Ten years since the orphan drug legislation was passed, FDAhas finally issued official regulations. For companies developingor producing orphan drugs, it will be business as usual.

For example, there will remain no profit limit on orphan drugs,nor will licenses be revoked if new indications are approved.

One biotech company representative, who requestedanonymity, said the regulations governing what constitutes a"same drug" as one already marketed are virtually identical tointerim regulations. The same is true, the source said, forprovisions regarding clinical superiority.

And Donna La Voie, spokeswoman for Genzyme Inc., said, "Offthe cuff, (the regulations do not) appear to impinge onGenzyme's orphan drugs."

Ceredase, one of the most expensive and controversial orphandrugs, earned Genzyme $100 million in 1992.

But Bill Small, executive director of the Association ofBiotechnology Companies, criticized the regulations as too lax."If there are two or more companies working on the same drug,obviously it isn't an orphan," he said, adding that "a couple oforphan drugs certainly are blockbusters."

Small also faulted the new regulations for failing to requirethat orphan status be revoked if approval of new indicationsraises the number of patients beyond the 200,000 statutorylimit.

And clinical superiority provisions should be liberalized so thatif 10 percent of an afflicted population fails to benefit from anorphan drug, but responds to a "same drug," the latter couldgain orphan status, said Abbey Meyers, director of the NationalOrganization for Rare Disorders.

"The regulations look like they are trying to protect monopoliesof the most lucrative drugs," she said.

The uncertainties surrounding orphan drug regulations havescared off at least one potential manufacturer. Edward West,director of corporate communications for Eli Lilly and Co., saidhis company has gotten out of the orphan drug businessbecause there have been too many threats of tinkering withthe legislation.

"We operate on very long time lines of 10 to 12 years to bringa new drug out, at a cost of $350 million," West said. "We can'tcope with the danger of having the rules change after we'vemade that kind of investment."

Although he said he'd prefer to see orphan status revokedwhen the number of patients exceeds 200,000 and when twoor more companies are developing a drug, Small has helpedSen. Nancy Kassebaum, R-Kan., with the Kassebaum-(Howard)Metzenbaum (D-Ohio) bill that would cap orphan profits.

Small said he anticipated the bill will be reintroduced. Thistime, he said, the bill's changes will benefit from Kassebaum'snew position as ranking Republican on the Senate Labor andHuman Resources Committee.

-- David C. Holzman Special to BioWorld

(c) 1997 American Health Consultants. All rights reserved.