Developing Better Evidence for Medical Product Safety ISPOR 16th - PowerPoint PPT Presentation

Developing Better Evidence for Medical Product Safety ISPOR 16th Annual International Meeting May 23, 2011 Joshua Benner, The Brookings Institution Judy Racoosin, U.S. Food and Drug Administration Jeffrey Brown, Harvard Medical School and Harvard Pilgrim Health Care Institute Paul Stang, Johnson & Johnson and Observational Medical Outcomes Partnership

Workshop Overview: Developing Better Evidence for Medical Product Safety ISPOR 16th Annual International Meeting May 23, 2011 Josh Benner, PharmD, ScD Fellow and Research Director, Engelberg Center for Health Care Reform The Brookings Institution Adjunct Scholar, Center for Clinical Epidemiology and Biostatistics University of Pennsylvania School of Medicine

FDA’s Post Market Safety Monitoring Tools • Phase IV Studies • Adverse Event Reporting System (AERS) – Voluntary reports submitted by patients and providers – Good at detecting very rare events that typically occur in association with medical products (e.g., Guillain Barre syndrome, agranulocytosis, Stevens Johnson syndrome) – Not good at picking up events that are common in the background or indicated population (e.g., acute myocardial infarction) • The Sentinel System – under development – Will use existing electronic health care data to more quickly identify relationships between medical products and adverse events

Sentinel Will Build Upon the Work of Others • CDC’s Vaccine Safety Datalink • HMO Research Network • Brookings forums and roundtables on postmarket evidence • Observational Medical Outcomes Partnership • Operational and pilot efforts conducted by numerous health plan-based organizations (I3 Drug Safety, Health Core) and numerous integrated delivery systems (Kaiser, GroupHealth, etc.) • AHRQ’s Effective Health Care Program and DEcIDE Networks • CERTS • eHI’s Connecting Communities for Drug Safety collaboration • Decades of pharmacoepidemiology research

FDA is Collaborating with Multiple Partners to Inform Development of the Sentinel System • FDA Pilots – Mini-Sentinel • Five year contract between FDA and Harvard Pilgrim Health Care Institute, launched in 2009 • Over 200 co-investigators from 27 institutions – Federal Partners Collaboration between FDA, CMS, DoD, and VA • Related Activities – Observational Medical Outcomes Partnership • Public-private partnership led by FDA, PhRMA, and FNIH with participation of academic and private sector investigators, launched in 2008 – Brookings • Convenes discussions under a cooperative agreement with FDA to inform development of a national active surveillance system

The Sentinel System will be an important new resource for evidence development

Workshop Objectives • The governance, scientific operations, and data infrastructure of the eventual Sentinel System will be informed by Mini-Sentinel, OMOP, and other related projects. • This workshop will provide an update on these activities and publicly available resources, featuring perspectives from scientists at FDA, Mini-Sentinel, OMOP, and the Brookings Institution.

Update on FDA’s Sentinel Initiative Judith A. Racoosin, MD, MPH Sentinel Initiative Scientific Lead US Food and Drug Administration May 23, 2011

FDA Amendments Act of 2007 Section 905: Active Postmarket Risk Identification and Analysis • Establish a postmarket risk identification and analysis system to link and analyze safety data from multiple sources, with the goals of including – at least 25,000,000 patients by July 1, 2010 – at least 100,000,000 patients by July 1, 2012 • Access a variety of sources, including – Federal health-related electronic data (such as data from the Medicare program and the health systems of the Department of Veterans Affairs) – Private sector health-related electronic data (such as pharmaceutical purchase data and health insurance claims data)

Sentinel Initiative • Improving FDA’s capability to identify and evaluate safety issues in near real time • Enhancing FDA’s ability to evaluate safety issues not easily evaluated with the passive surveillance systems currently in place • Expanding FDA’s access to subgroups and special populations (e.g., the elderly) • Expanding FDA’s access to longer term data • Expanding FDA’s access to adverse events occurring commonly in the general population (e.g., myocardial infarction, fracture) that tend not to get reported to FDA through its passive reporting systems **Will augment, not replace, existing safety monitoring systems

