LAUSR

Introduction Patient-reported outcomes (PRO) data are important in understanding patients’ experience of disease and treatment; however, PRO data are not universally collected or consistently included as part of a Health Technology Assessment (HTA) submission. Additionally, the HTA bodies’ response to PRO data vary, making the impact unclear. To understand the impact of PRO data on reimbursement decisions for Acute Myeloid Leukemia (AML) indications, an in-depth analysis of HTA bodies’ appraisals of AML and analogous indications was conducted. Methods This analysis was conducted using IQVIA's HTA Accelerator, which contains HTA appraisals from ≥100 HTA agencies in thirty-nine countries. Included in the analysis were single-technology assessments (original submissions, resubmissions, extensions of original indications, and renewals); relevant regulatory approvals and pivotal trials were also analyzed. Results Of the 185 AML appraisals from sixteen HTA bodies, 66 (36%) included PRO data. Within these, thirteen different PRO instruments were identified, none of which have been validated in patients with AML. For seven of twenty in-scope products, PRO evidence positively impacted ≥1 of the HTA decisions. Although the same HTA bodies (i.e., Scottish Medicines Consortium, pan-Canadian Oncology Drug Review, and the National Institute of Health and Care Excellence) generally accepted the PRO evidence, others were critical of the evidence (i.e., Haute Autorité de Santé and the Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen). The most common concerns raised by the HTA bodies regarding the PRO evidence included trial design and low patient response rate. Conclusions Of the products that included PRO evidence in their HTA submissions, 35% received positive feedback from ≥1 HTA body on their submitted PRO evidence. Attention to PRO data collection is key to demonstrate the value of AML products to HTA bodies. Without these data, a clear gap in the understanding of patients’ experience is evident.