Marcia Reinhart DPhil CMPP Principal Tantalus Medical Communications Many definitions H ealth T echnology A ssessments Technology assessment in health care is a multidisciplinary field of policy analysis It studies the medical social ethical and economic implications of developm ID: 737204
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Slide1
Health Technology Assessments and Systematic Reviews
Marcia Reinhart, DPhil CMPP
Principal, Tantalus Medical CommunicationsSlide2
Many definitions
H
ealth
Technology Assessments
“Technology assessment in health care is a multidisciplinary field of policy analysis. It studies the medical, social, ethical, and economic implications of development, diffusion, and use of health technology.”
– International Association of HTA (INAHTA)
“A form of policy research that examines short- and long-term consequences of technology. . . safety, efficacy, patient-reported outcomes, real-world effectiveness, cost, and cost-effectiveness as well as social, legal, the application of a health-care ethical, and political impacts.”
– International Society for Pharmacoeconomics and Outcomes Research (ISPOR)Slide3
Regardless of the technology assessed, HTAs include similar elementsSlide4
HTAs are used to support many health care decisionsSlide5
Governments/private insurers determine the
price
and
availability
of drugs via formulary designation
What is 'Market Access'?
Figure: Eichler GH
et al. Nat Rev Drug Discov
2010;9:277-91.Slide6
Just as health care systems vary worldwide, the payer assessment process differs from country to country
Market Access around the worldSlide7
National/regional government-based agencies
Independent academic
or consulting
groups
Who
conducts and uses
HTAs?Slide8
HTAs as part of the market access continuum
How do HTAs fit into the decision-making process?
Payer often agrees with HTA agency, although may be influenced by additional factorsSlide9
Cochrane Collaboration definition:“…
attempts to identify, appraise and synthesize all the empirical evidence that
meets pre-specified eligibility criteria to answer a given research question
. Researchers conducting systematic reviews use explicit methods aimed at minimizing bias, in order to produce more reliable findings that can be used to inform decision making
.”
Systematic review – an essential step of the HTA processSlide10
Define research
question
Develop study inclusion/exclusion
criteriaPatientsI
ntervention(s)
C
omparator(s)
OutcomesStudy typeDevelop literature search strategy Typically conducted across
multiple databasesSearch strategies should be broad enough to capture all relevant publications, but narrow enough to avoid excessive irrelevant informationScreen studies for inclusion and collect relevant data
Systematic review: process
PICOSSlide11
Define research
question
Example –
acupuncture for the treatment of fibromyalgiaSlide12
Define research
question
Develop study inclusion/exclusion
criteriaP
atients
Example –
acupuncture for the treatment of fibromyalgia
Age?
Diagnostic criteria?
Comorbidities?
Disease severity?Ethnicity?Slide13
Define research
question
Develop study inclusion/exclusion
criteria
P
atients
I
ntervention(s)Example – acupuncture for the treatment of fibromyalgia
Definition of acupuncture?
Frequency?Adjunct therapies?Slide14
Define research
question
Develop study inclusion/exclusion
criteria
P
atients
I
nterventionComparator(s)
Example – acupuncture for the treatment of fibromyalgiaNo treatment?
Placebo?
Other TCM techniques?Drugs?
Physical therapy?Slide15
Define research
question
Develop study inclusion/exclusion
criteria
P
atients
I
ntervention(s)C
omparator(s)OutcomesExample – acupuncture for the treatment of fibromyalgia
Pain reduction?
Days off work?Physical function?
Quality of life?
Adverse events?
Fatigue?Slide16
Define research
question
Develop study inclusion/exclusion
criteria
P
atients
I
ntervention(s)C
omparator(s)OutcomesS
tudy typeExample – acupuncture for the treatment of fibromyalgia
RCTs?Non-RCTs?
Quasi-randomized?
Economic evaluations?
