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Confronting High Costs And Clinical Uncertainty: Innovative Payment Models For Gene Therapies.
Health Aff (Millwood)
November 2023
Aaron S. Kesselheim Brigham and Women's Hospital.
Gene therapies offer potentially life-changing benefits for patients, but their unprecedented high prices exacerbate challenges for reimbursement. Payers must confront high budgetary impacts, as a large up-front payment for each patient makes it difficult to predict and absorb costs. Payers also face considerable clinical uncertainty, as evidence for efficacy and durability is limited at approval.
View Article and Find Full Text PDFKids Mod PAH trial: A multicenter trial comparing mono- versus duo-therapy for initial treatment of pediatric pulmonary hypertension.
Pulm Circ
October 2023
Departments of Pediatrics, Neurology, Anesthesiology and Critical Care Medicine, and Biostatistics Johns Hopkins University School of Medicine Baltimore Maryland USA.
Pulmonary hypertension (PH) is a significant health problem that contributes to high morbidity and mortality in diverse cardiac, pulmonary, and systemic diseases in children. Evidence-based advances in PH care have been challenged by a paucity of quality endpoints for assessing clinical course and the lack of robust clinical trial data to guide pharmacologic therapies in children. While the landmark adult AMBITION trial demonstrated the benefit of up-front combination PH therapy with ambrisentan and tadalafil, it remains unknown whether upfront combination therapy leads to more rapid and sustained clinical benefits in children with various categories of PH.
View Article and Find Full Text PDFLymph Node Metastasis in Extraperitoneal Rectal Cancer After Neoadjuvant Therapy: An Unsolved Problem?
Anticancer Res
June 2023
Department of Medical Surgical Science and Translational Medicine, Sant' Andrea Hospital, Sapienza University of Rome, Rome, Italy.
Background/aim: Thanks to the promising benefits obtained in terms of quality of life, there has been growing interest in organ-sparing approaches after neoadjuvant chemoradiotherapy (nCRT) in patients with locally advanced rectal cancer, mainly represented by transanal local excision and watch-and-wait. The main mandatory criterion is complete lymph nodal response (pN0). However, considering the reduced specificity of current radiological means in identifying one-to-one correspondence between clinical and pathological staging, the problem of underestimating lymph nodal involvement remains unsolved.
View Article and Find Full Text PDFENRICHing medical imaging training sets enables more efficient machine learning.
J Am Med Inform Assoc
May 2023
Department of Medicine, Division of Cardiology, Department of Radiology, Bakar Computational Health Sciences Institute, University of California, San Francisco, San Francisco, California, USA.
Article Synopsis
- Deep learning (DL) is increasingly used in biomedical imaging, but the scarcity of labeled data from expert annotators makes training models difficult, leading to a need for instance selection to improve model performance.
- The authors introduce ENRICH, a method designed to prioritize medical images based on the diversity they contribute to the training dataset, addressing the unique challenges posed by medical data.
- ENRICH significantly enhances classification and segmentation results by using fewer images compared to random selection, while also helping to identify errors in medical imaging datasets efficiently.
Proposing An Innovative Bond To Increase Investments In Social Drivers Of Health Interventions In Medicaid Managed Care.
Health Aff (Millwood)
March 2023
Richard Thakor, University of Minnesota.
Interventions to address social drivers of health (SDH), such as food insecurity, transportation, and housing, can reduce future health care costs but require up-front investment. Although Medicaid managed care organizations have incentives to reduce costs, volatile enrollment patterns and coverage changes may prevent them from realizing the full benefits of their SDH investments. This phenomenon results in the "wrong-pocket problem," in which managed care organizations underinvest in SDH interventions because they cannot capture the full benefit.
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