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Concept Paper on Risk Management Programs

Concept Paper on Risk Management Programs. CDER/CBER PDUFA3 Risk Management Working Group. Anne Trontell, M.D., M.P.H. Chair: Anne Trontell CDER lead: Florence Houn CBER lead: Mark Weinstein OTCOM support Nancy Smith Colleen Pritchard ORP support Aileen Ciampa Project Manager

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Concept Paper on Risk Management Programs

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  1. Concept Paper on Risk Management Programs CDER/CBER PDUFA3 Risk Management Working Group Anne Trontell, M.D., M.P.H.

  2. Chair: Anne Trontell CDER lead: Florence Houn CBER lead: Mark Weinstein OTCOM support Nancy Smith Colleen Pritchard ORP support Aileen Ciampa Project Manager Chris Bechtel CDER Susan Allen Jonca Bull Yuan-Yuan Chiu Toni Piazza Hepp Deborah Leiderman Claudia Karwoski Denise Toyer Kathleen Uhl CBER Richard Diamond Theresa Finn Brian Harvey Toni Stifano Robert Wise Risk Management Working Group

  3. Overall Context: PDUFA3 Guidances • PDUFA3 calls for three interrelated guidances on risk management • Yesterday’s and tomorrow’s concept papers focus on risk assessment components of risk management • Premarketing Risk Assessment • Pharmacovigilance/pharmacoepidemiology

  4. Context: Today’s Concept Paper • Focuses on actions taken to reduce risks in drug product use • based on assessments described in other two concept papers • Distinguishes itself from overall efforts by using the term Risk Management Programs • Calls overall process RM planning

  5. Process Highlights • Comments --> solicited today and through April 30, 2003 • Draft guidances --> Fall 2003 • Public comment period on draft guidances • Final guidances --> September 30, 2004

  6. Format of Today’s Meeting • No external panel • Workgroup and Steering Committee only • Two half-day sessions each including: • Two key sections of concept paper • Presentation of highlights by WG members • Clarifying questions on presentations • Presentations by registered public speakers • Qs & As, discussion • Afternoon concludes with brief wrap-up

  7. Scope of Concept Paper • Considerations for initiating and designing a risk management program - definitions and when appropriate • Selection of tools and levels • Evaluation processes and methods • Elements of submissions to FDA

  8. Agenda Morning Workgroup Speakers: Concepts, Definitions & When RMPs are Appropriate Dr. Robert Wise, CBER Tools and Levels of RM Programs Dr. Toni Piazza-Hepp, CDER

  9. Agenda Afternoon Workgroup Speakers: Evaluation Processes and Methods Dr. Richard Diamond, CBER Submission Elements Dr. Claudia Karwoski, CDER

  10. Morning Session: Commentary Invited • Definition of an RM Program (RMP) • Clarity of RMP goals, objectives • Determining when an RMP is needed and Sponsor/FDA roles in decision-making • Best ways for tools to be in guidance • Classification of RMP tools into Levels

  11. Afternoon: Invited Commentary • Pretesting of RMP tools • Recommended evaluation of all RMPs • Recommendation for 2 independent methods to evaluate key RMP goal(s) • Role of qualitative data in evaluation • Elements of RMP submissions & reports

  12. RISK MANAGEMENT PROGRAM CONCEPTS Robert P. Wise MD, MPH CBER

  13. OVERVIEW • Definitions • Risk Management Program (RMP) • component goals and objectives • when appropriate

  14. Definitions • Product: drug or biological regulated by CDER or CBER • Includes vaccines and plasma derivatives • Excludes other blood products • FDA approval (or licensure) means a product is : • safe and effective • for labeled indications • under labeled conditions of use • Safe means that beneficial actions: • outweigh harmful or undesirable side effects; • does not suggest absence of risk.

