Marketing Exclusivity Provisions for Rare Disease Drugs Back in 21st Century Cures Bill

Posted 13 May 2015 By Alexander Gaffney, RAC


An updated draft of the House Energy and Commerce (E&C) Committee's 21st Century Cures Act has reintroduced a provision which would extend marketing exclusivity for some drugs repurposed to treat rare diseases or conditions by six months.


The provision, popularly known as the Orphan Product Extensions Now Act, was originally introduced in the US House of Representatives in November 2014. Under the bill, existing pharmaceutical products would be granted an additional six months of exclusivity if a company is able to demonstrate that the product is able to treat or prevent a rare disease or condition.

The OPEN Act is heavily based on a framework in place for pediatric products known as the Pediatric Research Equity Act (PREA). Originally passed under the Food and Drug Administration Modernization Act (FDAMA), PREA requires some companies to conduct studies regarding the safety and efficacy of their products in pediatric populations. If a company successfully shows its product to be safe and effective in these populations, all of its products' remaining marketing exclusivity is eligible for a six-month extension.

The OPEN Act

The OPEN ACT operates on a similar concept, with some differences.

Like the pediatric exclusivity provision, FDA would be permitted to extend a drug's exclusivity period by six months if a product—initially not approved as an orphan drug product—is later approved as such. As defined by the Federal Food, Drug and Cosmetic Act (FD&C Act), orphan drugs are those approved to treat "rare" conditions affecting fewer than 200,000 people in the US.

Unlike PREA exclusivity, which is only granted if a company submits a pediatric written request (PWR), FDA would not have to issue a written request for a company to be eligible for added exclusivity under the OPEN Act.

The legislative language indicates the bill is intended to repurpose drugs already known to be safe and effective in humans, and to determine if they are effective at treating other indications. In theory, that could accelerate drug development by decreasing the chances that a drug fails a clinical trial due to safety issues.

The language could be of a substantial benefit for marketers of rare disease drugs. Some of those drug makers have already adopted iterative drug development pipelines in which a single drug is tested (and approved) in multiple rare disease subsets. Under the OPEN ACT's language, marketing exclusivity for existing products could be extended by an additional six months.

The bill does not make clear if there is a limit to the number of six-month extensions a product is eligible for. OPEN ACT exclusivity would, however, be in addition to other types of exclusivity, such as pediatric exclusivity. FDA is to promulgate regulations on the extensions within two years of the bill's passage.

The inclusion of the legislative language in the 21st Century Cures Act comes after it was stripped from a second draft of the legislation released on 30 April 2015. After an outcry from rare disease groups, including the advocacy group Rare Disease Legislative Advocates (RDLA), legislators re-introduced the language into the third and latest draft of the bill.


Latest Draft of the 21st Century Cures Act (Section 2151)

Read Regulatory Focus's Regulatory Explainer on the 21st Century Cures Act

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Categories: Biologics and biotechnology, Drugs, Orphan products, News, US, FDA

Tags: Congress, 21st Century Cures Initiative, 21st Century Cures Act, Legislation, Bill, Energy and Commerce Committee, E&C

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