- People in England and Wales with a rare inherited eye disorder could soon be covered to receive treatment with Spark Therapeutics' pricey gene therapy Luxturna, following an agreement between Novartis, which sells the drug outside the U.S., and a U.K. cost-effectiveness agency.
- NICE, or the National Institute for Health and Care Excellence, on Wednesday recommended Luxturna for use through the National Health Service. The gene therapy, which secured marketing authorization in Europe last fall, costs about $746,000 per patient at list price in England.
- That's cheaper than the $850,000 per-patient U.S. price tag. Novartis also offered an undisclosed discount to NICE to secure a favorable recommendation, the cost agency said.
Securing NICE's backing isn't a sure thing for drugmakers.
The agency, which evaluates cost-effectiveness of new drugs for national insurer NHS, has clashed often with pharmaceutical and biotech companies, including Novartis. Unlike in the U.S. where there is no government body charged with determining cost-effectiveness, NICE functions as a gatekeeper to coverage for new medicines.
In the case of Luxturna (voretigene neparvovec), NICE evaluated the gene therapy through its "highly specialized technology program," a pathway previously used to evaluate Strimvelis, an ex vivo gene therapy made from patient cells.
Luxturna treats a specific type of inherited retinal dystrophy that stems from mutations in a gene known as RPE65. The condition is usually diagnosed in childhood and progresses to near-total blindness over time.
Injected directly into the retina, Luxturna is designed to be curative, delivering a functional copy of the gene to spur production of the needed RPE65 protein. In clinical testing used to secure U.S. approval, Luxturna improved sight in 27 of 29 patients after at least one year of follow-up.
Novartis estimates roughly 180 people in the U.K. have mutations in both copies of the RPE65 gene, a population that could soon access the treatment through NHS coverage.
NICE's recommendation was published as a draft guidance, and can be appealed by stakeholders including healthcare professionals and patient groups. If none are received, NICE plans to publish final guidance in October.
To secure a favorable ruling from NICE, Novartis offered a discount on Luxturna's list price of 613,140 pounds per patient, as that price would have broken NHS budget rules. NICE said it would keep the size of the discount confidential.
In the U.S., Luxturna costs $425,000 per eye at list price.
A key part of assessing Luxturna's cost-effectiveness is determining how long the treatment's benefit might last. Currently, follow-up of treated patients extends out through seven-and-a-half years. While negotiating with NICE, Novartis assumed a treatment effect would last 40 years — about midway between that seven-and-a-half year figure and an assumed 70-year lifetime.
Clinical experts convened by NICE considered a lasting benefit to be biologically plausible provided Luxturna is effectively delivered into the eye. As photoreceptor cells don't replicate or regenerate, introduction of the RPE65 gene into the cell nucleus should ensure lasting production of the missing protein.
NICE could soon face decisions on other costly gene therapies. Bluebird bio's treatment for beta-thalassemia, Zynteglo, was approved in the EU in June and costs $1.8 million at list price. The biotech has delayed launching the therapy until early 2020, however, to sort out commercial manufacturing.
And Novartis' own gene therapy Zolgensma (onasemnogene abeparvovec) could receive EU approval later this year. That treatment costs $2.1 million per patient in the U.S., and has recently come under scrutiny over manipulated preclinical data used to win its Food and Drug Administration approval.