Abstract
Payers and providers face challenges in enabling appropriate and sustainable access to new medicines. To help enable rational use of new medicines various policy options exist. In Sweden, horizon scanning, forecasting, value-based pricing and reimbursement, treatment recommendations, and assessment of drug utilization patterns and patient outcomes in routine clinical practice have been used to facilitate rational introduction of new medicines. Such activities, however, should be informed by research and be subject to continuous evaluation.
This thesis aims to examine selected elements of the process for managed introduction of new medicines. Study I provides an evaluation of the Swedish Horizon Scanning System. Study II assesses the impact of treatment recommendations on the use of new medicines in the specialized care setting. Finally, studies III and IV explore the utility of healthcare databases in the assessment of real-world use and outcomes of two specialist medicines prioritized for managed introduction. Different types of data were used in these studies, including public assessment reports published by the European Medicines Agency, early assessment reports prepared by the Swedish Horizon Scanning System, national sales data on all inpatient and outpatient medicines, regional administrative healthcare services data, and national registers of Statistics Sweden and the National Board of Health and Welfare. The evaluation of the Swedish Horizon Scanning System demonstrates that all innovative medicines that had substantial economic impact were identified and assessed prior to their introduction. The assessment of the impact of treatment recommendations shows that both local and regional treatment recommendations were associated with changes in the use of new medicines. Both regional and national healthcare databases provide the opportunity to study the use and outcomes of new medicines in routine clinical practice.
The findings indicate that healthcare decision makers can rely on the outputs of the Swedish Horizon Scanning System to keep informed of new medicines. Moreover, treatment recommendations appear to influence the uptake and utilization of new specialist medicines. Finally, even though the existing Swedish data sources provide unique research opportunities, the assessment of appropriate use and relevant outcomes of the growing number of new specialist medicines may still be impeded by a lack of fit-for-purpose data.
