Practical Evaluation of a Pharmacoeconomic Paper
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Transcript of Practical Evaluation of a Pharmacoeconomic Paper
Practical Evaluation of a Pharmacoeconomic Paper
Dr J Miot
Wits University
May 2011
What is “Health”?
World Health Organisation:Health is a “state of complete physical, mental and social well-being”
HEA PTP: M207 Health Economics
Everyone wants it!
Why do we need Health Economics?
“All effective health technologies should be free” Archie Cochrane
But The introduction of new effective technology is
faster than the increase in our ability to pay for them
Uncertainty about both effects and resource use for new technologies
Economic Drivers of Healthcare Costs
• Increasing population• Changing population• Increase in pace and price of new technology• Patient choice and expectation• Economies of scale (pvt vs public)• Tensions between different fund options• Lack of legislative bodies to control price and quality of
new health treatments • Role of country’s economic well being
Its all about choices
Resources are scarce
What we “want” is unlimited
Therefore involves “choice”
How do we choose?
HEA PTP: M207 Health Economics
Unrestricted Access Spending scarce healthcare resources on technologies that
provide little if any benefit or may even harm
Restricted Access Delaying benefits which could be accrued whilst generating
further evidence on effectiveness
Need to explore ways to take into account uncertainty while also offering chance for earlier/increased access to novel treatments
Trueman P. ISPOR SA 3rd Annual Conference 2010
Balancing Uncertainty and Access
Health Economics and Choices
“Assessment of the overall value of a healthcare intervention for the allocation of resources in a given environment”
•Maximise the benefits from available resources
•Provides tools to make consistent decisions
•Provides value for money based on cost-effectiveness and not on the basis of cost alone
Uses of Health Economics Studies
Submission for Re-imbursementSubmission for Regulatory approvalAcademic and EducationalPublication for informationNational GuidancePolicy DeterminationCost-Benefit Analysis
Lets start with an example....
This workshop is giving you a headache! You need treatment for immediate relief.
Your treatment options are:
Which is the most cost-effective option?
Treatment Dose
Paracetamol 1000mg
Ibuprofen 400mg
Tramadol 50mg
Lavender essence 1 drop
Which is the most cost-effective option?What do you look at first?
Clinical Evidence! • Which products have proven clinical benefits?• Are they all the same or are there differences in outcome?• Paracetamol = ibuprofen > tramadol• Now look at the costs• Which is the most cost-effective option?
Sachs C. Oral analgesics for acute non-specific pain. Am Fam Physician 2005;71:913-18.
What are the costs....
The costs for your treatment options are:
Which is the most cost-effective option?
Treatment Dose Cost/dose
Paracetamol 1000mg R 1 (Panado)
Ibuprofen 400mg R 2.54 (Nurofen)
Tramadol 50mg R4.11 (Tramal)
Lavender essence 1 drop R0.02
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The COMPARATIVE analysis of alternate treatments in terms of COSTS and CONSEQUENCES ( can be more than one alternative).
What are the components of Health Economics?
CHOICECHOICE
A
B
Costs A
Costs B
Drug
Comparator
Consequences A
Consequences B
Incremental benefits and costs
Generally going to spend more money so have to ask
“Is the increased benefit worth the increased cost?”
Incremental Cost Effectiveness Ratio = (Costs B – Costs A)
ICER (Effects B- Effects A)
The Cost-effectiveness PlaneMore Costly
Less Costly
More Benefit
Less Benefit
TX is less effective and more costly
TX is more effective and less costly
Upper T
hresh
old
Moderate
WeakModerate
Strong
Weak
StrongUpper
Thr
esho
ldLower Threshold
Lower Threshold
DOMINANT
DOMINANT
Part of a Decision-making Process
Clinical Evidence
Epidemiology
Health Economics
Financial Costs
Patient AccessBudget Impact
Communication and Implementation
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Type of HE Study Design:
• Similar to CUA but the output measure expressed in monetary units.
