Managing the challenges of paying for gene therapy: strategies for market action and policy reform in the United States

Eric NormanFoCUS, Publications

New open access policy analysis from Institute for Clinical and Economic Review (ICER) and NEWDIGS Initiative at Tufts Medical Center analyzes the challenges and potential policy options for paying for … Read More

Implementation Brief: Considerations for developers in applying the Multiple Best Price (MBP) rule to improve access to novel therapies

Eric NormanFoCUS, Implementation Briefs

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

Managing the Challenges of Paying for Gene Therapy: Strategies for Market Action and Policy Reform

Eric NormanFoCUS, Publications, Whitepapers

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

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

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

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

Tools for Implementation of Precision Financing Solutions Within Medicaid Plans

Eric NormanFoCUS, Whitepapers

Implementation of precision financing solutions is a challenge for state Medicaid payers given the complexity of the environment. This white paper provides example templates to support state Medicaid plans, manufacturers, and … Read More

Payer Perspectives on Financing and Reimbursement of One-time High-cost Durable Treatments

Eric NormanFoCUS, Whitepapers

An online 15-question survey was conducted with clinical and financial health plan leaders from US payers between September 2018 and April 2019. The survey was focused on assessing payer perspectives … Read More

Tracking Medicaid Coverage of Durable Cell and Gene Therapies

Eric NormanFoCUS, Research Briefs

Durable cell and gene therapies represent a unique challenge to the US healthcare insurer/payer landscape, given the one-time treatment and associated high upfront cost but potentially durable outcomes. This study … Read More