Gene therapies delivered through a single administration have revolutionized treatment possibilities for many patients living with serious or fatal conditions such as spinal muscular atrophy, hemophilia, and sickle cell disease. … Read More
Better Outcomes Tracking: A First Step In Improving Access To New Treatments
New article in Health Affairs Forefront Transformative new pharmaceutical treatments hold the promise of changing patients’ lives by slowing and curing disease, improving well-being, and advancing quality of life. Yet, … Read More
Payer and developer perspectives on alternative payment models
Expert Review of Pharmacoeconomics & Outcomes Research, Volume 24, 2024 – Issue 2Moradian, R., Meshesha, T., Trusheim, M., & Barlow, J. F. The paper reports the results of a survey … Read More
Medicaid best price reforms to enable innovative payment models for cell and gene therapies
Expert Review of Pharmacoeconomics & Outcomes Research, 23(2), 191–203.Quinn, C., Ciarametaro, M., Sils, B., Phares, S., & Trusheim, M. Cell and gene therapies promise durable benefits but face financial challenges … Read More
Broadly Engaged Team Science Comes to Life in a Design Lab
The objective of broadly engaged team science is to bring a diverse group of stakeholders into a project early and continue to include their expertise throughout to inform and propel … Read More
Data for modelling US projections of product approvals, patients treated, and product revenues for durable cell and gene therapies
Data in Brief, 41, 107891.Young, C. M., Trusheim, M., & Quinn, C. (2022). The recent marketing approval of several durable gene and cell therapies (2017-2020), together with observations that 7,000 … Read More
Durable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues
Drug Discovery Today (2021). DOI: 10.1016/j.drudis.2021.09.001C. Young; C. Quinn; M. Trusheim. Durable cell and gene therapies potentially transform patient lives, but payers fear unsustainable costs arising from the more than … Read More
Precision Reimbursement for Precision Medicine: Using Real-World Evidence to Evolve From Trial-and-Project to Track-and-Pay to Learn-and-Predict
Basic scientists and drug developers are accelerating innovations toward the goal of precision medicine. Regulators create pathways for timely patient access to precision medicines, including individualized therapies. Healthcare payors acknowledge … Read More
Tracking the CAR-T Revolution: Analysis of Clinical Trials of CAR-T and TCR-T Therapies for the Treatment of Cancer (1997–2020)
Ivica NA, Young CM. Healthcare. 2021; 9(8):1062. Chimeric antigen receptor and T-cell receptor (CAR-T/TCR-T) cellular immunotherapies have shown remarkable success in the treatment of some refractory B-cell malignancies, with potential … Read More
Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop
Forum on Drug Discovery, Development, and Translation and the Roundtable on Genomics and Precision Health of the National Academies of Sciences, Engineering, and Medicine, March 8, 2017 Read the meeting … Read More
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