The God Doctor does not have some magic formula that will allow "us", meaning him, to do these trials on the cheap or in a hurry. Bottom line, the trials, assuming they ever happen, will take many years and cost tens of millions of dollars. Without a big pharma partner to pay the freight, it is hard to see how Anavex does this for its own account without massive dilution.
Are Big Clinical Trials Relevant? Researchers Disagree Amid health care tailored to an individual’s DNA, do massive clinical trials that take years and involve thousands of patients still matter?
By Lucette Lagnado May 29, 2018 9:15 a.m. ET
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.
“You can’t wait years to get these medications approved. What we are dealing with are women with cancers and their lifespans are limited. They need medications and they need them now, and they are not looking to wait for five years,” says Dr. Matulonis, chief of gynecologic oncology.
That is why flexibility in a trial’s size is crucial, she contends. “As we become more genetically astute, and understand a type of cancer better, I think those large randomized trials will be hard to do. There won’t be that many patients,” that fit into one big group, she added.
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.
She has been taking capsules twice a day and going for check-ups every 28 days since then. Despite side-effects, she has been able to work and carry on. “I have seen my children [grow] and I have seen grandchildren I didn’t have then,” she says. Ms. Sheehan was on a randomized trial where both groups of patients received treatment with Olaparib. One group got the drug only, the other received Olaparib in combination with another drug, her doctor said, adding, “there was no placebo.”
In remission, Ms. Sheehan has become a fan of small trials that offer women such as herself options. She also is a realist. If Olaparib fails, she hopes other trials now going on may yield treatments for her.
The big randomized clinical trial is an “endangered species,” says the senior author of a recent study that charted a decline in clinical trials funded by the National Institutes of Health. “Everyone wants an answer in a hurry. Everyone wants to study a few patients,” says Curtis Meinert, a professor at the Johns Hopkins Bloomberg School of Public Health.
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