The clinical research enterprise is “at best the sickliest link” in the chain between patients, the healthcare system, and science that can and should bring cures and better therapies, according to Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research (CDER).
“We have a sicker population, we have expensive new interventions and we have wonderful new science that is begging to get translated and evaluated,” said Woodcock in her keynote address at the recent Bridging Clinical Research and Clinical Health Care conference. “All these forces are coming together and converging on the clinical research enterprise. We must bridge clinical research and healthcare, or the entire enterprise is going to fall down.”
To illustrate how little research is getting translated into new directives for healthcare providers, Woodcock explained that only about 15 percent of professional guidelines for healthcare providers in the U.S. are based on evidence from evaluation of trials or actual data; the rest are based on expert opinion. Innovations abound, she said, but we still lack the means to evaluate their impact, and that is a shortcoming of the clinical research space.
“We keep trying things and changing things, but we aren’t ever really sure whether we’ve gone in the right direction or actually taken a step backward,” Woodcock said. “For new pharmaceuticals, despite the billions of dollars spent developing them, we often lack important information when they come on the market about how they’re going to perform in the real world. And we don’t really have a good way of evaluating how they’re performing in the real world.”
The crux of the problem, she said, is that the goals sought by each sector of the healthcare and clinical research arenas have far more to do with other priorities — such as financial gain, tenure, business goals — than they do the kind of collaborative learning that will bring new cures.
“Currently I think almost all the incentives, financially or otherwise, in this whole vast healthcare ecosystem are aligned against collaborative learning,” she said. Woodcock listed the stakeholders that interact with patients and study subjects, explaining where they err.
For academic clinical researchers, she said, the focus is often primarily on tenure, obtaining grant support and productivity in the clinic. And insurers and payers, while they could benefit from increased knowledge from clinical research about what works best, do not focus on learning. “It’s not their business,” said Woodcock. “It’s not how their incentives are aligned.”
Healthcare providers, meantime, work in a fee-for-service environment that is antithetical to research, adding, though, that she expects large healthcare networks to eventually emerge as entry points for bridging research into healthcare more rapidly.
The medical product industry, she said, is now mired in increased research complexity coupled with an overarching desire to create new revenue streams, and these get in the way of maintaining a pure focus on learning and quickly sharing new information gleaned from research.
So NIH then? Well, no. NIH, said Woodcock, has spent the last 40 to 50 years building up the basic biomedical research enterprise, and now that must be sustained.
“The enterprise is like a hundred and fifty baby birds — it has to be fed,” said Woodcock, mentioning laboratories, thousands of post docs, securing of grants and research centers all across the U.S. “I don’t think you can look to NIH and say they are going to fund this enterprise and bring about these transformations. They have other incentives, other pressures on them,” said Woodcock.
Given all these badly misaligned pressures on the industry’s key stakeholders, Woodcock offered her thoughts on how to proceed in building “a nice, sturdy bridge instead of this very flimsy and teetering bridge.”
She added that ignoring community physicians and not sharing study results with them is part of the problem, and must stop. “Their marginalization has lead to the fact that most people are not referred into clinical research in any way because their healthcare practitioners are marginalized from this enterprise and don’t want anything to do with it,” she said.
Said Woodcock, “People are really eager for answers; They don’t just want to wait ten years to hear: do this. But right now, what do we tell them? ‘Here’s what we found in this study but more research is needed.’ How does that help a practitioner or patient? We need to say, ‘This is what we know now, and if you participate, in one year we’ll know more and we can feed that back to you and to your doctors, and we will build on that so the knowledge we gained from you will count. It will make a difference.’”
If those in clinical research can do that, the rapid knowledge feedback loop will start sustaining itself, she said.