By Lisa Seachrist

Washington Editor

WASHINGTON — There was a time when FDA approval signaled a drug's or a biologic's graduation from an experimental therapy to a safe and effective new treatment.

Those days may be ending, as some managed health-care plans have resorted to keeping the "investigational" label on newly approved drugs that lack data related to long-term outcomes.

Such policies prompted a group of physicians to publish a paid-for open letter to Congress in the congressional newspaper, Roll Call. "Increasingly," the letter declares, "managed care organizations — not FDA — are the obstacle to patient access to safe and effective treatments."

That letter, the publication of which was funded by Cambridge, Mass.-based Genzyme Corp., urges Congress to add a provision in any managed care legislation prohibiting health plans from deeming FDA-approved drugs "investigational."

"We are really quite concerned," said Lisa Raines, vice president for government relations for Genzyme. "We fear that, when there are new FDA-approved treatments that are more expensive, they will be excluded from coverage at the outset as experimental or investigational, without a federal standard establishing that FDA-approved therapies may no longer be considered experimental treatments."

Genzyme became involved with the issue when it began to hear from orthopedic surgeons that Carticel, the company's cellular therapy approved in August 1997 for restoring articular cartilage in knees, was considered an experimental therapy that did not merit coverage by some health-care plans.

HMOs Skirt Questions, Claiming 'Proprietary' Data

Upon further investigation, Raines said, the company discovered that a therapeutic evaluation committee for the Blue Cross plans had decided there was insufficient evidence supporting Carticel use in treating osteoarthritis in the knee. As a result, some Blue Cross plans, as well as some other managed health care plans, refuse to cover treatment with Carticel.

Until Carticel's approval, the only treatment for damaged cartilage in the knee was a surgical procedure that created scar tissue to cushion the joint. Carticel, on the other hand, involves harvesting cartilage cells from a patient, growing those cells in culture and implanting the new cartilage into the patient's knee. (See BioWorld Today, Aug. 26, 1997, p. 1.)

When the company asked health care plans to show them the data used to prove that Carticel's efficacy was in doubt, some plans refused, saying the data was "proprietary," Raines said. Some plans have refused to cover Carticel because it isn't the "accepted practice of medicine," she added.

"We think that this refusal is especially egregious when there is only one FDA-approved therapy for treating people with this sort of injury," Raines said. "They are basically saying that, notwithstanding FDA approval, they can consider a new treatment experimental."

This is not to say Genzyme and the signatories of the letter believe health-care plans should be required to cover every approved therapy. Raines noted many health plans state in their policy that they will not cover certain procedures such as cosmetic surgery, or drugs such as contraceptives. If a company excludes Carticel, Raines said, it should be required to state as much in its policy, so consumers know about it, rather than simply deem the FDA-approved treatment "investigational."

If a health-care plan decides Carticel is not cost-effective, the plan can opt not to cover it for that reason, Raines noted, but the current policy bypasses such analysis. "There is no need to consider a therapy if it is experimental," she said.

Carticel isn't the only newly approved therapy that has been labeled investigational, Raines told BioWorld Today. The tag has been applied to Princeton, N.J.-based Cytogen Corp.'s ProstaScint, an approved cancer diagnostic, and to Synvisc, the osteoarthritis treatment marketed by Wyeth-Ayerst Laboratories for Ridgefield, N.J.-based Biomatrix Inc. Wyeth-Ayerst is a subsidiary of American Home Products Corp., of Madison, N.J.

"We'd like to nip this problem in the bud before it becomes endemic," Raines said. "Our industry is at particular risk, because the therapies we develop are often pricey. We've all proceeded under the assumption that if we build a better mousetrap, the world will beat a path to our door. That may not be the case."

However, with the number of working days for Congress waning, and a presidential scandal occupying precious legislative time, Raines noted that a managed health-care bill is unlikely to pass this year.

"We want to be ready in January when the next Congress arrives," Raines said. *