News & Events

Your experiences of accessing rare disease medicines

Last Reviewed 4/07/2019

By Toni Qualey

Is there a treatment for your rare condition? If so, have you experienced any problems trying to access it?

We’re launching a survey to capture your experiences of accessing rare disease medicines. The responses you provide will be incorporated into our project on access to rare disease medicines.

The survey deadline has been extended to 17:00, Friday 26 July 2019.

Why should I take part?

Genetic Alliance UK are working on a policy project (‘Resetting the Model’) to improve access to medicines for rare diseases.  We plan to deliver a new vision for making decisions about which rare disease medicines should be made accessible through the NHS.

We have carried out extensive research on the decision-making processes that are currently used. We also previously conducted a survey to understand individuals’ preferences about these processes. From this work, a selection of key issues with the current system have emerged.

We are launching this survey to ensure that our list reflects and completely captures any problems you might have faced accessing a medicine for your rare condition. This is the final chance to share your experiences for this piece of policy work, so your response is of the upmost importance.

Who can take part?

We want to hear from patients, parents/carers and patient representatives about your experiences of accessing rare disease treatments. This survey is for individuals where a treatment for their condition has been deemed safe (it is licensed), and may, or may not, be accessible via the NHS.

If you are responding on behalf of an organisation, we welcome both organisation-wide responses and responses from individuals within the organisation.

This survey was previously open only to members, but we are now seeking wider participation, so we welcome responses from non-member patient organisations as well as individual patients and carers themselves.

Take part in the survey here

For any more information about the survey or the ‘Resetting the Model’ project, please contact [email protected].

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