OBJECTIVES: This study evaluates the role of patient-reported outcomes (PROs) and patient-group submissions in reimbursement decisions across health technology assessment (HTA) agencies in Acute Myeloid Leukemia (AML).

METHODS: HTA reports from G-BA, IQWiG (Germany), HAS (France), NICE, SMC (United Kingdom), PBAC (Australia), pCODR (Canada), TLV (Sweden), GENESIS (Spain), and CF AVEN – Emilia Romagna, ESTAR (Italy) were reviewed for drugs approved for the treatment of AML. HTA reports from 2009 to May 2020 were collected. Abbreviated submissions, non-submissions, and reviews without details were excluded. Data regarding the use of PROs and participation of patient-groups were included.

RESULTS: A total of 50 reviews were assessed in the final sample, of which 80% of decisions were positive. The most-used PRO in the clinical model was the European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30), and in the economic model was the EuroQol-five dimensions questionnaire (EQ-5D). Only 15% (n=6) of reviews with positive decisions used PROs in their clinical evidence. Thirty-two out of 50 reviews assessed the cost-effectiveness of intervention, of which 75% (n=24) of decisions were positive. Approximately 79% (n=19) of these reviews with positive decisions did not use PROs in their economic model. Patient-group submissions were observed in 64% (n=32) of reimbursement decisions, of which approximately 81% (n=26) of decisions were positive. Patient groups were found to be localized. Patient groups commented on the following areas: patient need, disease burden, quality of life, tolerability, disease control, and health outcomes.

CONCLUSIONS: From this study, it was observed that the use of PROS does not have a significant impact on recommendation of intervention. Even though there were many patient advocacy groups consulted in the HTAs, the impact of their feedback on HTA decisions is unclear.