Former FDA Deputy Chief Says More Surveillance is Badly Needed

Despite a pressing need in the device sector for postmarketing surveillance, studies remain infrequent and limited in scope, and though the FDA has envisioned a robust system using medical records to track device safety, any such system is years from adoption, would need additional funding, and may not survive the new administration’s interest in deregulation.

That’s according to Joshua Sharfstein — who served as FDA deputy commissioner from 2009 to 2011, was then secretary of the Maryland state Department of Health and Mental Hygiene, and is now associate dean at the Johns Hopkins Bloomberg School of Public Health.

To illustrate the point, Sharfstein and researchers Rita Redberg and Alison Jacoby use power morcellators as an example, in a Journal of the American Medical Association article.

Morcellators

The laparoscopic surgical devices that allow minimally invasive hysterectomy and myomectomy for large uteri and fibroids, were first described in 1993 and were then widely adopted by gynecologists. Uptake of the new technology was rapid. From 2005 to 2013, the rate of outpatient laparoscopic hysterectomy increased from 31.4 to 161.6 per 100,000 adult women, whereas the rate of inpatient hysterectomy declined from 172.1 to 72.1 per 100,000 adult women, with doctors using power morcellators for an estimated 55,000 to 75,000 procedures annually in the United States at peak, wrote Sharfstein et al.

In 2013, however, power morcellation came under scrutiny when a highly publicized case raised awareness that some masses diagnosed as fibroids might actually be undiagnosed cancers — and the use of the devices might spread malignant cells from these tissues throughout the abdomen. The FDA investigated the issue, and, in April 2014, issued a safety communication estimating the risk of an unexpected uterine sarcoma at 1 in 350 patients undergoing hysterectomy or myomectomy.

Later that year, the agency required a “black box” warning label on power morcellator devices. After that, the use of power morcellators saw a steep drop, with the largest manufacturer of the devices withdrawing them from the market.

Much controversy ensued. Many charged that the FDA failed to act sufficiently to protect patients, saying the agency should have removed the devices from the market. Others claimed FDA acted too aggressively and interfered inappropriately with patient care. Some said women should have been able to choose the technology for their procedures if they wanted it and understood the risks.

Competing Views

“The dispute reveals, in part, that there remain competing views about how regulatory decisions should be made about the use of medical products as evidence emerges on safety risks,” wrote Sharfstein et al. “But it also indicates how such ideological disagreements become more prominent when there is a lack of evidence on risks and benefits at the time of approval, coupled with a lack of ongoing data collection to address key questions.”

Other information clouded the matter: The FDA estimated the risk of an unexpected uterine sarcoma at approximately 1 in 350 women undergoing hysterectomy or myomectomy, while others have estimated the risk at approximately 1 in 2000.

Which is correct and should be weighed more heavily?

“These and other empirical questions are unresolved in part because of what is missing: a comprehensive system of data collection based on the experience of many thousands of women who have undergone these procedures,” wrote Sharfstein et al.

Morcellators, like many devices cleared through the 510K pathway, entered the market based on their “substantial equivalence” to prior devices; in this case, the equivalence was to a device used in arthroscopic joint surgery.

Even though the risk of potentially spreading cancer was known at the time of FDA clearance of the first of these devices in 1991, no clinical studies to assess the issue were required and no postmarketing requirements for data collection were put into place, wrote Sharfstein et al.

“Without such requirements, there was little incentive for the industry sponsors of the device to voluntarily gather such data,” the researchers wrote.

“Moreover, physicians did not demand this information, instead relying on individual clinical experience. If the FDA had established a strong surveillance system, the agency could have reviewed outcomes years earlier, with far higher–quality data than are available today.”

Sharfstein told IDDM that unfortunately, this cycle may need to be repeated a few more times before meaningful change will come.

“The FDA is clearly interested in better post-market surveillance,” he says. “It may take a few more controversies like this one to convince device makers and the clinical community of the value of this type of data.”

Until a postmarketing surveillance approach for medical devices is established that can answer essential questions quickly, clinical medicine will remain in data purgatory, vulnerable to the next dispute over the safety of a new device or medical technology, wrote Sharfstein and his colleagues.

“Escaping this state requires recognizing that a common ground exists beyond ideological debates about regulatory decision making: strong data requirements that benefit patients, physicians, and industry alike,” Sharfstein et al wrote.

Sharfstein told IDDM that unfortunately, this cycle may need to be repeated a few more times before meaningful change will come.

“The FDA is clearly interested in better post-market surveillance,” he said. “It may take a few more controversies like this one to convince device makers and the clinical community of the value of this type of data.”