Introduction: Until recently, adjuvant treatment options for higher stage resectable cutaneous melanoma were limited. Two studies with a similar set-up, published 2017, led to registration of targeted therapy for BRAF-mutated melanoma with dabrafenib and trametinib as well as of the...

We empirically assess the potential financial impact of future gene therapies on the US economy. After identifying 109 late-stage gene therapy clinical trials currently underway, we estimate the number of new and existing patients with corresponding diseases to be treated by these gene...

While innovation is central for the quality of healthcare and improving health outcomes, it is also a source of increasing costs for governments. Confronted by fiscal pressures, governments have made efforts to restrict access to innovative treatments. While such policies are understandable,...

The threshold approach to medical decision-making, in which treatment decisions are made based on whether the probability of sickness exceeds a predetermined threshold, was introduced by (Pauker and Kassirer, N Engl J Med 293:229-234, 1975) and (Pauker and Kassirer, N Engl J Med 302:1109-1116,...