Generating reproducible real-world evidence with multi-source data to capture unstructured clinical endpoints for chronic diseases

Ended Cooperative Agreement Awarded Last action

Award ID
U01FD007929
Recipient
Awarding agency
Obligated
$2,157,873

Award facts

Assistance listing
93.103 Food and Drug Administration Research
Completion date
Funding agency
Center for Drug Evaluation and Research Food and Drug Administration, Department of Health and Human Services
Place of performance
Boston, MA 02115, USA

Award details come from USAspending.gov.

Description

As the agency reported it.

Abstract
Randomized clinical trials (RCTS) are the gold standard for assessing treatment safety and efficacy and are the primary evidence supporting FDA's regulatory decisions. However, RCTS have a number of limitations, including the lack of generalizability of study findings and insufficient follow-up to assess long-term outcomes. With the growing availability of disease modifying treatments (DMTS), novel approaches are needed to monitor long-term safety and efficacy of agents used in chronic diseases. Electronic health record (EHR) data present the opportunity to capture longitudinal treatment response in heterogeneous patient populations and real-world settings and can be used to generate real-world evidence (RWE) to augment RCT data for these drugs. However, availability of RWE for DMTS has been limited by the lack of computable information on disease progression measures, which are the clinical outcomes monitored by physicians directing therapy. This information is typically captured only in unstructured text during clinical visits and may also not be consistently documented at every encounter, resulting in incomplete data even with labor-intensive manual abstraction. Further, IT is critical that RWE resources for robust post-market assessments of DMTS ensure reproducibility of findings across healthcare systems. In this proposal, we address this unmet need by developing methods to generate reproducible and generalizable RWE on unstructured efficacy and adverse event (AE) endpoints used in the evaluation of therapies for rheumatoid arthritis and multiple sclerosis. We will create scalable disease progression endpoints from EHR data by linking information in ehrs to registry data and building algorithms for ordinal disease activity scores using features derived from scoring guidelines. In aim 1, we integrate disease activity and progression data from registries to generate scalable RWE on disease progression endpoints leveraging structured and free-text EHR data. In aim 2, we develop strategies to correct for noise in medication prescriptions for DMTS in RWE studies. Aim 3 combines EHR data from multiple healthcare systems through federated learning to ensure generalizability of RWE. We intend for the methods to build new capabilities for use of RWE in FDA's regulatory decisions on drug effectiveness, providing an efficient, scalable, and robust approach to using real-world clinical data to support approval of new drug indications and conduct of postmarket studies for DMTS.

Transactions

Action Obligated Date
Amendment $0
Amendment $1,103,420
Original award $1,054,453

Subawards

Subrecipient Amount Awarded
Upmc Community Medicine, Inc. University of Pittsburgh hosts the expertise of project co-I Dr. Xia who is a board-certified clinical neurologist and a physician-scientist with research… $111,879
Regents Of The University Of Minnesota Dr. Hou and the GRA and will be working on the methodology development and perform analysis using the MS data from UPMC as use case. $117,667
Brigham & Womens Hospital Inc. Dr. Liao (site Pl} at Brigham and Women's Hospital is the Pl of the VERlTY RA cohort, an electronic health record (EHR} based cohort of subjects with rheumatoid… $402,750
Boston VA Research Institute, Inc. Goals: To advance the use of generate real-world evidence (RWE) as a complementary source of evidence to those from clinical trials, we propose to develop… $31,564
Brigham & Womens Hospital Inc. Dr. Liao (site Pl} at Brigham and Women's Hospital is the Pl of the VERlTY RA cohort, an electronic health record (EHR} based cohort of subjects with rheumatoid… $358,000
Upmc Community Medicine, Inc. University of Pittsburgh hosts the expertise of project co-I Dr. Xia who is a board-certified clinical neurologist and a physician-scientist with research… $110,324
Regents Of The University Of Minnesota Dr. Hou and the GRA and will be working on the methodology development and perform analysis using the MS data from UPMC as use case. $115,361

Quick answers

Who received award U01FD007929?

U01FD007929 was awarded to President And Fellows Of Harvard College by FDA Office of Acquisition and Grant Services (Food and Drug Administration) on September 1, 2023.

How much is award U01FD007929 worth?

$2,157,873 has been obligated on U01FD007929.

Which agency awarded U01FD007929?

FDA Office of Acquisition and Grant Services (Food and Drug Administration) awarded U01FD007929, with funding from Center for Drug Evaluation and Research (Food and Drug Administration).

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