Vertex clinches Trikafta and other cystic fibrosis drug reimbursement deal in England

Cystic fibrosis (CF) patients and their families in England can rest a little easier this week, thanks to an extended long-term reimbursement deal inked by Vertex Pharmaceuticals and the country’s National Health Service (NHS).

The accord comes after years of back-and-forth in the United Kingdom over the cost of Vertex’s life-saving—yet expensive—CF drugs.

Vertex announced the updated reimbursement deal with the NHS on Thursday, which ensures access to the drugmaker’s CF meds Trikafta (known as Kaftrio in the U.K.), Symkevi and Orkambi for all existing and future eligible cystic fibrosis patients in England.

It’s expected that similar deals will soon materialize in Wales, Scotland and Northern Ireland, according to the British Broadcasting Corporation (BBC).

The agreement comes after the U.K.’s drug cost gatekeeper, the National Institute for Health and Care Excellence (NICE), issued a positive recommendation for Vertex’s roster of CFTR modulators in June.

In November 2023, NICE acknowledged in draft guidance the effectiveness of Vertex’s CF cocktails, but the agency stopped short of dubbing the medicines cost-effective.

“Even when considering the condition’s severity … the most likely cost-effectiveness estimates for [Trikafta], [Symkevi] and [Orkambi] are above the range that NICE considers an acceptable use of NHS resources,” the government agency said at the time.

The situation mirrored a yearslong standoff between Vertex and the NHS over the pricing of Orkambi and other CF drugs that was resolved in 2019 with a four-year government reimbursement deal. Trikafta was added to the scheme in 2020.

At the time of NICE’s decision last fall, David Ramsden, CEO of the Cystic Fibrosis Trust, argued that Vertex, NICE and the NHS must “urgently work together to find a solution to make these treatments available for all those who could potentially benefit.”

Under the latest deal, patients will also receive access to any future license extensions on Vertex’s CF meds, the company explained in its release.

Moreover, Vertex, NICE and the NHS are working on a path towards “rapid access” for Vertex’s next investigational cocktail for CF, which combines vanzacaftor, tezecaftor and deutivacaftor, should the combo therapy pass muster with England’s drug regulator.

CF is a genetic condition in which a faulty protein affects the body’s cells, tissues and mucus- and sweat-producing glands. Patients with the disease make thick, sticky mucus that can build up and cause damage or infection to organs like the lungs and pancreas. The most serious and common complications of CF are respiratory in nature, including serious lung infections.

Vertex’s flagship drug Trikafta is a combination of elexacaftor, ivacaftor and tezacaftor. It works by helping defective CF transmembrane conductance regulator proteins work more effectively.

Vertex’s roster of CF medicines is currently “broadly available” in more than 60 countries, including Australia, France, Italy, Germany, the Republic of Ireland, the Netherlands, Spain and the U.S., the company explained.

Meanwhile, another pricing standoff could be brewing between NICE and Vertex on the drugmaker’s CRISPR Therapeutics-partnered gene therapy for sickle cell disease, Casgevy.

In draft guidance unveiled in March, the cost watchdog did not endorse Casgevy—which gained its U.K. approval in November 2023—but caveated that it was “exploring further data collection” on the therapy’s effectiveness and a potential commercial arrangement.