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Regulators Should Better Leverage Effectiveness Standards to Enhance Drug Value
Author(s) -
Naci Huseyin,
Alexander George Caleb
Publication year - 2014
Publication title -
pharmacotherapy: the journal of human pharmacology and drug therapy
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.227
H-Index - 109
eISSN - 1875-9114
pISSN - 0277-0008
DOI - 10.1002/phar.1467
Subject(s) - leverage (statistics) , authorization , flexibility (engineering) , risk analysis (engineering) , clinical trial , business , drug , value (mathematics) , actuarial science , prior authorization , medicine , computer science , pharmacology , computer security , economics , pathology , management , machine learning
Regulators show some flexibility in the evidentiary standards of effectiveness that must be demonstrated for a drug prior to its market authorization. Adopting a more formal framework for when and how effectiveness standards should vary would improve the therapeutic value of new medicines at the time of market entry. We identify three factors—the number and effectiveness of existing treatment alternatives, magnitude of unmet need, and expected clinical application—to guide the effectiveness threshold for a given therapy. Using these factors, regulators should actively guide sponsors regarding appropriate comparators and end points in pivotal trials, as well as determining the size and characteristics of the patient populations enrolled.