Raising orphans: How clinical development programs of drugs for rare and common diseases are different

Clinical Pharmacology and Therapeutics

Volumn 92, Issue 2, 2012, Pages 262-264

  Orfali, M ^a   Feldman, L ^b   Bhattacharjee, V ^c   Harkins, P ^c   Kadam, S ^c   Lo, C ^c   Ravi, M ^c   Shringarpure, D T ^c   Mardekian, J ^a   Cassino, C ^a   Cote T ^c  

^a PFIZER INC   (United States)

^b TUFTS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c KECK GRADUATE INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DRUG; NONORPHAN DRUG; ORPHAN DRUG; UNCLASSIFIED DRUG; VACCINE;

ARTICLE; CARDIOLOGY; CLINICAL TRIAL (TOPIC); DERMATOLOGY; ENDOCRINOLOGY; FOOD AND DRUG ADMINISTRATION; GASTROINTESTINAL TRACT; HEMATOLOGY; HUMAN; IMMUNOLOGY; INFECTION; METHODOLOGY; NEUROLOGIC DISEASE; NEUROSCIENCE; ONCOLOGY; OPHTHALMOLOGY; PRIORITY JOURNAL; PULMONOLOGY; RANDOMIZATION; RARE DISEASE; RESPIRATORY SYSTEM; UROGENITAL SYSTEM PARAMETERS;

DRUG APPROVAL; DRUG DISCOVERY; HUMANS; ORPHAN DRUG PRODUCTION; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RARE DISEASES; RESEARCH DESIGN; SAMPLE SIZE; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84864133268     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2012.87     Document Type: Article

References (4)

1. Coté, T., Kelkar, A., Xu, K., Braun, M.M. & Phillips, M.I. Orphan products: an emerging trend in drug approvals. Nat. Rev. Drug Discov. 9, 84 (2010).
2. S asinowski, F.J. Quantum of effectiveness evidence in FDA's approval of orphan drugs DIA: National Organization for Rare Disorders (NORD). US Conference on Rare Diseases and Orphan Products (2011). Washington, DC, 11-13 October 2011.
3. Kesselheim, A.S., Myers, J.A. & Avorn, J. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. JAMA 305, 2320-2326 (2011).
4. Tufts Center for the Study of Drug Development. Approved Products Database and Orphan Products Database 〈http://csdd.tufts.edu/research/ databases〉 (2012).