Bill Aimed to Extend Market Exclusivity for Rare Disease Drugs

The House Energy and Commerce (E&C) Committee released an updated draft of its 21st Century Cures Act, reintroducing a provision that would extend market exclusivity for drugs modified to treat rare diseases.

House lawmakers agreed to extend drug exclusivity perks for manufacturers on their products by six months if they are altered to treat a rare disease. The provision, known as the Orphan Product Extensions Now (OPEN) Act, was originally introduced last November.

Under the bill, existing drugs would be granted an additional six months of market exclusivity if a company is able to demonstrate that the product is able to treat or prevent a rare disease or condition. During that time, the US Food and Drug Administration (FDA) would be prohibited by law from approving a generic version of the drug. The bill is based on a framework currently in place for products among pediatric patients, known as the Pediatric Research Equity Act (PREA), which requires some companies to conduct studies evaluating the safety and efficacy of their products in pediatric populations.

Like the PREA, the OPEN Act would permit the FDA to extend a drug’s exclusivity by six months if the drug was not originally approved for the indication. However, under the OPEN Act, the FDA would not have to issue a written request for a company to be eligible for added exclusivity, which is required under PREA. The bill could accelerate drug development, since it provides incentives to manufacturers to repurpose drugs that are already known to be safe and effective in humans decreasing the chances that a drug fails a clinical trial based on safety issues.

The FDA defines a rare condition as one that affects fewer than 200,000 people in the US.

In a second draft of the bill, released in April, the language regarding rare diseases was removed. After rare disease advocacy groups expressed their discontent, the legislators re-introduced the language into the third draft version of the bill. Other provisions in the bill, including an extra $10 billion in research funding, remain.

Source: The House Energy and Commerce

Last updated: 5/14/15; 2:45pm EST


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