England Approves Expensive Hemophilia B Gene Therapy via Outcomes-Based Model

The National Health Service (NHS) in England has approved CSL Behring's Hemgenix, a costly gene therapy for hemophilia B, under an outcomes-based payment model. This arrangement provides temporary access while further data is collected to confirm effectiveness. Hemgenix, priced at $3.5 million, is a one-time treatment that replaces the missing clotting protein for individuals with this inherited bleeding disorder. This model ensures the NHS's financial protection and value of the treatment's benefits.

The National Health Service (NHS) in England has granted approval for CSL Behring's Hemgenix, a costly gene therapy for hemophilia B, under an outcomes-based payment model [1]. This arrangement allows temporary access to the treatment while further data is collected to confirm its effectiveness. With a price tag of $3.5 million, Hemgenix represents a significant investment for the NHS [1].

Hemophilia B is an inherited bleeding disorder characterized by the deficiency of clotting factor IX. Traditionally, individuals with this condition have relied on lifelong factor IX prophylaxis treatments to manage their condition and prevent bleeding incidents. Hemgenix, a one-time gene therapy, aims to replace the missing clotting protein and potentially offer long-lasting therapeutic benefits [1].

The NHS's decision to approve Hemgenix under an outcomes-based payment model demonstrates a commitment to evaluating the long-term value and effectiveness of innovative treatments while ensuring financial protection for the healthcare system [2]. This arrangement allows the NHS to reassess its initial decision based on the additional input and data collected over time [1].

While the cost-effectiveness of Hemgenix is a subject of debate, with some expressing concerns about its comparison to factor IX prophylaxis treatments [1], it is essential to consider the potential long-term benefits and reduced burden on the healthcare system that come with a one-time treatment [3].

It is important to note that this decision is not final, as the draft guidance is open to comment for the next 15 days before the NICE committee reassesses its initial recommendation [1]. However, the positive step towards approving Hemgenix under an outcomes-based payment model signifies a potential shift in the NHS's approach to evaluating and reimbursing innovative treatments.

References:

[1] FiercePharma. (2023, April 20). UK's NICE initial rejection of CSL Behring, uniQure's gene therapy Hemgenix. Retrieved from https://www.fiercepharma.com/pharma/uks-nice-initially-rejects-csl-behrings-pricey-gene-therapy-hemgenix

[2] NICE. (2023, April 20). Technology appraisal guidance 623: etranacogene dezaparvovec (Zynaglo®) for the treatment of paediatric and adult patients with severe haemophilia B. Retrieved from https://www.nice.org.uk/guidance/ta623

[3] National Health Service. (2023, April 20). Hemophilia B: Causes, diagnosis and treatment. Retrieved from https://www.nhs.uk/conditions/haemophilia-bleeding-disorders/hemophilia-b/