It took an instant for 58-year-old Gailanne Reeh to go from the picture of health to death's door.
By chance, Reeh's doctor noticed a lump under her arm during a routine exam. It turned out to be advanced breast cancer.
The surgeon, Reeh recalled, “looked at me and said: 'This is not a conversation I like to have. But I can't do anything for you. You can't be cured. You can't be treated. All we can do is manage your cancer.' ”
And so, like many others in that situation, Reeh, the vivacious owner of a staffing agency in Boston, was given bevacizumab, also known as Avastin, a drug that signifies both the hopes and dilemmas of modern medicine.
Looked at one way, Avastin, made by Genentech at an Oceanside facility, is a wonder drug. Approved for patients with advanced lung, colon or breast cancer, it cuts off tumors' blood supply, an idea that has tantalized science for decades. Despite its price, which can reach $100,000 a year, Avastin has become one of the most popular cancer drugs in the world, with sales last year of about $3.5 billion, $2.3 billion of that in the United States.
But there is another side to Avastin. Studies show the drug prolongs life by a few months, if that. Some newer studies suggest the drug might be less effective against cancer than the Food and Drug Administration had understood when the agency approved its uses.
While many patients and their doctors say the drug can improve the quality of life, such as a sense of well-being and an ability to carry out daily tasks without exhaustion or pain, such effects can be hard to document.
Avastin also has serious, if infrequent, side effects, some of which can be lethal.
“I still use Avastin routinely, but it's sobering,” Dr. Leonard Saltz, a colon cancer specialist at Memorial Sloan-Kettering Cancer Center in New York, said of the new data. “It's not a slam-dunk and, in fact, the incremental benefit may be more modest than we want to admit.”
If Avastin were inexpensive or if it cured cancer or even held it at bay, as the drug Gleevec does for blood cancer, few might care. But like a half-dozen or so new biotechnology drugs with a similar combination – alluring promise, high price and only arguable benefits – Avastin raises troubling questions:
What does it mean to say an expensive drug works? Is slowing the growth of tumors enough if life is not significantly prolonged or improved? How much evidence must there be before billions of dollars are spent on a drug? Who decides? When, if ever, should cost come into the equation?
For a patient like Reeh, fighting for her life, the cost is not the main concern. If her insurer didn't pay, she said, she would go into debt, find a way to raise the money.
Some in the pharmaceutical industry worry such prices will raise concerns about whether the drugs are worth it, leading to a backlash such as price controls or restrictions on use.
Roy Vagelos, a former chief executive of Merck considered an elder statesman of the industry, said in a recent speech that he was troubled by a drug, which he wouldn't name but which was a clear reference to Avastin, that costs $50,000 a year and adds four months of life.
Some patient advocates also are troubled by very expensive treatments, such as Avastin, coming into routine use on what they see as little more than a hope and an expensive prayer.
“It's absolutely critical that we start having a public discussion,” said Barbara Brenner, executive director of Breast Cancer Action, an advocacy group. “I think of Avastin as a model that is showing us where the problem is.”