Tuesday 26 February 2013
Parliamentary inquiry into future of access to high cost drugs for rare diseases launched
Today marked the launch of the All Party Parliamentary Group (APPG) for Muscular Dystrophy's Inquiry into accessing high cost drugs for rare diseases. The inquiry will be conducted over the next few months exploring the challenges and barriers that may prevent access to potential future treatments.
The APPG provided an opportunity for MPs and families living with muscle-wasting conditions to quiz an expert panel, consisting of clinicians, researchers and representatives from pharmaceutical companies about how access to treatments in the future could be facilitated. Baroness Thomas of Winchester, who chaired the session, highlighted that this is an extremely exciting time in research for research into neuromuscular conditions, with new treatments for Duchenne muscular dystrophy on the horizon.
Lord Walton of Detchant shared his concerns about the National Institute for Health and Clinical Excellence (NICE) controlling the regulation of medicines for rare diseases and in particular stressed the need for 'special' treatment for rare disease drugs given their high cost and low incidence.
The Group was updated on the exciting developments in research by the panel of experts including Professor Francesco Muntoni.
Dr Rohit Batta, the Global Medical Leader for Neuromuscular Disorders at Glaxo Smith Klein Rare Diseases Unit, gave the Group an idea about the breadth of clinical trials GSK are currently undertaking.
Allison Morgan, the Vice President of Clinical Research & Development at Prosensa Therapeutics, described how the group's approach to clinical work was having positive results and voiced her hope that the inquiry sessions could be a platform for policy change.
Allison Morgan commented after the session:
It was very encouraging to see the level of passion and commitment displayed by the attending MPs, and their understanding of the issues at hand. This is a timely inquiry. We are on the brink of great therapeutic discoveries and we need to decide now how these will be handled. Personalised therapies are not only relevant to the treatment of conditions like Duchenne muscular dystrophy - they are the future of medicine.
We need to work together to move personalised treatment forward, and ensure that the care needed to optimise the effectiveness of these therapies, such as physiotherapy, cardiac and respiratory care, is also developed. Care for orphan disease should be elevated within the NHS and allocated its own ring-fenced budget.
A key element to the Inquiry session was the importance of time. Emily Crossley, whose son has Duchenne muscular dystrophy, asked the panel how long accessing potential treatments would take once the drugs were licensed, highlighting how critical time is.
The meeting was productive in bringing together experts and MPs, with both Graham Evans MP and Alison Seabeck MP requesting that the panel works with them to provide details and information of the potential barriers that might obstruct swift access to promising treatments for patients, so that they can play their part in Parliament.
Dr Marita Pohlschmidt, Director of Research at the Muscular Dystrophy Campaign, said:
This is a time of exciting developments in the field of neuromuscular research, with potential treatments for Duchenne muscular dystrophy on the horizon. Today we heard from leading researchers and the pharmaceutical industry, on all the issues from gaining approval for rare disease treatments to ensuring funding for their prescription through the NHS. It was a fascinating and encouraging first session, of what promises to be a landmark parliamentary inquiry.
The Muscular Dystrophy Campaign provides all the organisational support for the inquiry and the All Party Parliamentary Group for Muscular Dystrophy. For more information, please get in touch on 020 7803 4839 or by email at j.kingsley@muscular-dystrophy.org
