Economic Evaluation of Drugs Canada’s Experience

							Economic Evaluation of Drugs:
     Canada’s Experience

            Bruce Brady
   Canadian Coordinating Office for
   Health Technology Assessment
             (CCOHTA)

             Sao Paulo
            Nov 22, 2002
               Outline

1.   The drug evaluation process in Canada

2.   Canada’s experience with economic
     evaluation (EE) of drugs

3.   CCOHTA’s role in evaluating drugs

       thoughts for Brazil to consider
    Canadian Health Care System
    Federal system - 13 provinces (states)
     & territories
    Govts of provinces / territories
     responsible for providing health care
    Drugs partly covered & paid by govt
     through public drug plans:
    –   some drugs  listed on formulary
    –   some identified groups (based on high age,
        low income, or special needs)
         Goals of Public Drug Plans

1.   Identify cost-effective drugs
     –    focus on effectiveness in relation to price
     –    economic evaluations (EE) used


2.   Ensure proper prescribing & use of
     these drugs

3.   Ensure availability of these drugs at
     reasonable prices to those in medical &
     financial need
        Drug Review Process (1)
  Question #1: Can we use this drug?
 Federal govt
 Decision to approved drugs for use in
  Canada based on:
    –   safety
    –   efficacy
    –   quality
   Compared with placebo
       Drug Review Process (2)
 Question #2: Should we use this drug?
 Drug plans of provinces & territories
   decide which drugs to pay for & list
 Decide which drugs to pay for based on:
    1. relative clinical effectiveness
    2. relative cost-effectiveness  EE used
    3. other considerations:
        impact on plan budget
        ethical / equity; legal; political
    Compared with appropriate alternative
      Drug Review Process (3)

   Recommendations for listing can be:
      1. No restriction – listed for general use
      2. List with restrictions - use for patients
         meeting predefined clinical criteria
      3. Not to list
      4. No decision pending further deliberation

   Restricting listing – prescribing to sub-group of
    patients for who drug is most cost-effective
 Drug Review Process
                            Approved
                            New Drug

  Expert                                           Expert
Pharmaco-                                          Clinical
Economic                  Drug Review             Reviewers
Reviewers
                           Committee
                                                         External review
                                                       capacity can be used

                       Expert Committee
                       Recommendation




            Decide    Decide       Decide    Decide
            Listing   Listing      Listing   Listing
 Canadian Model of Drug Review
Key features:
1.    Manufacturer submits information to
      committee of independent experts
2.    Information submitted on drug:
         1. pharmacologic & clinical effect
         2. economic:
            – budget impact analysis
            – economic evaluations (EE)
3.    Committee reviews information of companies
4.    Limited additional information & analysis
     – problem of bias in EE
            Strengthening the
         Drug Review Process (1)
1.   Use standard forms & guidelines for
     submission & review of information

2.   Send unsatisfactory submissions back
     to company (Ontario)

3.   Ask company to submit EE model
     –   validate model & test sensitivities
        Strengthening the
     Drug Review Process (2)
4.   Review / re-evaluate listed drugs after
     obtaining more “real-life” evidence
     –   probationary listing??


5.   Seek additional economic analysis
     from independent 3rd parties
     (CCOHTA)
     Economic Evaluations for
        Decision-Making
   Compares costs & benefits of alternatives

   Value for money  to improve efficiency

   Addresses question: “Does the cost justify
    the benefits relative to alternatives?”
    – answer: “Yes”, “No” or “Uncertain”

   Helps decision-makers to make choices
    with limited resources
Economic evaluations
   Other benefits:
    – makes decision-making explicit & transparent
    – a structured way of thinking
   Can be used with other techniques
    – program budgeting marginal analysis
 More useful in some situations than
  others (inexact science)
 A tool in the health technology
  assessment (HTA) toolkit
    Economic Evaluations are Not:

   EE not intended to stifle industry
    innovation or research & development
    – focus on giving incentives (paying for) drugs
      that are clinically effective & cost-effective


