CSL Behring’s one-time gene therapy for hemophilia B, Hemgenix (etranacogene dezaparvovec), is to be made available to eligible patients in Spain after the country’s drug pricing committee, the CIPM, delivered a positive recommendation on national reimbursement.
The product, which has a list price of around $3.5m, was recommended at the committee’s 26 September meeting. Hemgenix has already secured reimbursement in a number of European countries – France, Austria, Denmark, England and Scotland – although its path to reimbursement has not always been easy.
“This significant milestone underscores the value of Hemgenix and means that patients in Spain will have access to this groundbreaking gene therapy through the local health care system,” CSL Behring commented. It added that the recommendation was based on the results of the pivotal HOPE-B trial and “consideration of the therapeutic and social value of Hemgenix and unmet need in the current treatment landscape.”
Describing the therapy as an “innovative one-time treatment option,” Maria Jose Sanchez Losada, general manager of CSL Behring Iberia, said the company’s priority in Spain now was to “work with health care authorities to conclude the agreement and ensure Hemgenix can reach patients and health care professionals throughout the country.”
Asked about the nature of the Spanish reimbursement agreement, the company said it could not give more details, but it was “confident in the clinical, economic, and societal value of Hemgenix.”
Noting that about 5,900 people have hemophilia B in Europe, it said would continue working to enable access to the therapy for “all appropriate hemophilia B patients.”
“We are actively engaging in a number of ongoing discussions with stakeholders in European markets and, while we are unable to comment on the current status, we are confident in finding solutions to patient access throughout the European region,” it added.
Overcoming Hurdles To Access
In February 2023, Hemgenix was granted a conditional EU marketing authorization for the treatment of severe and moderately severe hemophilia B in adult patients without a history of factor IX inhibitors. (Also see "CSL/UniQure’s Hemgenix To Enter Uncertain EU Market After Regulatory Nod" - Scrip, 21 February, 2023.)
But the therapy’s path to availability across Europe has not been trouble free. Last year, for example, the health technology assessment (HTA) institute for England, NICE, recommended against National Health Service funding, citing uncertainties about the clinical data and cost-effectiveness estimates and the therapy’s high cost. (Also see "England: CSL Undeterred By Draft NICE Rejection Of Its £2.6m Hemophilia B Gene Therapy" - Pink Sheet, 2 August, 2023.)
In June this year, though, the institute reached a “first-of-its-kind“ agreement with the company, involving a managed access scheme under the UK’s temporary funding mechanism, the Innovative Medicines Fund. (Also see ”Hemophilia Gene Therapy Hemgenix Gets Funding In England, With More Talks Underway In Europe" - Pink Sheet, 1 July, 2024.)
Denmark’s Results-Based Deal
Also in June, Denmark became the first Scandinavian country to recommend Hemgenix for reimbursement after the Medical Council, which provides guidance on the use of new medicines in the hospital sector, said CSL Behring had offered a results-based agreement where hospitals would pay for the therapy only if it was effective.
“It is not known how many years Hemgenix will work, and thus how many patients will have to resume preventive treatment with factor IX preparations,” the council said at the time. “There is also very little knowledge about side effects, especially in the long term, of treatment with Hemgenix. It is therefore important that the patient is followed closely in the clinic with frequent blood tests, especially in the first years.”
But the Medical Council too had initially turned down the gene therapy. In November 2023, it said Hemgenix was “not recommended due to uncertainty about long-term effects combined with a very high price.” The official list price for the therapy in Denmark was DKK21.5m ($3.2m).
“At that time, the Medical Council called on the pharmaceutical company to return with a new price agreement,” it noted.
“The new gene therapies have great potential and may in some cases be able to cure the patients, but they are also often extremely expensive, and there is great uncertainty about the effect and side effects in the long term,” said Birgitte Klindt Poulsen, deputy chair of the council.
“It is therefore very good that we have now entered into an agreement on Hemgenix, where the payment depends on whether the effect lasts.” Poulsen said she expected the first patients to begin treatment with Hemgenix in the autumn.
Scotland’s ‘Interim Basis’ Recommendation
Hemgenix has also been accepted for use on the National Health Service in Scotland, but only on an interim basis “subject to ongoing evaluation and future re-assessment,” according to the country’s HTA body, the Scottish Medicines Consortium.
Interim acceptance can be granted if the SMC considers that a medicine might provide value for money and if additional evidence is expected that may address the key uncertainties in the evidence presented by the company.
The SMC said its August 2024 decision took account of a confidential discount offered by the company, noting that it was able to be “more flexible in its decision-making because the medicine is for a rare condition.” The list price of the therapy per treatment is £2.6m ($3.5m), excluding any patient access schemes, it added.
It said that it considered the benefits of the therapy “in the context of the SMC decision modifiers that can be applied when encountering high cost-effectiveness ratios and agreed that as etranacogene dezaparvovec is an orphan medicine, SMC can accept greater uncertainty in the economic case.”
Beneluxa: Talks Underway
Hemgenix is also being assessed for reimbursement in Belgium and the Netherlands, according to the Beneluxa initiative, a five-country collaboration that aims to improve access to medicines by sharing best practices and conducting joint negotiations with companies.
On 1 August, Beneluxa said that the two countries had completed a joint HTA report on the gene therapy, involving a pharmacotherapeutic and budgetary assessment. But on 17 September, it announced that while joint price negotiations were the preferred approach of both governments after a joint HTA, “differences in health care systems can occasionally present challenges to this process.”
In this case, it said, there was “a clear alignment on principles for establishing the willingness to pay, as described in the HTA report.”
However, it noted that the financing of the current standard of care – factor IX products – was “organized differently in both countries” and therefore produced a “different outcome of the recommendation for reimbursement. Therefore, the decision was made to proceed individually with the negotiations for Hemgenix.”
Novel access approaches have also been applied to the gene therapy in Austria and France. (Also see "CSL Praises Austria’s ‘Pioneering Funding Solution’ For Gene Therapy Hemgenix" - Pink Sheet, 11 March, 2024.) (Also see "CSL Nabs First ‘Direct Access’ Funding Agreement In France For Its Gene Therapy" - Pink Sheet, 15 December, 2023.)
