Title |
Accelerating development, registration and access to medicines for rare diseases in the European Union through adaptive approaches: features and perspectives
|
---|---|
Published in |
Orphanet Journal of Rare Diseases, February 2014
|
DOI | 10.1186/1750-1172-9-20 |
Pubmed ID | |
Authors |
David Uguen, Thomas Lönngren, Yann Le Cam, Sarah Garner, Emmanuelle Voisin, Carlo Incerti, Marc Dunoyer, Moncef Slaoui |
Abstract |
There is growing recognition that the current research-and-development (R&D) and innovation-regulation ecosystem could be made more efficient to stimulate and support access to innovative therapies for those patients with rare, life-threatening diseases for which there are no adequate licensed therapies. New and progressive thinking on the principles and processes of drug development and regulation are needed in rare disease settings in order to ensure developments are financially sustainable. This paper presents perspectives on the current and emerging schemes for accelerating development of and access to medicines for rare diseases in the European Union. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 2 | 22% |
United Kingdom | 1 | 11% |
Canada | 1 | 11% |
Unknown | 5 | 56% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 5 | 56% |
Scientists | 3 | 33% |
Practitioners (doctors, other healthcare professionals) | 1 | 11% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Spain | 1 | 3% |
Brazil | 1 | 3% |
Unknown | 32 | 94% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Master | 12 | 35% |
Student > Ph. D. Student | 5 | 15% |
Student > Bachelor | 4 | 12% |
Researcher | 3 | 9% |
Other | 2 | 6% |
Other | 6 | 18% |
Unknown | 2 | 6% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 17 | 50% |
Pharmacology, Toxicology and Pharmaceutical Science | 4 | 12% |
Nursing and Health Professions | 3 | 9% |
Biochemistry, Genetics and Molecular Biology | 2 | 6% |
Social Sciences | 2 | 6% |
Other | 3 | 9% |
Unknown | 3 | 9% |