This guest blog has kindly been written by Robert Meadowcroft, Chief Executive of the Muscular Dystrophy Campaign on how the campaign is at the forefront of fighting for quicker access to drugs for those with rare disease.
The Muscular Dystrophy Campaign is at the forefront of the campaign for faster access to rare disease treatments. The charity instigated a six-months long parliamentary inquiry by the All Party Parliamentary Group (APPG) for Muscular Dystrophy and supported the APPG in the production of its report on Access to high-cost drugs for rare diseases in September 2013, putting forward recommendations to ensure that patients with rare diseases such as muscular dystrophy and related neuromuscular conditions are able to access potential treatments without delay when they become available.
We heard during the inquiry about the challenges of moving promising treatments into clinical trials and through the regulatory, appraisal and funding processes. People with muscle-wasting conditions and their families gave powerful accounts about their fears that inconsistency of specialist care across the UK could also delay – or even prevent – the NHS delivering therapies effectively once they become available.
Bringing forward the Early Access to Medicines Scheme was a key recommendation of the APPG report and we welcomed its announcement by the Department of Health as a key step towards addressing the lengthy and at times convoluted process of bringing a drug to market. The scheme could be of significant benefit to people affected by muscular dystrophy and related neuromuscular conditions in allowing early access to groundbreaking new medicines, once the Medical and Healthcare Products Regulatory Agency indicates that safety and effectiveness have been satisfactorily demonstrated.
The Muscular Dystrophy Campaign is also encouraged to learn that the European Medicines Agency is conducting an adaptive licensing pilot project. Adaptive licensing is a very important part of the process of speeding up access to emerging treatments and we hope that this pilot will lead to a wider roll out of adaptive licensing in the near future.
The pipeline for groundbreaking potential treatments for muscular dystrophy and neuromuscular conditions looks increasingly strong and it is vital that patients affected by these conditions do not lose out should treatments reach the market.
For people with some forms of muscular dystrophy, every day counts and a year could be the difference between walking unaided and using a powered wheelchair, between breathing independently and needing a ventilator to support lungs through the night. The long wait for potential treatments to make their way through three stages of clinical trials can be agonising – particularly when followed by a drawn-out licensing and reimbursement process. We need urgency in the delivery of safe and effective treatments to these families, benefiting companies at the same time. We must never forget that every day is vital for each individual and family affected by a muscle wasting condition.
Robert will be speaking at the inaugural Fast Tracking Orphan Drugs Briefing on 10th July in London about the Early Access to Medicines Scheme. If you would like to discuss new regulation and how it will impact orphan drugs download a brochure for the event and join us to hear from leading industry experts.
Image: From Google, Trailblazers Muscular Dystrophy Campaign