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
Innovating methods for planning, producing, and using real-world evidence
Real-world data (RWD) are a critical element of the evidence needed to help reach this goal, potentially informing a new drug’s development or providing insight for its use after it … 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
Precision for Whom? Partnering with patients to ensure that Precision Medicine targets what matters
Despite decades of growth and adaptation in patient engagement, knowing or being able to find guidance for whichmodel or approach will be most impactful in which healthcare or research context … Read More
Payer perspectives on outcomes tracking for value-based payment arrangements (VBPs)
Share the researchWith the new CMS rule changing Medicaid Best Price reporting to encourage VBPs, many options are available for which healthcare system players will collect, protect and adjudicate the … Read More
The resource navigation challenges for patients and caregivers
Share the researchWhile the problem of finding system-wide, implementable precision financing models for curative and durable therapies involves all stakeholders, patients and caregivers are directly affected by many different elements … 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
Downstream Innovation Part II
December 2019 marked the transition from the LEAPS two-year design and feasibility assessment phase to the rollout of the rheumatoid arthritis in Massachusetts (RA MA) Pilot. Some important themes surrounding … Read More