  15. Definition: Risk Management (RM)Methods used throughout a product’s lifecycle to: • minimize risks • optimize benefit/risk balance

  16. What is Risk Management? • Use of risk information from investigational and marketing phases • Identification and interpretation of risks and benefits in actual use • Interventions to minimize risks as necessary • Intervention phases: • Design • Implementation • Evaluation • Revision as appropriate

  17. Risk Management Planning • All sponsor efforts to minimize product risks: • Risk assessment • Pharmacovigilance/pharmacoepidemiology • Special studies or interventions • Professional product labeling (PI) • All products have risk management planning • Traditional: PI and postmarketing surveillance (PMS) • Risk management program when PI and PMS are not sufficient to minimize risks

  18. Risk Management Program (RMP) • Strategic safety effort to reduce risk: >1 risk reduction goal >1 intervention (tool) in addition to PI • Tool examples: education, forms, processes, and other methods to influence or control a product’s: • prescribing • dispensing • use Note: the package insert (PI) is that portion of the approved product labeling described in 21 CFR 201.57, that is directed primarily to health professionals. The PI should not be confused with approved product labeling which my incorporate RMP materials such as Medication Guides and patient agreements in addition to the PI.

  19. Definition: RMP Goal(s) • Tailored to specific risk concerns • Describe desired end result of RMP • Include “Vision statement” of optimal drug use scenario, e.g: “No patient with condition A (e.g. pregnancy) should receive product B (e.g. teratogen).”

  20. Definition: RM Program Objectives • Intermediate steps to an RMP goal • Affect processes, behaviors, or outcomes • Multiple objectives can serve one goal (e.g. MD training, pharmacy practice)

  21. When is an RM Program Appropriate? • Whenever risk reduction needs emerge (throughout product lifecycle) • Sponsor may volunteer or FDA propose • “When the number or severity of a product’s risks appears to undermine the magnitude of benefits in an important segment of actual or potential users.”

  22. How to Assess WhetherRisks Undermine Benefits? • No simple formula compares risks to benefits • Risk and benefit numbers, types, measures vary • Case-by-case judgments required by sponsor and/or FDA on whether to develop, submit, and implement an RMP • FDA expects: • Most products will be handled by PI. • PI revision will not automatically imply a need for RMP

  23. Risk Management Tools Toni Piazza-Hepp, Pharm. D. CDER

  24. Definitions • Risk management intervention (tool): a process or system intended to enhance safe product use by reducing risk • Risk management programs use one or more tools • Choice of tools influenced by severity, reversibility, and rate of risk

  25. Types of Tools in Current Programs

  26. Education and Outreach • Beyond professional labeling (“package insert”, PI) • HCP letters and other public notices • Training programs and CE for credit • Patient-oriented labeling • Medication Guides (MG) • Patient Package Inserts (PPI)

  27. Systems Guiding Prescribing, Dispensing, Use • Patient agreements/informed consent • Enrollment of one or more stakeholders in special program • Practitioner certification programs • Special conditions of dispensing • special packaging • limited supply / no refill • check mechanisms to assure appropriate prescribing

  28. Restricted Access Systems • Designed to enforce compliance with program elements • May include registration/enrollment of physicians/pharmacists/patients • May include documentation of safe use conditions (such as lab tests) before dispensing to patients

  29. Suspension of Marketing With or without application withdrawal

  30. Selecting and Developing ToolsConsider: • Stakeholder input: feasibility, acceptance • Consistency: with existing/accepted tools • Evidence of success in achieving desired objective based on other RMP • Evidence of success in ability of novel tool to achieve desired objectives • based on application in non-RMP settings • Variability, validity, reproducibility

  31. Proposed “Levels” for RMP Classification • Broad categorization; a continuum • Risk management planning considers all levels; basis is product risk • Risk management program (RMP) • begins at Level 2 • beyond the “package insert” • Increased level - increased divergence from conventional prescribing and dispensing

  32. Proposed “Levels” for RMP Classification Level 1: Package insert only Level 2: Adds education and outreach tools Level 3: Level 2 plus systems guiding prescribing, dispensing and/or use Level 4: Access to product requires adherence to specific program elements

  33. Morning Session: Commentary Invited • Definition of an RM Program (RMP) • Clarity of RMP goals, objectives • Determining when an RMP is needed and Sponsor/FDA roles in decision-making • Best ways for tools to be in guidance • Classification of RMP tools into Levels

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