Informing Canadian Rare Drug · health technologies for rare diseases which optimize ... Europe) in...
Transcript of Informing Canadian Rare Drug · health technologies for rare diseases which optimize ... Europe) in...
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I Informing Canadian Rare Drug Policy Through Research
Objectives • To provide an overview of some key PRISM research findings, with a
focus on their contributions to policy making and/or the broader health care system
• approaches to rare disease insurance design • using Citizen’s Juries to understand values in rare disease decision making • interplay between innovation and pricing for rare disease products
• To share stakeholder perspectives on the impact of this research on knowledge, activities, decisions and/or dialogue in the rare disease public policy environment
• To obtain input on areas for future PRISM research
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Panelists Presenters: • Dr. Devidas Menon, Principal Investigator - PRISM, School of Public Health, University
of Alberta • Dr. Tania Stafinski, Health Technology and Policy Unit, School of Public Health,
University of Alberta • Dr. Aidan Hollis, Department of Economics, University of Calgary; President -
Incentives for Global Health
Respondents: • Durhane Wong Reiger, President & CEO, Canadian Organization for Rare Disorders· • Chad Mitchell, Executive Director, Pharmaceutical & Health Benefits, Alberta Health • David Lee, Executive Advisor to the Assistant Deputy Minister, Health Products and
Food Branch, Health Canada
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Agenda
• PRISM overview • PRISM Researchers
• Dev - policy comparisons • Tania - citizen juries and patient engagement • Aidan – pricing, innovation, R&D, etc.
• PRISM Stakeholders • Durhane – patient perspective • Chad – payer perspective • David – federal regulator perspective
• Q&A • Wrap-up comments
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What is PRISM?
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PRISM
• PRISM = “Promoting Rare-Disease Innovations through Sustainable Mechanisms”
• CIHR-funded health services research initiative
• Goal (2012) • “To create a knowledge base for supporting the design of public
policies & processes that enable the development & introduction of health technologies for rare diseases which optimize both outcomes & the use of healthcare resources in Canada.”
• Key principle: engagement of stakeholders to generate knowledge that
supports innovative policies for the development, introduction, and use of technologies for rare diseases
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Knowledge Integration Core/Hub
Key Differentiators
• Involvement of government decision makers from multiple jurisdictions across the country (i.e., provincial and federal governments)
• Engagement of international experts (e.g., UK, US, Europe) in PRISM’s governance structures as well as research activities
• Collaboration with key Partners in PRISM and/or partner-driven initiatives
• Involvement of patient groups and industry stakeholders through standing liaison working groups
• Creation of a network of national and international research collaborators to support PRISM’s research activities
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Rare Disease Policy Assessment
Dev Menon
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PRISM ACTIVITIES RELATED TO POLICY
• Policy landscape
• Policy development
• Pan-Canadian Rare Disease Strategy
IN CANADA • 5 provinces have DRD “programs”
• Separate plan containing specified list of DRDs (Alberta, New Brunswick)
• Separate decision-making process (British Columbia, Ontario, Saskatchewan)
• “Safety-net” programs
• Proportion of CDR-reviewed DRDs (non-oncology)
reimbursed varies from 24% to 73% across jurisdictions
INTERNATIONALLY
Country Reimbursement decision-making mechanisms
DRD specific mechanisms or considerations
Conditions of reimbursement: Conditional listing agreements/Managed Entry Agreements (MEAs)
France • General reimbursement • Case by case (individual or cohort)
None • Frequently used • Common framework for all MEAs based
on payment by results (financial) • Few payment by results (clinical)
Netherlands • General reimbursement
No economic evaluation required
• Frequently used • In past, mainly coverage with evidence
development • Increasing use of payment by results
(clinical)
Denmark • General reimbursement • Case by case (individual or cohort)
None • Rarely used • Not formally a part of reimbursement
system
Italy • General reimbursement • Case by case (individual or cohort)
AIFA 5% Fund • Frequently used • Payment by results (mainly clinical) • Monitoring registries
INTERNATIONALLY Proportion of DRDs with regulatory approval reimbursed by country
Source:
INTERNATIONALLY National price versus mean EU price in countries with greatest number of reimbursed DRDs
Source:
INFORMING PUBLIC POLICY Provincial-Territorial Working Group: PRISM has been supporting the workplan of this group of directors of public pharmaceutical programs. Engagement of policy-makers: PRISM coordinated a meeting of provincial drug plan managers to discuss approaches to providing access to new orphan drugs. Meetings have also taken place between PRISM researchers and senior government officials, including Deputy Ministers at both provincial and federal levels. Rare Disease Strategy: PRISM was a partner with CORD, CIHR, Genome Canada, Innovative Medicines Canada, BIOTECanada, Care4Rare, and the Rare Disease Foundation in developing Canada’s Rare Disease Strategy (issued in May 2015).
