"Patients and Researchers: Partners for Life!"
Patients need Research...
Research represents hope for the millions of rare disease patients who are without cure today. In the last decade huge leaps have been made in genetics and medical technology, raising great hopes from scientific and therapeutic progress.
However, existing public research programmes for rare diseases are not sufficient; drug development to treat a small number of patients remains very limited and the fact that patients and researchers are scattered makes it difficult to aggregate the value of the knowledge already acquired.
Research needs Patients…
Rare disease patients are the best partners researchers can ask for – Without their participation in clinical trials, bio banks, databases and registries, research would be brought to a standstill. In addition to being the subjects of the research, patients are ‘experts by experience’ – they and their families know their disease better than anyone else. They can therefore help find therapeutic clues and advance basic research by furthering understanding into the epidemiology and natural history of the disease.
“Families had reported the existence of breathing difficulties in patients without obesity problems. We passed on this information to clinicians. At a subsequent meeting, a scientist decided to research this symptom on a mouse model and got confirmation.” Parent of a Prader Willi Syndrome patient
Our goal: Make each of our individual stories a milestone in the general progress of sciences
Rare disease patient organisations can be valuable partners in research projects – by funding research teams, equipment and training or by helping constitute cohorts and organising campaigns to collect biological samples. Patients often act as catalysers for increased collaboration amongst researchers, clinicians, industry and patients. This is why it makes sense for patients and researchers to work closer together!
Rare Disease Research is an important area of research
Rare Disease Research needs to be better funded
Patients and Researchers win by working together
Find out more...
- To learn more about who is working in rare disease research today, visit the Rare Disease Day Hall of Fame
- To see the results of the Patients in Research Survey conducted by EURORDIS, click here
SURVEY RESULTS: Patients & Research
View the results of the EURORDIS Survey of European Patient Organisations.
Interview with Orpha.net founder
Video interview with Ségolène Aymé, Founder & Director of Rare Disease Online Database Orpha.net.
A View from Industry
"Bridging Patients and Researchers". Video Interview with Wills Hughes-Wilson (Genzyme).