Access barriers to ultra-orphan medicines for patients in England
On 6 July, the MPS Society brought together patients, clinicians, MPs, campaign groups and industry representatives to discuss access barriers to ultra-orphan medicines for patients in England.
This event follows months of extensive campaigning on access to treatments for patients with ultra-rare diseases. The focus of the event was largely on access to Vimizim™ for patients with Morquio A syndrome, who continue to face uncertainty as they wait for a decision on funding for the treatment they need.
The event was chaired by Greg Mulholland MP, who led a panel of clinicians and campaigners – representing both the Morquio and Duchenne Muscular Dystrophy communities – who shared their perspectives on the challenges facing those fighting for access to ultra-orphan treatments in the UK today. The panellists were: Professor Chris Hendriksz, Consultant Transitional Metabolic Medicine, Manchester Academic Health Science Centre and Salford Royal Foundation NHS Trust, Paul Lenihan MBE, CEO, and Mark Silverman, Trustee, Action Duchenne, Katy Brown, Morquio campaigner and Christine Lavery MBE, CEO, MPS Society.
Why are we campaigning?
• The system for evaluating treatments for rare and ultra-rare diseases in England is fundamentally broken. Current processes discriminate against people living with rare and ultra-rare diseases, creating a two-tier NHS.
• Patients with the ultra-rare disease Morquio have waited 15 months, since the approval of Vimizim by the European Medicines Agency, for a decision on funding from NHS England. This wait has been extended by a further 6 months, as we now wait for the NICE process. All the while patients are missing out on life saving treatments and are likely to see their condition deteriorate
• The UK is a world leader in life sciences, yet patients are not reaping the benefits, as ground breaking treatments are left unfunded by the NHS and out of reach for the people who need them most. The UK has proven an attractive prospect for the life sciences sector, however this world leading position is being eroded by a slow and opaque access system.
• The first infusion of Vimizim was in the UK and as a result of clinical trials, run in the UK, the treatment is now funded and available in 20 other European countries.
The reason for protesting and crowdfunding. Patients should not be suffering the consequences of process failure. Ministers need to intervene to address the issue of interim funding. They have been hesitant to get involved in clinical decisions; however it is clear the process is broken. They now have a duty and a responsibility to investigate and advise.
• Charities and patient groups are fundraising for research, but their efforts are fruitless when treatments do not reach the patients that need them.
• NHS England, NICE and the Department of Health need to engage collectively with patient groups and industry to gain a more complete understanding of the issues.