The FDA is challenging drug sponsors to come up with their own ideas for identifying and gathering the kind of patient experience data that can bring ordinary people closer to the clinical trial process without sacrificing safety or efficacy.
The agency, in a draft guidance released in late December, is encouraging sponsors to propose new draft guidances that focus on points in a treatment or device’s lifecycle that “could be particularly informed by” patient’s experiences.
Sponsors interested in doing so should think about whether they already have expertise in such areas as patient registries, natural history studies and work coordination between patient advocacy groups and trials officials or regulators, the 12-page guidance states.
According to the FDA, a draft guidance might be helpful if:
There already is clinical guidance in a disease area, but it doesn’t address the needs of specific subpopulations;
There’s a methodological guidance — such as adaptive trial designs — but sponsors still need specific ideas for how such methods can be applied to a specific disease or subpopulation; and
If there seems to be a need for a disease-specific guidance the FDA hasn’t addressed.
Developing such draft guidance is an important step, says Mark Summers, president of patient engagement at WCG Clinical.
“Nobody in the clinical research world is looking for extra things to do,” he says. “But I think the idea behind [the draft guidance] is tremendous.”
Sponsors should probably focus their efforts on coming up with common measures of patient experience, Summers says.
“You want to be sure that the information is standardized as much as possible,” he says. A seven on one person’s pain scale could be another person’s four, and that kind of variability must be eliminated.
Equally excited about the new draft guidance is Dana Dornsife, founder and chairperson of the Lazarex Foundation, a California-based nonprofit group that helps patients absorb the costs of participating in clinical trials.
“It really reflects a new attitude at the FDA,” she tells CenterWatch. “I think this is a great development for patients. I think that what they’re doing is really trying to create organization around a very chaotic phenomenon right now, which is all these big data projects. Everybody’s collecting data right now, but they’re asking ‘What do we do with it?’”
It’s early in the process, but Dornsife says she hopes the ongoing understanding of how real patients react to treatments will get sponsors to rethink some of their enrollment criteria for trials.
“We don’t want to walk away from a promising treatment. But that clinical trial nirvana doesn’t reflect our larger society,” she says. “People are being left out of the equation on the statistical and scientific impact on their communities.”
Once new draft guidances have been proposed, the FDA will open a new docket for public comment, but the agency won’t commit to issuing guidance of its own.
