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Regulatory Focus™ > News Articles > Bill Would Benefit Developers of Drugs for Rare Diseases

Bill Would Benefit Developers of Drugs for Rare Diseases

Posted 24 November 2014 | By Alexander Gaffney, RAC

Bill Would Benefit Developers of Drugs for Rare Diseases

A new bill introduced in the US House of Representatives would grant existing pharmaceutical products an additional six months of marketing exclusivity if a company is able to demonstrate the product is able to treat or prevent a rare disease or condition.


The bill, the Orphan Product Extensions Now Accelerating Cures and Treatments Actof 2014 (OPEN ACT), appears to be loosely modeled off a similar plan in place for pediatric products. Under the US Food and Drug Administration's pediatric exclusivity provisions, sponsors are eligible to receive an additional six months of exclusivity if they provide data indicating their product is safe and effective in children.

The provisions, passed under the 1997 Food and Drug Administration Modernization Act (FDAMA), are meant to incentivize the development of safe and effective therapies for children. Notably, the six months of exclusivity applies (in most cases) to all approved indications for the drug, giving drug sponsors a powerful incentive to conduct studies. However, potential abuse of the program is limited by a requirement that FDA must first issue a pediatric written request (PRW) for the pediatric studies to be conducted.

For more, please read FDA's 1997 guidance document, Qualifying for Pediatric Exclusivity Under Section 505A of the Federal Food, Drug and Cosmetic Act.

Additional Exclusivity

The OPEN ACT operates on a similar concept, with some key 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. Orphan drugs are those approved to treat rare conditions, defined as affecting fewer than 200,000 people in the US.

Unlike the pediatric provisions, the approval would have to be for an entirely new indication, and not just show the product works in a rare subset of the existing population.

In addition, FDA would not have to issue a written request for a company to be eligible for the added exclusivity.

The language appears to be of a substantial benefit for marketers of rare disease drugs. Some of those drug makers, such as Vertex Pharmaceuticals, 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. However, FDA is to promulgate regulations on the extensions within two years of the bill's passage.

The bill applies to both drugs approved under Section 505 of the Federal Food, Drug and Cosmetics Act (FD&C Act) and biologics approved under Section 351(k) of the Public Health Service Act (PHSA).


Orphan Product Extensions Now Accelerating Cures and Treatments Act


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