Patient and Citizen Engagement in Rare Disease Drug Policy Tania Stafinski
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PATIENT AND CAREGIVER PREFERENCES
What matters to patients and
families?
• Conducted scoping review • Teamed up with CORD • Held focus groups and workshops • Created Patient and Caregiver Liaison Group
Active involvement in policy
development for new therapies
What could active involvement look
like?
• Explored and mapped possible activities onto technology lifecycle – uncertainty matrix
• Prioritized possible activities • Selected one activity to explore further
Preference for activities that address uncertainties around
real world effectiveness
WHERE COULD PATIENTS AND CAREGIVERS PLAY A ROLE?
Step in technology lifecycle
Type of Uncertainty Clinical Benefit Value for Money Affordability Adoption/diffusion
Pre-clinical phase X Clinical phase X X X Regulatory approval X X Real world studies X X X X Reimbursement decision-making processes
X X X X
Introduced as insured service/ use in routine clinical practice
X X
X
X
Obsolescence X X X
MANAGED ACCESS PROGRAMMES
PATIENT AND CAREGIVER-LED DEVELOPMENT OF A MODEL FOR MAPS
•Held workshops across the country •Presented learnings from international experience with
MAPs •Discussed ways of addressing potential challenges
related to the implementation of MAPS in Canada
Workshops
•Combined findings from workshops •Conducted thematic analysis •Organized themes into a checklist
Draft model
Patient and caregiver liaison group meeting
Final model
•Reviewed checklist •Considered factors such as feasibility and relevance
•Sought input from PRISM team members • Prepared final checklist
MODEL = CHECKLIST FOR DECISION-MAKERS
• It isn’t all about numbers
• It is about ensuring that the gain in quality of life is significant
• Views on MAPs are mixed – great idea, but hard to implement • Checklist contains
questions to ask when developing a MAP
• Continued provision of information that reflects the best available evidence to support the group’s work
SYSTEM ORGANIZATIONAL DESIGN • Program goals • Program specific
committee • Ongoing
monitoring and registries
• Disease/drug priorities
• Individual patient input
• Outcome measures and stopping criteria
• International collaboration
Each bullet corresponds to a set of questions for decision-makers to consider
PUBLIC VALUES Will the public
support decisions to fund high cost
therapies for ultra-rare
conditions ?
• Held 2 citizens juries • Convened multi-stakeholder steering committee to guide
jury planning • Selected two groups of ordinary citizens to broadly
represent demographic characteristics
Answer: sometimes
Why citizens juries? Random selection of jury pool:
Members of the jury pool are randomly selected using scientific polling techniques
Representative: Jurors are carefully selected to represent public at large
Informed: Witnesses provide information to jury on key aspects of the issue Jurors engage witnesses in dialogue
Impartial: Witness testimony is carefully balanced to ensure fair treatment to all sides of an issue
Deliberative: Jury deliberates in a variety of formats and is given enough time to ensure all jurors’ opinions are considered
WHAT DID WE DID
• It isn’t all about numbers
• It is about ensuring that the gain in quality of life is significant
• Views on MAPs are mixed – great idea, but hard to implement • Checklist contains
questions to ask when developing a MAP
• Continued provision of information that reflects the best available evidence to support the group’s work
DAY 1 DAY 2 DAY 3 MORNING Both juries
• Presentations from expert witnesses • Expert witness panel
Both juries • Exercise 3: Defending decisions • Exercise 4: Ranking the ‘severity’ of
diseases
AFTERNOON Both juries • Exercise 1: Identifying decision
factors
Jury 1 • Exercise 5:
Trading off health benefits to small and large populations
• Exercise 6: Willingness to pay for incremental benefits
Jury 2 • Exercise 5:
Trading off health benefits when they are uncertain
• Exercise 6: Differentiating between types of uncertainty
Jury 1 • Exercise 2:
Trading off benefits to patients, caregivers, and society
Jury 2 • Presentation on
uncertainty in decision-making
• Exercise 2: Reconsidering choices when benefits are uncertain
Both juries • Wrap-up • Evaluation
EVENING Both juries • Welcome and
introductions • Icebreaker exercise • Presentations - The need for priority- setting in healthcare - Challenges related to rare diseases
