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Engaging patients in therapeutic development 

Engaging patients in therapeutic development
Chapter:
Engaging patients in therapeutic development
Author(s):

Emil Kakkis

, and Max Bronstein

DOI:
10.1093/med/9780198746690.003.0015
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date: 02 March 2021

Engagement of patients during the development of new treatments has evolved rapidly in the last decade. No longer should patients be viewed as passive subjects of research: they should be active participants in a more open and ambitious ecosystem of academic investigators, companies, and government entities. Enhanced engagement of patients throughout the development process provides important benefits for the introduction of useful and effective drugs by changing the understanding of the disease, the measurement of the disease during treatment, and the interpretation of benefit-risk. This system is critical for the discovery, study, development, regulation, delivery, and use of innovative therapies.

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