Risk Management Considerations for Romiplostim

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Risk Management Considerations for Romiplostim

• Suzanne Berkman, PharmD
• Senior Risk Management Analyst
• Division of Risk Management
• Office of Surveillance and Epidemiology

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What is Risk Management?

• Risk assessment Risk minimization
• Routine risk management
• Labeling
• Spontaneous adverse event monitoring
• Additional measures
• Education and communication plans
• Distribution linked to safe use
• Risk assessment studies

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Amgens Proposal

• Risk minimization
• Education and communication plan
• Medication Guide, training kit, pre-use checklist
• Controlled distribution
• Risk assessment
• Evaluation of program data
• Further studies
• No direct-to-consumer advertising

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Further Considerations for Romiplostim
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Possible Criteria for Appropriate Patient
Selection

• Should the duration of ITP be specified?
• Limit to splenectomized patients?
• How should insufficient response, and
  intolerance be defined?
• Should a particular platelet count be specified?
• Should a bone marrow biopsy be required?
• Should patients with a known history of bone
  marrow stem cell disorder be considered?
• Should patients with active malignancy be
  considered?
• Should certain concomitant medication use be
  discouraged?
• Other considerations?

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Patient Monitoring

• What to monitor?
• How often to monitor?
• How to collect the data?

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Risk Management Example Tysabri (natalizumab)
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Tysabri (natalizumab)

• Indications
• Multiple Sclerosis (MS)
• Crohns disease
• Risk
• Progressive Multifocal Leukoencephalopathy (PML)
• Risk Management Program
• Tysabri Outreach Unified Commitment to Health
  (TOUCH) Prescribing Program

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TOUCH Prescribing Program

• Goals
• Minimize the risk and health consequences of PML
• Promote informed risk-benefit decisions
• Further assess the safety profile of natalizumab
• Reinforces
• Appropriate patient selection
• Risk communication
• Close patient monitoring and data collection

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Prescriber/ Patient
TOUCH Prescribing Program
Infusion Site

• Assigns patient to authorized infusion site
• Changes patient status to authorized in TOUCH
  online or provides Notice of Patient Authorization

Prescriber/ patient complete Enrollment Form
Infusion Site confirms patient is authorized to
receive natalizumab
Patient receives natalizumab infusion based on
answer to Pre-Infusion Checklist
6-month authorization periods begins and
Pre-Infusion Patient Checklists are tracked
monthly
Pre-Infusion Patient Checklist is submitted via
TOUCH online or faxed by Infusion Site
Prescriber completes Reauthorization Form every 6
months
Tracks all patients every 6 months
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Differences to Consider

• Romiplostim
• Weekly subcutaneous injection
• Administered in prescribers office
• Specific laboratory monitoring?
• Multiple serious risks
• Preventable, manageable?
• Long term safety is a concern
• Off-label use within prescribers expertise

• Tysabri (natalizumab)
• Monthly infusion
• Administered in infusion center
• No specific laboratory monitoring
• One serious known risk
• Preventable, manageable?
• Long term safety is a concern
• Off-label use outside of neurology

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Summary

• Mandatory enrollment of prescribers, patients,
  other healthcare providers, and distributors
• Clear criteria to identify the target patient
  population
• Mandatory education
• Mandatory regular monitoring
• Long-term data collection on all treated patients

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