Lin Wang, ScM, MD

Prostate cancer is the most common cancer and the second leading cause of cancer death among men in the United States. It puts a tremendous burden on the health system, with 3.7+ million living cases, 248,530 new cases, and 34,130 new deaths expected in 2021. Most prostate cancer deaths were due to metastases. Progress in research has led to several chemohormonal therapies that can delay disease progression. The availability of these therapies has improved prostate cancer survival. However, owing to the lack of head-to-head comparison in clinical trials, little is known about the optimal choice weighing effectiveness and safety. As a result, clinical guideline committees hesitate to recommend one drug over others. Meanwhile, costs vary widely across therapies, ranging from hundreds of U.S. dollars (USD) to hundreds of thousands of USD, for a patient to complete the standard treatment courses. Identifying the value of the therapies is critical to inform clinical practice and insurance policy. This study compared the efficacy, safety, and cost-effectiveness of competing treatments. Bibliographic databases, trial registries, and regulatory documents were systematically searched to identify a comprehensive body of clinical trial evidence. Relative efficacy and safety of treatments were assessed by synthesizing clinical trial data using state-of-art evidence synthesis methods. The cost-effectiveness of treatments was assessed by advanced economic modeling. Besides answering the clinical and economic questions, this study addressed methodological challenges encountered by decision-makers in oncology and rare disease areas. That is, 20% of cancer treatments and most rare disease treatments were approved based on single-arm trials. A lack of head-to-head comparison with other treatment options precludes decision-makers from understanding the relative value of different treatments and investing limited health resources in an efficient way. This study involved a treatment that had only single-arm trial evidence and used novel statistical methods to assess its value relative to other treatments. The findings and the methodology of this study shall have a significant impact on clinical practice, health policy, and health outcomes research.