Session (CTI)

ISSUE: The introduction of the IRA concerns rare disease stakeholders as the only exemption from Medicare negotiations applies to orphan drugs with a single approved indication. Many medicines launched in rare conditions are eventually approved in multiple indications, ultimately allowing for a greater financial return on investment. Following the IRA’s 2022 rollout, there was a knee-jerk reaction by rare disease stakeholders, including Eli Lilly and Alnylam Pharmaceuticals which suspended clinical programs in rare diseases. The National Organization for Rare Disorders subsequently declared in an open 2023 letter to the CMS that the IRA could inadvertently reduce investment, and subsequently access, to rare disease therapies. Three years after the IRA’s introduction, this issue panel will address to what extent these concerns will impact investment, access, and pricing in rare diseases. Has the rare diseases community seen further signs of reduced investment that will result in access to fewer therapies? Or where these concerns an ‘overreaction’ with the IRA now expected to have a smaller impact on innovation and access than what was initially anticipated? OVERVIEW: Kate will provide a 10-minute overview highlighting the IRA’s influence on orphan drugs and concerns from the rare diseases community. Richard will share real-life examples demonstrating how the IRA is leading to reduced investment in orphan drugs, and its anticipated impact on pricing and patient access to new therapies. Sara will highlight Chiesi’s global rare disease’s commitment to their rare diseases pipeline and drugs, including the current impact of the IRA on US access and Chiesi’s investment in R&D. John O’Brien will discuss the IRA’s intended benefits for ensuring greater affordability, along with how CMS could mitigate any of the unintended consequences the IRA will have on innovation and access for rare disease treatments. There will be interactive polling and a 20-minute Q&A session with the audience.