Skip to main content

Your views on access to rare disease medicines

Genetic Alliance UK are working on a policy project to improve access to medicines for rare diseases. This project is called Resetting the Model. To inform this project, we are launching a survey to find out the views of patients, carers, patient organisations and industry members  on access to medicines for rare diseases.

This survey is now closed.


Through the Resetting the Model. project, we are looking at the decision-making process which determines whether licensed products are reimbursed and made accessible through the NHS. We want to deliver a vision for a single, new pathway for making decisions about rare disease medicines that is fair and transparent.

To ensure this vision comes from the whole rare disease community, we wish to understand your views on how decisions should be made around access to rare disease medicines. To do this, we are launching a survey to capture your preferences about the decision-making process. We will be releasing another survey at a later date, where you will be given the opportunity to expand upon your views. More information about the subsequent survey will be released in due course.


The survey we are using is known as a discrete choice experiment (DCE).

This survey follows a slightly unusual format:

  • For each question, you will be presented with two scenarios and asked to pick your preferred choice, by weighing up the characteristics in each scenario.
  • You will be repeatedly asked to choose your preferred scenario, when faced with different combinations of characteristics.
  • You may find yourself choosing between two non-ideal scenarios, this is so that we can understand what your priorities are.

We will use the data from the DCE to analyse which aspects of the decision making process for rare diseases are most important to you. We aim to incorporate this information into our design of a new method of decision-making. In order for us to design a new method of access to suit your needs, it is vital that you answer the survey at face value.


The survey is now closed to patients, carers and patient organisations.

If you are an industry member and produce a medicine to treat a rare or genetic condition, please select this survey.

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

Join our alliance of 200+ SUPPORT ORGANISATIONS AND charities