Government voices concerns about using legal powers for affordable access to cystic fibrosis drug

The health minister Steve Brine has said that although the government had considered the option, using a Crown use licence would be a complicated route to ensuring patient access to the cystic fibrosis drug Orkambi.

steve brine

The government has said that using its legal power to “break the monopoly” held by drug company Vertex Pharmaceuticals over its combination cystic fibrosis treatment Orkambi would not represent “a quick solution”.

Orkambi, which is a lumacaftor/ivacaftor therapy, currently costs £104,000 per patient per year — a cost which the National Institute for Health and Care Excellence (NICE) said in 2016 is
not justified by the benefits of the treatment.

Vertex later rejected NHS England’s offer of £500m over five years to license the drug in the UK, with the NHS describing the offer in July 2018 as the “largest ever financial commitment” in its 70-year history.

As a result, campaigners have called for the government to enact a Crown use licence that would give another company permission to make and sell an exact high-quality, generic version of Orkambi to the NHS.

Just Treatment, a campaign group demanding fair access to medicines, launched a petition demanding the enactment of a Crown use licence, claiming that doing so could see the drug’s price to drop from £104,000 per patient per year to less than £5,000, saving the NHS nearly £4bn over ten years.

However, speaking in Parliament on 4 February 2019, health minister Steve Brine said that although the government had “considered” the option, using a Crown use licence is a complicated process and “would not represent a quick solution to ensuring patient access” to lumacaftor/ivacaftor.

“Crown use [licensing] could have the effect of putting patients at a disadvantage, jeopardising access to future medicines and potentially setting a precedent of issuing further licences at very high cost,” he said and maintained that Vertex should instead take up the “very generous offer” made by NHS England in 2018.

He said: “That offer will improve the lives of eligible cystic fibrosis patients and their families. For all the reasons I have set out, it is far and away the quickest and simplest way to resolve this matter.”

Brine added that the government “wholeheartedly” shared the view that patients should benefit from Orkambi.

David Ramsden, chief executive of the Cystic Fibrosis Trust, said it was “pleased to see further support” from MPs on accessing Orkambi and future cystic fibrosis drugs through the NHS.

He said: “However, the cystic fibrosis community requires support to turn into concrete action to end the wait.

“If people with cystic fibrosis and their families cannot see genuine efforts to make progress then it is inevitable they will call for drastic action to be taken.”

In November 2018, the House of Commons Health and Social Care Select Committee announced an inquiry into the availability of lumacaftor/ivacaftor on the NHS.

The committee requested technical documents from NHS England, Vertex, and NICE and also asked for information about the negotiations, including formal offers made by Vertex to NHS England and NICE and the responses to those offers from NHS England.

The committee will also be considering the experiences and views from the general public and will be holding a public hearing in March 2019.

  • This story was amended on 11 February 2019 to highlight that the government has not explicitly ruled out using a Crown use licence
Last updated
The Pharmaceutical Journal, Government voices concerns about using legal powers for affordable access to cystic fibrosis drug;Online:DOI:10.1211/PJ.2019.20206110

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