DUBLIN – A novel real-world study methodology that marries digital technology with a citizen science or do-it-yourself approach to “drug” procurement, formulation and administration has found – yet again – that the placebo remains one of the great wonders of the medical world.
LONDON – After a year in the mill, the U.K. National Institute for Health and Care Excellence (NICE) has put proposals for how it intends to evaluate drugs, devices and diagnostics in the future out for consultation.
Tracking the pandemic in all its manifestations – from symptoms and spread, to viral genomics and repurposing drugs – has massively increased appreciation of the importance of real world data, with significant implications for the future of drug discovery and clinical development, the use of patient data and health care as a whole.
The U.S. FDA has been easing gently down the road of real-world evidence (RWE) in regulatory decision-making, and the case of the Watchman left atrial appendage device is instructive in this regard. Robert Shipley, of Boston Scientific Corp. (BSX), said on webinar hosted by the Advanced Medical Technology Association that the FDA changed gears and agreed to a registry for a post-approval study for the first generation Watchman, but added that the use of registry data and other RWE for approval of a next-generation device is as yet more aspirational than practicable.
China’s provincial government for Hainan Province has two sets of medical devices going through the urgent medical device needs program, but leveraging this program for regulatory approval in China is still no small undertaking. One complication is that any real-world evidence gathered in Hainan might not suffice for nationwide regulatory approval, but patient recruitment is also difficult.
Real-world evidence (RWE) and clinical trial data might seem to bear little resemblance to each other, but Naomi Aronson, executive director for clinical evaluation at the Blue Cross and Blue Shield Association, said there are problems common to both. The most significant of these is an absence of validated outcomes measures for many conditions, measures Aronson said are “desperately” needed in order to make an appropriate coverage determination.
The U.S. FDA has put a considerable amount of emphasis on patient perspectives regarding medical devices in recent years, the subject of a recent webinar hosted by the Medical Device Innovation Consortium (MDIC). One of the speakers on the webinar, Carrie Kuehn, of Evidation Health of San Mateo, Calif., said that not only do device makers have a trove of valuable data at their fingertips with patient preference data, but also that the FDA’s message is, “if patients say something, we want to hear about it.”
The use of real-world data (RWD) and real-world evidence (RWE) in regulatory decision-making may seem more aspirational than practical at times, but that state of affairs is changing rapidly. Wendy Rubenstein, director of personalized medicine at the U.S. FDA’s Center for Devices and Radiological Health, said contemporary versions of these real-world sources of evidence “are of progressively higher quality” in recent years, and thus RWE and RWD may soon hit their strides as mature sources of data with which regulators are increasingly comfortable.
The FDA has typically used real-world evidence (RWE) as a way of monitoring safety issues post-approval, especially through the Sentinel Initiative, which started in response to the FDA Amendments Act of 2007.
