Wed Jun 9 11:58:32 2021
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(*5e05c158*):: +public! The first vaccine to prevent rotavirus infection in infants was licensed in 1998, but withdrawn in 1999 due to risk of intussusception, a form of bowel obstruction, said Platt. Alternative rotavirus vaccines showed no increased risk in clinical trials, but postlicensure studies in other countries suggested an increased risk. In 2010, FDA began a study to quantify the possible risk among U.S. infants. Researchers used data from three Sentinel partners, and gathered data using the Current Procedural Terminology (CPT) codes for immunization and the _International Classification of Diseases, Ninth Revision_ (ICD-9) codes for intussusception and related issues. Because CPT and ICD-9 codes are “not sufficiently specific enough to do high-quality epidemiology,” said Platt, the researchers actually reviewed the full-text medical records of patients after redacting direct identifiers. Using data on 500,000 patients, the algorithm identified potential cases, and researchers obtained full medical records for 80 percent of these. Pediatricians adjudicated the records, and they found a risk of intussusception of about 1.5 per 100,000 children immunized. The clinical trials that had been performed for these vaccines, said Platt, had enrolled only 60,000 children, which may not have been sufficient to find the actual risk. This study demonstrates the value of being able to link administrative data to full-text records, said Platt.
(*5e05c158*):: +public!
— Learning from Success
— In this session of the first workshop, participants heard about successfully completed and ongoing initiatives that generated, collected, and/or analyzed real-world data (RWD) and real-world evidence (RWE). Speakers were asked to describe the features that led to success in their particular program, and to consider how these successes could be generalized and scaled for future projects.
(*5e05c158*):: +public! The Sentinel Initiative is a national medical product monitoring system that was launched in 2008 by the U.S. Food and Drug Administration (FDA) in response to legislation that required FDA to use electronic health data to support postmarketing medical product evaluation, said Richard Platt, professor and chair, Harvard Medical School, and executive director, Harvard Pilgrim Health Care Institute. While the mandate asked FDA to assess the use, safety, and effectiveness of regulatory medical products, FDA has focused Sentinel primarily on safety. However, FDA has always intended that Sentinel would be used to support a variety of activities, including clinical research, randomized trials, and public health surveillance.
Sentinel is the product of a collaboration between FDA and a large number of organizations that bring both data and scientific expertise. In essence, Sentinel is a curated, distributed dataset that adheres to a common data model, said Platt. Sentinel is a fairly simple system, with a set of linked flat file records.
(*5e05c158*):: +public! The Sentinel Initiative is a national medical product monitoring system that was launched in 2008 by the U.S. Food and Drug Administration (FDA) in response to legislation that required FDA to use electronic health data to support postmarketing medical product evaluation, said Richard Platt, professor and chair, Harvard Medical School, and executive director, Harvard Pilgrim Health Care Institute. While the mandate asked FDA to assess the use, safety, and effectiveness of regulatory medical products, FDA has focused Sentinel primarily on safety. However, FDA has always intended that Sentinel would be used to support a variety of activities, including clinical research, randomized trials, and public health surveillance.
Sentinel is the product of a collaboration between FDA and a large number of organizations that bring both data and scientific expertise. In essence, Sentinel is a curated, distributed dataset that adheres to a common data model, said Platt. Sentinel is a fairly simple system, with a set of linked flat file records.
(*5e05c158*):: U.S. Food and Drug Administration: Biologics
The 21st Century Cures Act is a main driver of the RWD and RWE initiatives at FDA, said Steven Anderson, director of the Office of Biostatistics and Epidemiology at Center for Biologics Evaluation and Research (CBER). CBER, which regulates vaccines, blood and blood products, tissue and tissue products, and cellular and gene therapy products, has undertaken several RWE initiatives in recent years, he said.
CBER uses a number of population-based data systems to conduct RWE safety and effectiveness studies, including a system being developed specifically for CBER called the Biologics Effectiveness and Safety (BEST) program. BEST is part of the Sentinel initiative, and was launched in September 2017. One goal of BEST, said Anderson, is to build data infrastructure and tools and develop expertise to conduct queries and studies of biologic products. The second goal is to automate adverse event reporting by using methods such as machine learning and natural language processing in order to mine adverse events related to biologics from EHRs and automatically submit them to FDA. The BEST program, said Anderson, will help CBER to better meet its regulatory needs by building “better, faster, cheaper systems” to generate evidence about safety and effectiveness of biologic products. CBER also uses the Sentinel system (described in
— Learning from Success
— In this session of the first workshop, participants heard about successfully completed and ongoing initiatives that generated, collected, and/or analyzed real-world data (RWD) and real-world evidence (RWE). Speakers were asked to describe the features that led to success in their particular program, and to consider how these successes could be generalized and scaled for future projects.
— Looking Ahead
— The workshops’ discussions on real-world evidence (RWE) concluded with sessions about how RWE can be used to improve different facets of the health care system, including any potential to improve health technology assessment, to transform product research and development, or to bring patients in as partners for research. Workshop participants also discussed how—based on U.S. Food and Drug Administration (FDA) input—RWE can inform regulatory decisions for biologics, drugs, and devices in the United States and abroad.
(*5e05c158*):: +public! The Sentinel Initiative is a national medical product monitoring system that was launched in 2008 by the U.S. Food and Drug Administration (FDA) in response to legislation that required FDA to use electronic health data to support postmarketing medical product evaluation, said Richard Platt, professor and chair, Harvard Medical School, and executive director, Harvard Pilgrim Health Care Institute. While the mandate asked FDA to assess the use, safety, and effectiveness of regulatory medical products, FDA has focused Sentinel primarily on safety. However, FDA has always intended that Sentinel would be used to support a variety of activities, including clinical research, randomized trials, and public health surveillance.
Sentinel is the product of a collaboration between FDA and a large number of organizations that bring both data and scientific expertise. In essence, Sentinel is a curated, distributed dataset that adheres to a common data model, said Platt. Sentinel is a fairly simple system, with a set of linked flat file records.
(*5e05c158*):: Anderson highlighted CBER’s use of RWE for real-time analysis. CBER uses the CMS claims data system to conduct near-real-time analysis of the annual flu vaccine and related adverse events such as Guillain-Barre syndrome, he said. One specific study that was performed in 2017–2018 was a rapid response effectiveness study of cell versus egg-based influenza vaccines. Using CMS data, FDA and CMS examined about 13 million vaccine doses, and found that cell-cultured vaccines were slightly more effective. These types of studies, said Anderson, can provide near-real-time information (within 4 to 6 weeks) to inform regulatory decisions concerning current and future influenza vaccines.