On July 9, 2024, the House of Representatives Committee on Appropriations released its Fiscal Year 2025 draft report, which includes important language regarding protecting access to genetic therapies such as antisense oligonucleotides (ASOs). While the IRA exempts drugs from the negotiation process for the first seven years (small molecule—prescription drugs) and eleven years (large molecule—biologics), these exemptions do not address how certain rare disease treatments, such as ASOs will be treated.
Genetic therapies, like ASOs can be more expensive and time-consuming to develop because they modify the gene expression to ensure the gene either produces the correct protein or stops the gene from producing the wrong protein. As of 2022, only 14 ASOs had been approved by the FDA, therefore, Congress must continue to protect access to these novel treatments which may help address genetic disorders in ways other therapeutics (small molecule and biologics) cannot. The below Congressional report language recognizes the need to protect access to these novel treatments:
Genetically Targeted Technologies.—Genetically targeted drugs, defined for the first time in the 21st Century Cures Act (P.L. 114– 255), include a set of complex small molecule products that target predominantly rare, genetic-based diseases, but can also target broader populations in novel ways. These genetically targeted technologies are in the infancy of their commercialization and subjecting these highly complex drugs to a negotiation timeline intended for simpler drugs so early in their commercialization will disproportionally impact these innovative ways to treat diseases with high unmet medical need. The Committee encourages CMS to work with Congress to align the timeline for which drugs made with genetically targeted technologies are eligible for negotiations, equivalent to that of large molecules.
Read the report (see page 147).
Last Updated on August 13, 2024 by Aimed Alliance
