NY Times: New drug for macular degeneration -- but expensive

June 29, 2006

Questions Over New Eyesight Drug That May Be as Good as Older, Cheaper One

By ANDREW POLLACK

New York Times

A drug that can restore eyesight to some elderly people, even allowing them to read or drive again, is expected to win federal approval this week.

But for patients, doctors, Medicare and other insurers, the drug's arrival will pose a conundrum. That is because the medicine, Lucentis, is expected to be 10 to 100 times as expensive as a similar drug that many ophthalmologists say is every bit as good.

Lucentis, made by Genentech, is the first drug demonstrated in clinical trials to improve vision in people with so-called wet macular degeneration, a form of bleeding behind the retina that is the leading cause of blindness in people over 65. The condition afflicts an estimated 1.2 million people in this country, with an 200,000 new cases each year.

While the drug is not a cure and does not help everyone, specialists say it represents a breakthrough against a disease that once almost inexorably led to an incapacitating loss of eyesight.

"I have people who are reading the paper every morning who would have been totally legally blind two years ago," said Dr. Julia A. Haller, a professor of ophthalmology at Johns Hopkins University, who has had patients testing Lucentis.

But the big question is whether insurers and patients will consider Lucentis worth prices that may be $10,000 a year or higher, compared with around $1,000 or less for the drug already on the market that many ophthalmologists say is just as good.

That drug, Avastin, is approved only to treat cancer. But used for the eye condition, which is legal and known as an off-label use, it works the same way as Lucentis. In fact, Lucentis is a derivative of Avastin tailored to be used in the eye. Like Lucentis, Avastin was developed by Genentech.

With Lucentis not yet available, off-label use of Avastin has become the treatment of choice for macular degeneration. Since last fall, thousands of eye patients have been treated with Avastin, with good results and minimal side effects, experts say.

But Genentech has no interest in getting Avastin approved for macular degeneration, because that would undermine the sales of Lucentis, which some analysts predict will have annual sales of several hundred million dollars.

Avastin is not a cheap drug. It costs about $50,000 a year when used intravenously to treat colon cancer.

But for an injection into the eye to treat macular degeneration, only about $20 to $100 worth of the drug is needed.

Genentech has not announced the price for Lucentis, which the F.D.A. is widely expected to approve by tomorrow. But retina specialists expect an injection of Lucentis to cost from $1,500 to well over $2,000.

For both drugs, an injection is needed as often as every four weeks.

The number of patients with wet macular degeneration is expected to mushroom as the baby boomers age. About 85 percent of people with wet macular degeneration are covered by Medicare, Genentech says.

Use of Avastin instead of Lucentis could save patients and insurers hundreds of millions of dollars a year. Yet, despite the potential cost savings, several specialists said they expected most doctors to switch to Lucentis after its approval because there are solid data from clinical trials on the safety and efficacy of that drug. Using an approved drug, instead of using another drug off-label, also means less paperwork and less risk of malpractice lawsuits. What is more, apportioning Avastin into small sterile doses for ophthalmic use requires its repackaging by special druggists known as compounding pharmacists.

"The whole experience really opens your eyes to how our whole health care system is operating," said Dr. Philip J. Rosenfeld, an associate professor of ophthalmology at the University of Miami who pioneered the use of Avastin for macular degeneration. "We could be incentivized to use the most effective therapy at the most reasonable cost. But that's not how our system is set up."

Genentech's disinclination to test Avastin as an eye treatment has angered some doctors. "The part that really bothers me most is that Genentech has steadfastly refused to do a head-to-head study between Avastin and Lucentis," said Dr. Raj K. Maturi, a retina specialist in Indianapolis.

But Dr. Susan Desmond-Hellmann, the president of product development for Genentech, said the company's priority was "to innovate and do better things for patients" — not to show one drug was equivalent to another.

Dr. Desmond-Hellmann said Lucentis would save the health care system money by preventing blindness, which can lead to other injuries and to nursing home admissions. She also said the company would support programs to help patients with their insurance copayments for Lucentis and would provide the drug free to patients who could not otherwise pay for it.

Wet-form macular degeneration is characterized by abnormal, leaky blood vessels that form under the retina, the light-sensing part of the eye. The vessels leak fluid into the eye, causing warped vision, and damage the macula, the part of the retina responsible for the straight-ahead vision used for tasks like recognizing faces, reading, driving and watching television. People with the condition usually retain some peripheral vision.

"I could look at my husband but not see his face unless I looked to the right to bring him into view," said Helen Nicolosi, 77, of Marathon, Fla., whose eyesight was affected by the disease.

She said monthly injections of Lucentis in a clinical trial improved vision in her affected eye to 20/50 from 20/150, letting her drive again and do the crossword puzzles she loves. Both Avastin and Lucentis work by stopping the growth of new blood vessels. That retards the leaking in eye vessels, and in cancer it halts the supply of nourishment to tumors. Lucentis is a smaller-protein version of Avastin, designed to penetrate the retina better.

In clinical trials, one-quarter to one-third of patients who received Lucentis gained three or more lines of vision on an eye chart, benefits that have lasted two years so far with continued injections. About 40 percent of the patients ended up with at least 20/40 vision, the usual minimum for a driver's license. Only about 10 percent had a degradation of vision of at least three lines on the eye chart after two years.

By contrast, the two drugs already specifically approved for macular degeneration — Visudyne from QLT and Novartis, and Macugen from OSI Pharmaceuticals and Pfizer — slow the deterioration of eyesight but do not usually improve vision.