ARM Foundation for Cell and Gene Medicine Releases Health Economic Impact Landscape Analysis Led by IQVIA

WASHINGTON, D.C. – August 6, 2018

Health Economic Impact Landscape Analysis is the first step to developing macroeconomic value model for regenerative medicine advanced therapies.

Washington, D.C., August 6, 2018 – The ARM Foundation for Cell and Gene Medicine (ARM Foundation) today released its first-of-its-kind Health Economic Impact Landscape Analysis of regenerative medicine advanced therapy.

The analysis includes a comprehensive review of published academic literature, health technology assessments, and value frameworks related to the global health economic impact of cell and gene therapies. Performed by IQVIA on behalf of the ARM Foundation Economic Impact Working Group, the landscape analysis is the initial step in the Foundation’s broader Economic Impact Project, which will ultimately provide a framework to measure and forecast the effect that breakthrough and potentially curative therapies will have on national and global healthcare economies.

“We are in a new era of value-based medicine. A macroeconomic analytical framework is necessary to objectively assess the clinical, economic, and social impact that regenerative therapies will have on the healthcare economy,” said Morrie Ruffin, Executive Director of the ARM Foundation.

John Doyle, Dr. P.H., Senior Vice President and General Manager, IQVIA, and Co-Chair of the Foundation’s Economic Impact Steering Committee added, “Value inputs must be identified and validated so they can be used to measure the impact of certain therapies or classes of products as novel market access models are considered. This health economic impact landscape analysis is a critical initial step to help us better understand current information gaps, what new value constructs we need to operationalize, and how we should prioritize our research to maximize value for patients.”

The landscape analysis reveals that current models fail to assess the impact of changing the treatment paradigm. Additional key findings include:

– The analysis identifies 12 additional key value inputs that should be measured to develop an accurate and useful macroeconomic value framework;
– To validate the model, consensus should be built around novel indicators such as:
– Patient-centered endpoints, including indirect costs and non-medical costs;
– Opportunity costs associated with less effective treatments;
– Patient access costs/barriers, including both financial and non-financial (sites of care, access for underserved, diagnostics coverage required to qualify, etc.); and
– System-impact value.

“It is critical to have better tools for assessing economic value,” said David Smith, JD, Pepper, Hamilton, LLP, and the Economic Impact Steering Committee’s other Co-Chair. “The increasing availability of safe and effective cell and gene therapies will offer potential benefits and outcomes that in aggregate could profoundly impact the overall cost of care for a given disease or family of diseases. Therefore, more comprehensive macroeconomic models that include additional value inputs, such as indirect patient and caregiver costs, and that look at cost-effectiveness over lifetime horizons should be built to better quantify that impact to support development and application of these therapies.”

The Economic Impact Project is led by a steering committee of 12 experts in healthcare economics and market access from academia, industry, and the patient community. The project’s first goal is to develop a comprehensive consensus value framework that will enable thought leaders and policy makers to apply key value inputs that are not captured in the narrower pharmacoeconomic models used to evaluate traditional therapies.

The next step for the Economic Impact Project will be to convene a national and international Stakeholder Value Input Consensus Panel to examine macroeconomic value inputs that can be measured and optimized for maximum utility in model development.

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About the ARM Foundation for Cell and Gene Medicine (ARM Foundation)

The ARM Foundation for Cell and Gene Medicine is an independent, 501(c)(3) non-profit organization dedicated to providing education and research that will accelerate patient access to safe, efficacious and potentially curative therapies. Its programs engage, educate and empower patients, caregivers, industry leaders and other stakeholders to help advance the science and benefits of gene therapy, gene editing, cell therapy, tissue-engineering and organ regeneration. By increasing understanding and acceptance of these transformative technologies, the Foundation hopes to involve more people in the clinical trial process and therefore help expedite the development of life-saving therapies. To learn more, visit