• Measured in terms of “Willingness to pay”• e.g. cost of diabetic counselling
• Multiple outcomes, different costs• ‘soft’ measures - pain, suffering and disability• ‘hard’ measures - years of reduced life,
restenosis• Combined into a single outcome measure:
Quality Adjusted Life Year (QALY)• e.g. biologics in Rheumatoid Arthritis
Cost Benefit Analysis (CBA)Cost Utility Analysis (CUA)
• Different outcome, different costs• Usually measured in events prevented, lives
saved• e.g. Open vs. laparoscopic surgery
• Same outcome, different costs• e.g. antibiotics, generics• “the cheapest option”
Cost Effectiveness Analysis (CEA)Cost Minimisation Analysis (CMA)
Cost Minimisation Analysis
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Bradley J et al. NEJM, 1991;325(2):87-91
Amoxicillin/Clavulanic acid
Levofloxacin
Acute Sinusitis 1000mg bd 500mg daily
Cure within 21 days 86% 86%
10 day course Augmentin BD Tavanic
Cost per pt
Cost per pt
• Meta-analysis flouroquinolones vs beta-lactam antibiotics in acute bacterial sinusitis. OR 1.09 (0.85-1.39)
• Canadian Medical Association Journal 2008;178(7):845-854
402.09255.62
268.56224.45
Forcid Tavaloxx 500
Cost-minimisation analysisRx 1 Cost Rx 2 Cost Rx 3 Cost
Nasonex 229.50 Beclate Aquanase
65.68 Salex 39.03
Myprodol 95.29 Mybulen 47.19 Betagesic 25.26
Tavanic 402.09 Tavaloxx 268.56 Forcid 224.45
Total
Now add:
GP consult R211.30 – R332.00
And maybe even;
CT Sinus R534 – R3044
MRI Sinus R4474 – R7171
726.88 381.43 288.54
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Type of HE Study Design:
Cost Benefit Analysis (CBA)Cost Utility Analysis (CUA)
• Different outcome, different costs• Usually measured in events prevented, lives
saved• e.g. Open vs. laparoscopic surgery
Cost Effectiveness Analysis (CEA)Cost Minimisation Analysis (CMA)
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Cost-effectiveness Analysis
Intervention Outcomes/100pts Drug Costs/pt
No treatment 15 deaths -
Thrombase 10 deaths R 2000
Klotgon 7 deaths R10 000
What is the ICER? (C1-C2)/(E1-E2)
Cost/Lives saved = (10 000 – 2 000)/ (0.07 – 0.1)
= R266 666/additional deaths prevented
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Type of HE Study Design:
• Multiple outcomes, different costs• ‘soft’ measures - pain, suffering and disability• ‘hard’ measures - years of reduced life,
restenosis• Combined into a single outcome measure:
Quality Adjusted Life Year (QALY)• e.g. biologics in Rheumatoid Arthritis
Cost Benefit Analysis (CBA)Cost Utility Analysis (CUA)
Cost Effectiveness Analysis (CEA)Cost Minimisation Analysis (CMA)
QALYs are generally considered the standard unit of comparison for measuring quality of life outcomes in health economic evaluations
QALYs = time (years) x quality (utilities)
e.g after amputation above the knee – LE is 40 years but utility is 0.875
40 x 0.875 = 35 QALYs
Quality Adjusted Life Years (QALYs)
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Years of Life at Full QualityU
tility
Years of Life
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Loss of years and quality of lifeU
tilit
y Catastrophic illness starts
Years of Life
Reduced Quality of Life
Reduced Years of Life
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Current Treatment A
*Quality Adjusted Life Year
Uti
lity
QALY’s* gained withtreatment A = 3.5
Cost: R200,000
No treatment
Years of Life
Improved Quality of Life
Improved Years
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
0.8
0.9
1
0 1 2 3 4 5 6 7 8 9
Improved Quality of Life
Improved Years of Life
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New Treatment B
*Quality Adjusted Life Year
QALY’s* gained withtreatment B = 3.65
Cost: R290,000
No treatment
Improved Quality of Life
Improved Years of Life
U
tilit
y
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
0.8
0.9
1
Years of Life
0 1 2 3 4 5 6 7 8 9
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Choice of Treatment:
Treatment A = R200,000 per 3.5 QALY’s*
Treatment B = R290,000 per 3.65 QALY’s*
Incremental Cost/QALY* = R600,000/QALY*
Incremental Cost-Effectiveness Ratio
= (290,000-200,000)/(3.65-3.5)
*Quality Adjusted Life Year
QALY’s* gained withtreatment B = 3.65Cost: R290,000
No treatment
Uti
lity
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
0.8
0.9
1
Years of Life
0 1 2 3 4 5 6 7 8 9
QALY’s* gained withtreatment B = 3.65Cost: R290,000
No treatment
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
0.8
0.9
1
Years of Life
0 1 2 3 4 5 6 7 8 9
QALY’s* gained withtreatment A = 3.5Cost: R200,000
No treatment
Years of Life
Improved Quality of Life
Improved Years of Life
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
0.8
0.9
1
0 1 2 3 4 5 6 7 8 9
Uti
lity
QALY’s* gained withtreatment A = 3.5Cost: R200,000
No treatment
Years of Life
Improved Quality of Life
Improved Years of Life
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
0.8
0.9
1
0 1 2 3 4 5 6 7 8 9
Is it a health economics study?
Are both costs and consequences examined?
No No Yes
Examines only consequences
Examines only costs
PARTIAL EVALUATION PARTIAL EVALUATION
Outcome description
Cost description
Cost-outcome description
Yes PARTIAL EVALUATION FULL ECONOMIC EVALUATION
Efficacy or effectiveness evaluation
Cost analysis
Cost -minimisation
Cost-effectiveness
Cost-utility
Cost-benefitCo
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M. Drummond et al, Methods for Economic Evaluation of Health Care Programmes. 2nd Ed. 1997
Steps to Evaluating Health Economics
1. What is the question being answered?
Was a well-defined question posed in answerable formLook in the introduction and methodology for this info
Drummond et al, 1996. Methods for the Economic Evaluation of Health Care Programmes. Chap 3.
