Kendalle Burlin O’Connell, CEO and president of MassBio, today released the following statement after introduction of the ORPHAN Cures Act (H.R. 946):
Getting to a rare disease diagnosis is an odyssey. It takes time, several misdiagnoses, and an immeasurable emotional toll beyond a patient’s physical symptoms. And then you’re most likely to learn that there isn’t yet a treatment – 6,000+ rare diseases don’t. The Orphan Drug Act of 1983 has been an indisputable success in advancing the development of treatments for rare diseases, which represent a smaller patient pool and larger risk for investment without the proper economic incentives. Unfortunately, one of the most problematic elements of the drug price negotiation program introduced through the IRA threatens to curtail the Orphan Drug Act’s effectiveness.
The specter of negotiation significantly disincentivizes research to identify new uses for existing orphan drugs, even additional indications for rare disease, contrary to the intent of the Orphan Drug Act. Today, a bipartisan group of legislators – including Congressman Bill Keating (D-MA-10)—have reintroduced the ORPHAN Cures Act (H.R. 946) to address this unintended consequence.
MassBio represents many of the biotechnology companies most deeply invested in the rare disease space. We know the odds are already stacked against them when it comes to developing drugs for extremely small patient populations. We believe it is essential for the federal government to restore these incentives and we applaud the refiling of this legislation in the new Congress.
MassBio looks forward to marking the annual Rare Disease Day with an event on February 28, in solidarity with the 30 million Americans living with a rare disease and their families and caregivers. We call on Congress to pass this bill quickly to stand on the side of patients and innovation once again.