Abstract
The European pharmaceutical market is strictly regulated. Sufficient levels of quality, safety, and efficacy will have to be demonstrated before a pharmaceutical is allowed to enter the market. There is little evidence, however, that the current drug regulatory framework is achieving its goals of protecting and promoting public health in
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a cost-effective manner. Health Technology Assessment (HTA) is commonly used to assess the cost-effectiveness of medical interventions, but any policy aimed at increasing public health can be subjected to a cost-effectiveness analysis, including drug regulation. The aim of this thesis therefore was to assess the cost-effectiveness of several parts of the drug regulatory framework in order to identify whether the aims of safety-related drug regulation are met. Furthermore, several other methodological approaches were used to assess the monetary valuation of safety-related regulatory actions, as well as trade-offs made with regard to benefit-risk assessment of pharmaceuticals. Two regulatory cost-effectiveness analyses are reported in this thesis. First, the cost-effectiveness of thorough QT/QTc (TQT) studies was assessed. These clinical trials evaluate a pharmaceutical’s QT-prolonging potential, which can increase the risk of a drug-induced sudden cardiac death, and have been part of the drug regulatory framework since 2005. Furthermore, the cost-effectiveness of Periodic Safety Update Reports (PSURs) for biologicals was assessed. Both regulatory cost-effectiveness analyses found incremental cost-effectiveness ratios (ICERs) that exceed commonly applied willingness to pay thresholds for medical interventions. However, other research reported in this thesis found that the societal valuation of safety-related regulatory actions is high, and there is evidence that health gains resulting from medical interventions are not valued equally to health losses resulting from adverse drug reactions. Therefore, it cannot be assumed that the willingness to pay for safety-related drug regulation (in terms of costs per QALYs gained) is equal to the willingness to pay for medical interventions. Societal developments during the last decade have resulted in an increasingly risk-averse drug regulatory framework. As more stringent regulatory requirements could negatively affect drug prices, more efforts should be made to identify the societal willingness to pay for drug safety. The research performed in this thesis indicates that the regulation of small drug safety risks can yield substantial opportunity costs, but that it is unlikely that these opportunity costs are considered in the development of regulatory measures
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