[NAME] MLA Northern Ireland Assembly Parliament Buildings Ballymiscaw Stormont Belfast BT4 3XX

Dear [NAME]

Re: Faster access to potential treatments for muscle-wasting conditions

I am writing as a constituent and as a supporter of the Muscular Dystrophy Campaign regarding future access to high cost, specialist treatments for the estimated 1 in 1,000 people living in your constituency with muscle wasting conditions.

Muscle diseases weaken and waste muscles. They can cause lifelong disability or premature death, usually in childhood of young adulthood. The conditions can be inherited or acquired and can affect people of all ages, backgrounds and nationalities.

Patients and their families with neuromuscular conditions are waiting desperately for a cure or treatment to slow down the progress of their condition. Now, for the first time, treatments which could slow the progression of some of these conditions, such as certain mutations of Duchenne muscular dystrophy, are at advanced stages of clinical trial. Time is not a luxury that patients and their families have. We must ensure that any effective treatments reach them as quickly as possible.

[You could include some more personal details here, for example your need to access potential treatments without delay, your fears about the inconsistency of specialist care causing delays and obstacles in receiving treatment and how the wait is exacerbating yours or your family member’s condition]

The Muscular Dystrophy Campaign has recently launched a new initiative, Fast Forward, which is backed by families, clinicians, researchers and politicians. The charity is pressing for greater support for clinical trials for muscle-wasting conditions, as well as minimising delay at regulatory, approvals and funding stages. This follows concerns that if medical breakthroughs are made, slow appraisals and lack of funding and capacity could lead to patients in desperate need of treatments being denied access to a drug that could extend their life.

Fast Forward sets out ten actions aimed at faster access to specialist treatments. These include:

 The Health and Social Care Board (HSCB) to identify a model of sustainable funding to help meet the costs of delivering high-cost treatments for rare conditions  NICE to assess high-cost drugs for rare conditions separately from drugs for more prevalent conditions and not subject them to an inappropriate cost benefit analysis

 HSCB to ensure that optimum standards of care are delivered so that all patients in Northern Ireland have access to essential care and support from specialist multi- disciplinary teams. Consistent, high standards of care will also ensure that patients can participate in clinical trials for their particular condition.

 Funding for patient registries and databases from statutory bodies, for example HSCB.

I would greatly appreciate your support in highlighting these issues issue by writing to Edwin Poots MLA, Minister for Health, Social Services and Public Safety. I would be grateful if you could ask him what steps the Northern Ireland Executive is taking to work with other Governments in the UK, through the UK Strategy for Rare Diseases, to speed up funding and appraisals processes for high-cost treatments, foster greater links between research and care at specialist centres and ensure that vital clinical trials infrastructure is supported.

For more information on the ten actions towards specialist treatments outlined in Fast Forward, as well as priorities on faster access to raise in the Northern Ireland Assembly, please get in touch with Peter Sutton, Policy and Campaigns Officer, on 020 7803 4838 or by email at [email protected]

Thank you for your support and I look forward to hearing from you.

Yours sincerely,

[YOUR NAME]