Analyzing 340B and ASP interactions: Do Federal program rules disincentivize the use of VBCs, despite Medicaid Best Price reform?

Eric NormanFoCUS, Research Briefs

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?

Eric NormanFoCUS, Research Briefs

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

Eric NormanLEAPS, Research Briefs

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?

Eric NormanFoCUS, Research Briefs

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

Eric NormanLEAPS, Research Briefs

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)

Eric NormanFoCUS, Research Briefs

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

Eric NormanFoCUS, Research Briefs

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

Eric NormanFoCUS, Research Briefs

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

Eric NormanLEAPS, Research Briefs

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

Impact of FDA Guidelines on Communication between Developers and Payers on Metrics in Performance-Based Agreements

Eric NormanFoCUS, Research Briefs

The FDA regulates communications from pharmaceutical developers to ensure that statements about products are appropriately supported by evidence. In this Research Brief, we explore the possible impacts of these regulations … Read More