A new campaign report was presented to Health Minister Norman Lamb MP in Parliament last week by the All Party Parliamentary Group (APPG) for Muscular Dystrophy, warning that people with muscle-wasting conditions could be denied cutting-edge therapies owing to drastic changes to the way drugs are funded and assessed for rare diseases. Jonathan Kingsley, Senior Policy, Campaigns and Parliamentary Officer of the Muscular Dystrophy Campaign, outlines the report and its recommendations. 

Following a comprehensive six month inquiry supported by the Muscular Dystrophy Campaign, the APPG for Muscular Dystrophy examined the needs of patients with rare diseases such as muscular dystrophy and related neuromuscular conditions to access potential treatments without delay if they become available. The APPG heard about the stages before the licensing of treatments and the importance of streamlining and speeding up the process from basic research to clinical trials.

Evidence was taken from leading clinicians and researchers in the neuromuscular field and representatives of the pharmaceutical industry. We heard about barriers to treatments and the challenges of moving promising treatments into clinical trials – and then the approval of such treatments by regulatory authorities. People with muscle-wasting conditions and their families gave powerful accounts about their fears that inconsistency of specialist care across the UK could delay – or even prevent – the NHS delivering therapies effectively once they become available.

Robert Meadowcroft, Chief Executive of the Muscular Dystrophy Campaign, commented:

“This inquiry is a major step in the right direction, highlighting issues that need to be addressed with a sense of utmost urgency. These will not be simple medications, and alongside licensing and funding issues, we must also address the need for access to specialist care for the 3.5 million people living with rare conditions such as muscular dystrophy in the UK. Specialist care is needed in each region, to ensure that these emerging treatments are as effective as possible in extending lives and improving people’s health.”

Leading figures involved in the regulation, appraisal and funding processes gave encouraging indications that there are ways in which processes can be streamlined and simplified.

The APPG for Muscular Dystrophy is calling on:

  • the Government to establish a ring-fenced fund for rare disease drugs to ensure that patients affected by rare diseases are not denied treatments
  • NICE to assess treatments for rare conditions in a different way from less rare conditions – ensuring an appropriate and effective cost benefit analysisthe Medicines and Healthcare Products Regulatory Agency, NICE and NHS England to speed up access to life-changing drugs after final stages of clinical trials to ensure there are no major delays in treatments reaching children
  • NHS England to ensure specialist centres are equipped with an appropriate range of health professionals to deliver treatments