# Presentation: 337064

## Risk Assessment of Observational Data: Good Pharmacovigilance Practices and Pharmacoepidemiologic Assessment

**CDER / CBER Pharmacovigilance  Working Group**

## Working Group Members

**Group Leads:**
**Julie Beitz**
**Mark Goldberger**
**Miles Braun**
**Project Manager:**
**Patrick Guinn**

**Kathy Miracco**
**Yi Tsong**
**Edward Cox**
**Dan Shames**
**Susan Lu**
**Joyce Weaver**
**Marianne Mann**
**Judy Staffa**
**Carol Holquist**
**Min Chen**
**Melodi McNeil**
**Ralph Schmid**

**Jeff Siegel**
**Bette Goldman**
**Jerry Davis**
**Roselie Bright**
**Mary Beth Jacobs**
**Support:**
**Aileen Ciampa**
**Jane Axelrad**
**Virginia Beakes**
**Nichelle Cherry**
**Jane McCarthy**

## Workshop Agenda

**Morning**
**Overview**
**Good Pharmacovigilance Practices **
**Presentations, Qs and As**
**Afternoon**
**Pharmacoepidemiologic Assessment**
**Presentations, Qs and As**
**Closing Remarks**

## Scope of Concept Paper

**Present FDA’s preliminary thoughts on:**
**Important pharmacovigilance concepts**
**Safety signal identification**
**Pharmacoepidemiologic assessment and interpretation of safety signals**
**The development of pharmacovigilance plans**

## Scope of Concept Paper

**Focus on risk assessment based on observational data sources**
**Case Reports, Case Series**
**Pharmacoepidemiologic Studies**
**Registries**
**Surveys**

## What is Pharmacovigilance?

**	****All post-approval scientific and data gathering activities relating to the detection, assessment, understanding, and prevention of adverse effects or any other product- related problems  **
**	**
**	****This includes the use of pharmacoepidemiologic studies**

## Pharmacovigilance: Why?

**At the time of approval, clinical trial data are available on limited numbers of patients treated for relatively short periods**
**Once a product is marketed, large numbers of patients may be exposed, including:**
**Patients with co-morbid illnesses**
**Patients using concomitant medications**
**Patients with chronic exposure**

## Pharmacovigilance: Why?

**After marketing, new safety information may become available:**
**Through use of the product domestically or in other countries**
**Through use of other drugs in the same class**
**From preclinical studies**
**From pharmacologic studies**
**From controlled clinical trials**

## What is a Safety Signal?

**An apparent excess of adverse events associated with use of a product**
**Even a single well-documented case report may be viewed as a signal**

## What is a Safety Signal?

**Preclinical findings or experience with other products in the class may be sufficient to generate a signal, even in the absence of case reports in patients**
**A product’s risk profile may be characterized by several safety signals**

## Safety Signals

**Are ****identified**
**From case reports, other sources**
**Are ****evaluated**
**In pharmacoepidemiologic studies, registries, surveys**
**Are ****interpreted**
**In the context of all available safety information**
**Are ****monitored**
**Through enhanced pharmacovigilance efforts**

## What is a Pharmacovigilance Plan?

**A plan proposed by a sponsor **
**for the ongoing evaluation of safety signals identified with the use of a product**
**to monitor at-risk populations which have not been adequately studied**
**Plan may be developed at the time of product launch or after a signal is identified**

## What is a Pharmacovigilance Plan?

**A sponsor’s plan may involve:**
**Expedited reporting of serious adverse events of interest**
**Submission of adverse event report summaries at more frequent, pre-specified intervals**
**Conduct of additional observational studies or clinical trials**
**Implementation of active surveillance activities to identify as yet unreported adverse events**

## Active Surveillance May Involve...

**Product-based activities**
**Follow cohorts of patients exposed to specific products**
**Setting-based activities**
**Surveillance of healthcare settings (e.g., EDs) for events likely to present there**
**Event-based activities**
**Monitor registries for events that are often drug-related (e.g., liver transplant registries)**

## 	Emerging new safety data may result in ongoing revisions to the sponsor’s pharmacovigilance plan for a product

## 	While additional safety information is being developed, FDA will work with sponsors to: 
communicate information about safety signals and
minimize events occurring in users of a product through risk management programs1

**communicate information about safety signals and**
**minimize events occurring in users of a product through risk management programs****1**

**1****Concept Paper: Risk Management Programs**

## Workshop Agenda

**Morning**
**Overview**
**Good Pharmacovigilance Practices**** **
**Presentations, Qs and As**
**Afternoon**
**Pharmacoepidemiologic Assessment**
**Presentations, Qs and As**
**Closing Remarks**