The importance of natural histories for rare diseases

Expert Opinion on Orphan Drugs

Volumn 3, Issue 8, 2015, Pages 855-857

  Gavin, Pamela ^a  

^a National Organization for Rare Disorders   (United States)

Author keywords

Clinical trials; FDA; natural history studies; rare diseases

Indexed keywords

DISEASE COURSE; FOOD AND DRUG ADMINISTRATION; HEALTH CARE QUALITY; HISTORY; HUMAN; LONGEVITY; PRIORITY JOURNAL; PRODUCT DEVELOPMENT; QUALITY OF LIFE; RARE DISEASE; REVIEW;

EID: 84937485232     PISSN: None     EISSN: 21678707     Source Type: Journal    
DOI: 10.1517/21678707.2015.1063415     Document Type: Review

References (3)

1. EURORDIS, Rare Diseases Europe website. Available from: http://www. eurordis.org/about-rare-diseases
2. Sasinowski FJ. Quantum of Effectiveness Evidence in FDA's Approval of Orphan Drugs. Chairman of the Board, NORD. Available from: http://nordphysicianguides.org/wp-content/uploads/2012/02/NORD-study-of-FDAapproval-of-orphan-drugs.pdf
3. Workshop on Natural History Studies of Rare Diseases: Meeting the Needs of Drug Development and Research. Available from: https://events-support. com/Documents/Summary-NHS.pdf