ORPHAN DISEASE DRUGS

Two hundred thousand dollars is a lot of money for a year’s supply of a biotechnology drug, even considering that it can save a life and is costly to produce. The Democratic-controlled Congress, with support from several Republicans, including Maine’s Sen. Susan Collins, is considering legislation that could cut the prices of such drugs.

Much attention has centered on Cerezyme, one of the most expensive of the biotech drugs. At an average price of $200,000 a year, it can slow the course of Gaucher disease, a so-called “orphan disease,” affecting only about 10,000 people worldwide. It results from a hereditary deficiency of a vital enzyme. Genzyme Corp., a biotech firm based in Cambridge, Mass., produced this genetically engineered form of the crucial enzyme after the National Institutes of Health had spent more than 25 years of research to develop the therapy.

The Senate bill, as well as a similar House measure introduced by Rep. Henry Waxman, D-Calif., are analogous to a 1984 law that has saved consumers billions of dollars by authorizing the Food and Drug Administration to approve low-cost generic versions of patented conventional drugs.

When Sen. Collins announced her support for the bill she cited widespread agreement that “legislation creating a pathway for approval of generic biologics is critically important to assure access to more affordable drugs.”

She noted that the bill establishes a rigorous, case-by-case scientific process for approving these products to make sure that they are as safe and effective as their brand-name counterparts.

She said the bill had been endorsed by the AARP, Aetna, the AFL-CIO, the Coalition for a Competitive Pharmaceutical Market, Consumers Union, General Motors, Generic Pharmaceutical Association, Express Scripts Inc. and the National Organization of Rare Disorders.

Still, there are skeptics. An April 10 editorial in The Wall Street Journal called the bill “an attempt to fit a new problem into a framework for which it wasn’t designed.” It said that the biologics, being made from living cells, will not be biochemically identical to the original. It said that variations could bring side effects and cited a 1990 incident in which a minor change in a licensed version of a dialysis drug triggered serious reactions in several hundred patients.

Public hearings on the bills should explore this safety issue and get independent scientific testimony as to any difficulties and hazards in trying to replicate a biological drug into a synthetic pill.

Costs of producing these patented biologics should also be explored. Dan Quinn, Genzyme’s corporate communication director, says the company does not disclose its cost of developing a drug. He says the NIH version wasn’t a successful therapy, but Genzyme improved and developed it and found how to produce it on a commercial scale.

Synthetic substitutes would introduce competition into a field that now is limited to a few companies. But approval of new and cheaper pills must be done with careful respect for safety considerations as well as the rights and interests of firms that are seeking cures for orphan diseases.