Neglected diseases - letter to The Times

    Heads of more than 35 charities that advocate for patients with rare diseases have written to The Times to ask NICE that rare diseases get fair and equal access to treatment. Read the letter and an article from a mum who fought for a life-changing treatment for her children who are living with Batten disease.

    Sir, We are concerned that patients with rare and complex health conditions will continue to miss out or face lengthy delays in accessing new treatments unless urgent action is taken. In the UK, three million people will be affected by a rare disease in their lifetime. Many of these diseases are life-threatening and have few, if any, treatment options. The National Institute for Health and Care Excellence (Nice) has shown willingness to adapt to scientific progress and the changing nature of the treatments that it assesses. As Nice takes on additional responsibility for rare disease medicines, we urge it to employ increased flexibility to ensure that people with rare conditions have equal access to drugs as people with common conditions.


    Because of the small number of patients with such disorders, there is greater uncertainty about whether a particular rare disease treatment will be effective. Drugs are often rejected because not enough is known about their long-term benefit, even if no other treatments are available. Where uncertainty is the primary issue blocking access, we urge Nice to offer conditional access to the new drug until more data is gathered.

    Visit our page on access to life changing treatment to find out more about our ongoing campaign.


    We are the only registered charity providing professional support to individuals and families affected by MPS, Fabry or a related disease in the UK.

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