Jeff Leiden, M.D., Ph. D.

President, CEO, and Chairman

The Rt Hon Theresa May MP

Prime Minister & First Lord of the Treasury
10 Downing Street
London
SW1A 2AA
6 July 2018

Dear Prime Minister,

Access to life-changing medicines for the treatment of cystic fibrosis

I am writing to request your urgent intervention to ensure patient access to the portfolio of
Vertex medicines for the treatment of cystic fibrosis in England.

This matter is a test case for the Government’s delivery of accelerated access to innovative
medicines in the NHS.

Cystic fibrosis (CF) is a rare, life-threatening genetic disease that disproportionally impacts
the UK with the second highest patient population in the world – with 12% of the CF
population compared to 1% of the world’s population. By investing years of research and
billions of dollars, both in the UK and US, we have discovered and developed the first
medicines to treat the underlying cause of cystic fibrosis, which are already available to tens
of thousands of patients in over 30 countries around the world. We continue to invest with
the ambition to create more medicines that may ultimately cure the disease.

As you are aware, Vertex has been negotiating with NHS England on access to our portfolio
of all existing and future licensed medicines including Orkambi® (lumacaftor/ivacaftor),
which has been in process for three years. At Prime Minister’s Questions on 16 May, you
made clear that you wanted to see a swift resolution to these negotiations. I am sorry to
inform you we have made no progress.

In the latest negotiation meeting on 4 July, Vertex made the most innovative and best offer
in the world to NHS England – covering all patients, for all of our medicines today and
tomorrow – at a price that is reflective of the exceptionally high prevalence of CF in the UK.

50 Northern Avenue, Boston, MA, 02210-1862, Tel: 617-341-6100, Jeff_Leiden@vrtx.com

Jeff Leiden, M.D., Ph. D.
President, CEO, and Chairman

At a Westminster Hall debate on 19 March, parliamentarians from across the political

spectrum called for flexibility on the part of NHS England in how they appraise and value
innovative new medicines. Regrettably, however, NHS England has shown no flexibility by
adhering to arbitrary value thresholds, which were so widely challenged and criticized in the
debate. They are placing a lower value on the life of a CF patient than other countries
around the world – for example, patients have access to Orkambi in Ireland, Sweden,
Germany, Austria, Italy, Denmark and the Netherlands. In some of these countries, people
have had access to the medicines for nearly three years.

We welcome your commitment to fostering the UK’s global leadership in science and
innovation and the signals that Ministers have given to support the adoption and diffusion of
innovative medicines in the NHS, including through the implementation of the Life Sciences
Industrial Strategy and Sector Deal. We as a company have a significant presence in
England and have done so for over 20 years. Vertex is heavily committed to the UK with its
international HQ in London, an R&D site near Oxford and runs many clinical trials in the
NHS. Since 2006, Vertex has invested over £1.2bn in the UK and now employs 250 people,
of whom over 110 are scientists. In the light of our commitment to science in the UK, and
your commitment to the Life Sciences, we are questioning this ecosystem - as it is unable to
value life changing medicines for the patients that need them.

For the UK to develop its science and innovation ecosystem post-Brexit, these policy
statements and plans must translate to improved patient access to innovative therapies,
such as the new generation of precision medicines which work on different genotypes of
rare diseases. This requires appraisal processes to keep pace with the science; current
processes have significant limitations in how they capture and value the full medical,
societal, economic and innovation benefits of such precision medicines. Without this, any
future biotech investment in the UK is at significant risk.

We believe that a more flexible approach on the part of NHS England is urgently needed
and will yield significant benefits in transforming patients’ lives, as well as incentivizing
future research and innovation in the UK.

We are confident our proposal to NHS England would achieve rapid patient access, budget
certainty for the NHS, and fair reward for innovation. We have provided the most innovative
offer in the world to the NHS, yet have seen no receptivity from NHS England. In one of the
most prosperous countries in the world, NHS England’s level of interest in our offer
represents a lack of commitment to children and young people with this devastating
50 Northern Avenue, Boston, MA, 02210-1862, Tel: 617-341-6100, Jeff_Leiden@vrtx.com
Jeff Leiden, M.D., Ph. D.
President, CEO, and Chairman

disease. We respectfully request, therefore, that you urge NHS England to properly value
and realise the potential of our portfolio of cystic fibrosis medicines.

It would be disappointing in the extreme that, as the NHS turns 70, it is found to be shutting
the door to a new era of precision medicines that stand to revolutionize healthcare.

For people with cystic fibrosis, every day counts. I stand ready to meet with you to discuss
how we can work together on behalf of people with cystic fibrosis to fulfill the Government’s
stated ambition for NHS patients to be “among the first in the world to get life-changing
treatments”.1

This letter will be made available to the Cystic Fibrosis Trust and Members of Parliament.

Yours sincerely,

Jeff Leiden, MD, PhD

Chairman, CEO and President
Vertex Pharmaceuticals

Cc Rt Hon Jeremy Hunt MP, Secretary of State for Health and Social Care
Steve Brine MP, Parliamentary Under Secretary of State for Public Health and Primary Care
Lord O’Shaughnessy, Parliamentary Under Secretary of State for Health (Lords)

1
Department of Health/Department for Business, Energy and Industrial Strategy: Making a reality of the Accelerated Access Review, Nov. 2017
50 Northern Avenue, Boston, MA, 02210-1862, Tel: 617-341-6100, Jeff_Leiden@vrtx.com