Patients and their advocates are getting an ever-larger voice in how medical research is carried out. They participate in the design of experiments and have a greater say in what outcomes they care about most — and it's not always simply living longer.
There are now hundreds of patient groups, focusing on everything from arthritis to xeroderma pigmentosum. And patient participation in medical research is no longer an informal matter. The concept is written into federal laws, including one that funds the Food and Drug Administration. Since 2013, the FDA has held more than 20 meetings in which patients are invited to lay out what actually matters to them.
The FDA historically hasn't considered its work from the patient's point of view. It generally starts with a company pitching a new drug and is supposed to answer the narrower question: Is the product safe and effective? Now that the FDA knows what patients care about, it can suggest that drug companies measure those outcomes in the course of testing new products.
The FDA has already demonstrated that it's paying more attention to what patients want. Earlier this year, after hearing impassioned pleas from parents and their scientific allies, the agency temporarily approved a drug for Duchenne muscular dystrophy, even though the science in hand didn't provide proof positive that the drug was effective (read “FDA Succumbs to Industry-Sponsored "Patient Power" & Accelerates Approval of Sarepta's Duchenne Drug”; http://sco.lt/4mgzvV).
Patients considered that a victory. And the company could start selling its drug for $300,000 per patient per year.
"The bad news about what's happening is that so much of patient input is involving patients who are working with or who are recruited by drug companies," cautions Diana Zuckerman, who runs a consumer group called the National Center for Health Research.
These companies pour millions of dollars into disease advocacy organizations and provide information to patients. So the patient voices are sometimes inadvertently channeling commercial interests, Zuckerman says.
With that system, patients also hear more about potential benefits than risks, she says. Clearly patients should have a voice in how big a risk they are willing to take, she says, but within limits.