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House Reviews the ORPHAN Cures Act

On Feb. 29, the House Energy and Commerce Subcommittee on Health held a hearing to review a number of bills that impact those living with rare diseases. Among them was H.R. 5539, the Optimizing Research Progress Hope and New Cures Act (ORPHAN Cures Act).


Since the enactment of the Orphan Drug Act of 1983, we’ve witnessed a revolution in rare disease treatment options for the nearly 30 million Americans living with these conditions. In the 1980s, fewer than 40 medications for rare disease were available. Fast forward to today, more than 600 drugs have been approved by the FDA to treat more than 1,000 rare conditions, with another 700 in various stages of development. However, this extraordinary advancement will be impeded without fixes to the Inflation Reduction Act’s (IRA) Medicare Drug Pricing Negotiation Program. That’s what H.R. 5539 seeks to do.


While the IRA extends exemptions from the price-setting provisions to products designated and approved for a single rare disease or condition, the ORPHAN Cures Act represents a crucial fix by allowing orphan drugs, regardless of the number of orphan drug designations or indications for rare diseases the medicine has, to be exempt from price-setting selection. Further, the bill would provide clarity by ensuring the clock for price-setting provisions would start only when the orphan-designated drug is approved for a non-rare condition.


These changes are essential. Today, more than 45% of all orphan-designated drugs are approved for additional rare conditions beyond the initial indication, offering hope to additional patients and families. However, these additional uses require investment and typically another 4.5 years of testing before they can be approved and made available to patients. Without changes to the IRA, analysts predict 41% fewer orphan drugs will be approved and subsequent post-approval research in rare conditions will be abandoned.


Despite the achievements of the Orphan Drug Act, a stark reality remains. More than 90% of all rare conditions still lack approved treatment options. Preserving incentives to pioneer new treatments, explore additional indications for orphan drugs, and seek approval for other rare conditions is critical if we want to continue progress for all patients, irrespective of the rarity of their disease.


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