WASHINGTON -- Proposed regulations on orphan drugs havereceived generally favorable reviews from the biotechnologyindustry, according to Marlene Haffner, director of the office incharge of writing and administering the regulations.

In an interview, Haffner said her office has conducteddiscussions with the industry about the proposed regulationsthat will govern administration of the Orphan Drug Act of 1983.

Haffner said FDA officials and industry representativesdiscussed the regulations at a Food and Drug Law Institutemeeting on March 20. "In general, the comments heard at thatmeeting were very positive," Haffner said.

The proposed rules would grant orphan status to a drug if itwere shown to be "clinically superior" to an already approveddrug, which could then lose its marketing exclusivity. Underthe law, companies have been granted seven-year exclusiverights to market drugs developed to treat rare diseases.

The period for submitting comments to the FDA ended April 1.BioWorld reported that the final regulations would be issuedlast week. However, said Haffner, once her office completesanalyzing the comments, approvals still must be obtained fromthe FDA, the Department of Health and Human Services, andthe Office of Management and Budget. Until then, the proposedregulations will be in force.

Comments submitted to the FDA generally supported theproposals, while calling for due process procedures governingthe agency's prerogative to grant and revoke marketexclusivity.

The Industrial Biotechnology Association (IBA) "stronglysupports the FDA decision to propose ... clinical superiority" asthe criterion for granting orphan status. "It strikes a fairbalance between the need to provide a meaningful marketingexclusivity incentive to pioneering developers of biotechnologyproducts and the public's interest in obtaining access to follow-on products that provide clinically important advantages," IBAPresident Richard Godown wrote.

The IBA recommended that the FDA create a procedure thatwould enable orphan drug owners to challenge incipientapprovals of products that "arguably are the same drug." It alsoasked for provision of detailed information on the FDA'sdetermination that two drugs are not the same and the right toa hearing before the approval of the second product.

Representatives of Fujisawa Pharmaceutical Co., which said itfaced challenges to its pentamidine AIDS treatment, called forclear due process procedures in determining marketingexclusivity. Amgen Inc., whose Epogen erythropoietin hasorphan status, also urged adoption of due process protections.

-- Steve Usdin BioWorld Washington Bureau

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