Proposed rule advances longstanding priority for gene therapy stakeholders
FOR IMMEDIATE RELEASE
July 20, 2020
CONTACT: Macey Kostbar, msevcik@gene-therapies.org
WASHINGTON, DC (July 20, 2020) – Today, the Institute for Gene Therapies (IGT) submitted comments to the Centers for Medicare & Medicaid Services (CMS) on the proposed rule that makes changes to the Medicaid Drug Rebate Program (MDRP) to facilitate value-based purchasing (VBP) arrangements.
Advancing VBP has been a high priority for stakeholders in the gene therapy field over the course of the past decade. However, a primary hurdle has been the Medicaid Best Price policy. The proposed rule is significant in that it sets forth a potential pathway for addressing Best Price as it applies to VBPs and setting forth a proposed definition for qualifying VBP arrangements.
“IGT commends CMS for taking action to ameliorate price reporting barriers to VBP arrangements,” said IGT Chairman Erik Paulsen. “The Proposed Rule is a pivotal moment in the transformation of price reporting methodologies. These innovative arrangements are absolutely needed to help patients living with devastating conditions benefit from the incredible potential of gene therapies.”
In its comments, IGT also highlighted operational considerations that are important for CMS to address to ensure that the proposed approach results in meaningful change and progress.
On Monday, July 13, IGT hosted a webinar featuring recognized experts in the field of gene therapy policy and advocacy. IGT Chairman Erik Paulsen, led the discussion about how this rule will impact patient access to and coverage of gene therapies. The webinar speakers included:
Lauren Randall Buckley, Director of Health Policy & Advocacy, IGT
Stuart Portman, Health Policy Advisor, U.S. Senate Finance Committee
Jamie Sullivan, Director of Policy, EveryLife Foundation for Rare Diseases
Mark Trusheim, Strategic Director, MIT NEWDIGS
Thomas Evegan, VP, Integrechain
Stuart Portman, Health Policy Advisor for the U.S. Senate Finance Committee noted that the work in Congress continues. “I have had a number of conversations when it comes to gene therapy – both with the FDA along with the payer side at CMS, and now with Senator Grassley’s very strong work trying to bring to the forefront conversations related to gene therapy on Capitol Hill," Portman said. “This has been a great endeavor – we have made lot of progress over the last 18 months and we anticipate more progress to be made.”
Jamie Sullivan, Director of Policy for the EveryLife Foundation for Rare Diseases, provided the patients’ perspective. “We know that many of the transformational therapies that have been recently approved have faced access barriers that end up prohibiting or delaying life changing treatments from reaching patients,” Sullivan stated. "We are pleased to see CMS recognize the need to advance development of value-based payment arrangements. At the same time, we recognize the complexity of what is being proposed and would like to see additional details and clarity prior to finalization of any model. We also want to emphasize that patient communities must play a central role in these discussions. We now must engage with patients, payers and value assessors. This should be done early, often and with the right evidence.”
Mark Trusheim, Strategic Director of MIT NEWDIGS works closely with a cross-section of gene therapy stakeholders. He offered, “We believe these valuable therapies, no matter how that negotiation comes about, are going to wind up causing some financial challenges and we want to hear from all our different stakeholders so that we design solutions that work for everyone.”
IGT will continue to host discussions on policy developments in the gene therapy space with key stakeholders. To watch the full discussion, click here. To read IGT’s comments to CMS, click here.
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About the Institute for Gene Therapies
The Institute for Gene Therapies brings together experts from across the healthcare system to advocate for a modernized policy framework that encourages innovations and promotes patient access to the treatments they need. We represent innovators and patients, business leaders and academics working to ensure policies reflect medical advances, creating a new reality for patients. Follow us on Twitter @gene_therapies.