Making Cell and Gene Therapies Financially Accessible and Sustainable

Eric NormanFoCUS, News

CAMBRIDGE, Massachusetts, December 5—The Financing and Reimbursement of Cures in the US Consortium (FoCUS) launched the Paying for Cures Toolkit today. The free online resource helps payers, patient advocates, drug developers, policymakers, and other healthcare system stakeholders explore strategies to sustainably finance new cell and gene therapies with durable—and potentially curative—effects.

Such durable and curative treatments present the possibility of a short, sometimes single dose, treatment regime that provides lasting benefits to patients. Yet, these therapies pose significant financing challenges for many consumers and payers.

The Toolkit (available enables users to:

  • Understand the issues, possible solutions, and potential implementation challenges
  • Easily access FoCUS research and findings via interactive tools
  • Jump start needs evaluation, customize options, and simulate the potential financial impact of new therapies on their business

The NEWDIGS initiative at MIT formed FoCUS as a cross-stakeholder consortium to assist in bringing new therapies to patients faster in ways that are sustainable for all players in the system. The Toolkit, offered as a public service in this mission, provides information and simulations to assist in planning and implementing financing options.

“The simulation and analysis capability in the Toolkit provides significant insights to an insurer like Harvard Pilgrim with regard to what to expect over the next few years, as well as information to inform our decisions around when to employ solutions such as performance-based annuities or milestone-based contracts,” said Michael Sherman, MD, MBA, Chief Medical Officer, Harvard Pilgrim Health Care. “It is a useful complement to our internal analyses.”

The Toolkit also empowers patient advocacy organizations, eager to make new therapies accessible to their communities. “For patients, the Toolkit demonstrates there are viable financing solutions for these therapies. By understanding the solutions available, patients are better positioned to advocate for themselves,” said Durhane Wong-Rieger, President & CEO, Canadian Organization for Rare Disorders.

“Helping implement, not just envision, precision financing solutions for durable cell and gene therapies is a critical part of the FoCUS mission,” said Mark Trusheim, Strategic Director, NEWDIGS. “The Toolkit can help ensure fast and appropriate patient access to these transformative therapies by helping payers determine how to cover and creatively pay for these new therapies in ways that also work for providers, developers, and intermediaries.”

“As a pharmacy benefit manager, it is critical to understand the impact new therapies may have on our clients and members,” said Karen Geary, Vice President of Strategy and Innovation, MedImpact Healthcare Systems, Inc. “The Toolkit will be useful in terms of estimating what our costs could be for known populations as well as understanding how to underwrite unknown risk. The Toolkit is a valuable planning resource.”

“I’m excited to use the Toolkit to better simulate the potential impact of the launch of durable and curative cell and gene therapies over the next 5 to 10 years,” said Mike Ciarametaro, Vice President of Research, National Pharmaceutical Council. “Leveraging the FoCUS clinical trial research and pipeline analysis, the Toolkit is a great resource for payers, developers, and policymakers.”

“The Toolkit helps educate our stakeholders on the financial challenges of paying for cell and gene therapies, an issue we need to better understand,” said Esther Krofah, Senior Director at FasterCures, a Center of the Milken Institute, which convenes a cross section of stakeholders to improve health outcomes from biomedical innovation. “We look forward to using this valuable resource.”

Visit the Paying for Cures Toolkit at

About the FoCUS Consortium
The MIT NEWDIGS consortium FoCUS Project (Financing and Reimbursement of Cures in the US) seeks to collaboratively address the need for new, innovative financing and reimbursement models for durable and potentially curable therapies that ensure patient access and sustainability for all stakeholders. Our mission is to deliver an understanding of financial challenges created by these therapies leading to system-wide, implementable precision financing models. This multi-stakeholder effort gathers developers, providers, regulators, patient advocacy groups, payers from all segments of the US healthcare system, and academics working in healthcare policy, financing, and reimbursement in this endeavor.

MIT NEW Drug Development ParadIGmS (NEWDIGS) is an international “think and do tank” dedicated to delivering more value faster to patients, in ways that work for all stakeholders. NEWDIGS designs, evaluates, and initiates advancements that are too complex and cross-cutting to be addressed by a single organization or market sector. Its members include global leaders from patient advocacy, payer organizations, biopharmaceutical companies, regulatory agencies, clinical care, academic research, and investment firms. For more information, visit ###