First comes the shock of a cancer diagnosis. Then comes the flurry of questions: Is there a cure? Is there a treatment? What drug do I take? Will it work?

Sometimes the answers are easy to find. People with chronic myeloid leukemia, for example, can take imatinib (Gleevec) and have an 80% chance of surviving for 10 years. 

But other cancer patients aren’t so lucky. They may be given an option of taking a chemotherapy medication that won’t help them live longer—and that will make their lives more miserable in the bargain.

This is especially true of the spate of cancer therapies that have come on the market in the last 10 years, according to recent studies. Despite these new drugs, cancer deaths for men and women decreased by just approximately 1% from 2004 to 2013, according to the National Cancer Institute.

We asked Diana Zuckerman, PhD, president of the National Center for Health Research, a nonprofit organization in Washington, DC, to explain why many new cancer medications have so few benefits…and what you or your loved ones can do to get the treatment you need and deserve—at a reasonable cost.


One big reason these drugs aren’t very effective is a sped-up approval schedule that puts many new cancer drugs in the hands of patients before they’ve been thoroughly vetted. 

Consider this: From 2002 to 2014, the Food and Drug Administration (FDA) approved 71 drugs for a variety of types of cancer—but fewer than half of these drugs have been shown to extend patients’ lives by at least two-and-a-half months, the minimum standard. Many of them came with hefty price tags of more than $10,000 a month. For some of these chemo drugs, the story is even worse—they didn’t improve survival at all. As if that weren’t bad enough, some of these new drugs have side effects that are as dangerous as the cancer they are supposed to treat.

How did this happen? In the quest to get potentially lifesaving medications to people who desperately need them, the FDA approves drugs before the information about their real benefits has been determined. Two-thirds of these drugs were approved because they showed some evidence that they might work, such as shrinking the tumor in the first few months, but without any evidence that they helped patients live longer.

While stopping tumor growth or reversing it is a promising sign, physicians know that it can be temporary and sometimes followed by rapid tumor growth. In some cases, the treatment is so toxic that it kills healthy cells in addition to cancer cells and the patient may die sooner or with terrible side effects. Nevertheless, pharmaceutical companies prefer to measure the effect on tumor growth because the results can be measured months or even years sooner than overall survival.

But that’s a mistake. If you have cancer, you’re probably more interested in living longer than in simply avoiding death from cancer. For example, if the drug you take causes liver toxicity or a stroke that can kill you before the cancer would, it’s not doing you any good.


When drugs are approved only on the basis of the way cancerous tumors respond, the FDA usually requires follow-up studies to prove that the drugs are truly beneficial, such as helping patients live longer or improving their quality of life for the weeks, months or years that they have left.

That process makes sense—as long as the FDA enforces it. Example: In 2008, bevacizumab (Avastin) was approved to treat metastatic breast cancer because studies showed that it improved progression-free survival (how much time the patient lives with breast cancer without the disease getting worse). Later research showed that the drug offered no survival benefit and it came with an increased risk for heart attacks and strokes, among other harmful and debilitating side effects. In 2011, the FDA revoked the drug’s approval for breast cancer. That follow-up decision has helped breast cancer patients avoid an expensive, ineffective drug, although thousands of breast cancer patients took the drug between 2008 and 2011.

Unfortunately, that’s a rare example. Between 2008 and 2012, 36 out of 54 cancer drugs were approved on the basis of tumor shrinkage and similar preliminary measures. Later studies showed that only five of those 36 drugs improved overall survival. Eighteen of the drugs proved unable to improve survival, and there’s no information about the final 13 because they either haven’t been tested or the results haven’t been reported.

Yet all these drugs are still on the market. One reason is that postapproval studies that could result in removing a drug from the marketplace are very difficult to conduct. Example: It is difficult to conclusively prove whether these cancer drugs are effective because patients are less willing to participate in further clinical trials on a drug that’s already been approved.

