Patient group criticises decision to reject new rare disease drug in England
9 July 2018
A group that represents people living with the rare disease pulmonary hypertension has branded an NHS England decision to reject a new drug that could improve quality of life ‘exceptionally unjust and grossly unfair’.
The Pulmonary Hypertension Association (PHA UK) has been campaigning hard for Selexipag to be available in England, following positive decisions in Scotland and Wales. Today, 7th July, NHS England announced that an earlier decision not to approve the drug would be upheld.
Pulmonary hypertension 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.
The PHA UK has been campaigning in collaboration for the first negative decision in July 2016 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, 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: “As a patient organisation we know the rejection of this drug is exceptionally unjust and grossly unfair. The symptom burden of pulmonary hypertension is substantial, so access to any drug that may improve outcomes for these patients is vitally important for their health and wellbeing. An urgent review is needed on decisions regarding new therapies available for PH currently blocked or deferred by national funders. As a PH community we want the same equality and access to treatment as other disease areas like cancer. We are extremely disappointed by this decision and as a dynamic patient organisation we will continue to fight for equality and equity of access to service provision in the UK.”
The decision to reject Selexipag in England comes just weeks after the Scottish Medicines Consortium and All Wales Medicines Strategy Group approved the drug in Scotland and Wales.
Tess Jewson (pictured above), 27, lives in Chelmsford in Essex and was diagnosed with PH ten years ago. She said: “Living with PH is difficult enough without having drugs being rejected. My message to NHS England commissioners is this: How do you think it makes us feel knowing there are drugs out there that can keep us stable and you’re keeping them from us?
“Every day we struggle to live a ‘normal’ life with PH. Think about what we have to go through - the chronic pain and fatigue, the severe breathlessness, the tears, the frustration and anger and maybe you would understand why I, as a PH patient, am distraught at this decision. You can’t put a price on a life - but you’re making us pay with ours.”
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.
Notes to editor
Images shows Tess Jewson and Iain Armstrong, Chair of the PHA UK
Media contact: Mary Ferguson on 07496 872142 or at email@example.com
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.
For further information about pulmonary hypertension visit www.phauk.org, call the PHA UK office on 01709 761450 or e-mail firstname.lastname@example.org.
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 more information about PHocus2021, visit www.phocus2021.org.uk
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.