Evidence from clinical practice to support healthcare decision making

The growing pressure on healthcare budgets creates great tension between financial sustainability of healthcare systems and accessibility to (new) treat¬ments. To ensure equal and timely access to promising but costly inpatient drugs, conditional funding was introduced in the Netherlands in 2006. At that time, conditional funding implied the additional funding of innovative drugs (i.e. reimbursing hospitals [most of] the drug costs) for a period of three years on the condition that data regarding uptake, use and outcomes (in terms of effectiveness and cost-effectiveness) of these drugs in clinical practice were to be collected. Conditional funding applied to two innovative but costly drugs for patients with metastatic renal cell carcinoma, i.e. bevacizumab and temsirolimus. This dissertation shows that the traditional treatment with interferon-alpha (IFN-α) has been largely replaced by treatment with so-called targeted therapies, mainly sunitinib. It also shows that few patients were treated with bevacizumab (in combination with IFN-α) or temsirolimus, even though these therapies were added to European guidelines in 2009, and Dutch guidelines in 2010 (besides sunitinib). Due to the limited use of bevacizumab and temsirolimus in clinical practice, data collection yielded little additional evidence regarding the effectiveness and cost-effectiveness of these drugs. This dissertation did show that new therapies that prolong progression-free survival (as demonstrated in other studies), postpone reductions in health-related quality of life. This is because symptoms, such as fatigue and pain, increase with progression of disease. In addition, the dissertation showed that the overall mix of new drugs has led to an increase in quality-adjusted life-years of patients with metastatic renal cell carcinoma, as well as an increase in costs. In line with the current policy of so-called specialist drugs (including costly inpatient and outpatient drugs), this dissertation states to restrict the use of conditional funding of expensive drugs. Only in specific cases is outcomes research able to provide a more robust estimate of cost-effectiveness. These cases should be selected carefully in order to minimize the costs of extensive data collection.

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