Cost effectiveness in practice and its effect on clinical outcomes

Abstract

a b s t r a c tThe 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 evidencebased and cost-effective cancer care in clinical practice.

Mission‐oriented translational cancer research – health economics

“…However, the healthcare system is not yet prepared for this new role. Although data on use of resources and actual outcomes from specific interventions are rapidly evolving, and methods for comparative effectiveness analysis are being presented, the actual use of such studies is still in an early stage (J€ onsson et al, 2014;Luce et al, 2010). There are many reasons for this.…”

Economic considerations and patients' preferences affect treatment selection for patients with rheumatoid arthritis: a discrete choice experiment among European rheumatologists

“…A recent article revealed a strong association between access to (expensive) biologics and the country’s wealth 8. Interestingly, however, another review found that drug coverage for innovative and expensive drugs does not necessarily translate into its use; there were differences in use between countries with comparable reimbursement criteria, and also between regions within a country 9. Differences in attitudes among clinicians likely contribute to differences in treatment choices 10…”

Context matters in outcome and management of rheumatoid arthritis

“…38 Relative differences in parameter estimates of ≥10% were considered as indicating relevant bias. 39 Regression analyses were estimated in Stata 12. 40 …”

“…35,36 Only a few contextual factors, namely gender, 37 education, [38][39][40] socioeconomic status, 39 or coping and helplessness as personal factors, 41 have been studied with regards to their influence on (a limited number of) outcomes. They have shown to contribute importantly to the outcomes studied.…”

“…38 The high heterogeneity is of some concern as it jeopardizes equality in care, a common principle that can be found in all European healthcare systems. 39 In simple words, it should not be the lucky incidence of a patient to be treated by a doctor who is more open to patients' preferences, or who is less cost-sensitive when proposing a treatment. Certainly, some variations in attitudes between clinicians are acceptable but our data suggest that the variations are significant and concerning.…”