MassBio, nation’s largest life sciences trade association, endorses ORPHAN Cures Act

Sep 28, 2023

Bipartisan federal legislation will mitigate harmful effects of IRA on rare disease research

CAMBRIDGE, MA (September 28, 2023)—MassBio, the nation’s largest and oldest life sciences trade association, has endorsed The Optimizing Research Progress Hope and New Cures (ORPHAN Cures) Act (H.R. 5539), sponsored by Reps. John Joyce, MD (R, PA-13) and Wiley Nickel (D, NC-13). The ORPHAN Cures Act would allow orphan products to remain exempt from Medicare negotiation so long as approved uses are exclusively for rare diseases. The bipartisan legislation would also clarify that the negotiation clock starts for an orphan product only once it loses the exemption.

“The ORPHAN Cures Act addresses one of the most problematic elements of the IRA’s drug price negotiation program. MassBio, which represents many of the biotechnology companies most deeply invested in the rare disease space, is eager to work with the Massachusetts delegation and Congress to quickly advance this bipartisan bill,” Kendalle Burlin O’Connell, MassBio CEO and President, said. “Without this change, the law creates significant disincentives to identify new uses for existing orphan drugs, even additional indications for rare disease, contrary to the intent of the Orphan Drug Act. By restoring incentives to develop therapies for rare diseases the federal government will once again stand on the side of patients and innovation.”

MassBio has been outspoken on behalf of our members and the Massachusetts ecosystem about the unintended consequences of the IRA, engaging with the Centers for Medicare & Medicaid Services (CMS) throughout the implementation of the Medicare Drug Price Negotiation program. In April, MassBio submitted a letter to CMS Deputy Administrator & Director of the Center for Medicare Meena Seshamani expressing deep concern that the narrow scope of the orphan drug exclusion, combined with CMS’s overly broad QSSD definition, created a strong disincentive for developers to continue to develop new indications and formulations for existing orphan therapies. CMS ultimately did not adopt MassBio’s recommendations that would have remedied the issue.

Rare diseases, as defined by the FDA are conditions that impact fewer than 200,000 patients nationwide, and are inherently under-researched, under-diagnosed, and under-treated. Although much progress has been made since the enactment of the Orphan Drug Act 40 years ago, over 90 percent of known rare diseases do not have therapies or treatments. There has been a recent surge in the development of drugs for rare disease populations, with much of this development occurring in Massachusetts. Neglecting to resolve how the IRA treats orphan drugs would significantly curtail this development and stymie the progress made through the Orphan Drug Act.

About MassBio

MassBio’s mission is to advance Massachusetts’ leadership in the life sciences to grow the industry, add value to the healthcare system, and improve patient lives. MassBio represents the premier global life sciences and healthcare hub, with 1,600+ members dedicated to preventing, treating, and curing diseases through transformative science and technology that brings value and hope to patients. Founded in 1985, MassBio works to advance policy and promote education, while providing member programs, events, industry thought leadership, and services for the #1 life sciences cluster in the world.

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