sandra anderson: rare disease day 2016 conference
TRANSCRIPT
Orphan Drugs & Specialized Needs Innomar Strategies
Rare Disease Day 2016 Conference
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
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
Orphan Drugs
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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
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
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Cost Containment Is Key PPNs; Capped Mark-Ups; Pooling...
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
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Green Shield Cooperators Medavie Sun Life Manulife
INSURERS REFERENCING
CADTH/CDR
HEALTH CASE MANAGEMENT PPNs MANAGING CHRONIC
DISEASE
Potential Solutions: Managed Access Programs and Adaptive Listing = Evidence Generation Considerations
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.
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Example of Adaptive Listing Model: Start, Stop Criteria to be negotiated with Manufacturer
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
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The HealthForward Model in Canada
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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
Cost-Effectiveness of the HealthForward Model is under Evaluation
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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
Opportunities to Leverage Existing Patient Support Program Natural Evidence Generation Approach
PSP Evidence Generation to support market access of orphan drugs Closing the gap between regulatory needs and payer needs
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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
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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