Under the Information Exchange and Data Transformation initiative, existing clinical trial datasets will be standardized and integrated with real-world sources in a new data environment.
FDA’s Office of Hematology and Oncology Products is trying to better leverage the clinical data already in-house while also integrating it with real-world data sources under a new initiative.
OHOP’s Information Exchange and Data Transformation (INFORMED) program is designed to support novel scientific research and advance regulatory science by building an organizational and technical infrastructure. This infrastructure will enable research using aggregated clinical trial datasets and emerging pipelines of real-world data, such as from electronic medical record systems, biometric monitoring devices, wearable technologies and social media networks.
At a June 16 Friends of Cancer Research/Alexandria Summit-sponsored meeting on real-world evidence, FDA Commissioner Robert Califf described INFORMED as a good example of taking advantage of data that’s already at FDA, albeit mostly in context of randomized trials.
- Transform existing clinical trial datasets for oncology products into a common standard.
- Develop a data environment for storage and mining of transformed and standardized datasets.
- Incorporate and organize diverse pipelines of data, including omics, electronic medical records and biometric sensor technologies, into the data environment.
Califf cited three objectives for the initiative (see box).
FDA told Pink Sheet that conceptualization and planning for the initiative began in Summer 2015, and core programming and development activities started in March. Data transformation occurs in parallel to research efforts, and the agency has been able to conduct descriptive analyses on aggregated datasets and is in the process of developing and testing formal hypotheses.
“With INFORMED, we are developing both technical and organizational capabilities, and the initiative is not just about data science and technology but also stakeholder engagement,” the agency said. “The lessons learned from the experience since the planning stages in the Summer of 2015 have allowed us to seed multiple internal and external collaborative research projects.”
Pilot Project On Pre-Market Safety Reports
One of these internal collaborations is with the Office of Surveillance and Epidemiology on a pilot to digitize all pre-market expedited safety reports in oncology, FDA said. This pilot is in its final stage, with four companies volunteering to submit pre-market safety reports via an electronic gateway as datasets according to technical specifications developed in the pilot’s first stage rather than the usual way they are sent to FDA, as pdf files or on paper.
“The goal of the pre-market safety project is to allow the FDA and sponsors to exchange safety data efficiently and in near-real time and reduce the number of uninformative reports that can potentially mask true safety signals,” the agency said.
The agency is currently evaluating proposals from industry, initiated as a direct result of outreach activities in conjunction with INFORMED, for use of real-world data as supportive evidence for oncology product approval.
Sean Khozin, an OHOP senior medical officer who is co-founder and project lead for INFORMED, told the FOCR/Alexandria Summit conference that OHOP is experimenting with novel pipelines of data and has a real-world data working group that frequently meets to explore needs and opportunities where such evidence can potentially support regulatory decisions.
“You can easily envision that once we do have … more experience with these new pipelines of data, that data can come as part of formal regulatory submission and the FDA can use that data in its decision-making process,” Khozin said.
The agency sees a role for real-world data beyond product approval and labeling expansions to include informing labeling language and risk management plans (see related story, “Real-World Evidence May Find A Home On Breakthrough Pathway” — “The Pink Sheet,” Jun. 27, 2016).
FDA is talking to sponsors of PD-1 immune-checkpoint inhibitors about a real-world study that would be conducted through clinical practice (see related story, “FDA, PD-1 Inhibitor Sponsors Discussing Real-World Study” — “The Pink Sheet,” Jun. 27, 2016).
OHOP’s experience with INFORMED could serve as a model for other review divisions.
“We are looking pretty closely at this as a model perhaps for other areas,” Califf said. Noting that he recently met with the Institute of Medicine’s neurosciences forum, Califf said: “If you want to talk about a field that’s in desperate need of better information, it’s the field of mental health and chronic neurologic diseases.”