Access to treatments

Every day is vital for people with muscle wasting and weakening conditions. We work tirelessly to ensure members of our community can access the treatment they need, when they need it.

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How treatments become available in the UK

Exceptions for rare conditions

Our Access to Treatments campaign

Treatments currently in our campaign

Successful campaigns

How treatments become available in the UK

In recent years, there has been significant progress in the development of treatments for some muscle wasting and weakening conditions. Many potential treatments are currently being tested in clinical trials, and several are available or emerging very soon.

Once a treatment has been tested in clinical trials, it is reviewed by the Medicines and Healthcare products Regulations Agency (MHRA). This ensures the treatment is safe, effective, and any benefits outweigh possible side effects. If approved, the treatment will be granted a license so it can be prescribed in the UK. However, at this stage the treatment cannot be accessed free-of-charge through the National Health Service (NHS). An assessment must take place to decide if the treatment is cost-effective.

The assessment is completed by the National Institute of Health and Care Excellence (NICE) for England, and Northern Ireland and the Scottish Medicines Consortium (SMC) for Scotland. Although their processes are slightly different, both bodies review the benefits and impact on quality of life the treatment would provide and weigh these against the cost of the treatment. You can find out more on the NICE and SMC websites.

Once NICE or SMC has recommended a treatment, patients have a legal right to access it through the NHS if a doctor says the treatment is appropriate. However, it is important to remember that NICE, SMC and MHRA continue to review medicines when new patient experiences and evidence arise. Although rare, it is possible for a previously approved treatment to be withdrawn.

Exceptions for rare conditions

Because muscle wasting and weakening conditions are rare, NICE and SMC may sometimes think there isn’t enough evidence from sources like clinical trials to make a firm recommendation, even if they find the evidence that is available convincing. So, they may ask for further evidence to be collected.

As treatment options for muscle wasting and weakening conditions are limited, NICE and SMC have processes which allow patients to access the treatment through the NHS, while the additional evidence is gathered. This is known as ‘managed access’ by NICE and the ‘ultra orphan medication pathway’ by SMC. Access is normally limited to a defined period of time (usually three to five years) and then another assessment takes place to take into account the new evidence.

Our Access to Treatments campaign

We’re campaigning for all people in the UK with muscle wasting and weakening conditions to have access to the best treatment. As quickly as possible.

Insights into the experiences of people living with the condition are critical to the NICE and SMC appraisals. We work with our community, healthcare professionals, researchers, other charities and patient groups to represent people with lived experience at various stages of the drug approval process. We carry out patient surveys, share data and support people with lived experience, as well as their families and carers, to share their knowledge and experiences as patient experts.

To find out how you can get involved in our Access to Treatments campaign work, please contact our campaigns team, who will be happy to share more information.

Treatments currently in our campaign

We are currently campaigning for access to six treatments across three muscle wasting and weakening conditions.