The Importance of Measuring Indirect Treatment Effects
Rahul Dhanda* and Donald E Stull
Published: December 19, 2022
DOI: 10.55162/MCMS.04.092
Abstract
The current FDA model of drug approval relies on determining the direct effect of treatment on outcomes of interest, including an evaluation of patient experience. The Cures Act (among others) that amends the Federal Foods and Cosmetic Act defines patient experience data as:
“data that are collected by any persons (including patients, family members, and caregivers of patients, patient advocacy organizations, disease research foundations, researchers, and drug manufacturers); and are intended to provide information about patients’ experiences with a disease or condition, including (A) the impact (including physical and psychosocial impacts) of such disease or condition, or a related therapy or clinical investigation, on patients’ lives; and (B) patient preferences with respect to treatment of such disease or condition.
“(source:https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development;
https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical).