The value of evaluating and implementing pharmaceuticals

Sammanfattning: Pharmaceuticals are a central part of high-quality health care and a resource for improving population health. However, high prices set by private companies who develop and own the rights to new pharmaceuticals question the value that they contribute to the health care system. Publicly funded health care systems need to get the most from limited health care resources, which has become even more apparent in recent years with ageing populations, rapid technological development, and more recently the impact of COVID-19. Reducing pharmaceutical prices increase the current value that they offer to health care systems, but price reduction also decreases incentives to develop future treatments. Hence, the health care systems must balance the objective of improving the value from the treatments available today and incentivising the development of future treatments. Governments and health care decision makers use a variety of policies to control prices and use of pharmaceuticals. However, these policies are rarely the focus of formal analysis and their effect on short- and long-term population health is often unclear. The aim of this thesis was to investigate how policies that control pharmaceutical prices and implementation impact population health and incentives for pharmaceutical research and development (R&D). The first study in this thesis outlines a framework for assessing the effect of pharmaceutical policies on population health and pharmaceutical earnings and shows that price reducing policies can increase the current value of pharmaceuticals to health care systems while lowering R&D incentives. The design of specific policies determines the impact as well as the distribution of the gains of lower prices across patients, health care providers, pharmacies, and other affected parties. The second study analyses the trade-off between accuracy and cost of the cost-effectiveness appraisals for pharmaceuticals by viewing it as a diagnostic test that aims to identify costeffective treatments. The study identifies some policy relevant conclusions, including that the process should be flexible over time and depend on characteristics of the treatment undergoing assessment. Study three, investigating the impact of regional implementation variation of the antiplatelet ticagrelor, found that an additional 1,100 Quality Adjusted Life Years (QALYs) could have been gained from achieving equal implementation across health care regions. This represents a value of SEK 285 million from avoiding regional implementation variation of ticagrelor (given a value of SEK 250,000 per QALY). The study also shows that avoiding delays due to sequential decisions on reimbursement, treatment guidelines, and funding could have significant value. Finally, the fourth study investigates the comparative effectiveness of ticagrelor using observational data collected as part of routine clinical care in the SWEDEHEART registry. The study finds similar reduction in mortality as observed in the pivotal randomised clinical trial of ticagrelor, the PLATO trial. Furthermore, the importance of appropriate methods for observational research on comparative effectiveness are demonstrated, highlighting the importance of using appropriate methods when investigating the effectiveness of treatments used in clinical practice. In conclusion, this thesis shows the importance of analysing and understanding the effect of policies that control price and implementation of pharmaceuticals, whether the goal is to maximise the value from currently available pharmaceuticals or to also incentivise the development of new pharmaceuticals. Although the value of improving implementation may not be as obvious or tangible as savings from lowering pharmaceutical prices, improving implementation may contribute more to population health than reinvesting potential savings from price reductions.