Title |
Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints
|
---|---|
Published in |
Orphanet Journal of Rare Diseases, February 2015
|
DOI | 10.1186/s13023-014-0195-4 |
Pubmed ID | |
Authors |
Emil D Kakkis, Mary O’Donovan, Gerald Cox, Mark Hayes, Federico Goodsaid, PK Tandon, Pat Furlong, Susan Boynton, Mladen Bozic, May Orfali, Mark Thornton |
Abstract |
For rare serious and life-threatening disorders, there is a tremendous challenge of transforming scientific discoveries into new drug treatments. This challenge has been recognized by all stakeholders who endorse the need for flexibility in the regulatory review process for novel therapeutics to treat rare diseases. In the United States, the best expression of this flexibility was the creation of the Accelerated Approval (AA) pathway. The AA pathway is critically important for the development of treatments for diseases with high unmet medical need and has been used extensively for drugs used to treat cancer and infectious diseases like HIV. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 5 | 28% |
Canada | 2 | 11% |
France | 1 | 6% |
Spain | 1 | 6% |
Unknown | 9 | 50% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 15 | 83% |
Science communicators (journalists, bloggers, editors) | 2 | 11% |
Scientists | 1 | 6% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 2 | 2% |
Netherlands | 1 | <1% |
Belgium | 1 | <1% |
Unknown | 115 | 97% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 19 | 16% |
Researcher | 17 | 14% |
Other | 13 | 11% |
Student > Master | 9 | 8% |
Professor | 6 | 5% |
Other | 28 | 24% |
Unknown | 27 | 23% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 26 | 22% |
Biochemistry, Genetics and Molecular Biology | 9 | 8% |
Nursing and Health Professions | 9 | 8% |
Social Sciences | 9 | 8% |
Agricultural and Biological Sciences | 6 | 5% |
Other | 19 | 16% |
Unknown | 41 | 34% |