   Cost-effectiveness ≠ cost savings
    – few drugs actually save money


   EE ≠ budget impact analysis
    – both are needed
    Budget Impact Analysis vs.
      Economic Evaluation
           Budget impact       Economic
           analysis            evaluations
Question   Is it affordable?   Is it good value for
                               money?
Goal       Cost containment    Efficiency of
                               alternatives
Measure    Total expenditure   Incremental cost
           (P x V)             per unit of benefit
Time       Usually short       Usually longer term
horizon    (1 to 3 years)      (may be lifetime)
     Economic Evaluations do not
      Replace Decision-making
1.   Practical difficulties with EE:
     – flawed studies; lack of “real-life” data;
       poor generalizability
2.   Technical / methodological limitations:
     – no budget impact analysis
     – valuing productivity; analysis partial or static
3.   Political / philosophical considerations:
     – Public participation & preferences
     – Equity issues (group vs individual rights)
     – Political feasibility - legal; interests; values
       Decision-makers need to
       use their best judgment
   Weigh the following:
    – total sum of the evidence in a HTA
      (clinical, as well as economic)
    – resources availability
    – health needs of population
    – population preferences
    – political feasibility
    – their own values
         When to Conduct an
         Economic Evaluation

1.   The amount of resources at stake justify the EE
2.   There are clear alternative uses for the
     resources to be evaluated
3.   The technology underlying each alternative is
     reasonably well understood
4.   A reasonable length of time is available to
     conduct the EE
5.   Decision-makers are receptive to the EE
     results & have not already made up their minds
Survey of Economic Evaluations
    in Decision-Making (1)
   Survey of public drug plan managers in Canada
    in 2002
   Policy for submission of EE:
     – Required:
        • Ontario
        • BC (not generics)
        • Manitoba & Atlantic provinces (except line
          extensions, generics)
     – Recommended / encouraged:
        • Alberta (for NCE) & all other jurisdictions
    Survey of Economic Evaluations
        in Decision-Making (2)
   Use of EE in decision making:

    – Used for all listings:
       • Ontario
       • BC (not for generics)
       • Manitoba (not for line extensions, generics)

    – Used only selectively:
       • All other jurisdictions
    Use of Economic Evaluations
            (continued)
   Lack of information on use by decision
    makers
    – difference between “considered” vs “used”


   Economic considerations generally:
    – follow clinical value
    – of lower priority than budget impact analysis


   May be simplistic (cost-minimization
    analysis)
Use of Economic Evaluations
  Varies by Drug & Place
   More likely to be used if drug has:
    – high budget impact
    – marginal benefit but at high cost
    – innovative
   Less likely to be used if drug has:
    – weak clinical evidence
    – already penetrated market
    – or where other decision criteria are key
      (ethics, politics)
        What is the Problem? (1)

   Silo budgeting
    –    focus on limited outcomes
   Lack of timely information when needed
   Weak dissemination of results
   Lack of economic expertise in some
    places
     What is the problem? (2)

   Deficiencies in conduct & reporting EE:

    – Reliability – inappropriate use of clinical data
      (extrapolation), assumptions, transparency

    – Relevance - comparator, sub-group analysis,
      transferability of results, timing

    – Example: province of BC in 1998 - only 5 of
      21 EE submissions complied with guidelines
Improving Economic Evaluations

   Follow economic guidelines
    –   CCOHTA Economic Guidelines
    –   …..…but it will not eliminate bias

   Provide decision makers with economic
    expertise & training

   Improve communication & dissemination
    of results to decision-makers
Summary: Economic Evaluations
    for Decision -making
1.   EE do not replace decision-making
     ….. BUT there is no ideal method

2.   EE is a useful tool for decision making
     ……BUT it has not yet been fully
     exploited by public drug plans

3.   The time is ripe to use EE to strengthen
     decision-making
     …BUT their quality needs to be improved
     Overview of CCOHTA (1)
   Canada’s national heath technology assessment
    (HTA) agency
   What do we do?
    – assess evidence of clinical effectiveness & cost-
      effectiveness of health technologies (drugs)
   For Whom?
    – decision & policy makers in govt
    – drugs: govt drug plan & hospital formulary
      committees
   Goal:
    – to encourage the appropriate use of clinically
      effective & cost-effective technologies
    Overview of CCOHTA (2)

 Funded by govts & responds to priorities
  set by Ministries of Health
 Operates independently
 In 2002:
   – C$ 4.3 million budget
   – > 40 assessment a year
   – 30 staff
                What is HTA?

