FDA’s MyStudies release is a ‘major milestone’ with ‘profound’ implications, says Iqvia
The US Food and Drug Administration (FDA) partnered with Kaiser Permanente on a pilot study to measure the app’s functionality and subsequently released the open source code and technical documents. This release will allow researchers to customize and use the MyStudies app for real-world evidence collection via patients’ mobile devices, according to the agency.
John Doyle, senior vice president and general manager at Iqvia, said the announcement is a major milestone on a few fronts. As it pertains to real-world evidence (RWE) guidance, the release broadens the channel options for real-world data (RWD) collection, he said.
“On the patient-centricity initiative, it amplifies the patient voice, providing them with the ability to contribute patient-generated health data (PGHD) from their own wearable devices and medical apps,” Doyle told us.
The tool also enhances the FDA’s ability to measure the quantitative benefit-risk profile of new products with “a more holistic view of the patient experience,” he said, which furthers the patient-centric approach.
Additionally, because it is open-source, the tool provides all stakeholders the ability to participate in co-creating novel digital tools, catalyzing what Doyle calls a “network effect” in RWE research.
What are the implications for the CRO industry, pharma, and patients?
Doyle said the implications are profound. “I believe this digital tool will serve as a gateway to development of next-generation digital tools for collecting RWD and facilitating ‘translation’ to RWE,” he said.
“Better capture of real-world data, collected from a variety of sources, has the potential to make our new drug development process more efficient, improve safety, and help lower the cost of product development,” FDA Commissioner Scott Gottlieb, MD, said in the press release.
In addition, Doyle said the tools will facilitate better studies by capturing more relevant patient-centric endpoints.
“In the late-phase space, it will also enable more direct-to-patient studies, reducing research time and cost burden so larger populations can be studied across more heterogeneous settings,” he added. This should augment the volume of comparative effectiveness studies as well as the robustness and generalizability of benefit-risk product profiles.
However, Doyle said there is a question about the feasibility of implementation.
“While Kaiser Permanente is an ideal place to evaluate the ‘internal validity’ of the application, that is, ‘does it work?’ External validity or generalizability of those findings to other hospital systems is uncertain,” he said.
“With the exception of a few of the largest and most advanced integrated delivery networks, most health care systems in the US do not have access to the high level of technology and integrated data as Kasier.”
