Patient activists in the UK have called for government support to work round the deadlock over Vertex’s cystic fibrosis drug.
The Cystic Fibrosis Buyers’ Club has asked health secretary Matt Hancock to help patients circumvent Vertex and find alternative ways to gain access to Orkambi – including the import of generic versions of the drug from Argentina.
It is three and half years since Orkambi was first approved across Europe, but a long-running row between Vertex and NICE and NHS England means most eligible patients in the UK aren’t receiving the treatment.
Vertex’s UK list price for Orkambi is £104,000 ($136,000) for a year’s treatment, but NHS England’s attempts to negotiate a much lower price have hit long-term refusal from the US pharma company.
Orkambi treats those with the F508del mutation, which represents around 50% of the CF population in the UK.
Now families of children with CF and adult patients and activists have set up the CF Buyers’ Club, and have called on the government to implement its three proposals: invoke a (compulsory licensing) crown use licence, launch a large scale clinical trial and provide direct support for individuals using the buyers’ club.
These would be aggressive moves against Vertex, with a ‘crown use’ licence representing an overriding of the company’s UK patent, and the importing plan similarly circumventing the company’s intellectual property.
The government has ruled out a crown use licence until recently – not least because of the signal it would send out regarding the security of patents in the UK – but in a House of Commons debate last week a health minister said it may have to be considered.
Health Minister Seema Kennedy said it would be an extreme measure, but that she had a ‘moral obligation’ to consider such measures if Vertex cannot agree a deal with NHS England soon. She repeated NHS England’s remarks from earlier this year, calling the US pharma company ‘an extreme outlier extreme in both pricing and behaviour.”
A letter penned by the CF Buyer’s Club in response to the debate has called for urgent action. The letter, signed by ‘110 Parents, Patients and Campaigners’ calls for the government to back its three point plan.
The Buyers’ Club has already established contact with Gador, a generic manufacturer in Argentina (where Orkambi is not patent protected), which will supply their copy of the drug for £23,000 per patient per year. It says it will lower the price of its version, Lucaftor, to £18,000 if the club can get at least 500 members.
These prices are a fraction of Vertex’s list price, but parents will have to pay for the treatment privately out of their own pockets.
The group has enlisted the help of Dutch medicines expert Dr Wilbert Bannenberg to help ensure the safety and efficacy of Gador’s medicine.
UK regulations allow patients or their parents to import non-UK-registered medicines for personal use, provided they have a valid prescription, and takes the medication under regular supervision of a physician.
Meanwhile, other activist groups plan to keep the pressure up on politicians as well as Vertex. A ‘Lay down for CF’ demonstration will be taking place in Parliament Square on 11 July to protest about the continuing lack of access to Orkambi.