Only a few of the oncology drugs FDA has approved in recent years have evidence showing the drugs significantly extend patients' survival, Liz Szabo reports for Kaiser Health News.
According to KHN, FDA in recent years has approved "a flurry" of oncology drugs in part because of pressure from patient advocates to get access to new medications. However, research shows cancer patients' life expectancy rates have barely improved over the past decade. For instance, a study published in JAMA Otolaryngology–Head & Neck Surgery in 2014 found that the 72 cancer therapies FDA approved between 2002 and 2014 added just 2.1 months to patients' life expectancies.
Vinay Prasad, assistant professor of medicine at the Oregon Health and Sciences University, said many oncology drugs FDA has approved in recent years have minimal patient benefits, and two-thirds of cancer drugs approved in the past two years have no evidence showing they extend survival.
Further, a research letter published this month in JAMA Internal Medicine found that some cancer drugs are approved without evidence that they improve patients' lives. The researchers reviewed 18 cancer drugs that lacked survival-related evidence and found just one had evidence showing it improved patients' lives, while two had evidence showing they harmed patients' quality of life.
Stakeholders raise concerns about rush to approve new drugs
Stakeholders say the findings raise concerns about industry efforts to get approval for new cancer drugs.
Fran Visco, president of the National Breast Cancer Coalition, said, "We are very concerned about the push to get more drugs approved, instead of effective drugs approved."
American Cancer Society (ASC) CMO Otis Brawley expressed concern that FDA is lowering its standards for drugs. He said, "We're getting less rigorous scientifically because we want to get these drugs out to people faster."
For example, Brawley said FDA has increasingly approved more drugs based on "progression-free survival," or how long patients live with cancerous tumors under control. According to Brawley, physicians consider tumors to be under control if they do not grow by more than 20 percent. "The problem with approving a drug based on a progression-free survival is that you don't know if the drug is actually doing anything positive for the patient," Brawley said.
Stakeholders raise concerns about cancer drugs' prices
Further, some stakeholders have raised concerns that ineffective drugs carry high prices. According to the Center for Health Policy and Outcomes at New York's Memorial Sloan Kettering Cancer Center, the average cost for a cancer drug approved in 2016 was $171,000 annually. However, research indicates that the drugs' prices do not correlate with their effectiveness.
Hanna Sanoff, an associate professor and section chief of the University of North Carolina's School of Medicine's Gastrointestinal Medical Oncology Program, said the effectiveness achieved in clinical trials might not translate to drugs' real-world effectiveness because clinical trials often draw patients who are younger and healthier. For instance, Sanoff in a study published last year in The Oncologist found a drug extended liver cancer survival by three months in a clinical trial did not increase survival among Medicare beneficiaries outside of the trial.
Richard Schilsky, senior vice president and CMO at the American Society of Clinical Oncology, said, "You begin to question what is the real value of a therapy when the benefit is small, the toxicity may be similar to a previous drug and the cost is much higher."
FDA officials have offered several explanations for why many cancer drugs lack evidence showing improved survival, KHN reports.
Richard Pazdur, director of FDA's Oncology Center of Excellence, said it can take several years to tell whether a new drug extends survival because some cancers grow slowly.
Further, Pazdur said cancer trials' designs can make it challenging to gather survival data, as many trials allow patients in a control group to begin taking the drug being tested if preliminary data indicates the treatment could be beneficial.
Pazdur also said overall survival rates do not show that some drugs, such as certain targeted therapies, extend some patients' lives for years instead of months (Szabo, Kaiser Health News, 2/9).
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