Vertex has presented NHS England with an “innovative long-term framework proposal” that hopes to secure access for all eligible patients to its current and future portfolio of cystic fibrosis (CF) medicines.
Specifics of the offer haven’t been revealed at this stage, but according to the firm the approach should ensure timely access to its medicines while providing budget certainty and value to the NHS.
Crucially, it “offers fair and equal access for CF patients to precision medicines that are tailored to specific genetic mutations,” a spokesman said.
The UK has the second highest number of CF patients in the world and Vertex says its medicines are the first – and only licensed medicines – to treat the underlying cause.
However, just 5 percent of patients in England can currently access its CF therapies routinely through the NHS, and yet it is widely available in several other European countries. A portfolio approach would “put an end to this inequity of access to Vertex medicines”, the firm noted.
The proposal follows “significant engagement with NICE, NHS England and Government Ministers over the past two years,” and a similar successful deal with the Republic of Ireland, which provides access to all patients for whom Kalydeco (ivacaftor) and Orkambi (lumacaftor/ivacaftor) are approved, and enables rapid access for new medicines and extensions to currently approved therapies.
“We share the CF community’s sense of urgency on access to new Vertex medicines and we are committed to working with the NHS to find a sustainable funding solution. That is why we have proposed to the Government a bold new portfolio approach that could make our medicines available to patients as soon as possible,” a company spokesperson said.
“We believe that our approach is a first for England, and a first for the UK. We have already demonstrated it works in other countries − and offers CF patients who could benefit, access to Orkambi. The proposed portfolio approach is for England but we continue to engage in meaningful discussions with the devolved nations.”