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Simple Summary: The prognosis of children with relapsed/refractory (R/R) acute myeloid leukemia (AML) remains poor, and innovative treatments are needed. Most drugs approved for adults with AML over the last decade are not available or not licensed for use in children. Clinical trials in pediatric AML to assess the safety and/or efficacy of new drugs may take a long time to recruit given smaller patient numbers. The overarching aim of the Pediatric Acute Leukemia (PedAL) program, supported by the Leukemia and Lymphoma Society as part of its Dare to Dream Project, is to establish new standards of care for children with R/R AML via international collaboration in Europe, North America, and Asia-Pacific to accelerate clinical trial conduction, increase access to promising new therapies, and create a registry of uniformly-collected data. These efforts will facilitate biomarker-driven approaches of targeted therapies, of which an overview is provided in this manuscript, with the intent to obtain regulatory approvals. The prognosis of children with acute myeloid leukemia (AML) has improved incrementally over the last few decades. However, at relapse, overall survival (OS) is approximately 40–50% and is even lower for patients with chemo-refractory disease. Effective and less toxic therapies are urgently needed for these children. The Pediatric Acute Leukemia (PedAL) program is a strategic global initiative that aims to overcome the obstacles in treating children with relapsed/refractory acute leukemia and is supported by the Leukemia and Lymphoma Society in collaboration with the Children's Oncology Group, the Innovative Therapies for Children with Cancer consortium, and the European Pediatric Acute Leukemia (EuPAL) foundation, amongst others. In Europe, the study is set up as a complex clinical trial with a stratification approach to allocate patients to sub-trials of targeted inhibitors at relapse and employing harmonized response and safety definitions across sub-trials. The PedAL/EuPAL international collaboration aims to determine new standards of care for AML in a first and second relapse, using biology-based selection markers for treatment stratification, and deliver essential data to move drugs to front-line pediatric AML studies. An overview of potential treatment targets in pediatric AML, focused on drugs that are planned to be included in the PedAL/EuPAL project, is provided in this manuscript. [ABSTRACT FROM AUTHOR]

Simple Summary: There is a scarcity of economic analysis on the use of non-invasive biomarkers to inform policy-making with respect to their application in population-based colorectal cancer (CRC) screening programs. The objective of this study is to conduct a comparative cost-effectiveness analysis on using M3CRC (m3 + Fn + Ch + Bc + FIT) as a primary test for population-based screening, when compared with FIT and colonoscopy. The study can provide economic analysis regarding the use of non-invasive biomarkers in population-based CRC screening programs. This study aimed to examine the cost-effectiveness of fecal biomarker M3 panel compared to fecal immunochemical test (FIT) and colonoscopy in an Asian population. In a hypothetical population of 100,000 persons aged 50 years who received FIT yearly, M3 biomarker yearly, or colonoscopy every 10 years until the age of 75 years. Participants with positive FOBT or a result of "high risk" identified using the M3 biomarker are offered colonoscopy. We assumed surveillance colonoscopy is repeated every 3 years, and examined the treatment cost. A comparison of various outcome measures was conducted using Markov modelling. The incremental cost-effectiveness ratio (ICER) of FIT, M3 biomarker, and colonoscopy was USD108,176, USD133,485 and USD159,596, respectively. Comparing with FIT, the use of M3 biomarker could lead to significantly smaller total loss of cancer-related life-years (2783 vs. 5279); a higher number of CRC cases prevented (1622 vs. 146), a higher proportion of CRC cases prevented (50.2% vs. 4.5%), more life-years saved (2852 vs. 339), and cheaper total costs per life-year saved (USD212,553 vs. 773,894). The total costs per life-year saved is more affordable than that achieved by colonoscopy as a primary screening tool (USD212,553 vs. USD236,909). The findings show that M3 biomarkers may be more cost-effective than colonoscopy. [ABSTRACT FROM AUTHOR]