2. Are these results useful to me in my setting?
• Only if methodology is appropriate and results are valid
• Not every study will answer every question• Will reveal weaknesses and strengths of the
study
Study Perspective
Societal : all costs and outcomes
Third party payer : public or private
Health care provider : hospital
Benefits manager in private industry : direct medical costs to employees, productivity of employee
Patients : out of pocket expenses, travel and waiting time
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Time horizon
Depends on the intervention and the associated harm or benefit
Days
Weeks
Months
Years
Lifetime
3. What are the Clinical Comparators?
• Are they properly described?• Were any important alternatives omitted?• Was (should) a do-nothing alternative be
considered?• Was an appropriate alternative chosen?
4. Is there evidence of effectiveness?
• Is it from randomized clinical trials, meta-analysis or expert opinion?
• Is it effective in clinical practice?• Were observational data or assumptions used to
establish effectiveness? What about potential biases?• Is the appropriate patient population being evaluated?
If the treatment is not safe or effective – don’t go any further!
5. What are the Clinical Measures?
• Hard endpoints – survival, events, cures• Surrogate markers – BP, TC, Viral load etc
• Are they relevant and appropriate?• How are they presented? OR, RRR, AR etc
6. What costs were measured?
• Depends on Perspective
• Direct medical costs: drug acquisition costs, pharmacy dispensing costs, lab costs, physician visits for monitoring, treatment of side-effects etc
• Indirect cost: decreased productivity, absenteeism, income lost, forgone leisure time, time spent by pt seeking medical services, time spent by family and friends attending the pt
• Intangible cost: psychosocial costs, apprehension, anxiety, grief, loss of well-being, social isolation, family conflict, pain, changes in social functioning and activities of daily living
What costs were measured?
Cost = price x utilisation
• Were the costs measured correctly in appropriate physical units
• What measures were used? Were they appropriate? • What was omitted from measurement – why?• Any special circumstances that make measurement
difficult?
Were the costs and consequences adjusted for differential timing
• Were costs and outcomes occurring in the future “discounted” to present value
• Was there any justification of discount rate used?• Were all cost brought to a fixed time period?
7. What are the Health Economic Measures?
Was an incremental analysis performed?
Incremental Cost/Life year gained (LYG)Incremental Cost/QALYIncremental Cost/event preventedIncremental Cost/procedure
8. How confident are you that this is a valid outcome?
• Was a sensitivity analysis performed?• Was justification provided for the range of values?• Which parameters were sensitive to change and reasons
given for why?• Has a statistical analysis been done?
9. Presentation and Discussion
• Did the presentation and discussion of study results include all issues of concern to users?
• Interpreted intelligently or mechanistically?
• Results compared with other similar studies? What were differences/similarities
• Discussion on generalisability of results to other settings or pt groups?
• Account for other important factors?
• Discuss impact and feasibility of implementation?
• Conflict of Interests?
Pessimist: bottle ½ empty
Optimist: bottle ½ full
Economist: bottle ½ wasted
inefficient!
HEA PTP: M207 Health Economics
What is your perspective?
Lack of direct comparisons to relevant alternative Head-to-head trials often not available Comparator in trials may not be relevant in our setting
Measuring relevant costs and benefits Vary from country to country ( transferability) Value of surrogate endpoints
Lack of long-term follow-up – extrapolation beyond clinical trials Especially important in chronic disease Some modelling is required Decision needed now - can’t wait until long-term data is available
Relevance to local settings – adapting from other settings Local costs and resource utilisation
Reducing uncertainty to improve confidence in outcome Sensitivity analysis Cost-effectiveness acceptability curves plotted from probabilistic models
Challenges in HE Analysis
•Using EBM and Health Economics leads to;
•Better clinical outcomes
•More efficient use of resources
•Reduce over-utilisation
•Reduce perverse incentives
•Improved re-imbursement structures
•Improved training and skills
Challenges are Opportunities
What Levels of Training are needed?
Level of Competency Level of Training
CPD Certificate Fellowship Formal Degree
Awareness;• Industrial field force• Healthcare practitioners• Healthcare administrators•Clinical & marketing industry team•Patient groups•Benefits managers
√√√√√√
Application;• Decision-makers for populations• Applied researchers
√√
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Conceptualise;• Academic/faculty•Senior industry scientists•Senior research consultants
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Education and Skills needed to conduct, interpret and use economic evaluations in healthcare. ISPOR Panel 4. Value in Health, 1999. 2 (2):88-91
Websites and other useful info
ISPOR www.ispor.orgNICE www.nice.org.ukCochrane Databasehttp://www.healtheconomics.com/
Guidelines for authors and peer reviewers of economic submissions to the BMJ. Drummond M and Jefferson TO. BMJ, 1996:313:275-283 - Notes in file
Challenges in systematic reviews of economic analyses. Pignone et al. Ann Intern Med, 2005:142:1073-1079
Methods for Economic Evaluation of Health Care Programmes. Second Edition. Drummond et al. Oxford Medical Publications
Questions?