The use of patient registries for orphan drugs and rare diseases

EJHP Practice

Volumn 16, Issue 4, 2010, Pages 30-31

  Clarke, Joe T R ^a   Hernberg Stahl E ^b  

^a CENTRE HOSPITALIER UNIVERSITAIRE DE SHERBROOKE   (Canada)

^b Late Phase Solutions Europe AB   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

ORPHAN DRUG;

ACCURACY; ARTICLE; DATA ANALYSIS; DRUG EFFICACY; DRUG SAFETY; RARE DISEASE; REGISTER;

EID: 78349303068     PISSN: 17819989     EISSN: None     Source Type: Trade Journal    
DOI: None     Document Type: Article

References (8)

1. Orphan Drug Act, Public Law 97-414. Sect. J(b). 1983 or Title 21, US Code, Sect. 360aa, note, Pub. L. No. 97-414 (1984, 1983).
2. Haffner ME, Whitley J, Moses M. Two decades of orphan product development. Nat Rev Drug Discov. 2002;1(10):821-5.
3. Gliklich RE, Dreyer NA, editors. Registries for Evaluating Patient Outcomes: A User's Guide (Prepared by Outcome DEcIDE Center [Outcome Sciences, Inc. dba Outcome] under Contract No. HHSA29020050035I TO1). Rockville, MD: Agency for Healthcare Research and Quality. April 2007.
4. Mehta A, Ricci R, Widmer U, Dehout F, Garcia de Lorenzo A, Kampmann C, et al. Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey. Eur J Clin Invest. 2004;34(3):236-42.
5. Eng CM, Fletcher J, Wilcox WR, Waldek S, Scott CR, Sillence DO, et al. Fabry disease: baseline medical characteristics of a cohort of 1765 males and females in the Fabry Registry. J Inherit Metab Dis. 2007;30(2):184-92.
6. Concato J. Observational versus experimental studies: what's the evidence for a hierarchy? NeuroRx. 2004;1(3):341-7.
7. Vandenbroucke JP. Observational research, randomised trials, and two views of medical science. PLoS Med. 2008;5(3):e67.
8. Mehta A, Beck M, Elliott P, Giugliani R, Linhart A, Sunder-Plassmann G, et al. Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data. Lancet. 2009;374(9706):1986-96.