History may repeat for Vertex Pharmaceuticals in the U.K., where a new decision by the country’s cost-effectiveness gatekeeper threatens to spark another dispute over the cost of lifesaving cystic fibrosis (CF) drugs.
Last week, the National Institute for Health and Care Excellence (NICE) unveiled draft guidance that acknowledged the effectiveness of Vertex’s CF cocktails Trikafta (known as Kaftrio in the U.K.), Symkevi and Orkambi. But the agency stopped short of labeling the medicines cost-effective.
The decision is not a final recommendation. NICE pointed out that its guidance shouldn’t affect treatment with Vertex’s drugs started in the National Health Service (NHS) before the recommendation’s publication.
NICE will now go through a four-week consultation period followed by a second committee meeting to discuss the responses it receives, the U.K.’s Cystic Fibrosis Trust said in a separate statement responding to NICE’s appraisal. During this time, the public and organizations like the trust will be able to chime in and advocate for people with CF and their families.
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.
NICE admits that clinical data suggest Trikafta improves lung function, growth and weight gain while reducing the number of lung infections patients endure compared to standard CF treatment. The same goes for Vertex's Symkevi and Orkambi, according to the cost watchdog.
That said, “[e]ven 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.
Vertex did not immediately respond to Fierce Pharma’s request for comment on NICE’s draft guidance.
The situation resembles a yearslong standoff between Vertex and the NHS over pricing of Orkambi and other CF drugs—an ordeal that was ultimately resolved in 2019 with a four-year government reimbursement deal. Trikafta was added to the deal in 2020.
Because of the stalemate, CF patients in the U.K. were previously unable to access Vertex’s medications despite Orkambi’s EU approval in 2015. But, early in 2019, the situation began to thaw, with Vertex’s then-CEO Jeffrey Leiden agreeing to attend a hearing on Orkambi alongside officials from NHS England and NICE.
In March 2019, the situation came to a head when Vertex revealed it was forced to destroy nearly 8,000 Orkambi packs that had expired in 2018.
Meanwhile, NICE’s latest appraisal hasn’t received the warmest reception across the pond. The same day the agency released its draft guidance, David Ramsden, CEO of the Cystic Fibrosis Trust, said the decision “creates uncertainty for those not yet on treatment.” He 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.”
The NHS said it’s committed to maintaining patient access to Vertex’s therapies, with the aim to work through the appraisal process in a way that’s both “fair to patients, and fair to taxpayers.”
The cost of Vertex’s CF therapies has proved a point of contention elsewhere, too. In February, a coalition of CF patients and their families, helmed by Vertex Save Us and U.K.-based patient advocacy group Just Treatment, petitioned governments in South Africa, Brazil, India and Ukraine to either revoke or suspend Vertex patents surrounding Trikafta.
The groups claimed Vertex’s therapies were out of reach for patients in low- and middle-income countries as well as some high-income countries. The initiative sought to secure affordable generic access to what it branded “extremely expensive medicines.”