Patient voice secures ‘yes’ for rare disease drug in Wales

19 June 2018

Patient voices have been victorious in securing the approval of a new drug to treat pulmonary hypertension (PH) in Wales.

The All Wales Medicines Strategy Group (AWMSG) announced last week (13 June) that Selexipag has been approved for use by the NHS. The decision follows evidence of the symptom burden of PH that was submitted by patient organisation the Pulmonary Hypertension Association (PHA UK) in the form of its Living with PH survey.

Pulmonary hypertension is a serious, life-limiting disease that affects just 7,000 people in the UK. Symptoms typically involve breathlessness, fatigue, black-outs and swelling around the ankles, arms and stomach. PH affects the ability to carry out basic tasks and get around.

The announcement to approve Selexipag in Wales comes just a few weeks after the Scottish Medicines Consortium (SMC) overturned its initial decision to deny the drug to patients in Scotland. A decision by NHS England is expected in the coming weeks.

Selexipag, also known as Uptravi, helps to relax and widen the pulmonary arteries, relieving symptoms of PH and slowing down progression of the disease. It is taken as a tablet, providing an alternative for the first time to drugs usually only available intravenously or by inhalation.

The PHA UK has campaigned hard and in collaboration to secure a positive outcome in Wales, and the results of the Living with PH survey provided key evidence. The AWMSG said it delivered one of the strongest patient organisation responses it had ever received.

Iain Armstrong, chair of the PHA UK, said: “This is absolutely the right decision by Wales and shows once again just how powerful the patient voice can be. We will be writing to the AWMSG to thank them for listening to us, during what is a new process for them. They should be congratulated for being so engaged with the patient population.”

Katie Annett, 27, lives in Ebbw Vale in South Wales. She was diagnosed with PH nine years ago, when pregnant with her daughter.

She said: “I am over the moon that Selexipag has been approved for patients in Wales. Our voices have been heard when deciding how much this drug would benefit patients in Wales, and the PHA UK has put in so much hard work to support us - which I am extremely grateful for. I hope the right decision is made for patients who live in England because it shouldn’t matter where you live - if it’s going to benefit a patient in any way, why deny them a much easier and safer way to take the medication?”

Neil Hamilton, consultant pharmacist at the Sheffield Pulmonary Vascular Disease Unit said: “We will now be able to treat Welsh patients here in Sheffield with a new drug that could slow progression of the disease, but we want to ensure that all our patients have equal access – regardless of their postcode. The PHA UK have used patient voices to prove that the drug is needed for everybody, so it’s important that NHS England now follow the positive outcomes for Scotland and Wales with the same fair decision on Selexipag.”

Selexipag is manufactured by Actelion Pharmaceuticals. Robin Bhattacherjee, general manager at Actelion Pharmaceuticals UK, said: “Working in collaboration with the PHA UK has been vital in helping us secure a positive decision on Selexipag in Wales. They brought really important evidence of the significant symptom burden of PH to the table, using the voices of patients themselves in the form of their major survey into life with pulmonary hypertension. This has proven the importance of working collaboratively in a transparent, ethical manner with a patient organisation and the need for the patient voice to drive home the impact of living with a rare condition such as PH".

The PHA UK has also been working in collaboration with the National Institute of Clinical Excellence (NICE) to secure funding for Selexipag in England – the third piece in the jigsaw.

Iain added: “It is essential that PAH patients receive the best quality and evidence-based care for this debilitating, life-shortening disease - and although we feel the decisions by Wales and Scotland are steps in the right direction, as a dynamic patient organisation we will continue to fight for equality and equity of access to service provision across the UK. We now eagerly wait for the right decision to be made in England.”

Notes to editor

Image shows Iain Armstrong, Chair of the PHA UK.

Media contact: Mary Ferguson on 01226 766900 or at mary@capitalbmedia.co.uk

About pulmonary hypertension (PH)

People can be born with PH or develop it at any time and it can affect anyone, regardless of age or ethnic background. It affects more women than men.

The seven adult specialist PH centres in the UK are within the Freeman Hospital in Newcastle; Golden Jubilee National Hospital in Glasgow; Royal Hallamshire Hospital in Sheffield; Papworth Hospital NHS Trust in Cambridgeshire; and Hammersmith Hospital, Royal Brompton Hospital and Royal Free Hospital in London.

Patients from Wales are generally treated at the specialist centres in either London or Sheffield.

About the Living with PH survey

Almost 600 patients responded to the Living with Pulmonary Hypertension survey, which reveals that concerns about life expectancy have the biggest impact on quality of life - with over 50 per cent reporting a ‘major impact’. Almost two thirds (63 per cent) said that financial worries had an impact on their lives.

The survey, which looked at all aspects of life with PH, also discovered that almost half of patients had to wait over a year after first experiencing symptoms before being diagnosed. 40 per cent had to see four or more doctors.

The full results of the Living with PH survey can be accessed here

For further information about pulmonary hypertension visit www.phauk.org, call the PHA UK office on 01709 761450 or e-mail office@phauk.org.  

The full results of the Living with PH survey can be accessed here

PHA UK is a registered charity no: 1120756

This press release was prepared by Capital B Media, a public relations and media training agency based in Sheffield, South Yorkshire. For more information, please visit www.capitalbmedia.co.uk.

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