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
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
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
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
The Janus initiative: A multi-stakeholder process and tool set for facilitating and quantifying Adaptive Licensing discussions
Health Policy and Technology, December 1, 2014Mark R. Trusheim, Lynn G. Baird, Sarah Garner, Robyn Lim, Nitin Patel, and Gigi Hirsch Successfully advancing Adaptive Licensing requires coordinated planning and action … Read More
Legal Foundations of Adaptive Licensing
Clinical Pharmacology & Therapeutics, September 1, 2013Oye, L. G. Baird, A. Chia, S. Hocking, P. B. Hutt, D. Lee, L. Norwalk, and V. Salvatore In April 2012, MIT’s Center for … Read More
Curing Consortium Fatigue
Science Translational Medicine, August 28, 2013Magdalini Papadaki and Gigi Hirsch The complex pathology of consortium fatigue provides diagnostic data on how to improve collaboration in biomedical innovation. Read the publication
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