Are Cancer Drugs Worth The Money?

Matthew Herper and Robert Langreth 05.30.09, 4:15 PM ET

ORLANDO - At the annual meeting of the American Society for Clinical Oncology, giant banners with pictures of heroic cancer patients proclaim doctors are "Personalizing Cancer Care."

But many companies seem to be maximizing cancer profit instead. Big drug companies are making big money off smaller and smaller improvements in cancer care. Newfangled cancer drugs can cost $50,000 a year, and that doesn’t mean they will add a year to the patient’s life--you might spend $50,000 for a year and extend the patient's life by only weeks.

The numbers would look better if drug companies did a better job of targeting drugs at the patients most likely to benefit. But that targeting has occurred in only a few scattered examples.

The skyrocketing costs for limited benefit are leading some experts to worry about whether the medical system has the right incentives.

"We are wasting a lot of resources treating people with treatments they don't need," says Otis Brawley, chief medical officer at the American Cancer Society. Novartis, Wyeth and Pfizer all sell very expensive cancer pills. "We would like to believe that cost should be no object, but that is not reality," says Leonard Saltz, a colon-cancer expert at Memorial Sloan-Kettering Cancer Center.

The poster child for high-cost cancer drugs is Roche's Avastin. The drug has $4.8 billion in sales and is approved to treat breast, colon, lung and brain cancer. It was first approved in 2004 after research showed it could extend survival in advanced colon-cancer patients by five months.

The hope was that further studies of Avastin in other types of cancer or in earlier stages of the disease would show even greater survival benefits. But they haven’t. In several breast-cancer trials--including a new one being presented at the meeting this weekend--Avastin slowed the progression of disease but did not extend patient survival at all. But doctors still use the drug in treating breast cancer because they figure it helps symptoms, even if patients don’t live longer. Avastin costs up to $55,000 a year.

In another major study presented at the meeting, Avastin failed to prevent colon cancer from recurring after surgery. In the first year of treatment, more patients who got Avastin remained free of detectable cancer. But after a year, the drug was stopped, and by the end of the study, the apparent initial benefit had faded completely.

Historically, the goal of therapy in early-stage cancer has been to increase the cure rate. But results hint that Avastin may not be curing anyone--it may suppress microscopic cancer deposits without killing them by preventing the growth of their blood vessels. The doctor who conducted the trial, Norman Wolmark of Allegheny General Hospital, is talking about testing Avastin for twice as long in order to get an effect, but that may not be enough. To make a difference in early-stage disease, "the patient may need very, very long-term or even permanent treatment with Avastin," says Saltz. "It gets phenomenally expensive."

Roche says the hints of efficacy support Avastin's future prospects. "It is a validation of the fact that Avastin has a role in early-stage disease," says William Burns, head of Roche's pharmaceuticals division. He forecasts Avastin could still reach sales of $8 billion by 2012 or 2013, even without use in earlier stages of cancer. "With Herceptin, we have learned that it is a drug to start with and stay with" at multiple stages of disease, says Burns, referring to the company's targeted breast cancer drug. "We are start[ing] to piece together exactly the same journey for Avastin."

Burns says the company will consider altering its ongoing trials of Avastin in early-stage breast and lung cancer to include longer-term Avastin use.

Roche is also combining other expensive drugs with Avastin. One study at the meeting showed that adding Tarceva, from OSI Pharmaceuticals and Roche, delayed by one month the median time it takes lung tumors to grow. While it is not yet possible to tell whether there is an effect on survival, the results were statistically significant and financially momentous. U.S. sales of Tarceva were $457 million last year. "We estimate we have the potential for at least $500 million in new [U.S.] business from this study," says OSI Chief Executive Colin Goddard.

One problem is that doctors have no idea which patients are likely to benefit from Avastin, so they give it to everyone in hopes that a few will benefit. Another strategy might be to amp up the search for blood-test results that could predict whether a patient will respond to Avastin, says Angela DeMichele, an oncologist at the University of Pennsylvania whose research focuses on breast cancer. There are clues as to how patients most likely to benefit from Avastin might be identified, she says, but these avenues haven't been pursued yet. "We're still treating people in an unselected way," DeMichele says.

"Avastin is probably not a targeted therapy, as far as I am concerned," says MD Anderson Cancer Center breast cancer expert Franciso Esteva.

Drugs like Tarceva and AstraZeneca's Iressa, which is no longer used in the U.S., work best in patients whose tumors have a mutation in the gene for the epidermal growth factor receptor (EGFR), which is involved in cell growth. In another lung cancer study, Tarceva only slightly delayed the median progression in the overall group of patients, but the small subset of patients with EGFR mutations in their tumors had a tenfold lower risk of the tumor progressing when they took Tarceva.

Since Iressa, AstraZeneca has made a point of pairing drugs with diagnostic work. But it didn’t with Zactima. This experimental drug delayed the progression of lung cancer by three weeks to 17 weeks. Although promising--and viewed as one of AstraZeneca’s most important new products--it has not been paired with a test. Doctors hope for a greater benefit if it is used in earlier stages of the disease.

One of the most impressive results was not from a fancy new therapy at all. Alimta, a more traditional chemotherapy pill sold by Eli Lilly, didn't just slow tumor progression but also increased survival for patients with non-small-cell lung cancer by a median 2.8 months, extending survival to 13.4 months.