Are Big Clinical Trials Relevant? Researchers Disagree

[question everything]

Sweeping clinical trials in which thousands of patients are studied for years at a stretch have long been the Holy Grail of medical research—especially when funded by the federal government.

But in the era of personalized medicine, where care can be tailored to a person’s genetic make-up and doctors analyze a patient’s DNA to figure out treatments, big trials are falling out of favor.

Instead, many researchers are arguing for smaller, nimbler trials that involve fewer patients and take less time. This has pitted those who believe small trials make more sense against those who worry that large trials are being cast aside in favor of flash-in-the-pan approaches that won’t stand the test of time or efficacy.

To Ursula Matulonis, who treats ovarian cancer and other women’s cancers at Dana-Farber Cancer Institute in Boston, the debate over trial size has a special urgency: Many of her patients are desperately sick.

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One of her patients, Janet Sheehan, is grateful for the small clinical trial she has taken part in for the past five years. Ms. Sheehan, a 63-year-old nurse near Boston, was diagnosed with advanced ovarian cancer a dozen years ago. It has come back three times, and at one point she learned that she had a mutation in the BRCA1 gene which indicates a strong predisposition to breast and ovarian cancer. Dana-Farber suggested in 2013 that she go on a randomized 90-person trial for a drug named Olaparib that showed promise among women with a BRCA1 gene mutation.

(snip)

At a recent symposium at the annual meeting of the American Society of Breast Surgeons titled “Why Big Trials Still Matter,” Dr. Norton (a breast-cancer specialist and senior vice president at Memorial Sloan Kettering Cancer Center) cast a critical eye on small trials and the current shift toward them. He warned that most discoveries and advances are modest—for example, a drug that reduces the risk of death by 25%—and statistically, they are more likely to be uncovered in the course of large trials and missed in small trials. “I think that we are throwing away a lot of good drugs and a lot of good diagnostics because our trials are too small,” he said.

More..

https://www.wsj.com/articles/are-big-clinical-trials-relevant-researchers-disagree-1527599699 (paid subscription)

[shraby]

1. One only needs to remember thalidomide for their answer to the question.

It became a disaster beyond imagination.

[question everything]

2. Except.. thalidomide never made it to these shores

but was the push for clinical studied that had to be not only, safe, but also effective, resulting in many delays of life saving drugs. And, of course, we still have many recalls.