Real-world evidence?Slide17
Small group exercise – develop PICOS criteriaSlide18
Develop literature search strategy
Use both indexing terms (e.g. MeSH) and plain text terms
Draft searches to identify studies in categories (e.g. disease terms, intervention terms), combined at end
Example – acupuncture for the treatment of fibromyalgia
Disease termsSlide19
Develop literature search strategy
Example – acupuncture for the treatment of fibromyalgia
T
herapy terms
CombinedSlide20
Develop literature search strategy
Example – acupuncture for the treatment of fibromyalgiaSlide21
Screen studies for inclusion
Example – acupuncture for the treatment of fibromyalgiaSlide22
Multiple studies are combined statistically to reveal the overall effect of an intervention
A
summary (pooled)
effect estimate is calculated as a
weighted average
of the
effects
estimated in the individual studiesData extraction and synthesis: The meta-analysis
Individual studiesBox represents estimated effectLines represent 95% CI
Size of box represents weightingSummary intervention effectSlide23
Meta-analysis for mean difference in pain
Example – acupuncture for the treatment of fibromyalgiaSlide24
A bias is a systematic error, or deviation from the truth, in results or inferencesSelection, performance, detection, attrition, reporting
Several tools have been developed to assess bias
Risk of biasSlide25
NICE
single technology appraisal (STA
)
Section A – Decision problemDescription of technology under assessment
Context
Equality
Innovation
Statement of decision problemMay include >1 research question
Role of medical writers in HTAs Slide26
NICE
single technology appraisal (STA
)
Section B – Clinical and cost effectivenessClinical evidence
Cost effectiveness
Section C – Implementation
Assessment of factors relevant to
NHS/other partiesReferences
AppendicesRelated procedures for evidence submissionRole of medical writers in HTAs
Systematic reviewMeta-analysis
Non-RCT evidenceSafety data
Systematic
review
for published analyses
Report and interpret results of own economic model(s)Slide27
Section B – Clinical and cost effectiveness
Clinical evidence
Example – new class of drug to treat rheumatoid arthritisSlide28
Section B – Clinical and cost effectiveness
Clinical evidence
Results from the systematic review:
Phase III clinical trial of new drug (B) vs placebo (A)
Phase II clinical trial of new drug (B) vs placebo (A)
3 RCTs of competitor (C) vs placebo (A)
2 RCTs of competitor (C) vs other competitor (D)
3 single-arm non-RCTsHow do we use this information to compare the new
drug (B) to competitors C and D??Example – new class of drug to treat rheumatoid arthritisSlide29
Network meta-analysis can be used to infer the comparative effectiveness of
treatments of interest that have
not been directly compared in trials
Network meta-analyses/indirect treatment comparisonSlide30
Section B – Clinical and cost effectiveness
Clinical evidence
Safety data
AEs from RCTs and non-RCTs
Post-marketing surveillance data (if available)
Other sources of real-world evidence
Example – new class of drug to treat rheumatoid arthritisSlide31
Section B – Clinical and cost effectiveness
Clinical evidence
Cost effectiveness
Example – new class of drug to treat rheumatoid arthritisSlide32
Cost difference (+)
Cost difference (−)
Effect
difference (+)
Effect
difference (
−
)
Dominated
Dominant
Trade-off
Trade-off
SC
A new treatment can be compared with an existing therapy based on its effect on
cost
and
effect differences
The cost-effectiveness plane
SC = standard careSlide33
In cost-utility
analyses, effectiveness is measured in quality-adjusted life years (QALYs)
QALYs incorporate
both quality and quantity of life gained from an intervention
Key concept: QALYs
Improvement of
quality
of life with new treatment
Estimated using
utility values
Improvement of
quantity
of life with new treatment
QALYs
without
new treatment
(standard care)
Time
QoL
Figure: adapted from McCabe C. Hayward Group Ltd.
