Exploring clinical and economic value of novel therapies in oncology; contemporary challenges and applications

Organizations that evaluate health technologies face significant challenges when dealing with uncertain evidence. The evidence they evaluate is often based on short-term measures instead of long-term ones, single-arm trials instead of randomized clinical trials, or incomplete data without enough long-term follow-up. This reality means these national agencies must make recommendations based on incomplete or immature information. This thesis addresses ways to handle incomplete or immature survival data in oncology, where prolonging survival is the ultimate goal of novel cancer treatments.
The thesis is divided into three main parts.
Part I focused on understanding how surrogate measures relate to overall survival. My studies highlighted the role of post-progression treatments in assessing surrogate endpoints in lung cancer, which can apply to other cancer types and offer a valuable tool for decision-makers to assess observed treatment effects on surrogate endpoints. In addition, I shed light on new candidate surrogate outcomes in lung cancer and multiple myeloma.
Part II focused on comparing treatments by considering differences in population characteristics. My studies employed the best approach to promptly inform decision-makers regarding treatment efficacy in newly diagnosed multiple myeloma patients. In the future, I expect the use of alternative methods suitable for the evolving clinical evidence available in the public domain.
Part III focused on the inclusion of historical data to estimate overall survival. Using external data to improve survival extrapolations represents a sensible alternative to trial-oriented analyses - for less uncertainty and better decision-making. This approach is contingent upon their relevance to the decision problem and method choice. My studies were further used for HTA interaction for reduced uncertainties and better decision-making.
These topics are some of the biggest challenges in evaluating health technologies in cancer treatment. The findings from this thesis should assist manufacturers and health technology assessment agencies in better determining the value of new therapies in terms of effectiveness and cost-effectiveness.