This document discusses access challenges for orphan drugs and specialized therapies in Canada. It proposes alternative models like managed access programs and adaptive listings to generate real-world evidence for these treatments. Managed access programs in other countries tie reimbursement to ongoing data collection on safety, effectiveness and appropriate use. Challenges include defining patient eligibility, setting stopping criteria, and assigning stakeholder roles. The document also describes private insurers' initiatives in Canada like chronic disease management programs and references to CADTH reviews. It argues collaborative solutions are needed to provide timely, equitable access to promising rare disease therapies in a sustainable way.

1. Orphan Drugs & Specialized
Needs
Innomar Strategies
Rare Disease Day 2016 Conference

2. 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

3. Increased Management of Drug ProgramsPrivate
Payer
Value DATA, and Customization will be KeyManufacturer
Cost-Containment Environment – Price DrivenPublic PayerPublic
Payer
The Payer Framework in Canada

4. Orphan Drugs
3/16/16 CONFIDENTIAL4
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

5. 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...

6. 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

7. Potential Solutions:
ManagedAccess Programs and
Adaptive Listing = Evidence
Generation Considerations

8. 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

9. 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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10. 3/16/16 CONFIDENTIAL10
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

11. Cost-Effectiveness of the HealthForward Model is under
Evaluation
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GWL-insured patient
cohort
HCM patient cohort
Non-HCM patient
cohort
Full reimbursement
for six months
Full reimbursement
without limited time
Treatment
responders
Treatment non-
responders
Continuous
reimbursement
Reimbursement
discontinued
Continuous
reimbursement
Reimbursement
discontinued
Ongoing treatment
Treatment discontinued
Treatment adherence
associated with HCM
Ongoing treatment
Treatment discontinued
Treatment adherence
associated without HCM
Accepted abstract at coming 21st ISPOR international conference:
INDEPENDENT FACTORS AFFECTING PATIENT COMPLIANCE TO PRIVATE INSURER-
FUNDED HEALTH CASE MANAGEMENT IN CANADA

12. Opportunities to Leverage Existing Patient Support Program
Natural Evidence Generation Approach

13. PSP Evidence Generation to support market access
of orphan drugs
Closing the gap between regulatory needs and payer needs

14. 3/16/16 CONFIDENTIAL14
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

15. How can We Support the Rare Disease Strategy?
Private
Payer
Manufacturer
Public PayerPublic
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