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

Precision Financing challenges for solid tumor adoptive T-cell therapies

Eric NormanFoCUS, Research Briefs

Durable gene and cell 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. Previous research … Read More

Downstream Innovation Part I

Eric NormanLEAPS, Research Briefs

There is a growing recognition of the importance of augmenting product- focused learning with disease-focused learning, incorporating the value of advances in real-world evidence (RWE). In LEAPS, we aim to … 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

Transforming Real-World Evidence in Biomedical Innovation: Advancing the Design of Collaborative Disease-Focused Learning Ecosystems

Eric NormanLEAPS, Research Briefs

The LEAPS methodology begins by answering two structured questions – WHAT evidence to produce, and HOW to produce it, as outlined in this two-part Research Brief series: • Part I: … Read More

State Insurance Regulations Regarding Benefit Design (Deductible and Co-Pay Waivers)

Eric NormanFoCUS, Research Briefs

FoCUS has identified an issue that may be a barrier to timely patient access to durable therapies. The advent of expensive, one-time durable therapies will require changes in private payer … Read More

Baking the Cake: A decision driven framework for planning fit-forpurpose evidence across stakeholders

Eric NormanLEAPS, Research Briefs

 The LEAPS methodology begins by answering two structured questions: what evidence to produce, and how to produce it, as outlined in this two-part Research Brief series: • Part I: Baking … Read More

Payer Perspectives Survey

Eric NormanFoCUS, Research Briefs

A survey of 77 payers representing over 280 million lives, completed in April 2019, explored perspectives concerning financing challenges and management strategies associated with durable cell and gene therapies. Download … Read More