FoCUS Monthly Recap: February 2022

Eric NormanFoCUS, News


Paying for Cures Toolkit updated with new pipeline modeling and financing strategies

FoCUS released an updated and expanded version of its Paying for Cures Toolkit, which is designed to help patient groups, payers, policymakers, and drug developers make financial plans for promising but potentially costly new cell and gene therapies.

The update features: 

  • Thorough analysis of the new therapies’ potential financial impact 
  • Precision Financing Solutions describing innovative financing approaches
  • Interactive tools enabling stakeholders to calculate customized scenarios to aid decision-making

“The Paying for Cures Toolkit shares in one place a combination of insights and tools not found elsewhere, the innovative product of the FoCUS community of patients, payers, developers and policymakers.”
—Karen Geary, lead update editor; formerly, Vice President, Strategy and Innovation, MedImpact

Visit the Toolkit


FoCUS contributes to new national framework for ensuring access for high-cost medicines

Members of the FoCUS Project contributed to the new framework “Breakthrough Cures, Blockbuster Costs: Future Directions” published by The Aspen Institute and Blue Cross Blue Shield Association.The new report was co-chaired by Scott Gottlieb and Margaret Hamburg, M.D.


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. 


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

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. 


The next FoCUS Design Lab will be held on April 13–14, 2022 at MIT. By invitation only.