Introduction

This book explores topics of importance to all who have an interest in economic methods for assessment of the efficacy and effectiveness of new cancer treatments and in regulatory measures relating to their marketing authorization and pricing. Targeted therapies and modern immunotherapy are placing a substantial strain on health care budgets. Regulation and economic methods to assess the parameters for establishing efficacy and effectiveness are therefore of prime importance. Payer authorities have to determine whether the use of these novel therapies yields clinical benefits that justify their increasing cost. In the simplest terms, cost-effectiveness analyses quantify the ratio between the extent to which an intervention raises healthcare costs and the extent to which it improves health outcomes. Rigorous cost-effectiveness analyses translate all health outcomes into quality-adjusted life years. On the other hand, in order to sustain innovation, price regulations must be coupled with efforts to ensure that drug companies are still able to recoup their investments in high-risk and high-costs research programs. Ultimately, decisions regarding health care expenditures are also a question of society’s willingness to pay.