TY - JOUR
T1 - Don’t Do Different Things…Do Things Differently! Drug Development in Rare Diseases
AU - Rudek-Renaut, Michelle
AU - Korth-Bradley, J. M.
PY - 2016/10/1
Y1 - 2016/10/1
N2 - Although definitions of rare disease vary, most acknowledge that there are small numbers of affected patients compared with other conditions. Small numbers of patients, overlapping involvement of investigators as researchers and caregivers, as well as close relationships between researchers and manufacturers require a different pattern of drug development. Regulatory guidances for rare diseases are available, as well as ones for specific rare diseases. Maintaining drug supply for rare diseases also demands innovative approaches.
AB - Although definitions of rare disease vary, most acknowledge that there are small numbers of affected patients compared with other conditions. Small numbers of patients, overlapping involvement of investigators as researchers and caregivers, as well as close relationships between researchers and manufacturers require a different pattern of drug development. Regulatory guidances for rare diseases are available, as well as ones for specific rare diseases. Maintaining drug supply for rare diseases also demands innovative approaches.
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U2 - 10.1002/cpt.424
DO - 10.1002/cpt.424
M3 - Comment/debate
C2 - 27393720
AN - SCOPUS:84986596234
SP - 333
EP - 335
JO - Clinical Pharmacology and Therapeutics
JF - Clinical Pharmacology and Therapeutics
SN - 0009-9236
ER -