Cost effectiveness in practice and its effect on clinical outcomes

Abstract

AbstractThe value of new cancer drugs is maximized when they are used for the right patient in the right way in clinical practice. Clinical trials conducted during drug development are the most important source of information to predict value at the time a drug is introduced in practice. Regulatory approval is an indication of value, which lately has been complemented with an assessment of clinical value for decisions about reimbursement, using the methodology of health technology assessment (HTA). Formal cost-effectiveness studies are an important part of this methodology, aiming to assist decisions about value for money in health care spending. The question is if the addition of a complementary HTA and cost-effectiveness study increases the value realized by the drug in practice compared to how it would be used without these assessments.We review the issues involved in providing an answer by using the introduction of new targeted therapies for metastatic renal cell cancer (mRCC). Specifically, we examine the link between clinical trial data and estimations of cost-effectiveness at drug launch, reimbursement decisions, uptake and use in different countries and evidence about impact on outcome in patient populations for which the new drugs are indicated.We conclude that there is a weak link between the assessments used at drug launch and the value created in clinical practice. We suggest measures that are necessary for the achievement of evidence-based and cost-effective cancer care in clinical practice