Patient group welcomes decision to fund new rare disease drug in Scotland

PRESS RELEASE: Patient voices have helped secure the approval of a new drug designed to improve the lives of people affected by a rare, life-limiting disease in Scotland.

The Pulmonary Hypertension Association (PHA UK) has welcomed today’s decision by the Scottish Medicines Consortium (SMC) to approve a new treatment for people with pulmonary hypertension, which causes high pressure in the blood vessels connecting the heart and lungs. Just 7,000 out of 64 million people in the UK live with a diagnosis.

Today, Monday 7 May 2018, the SMC has announced that selexipag will now be made available on the NHS. The drug was rejected in July last year because evidence of the clinical and economic benefits was ‘not robust enough’.

Over the last few months the PHA UK has been campaigning hard and in collaboration for the decision to be overturned by submitting evidence, responding to questionnaires and fighting to represent the PH community at various agency and key stakeholder meetings.

A major piece of research into what it is like to live with pulmonary hypertension, conducted by the PHA UK and published in 2017, formed the major basis of the evidence. The study, ‘What it means to live with PH today’, harnessed the voices of 563 patients to paint an accurate picture of the impact of the disease.

Sixty per cent of survey respondents said the serious condition has a ‘major impact’ on their overall quality of life.

Symptoms of PH 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.

Iain Armstrong, chair of the PHA UK, said: “The symptom burden of pulmonary hypertension is massive, so access to any drug that improves life for these patients is fair. Last year’s rejection of selexipag was grossly unfair and the depth and insight that our evidence brought to the decision-making process was crucial in securing this turnaround. We’ve been told by decision-makers that it was enlightening and refreshing to see compelling, relevant evidence that harnessed the patient voice.

“As a PH community we don’t ask for much; we just want the same equality and access to treatment as other disease areas like cancer. This is a step in the right direction but we want to see equality across the UK.”

England and Wales have already reviewed selexipag once and said no, so the PHA UK is also working in collaboration with the National Institute of Clinical Excellence (NICE) and All Wales Medicines Strategy Group (AWMSG) to help them understand why funding the drug is so vital.

Pauline Harrison, 42, lives in Milton of Leys in Inverness and was diagnosed with pulmonary hypertension when she was seven months pregnant with her son.

She said: “This is really good news. One of the scary things about having PH is not knowing whether there is anything that will make you better, as you don’t know how your body will react. That’s why it’s so important that new drugs are developed and approved, as we need to feel that there is hope at the end of the tunnel.

“Although my PH has progressed too far for me to be able to use this drug myself, it’s good to know that there is something out there that can benefit other people. An alternative to intravenous medication can give someone their life back and their freedom back.

“My intravenous medication is keeping me alive and allowing me to live well but it does have its challenges. I know it’s too late for me but I hope having access to this new medicine helps other patients.

“You can’t put a price on a life and if the tables were turned, wouldn’t those wearing suits and making the decisions put up the same fight?”

The PHA UK campaigned for access to selexipag via its own internal advocacy group PHocus2021, a consortium of health professionals and patients pushing for public policy changes to improve lives for people affected by PH.

Thanks to a range of highly advanced treatments developed over the last 15 years, people with pulmonary hypertension have seen quality of life improve and life expectancy double to around six to seven years on average, with many living longer. But there is no cure. A few people with PH undergo heart and lung transplants.

Selexipag, also known as uptravi, helps to relax and widen the pulmonary arteries, relieving symptoms of pulmonary hypertension 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.

Adult patients with PH are treated at seven specialist centres across the UK including the Golden Jubilee National Hospital in Glasgow. Children with the disease are treated at Great Ormond Street Children’s Hospital in London.

The PHA UK is based in Sheffield, South Yorkshire, and is the only charity in the UK dedicated to supporting the PH community made up of patients, their family and carers and NHS professionals.



Media contact: Mary Ferguson on 01226 766900 or at 

Last medically reviewed: May, 2018 • May, 2021