This document summarizes a workshop on designing managed access programs (MAPs) for rare disease drugs. The workshop objectives were to enhance understanding of MAPs' role in patient access and work in groups to design MAPs within national pharmacare. The agenda included case study discussions, working groups to refine MAP proposals, and summarizing recommendations. Case studies examined complex therapies, gene therapies, and rare disease drugs. Groups addressed questions on stakeholder involvement, eligibility, stopping criteria, ensuring compliance, and using data for decisions. Feedback informed further discussion on feasibility and a closing discussion reflected on progress and remaining questions around health technology assessments, clinician input, expertise, and patient experiences.
2. Facilitators
• Tania Stafinski, University of Alberta, PRISM
• Judy Glennie, PRISM
PRISM – Promoting Rare-Disease Innovations through
Sustainable Mechanisms
3. Objectives
1. To do a deep-dive to enhance our understanding of the
potential role of MAPs for enhancing patient access to drugs
for rare diseases (DRDs)
2. To work in multi-stakeholder groups to apply principles and
methods of designing and implementing Managed Access
Programs within the context of National Pharmacare, CORD’s
Proposal for Access to Rare Disease Drugs, and the proposed
FPT Process for Complex/Specialized Drugs
3. To place particular emphasis on discussion of the role of
patients in MAPs.
4. Agenda
Time Topic Presenters
1045am –
1245pm
Workshop Introduction and Case Studies
• Overview of MAPS
• Workshop instructions
• Case study group work
• Report back
Tania Stafinski, Judy
Glennie
PRISM
1245-130pm Lunch
130-215pm Working Groups: Refining MAPs
• Participants will refine their proposed MAPs
based on feedback and other proposed MAP
designs with the goal of improving feasibility and
sustainability.
Tania Stafinski, Judy
Glennie
PRISM
215-3pm Summary and wrap-up
• Review of key recommendations
• Closing discussion
Tania Stafinski, Judy
Glennie
PRISM
6. WHAT HAVE WE HEARD SO FAR
• Patients need treatment options
• There are lots of promising therapies
• But significant uncertainty exists
- Uncertainty over who and how many could benefit
- Uncertainty over benefit and magnitude of benefit ->
uncertainty over the value proposition
7. Working towards a shared goal of patient access that is:
• Appropriate
• Responsible
• Sustainable
WHAT HAVE WE HEARD SO FAR
8. WHAT WE HAVE HEARD SO FAR
Regulatoryapproval
Coverage
Managed Access
Programs (MAPS)
9. WHAT ARE MAPS?
• Form of contractual agreement between manufacturers and
payers
• Provides patients with access to a drug: if it does what it is
supposed to do, they stay on it, but if it doesn’t, they don’t
• Ties outcomes to ongoing reimbursement
• Real world evidence collected on outcomes around which
there is uncertainty
• Outcomes pre-specified
• May be related to clinical benefit, cost-effectiveness, cost/patient,
budget impact, etc.
• Have been used elsewhere
• Many different names
10. WHEN ARE MAPS USED?
Unmet
need
Severe
disease
Small
patient
population
Promising
evidence
of clinical
benefit
Limited
evidence
High
decision
uncertainty
11. THE BURNING QUESTION
How can we create MAPs that ensure
appropriate, responsible, sustainable
access to therapies for rare diseases?
13. Case Studies
1. Complex (combination) Therapies
2. Gene Therapy #1
3. Drugs for Rare Diseases
4. Gene Therapy #2
• See case descriptions on tables
• 2 groups will review each case
14. Process
1. Please take 5 minutes on your own to review
your assigned case study
2. Pick at time-keeper
3. Pick a note-taker/reporter
4. Review the questions
5. Address the questions as a group
ASSUMPTIONS:
• We are assuming that, in fact, there should be a MAP for the drugs in
each of the case studies.
• We are starting with a blank slate – i.e., we are starting at the first part
of the process for developing a MAP.
15. Questions – Part 1
1. Who should be involved in the design of your MAP? What would be role of
each group involved? (NOTE: please do not spend any more than 5 minutes
on this question!)
2. How should we decide who is eligible to participate in the MAP? Why?
3. How should we determine the “stopping criteria” for the MAP? Why?
4. What needs to be put into place to ensure that all stakeholders abide by the
“rules” of the MAP? What kind of accountabilities should patients have as
part of their role in the MAP?
5. How should the data from the MAP be used for decision-making?
16. Report Back
• Short summary of case by facilitator
• Comments on each case study by 2 groups
assigned
• Comments from other participants
19. Questions – Part 2
1. How would you change your considerations
for your MAP based on what you heard
during the report back?
2. What do you think needs to be
considered/done in order to make your MAP
feasible?
24. New proposed “supplemental process” for complex /
specialized meds – including rare disease drugs
• Background
• Created by the provincial and territorial health ministers
• Led by BC, Alberta and Ontario public drug programs
• Objective
Implement a proactive, consistent, fair, transparent process to facilitate
responsive funding decisions
• Next steps
• Consultation beginning this week and through late 2018 (CADTH
supporting)
• Working group will develop recommendations to health ministers
• Potential implementation in Spring 2019
2
25. What the process envisions
• Concurrent regulatory, evaluation and pricing reviews
(Health Canada, CADTH, and PMPRB)
• Followed by sequential pCPA negotiations and
government drug plan listing decisions
• Enhanced health technology assessment review
• Consideration of managed access agreements
• Enhanced use of real-world evidence
• Adjudication process to address individual patient
coverage requests by expert panels
27. Encouraging progress for rare disease
community
…but questions remain to be explored – for example:
• How will health technology assessments be better equipped to
evaluate rare disease drugs?
• What will change for clinicians? (e.g., adjudication / exceptional
access / input into process)
• How will the process ensure that the right experts are providing
advice?
• How can patients and clinicians participate in real world evidence
programs?
• How will patients know when & how they can expect a decision on a
specific rare disease treatment?