sandra anderson: rare disease day 2016 conference
TRANSCRIPT
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Orphan Drugs & Specialized Needs Innomar Strategies
Rare Disease Day 2016 Conference
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Agenda
§ Review of Payer Framework
§ Review of Orphan Drug Characteristics- why are they different?
§ Consideration of Current Access Challenges
§ Consideration of alternative models for access:
- Managed Access
- Adaptive Listings
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Increased Management of Drug Programs
Private Payer
Value DATA, and Customization will be Key
Manufacturer
Cost-Containment Environment – Price Driven Public Payer
Public Payer
The Payer Framework in Canada
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Orphan Drugs
3/16/16 CONFIDENTIAL 4
The “Why” and The “How”
SPECIALTY DRUGS ORPHAN DRUGS
Small Population <500 ~100-300 Patients
Burden of Illness / Population Impact Definitive studies Non-definitive studies
New Born Screening Not Required Required
Delayed Diagnosis / Definitive Diagnosis Definitive Diagnosis Delayed Diagnosis
Genetic Testing / Component Not Applicable ~80% of Rare Disease Population has a genetic component
Reimbursement Challenges Established Reimbursement Framework No clear path, despite other established markets (FDA, EU)
Financial Assistance Yes Yes, with conditions
Health Outcomes Studies Not Required Required
Integrated / Closed Distribution Recommended *Required for Health Outcomes
Measurements and Adherence
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Specialty Reimbursement through Private Payers
Timelines
• Up to 100 days to gain access
• Variation Private vs. Public
Medical Policy
• Defined criteria for use (e.g. clinical effectiveness, selected specialties, 3° centers, etc.)
• Prior Auth.
• Published
PLA
• Additional conditions to limit exposure for a specific payer (e.g. volume-based price, capped usage, etc.)
• Prior Auth.
• Terms are usually confidential
Case Management
• Reimbursement decision based on individual patient assessment
• Requires prior auth.
• Subject to periodic review
3/16/16 CONFIDENTIAL 5
Cost Containment Is Key PPNs; Capped Mark-Ups; Pooling...
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Medavie Blue Cross • Managing Chronic
Disease Program: 56% of workforce, ≈
• Self-care approach for chronic disease; targeted health services delivered by specialized HCPs
• Diabetes Management Program (pharma partnership)
Manulife § Designed to delay listing
& reimbursement until CADTH has reviewed
§ Manulife will review
based on its own schedule
Sun Life Provincial Integration Program
Great West Life • Monitoring a patient for
a specific period of time to ensure the best health outcome Sun Life Manulife GreenShield Medavie
Private Sector Policy Initiatives
3/16/16 CONFIDENTIAL 6
Green Shield Cooperators Medavie Sun Life Manulife
INSURERS REFERENCING
CADTH/CDR
HEALTH CASE MANAGEMENT PPNs MANAGING CHRONIC
DISEASE
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Potential Solutions: Managed Access Programs and Adaptive Listing = Evidence Generation Considerations
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Italy
§ Drug-monitoring to asses and track patient eligibility, evaluate utilization in clinical practice, collect epidemiological data (safety data and post marketing info)
§ Meant to guarantee appropriate use of medicines according to its therapeutic indication
§ Providing important information on the tolerability of a new drug and prescribing appropriateness.
3/16/16 CONFIDENTIAL 8
Example of Adaptive Listing Model: Start, Stop Criteria to be negotiated with Manufacturer
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Challenges with Managed Access Programs
Defining patient eligibility
• Collecting data on outcomes
• Who will be responsible for the patient registries?
• How will confidentiality be maintained?)
Setting stopping criteria
• Is it possible to achieve a consensus on stopping criteria when rare diseases are so heterogeneous?
• How will decisions be made for those who cannot speak for themselves?
Assigning stakeholder roles
and responsibilities
• What roles will Health Canada, payers, and pharmaceutical companies have?
• How will patient involvement be organized?
MAPs must work for both patients
and decision-makers.
• To avoid some of the issues such as backlash when the evidence does not support continued funding
• There is need to incorporate feedback from all stakeholders, including patients.
MAPs should consider all
support
• Support for patients but not just the drug alone
3/16/16 CONFIDENTIAL 9
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3/16/16 CONFIDENTIAL 10
The HealthForward Model in Canada
3/16/16 CONFIDENTIAL 10
Collaborate with the patient and their healthcare team
during all phases of the patient journey from diagnosis to
treatment and beyond
Collecting meaningful insights through well-defined
feedback mechanisms and action planning
Exceptional patient and healthcare
professional quality of care equates to
better overall adherence
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Cost-Effectiveness of the HealthForward Model is under Evaluation
3/16/16 CONFIDENTIAL 11
GWL-insuredpatientcohort
HCMpatientcohort
Non-HCMpatientcohort
Fullreimbursementforsixmonths
Fullreimbursementwithoutlimitedtime
Treatmentresponders
Treatmentnon-responders
Continuousreimbursement
Reimbursementdiscontinued
Continuousreimbursement
Reimbursementdiscontinued
Ongoingtreatment
Treatmentdiscontinued
TreatmentadherenceassociatedwithHCM
Ongoingtreatment
Treatmentdiscontinued
TreatmentadherenceassociatedwithoutHCM
Accepted abstract at coming 21st ISPOR international conference: INDEPENDENT FACTORS AFFECTING PATIENT COMPLIANCE TO PRIVATE INSURER-FUNDED HEALTH CASE MANAGEMENT IN CANADA
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Opportunities to Leverage Existing Patient Support Program Natural Evidence Generation Approach
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PSP Evidence Generation to support market access of orphan drugs Closing the gap between regulatory needs and payer needs
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3/16/16 CONFIDENTIAL 14
Considerations for Orphan Program Solutions Cover certain drugs by therapeutic class based on: Adaptive Listing Scheme following a framework based on: • Agreed upon criteria per Orphan category; • General framework for post marketing surveillance
requirements
Central Registry/PSP model
• Testing requirements vary – will not be able to standardize consistent approach
Genetic Testing requirements
• Link to Payer validated data requirements • Consider Central Data Collection • Monitoring tool to provide ability to cover drugs based on
evaluation of outcomes- proactively agreed upon data points
Data Collection criteria based on approved guidelines
3/16/16 CONFIDENTIAL
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How can We Support the Rare Disease Strategy?
Private Payer
Manufacturer
Public Payer
Public Payer
Provide timely, equitable and evidence-informed care: Right patient, Right drug, Right time
Providing sustainable access to promising therapies Consider offering a unique model based on outcomes for Rare Diseases
Generate real-world evidence to meet market access needs Disease burden studies to demonstrate unmet medical needs and support advocacy of rare disease