Alexandria, VA – February 7, 2023 – The Innovation and Value Initiative (IVI), a non-profit research organization committed to advancing the science, practice, and use of health technology assessment in healthcare, is announcing their Engaging Patients with Rare Disease in Identifying Meaningful Approaches to CER and Value Assessment launch as part of their Rare Disease Initiative, made possible through funding support from the Eugene Washington PCORI Engagement Awards program, an initiative of the Patient-Centered Outcomes Research Institute (PCORI) .

There are an estimated 10,000 rare diseases and disorders that are often irreversibly progressive, physically disabling, negatively impact life expectancy and effect an estimated 30 million Americans and 300 million people worldwide, a disproportionate percentage of whom are children. As therapy options continue to grow, using the value frameworks for decision-making within the US healthcare system requires consideration of the potential direction with a full range of possible patient health outcomes.

“As the approval of treatments for rare diseases accelerates, including cell and gene therapies, it will become increasingly important for decision-makers to engage with patients, caregivers and families to understand the full range of outcomes that matter to those living with rare disease,” said Rick Chapman, Chief Scientist at IVI.

This initiative is part of collective effort between IVI and EveryLife Foundation for Rare Diseases. IVI and EveryLife will serve as co-chairs of the steering committee and work together with patients and other stakeholders in a series of roundtable dialogues to inform project outcomes.

“The only therapies of value are those that are readily accessible to eligible patients. We are delighted to be collaborating with IVI to build an innovative approach that ensures the methods used to inform such decisions within our rare disease space are reflective of timely, patient-centered evidence,” said Annie Kennedy, Chief of Policy, Advocacy & Patient Engagement at the EveryLife Foundation.

Through collaboration with leading experts from across the patient and family, research, and healthcare communities, IVI will engage in a cooperative process to plan for the development of new approaches in patient research and value assessment, ultimately advancing our understanding of patient-centered outcomes in rare disease.

The Engaging Patients with Rare Disease in Identifying Meaningful Approaches to CER and Value Assessment is part of a portfolio of projects that PCORI has funded to help develop a community of patients and other stakeholders equipped to participate as partners in comparative clinical effectiveness research (CER) and disseminate PCORI-funded study results.  Through the Engagement Award Program, PCORI is creating an expansive network of individuals, communities and organizations interested in and able to participate in, share, and use patient-centered CER.

This project is partially funded through a Patient- Centered Outcomes Research Institute® (PCORI®) Eugene Washington PCORI Engagement Award (#EASCS-26726).


About IVI
The Innovation and Value Initiative (IVI) is a 501(c)(3) tax-exempt, non-profit research organization dedicated to advancing the science and improving the practice of health technology assessment through development of novel methods and the creation and application of enhanced health technology assessment models to support local decision-making needs in healthcare.

Contact: Smita Sanwardeker
Phone: 267.909.7661

PCORI is an independent, nonprofit organization authorized by Congress in 2010 to fund comparative effectiveness research that provides patients, their caregivers, and clinicians with the evidence needed to make better-informed health and healthcare decisions. PCORI is committed to seeking input from a broad range of stakeholders to guide its work.

About the EveryLife Foundation for Rare Disease
The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments, and cures.