Researchers say many new cancer drugs don’t improve life or increase lifespan. Some doctors suggest we need to speed up the cancer drug approval process.
There’s no clear evidence that most new cancer drugs extend or improve life.
According to a research team in London, when drugs do show survival gains over other treatments, those gains are often marginal.
The researchers looked at reports on cancer approvals by the European Medicines Agency (EMA).
Of 68 medications approved by the EMA from 2009 to 2013, 39 came on the market on the basis of a surrogate endpoint, such as shrinking tumors or lowering biomarker levels.
There was no evidence that they significantly extended survival or improved quality of life.
After a median five-year follow-up, 51 percent showed gains in survival or quality of life. The rest remain uncertain.
“When expensive drugs that lack clinically meaningful benefits are approved and paid for within publicly funded healthcare systems, individual patients can be harmed, important societal resources wasted, and the delivery of equitable and affordable care undermined,” wrote the researchers.
Dr. Vinay Prasad, assistant professor at Oregon Health & Science University, wrote an
He referenced his own 2015
Of 36 approvals, only 14 percent were eventually shown to improve survival when compared with existing treatments or placebo after more than four years on the market.
Dr. Santosh Kesari is a neurologist and neuro-oncologist and chair of the Department of Translational Neurosciences and Neurotherapeutics at the John Wayne Cancer Institute at Providence Saint John’s Health Center in California.
Kesari told Healthline that in the United States, the move is to try to get drugs to patients earlier rather than later.
He pointed out that studies focus on averages and medians.
“Most of these drugs have marginal benefits on average for a statistical endpoint. But if you look at specific drugs, the benefits are more in long-term overall survival. That’s one of several metrics we use to qualify a drug as being important to the patient,” said Kesari.
“When you look at the tail of the curve, who’s living at three or five years? This is not always reflected in that one number. The FDA and EMA approve drugs because there’s more data than just that number,” Kesari continued.
Dr. Jack Jacoub is a medical oncologist and medical director of MemorialCare Cancer Institute at Orange Coast Medical Center in California.
He told Healthline he’d also like to see cancer drugs get approved more quickly.
“This happens to be an era of rapid drug approval because drugs have advanced so much in the last three- to five-year window where it is remarkable,” said Jacoub.
“These trials are well designed and scrutinized. It’s not something where you can fudge the numbers. There are benchmarks that drug trials have to prove. The FDA is rigid. There are financial and patient safety issues, so they’re very careful. They have hundreds of applications at a time and don’t approve the majority,” he added.
“There’s a significant need for people with potentially curable cancer and metastatic disease. Some can live one, two, three years, depending on the type of cancer therapy being offered. To wait for clinical trials to be completed, the regulatory phase, and approvals. Unfortunately, some of these patients die waiting,” said Jacoub.
“We want early access for cancer drugs that have potential, because it takes 5, 10, or 20 years to get drugs approved while patients are dying,” he said.
Kesari pointed to the
“It lets people who don’t have other options to have access to a drug once you know it’s safe. The drug company still has to do phase 3 clinical trials to get full approval,” he explained.
“This is also about quality of life. Many people look at survival and miss quality of life. But that is important as well. I think patients generally put up with a lot of side effects and we can manage most side effects. So, they can deal with a quality-of-life issue if there’s a clear survival benefit,” said Kesari.
Do some of these new drugs give cancer patients and their families false hope?
“There are some like that,” said Kesari. “But few, if you look at the data carefully. There are always subsets of patients that really, truly benefit. They may live six months, a year, or many years.”
Jacoub said when treating a large group of patients with a particular drug, it’s with the understanding that only a certain percentage will benefit.
“But we don’t know who. We need to do a better job of figuring that out,” he said.
“As the field moves more from broad stroke to molecularly, or target driven, types of therapy, you’re likely to find more benefit. Having therapy specifically for certain targets enriches enrollment with people who have the specific target for your drug. You can get to an answer pretty quickly,” continued Jacoub.
Jacoub also suggests that trials should involve people with earlier stages of disease.
He explained that by testing drugs before surgically removing a tumor, there’s a better window to see what the drug can do, as opposed to having surgery and then using a drug.
“When switching the sequence of therapy, you can test how the tumor is changing in a person’s body. It can be a rapid way of understanding if a drug works,” added Jacoub.
He’d also like to see trials end when there’s clearly no benefit, rather than continuing for years to the trial’s completion date.
New cancer drugs are expensive, and Jacoub acknowledged there may be a pushback from payers.
“But that’s a different argument,” he said. “The more important question is, ‘how do we find those who will benefit?’”
Time is of the essence, according to Jacoub.
“Sometimes we ask drug companies to provide medicines on a compassionate basis — or you try to get them on a trial — those are the two options,” he said. “Trials can last years and some patients simply don’t have that.”