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Rare disease patient advocates & sponsors come together to co-produce clinical research

Oktober 2019

EURORDIS Community Advisory Board patient advocates

Over the last decade, EURORDIS has made great progress in empowering people living with a rare disease and their advocates to engage with the sponsors and bodies that carry out research and develop therapies for rare diseases.

Likewise, EURORDIS’ actions facilitate sponsors’ collaboration with rare disease patient advocates, ensuring that research developed takes into account the real needs of people living with a rare disease.

The EURORDIS Charter for Collaboration in Clinical Research in Rare Diseases

Ten companies working on rare disease therapies have now signed up to the revised EURORDIS Charter for Collaboration in Clinical Research in Rare Diseases, which was updated earlier this year.

The Charter sets out a common framework and aims to improve the quality of clinical research in rare diseases and to build transparent and effective dialogue between companies or academic sponsors and patient networks. By signing the Charter, a sponsor shows its determination to respect the principles of collaboration with patients.

>>Companies interested in signing up to the EURORDIS Charter for Collaboration in Clinical Research can contact Céline Schwob:

Community Advisory Boards (CABs)

Community Advisory Boards

(CABs) are groups established and operated by patient advocates. They facilitate an ongoing dialogue between patients and sponsors on research and policy in specific disease areas.

CABs are involved in scientific as well as policy-related issues such as access to therapies. They can have anywhere from seven to sixteen patient advocate members and help to design, carry out and communicate on clinical studies that are more rational and inclusive of the patient’s point of view. By doing so, a bond of trust between the patient and scientific community is formed and can result in higher quality research.

The EURORDIS Community Advisory Board Programme

What if a CAB does not exist in your disease area?

Or are you from a company looking to connect with patients to collaborate on research for a specific disease?

The EURORDIS Community Advisory Board Programme assists patient organisations in setting up and structuring a CAB for their disease and can connect research sponsors with patients.

Since the start of the Programme in 2018, EURORDIS has supported the creation of four CABs for cystic fibrosis, rare lymphomas, Duchenne muscular dystrophy and cystinosis. Discussions are also ongoing to create CABs for several other diseases in early 2020.

Hilde De Keyser of Cystic Fibrosis Europe created a CAB for cystic fibrosis with the support of EURORDIS. She said, ‘EURORDIS supported us to drive forward our own cystic fibrosis CAB. The cystic fibrosis CAB allows us, CF Europe, to establish a transparent and solid collaboration with drug developers, and allows patients to drive the research agenda, reversing the traditional model of companies alone deciding research priorities. Through our CAB we have progressed in discussions on issues like more realistic and patient-friendly outcome measures and the importance of measuring relevant quality of life markers. We are also working on improving access to clinical studies by getting more European and Eastern European sites involved in clinical studies’.

The CAB Programme also covers common principles and tools for patient groups and sponsors, including: training and capacity building of patient advocates, peer-to-peer exchange of experiences across CABs, quality monitoring of the process and outputs of CABs, transparency and prevention of competing interests, promotion of the programme, evaluation and possible eventual scientific publications.

As part of the CAB Programme, EURORDIS can offer specific training pre-meeting and at CAB meetings to facilitate patient-led interactions using EURORDIS Open Academy materials.

>>Patient organisations and companies interested in collaborating through the EURORDIS CAB Programme are encouraged to contact Rob Camp:

Ongoing dialogue with industry

Discussions at this Tuesday’s EURORDIS Round Table of Companies Workshop in Barcelona focused on the importance of patient engagement in the life cycle of medicines. This workshop included a capacity-building session on understanding and using metrics for evaluating the learnings and the utility of CABs. Read the Workshop concept paper.

>>Companies interested in joining the EURORDIS Round Table of Companies are invited to contact Céline Schwob:

Eva Bearryman, Communications Manager, EURORDIS