WASHINGTON -- Proposed rule changes governing the OrphanDrug Act have come under fire from a group of scientists whowarn that the new rules would adversely affect thedevelopment of biotech drugs.
The Food and Drug Administration is set to issue new rulesthis week that would allow new drugs to be approved even ifsimilar drugs had already been granted a seven-year exclusivemarket under the orphan act.
Under the proposed rules, companies submitting drugs withsimilar structures would need to show that theirs are"clinically superior" in order to break the orphan drugmonopoly.
The comment period on the proposed regulations ended April 1.
In a letter to the FDA, 21 academic scientists said, "Theresolution of this issue will have an enormous impact on theentire biotechnology industry." Scientists in the group includedPeter Albersheim, director of the Complex CarbohydrateResearch Center at the University of Georgia; Minoru Fukuda,director of the glycobiology and chemical program at the LaJolla Cancer Research Center Foundation; and Robert L. Hill,chairman of the biochemistry department at Duke UniversityMedical Center.
They said orphan drug exclusivity is especially important tobiotechnology because "patent protection for many of thesedrugs is uncertain, leaving exclusivity as the only remainingmarketing protection." The scientists also pointed out that "adisproportionate number of orphan drugs are biotechnologyproducts."
In an accompanying cover letter, Stuart Kornfeld, professor ofmedicine at Washington University School of Medicine in St.Louis, wrote that the "FDA's most recent pronouncements aboutthis issue suggest that the agency is ready to announce apolicy which would consider drugs to be different products ifthey differed in any respect of their structure.
"Since it is easy to evade exclusivity by inducing changes thatwould make FDA consider a protein drug to be 'different,' thevalue of exclusivity as an incentive for the development ofprotein drugs for rare diseases would be negligible."
Kornfeld said, "I believe this policy would represent a seriouserror because it would have the effect of writing most proteinproducts out of the Orphan Drug Act."
The researchers urged the FDA to establish an independentadvisory panel to investigate the issue of similarity.The only corporate comments involved a debate betweenGenetics Institute Inc. and its marketing partner, Chugai-Upjohn Inc., with Amgen Inc. over the impact of the new ruleson erythropoietin (EPO), an orphan-designated drug used totreat anemia in kidney dialysis patients.
Genetics Institute and Chugai-Upjohn, whose EPO patent wasrevoked in federal court in March, contended that the new rulesshould allow the FDA to grant marketing approval for theirEPO, which would compete with Amgen Inc.'s orphan-approvedversion of the drug.
-- Steve Usdin BioWorld Washington Bureau
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