Intended for healthcare professionals


NHS England explores funding options for cystic fibrosis drug

BMJ 2019; 366 doi: (Published 17 September 2019) Cite this as: BMJ 2019;366:l5600
  1. Deborah Cohen
  1. BBC Newsnight

NHS England is “committed to exploring and developing alternative routes” to enable people with cystic fibrosis to access Orkambi (ivacaftor-lumacaftor) on the NHS if Vertex, the US company that markets it, does not price the drug “fairly and responsibly,” a spokesperson has said.

The statement comes after the Scottish government agreed a deal with Vertex to provide around 400 people with cystic fibrosis access to Orkambi and Symkevi (ivacaftor-tezacaftor).1

There are several options open to the NHS if it fails to reach an agreement with Vertex.

These include suspending the patent and proceeding with a crown use licence—meaning that another company can manufacture the drug. Or NHS England could avoid the patent by using a research exemption to import an existing generic version of Orkambi to use in a clinical trial.

The UK has a history of using a research exemption to facilitate access to generic versions of patented drugs for NHS patients.

Meanwhile UK patients and their families outside Scotland have formed a buyers’ club to import a generic version of Orkambi from Gador, an Argentinian company, for their personal use. This tactic is being used by thousands of UK citizens to access generic drugs.

Andrew Hill, a senior visiting research fellow at the department of molecular and clinical pharmacology at Liverpool University, has worked with several buyers’ clubs. These include around 10 000 people importing pre-exposure prophylaxis drugs to prevent HIV infection through a large scale clinical trial and a smaller group importing hepatitis C drugs.

“UK buyers’ clubs were all set up because the NHS could not afford to pay the high prices demanded by pharmaceutical companies. So patients had the option of either importing these drugs legally for their personal use or remaining untreated with all the clinical consequences this involved,” Hill said.

The generic version of Orkambi is called Lucaftor, and patients can legally import three months’ supply under current law. Gador is charging around £24 000 (€27 000; $30 000) a year, which compares with the list price of £104 000.

A group of patients and parents picked up their first batch in Buenos Aires last week, on the same day the Scottish deal was announced.

Robert Long, a parent of a child with cystic fibrosis and one of the organisers of the buyers’ club, said, “I’m delighted for the people of Scotland that they have managed to do a deal with Vertex. But that doesn’t mean it’s going to happen for the rest of the UK, and we’ve got to fight to get access for our children. So whether it’s with Vertex or a generic—it doesn’t matter to us.”

The BBC programme Newsnight, which has been investigating the matter,2 understands that some NHS clinicians are nervous about the situation. The buyers’ club obtained a private prescription for Lucaftor after consultation with a private doctor.

However, NHS England has sought to reassure doctors. “We see no reason why clinicians working in the NHS should not provide ongoing clinical care for cystic fibrosis patients who may have been prescribed a drug privately,” said a spokesperson.

Hill said that there are things UK doctors need to be aware of, such as how the drugs are prescribed.

“UK doctors may need to supply a letter to confirm that the patient is eligible to be treated with an imported drug, and that they are likely to benefit from taking it. Prescriptions for the drugs can be organised in other countries,” he said. “UK doctors would need a plan to monitor patients while they are taking imported generic treatments. For some diseases (for example hepatitis C), this monitoring could be the same as used for routine patient management.”

In a statement, Vertex said that it understood and shared patients’ frustration and the need to resolve the access situation in England. It said it had reached agreements with 17 countries around the world and remained in talks with NHS England. The price of its drugs were based on the difference they bring to patients’ lives and the significant cost of research and development, said the company.


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