UPCOMING SYMPOSIUM: Ensuring Equity in Implementation of IRA Drug Price Negotiations

Tuesday, December 5, 2023 (In-Person Event), Washington, D.C.

The Innovation and Value Initiative, Alliance for Aging Research, Leavitt Partners, LLC, and the National Pharmaceutical Council are hosting a public event focused on health equity, patient engagement, and related methodological and evidence issues in CMS’ implementation of the Drug Price Negotiation Program (DPNP) as mandated by the Inflation Reduction Act (IRA).

The half-day event aims to bring together a range of perspectives, including practical on-the-ground experience and broader policy and scientific expertise, in three panel sessions. The objective is to identify critical steps that CMS, policymakers, and others could take to address equity in implementing the IRA drug price negotiations.




Rare disease represents a broad spectrum of chronic illness that can be progressively disabling and may negatively impact life expectancy. The label “rare disease” includes more than 7,000 diseases and disorders. While rare disease affects a relatively small patient population (defined as smaller than 200,000 individuals in the U.S.), more than 30 million Americans are living with rare diseases and disorder. In 2019, total economic burden in the U.S. of 379 rare diseases included in one study was estimated to be $997 billion.

Patient-centered outcomes research (PCOR), comparative effectiveness research (CER), and other real-world evidence can be difficult to identify for a variety of reasons. The burgeoning ability to identify, diagnose and develop novel therapies for rare diseases also increase the demand for new approaches in CER and tools to aid in health technology assessment (HTA) for healthcare interventions.

Using IVI’s open-source “working laboratory” approach to HTA model development, IVI, in partnership with the EveryLife Foundation for Rare Diseases, seeks to convene experts from across the patient and family, research, and healthcare communities to engage in an iterative process and planning for the development of new approaches in PCOR/CER and HTA that advance our understanding of patient-centered outcomes in rare disease.

This effort is partially supported by a Eugene Washington PCORI Engagement Award (#EASCS-26726). This project is also supported by contributions from Alexion Pharmaceuticals.

For more information about this initiative, please contact IVI’s Chief Science Officer, Rick Chapman, PhD at [email protected].


The primary objective of this initiative is to explore existing challenges for conducting comparative effectiveness research and the full range of outcomes that are important to patients living with rare disease.


This initiative follows a three-step approach:

  1. Convene a steering committee to assist and advise IVI on project implementation
  2. Conduct a targeted literature review to understand current comparative effectiveness research in rare diseases and explore common patient-centered outcomes.
  3. Host roundtable meetings with diverse stakeholders, including patient representatives, payers, and other stakeholder groups.

Key Dates