NHS England to fund Novartis' gene therapy Luxturna for inherited retinal disease

NHS England announced Wednesday funding for Novartis' Luxturna (voretigene neparvovec) after agreeing a deal with the company that will provide access to the gene therapy from January 2020. The one-time treatment gained European Commission approval last year for patients with vision loss due to a genetic mutation in both copies of the RPE65 gene and who have enough viable retinal cells.

Luxturna costs £613,410 ($747,440) per patient, with NHS England noting that the agreement was reached after Novartis entered into a licensing and supply agreement with Spark Therapeutics, making the therapy available to markets outside of the US. NHS England said that the treatment will initially be available from three national specialist centres across the UK, with the option to roll-out the treatment to other hospitals.  

NHS chief executive Simon Stevens remarked "this latest deal reinforces the benefits for companies willing to engage with us and be pragmatic with their pricing – which is good news for patients, tax payers and industry." In the US, where Luxturna is priced at $850,000, or $425,000 per eye, the Institute for Clinical and Economic Review has been critical of the gene therapy's cost, arguing that in most cases it should be 75% less than the proposed price.

The move coincides with the National Institute for Health and Care Excellence issuing draft guidance recommending Luxturna. NICE noted that in the absence of an undisclosed discount offered by Novartis, the therapy would have cost the UK more than £20 million ($24.4 million) over three years, a figure that failed the Budget Impact Test. According to the agency, results from clinical studies have shown that in the short term, Luxturna improves vision and prevents the condition from getting worse. NICE added while there is no long-term clinical evidence, its appraisal committee considered it is biologically plausible that the treatment effect is likely to continue for decades.

Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, commented "the company's willingness to work with us early and constructively has allowed us to publish this guidance on a much faster timeline than normal which is good news for patients." NICE indicated that its cost-effectiveness evaluation of Luxturna was completed within 20 weeks, opposed to an average of 38 weeks under the highly specialised technologies programme.

Luxturna is the second gene therapy to receive positive guidance under NICE's highly specialised technologies programme following backing of GlaxoSmithKline's severe combined immunodeficiency-adenosine deaminase deficiency therapy Strimvelis last year.

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