2009Slide34
Incremental cost-effectiveness ratio
“costs per outcome” (cost-
effectiveness
analysis) or “costs per QALY” (cost-utility analysis)
Key concept: ICER
Costs
Treatment
– Costs
Standard careQALYsTreatment – QALYs
Standard care
= ICERSlide35
Key concept: ICER
Costs
Treatment
– Costs
Standard care
QALYs
Treatment
– QALYsStandard care
= ICER
Total costs
Life-years gained (LYG)
Utility
for LYG
QALYs
Treatment
$20,000
7
0.5
3.5
Standard Care
$10,000
5
0.6
3.0
Incremental
cost, Treatment vs Std. Care
Incremental
life-years
Incremental
cost/LYG
Incremental
QALYs
ICER
cost/QALY
$10,000
2
$5000/year
0.5
$20,000/
QALYSlide36
Optional group exercise – calculate the ICER of our new arthritis drugSlide37
Economic analysis: “willingness to pay” threshold
Cost difference (+)
Cost difference (−)
Effect
difference (+)
Effect
difference (
−
)
Reject
Accept
WTP threshold
SC
Some
countries/health care systems
use an official or unofficial threshold of acceptable ICERs for new technology assessments Slide38
Example – new class of drug to treat rheumatoid arthritis
Cost difference (+)
Cost difference (−)
Effect
difference (+)
Effect
difference (
−
)
Reject
Accept
WTP threshold
SC
An economic model determines that the ICER for the new drug is $40,000/QALY
If the WTP was $50,000/QALY, this drug would fall into the range of acceptable cost/QALY
XSlide39
Calculating costs per QALY requires estimation of several parametersu
tility values
cost of treatment
duration of treatment/patient lifespan (“time horizon”)Base case = best estimate
of all parameters
Sensitivity analyses test alternative parameter estimates to assess the range of possible results
Cost-utility: sensitivity analysisSlide40
ICER ($thousand/QALY)
Each parameter varied one-at-a-time over plausible range
One-way sensitivity analysis
Figure: Reynolds MR
et al.
Circ-Arrhythmia Elec
2009;2:362–69.
Tornado
diagram
base caseSlide41
Example – new class of drug to treat rheumatoid arthritis
Cost difference (+)
Cost difference (−)
Effect
difference (+)
Effect
difference (
−
)
Reject
Accept
WTP threshold
SC
Sensitivity analysis shows that the new drug may rise above the WTP threshold in certain circumstances
X
X
X
X
X
X
XSlide42
Scenario analysisSimilar to a one-way sensitivity analysis, however, assessments are made by varying multiple parameters at the same time
Example = best-case and worst-case analyses
Probabilistic sensitivity analysis
“Drug X has a 86% probability of falling within the WTP threshold of $50,000/QALY”More statistically complex; assesses distributions of data
for multiple inputs over the course of multiple simulations
Other sensitivity analyses used in HTASlide43
Cost-utility is not the only economic analysis used in HTAs
Cost-consequences analysis
Estimates cost and value of interventions, but leaves it to the reader to draw conclusions
Cost-minimization analysis
Compares input costs, but assumes outcomes are equivalent (e.g., bioequivalent drug comparisons)
Cost-effectiveness analysis
Measures costs in dollars and reports outcomes in natural health units (e.g., mmHg reduction) or ratios (differences in cost/difference in outcomes)
Cost-utility analysis
Measures outcomes based on years of life and quality of life obtained with treatment
Cost-benefit analysis
Enumerates and compares costs and benefits achieved in monetary terms
Budget impact analysis
Estimates effect of intervention on overall cost to organization or health planSlide44
Well-versed in literature searchingExperience with multiple databases and multiple interfaces (e.g. PubMed vs Ovid)
Attention to detail – screening and data extraction
Understanding of evidence grading systems
Knowledge of statistical methods (e.g. meta-analysis, NMA, sensitivity analyses)Understanding of health economic modelsExcellent writing skills
Desirable qualities in HTA medical writersSlide45
Thank you