IMI PREFER – Including the patient perspective.


Last week we attended the closing conference for the 6 year long PREFER study. The final product is a very detailed manuscript (book!) on WHY Patient Preferences are important, WHEN preference studies should be used, and HOW to implement them. Patients and patient representatives should be involved in all stages of the medical product lifecycle, from initial idea, through study design and clinical trial, right past approvals and funding, to clinical use!

That’s a lot of information, and this is only the first publication. As research continues and the framework is put into use, we are likely to learn more and see updates.

From our perspective, as patient representatives in the rare disease community, this framework could (should) change the way we (as patients) guide future studies and are involved in our own care, accounting for what is important to us in terms of treatments and medications, what risks WE are, or are not, willing to take, and for which benefits or treatments. Often these decisions are made by those who may have a completely different viewpoint on what is important for us and our quality of life. A clinician or researcher may not fully appreciate the full spectrum of complexity as we do, living with it every day, for our whole lives. A trial designer may not be familiar with DM and may not fully understand the limitations or variability of the disease.

Emma-Jayne was a patient representative in this study from the start, on the NMD case study team, and has been involved at every stage. As a charity we are now in a position where we have a working knowledge of how patient preference studies can help our community, and how we can support clinical trials, research studies and even clinicians in implementing the framework and letting our voice be heard.

“I am very proud of the time and hard work I have put into this, both personally and professionally, and attending the closing conference really brought it all together. It was a celebration of achievement, but the work is not over. If anything, it is only just beginning. It is vitally important that expert patient representatives are involved in planning, design and implementation of preference studies aimed at their community, to avoid misrepresentation or unconscious bias from misunderstanding the disease. Now we need to be the voice of our rare disease patient community as more trials and studies are on the horizon.”

(Emma-Jayne, Cure Myotonic Dystrophy UK Charity)

You can read more about the PREFER project and download the recommendations here:



Past News:

Cure DM were delighted to be recognised as an official partner within the Prefer Project consortium.

It was an honour to be recognised for the work we are doing within the NMD sections of the study.

"PREFER will establish recommendations to support development of guidelines for industry, Regulatory Authorities and HTA bodies on how and when to include patient perspectives on benefits and risks of medicinal products. 

Over 5 years they will run patient preference studies in both academic and industry settings. This is to provide a better understanding of what will be a recommended best-practice approach to patient-preference studies. This will show how patient preference studies can give valuable information to support decision making for regulators and HTA bodies.