Third Findacure Patient Group Workshop – 24th July

Yesterday saw 32 representatives from patient groups and research teams assemble at White & Case LLP in London for Findacure’s third patient group workshop of the year. The focus of the day was on the role patient groups could play in the development of clinical trials into their neglected conditions.


Hana Ayoob, The AKU Society

Flóra Raffai, Project Manager at Findacure, opened the workshop by introducing the need for patient groups to represent the patient voice within clinical trials development, and how this will result in better designed trials leading to more treatments being development for the millions waiting in the UK. She explained how increasing focus on rare diseases not only benefits patients but has a wider impact on the health of us all as many rare diseases inform on more common conditions and human physiology as a whole. Flóra then gave a short update on Findacure’s work over the first half of 2014 and gave an overview of projects currently in development. [Click here to access her slides].

Dr Tony Hall, Co-founder of Findacure, followed by presenting on the steps involved in planning a clinical trial. He shared the key challenges facing rare disease clinical trial development, emphasising the need for trials to be part of a large, complete clinical-regulatory strategy. In highlighting these challenges, Tony explained the best ways to overcome them through innovative thinking and early dialogue with regulators. [Click here to access his slides].


Kamlesh Sheth, PharmacoMedics

Hana Ayoob, Clinical Trials Coordinator at The AKU Society, shared her experiences recruiting and retaining patients for the European Commission funded DevelopAKUretrials. She explained that 30% of the clinical development timeline is taken up with patient recruitment and as a result 90% of all trials are delayed due to problems of recruitment. Her top tips for recruiting and retaining patients were: being relentless, providing clear information, and keeping it personal with the patients. [Click here to access her slides] [Click here to access her notes].

Following a short networking break, Dr Kamlesh Sheth from PharmacoMedicspresented on the ways in which patient groups can interact with industry in developing clinical trials. He emphasised his belief that it is mutually beneficial for this interaction to be as open as possible due to the shared objective to develop a treatment that is safe, effective, and accessible to all. He then detailed how to achieve a win-win relationship between patient groups and industry to achieve this common objective. [Click here to access his slides].

Panel Discussion

Panel Discussion

The final talk was given by Oli Rayner from Cystic Fibrosis Trust, who gave a patient perspective on clinical trials and presented research the Cystic Fibrosis Trust has conducted on the factors patients consider in the decision to take part in trials. He stressed researchers need to remember that patients ‘donate’ themselves to clinical trials and so it is important that they receive something back. [Click here to access his slides].

The workshop concluded with a panel discuss, where attendees were able to question all the speakers from the day. Key topics discussed were the need for patient groups to develop registries, how to manage cross-border clinical trials, and aspects of clinical trials that were made easier through a focus on rare diseases.

Findacure would like to thank all the speakers for their informative and engaging talks, White & Case LLP for the lovely venue, as well as all the attendees for their lively contributions to the day’s discussions. We look forward to seeing you at our future workshops. If you would like to see more photos of the event, follow this link to our Facebook photo album.

Thank you for posting this exciting event! I look forward to hearing about something similar in the US.

JC, Alan, and All,

Thanks again for forwarding this Is there anything similar to this patient workshop in the US? If not,
have any gluten-free or glutenataxia patients ever tried to organize something similar
with the help of their doctors?

This forum and the gluten America is wonderful! i am educating my doctors with what I learn!

Here is a very helpful link from Dr. Tom O'Bryan. You may not have seen
this link i posted yesterday, but it should still be valid. it is very long
(3 hours).

Here's the Dr. Obryan link again:

Dr Tom O’Bryan Live Stream Video on Gluten