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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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US share of prescription drug expenditures covered by insurance

US share of prescription drug expenditures covered by insurance

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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