UK families form CF Buyers Club to import generic Orkambi

In the UK, parents of children with cystic fibrosis have formed a buyers club in a bid to access a lower-cost version of Orkambi, the drug at the centre of a three-and-a-half-year pricing standoff between its manufacturer Vertex and NHS England.

The club has been launched by more than 100 CF patients, parents and campaigners who are increasingly frustrated at waiting for an end to the widely publicised pricing deadlock over the cystic fibrosis drug that is already accessible to patients in other health systems across the globe, including Australia.

In Australia the reported $200,000+/year cost of Orkambi has been subsidised on the PBS since October 2018 for children over the age of 6. An application for subsidy in children aged 2-5 is about to be considered by the Pharmaceutical Benefits Advisory Committee (PBAC).

In the UK, where Orkambi is not available on the NHS, families estimate that  if they pool their collective purchasing power they will be able to purchase a generic version of lumacaftor/ivacaftor, made by Argentinean pharmaceutical company Gador and called Lucaftor, for a reported cost between £18,000 to £23,000 (A$33,000-$42,000) per patient per year.

The club notes that while this figure will still be out of reach for many families, it is dramatically lower than Vertex’s current UK price of £104,000 (A$188,000) per patient per year.

The families argue that the government could make the generic version available on the NHS by issuing a Crown use license or by conducting a large scale clinical trial which would allow the government to use a generic product through the research exemption in UK law.

Despite being in its infancy, the club has grabbed media attention and gained strong support from Seema Kennedy, the new Minister for Public Health and Primary Care, who earlier this month told parliament: “Unless Vertex changes their approach and behaves responsibly, then I have a moral obligation to look at these other options”.

The CF Buyers Club has asked for a meeting with Health Secretary Matt Hancock, Ms Kennedy and NHS England CEO Simon Stevens to “urgently discuss the next steps”, accepting that “there are complications to be overcome in undertaking any of these approaches”.

Are there valid concerns around safety and efficacy?

According to respiratory physician and leading CF expert Professor Stuart Elborn, from Queen’s University in Belfast, CF clinicians are generally comfortable using Orkambi, having had access to the drug for people with CF and low lung function for several years. There is also good data available from published studies on lumacaftor/ivacaftor.

“But I think we all have concerns as to whether the generic drug is of similar quality and will have the same efficacy and side effect profile of Vertex Orkambi,” he said, noting that the import of drugs from outside the EU is verified to “ensure compliance with the highest safety standards”.

According to the CF Buyer’s Club website, the group has “sought to take all reasonable steps to ensure the safety and efficacy of the generic products we have identified”.

“We [have] had independent tests to confirm the active pharmaceutical ingredients are directly comparable,” they state.

A looming challenge for CF Centres

There is little doubt that patients gaining access to Lucaftor will impact respiratory physicians and CF teams, but exactly how remains unclear. Some of the important but finer details, including how patients can access a private prescription are yet to be worked out.

The club says they are currently working to reach an agreement with a doctor who can issue prescriptions for all prospective Buyer’s Club members, but the details are yet to be confirmed.

And then there’s the issue of who should manage any ongoing care once the patient has commenced treatment.

According to Professor Elborn, further follow-up should be managed by the prescribing doctor, and this may or may not be the CF Centre doctor.

Nevertheless, he told the limbic it would be very important for any patient importing the generic drug to coordinate with their CF team as lumacaftor/ivacaftor can be associated with side effects, particularly shortness of breath.

“The introduction of lumacaftor/ivacaftor has to be carefully managed and will need to be done in close coordination with the CF clinical teams,” he said.

However, he noted that there were concerns that this approach could be challenging as follow-up tests to monitor treatment would need to be paid for as a private patient, yet all care through CF centres are provided on the NHS.

“There may also be ethical concerns for CF doctors about this,” Professor Elborn added.

Buyer’s clubs for hepatitis C treatment, sofosbuvir, and the HIV preventative medicine, PrEP, are already operating successfully in the UK.

And in Australia a similar Hep C buyer’s club was set up to import affordable generic versions of direct acting antivirals for hepatitis C.


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