Derry patients denied blood disorder treatment available in Scotland

CLINUVEL, the pharmaceutical company which has developed a successful treatment for the life-limiting condition erythropoietic protoporphyria (EPP) is preparing to mount a legal challenge in the form of a judicial review against the National Institute for Health and Care Excellence (NICE).

The company is also planning to issue civil proceedings against NICE, in November.

NICE is the organisation tasked with deciding which treatments should be available on the NHS in England. It also aims to make sure that people have the same access to treatment and care wherever they live.

Rare genetic blood disorder means Oran cannot go out in natural light

Derry porphyria patient faces being denied successful drug treatment

CLINUVEL manufactures SCENESSE®, the only approved medication for EPP, a rare genetic blood disorder which results in the sufferer being unable to go out in natural light.

The legal proceedings centre on the fact, SCENESSE® is available to EPP patients in Scotland however NICE has refused to approve it for patients in the North, Wales or England. 

Speaking to Derry News, Dr Philippe Wolgen, the CEO of CLINUVEL said the company had worked for nearly 20 years to develop the drug for adult patients. 

Dr Wolgen added: “SCENESSE® has a long-standing safety profile, with some patients receiving continuous treatment since 2006. It was first launched in Europe in 2016, with a US launch in 2020.

“Work is also underway to develop a SCENESSE® dose for children, focusing first on adolescent patients like Oran.”

Dr Wolgen was referring to Oran Carlin (15) from the Templegrove area of Derry who is living with an EPP diagnosis.

Describing the effects of Oran’s condition to this paper in July, his mum Breanda said: “EPP would start off with an inner itch, which leads to severe pain. Then his blood vessels leak, which causes swelling. 

“Depending on how much he has been out in the sun and how much he has been affected, an episode could last for up to 24 hours. The worst case ever resulted in him having to sit in the dark for a full week.

“Essentially, EPP means Oran cuts from the inside out. If he is going through an episode, it would look like his face has been scrapped along tarmac. This happens days into an episode whenever he is healing. 

“Whenever his blood vessels leak, Oran swells up and it looks as if he has been beaten up with a baseball bat. His skin stretches and then splits. 

“After a couple of days, he looks like he has got burn marks, from rubbing against the floor, on his nose, his lips and his chin,” said Breanda.

According to Dr Wolgen, it was CLINUVEL’s analysis that there were “400 active EPP patients in the UK”. 

He added: “The EPP story is a tragic story because no-one sees that patients are sick. You can’t see on the outside. 

“Unfortunately, NICE has refused to engage with CLINUVEL, patients or physicians in its review of SCENESSE®, despite more than eight years of discussions.

“It has also twice been found, on appeal, to have breached its own processes. 

“Most appallingly, the head of NICE’s Highly Specialised Technology (HST) Committee, a medically qualified professional, stated in 2018, he ‘did not see EPP as a disability as it was not a visible disability’.

“In doing so, he was found to have breached the Equality Act of 2010, but was never reprimanded and remains in his role to this day,” said Dr Wolgen.

Dr Wolgen added that CLINUVEL had taken “a transparent approach to drug access with organisations like the NHS”.

He said: “We have offered a uniform price - with no opaque discounts, cashbacks or rebates - and entering long-term agreements with payors. This approach has been accepted in many countries, including in Scotland, where EPP patients do receive treatment.

“EPP and SCENESSE® do not fit NICE’s utilitarian models for funding assessments. Despite having flexibility in their decision models, and being offered identical terms to Scotland, NICE has refused EPP patients in England, Wales and Northern Ireland access to the drug. 

“CLINUVEL is evaluating its options to enable patient treatment access long-term, including legal avenues available after the NICE process, finally, came to a conclusion. We have encouraged EPP patients and advocates to speak to their MPs to highlight the unfair and illegal conduct of NICE over eight years. 

“In reality, the game NICE plays is that it wants confidential rebates from pharmaceutical companies like CLINUVEL, and we do not pay any insurance company or agents in the world commissions, discounts or rebates,” said Dr Dr Wolgen.

According to Dr Wolgen NICE and NHS England wanted CLINUVEL to sign confidentiality on a rebate programme.

“This would mean these terms could then not be disclosed under this agreement,” he added.

“CLINUVEL wants to stop these practices used by government agencies, since we transparently disclose our uniform price across Europe and do not pay rebates via a second flow of cash going back to NICE / NHS.”

The British Porphyria Association (BPA), which is supporting Oran and his family, described patients with EPP as suffering from acute, unremitting pain and skin damage in areas exposed to sunlight.

“Symptoms translate into debilitating effects that make daily activities, including education, sport and personal relationships a huge challenge - leading to negative effects on physical health, relationships, financial stability and psychological and emotional wellbeing.”