Vertex Pharmaceuticals in the United Kingdom is facing a situation that echoes past disputes over the pricing of essential cystic fibrosis (CF) medications, potentially repeating history. NICE published preliminary recommendations that recognized the efficacy of Vertex’s combination cystic fibrosis drug regimens, including Trikafta (referred to as Kaftrio in the United Kingdom), Symkevi, and Orkambi.
It’s crucial to note that this decision is not a final recommendation. NICE clarified that its guidance should not impact the ongoing treatment of CF patients with Vertex’s drugs within the National Health Service (NHS) initiated before the guidance’s publication.
NICE will now proceed with a four-week consultation period, followed by a discussion of its responses in grave detail. During this time, the public and organizations like the Cystic Fibrosis Trust in the UK will have the opportunity to voice their opinions and advocate for individuals with CF and their families.
Cystic fibrosis is a hereditary ailment marked by a malfunctioning protein that impacts the cells, tissues, mucus production, and sweat glands in the body. Patients with CF produce thick, sticky mucus that can accumulate and lead to damage in organs.
The most common and severe complications of CF are respiratory, often involving serious lung infections. Trikafta, Vertex’s primary medication, is a blend of elexacaftor, ivacaftor, and tezacaftor, designed to enhance the functioning of malfunctioning CF transmembrane conductance regulator proteins. NICE recognizes that clinical evidence indicates Trikafta’s potential to enhance lung function, promote growth, and facilitate weight gain, while also reducing the incidence of lung infections when compared to conventional CF treatments.
However, NICE points out that even when considering the severity of the condition, the estimated cost-effectiveness of Trikafta, Symkevi, and Orkambi is likely above what NICE deems an acceptable use of NHS resources.
Vertex has not yet responded to NICE’s draft guidance, and this situation harkens back to the prolonged standoff between Vertex and the NHS over the pricing of Orkambi and other CF drugs. This dispute was eventually resolved in 2019 through a four-year government reimbursement deal, with Trikafta being added to the agreement in 2020.
The previous stalemate had left CF patients in the UK unable to access Vertex’s medications, even after Orkambi received EU approval in 2015. However, in early 2019, progress began when Vertex’s CEO at the time, Jeffrey Leiden, agreed to attend a hearing on Orkambi alongside NHS England and NICE officials. In March 2019, the situation intensified when Vertex had to dispose of nearly 8,000 expired Orkambi packs from 2018.
