RARE Daily

NICE Won’t Recommend Clinuvel’s Drug for Rare Skin Disorder

July 26, 2023

NICE Rejects Clinuvel’s Drug for EPP

Rare Daily Staff

The National Institute for Health and Care Excellence, the U.K.’s drug price watch dog, said it would not recommend Clinuvel’s afamelanotide for preventing phototoxicity in adults with the rare skin disorder erythropoietic protoporphyria.

Erythropoietic protoporphyria causes severe reactions to light. Patients with the condition suffer physical burns and ulcers following exposure to light. This usually occurs within minutes of exposure to bright lights, especially sunlight. Exposure to artificial light may be sufficient to evoke symptoms. Phototoxicity is unresponsive to traditional pain and burn management techniques and patients can be incapacitated for days before reactions subside.

Afamelanotide, marketed as Scenesse, is a melanocortin-1 receptor agonist. It increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light sources. It is an implant that is inserted under the skin. The FDA approved the drug in 2019 to increase pain-free light exposure in adult patients with a history of damage to their skin from erythropoietic protoporphyria. Scenesse won approval in Europe as an orphan drug for the prevention of phototoxicity in adult patients with EPP in 2014.

NICE said its recommendation is not intended to affect treatment with afamelanotide that was started in the U.K.’s National Health Service prior to its issuing the guidance. People getting treatment can continue without change to the funding arrangements in place for them before the guidance was published, until they and their doctor consider it appropriate to stop.

In its guidance, NICE acknowledged that there is some evidence from clinical trials that afamelanotide provides benefits for people with EPP, but said it’s difficult to quantify.

“Testimonies and evidence from people with EPP and clinical experts show that improvements in symptoms and quality of life would be of great importance to them,” NICE said, “But it is very difficult to measure the effects of the condition and treatment and, although afamelanotide is an effective treatment, the size of its benefits is highly uncertain.”

It called cost-effectiveness analyses for afamelanotide “very challenging.” Based on the available evidence provided by the company, the cost-effectiveness estimates are all substantially higher than the range normally considered acceptable for highly specialized technologies.

NICE also said the benefits of afamelanotide may not have been captured adequately in the formal analysis. But, even when considering the scenario based on additional evidence from stakeholders, the most optimistic estimates of cost effectiveness are still higher than those considered acceptable for highly specialized technologies.

“It is not possible to conclude that afamelanotide provides appropriate value for money,” NICE said.

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