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Cystic Fibrosis Trust

Demonstration for life-saving drugs

The Cystic Fibrosis Trust held a demonstration on 7 March, the day of the Health and Social Care Committee's Inquiry into the availability of Orkambi and other life-saving medicines.

Watch the inquiry

Watch the inquiry

The inquiry into the availability of Orkambi and other cystic fibrosis drugs took place on 7 March 2019 and was broadcast on Parliament TV, where you can now watch it again.

You can also read a full transcript of the inquiry here.

Demonstration for access to life-saving drugs 

On 7 March 2019, the Health and Social Care Committee held a public hearing on the availability and pricing of Orkambi and other cystic fibrosis drugs manufactured by Vertex Pharmaceuticals. On that day, the Cystic Fibrosis Trust led a public demonstration outside the Houses of Parliament to highlight this Inquiry as part of our ongoing campaign with the CF community to make Orkambi and other life-saving drugs available to people who could benefit from them.

About the Inquiry

The Health and Social Care Committee Inquiry heard evidence from Vertex Pharmaceuticals, NICE and NHS England regarding the ongoing discussions around Orkambi and other live-saving drugs. After the Inquiry, which is the first of its kind to be held, evidence will be considered by the Committee, who will then make public recommendations to the negotiating parties on how to move forward.

Who was involved?

Health and Social Care Committee - appointed by the House of Commons to examine the policy, administration and expenditure of the Department of Health and Social Care and its associated bodies.

Vertex Pharmaceuticals – the pharmaceutical company that manufactures Orkambi, Kalydeco and Symkevi.

The National Institute for Health and Care Excellence (NICE) – a public body responsible for recommending medicines to the NHS.

NHS England – a public body that oversees the budget, planning, delivery and day-to-day operation of the NHS in England.


These are the 'witnesses' that were called during the inquiry:

  • A patient representative nominated by the Cystic Fibrosis Trust
  • A clinician representative nominated by the Cystic Fibrosis Trust
  • Professor Stephen Powis, National Medical Director, NHS England
  • John Stewart, National Director, Specialised Commissioning, NHS England
  • Sir Andrew Dillon, Chief Executive, NICE
  • Meindert Boysen, Director of the Centre for Health Technology Evaluation, NICE
  • Dr Jeff Leiden, Chairman, President and Chief Executive Officer, Vertex Pharmaceuticals
  • Stuart A. Arbuckle, Executive Vice President and Chief Commercial Officer, Vertex Pharmaceuticals

What will the outcome be?

While this Inquiry will not see a decision made about making Orkambi and other medicines available, we hope the committee’s findings will exert further pressure on the negotiating parties to end the deadlock and make life-saving drugs available at the earliest opportunity. Now that the Committee has heard all the evidence, they will make a number of public recommendations, suggesting how progress could be made. We do not know how long the Committee will take to form its recommendations, but we hope they will act quickly. While these recommendations are not legally binding, they will send a very clear message to the responsible parties as to how the Committee would like to see this issue resolved.

Stopping the Clock

Find out more about our campaign to ensure that people with CF across the UK can have access to innovative precision medicines.

Tell me more


Orkambi is the second precision medicine to be licensed in the UK for people with CF and treats the F508del mutation, which around 50% of people with CF have. It is not currently available on the NHS.

More about Orkambi

Other precision medicines

A double combination therapy and a triple combination therapy are currently being trialed to see how they could help to treat up to 90% of people with cystic fibrosis. Recent results are positive.

More about other treatments