This brief highlights some key steps and considerations for biopharmaceutical developers of novel, high-value therapies seeking to successfully implement CMS’s Multiple Best Price rule. The rule, which went into effect … Read More
Implementation Brief: Understanding access issues for new therapies
Our first implementation brief outlines several major barriers to patient access for novel cell and gene therapies. It also describes how providers, payers, and developers may overcome these barriers, with … Read More
Emerging market solutions for financing and reimbursement of gene therapies for sickle cell disease: Why do payment innovation?
The FDA has approved two cell and gene therapies for sickle cell disease, marking a significant development for the largest population condition to date. However, the high treatment costs, … Read More
Analyzing 340B and ASP interactions: Do Federal program rules disincentivize the use of VBCs, despite Medicaid Best Price reform?
This paper describes the interactions among Medicaid Best Price rebate calculations and those for 340B Ceiling Prices and Average Sales Prices which can create disincentives for providers and therapy developers … 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
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
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