Our article compares the clinical development success rates of durable cell and gene therapies (dCGTs) from 1988 to 2023 with BIO and IQVIA’s published benchmarks for all therapeutic modalities. As … Read More
Ask PAM: “How many?”
Individual Indication Workbook in actionHow many treatment-eligible people are in my population?The Pipeline Analysis and Modeling (PAM) in the NEWDIGS Paying for Cures Toolkit maintaints a unique, detailed, indication-by-indication analysis … Read More
Are Cell and Gene Therapy programs a better bet?
Compared to traditional drug development programs, do durable cell and gene therapy (CGT) programs experience higher or lower clinical success rates? Estimating the Probability of Technical and Regulatory Success (PTRS) … Read More
How can self-insured employers prepare for the portfolio impact of highcost gene therapies coming to market?
Self-insured employers (SIEs) currently rely on traditional stop-loss insurance to protect against unexpected high-cost claims, including those for cell and gene therapies. Given that current approvals are in rare diseases, … 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
Updated projection of US durable cell and gene therapies product-indication approvals based on December 2019 development pipeline
Ongoing pipeline modeling by the MIT NEWDIGS FoCUS team projects 10X growth from July 2020 levels, but with large uncertainties. Download FoCUS Research Brief 2020F207v51Share the research
Tracking the CAR-T revolution: Analysis of clinical trials of CAR-T and TCR therapies for the treatment of cancer
Chimeric antigen receptor and T-cell receptor (CAR-T/TCR) cellular immunotherapies have shown remarkable success in the treatment of some refractory B-cell malignancies, with potential to provide durable clinical response for other … Read More
Estimating the Clinical Pipeline of Cell and Gene Therapies and Their Potential Economic Impact on the US Healthcare System
Value in Health, June 2019Casey Quinn, Colin Young, Jonathan Thomas, and Mark Trusheim Cell and gene therapies promise durable clinical benefit from a single treatment course. High upfront reimbursement for … Read More
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