The problem may get worse. The FDA is currently evaluating ways to overhaul its processes and speed up approval of all types of drugs, including cancer treatments, in its effort to get life-saving drugs on the market faster.


A drug that increases a cancer patient’s well-being and comfort is also considered worthy of FDA approval even in cases where living longer is not likely. But a recent study published in JAMA Internal Medicine looked closely at 18 drugs that had no survival benefit to see what effect they had on patients’ quality of life. The majority of the drugs either contributed to a worse quality of life—for example, patients suffered from diarrhea, fatigue, sleep disturbances or memory loss—or the evidence was mixed (which means the research was inconclusive, with some studies suggesting that treatment was helpful and other studies suggesting it was harmful).

Some of the worst offenders included…

Bendamustine (Treanda)

The drug is approved for non-Hodgkin lymphoma and chronic lymphocytic leukemia.
Cost: $53,000 to $85,000 a year, depending on diagnosis.
Results: Research shows that patients taking this drug for either condition do not live longer, even though it is FDA approved for these patients. The drug does not improve quality of life for leukemia and no studies were done to show whether it improves quality of life for patients with lymphoma.

Cabozantinib (Cometriq)

The drug is approved for thyroid cancer.
Cost: Nearly $170,000 a year.
Results: Even though it is FDA approved for these patients, research shows those who take this drug do no live longer. It makes patients’ quality of life worse, usually due to diarrhea, fatigue, nausea and other unpleasant side effects.

Everolimus (Afinitor)

The drug is approved for kidney, breast and pancreatic cancer.
Cost: Nearly $145,000 a year.
Results: Research shows that this drug does not help patients with kidney, breast or pancreatic cancer live longer, although it is FDA approved for them. There is no clear evidence that it improves quality of life for these patients, either.

Peginterferon (PEG-Intron)

The drug is approved for melanoma.
Cost: $26,000 a year.
Results: Research shows that this drug does not help patients with melanoma live longer. It makes their quality of life worse. Some of its scarier and more common side effects include anxiety, depression, bloody diarrhea, difficulty breathing, vision loss, stroke and life-threatening infections.


A cancer diagnosis is stressful under any circumstances, but if your cancer doesn’t respond to standard treatments, the choices get tougher. The choice is always the patient’s—for some people, living another two months is worth possible side effects. But there is no excuse for chemotherapy that won’t help extend life and makes you more miserable in the final days.

Unfortunately, you can’t always count on your doctor to help you sort through the options. Many doctors will list all the drugs out there because they want you to have a say in your own care. They also assume that FDA approval means that the advantages outweigh the dangers and may not realize that the studies are inconclusive.

To make an informed decision, though, you need to know the benefits and risks of the medications that are being offered. Here’s how…

Ask your doctor specific questions. Will this drug help me live longer, or will it just cause the tumor to shrink? What are the side effects and how dangerous or unpleasant are they? If you don’t get answers, ask your health-care provider to do some research and give you a summary of the results. Then do some digging on your own.

Use reputable sources for your research. Google searches can send you straight to a misleading advertisement for a drug as well as other misinformation from a variety of sources. Go to instead, where you’ll find the proven risks and benefits of each medication. Look for the sections on risks, side effects, adverse events and contraindications to find out what the problems might be. Check what the drug has been approved to treat. For example, some treatments used for prostate cancer are FDA approved to treat prostate “problems” or “health” but are not proven to treat prostate cancer.

You might also search on Google Scholar or PubMed to find journal articles written by scientists and medical experts who have studied the treatment you are considering. Again, read carefully to see whether there is any evidence that the drug improves “overall survival.”  If the drug improves “progression-free survival,” that doesn’t mean patients live longer.

Get impartial help. The Cancer Prevention and Treatment Fund, the primary project of the National Center for Health Research, offers a no-cost cancer help hotline to help patients sift through their choices. This organization doesn’t accept any funds from companies that make any medical treatments. Send an email to with the type of cancer treatment you’re considering, and someone will reply with relevant and meaningful information that can help you make a smart decision.

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