Pathway for approval of a gene therapy orphan product: Treading new ground

Molecular Therapy

Volumn 21, Issue 8, 2013, Pages 1465-1466

  Byrne, Barry J ^a  

^a UNIVERSITY OF FLORIDA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ORPHAN DRUG; PARVOVIRUS VECTOR;

DRUG APPROVAL; DRUG EFFICACY; DRUG INDICATION; DRUG LEGISLATION; DRUG QUALITY; DRUG RESEARCH; DRUG SAFETY; EDITORIAL; FOOD AND DRUG ADMINISTRATION; GENE THERAPY; HUMAN; PHARMACOGENOMICS; PROTECTION; RARE DISEASE; STUDY DESIGN; UNITED STATES;

DRUG APPROVAL; GENETIC THERAPY; HUMANS; ORPHAN DRUG PRODUCTION; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84881304982     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.157     Document Type: Editorial

References (2)

1. Sasinowski, FJ (2011). Quantum of effectiveness evidence in FDA's approval of orphan drugs. National Organization for Rare Disorders http://www.rarediseases.org/docs/policy/NORDstudyofFDAapprovaloforphandrugs.pdf
2. O'Reilly, M, Kohn, DB, Bartlett, J, Benson, J, Brooks, PJ, Byrne, BJ et al. (2013). Gene therapy for rare diseases: Summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther 24: 355-362.