Washington Editor

In an attempt to simplify the process for obtaining orphan status for medications targeting rare diseases, the FDA and the European Medicines Agency (EMEA) have created a common application.

Previously, drug and biologic makers seeking orphan product designation had to submit separate applications to the FDA and the EMEA using forms requiring different information for each, which created extra financial and time-consuming burdens for the companies.

The common application will permit manufacturers to apply to both jurisdictions at the same time with one application. The common format also will help the agencies gain a better understanding of the similarities and differences in each other's application process, U.S. and European regulators said in a joint statement.

"As regulatory agencies, we are working together to try to minimize the impacts of any challenges to harmonization, to eliminate unnecessary duplication and to leverage each other's scientific and human resources so that we can each better meet the public health expectations of our citizens," the FDA told BioWorld Today in an emailed statement. U.S. regulators, the agency said, have "enjoyed a very close and very productive working relationship" with the EMEA.

Unfortunately, said Abbey Meyers, president of the National Organization for Rare Disorders, the two agencies have not yet succeeded in agreeing on uniform standards for the drug approval process.

However, she told BioWorld Today, the new common application for the orphan product designation is a "good first step" in achieving that goal.

The common application, Meyers said, will help make the orphan product designation process easier and less costly for drug and biologic makers. She noted that most orphan drug developers are small biotech firms that are struggling to make a profit.

"So it's going to save them something to be able to file one application for both Europe and the United States," Meyers said.

The new joint form contains a section for common information required by both the FDA and the EMEA and also has sections for requirements unique to each agency, regulators said.

U.S. and European regulators still will conduct independent reviews of application submissions to ensure the data submitted meet the legal and scientific requirements of their respective jurisdictions, the agencies said.

Manufacturers are required to submit two paper copies of the application to the FDA or it may be submitted electronically to the agency. The EMEA requires firms to submit one original signed and dated paper application and two electronic copies.

Firms may apply for orphan designation of an unapproved medication or for an approved drug for a new orphan indication, the joint application stated. Companies also may seek and obtain orphan designation of a product containing the same active substance as one in an already FDA-approved drug or biologic if the firm can present a plausible hypothesis of clinical superiority.

Because rare diseases affect small numbers of patients - about 25 million in the U.S. and 30 million in Europe - it is difficult for drug and biological makers to turn a profit when developing drugs to treat those conditions.

The orphan product designation was created in the U.S. under the Orphan Drug Act of 1983 to provide financial and regulatory incentives, such as protocol assistance, tax credits and marketing exclusivity, to firms that develop orphan drugs. The European Union in 1999 adopted a similar regulation.

Historically, the U.S. approval time for orphan products has been considerably shorter than for that of other drugs, according to the FDA. The reduced approval time often is due to the fact that many orphan products receive expedited review or accelerated approval because they target serious or life-threatening diseases.

The 1983 law also established a research grants program in the U.S. whereby researchers may compete for funding to conduct clinical trials to support the approval of orphan drugs and biologics for rare diseases and conditions. A product does not have to be orphan designated to be eligible for the grant program, according to the FDA.

Meyers, who was a strong advocate for the orphan drug legislation and played a central role in getting the 1983 law passed, noted that even though Congress last year authorized $25 million for the grants program, lawmakers only appropriated $15 million.

"Congress has never funded the program the way it was intended," she charged.

The FDA Amendments Act of 2007, enacted Sept. 27, has authorized $30 million per year for the program. However, Congress has yet to determine the amount it will appropriate.

The orphan drugs grants program, Meyers said, has been "wildly successful" at helping to bring breakthrough therapies for rare diseases to the market. She noted that about 320 orphan drugs have been approved since the 1983 law was enacted. "That's 12 or more approved every year," Meyers said. "That's extraordinarily successful."