Muscular Dystrophy Campaign Secures Campaigning Success on Rare Disease Drugs

The Government’s announcement today that an Early Access to Medicines scheme is to be brought forward follows a year of campaigning on this issue by the Muscular Dystrophy Campaign and patients and their families.

In February 2013, the All Party Parliamentary Group (APPG) for Muscular Dystrophy, supported by the Muscular Dystrophy Campaign, began an inquiry into ‘access to high-cost drugs for rare diseases’. The APPG set out to explore the challenges and barriers that may prevent access to potential future treatments for muscular dystrophy and other rare diseases.

Over four evidence sessions, the APPG heard from parents of children with a muscle-wasting condition, practising clinicians, pharmaceutical companies, as well as the National Institute for Health and Care Excellence (NICE) and the NHS.

The report was launched in Parliament September 2013, and revealed that while potential treatments are finally in clinical trials, parents fear that the “race against time” for their children to be treated may be lost owing to unnecessary delays, funding issues and bureaucracy.

Unfortunately there are currently no potential treatments which can be fast-tracked via this scheme, but the pipeline for ground breaking potential treatments for rare diseases is set to grow and we now look forward to working with the Government to ensure that patients affected by these conditions do not lose out should treatments reach the market.

In a moving statement, a parent of a child with a rare muscle-wasting condition told MPs and Peers:

Time is running out. Please help me and my family – every day I witness my son struggle and every day we live in fear of the future”.

This underlined the urgency of the situation facing many families and the APPG’s report made a number of recommendations on how access to treatments could be sped up. Central to this was the introduction of an Earlier Access to Medicines Scheme.  Key recommendations read:

 ‘In order for the Government to fulfil its commitment to ‘creating a more progressive environment that supports and promotes innovation with a view to providing faster access to new medicines for patients’, we recommend that it bring forward the Early Access to Medicines Scheme. The scheme would be of real value to patients affected by muscular dystrophy and other rare diseases as it would allow faster access to emerging treatments which have shown clear scientific benefit to patients and a positive risk profile. We call on the governments in each of the UK countries to support this approach and for the Medicines and Healthcare Products Regulatory Agency (MHRA) to publish the findings of their recent consultation on such a scheme.

‘We support the efforts of the pharmaceutical industry and the regulators to try to reduce the timeframe of clinical trials for future molecular patches with the potential to treat Duchenne muscular dystrophy, while maintaining standards of quality, safety and efficacy. The development of these so-called personalised medicines requires a re-assessment of approval protocols to avoid delays for patients with rare mutations. We call on regulators to consider class approval and a fast-track approach for drugs with the same mechanics of action treating different mutations of conditions such as Duchenne muscular dystrophy.

At the report launch, these recommendations received the backing of Parliamentarians, including the Health Minister, Normal Lamb MP, who expressed his support for the inquiry, and the Deputy Prime Minister, Rt Hon Nick Clegg MP. Mr Clegg said:

Having been contacted by a number of constituents who suffer from Muscular Dystrophy, I know how this rare disease has a detrimental affect on families throughout the UK. It’s important that we all work together to do what we can for those affected. I therefore extend my praise to those involved in organising this reception to highlight this campaign.

Our campaigners, themselves families affected by a muscle-wasting condition, were able to secure high profile coverage in the national media on the need to speed up access to treatments. This included BBC Breakfast News, where Jo Ashton and her son Liam were interviewed, and a feature on BBC News with Mark Creswick and Robert Meadowcroft. BBC Radio 4’s Today Programme, BBC Radio 5, ITV and the Independent also covered the report and spoke to families affected by muscular dystrophy or a related condition.

This media coverage and the report launch helped to significantly raise the profile of the issue and left us in a strong position to move the campaign for early access to medicines forward. The Muscular Dystrophy Campaign subsequently held meetings with Dr Ian Hudson, Chief Executive of MHRA, Sir Andrew Dillon, Chief Executive of NICE, as well as representatives from the NHS, to discuss how the APPG’s recommendations could be implemented as soon as possible. 

The campaign for earlier access to treatments also continued to gain recognition in Parliament. For example, at a debate last December on Rare Diseases, Graham Stringer MP drew attention to the slow process in obtaining approval for rare disease drugs:An excellent drug, or one that is likely to be excellent, can be developed, but because of the nature of rare diseases-that very few people suffer from them-it is very difficult to get it through the trials process, because that is designed to see whether the drug works on large numbers of people, quite sensible…NICE should have different rules for drugs related to rare diseases.

A firm indication that the Government was carefully considering the APPG’s recommendations came in January, when the Health Minister, Earl Howe, confirmed that Government thinking was at an ‘advanced stage’ on an early access to medicines scheme.

This has culminated in today’s announcement, which means that our hard work in campaigning for an Early Access to Medicines scheme is starting to pay off and we would like to thank all those who have lent their support. 

For more information about the Muscular Dystrophy Campaign’s work in campaigning for an Early Access to Medicines scheme, please get in touch on 020 7803 4838 or by email

Currently, a campaign on early access to medicines is underway in the US to urge the Food and Drug Administration (FDA) to give Accelerated Approval Rating to emerging treatments for Duchenne muscular dystrophy.

Source: Muscular Dystrophy Campaign

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