Use of real-world evidence in pharmacoeconomic analysis: illustrations in The Netherlands and China

The main objective of the thesis is to assess the added value of use of observational data in pharmacoeconomic (PE) evaluations. Five sub-aims were addressed in the thesis:
1) a systematic literature review summarized results evidencing the cost-saving potentials of renin-angiotensin-aldosterone system (RAAS) inhibitors, including angiotensin converting enzyme inhibitors (ACE inhibitors) and angiotensin II receptor blockers (ARBs), in type 2 diabetes (T2D) patients with nephropathy compared with conventional therapy excluding a RAAS inhibitor;
2) a general framework was developed to illustrate the problems and solutions of data quality assessment and preprocessing, based on the ‘fit-for-use’ concept;
3) a comparative effectiveness study compared the effectiveness of ACE inhibitors vs. ARBs for protecting T2D patients from renal function decline in a real-world setting, the value of observational data in outcomes research studies was assessed;
4) a real-world study described the changes of diabetes cost in Chinese patients using electronic claims data, and the value of observational data in health economics studies was assessed;
5) a cost-effectiveness analysis of RAAS inhibitors (ACE inhibitors and ARBs) vs. other active anti-hypertensive agents and no anti-hypertensive treatment in patients with T2D and nephropathy in China was performed from a healthcare payer perspective, and a budget impact analysis was conducted alongside the PE evaluation to analyze the budget changes of Chinese urban employee basic medical insurance (UEBMI) when different strategies of medication use are applied; the use of observational data in PE evaluations was assessed.