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Editor of Pharmaceutical Technology Europe
After years of discussions around pricing, UK governmental bodies and Vertex have finally reached an agreement on cystic fibrosis treatments.
Editor’s Note: This article was published in Pharmaceutical Technology Europe’s November 2019 print issue.
In recent news, it was announced that after several years of clashing over pricing, National Health Service (NHS) England and Vertex have finally struck a deal, which will give patients in England access to treatments for cystic fibrosis, Orkambi, Symkevi, and Kalydeco (1). The definitive agreement covers three medicines from Vertex that are licensed in the United Kingdom for the treatment of cystic fibrosis.
For cystic fibrosis sufferers in the UK, a country that has the second highest prevalence of cystic fibrosis in the world, it has been a four-year long wait to gain access to Vertex’s Orkambi treatment, which gained European approval in 2015 (2). Orkambi (lumacaftor/ivacaftor) is the first medicine to treat the underlying causes of cystic fibrosis with certain mutations rather than the disease symptoms and complications.
The back and forth that occurred between Vertex and NHS England is somewhat unusual in that both sides were in a stalemate for a long period of time, using the disagreement over pricing as a defence of one position or the other. For Vertex, which had gained a deal for its Kalydeco treatment worth £400 million over a five-year period in 2013, the pushback from NHS England and National Institute for Health and Care Excellence (NICE) over pricing was not necessarily anticipated. The company refused the pricing offer of £500 million for five-years for Orkambi and other drugs in Vertex’s pipeline-the largest expenditure ever offered by the NHS-which was made public by NHS England, despite all other discussions being confidential. For NHS England, the pricing being demanded by Vertex for Orkambi was deemed to be unaffordable.
Questions over how treatments are assessed, namely the single technology appraisal system of NICE, were raised as a result of rare disease treatment prices being specified as ‘unaffordable’. In Vertex’s case, the company specified that special considerations to patient population sizes should be factored in when performing appraisals (3).
With Vertex and NHS England at an impasse, tensions were frayed and inflammatory remarks and bravado on both sides ensued. As campaigners persisted in their efforts to gain access to Vertex’s treatments, the UK government was called upon to break the deadlock on pricing, even with some calls for the government to invoke ‘Crown Use’, which would have enabled third parties to access intellectual property of Vertex for the creation of generic versions of the medicine, Orkambi (4).
The potential ramifications of Crown Use were not lost on Vertex, which announced its reaction in a statement, according to the BBC (5). “To invoke Crown Use and provide third parties access to Vertex’s intellectual property would seriously undermine our ability to achieve these goals and would significantly weaken incentives for future innovation,” the statement read.
An agreement has now finally been achieved, however, and despite exact pricing for the treatment being unknown publically, it has been hypothesized that it is an amount ‘significantly less than the sum originally asked for’ (5). As a result of the agreement, an important and vast market has ultimately now been penetrated by Vertex, adding to markets in Spain, Australia, and Scotland.
“The UK has the second highest prevalence of cystic fibrosis of any country in the world, so today is an important and long hoped for moment for children and adults living with cystic fibrosis,” said Simon Stevens, NHS chief executive (1). “That fact also means that any drug company wanting to succeed commercially in this field needs to work constructively with the NHS. I’m pleased that Vertex has now agreed a deal that is good for our patients and fair to British taxpayers.”
1. NHS England, “NHS England Concludes Wide-Ranging Deal for Cystic Fibrosis Drugs,” England.nhs.uk, 24 Oct. 2019.
2. Vertex, “Vertex Receives EU Approval for Orkambi (lumacaftor/ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Two Copies of the F508del Mutation,” vrtx.com, Press Release, 20 Nov. 2015.
3. Parliament UK, “Documents from Vertex Pharmaceuticals,” parliament.uk, 30 Nov. 2018.
4. Bill Wiggin, “Bill Wiggin MP Calls for Crown Use License Debate,” billwiggin.wordpress.com, Media Release, 31 Jan. 2019.
5. Nick Triggle, “Cystic Fibrosis Drug Given Green Light in England,” bbc.co.uk, 24 Oct. 2019.
Pharmaceutical Technology Europe
Vol. 31, No. 11
When referring to this article, please cite it as F. Thomas, “Striking a Landmark Deal,” Pharmaceutical Technology Europe 31 (11) 2019.