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Risk Assessment of Observational Data: Risk Assessment of Observational Data: Good Pharmacovigilance Practices Good Pharmacovigilance Practices
andand Pharmacoepidemiologic AssessmentPharmacoepidemiologic Assessment
CDER / CBER Pharmacovigilance CDER / CBER Pharmacovigilance Working GroupWorking Group
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Working Group MembersWorking Group Members
Group Leads:Group Leads:• Julie BeitzJulie Beitz• Mark GoldbergerMark Goldberger• Miles BraunMiles Braun
Project Manager:Project Manager:• Patrick GuinnPatrick Guinn
• Kathy MiraccoKathy Miracco
• Yi TsongYi Tsong
• Edward CoxEdward Cox
• Dan ShamesDan Shames
• Susan LuSusan Lu
• Joyce WeaverJoyce Weaver
• Marianne MannMarianne Mann
• Judy StaffaJudy Staffa
• Carol HolquistCarol Holquist
• Min ChenMin Chen
• Melodi McNeilMelodi McNeil
• Ralph SchmidRalph Schmid
• Jeff Siegel
• Bette Goldman
• Jerry Davis
• Roselie Bright
• Mary Beth Jacobs
Support:
• Aileen Ciampa
• Jane Axelrad
• Virginia Beakes
• Nichelle Cherry
• Jane McCarthy
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Workshop AgendaWorkshop Agenda
• MorningMorning– OverviewOverview– Good Pharmacovigilance Practices Good Pharmacovigilance Practices – Presentations, Qs and AsPresentations, Qs and As
• AfternoonAfternoon– Pharmacoepidemiologic AssessmentPharmacoepidemiologic Assessment– Presentations, Qs and AsPresentations, Qs and As– Closing RemarksClosing Remarks
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Scope of Concept PaperScope of Concept Paper
• Present FDA’s preliminary thoughts on:Present FDA’s preliminary thoughts on:– Important pharmacovigilance conceptsImportant pharmacovigilance concepts– Safety signal identificationSafety signal identification– Pharmacoepidemiologic assessment and Pharmacoepidemiologic assessment and
interpretation of safety signalsinterpretation of safety signals– The development of pharmacovigilance The development of pharmacovigilance
plansplans
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Scope of Concept PaperScope of Concept Paper
• Focus on risk assessment based on Focus on risk assessment based on observational data sourcesobservational data sources– Case Reports, Case SeriesCase Reports, Case Series– Pharmacoepidemiologic StudiesPharmacoepidemiologic Studies• RegistriesRegistries• SurveysSurveys
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What is Pharmacovigilance?What is Pharmacovigilance?
All post-approval scientific and data All post-approval scientific and data gathering activities relating to the detection, gathering activities relating to the detection, assessment, understanding, and prevention assessment, understanding, and prevention of adverse effects or any other product- of adverse effects or any other product- related problems related problems
This includes the use of This includes the use of pharmacoepidemiologic studiespharmacoepidemiologic studies
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Pharmacovigilance: Why?Pharmacovigilance: Why?
• At the time of approval, clinical trial data are At the time of approval, clinical trial data are available on limited numbers of patients available on limited numbers of patients treated for relatively short periodstreated for relatively short periods
• Once a product is marketed, large numbers Once a product is marketed, large numbers of patients may be exposed, including:of patients may be exposed, including:– Patients with co-morbid illnessesPatients with co-morbid illnesses– Patients using concomitant medicationsPatients using concomitant medications– Patients with chronic exposurePatients with chronic exposure
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Pharmacovigilance: Why?Pharmacovigilance: Why?
• After marketing, new safety information After marketing, new safety information may become available:may become available:– Through use of the product domestically or in Through use of the product domestically or in
other countriesother countries– Through use of other drugs in the same classThrough use of other drugs in the same class– From preclinical studiesFrom preclinical studies– From pharmacologic studiesFrom pharmacologic studies– From controlled clinical trialsFrom controlled clinical trials
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What is a Safety Signal?What is a Safety Signal?
