Marlene McCarthy’s breast cancer has grown relentlessly over the past seven years, spreading painfully through her bones and making it impossible to walk without a cane.
Although the 73-year-old knows there’s no cure for her disease, she wants researchers to do better. It’s been years, she said, since she has found a drug that has actually helped. McCarthy said she’s frustrated that the Food and Drug Administration is approving cancer drugs without proof that they cure patients or help them live longer.
Pushed by patient advocates who want earlier access to medications, the Food and Drug Administration has approved a flurry of oncology drugs in recent years, giving some people with cancer a renewed sense of hope and an array of expensive new options. A few of these drugs have been clear home runs, allowing patients with limited life expectancies to live for years.
Many more drugs, however, have offered patients only marginal benefits, with no evidence that they improve survival or quality of life, said Dr. Vinay Prasad, assistant professor of medicine at the Oregon Health and Sciences University, who has written extensively about the FDA’s approval process for cancer drugs.
Overall cancer survival has barely changed over the past decade. The 72 cancer therapies approved from 2002 to 2014 gave patients only 2.1 more months of life than older drugs, according to a study in JAMA Otolaryngology-Head & Neck Surgery.
And those are the successes.
Two-thirds of cancer drugs approved in the past two years have no evidence showing that they extend survival at all, Prasad said.
The result: For every cancer patient who wins the lottery, there are many others who get little to no benefit from the latest drugs.
In a November study published in JAMA Internal Medicine, researcher Diana Zuckerman looked at 18 approved cancer drugs that didn’t help patients live longer. Only one had clear data showing that it improved patients’ lives, such as by relieving pain or fatigue.
Two drugs harmed quality of life. For example, thyroid cancer patients taking the most expensive drug, cabozantinib, scored worse on a scale measuring five symptoms: diarrhea, fatigue, sleep disturbance, distress, and difficult remembering, Zuckerman said.
“We cannot have a system where drugs that may not even work are being sold for these amazingly crazy amounts of money,” said Zuckerman, president of the National Center for Health Research, a nonprofit in Washington that aims to explain research to consumers.
Recognizing the slow pace of progress, the American Society of Clinical Oncology has set goals for new cancer drugs of extending life or controlling tumors for at least 2.5 months. The bar was set relatively low because “it’s not very often that we come across a transformative treatment,” said Dr. Sham Mailankody, an assistant attending physician and myeloma specialist at Memorial Sloan Kettering.
Yet in a study published in September in JAMA Oncology, Mailankody found that only one in five cancer drugs approved from 2014 to 2016 met those standards.
The FDA wants to give patients the chance to benefit as soon as possible, rather than waiting for definitive proof of improved survival, Pazdur said. In some cases, the FDA requires pharmaceutical companies to perform long-term studies after drugs are approved, to measure whether drugs live up to their early promise.
But many of these studies never provide an answer, Zuckerman said. Once a drug is approved and is available to anyone, patients have no incentive to participate in a clinical trial. So studies can end with no clear conclusion.
Unless the FDA requires companies to provide survival data before approving a drug, “we may never have answers,” Zuckerman said. “We will have all of these expensive drugs on the market and we will never have the information we need about how well they work or even how safe they are.”
President Donald Trump has vowed to cut regulations at the FDA and recently told pharmaceutical industry leaders that he wants to further speed up the drug approval process.
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