Sentinel Initiative: A Collaborative Effort • Data Partners – Private: Mini-Sentinel pilot – Public: Federal Partners Collaboration • Industry – Observational Medical Outcomes Partnership • All Stakeholders – Brookings Institution cooperative agreement on topics in active surveillance

Mini-Sentinel www.mini-sentinel.org Harvard Pilgrim Healthcare • Develop the scientific operations needed for an active medical product safety surveillance system • Create a coordinating center with continuous access to automated healthcare data systems, which would have the following capabilities: – Provide a "laboratory" for developing and evaluating scientific methodologies that might later be used in a fully-operational Sentinel System. – Offer the Agency the opportunity to evaluate safety issues in existing automated healthcare data system(s) and to learn more about some of the barriers and challenges, both internal and external.

Federal Partners Collaboration • An active surveillance initiative via intra-agency agreements with CMS, VA, DoD • Small distributed system – Each Partner has unique data infrastructure – No common data model being utilized • FDA proposes medical product – AE pairs to evaluate • Develop a shared protocol • Assess interpretability of query findings resulting from a decentralized analytic approach and different patient populations

Convener on Active Medical Product Surveillance Brookings Institution http://www.brookings.edu/health/Projects/surveillance • Expert stakeholder conferences • Medical Product Surveillance “Roundtables” • Active Surveillance Implementation Meetings • Annual Public Workshop

Next steps • Long-term, complex initiative – Implement in stages as scientific methodologies and data infrastructure evolves – Ensure maintenance of privacy and security within the distributed system – Continue to address the concerns of stakeholders including patients and the public • Address how the eventual Sentinel System will function as a national resource and complement other HHS initiatives using distributed systems for comparative effectiveness and quality assurance

FDA’s Mini-Sentinel Program: Overview of Primary Data Resources and Distributed Data Approach ISPOR 16th Annual International Meeting May 23, 2011 Jeffrey Brown, PhD Harvard Pilgrim Health Care Institute and Harvard Medical School

Mini-Sentinel Coordinating Center Jeff_brown@hphc.org

Distributed Data Partners Jeff_brown@hphc.org

Additional Partners Institute for Health Jeff_brown@hphc.org

Investigators ~200, including:  Investigators from all data partner organizations  All Vaccine Safety Datalink Principal Investigators  12 AHRQ CERTs PIs  9 AHRQ DEcIDE center PIs  12 current/former FDA advisory committee members  3 IOM “Future of Drug Safety” committee members  4 International Society of Pharmacoepidemiology presidents  Critical Path Institute leadership Jeff_brown@hphc.org

Principles/Policies  Public health practice, not research  Protect confidentiality  Data partners participate voluntarily  Minimize transfer data  Maximize transparency  Public availability of findings, tools, methods, etc.  Avoid/manage conflict of Interest  Promote development of epidemiologic, statistical, and data knowledge Jeff_brown@hphc.org

Rationale for a Distributed Data System  Little or no exchange of person-level data is needed to answer many safety, effectiveness, and quality questions  A relatively small subset of items in electronic health data systems can answer most of these questions  Data Partners’ knowledge of their data is essential in protocol development and in interpretation of findings  Data Partners prefer to control access to, and uses of, their data  Analyses should be implemented identically at all sites  Secondary uses must minimize impact on data partner operations Jeff_brown@hphc.org

Approach to Data  No centralized dataset  Common data model  Distributed programs Jeff_brown@hphc.org

Mini-Sentinel Distributed Analysis FDA Operations Center 1 Mini-Sentinel Portal 2 5 Data Partner Firewall / Policies Review & Review & Run Program Return Results 4 Local Datasets 3 Local Datasets Local Datasets Local Datasets 1- Query (an executable program) is submitted by FDA to the Operations Center and loaded onto the Mini-Sentinel Portal 2- Data Partners retrieve the query on the Distributed Querying Portal 3- Data Partners review query and perform analysis locally by executing the distributed program 4- Data Partners review results 5- Data Partners return results to Distributed Querying Portal for review by Operations Center and sent to FDA Jeff_brown@hphc.org

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