• An apparent excess of adverse events An apparent excess of adverse events associated with use of a productassociated with use of a product– Even a single well-documented case report Even a single well-documented case report
may be viewed as a signalmay be viewed as a signal
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What is a Safety Signal?What is a Safety Signal?
• Preclinical findings or experience with Preclinical findings or experience with other products in the class may be other products in the class may be sufficient to generate a signal, even in the sufficient to generate a signal, even in the absence of case reports in patientsabsence of case reports in patients
• A product’s risk profile may be A product’s risk profile may be characterized by several safety signalscharacterized by several safety signals
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Safety SignalsSafety Signals
• Are Are identifiedidentified– From case reports, other sourcesFrom case reports, other sources
• Are Are evaluatedevaluated– In pharmacoepidemiologic studies, registries, In pharmacoepidemiologic studies, registries,
surveyssurveys
• Are Are interpretedinterpreted– In the context of all available safety informationIn the context of all available safety information
• Are Are monitoredmonitored– Through enhanced pharmacovigilance effortsThrough enhanced pharmacovigilance efforts
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What is a Pharmacovigilance Plan?What is a Pharmacovigilance Plan?
• A plan proposed by a sponsor A plan proposed by a sponsor – for the ongoing evaluation of safety signals for the ongoing evaluation of safety signals
identified with the use of a productidentified with the use of a product– to monitor at-risk populations which have not to monitor at-risk populations which have not
been adequately studiedbeen adequately studied
• Plan may be developed at the time of product Plan may be developed at the time of product launch or after a signal is identifiedlaunch or after a signal is identified
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What is a Pharmacovigilance Plan?What is a Pharmacovigilance Plan?
• A sponsor’s plan may involve:A sponsor’s plan may involve:– Expedited reporting of serious adverse events of Expedited reporting of serious adverse events of
interestinterest– Submission of adverse event report summaries at Submission of adverse event report summaries at
more frequent, pre-specified intervalsmore frequent, pre-specified intervals– Conduct of additional observational studies or Conduct of additional observational studies or
clinical trialsclinical trials– Implementation of active surveillance activities to Implementation of active surveillance activities to
identify as yet unreported adverse eventsidentify as yet unreported adverse events
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Active Surveillance May Involve...Active Surveillance May Involve...
• Product-based activitiesProduct-based activities– Follow cohorts of patients exposed to specific Follow cohorts of patients exposed to specific
productsproducts• Setting-based activitiesSetting-based activities– Surveillance of healthcare settings (e.g., EDs) Surveillance of healthcare settings (e.g., EDs)
for events likely to present therefor events likely to present there• Event-based activitiesEvent-based activities– Monitor registries for events that are often Monitor registries for events that are often
drug-related (e.g., liver transplant registries)drug-related (e.g., liver transplant registries)
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Emerging new safety data may result Emerging new safety data may result in ongoing revisions to the sponsor’s in ongoing revisions to the sponsor’s pharmacovigilance plan for a productpharmacovigilance plan for a product
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While additional safety information is While additional safety information is being developed, FDA will work with being developed, FDA will work with sponsors to: sponsors to: – communicate information about safety communicate information about safety
signals andsignals and– minimize events occurring in users of a minimize events occurring in users of a
product through risk management product through risk management programsprograms11
1Concept Paper: Risk Management Programs
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Workshop AgendaWorkshop Agenda
• MorningMorning– OverviewOverview– Good Pharmacovigilance PracticesGood Pharmacovigilance Practices – Presentations, Qs and AsPresentations, Qs and As
• AfternoonAfternoon– Pharmacoepidemiologic AssessmentPharmacoepidemiologic Assessment– Presentations, Qs and AsPresentations, Qs and As– Closing RemarksClosing Remarks