WP9 – Decision analysis in small population groups
The central challenge in clinical medicine is to find the optimal treatment for each individual patient. There are a number of challenges, however, especially for small population groups. To help overcome these challenges, an analysis of stakeholder decisions is instrumental. Different decisions (by regulators, payers, physician and patient) are based on the results from confirmatory clinical trials. Regulatory and payer decision rules may have to be adjusted for rare diseases and personalised health care settings.
We will therefore analyse the impact of different decision rules on e.g. expected total patient utility and optimal trial designs. Furthermore, we will consider the incentives for development of new treatments for small population groups.
The goal is to improve the rational basis for decisions, and help align different stakeholder perspectives. This, we think, will increase the number of novel drugs for small population groups, and improve the patient’s availability to optimally choose between different treatment options.