OBJECTIVES: Performance-based agreements (PBAs) allow for the management of uncertainty about the real‑life value of a new drug depending on its performance. As most PBAs apply to high‑cost, innovative therapies, especially in rare diseases or cancers, this study aimed to investigate the use of PBAs in common (non‑communicable, non-oncological) diseases in EU3 (Germany, France, UK) and the US.

METHODS: A targeted literature review with supplementary grey literature search focused on PBAs use in the top 5 common diseases that represent the leading causes of death according to the global data (i.e., heart disease, stroke, chronic obstructive pulmonary disease, Alzheimer’s disease, diabetes) during the last 10 years was conducted.

RESULTS: A total of 24 PBAs (22 in the US, 2 in the UK, none in Germany or France) for 14 products (sitagliptin, sitagliptin/metformin, dulaglutide, exenatide, empagliflozin, dapagliflozin, liraglutide, sacubitril/valsartan, ticagrelor, alirocumab, evolocumab, inclisiran, lecanemab, budesonide/formoterol fumarate dihydrate) used in common diseases were identified. The mechanisms of the identified PBAs were payment by results (UK and US), coverage with evidence development (US), and population health management (UK). The heart or cardiovascular disease was the main targeted indication, reflecting more than half (13) of PBAs. The other indications included: type 2 diabetes (9), Alzheimer's disease (1), and chronic obstructive pulmonary disease (1); no PBAs for stroke were found. Due to confidentiality, some agreements might not be identified.

CONCLUSIONS: Although rare diseases and cancers remain the main focus of PBAs, the study results have shown that those mechanisms are also implemented in common diseases. While the prevalence of common diseases is increasing due to population aging, the use of PBAs in both Europe and the US is expected to grow to enable patients access to new therapies, optimize their budget impact, and decrease payer uncertainty.