On Friday 30th January the AKU Society team were invited to attend a patient workshop organised by Findacure in London. The focus of the afternoon was on how to identify rare disease patients. Identifying patients is one of the biggest challenges faced by rare disease patient groups, and is crucial in order to provide patient support.

Clinical Trial Recruitment

After a fantastic introduction to the day from Flora Raffai, Head of Development at Findacure, we heard from Christa Van Kan from PSR. PSR is an organisation which specialises in orphan drug development and clinical trial recruitment for rare diseases. Christa started her talk by asking the audience why they think identifying rare disease patients is so difficult. There were plenty of responses.


Patients with rare diseases may still be awaiting diagnosis, and even if diagnosis rates are high, patient numbers are still low. This makes traditional advertising methods, such as putting posters up in GP surgeries, redundant. Despite this, rare disease patients and parents are very active on the internet, and have a very high motivation to be involved in research.

For researchers, Christa said, “Collaboration with rare disease patient groups is crucial to the success of clinical trial recruitment.” Christa works with us as part of our DevelopAKUre clinical trial consortium, so we were pleased to be given as an example of this working in practice. Christa said in her experience it is very important to have a good online presence in order to create a network for your rare disease, and successfully recruit patients.

Patient Support and Advocacy

Finding patients is not only crucial for clinical trials; it is important for the basic work of patient groups. The fundamental purpose of a patient group is to support its patients. In her talk, Christine Lavery, founder of the MPS Society, warned against focusing too heavily on research. Patient support is always important, and Christine made this clear to us when she said, “The advocacy service is the backbone of the MPS Society.”Christine

It was incredibly valuable to hear from Christine about how she built up the MPS Society from nothing, developing an important patient database, and making sure all patients had equal access to take part in research. A bit of key advice from Christine was not to be afraid of working closely with pharmaceutical companies.

Connecting Online

One of the best ways to connect with patients is online. The final two speakers highlighted this brilliantly, and gave some practical advice on how to identify patients using online tools.

Pete Chan from Tudor Reilly emphasised data from a recent survey showing the internet is now the top source of clinical trial information. He told us all of the importance of using Google Adwords to advertise your patient group or clinical trial. This can be very affordable for charities eligible for a Google Grant.


His advice was to focus key words on symptoms of disease you work for. This way patients who are googling their symptoms may discover your website, and get in contact. One of his key tips was to “…speak the language of the patient.”
Marta Campabadal from Eurordis was the final speaker of the day, and she spoke of how to unite your rare disease crowd and connect the isolated. Marta works on a Eurordis project called RareConnect, which is a website especially designed to allow rare disease patients to connect and share their experiences. Our alkaptonuria group on RareConnect has well over 100 members, and is a great platform for patients to ask questions, and share their stories. She told us how any rare disease group can have a community designed for them completely free.

Marta also spoke about the importance of using other social media tools to connect with patients. She said the biggest advantage of Facebook is its simplicity, but said to be aware of disadvantages such as lack of anonymity. Marta told us about the important use of social media management tools such as Hootsuite to keep track of social media accounts such as Twitter.

The discussions throughout the day were also incredibly helpful, as they allowed patient groups of different sizes to share ideas and advice. We found the day inspiring, and came away with a few new ideas on how to identify patients more effectively.


If you would like to attend the next Findacure patient workshop, you can send an email to [email protected] to find out more.