Prove It | Kate Willette

Posted by Reeve Staff in Research News on March 02, 2018 # Research

I admit it. I was a big fan of Downton Abbey, the British mini-series that came out in 2010 and played for the next six years. One of the characters in that show was a personal butler named John Bates – a stoic, determined man who struggled with a limp that was obviously painful. Early on in the series, we found out that he’d been forcing himself to wear a cruel-looking metal brace that he’d been promised would repair the leg he nearly lost in the Great War. Instead, it was causing agony.

The writers set that scene in the year 1913, back when there was nothing to stop anyone with a flair for invention from selling “cures” in Great Britain or in the USA. The situation in the USA only started to change in 1937, when a company called Massengill put a strep throat medicine on the market that contained antifreeze. It killed more than a hundred people before the small, ineffective agency called the Food and Drug Administration (FDA) was able to locate and destroy every last bottle that was sitting on the shelves of drugstores.

Massengill paid no price for that catastrophe because there were no laws on the books to regulate what medicine and device companies could and couldn’t sell. The attempt to remedy that problem over the last eighty years is how we got the 2018 version of the FDA, a vast federal agency that employs more than 13,000 people. Its stated mission, in part, is:

“… protecting the public health by assuring the safety, efficacy and security of … drugs, biological products, medical devices …”

Let’s unpack that a little bit. Suppose you run the FDA and you’ve agreed to be responsible for assuring that new medical devices are both safe and effective. Suppose also that someone came to you with what could turn out to be a 2018 version of Mr. Bates’ nasty leg brace and told you that it ought to be on the market. Your job is to make sure that what they’re selling is going to do the customers some good. That’s what“safety, efficacy, and security” means.

Turns out, though, that it’s very tricky to define exactly what we mean by “some good.” In terms of a device marketed to our community – people living with spinal cord injury -- does “some good” mean walking? Does it mean improved breathing? Improved by how much? Does it mean completely restored sexual function? How about just being able to stand unassisted, even if you can’t take steps?

And that brings us back to The Big Idea, because these are the kinds of questions that CDRF has been working with the FDA to answer. What would constitute sufficient proof for the FDA to put its stamp of approval on the epidural stimulator for use in spinal cord injury? We’re in an odd situation with respect to this device, because it’s already on the market, fully insured, for use in pain-management. Today, right now, people with certain kinds of chronic back pain can have it implanted. It’s safe, or at least the FDA has already determined that it’s safe enough.

Safety, though, is only half the equation. The goal for us is that epidural stimulation be shown to be effective enough to justify the cost. We have to prove that it lowers the cost of care – that injured people who have these implants are less expensive to care for than injured people who don’t have them. We have to show that it saves more money than it costs, or it will never be adequately reimbursed by CMS, Medicare, Medicaid and private payers.

The Big Idea is a project that was built to make that case.

It began when Dr. Harkema – whose work with Rob Summers showed what was possible – and Reeve representatives met with a panel of FDA experts. At that meeting Dr. Harkema presented some of her epidural stimulation data, discussed her proposed study design and engaged in a highly informative dialogue with panel members.Throughout the last many months, CDRF has been facilitating a team made up of basic scientists, physicians, physical therapists, quality of life experts, FDA regulators, and health economics experts. Together they’ve worked out a careful, comprehensive protocol for The Big Idea.

The idea was that if the FDA regulators gave input during the design phase, the exact outcomes they’d need at the end would be built into the process. Making the right choices up front would save time in the long run, in the same way that mapping out a route is more efficient than randomly heading in the general direction you want to go.

So, where are we now? What is the design of the study? How far along is the project, and what do we expect will happen next? That’s the subject for next time.

Stay tuned.

The National Paralysis Resource Center website is supported by the Administration for Community Living (ACL), U.S. Department of Health and Human Services (HHS) as part of a financial assistance award totaling $8,700,000 with 100 percent funding by ACL/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by ACL/HHS, or the U.S. Government.