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Don't Do Different Things – Do Things Differently! Drug Development in Rare Diseases: The Patient's Perspective
Author(s) -
Speid L
Publication year - 2016
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1002/cpt.403
Subject(s) - drug development , perspective (graphical) , attrition , pharmaceutical industry , intensive care medicine , disease , medicine , population , risk analysis (engineering) , drug , medical emergency , business , pharmacology , environmental health , pathology , computer science , dentistry , artificial intelligence
The pharmaceutical industry and regulatory authorities have traditionally made risk–benefit decisions without consulting patients. Today there is a movement to engage the patient in these decisions. This is particularly pertinent for the rare‐disease patient population, who have limited access to effective diagnostics and treatments. We argue that their involvement at the earliest point of drug development will lead to more appropriate decision making, and potentially reduce the attrition rate .