Inclusion of Health-Related Quality of Life (HRQoL) and Other Patient-Reported Outcomes (PROs) in Reimbursement Reviews From the Canadian Agency for Drugs and Technologies in Health (CADTH)
Author(s)
Nicole Fusco, ScD1, Brittany Galloway, PharmD, MSc, RPh1, Malia Gill, MS1, Sarah Cadarette, MPH1, Erika Wissinger, PhD1, Maria Koufopoulou, MSc2;
1Cencora, Conshohocken, PA, USA, 2Cencora UK, London, United Kingdom
1Cencora, Conshohocken, PA, USA, 2Cencora UK, London, United Kingdom
OBJECTIVES: CADTH incorporates patient input during the reimbursement review process to gain insights into patient needs and experiences. Patient involvement aids in identifying outcomes that hold significant importance to patients, ensuring their measurement in clinical trials of new medicinal products. This study aims to describe the outcomes measured and reported in products assessed in CADTH reimbursement reports for asthma, non-small cell lung cancer (NSCLC), and rheumatoid arthritis (RA).
METHODS: Reimbursement reviews detailing a specific intervention for asthma, NSCLC, or RA were identified from the CADTH website. Information on outcomes reported by pivotal trials was extracted from these reviews and summarized descriptively.
RESULTS: A total of 63 clinical reports (13 for asthma, 42 for NSCLC, and 10 for RA) described 117 unique trials across 44 treatments. Most trials across all indications reported at least one HRQoL outcome (84.2% for asthma, 80.0% for NSCLC, and 83.3% for RA). Other PROs were commonly reported for asthma (92.1%) and RA (95.8%), but less so for NSCLC (18.2%). PROs were rarely primary outcomes; all NSCLC trials used clinical outcomes focused on survival or response measured by standard criteria, while all RA trials focused on multidimensional outcomes combining patient-reported data with clinical data, like the American College of Rheumatology 20/50/70 Response Criteria and Disease Activity Score. Clinical outcomes were the primary endpoint in most (89.5%) asthma trials, with a few focused on HRQoL (5.3%) or another PRO (5.3%).
CONCLUSIONS: The selection of primary and secondary/exploratory outcomes varies significantly based on the disease area, with diseases like RA showing a higher inclination towards incorporating patient-reported data in primary outcomes, reflecting the close relationship between treatment efficacy and patient experience. This patient-focused approach can enhance the assessment of treatment impact and patient well-being in clinical practice, and should be considered in the evaluation of new medicinal products.
METHODS: Reimbursement reviews detailing a specific intervention for asthma, NSCLC, or RA were identified from the CADTH website. Information on outcomes reported by pivotal trials was extracted from these reviews and summarized descriptively.
RESULTS: A total of 63 clinical reports (13 for asthma, 42 for NSCLC, and 10 for RA) described 117 unique trials across 44 treatments. Most trials across all indications reported at least one HRQoL outcome (84.2% for asthma, 80.0% for NSCLC, and 83.3% for RA). Other PROs were commonly reported for asthma (92.1%) and RA (95.8%), but less so for NSCLC (18.2%). PROs were rarely primary outcomes; all NSCLC trials used clinical outcomes focused on survival or response measured by standard criteria, while all RA trials focused on multidimensional outcomes combining patient-reported data with clinical data, like the American College of Rheumatology 20/50/70 Response Criteria and Disease Activity Score. Clinical outcomes were the primary endpoint in most (89.5%) asthma trials, with a few focused on HRQoL (5.3%) or another PRO (5.3%).
CONCLUSIONS: The selection of primary and secondary/exploratory outcomes varies significantly based on the disease area, with diseases like RA showing a higher inclination towards incorporating patient-reported data in primary outcomes, reflecting the close relationship between treatment efficacy and patient experience. This patient-focused approach can enhance the assessment of treatment impact and patient well-being in clinical practice, and should be considered in the evaluation of new medicinal products.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
P7
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas