Niklas Zethraeus

Research problem and specific questionsHealth economic evaluations constitute an important basis for decisions on the reimbursement and use of oncology drugs in Sweden. At the time of decision there is high degree of uncertainty in survival effects from a lifetime perspective, which needs to be predicted from clinical trials with short follow-up time.The project aim is to investigate the accuracy in health economic evaluations in the assessment of oncology drugs and to investigate how these evaluations can be improved to guide decisions on the reimbursement and use of these drugs. The research questions are:how accurate are long-term survival predictions for oncology drugs compared to outcomes in clinical practice?how can relevant information outside the clinical trial and statistical methods be used to improve survival predictions of oncology drugs in health economic evaluations?Data and methodA database with longitudinal data from Swedish registries is used to answer the research questions. Question 1 is answered by studying long-term survival in clinical practice in cancer patients who have used drugs previously assessed by the Dental and Pharmaceuticals benefits agency (TLV) to inform decisions on the reimbursement or use of drugs at hospitals as recommended by the New Therapies Council (NT-Rådet). At the time of the decision the predicted long-term survival effect of the drug will be compared to the long-term survival effect in clinical practice. Question 2 is answered by validating two statistical methods which use external data outside the clinical trial.Societal relev,ance and utilisationThe project contributes to improving health economic evaluations supporting decisions on the reimbursement and use of oncology drugs in Sweden. This benefits TLV, NT-rådet, and the health care system and will increase the opportunity to attain the highest level of health benefits for limited resources in society. The project results will be published in scientific journals and will be presented on seminars at NT-rådet and TLV, which will support the dissemination of the results of the project to a wider community, authorities and healthcare providers.Plan for project realisationThe project is part of a doctoral project and will be conducted in collaboration with TLV and between three research groups at Karolinska Institutet, providing extensive experience in health economic evaluation, biostatistics and cancer survival, and strong clinical experience in oncology.