WHAT WE FOUND
Analysis of results Insights into public values
Choices made in trade-off exercises
Transcripts
Decision factors
Large group
discussions
Trade-off exercises
It is not just about
prevalence but
prevalence matters
Jury
FACTOR LEVELS
Life expectancy
Less than 1 year 1 to 5 years Greater than 1 year
Pain and discomfort
Extreme Moderate Mild
Ability to function
Severely impaired Moderately impaired Mildly impaired
Depression and anxiety
Severe Moderate Mild
Caregiver burden
High Medium Low
Benefit is a combination of: • Initial health state (without Tx) • Amount of gain (number and magnitude) • Final health state • Prevalence They don’t carry equal weight The small population had to do really well with Tx Jurors had to be okay with the initial state of the large population
Economics of Rare Diseases Aidan Hollis
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Some economics of orphan drugs
Aidan Hollis University of Calgary: Department of Economics and
O’Brien Institute of Public Health Disclosures: • I acknowledge CIHR funding through the PRISM research team • No conflicts
Why is the orphan market booming?
Top 3 answers: • #3: Orphan drug legislation?
• A lot of top sellers are getting orphan designation. • Of the 30 top-selling new drugs approved in Canada 2009-14, 12
had at least one orphan designation in the US.
• #2: Improvements in underlying scientific understanding of some conditions.
• #1: Expansion of insurance enables super-charged pricing.
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SOURCE: Centers for Medicare & Medicaid Services, Office of the Actuary, National Health Statistics Group.
Is the pricing fair and appropriate?
• Insurers do not face the same budget constraints as households, so high pricing becomes a feasible business strategy.
• Some firms are taking advantage of insurance systems. • The obviously unscrupulous players:
– Martin Shkreli – Valeant under Michael Pearson
• Which raised the price of trientine from around $11k per year to around $160k per year with no additional investment
• But everyone else faces the same commercial constraints…
• Roberts, E, M. Herder, and A Hollis, “Fair pricing of ‘old’ orphan drugs: considerations for Canada’s orphan drug policy."
Canadian Medical Association Journal 2015; 187(6):422-425.
Disproportionate pricing hurts almost everyone
• When firms get super-high prices, this is not a ”win” for industry.
• Instead, budgets become tighter for everyone else.
• So if Alexion gets $1m per patient, this leads to less money for other manufacturers.
• Two problems: • Industry needs to organize better to prevent excessive pricing by
some players. • Payers have a responsibility to limit mispricing so that they can afford
to buy the products that do the most good.
Hollis, A., “Sustainable Financing of Innovative Therapies: A Review of Approaches.” PharmacoEconomics, 2016; 34(10):971-80. doi: 10.1007/s40273-016-0416-x
Thinking about cost: an important tool
• Often high orphan drug prices are ”justified” by reference to the small number of patients. • “In explaining the sky-high price of Alexion’s drugs, [John Haslam,
GM of Alexion Canada] says that the company settles on a number after looking at the extreme rarity of the disease the drug treats…”
• If we are to consider that price must be high because the cost per patient of R&D is high, then there is no reason to pay more than the cost of the drug, adjusting for risk and the cost of capital. • In other words – a fair return that doesn’t destroy the budget so
that Alexion’s former CEO can earn $52m.
Why drugs for ‘orphan’ diseases are a booming business with colossal costs for patients - The Globe and Mail 2017-04-10 http://www.courant.com/business/hc-alexion-ceo-compensation-20160401-story.html Fellows, GK, and A. Hollis, “Funding innovation for treatment for rare diseases: adopting a cost-based yardstick approach.” Orphanet Journal of Rare Diseases 2013, 8:180.
Respondent Comments Durhane Wong-Reiger
Chad Mitchell
David Lee
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Questions from Audience
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Wrap-Up
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Wrap-up Comments
• What has been of greatest value in terms of what PRISM has brought to policy discussions so far?
THANK YOU!
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