Sylvia Syvenky went for a routine dental appointment in early October, expecting to have two caps on her teeth replaced, but something went terribly wrong. She was rushed by ambulance to University Hospital near her home in Edmonton, Alberta, where doctors placed a mask on her face and forced air into her lungs,The New York Times writes. They told her she had heart failure.
And after her condition improved, they asked her to sign up for a study of a new drug to help with breathing. Syvenky is like many with heart failure who arrive at hospitals, unable to breathe. Yet she is the last person who would normally be asked to join a research study, the Times notes, adding that, at age 70, she was much older than typical study participants and her symptoms were too complex.
But now there is a growing movement to gather a new kind of evidence to fill in some of the biggest gaps in medical science: What treatment is best for typical patients with complex symptoms? Many are elderly with several chronic conditions and taking several unrelated meds. And, the Times asks, what are the long-term effects, such as death rates, side effects and disease progression?
A group of advocates, including medical researchers, medical societies and insurers, is lobbying Congress to pay for an Institute for Comparative Effectiveness Research that would assess treatments and identify gaps in evidence, the Times reports. When there are gaps, the institute would initiate what are being called “real world,” or “pragmatic,” clinical research trials to gather the evidence.
Some leading researchers who used to defend the status quo say they have switched. “There has been a 90-degree turn” in thinking, Eugene Braunwald, an eminent cardiologist at Harvard Medical School, tells the Times. “I personally have swung around.”
Although thousands of medical studies are completed every year, most have relatively limited goals. They often carefully select patients who have few medical problems other than the one under study, making it easier to get one clear result, the Times notes, adding that studies may not look at effects over the long term, assuming that if a treatment helps initially, patients will be better off.
But while such studies can help a drug acquire approval or answer a restricted research question, they may not tell how a new drug will work once it is tried in real patients with complex problems. Such limited studies, while they can have value, may no longer be enough, particularly when care has become so expensive and real evidence more crucial.
“They are at the heart of why we have trouble making decisions,” Scott Ramsey, a professor of medicine at the University of Washington, tells the Times.
The FDA does not have a specific rule about what it takes to show that a drug is effective, according to Bob Temple, director for medical policy at the FDA’s Center for Drug Evaluation and Research. A lot depends on what is known about a drug’s short-term effects and how well it can predict long-term outcomes. But there are practical concerns with large trials because drugmakers have to look at a wide range of possible effects when they test a drug. “If you do a large outcome study in 10,000 people in the same way you do short-term studies, you’ll never finish,” Temple tells the Times.
“There’s no white hat, black hat here,” Kevin Weiss, president and chief executive of the American Board of Medical Examiners, tells the paper. “Pharmaceutical companies are trying to do what they are supposed to do. The FDA is trying to do what it is supposed to do. But they are not fully connected to what the public needs.”