New studies show that a drug approved this summer to treat age-related macular degeneration not only prevents vision loss but improves the sight of patients who suffer from this eye discorder, which occurs when the macula, or central portion of the retina, breaks down and hinders the ability to decipher fine details.
"This is a remarkable step forward," said Dr. Frederick Ferris, clinical director of the National Eye Institute at the National Institutes of Health.
The studies were published today in the New England Journal of Medicine.
Age-related macular degeneration, or AMD, is the No. 1 cause of vision loss and legal blindness in adults. One in three people will be touched by it by the time they reach age 75, and experts expect a virtual AMD epidemic as the population ages.
The disease gradually destroys a person's sharp central vision so that daily activities, such as driving and reading, become impossible. Recognizing faces also becomes difficult. But treatment is available for some.
The FDA approved a drug to treat the more advanced form of AMD, known as wet AMD, last June. Between 10 and 15 percent of AMD patients have this form.
Researchers gave monthly injections of the drug ranibizumab, or Lucentis, to nearly 500 patients with AMD and gave sham injections to about 200 more patients.
After two years, nearly 95 percent of the patients who received Lucentis saw more clearly than they did when the study began. The researchers measured visual acuity by asking patients to read letters off a doctor's eye chart.
The Lucentis patients could read more letters off the chart after they took the drug. Doctors said the finding is something to celebrate.
"Patients have done incredible with this drug. It is like nothing we have ever seen before," said Dr. Peter Kaiser at the Cole Eye Institute at Ohio's Cleveland Clinic.
Kaiser is the author of the studies published today and has been an investigator in all the Lucentis trials.
But the patients who did not take Lucentis saw the letters on the eye chart less clearly after two years. Their vision actually got worse over the course of the study.
The study authors concluded that Lucentis not only prevents vision loss but makes vision better.
Before Lucentis became available, some doctors treated wet AMD with the cancer drug bevacizumab, sold as Avastin.
Some still prescribe Avastin for AMD, even though that use is considered "off-label" because the FDA has not approved Avastin to treat AMD.
Without the FDA approval for AMD, most insurers won't pay for it to treat that disorder.
"FDA approval is required for most insurance companies to cover the cost," said Dr. Richard Bensinger, spokesman for the American Academy of Ophthalmology.
"Therefore Avastin is not covered by most plans."
Doctors said Lucentis really works, and since it has an FDA stamp of approval, most health plans will cover it. But Lucentis is significantly more expensive than Avastin.
"Companies will pay $1,500-$2,000 for the Lucentis rather than the $40-$80 for Avastin," said Bensinger.
"This means that if the patient is insured, the insurance coverage will be for the larger bill. If the patient is uninsured, Avastin is likely to be used."
The extra cost of Lucentis isn't something to sneeze at.
"Cost is an issue for our health care system as a whole and also, often, for the patient," said Dr. Naresh Mandava, at the Rocky Mountain Lions Eye Institute in Colorado.
"Many patients are unable to pay the co-pay for such an expensive drug," Mandava said.
Some doctors said that the extra cost of Lucentis is enough to make them shy away from prescribing it.
"The difference in cost between the drugs exceeds $1,800 and is a factor for patients who do not have complete drug coverage," said Dr. Jayakrishna Ambati, vice chairman of the department of ophthalmology and visual services at the University of Kentucky.
For patients who do have insurance coverage, Lucentis can still be too costly.
Said Ambati, "Even their 20 percent co-pay is a significant burden for a drug that requires 12 injections a year at nearly $2,000 per visit for the drug alone!"
So Ambati goes with the less-expensive but FDA-unapproved option.
"I offer Avastin to such patients while informing them that Lucentis is the FDA-approved drug: Most patients choose Avastin in this circumstance," Ambati said
But cost isn't the only thing that matters. An expensive drug might be worth it if it works better than the alternatives.
Experts couldn't say for sure if Lucentis works better than Avastin, but they suggested that it might.
"We are currently in the position of having a proven, FDA-approved treatment and a less expensive off-label treatment that has similar biologic activity and seems to have a similar treatment effect but has not been carefully studied in the eye," Ferris said
So scientists need to see more research that compares the two drugs side by side. But some doctors already believe that Lucentis might be a better choice.
And studies suggest it could also be safer than Avastin.
But only a controlled experiment that tests Lucentis against Avastin would tell doctors whether one drug is better than another.
Since the drug company Genentech makes both of the drugs, it's not likely to sponsor such an experiment.
But the government will. The U.S. National Eye Institute plans to compare the two drugs under controlled conditions.
"The NEI has received a grant application to study Avastin versus Lucentis, and that has been approved after peer review. Current evaluation of the feasibility of this trial is under way," Ferris said.
In the meantime, AMD patients have something that can save their clouding eyesight.