FoCUS Monthly Recap: April 2022

Eric NormanFoCUS, News


How can self-insured employers prepare for the portfolio impact of high-cost gene therapies coming to market?

Self-insured employers currently rely on traditional stop-loss insurance to protect against unexpected high-cost claims, including those for cell and gene therapies. However, as more cell and gene therapies treatments are approved in more prevalent patient populations, this status quo approach may not be sustainable for stop-loss vendors or employers.

Get the Research Brief


Paying for Cures Toolkit helps patients understand new therapy financing impacts and payment models

The Paying for Cures Toolkit provides forecasts of the number of therapies coming to market, new payment models to support payer financial stability and patient challenges to navigate access to these treatments.

“The Paying for Cures Toolkit is an outstanding tool that educates me on information I would need to effectively advocate with a payer.”
—Brendan Hayes, Director of Education–Innovative Therapies, National Hemophilia Foundation

Visit the Toolkit


Help us improve the Toolkit with this short survey

Have you used the Toolkit to learn more about cell and gene therapies, and to develop financial strategies to make them accessible to patient? You can help us improve the Toolkit by sharing your experience with it!

Take the survey


FoCUS Spring Design Lab considers project past and future

In the first in-person Design Lab held since October 2019, FoCUS Project members scanned the landscape of current and emerging needs, including chances in Medicaid Best Price. Planning is underway to evolve FoCUS as the durable / curative therapies market grows. 

The next FoCUS Design Lab will be held on October 18–19, 2022. By invitation only. 


New Payer and Developer Perspective Study: Input invited

FoCUS is launching its third “Payer Perspectives” Study—this time to include developers’ perspectives! The survey will gather feedback from payers and developers regarding the feasibility and attractiveness of Precision Financing Solutions. We invite members of payer & developer organizations to participate in the survey. Please contact Tsega Meshesha to learn more. Share this news with your colleagues!


Kim Caldwell, Michael Ciarametaro, and Mark Trusheim hosted an education session titled “Gene Therapy Value-Based Purchasing and the Impact of Medicaid Best Price Changes” at AMCP ’22 Chicago on March 30. 

Mark Trusheim will lead the discussion “Could Gene Therapy Value-Based Purchasing Become Common Thanks to the New CMS Rule on Medicaid Best Price?” at ISPOR 2022 (May 15–18), featuring Michael Ciarametaro, Dorothy Hoffman, and Michael Sherman. Meeting registration is open now. 

Mark Trusheim will participate on a panel at the 3rd Annual Gene Therapy for Blood Disorders conference hosted by Hanson Wade, March 29–31, and at the World Orphan Drug Congress in Boston July 11–13. 


Data for modelling US projections of product approvals, patients treated, and product revenues for durable cell and gene therapies” was published in Data in Brief. The article is available with open access.