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

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 England.

The Pulmonary Hypertension Association (PHA UK) has welcomed the decision by NHS England to approve Selexipag for people with pulmonary hypertension, which causes high pressure in the blood vessels connecting the heart and lungs. Approximately 7-8,000 out of 64 million people in the UK live with a diagnosis.

NHS England has announced that Selexipag will now be made available to patients from April 2019, after initially rejecting it during a prioritisation round in July of this year.

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.

This collaborative campaigning has also resulted in the drug being approved in Scotland and Wales this summer.

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 basis of the evidence submitted to all three commissioning bodies. 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 overturned decision by NHS England, following the positive decisions by the Scottish Medicines Consortium and All Wales Medicines Strategy Group, is the third piece in the jigsaw for equity of access to drug therapy for people living with pulmonary hypertension.

“The symptom burden of PH is significant, so access to any drug that improves life for these patients is fair. NHS England’s initial rejection of Selexipag was grossly unfair and the depth and insight that our evidence brought to the decision-making process – for all three commissioning bodies – was crucial in securing this turnaround.

“It’s vital that the PH community receives the same equality and access to treatment as other disease areas like cancer.”

Tess Jewson, 27, lives in Chelmsford in Essex and was diagnosed with PH ten years ago. She said: “I am ecstatic to hear that Selexipag has been approved by NHS England. It is brilliant that the Living with PH survey has helped to secure the decision, because it means PH patients are finally being listened to, and our opinions and lives are actually being taken into account. 

“We are very lucky to have the support of the PHA UK and I believe medical views and personal views together made our argument stronger. As patients we want to be given the chance to live a long life and if it means approving more drugs, then so be it. PH is incurable, but knowing we have different drugs available reassures us that we can continue living the best life we can.”

The PHA UK campaigned for access to Selexipag via its own internal advocacy strategy 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.