Due to the known success of buyers’ clubs in gaining emergency access to HIV/AIDS and Hepatitis C medicines, parents of Cystic Fibrosis (CF) patients have decided to follow suit and take matters into their own hands to access to a generic version of Orkambi (ivacaftor+lumacaftor), a life-saving medicine for the condition.

The branded version, which is patented in the UK, has been deemed too expensive to be provided via the National Health Service.

Since the drug was first approved in the European Union (EU) over three years ago, Orkambi’s developer Vertex and the NHS have been in negotiations over its price. However, no progress has been made and the stalemate continues despite parliamentary and patient advocacy.

“Patients have had enough of waiting, and of watching their health or their loved one’s heath irreversibly decline in the meantime,” said Emily Birchall, a member of the CF Buyers’ Club working group.

The club has found a way to bypass the NHS-Vertex stalemate by buying a generic version of the drug from Gador, a company based in Argentina, where Vertex has no patent for Orkambi. It is possible for patients to important this drug into the UK legally, as long as it will only be used for personal use.

Why the disagreement? NHS-Vertex Orkambi negotiations

Orkambi is a precision medication with a novel mechanism of action to treat the underlying genetic cause of the most common types of the condition: patients with two copies of the F508del mutation.

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Despite acknowledging the clinical effectiveness of the drug, in 2016 the NHS’ pricing regulator National Institute for Health and Care Excellence (NICE) concluded the drug’s benefits do not justify the £105,000 annual per patient price tag, which Vertex originally asked for. The usual maximum price that NICE would accept is £30,000 per patient per year.

As a result, Vertex and NHS England entered negotiations to attempt to agree an appropriate, mutually acceptable price for Orkambi and the company’s other, innovative CF drug, Kalydeco.

However, talks reached an impasse in July 2018 with Vertex claiming it made “the best offer in the world” – the exact price is protected by a non-disclosure agreement – and NHS England stating the pricing was still “unsupportable.” At the time Secretary of State for Health and Social Care Matt Hancock, called on Vertex to disclose their final offer to no avail.

As part of a House of Commons Health and Social Care committee inquiry into the issue, NHS England publicised that it offered Vertex £500m for five years of NHS use to its entire CF portfolio, including those currently in development. After which the company would be able to submit alternative prices to NICE for new medications based on relevant evidence.

During a committee hearing, NHS England national director of specialised commissioning John Stewart noted that Vertex’s price is miles away from this £500m offer.

Vertex CEO Dr Jeffrey Leiden defended the company’s position at the same hearing by emphasising its altruism in providing Orkambi on compassionate grounds to certain patients, and explaining: “The English offer is around £10,000 per patient per year…If we accept that offer, that has been made public by the NHS, of course every other country will want that same offer.

“If we were to accede to that [in all the countries where Orkambi is available], our total CF revenues would be £850m a year. Unfortunately, that would not allow us to develop the next set of CF medicines, because we are spending $1bn just on R&D.”

Practicalities of importing a generic from abroad

Frustrated by the lack of progress, parents of CF patients decided to set up a Buyer’s Club, “allowing patients to buy the drug themselves if they cannot wait any longer.”

After identifying Gador as a possible option, the club asked experts to independently confirm that the generic manufactured by the company was bioequivalent to Vertex’s branded Orkambi, and therefore equally safe and efficacious for patients.

As the law dictates that patients can only import a three-month supply of medicines into the UK for personal use, although the price is agreed collectively, patients will need to individually buy the drug from Gador.

This can only be done once they have a prescription for the medicine from a private practice; the buyers’ club is seeking to reach an agreement with a qualified doctor to do it for the entire membership.

Taking Orkambi will need to be coordinated with and supervised by the patient’s NHS medical team, “as the drug is not suitable for all patients and reactions to it need to be carefully monitored”; the buyers’ club notes that many NHS CF teams have confirmed their willingness to cooperate.

Gador’s generic ivacaftor and lumacaftor is currently priced at £23,000 per year, however, as Birchall explains “the generic manufacturer has promised to lower the price to £18,000 per year if we can get 500 patients together”.

Improved access, but only for the richest patients

Although the buyers’ club will improve access to Orkambi in the UK and “plug the gap left by failed negotiations,” campaigning group Just Treatment organiser Elizabeth Baines noted that, “these much cheaper generic medicines will remain unaffordable for many.”

For this reason, Birchall emphasised that the CF buyers’ club see this only as a temporary solution; “it’s not right that only the richest patients will be able to self-fund this, and the government need to find a way to ensure all patients can access the drug who need it.”

They are supporting Just Treatment’s campaign for the UK Ggovernment to issue a Crown use license, which will remove Vertex’s patent in the UK on public interest grounds, which Baines explains would “allow cheaper, generic drugs to be made available on the NHS.”

“The buyer’s club has proven that a safe and effective generic supply already exists – if the government took the step of overturning Vertex’s patent, the NHS could be supplying generic Orkambi to patients within months rather than years,” according to Baines.

Not only has the buyers’ club identified a generic that the NHS could use after removing Vertex’s patent, Birchall explains that the price of Gador’s generic is likely to be lowered further if it is bulk purchased by the NHS, making it even more cost-effective.

The buyers’ club and Just Treatment are optimistic that the creation of the club could genuinely lead to the universal access to Orkambi because, in the words of Birchall, “The Department of Health and Social Care recently said that ‘they note the suggestion to put this drug [the generic] on a clinical trial in the UK’ and that ‘in the absence of a deal [with Vertex] ministers have asked NHS England and NHS Improvement to continue to explore all options to ensure patients can access treatments as soon as possible.’”

Baines concludes: “Now we need the government to confirm how and when it will act on its promises. We need a solution that will ensure that every cystic fibrosis patient who needs Orkambi can get it, and in the long term that means making it available on the NHS at a fair price.

“Patients have already waited long enough and Vertex have demonstrated their refusal to drop the price to a reasonable level. It’s time for the government to act.”