-to-control bleeding episodes. Hemgenix gene therapy for hemophilia B is a one-time treatment that gave generally safe results in clinical trials and is confirmed to lower annual bleeding rates in men with moderate to severe hemophilia B.

Hemgenix Approval Process

Once a treatment is approved, the National Institute for Health and Care Excellence (NICE) determines if it should be made eligible for reimbursement for patients using the National Health Service (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In draft guidance issued last year, NICE indicated that despite Hemgenix’s positive clinical benefits, there wasn’t enough long-term data to show that its effects would last, making it difficult to determine if the therapy was cost-effective. The committee recommended that it not be covered.

However, in a reversal of its previous recommendation, recommending Hemgenix be covered under the IMF managed access program means that the treatment will be accessible to patients over a limited five-year period while more clinical data from HOPE-B are gathered. 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. This offers NHS assurance of the therapy’s value, and patients with moderate to severe hemophilia B without inhibitors will have access to the only gene therapy approved for hemophilia B in the UK as soon as possible.

The Cost of Treatment

Hemgenix’s list price in the UK is £2.6 million, or a little under $3.5 million. The agreed-upon price of the gene therapy wasn’t disclosed, but National Institute for Health and Care Excellence (NICE) guidelines indicate it may be available to the NHS at a discount.

First of its Kind

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 to use this type of outcomes-based payment model. The NHS could reap the benefits of innovation from the UK’s committed contributions to hemophilia B gene therapy. Eduardo Cabas, general manager of CSL Behring U.K. and Ireland, said that “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.”

Coverage and Future Development

Hemgenix is approved in all of the UK countries, but NICE’s recommendation only formally covers England. CSL Behring is working with authorities in Wales, Scotland, and Northern Ireland to ensure patients have equal access to Hemgenix gene therapy for hemophilia B.

Experts and Medical Professional Feedback

Professor of hematology at University College London, Amit Nathwani, PhD, said in a company press release, “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.”

Clinical efficacy and cost containment are critical issues in the gene therapy market that companies need to aim to solve. Hemgenix’s case and positive recommendation show that a new model like the IMF program, managed access, and outcome-based payment model could be a way forward in this critical area of healthcare.

Originally Post From https://hemophilianewstoday.com/news/about-face-hemophilia-b-therapy-hemgenix-covered-nhs/

NICE U-Turn: Hemgenix Reimbursement Recommendation for Hemophilia News Today