New Report Demonstrates Potential for Cell and Gene Therapies to Provide Cost Savings

WASHINGTON, D.C. – January 10, 2020

WASHINGTON, D.C. – January 10, 2020 – The Alliance for Regenerative Medicine (ARM), the leading international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector, today announced the release of a report, “A Transformative Therapy Value Model for Rare Blood Diseases.” The report uses a refined value model to assess the impact of durable and potentially curative cell and gene therapies on health system costs.

Produced by the Marwood Group with support from ARM, the report calculates the potential cost savings that a durable cell or gene therapy could provide when treating patients with multiple myeloma, hemophilia A, or sickle cell disease. Under the current standard of care, these three disease states are projected to cost the United States healthcare system $163B per year by 2029.

Key findings from the report include:

  • The total potential savings of cell and gene therapies range from 18% to 30% in annual total disease costs and productivity. This represents an aggregate cost savings of more than $33B over ten years.
  • The modeled cost savings were highest in multiple myeloma patients ($27B in cumulative savings per year by 2029) due to the high cost of the current standard of care and to greater productivity losses experienced by the adult children caregivers of older patients.
  • Innovative financing models, such as subscription models, payment-over-time, and value-based payments, are needed to help payers offset the potentially high upfront costs of these therapies and realize longer term cost-savings.

The model used by the Marwood Group advances traditional value assessments by quantifying the gains in patient and caregiver productivity afforded by these medicines.

Janet Lambert, CEO of the Alliance for Regenerative Medicine, comments: “Over the next ten years, we anticipate dozens of new cell and gene therapies for genetic disorders and chronic conditions alike will be approved in the U.S. These therapies, many of which will likely be delivered through a single administration, have the potential to have a profoundly positive impact on patients who currently have few or no alternative treatment options. In order to realize the life-changing effect of these therapies on patients, their families and caregivers, as well as the associated financial impact on the U.S. healthcare system, it is imperative that payers, both government and commercial, and other stakeholders adopt new value-based paradigms that reflect the benefits of a durable or curative therapy in place of a palliative care approach that simply treats the symptoms, rather than the root cause of disease. The model used in this report provides an innovative framework for evaluating the potential direct and indirect cost-savings these therapies offer in comparison to the current standard of care.”

The full report and associated infographics are available here.


About The Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) is international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works an to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit