New cancer drugs have been welcomed as breakthroughs. They often come with a hefty price tag. Many cost over $100,000 a year. That might be justified if there were solid evidence that they save lives.
How Well Do the New Cancer Drugs Work?
Cures for cancer have been few and far between. A new analysis in the BMJ evaluated 48 cancer drugs that the European equivalent of the FDA, the European Medicines Agency, approved before 2009 and 2013 (Davis et al, BMJ, Oct. 4, 2017). The EMA had approved the drugs for 68 different uses, eight on the basis of a single study each. Several studies were not double-blind, considered a standard for drug research.
Although it seemed that the medications were extending survival, later research showed that was true only for half of them. In addition, that benefit was judged clinically meaningful only in about half of those cases.
The authors concluded that many of these cancer drugs were approved “without evidence of benefit on survival or quality of life.” Even more than three years after approval there remains a paucity of solid scientific evidence of substantial benefit.
What About the Price?
We have written previously about the extraordinary cost of these medications. Some of these drugs cost thousands of dollars a month and have been hyped as important new advances.
Since cancer patients are desperate for breakthroughs, they are sometimes willing to pay out of pocket for these expensive new medications in the event that insurance companies are slow to approve the extraordinary costs. Are patients and their families getting what they pay for?
How the FDA Approves New Cancer Drugs:
Many of the new cancer drugs have been approved based on what are referred to as “surrogate end points.” That means the pharmaceutical company demonstrates that a drug can shrink a tumor, improve a blood marker or enhance something called “progression-free survival,” abbreviated PFS. The FDA defines this surrogate marker as:
“PFS is defined as the time from randomization until objective tumor progression or death.”
That usually means the time from when the patient starts treatment in the clinical trial until there is obvious growth of the tumor or the patient dies. In a great many clinical trials a drug company merely has to show that it extends the time till there is “tumor progression.”
What Did the Study Find?
In JAMA Internal Medicine (October 19, 2015) researchers reported on their analysis of new cancer drugs. The investigators reviewed FDA approval data from January 1, 2008 until December 31, 2012. Of the 54 new cancer drugs that were approved during that time, 36 (67%) were based on surrogate end points.
The results of their analysis are shocking:
“Our results show that most cancer drug approvals have not been shown to, or do not, improve clinically relevant end points.
“Since 2008, the FDA has approved a higher percentage of drugs than previously, and cancer drugs are approved on the basis of surrogates that have poor correlations with overall survival. Our results suggest that the FDA may be approving many costly, toxic drugs that do not improve overall survival. Enforcement of postmarketing studies is therefore of critical importance.”
The Bottom Line on New Cancer Drugs:
There are some new cancer drugs that really do represent an important advance in treatment. Sadly, many of the very pricey medications approved over the last several years do not lead to benefits that patients and their families really care about: improved survival. And such drugs are not only costly, they come with substantial toxicity.
Perhaps the FDA should reconsider its policy of approving new cancer drugs quickly based on surrogate end points and instead approve such medications only when they demonstrate substantial improvement in both quality and quantity of life.
We welcome your thoughts and your experiences below in the comment section.