   Systematic process of identifying, collecting,
    synthesizing & critically evaluating research &
    evidence about health technologies
   Focus on assessing clinical effectiveness & cost-
    effectiveness of health technologies
   Health technologies are used to deliver health
    care, & include:
     – drugs & vaccines
     – devices, equipment, procedures & systems
        Why is HTA important?
 Decision-makers face difficult decisions:
   – limited budgets, more options, higher costs
 Technology is a major driver of health care costs:
   – technology = 50% to 60% of health care cost
     inflation in Canada
 More options than ever before:
   – 50% of all diagnostics & treatments used
     today did not exit 10 years ago
 Information overload:
   – Over 2 million articles a year to stay informed
             HTA Delivers

   Reliable, impartial, understandable
    information that:
    – makes complex research accessible
    – cuts through information overload to provide
      the relevant information
    – is scientifically rigorous
    – provides the basis for informed decisions on
       “Should we use this drug?”
     What does HTA tell you?

About the technology & its clinical use
Which technologies are clinically
 effective & cost-effective
How it compares with other treatments
For which population group
What it costs
Information & methodological
 deficiencies (transparency)
   Government Awareness of
         HTA’s Value

“The committee agrees that HTA is a
  critical activity & that more HTA
  needs to be undertaken”

       Kirby Commission Report on
         the Health of Canadians
CCOHTA Assessment Process

   Assessment of both the clinical &
    economic aspects of the technology

   Multidisciplinary team

   Rigorous systematic approach used
CCOHTA Assessment Process (1)

 1.   Topic selection
      –   screened, selected & prioritized by
          advisory committees from provinces
      –   ensures that research is based on need &
          relevant to national priorities
 2.   Feasibility work-up (“pre-assessment”)
      –   define research questions
      –   conduct preliminary literature search
      –   outline proposed search protocol,
          methodology & resource needs
CCOHTA Assessment Process (2)

3.   Project team
     –   most projects “in-house”
     –   external clinical experts recruited
     –   some project involve collaborations
4.   Gathering literature
     –   systematic search for relevant documents
     –   comprehensive sweep to many databases
     –   includes “grey” literature, information from
         industry
CCOHTA Assessment Process (3)

 5.    Synthesis & interpretation
      –  systematic review - transparent methods to
         assemble & synthesize literature
      – meta-analysis - use of statistical methods to
         combine clinical findings of studies
      – economic analysis may include:
        • systematic review of economic evidence
        • full economic evaluation
        • budget impact analysis
CCOHTA Assessment Process (4)

6.   Expert review
     –   Guidelines supplied for reviewers
     –   Reviewers with a range of expertise

7.   Publication
     –   Synthesis of relevant information about:
         •   The technology & it clinical use
         •   Whether it is efficacious / effective & for whom
         •   How it compares with other treatments
         •   + / - economic aspects of the technology
         •   Information deficiencies
      Quality Assurance
     throughout Process
                      CCOHTA                       External
                       Staff                      Consultants




Literature search &            Form Project Team
refinement
                               Gathering Literature

  Standard method of            Systematic Review
  analysis & reporting            Meta-analysis
                               Economic evaluation
External experts
including SAP members            Review Cycles

    Editing,                      Final Product
    proofreading
Guidelines for Quality Assurance
   Red process binder:
    –   Guidelines for Authors
        •   report format; pointers on clinical analysis
            & searching literature
    –   Economic Guidelines
    –   National Costing List
    –   Other templates & checklists

   All available on CCOHTA website
CCOHTA’s Economic Guidelines
   To help doers produce standardized, reliable
    economic evaluations to meet needs of users
   Contents:
    1. Design & methodology for economic
       evaluations
    2. Standard reporting format
    3. Quality assurance tips
   2003 revised edition, to reflect:
    – methodological developments
    – experience with use
   Worldwide trend (> 25 Guidelines)
Communication & Dissemination

    Pro-active engagement of decision-makers is
     crucial
    Meet user needs:
    1.  User friendly report format (short, clear, transparent)
    2.  Outreach activities (workshops; education)
       • stakeholder feedback improves HTA output
       • user understanding increases report uptake
    3. Dissemination strategies
    4. Impact assessment
6. Challenges / issues

1.    Multidisciplinary skills required for HTA
2.    HTA must be relevant & timely for
      decision-makers
3.    Difficult balancing rigor & timeliness of
      reports
4.    Reaching decision-makers can be
      difficult
5.    Be wary of contracting out assessments
     – partner or collaborate - more control
7. Improving HTA
1.   Focus on user needs
2.   Encourage consistent, reliable
     assessments through best practice
     –   guidelines, checklists, templates
3.   Collaboration among HTA agencies can
     be useful
     –    produce better & more timely evidence
         (INAHTA & Euroscan)
4.   Commitment to communication &
     dissemination activities needed
“Muito Obrigado”




  www.ccohta.ca