Vertex Pharmaceuticals media statement NHS E reimbursement meeting July 4

In the latest meeting, we made the best offer in the world to NHS England, reflecting our ultimate goal to provide access to all of our medicines for all cystic fibrosis (CF) patients as soon as possible. It includes KALYDECO® (ivacaftor), ORKAMBI® (lumacaftor/ivacaftor), tezacaftor/ivacaftor and our future medicines still in development, which we hope may treat as many as 90 percent of all patients. Our offer also provides budget certainty to NHS England and assurance that patients will have rapid access to advances in medicines soon to come, just like agreements made in other countries.

We find it outrageous that NHS England does not see a path forward to provide access for thousands of children and young people to the only medicines that treat the underlying cause of CF. We find it unconscionable that the Government is unable to value the importance of these CF medicines and provide access. They are placing a lower value on the life of a CF patient than other countries around the world.

We call on the Government to intervene to ensure that CF patients in England have access to the medicines that exist now as well as the advances soon to come. We stand ready to meet any time, any place, to ensure patient access to these lifechanging and transformative medicines. Patients do not have time to wait and we share their urgency.

About cystic fibrosis
• Over 10,000 people in the UK have CF1 − the second highest number in the world.1,2
• CF is a debilitating, life-shortening inherited condition that causes progressive damage to organs across the body from birth.2
• Currently, there is no cure for CF and half of people in the UK with CF die before they are 31.3
• The daily impact of treatment is significant. It can take up to four or more hours involving, nebulisers, physiotherapy and up to 70 tablets a day.4
• CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.5

About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases.

In addition to clinical development programmes in CF, Vertex has more than a dozen ongoing research programmes focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., USA, Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognised as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row.

1 CF Trust. FAQs. Available at: https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/faqs. [Last accessed July 2018]
2 NHS Choices. Cystic Fibrosis overview. https://www.nhs.uk/conditions/cystic-fibrosis/. [Last accessed July 2018].
3 Cystic Fibrosis Trust. UK Cystic Fibrosis Registry 2016 Annual Data Report 2016.
https://www.cysticfibrosis.org.uk/registryreports. [Last accessed July 2018]
4 CF Trust. Transition and adherence. Available at: https://www.cysticfibrosis.org.uk/the-work-we-do [Last accessed July 2018]
5 BLF CF Statistics. Available at: https://statistics.blf.org.uk/cystic-fibrosis. [Last accessed July 2018]

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