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The ORPHAN Cures Act’s Promise for Rare Disease Patients

Congress has introduced legislation seeking to protect innovation for people battling rare diseases.  

Lawmakers recently unveiled the Optimizing Research Progress Hope and New Cures Act, known as the ORPHAN Cures Act. The bill would preserve the orphan drug status of medications that treat one or more rare diseases, exempting them from the Medicare drug price negotiation outlined in the Inflation Reduction Act.  

Price Negotiation, Orphan Drugs & Innovation 

The potential impact of price negotiation on drug development across the board has some advocates and experts worried. A study by Vital Transformation estimates that as many as 40% of drugs developed in the past decade could be jeopardized by price negotiation measures, and dozens of effective treatments in the future might never come to market. 

For people living with rare diseases, the risk is magnified. Potentially life-changing or life-saving medications might never be developed, or introduction may be delayed, because the patient population who could benefit from a drug is too small for drug developers to justify the investment.  

Under the Inflation Reduction Act, only orphan drugs that treat a single rare disease would be excluded from price negotiation. The provision could unintentionally discourage manufacturers from seeking additional indications, limiting treatment options for people with rare diseases. About one in five FDA-approve orphan drugs are also approved to treat additional diseases.   

Exempting rare disease treatments with multiple approved uses from price negotiation, on the other hand, can help encourage manufacturers to continue investing in groundbreaking treatments for people living with rare diseases.   

Advocates Support the ORPHAN Cures Act 

The ORPHAN Cures Act has generated widespread support from patients, providers and other stakeholders.  

The rare disease community eagerly awaits the outcome of the ORPHAN Cures Act, which will have a significant impact on patients both current and future. This legislative proposal is a vital step toward fostering innovation – safeguarding hope for people in desperate need of treatment options. 

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