Patient Reported Outcome Measures (PROM)

We aimed to investigate the role of patient reported outcome measures (PROM) in the German AMNOG assessment with a focus on oncologic indications. In which way are PROMs evaluated and how do they affect the German Federal Joint Committee's (Gemeinsamer Bundesausschuss, G-BA) added benefit decision?

We have screened the relevant documents of the German authorities for specifications regarding PROMs. Subsequently, we performed a database analysis to validate the requirements found. The analysis encompassed all conducted benefit assessments in Germany up to 06/2022. We evaluated the number of PROMs used for each procedure and their corresponding consideration by the G-BA. We further investigated the most evaluated indication NSCLC.

Until 06/2022, 294 oncological procedures with overall 1002 PROMs were evaluated by the G-BA. The EORTC QLQ-C30 was the most frequently used PROM (368 times). With 43 (11.7%) procedures, NSCLC was the most evaluated oncological procedure, with 41 (95.3%) procedures utilizing PROMs. For studies accepted by the G-BA for NSCLC, 83 (80.6%) PROMs were also accepted. The lung cancer specific EORTC QLQ-LC13 was accepted in 95.2% of the accepted studies and demonstrated positive effects of the investigated drug in 15 of 20 (75.0%) benefit assessments. This was also true for 20 of 39 (51.3%) of the EORTC QLQ-C30 evaluated for NSCLC procedures, which was also accepted in 95.1% of the 41 procedures in which the respective study was accepted. The EQ-5D was accepted in 60.0% of the accepted studies, and 40.0% did not fulfill the requirements. For only 4 of 15 (26.7%) of the accepted EQ‑5D, positive effects were shown.

Although PROMs are of profound importance to assess the added benefit of oncological products, they have no privileges in the German system. Nevertheless, PROMs are frequently used and can make the crucial difference when it comes to decide on an added benefit, as selected precedents demonstrate.