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Marcia Reinhart, DPhil CMPP
Principal, Tantalus Medical Communications
Health Technology Assessments
• Many definitions
“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)
Regardless of the technology assessed,
HTAs include similar elements
Clinical
efficacy
HTA
Safety
Costeffectiveness
HTAs are used to support many
health care decisions
Clinicians and
patients
Public and
private payers
• Prescribing
decisions
• Practice
guidelines
• Drug plan
formularies
• Level of
coverage
Hospitals
• Technology
acquisition
• Hospital
formularies
What is 'Market Access'?
• Governments/private insurers determine the price
and availability of drugs via formulary designation
Figure: Eichler GH et al. Nat Rev Drug Discov 2010;9:277-91.
Market Access around the world
• Just as health care systems vary worldwide, the payer
assessment process differs from country to country
Who conducts and uses HTAs?
• National/regional government-based agencies
• Independent academic or consulting groups
How do HTAs fit into the decisionmaking process?
• HTAs as part of the market access continuum
Manufacturer
submission
• Conducts literature
review, economic
analysis, etc.
• Submits HTA-like
document based
on agency template
HTA agency
report
• Assesses
manufacturer
submission
• May conduct
independent review
or rely on HTAs
from 3rd parties
• Submits
recommendation to
payer




Payer
• Makes final
decision on
reimbursement and
availability of drug
or device
Payer often agrees with
HTA agency, although
may be influenced by
additional factors




Systematic review – an essential step
of the HTA process
• Cochrane Collaboration definition:
“… attempts to identify, appraise and synthesize
all the empirical evidence that meets prespecified 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: process
Define research question
Develop study inclusion/exclusion criteria
1.
2.





Patients
Intervention(s)
Comparator(s)
Outcomes
Study type
PICOS
Develop literature search strategy
3.

Typically conducted across multiple databases
 Search strategies should be broad enough to capture all
relevant publications, but narrow enough to avoid excessive
irrelevant information
4.
Screen studies for inclusion and collect relevant data
Example – acupuncture for the
treatment of fibromyalgia
1.
Define research question
Example – acupuncture for the
treatment of fibromyalgia
Define research question
2. Develop study inclusion/exclusion criteria
1.

Patients
Diagnostic
criteria?
Age?
Disease
severity?
Comorbidities?
Ethnicity?
Example – acupuncture for the
treatment of fibromyalgia
Define research question
2. Develop study inclusion/exclusion criteria
1.

Patients
 Intervention(s)
Frequency?
Definition of
acupuncture?
Adjunct
therapies?
Example – acupuncture for the
treatment of fibromyalgia
Define research question
2. Develop study inclusion/exclusion criteria
1.

Patients
 Intervention
 Comparator(s)
Placebo?
No treatment?
Other TCM
techniques?
Drugs?
Physical
therapy?
Example – acupuncture for the
treatment of fibromyalgia
Define research question
2. Develop study inclusion/exclusion criteria
1.

Patients
 Intervention(s)
 Comparator(s)
 Outcomes
Pain reduction?
Quality of life?
Adverse events?
Physical
function?
Days off work?
Fatigue?
Example – acupuncture for the
treatment of fibromyalgia
Define research question
2. Develop study inclusion/exclusion criteria
1.





Patients
Intervention(s)
Comparator(s)
Outcomes
Study type
RCTs?
Non-RCTs?
Economic
evaluations?
Real-world
evidence?
Quasirandomized?
Example – acupuncture for the
treatment of fibromyalgia
Develop literature search strategy
3.


Disease
terms
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
3.
Develop literature search strategy
Combined
Therapy
terms
Example – acupuncture for the
treatment of fibromyalgia
3.
Develop literature search strategy
Medline
Cochrane
Embase
congress
abstracts
Other
sources?
ALL COMBINED
(duplicates removed)
Example – acupuncture for the
treatment of fibromyalgia
4. Screen studies for inclusion
Data extraction and synthesis: The
meta-analysis
• 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 studies
Individual studies
• Box represents estimated effect
• Lines represent 95% CI
• Size of box represents weighting
Summary intervention effect
Example – acupuncture for the
treatment of fibromyalgia
•
Meta-analysis for mean difference in pain
Risk of bias
• A bias is a systematic error, or deviation from the
truth, in results or inferences

Selection, performance, detection, attrition, reporting
• Several tools have been developed to assess bias
Role of medical writers in HTAs
• NICE single technology appraisal (STA)
Section A – Decision problem
1. Description of technology under assessment
2. Context
3. Equality
4. Innovation
5. Statement of decision problem

May include >1 research question
Role of medical writers in HTAs
• NICE single technology appraisal (STA)
Section B – Clinical and cost effectiveness
6. Clinical evidence
7. Cost effectiveness
Section C – Implementation
8. Assessment of factors relevant to
•
NHS/other parties
•
9. References
10. Appendices
11. Related procedures for evidence
submission
•
•
•
•
Systematic review
Meta-analysis
Non-RCT evidence
Safety data
Systematic review for
published analyses
Report and interpret
results of own economic
model(s)
Example – new class of drug to treat
rheumatoid arthritis
Section B – Clinical and cost effectiveness
6. Clinical evidence
Example – new class of drug to treat
rheumatoid arthritis
Section B – Clinical and cost effectiveness
6. 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-RCTs
How do we use this information to compare the new
drug (B) to competitors C and D??
Network meta-analyses/indirect
treatment comparison
• Network meta-analysis can be used to infer the
comparative effectiveness of treatments of interest
that have not been directly compared in trials
Example – new class of drug to treat
rheumatoid arthritis
Section B – Clinical and cost effectiveness
6. 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 arthritis
Section B – Clinical and cost effectiveness
6. Clinical evidence
7. Cost effectiveness
The cost-effectiveness plane
• A new treatment can be compared with an existing
therapy based on its effect on cost and effect
differences
Cost difference (+)
Dominated
Effect
difference
(−)
Trade-off
SC
Trade-off
Dominant
Cost difference (−)
Effect
difference
(+)
SC = standard care
Key concept: QALYs
• 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
QoL
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
Figure: adapted from McCabe C. Hayward Group Ltd. 2009
Key concept: ICER
• Incremental cost-effectiveness ratio
 “costs per outcome” (cost-effectiveness analysis) or
“costs per QALY” (cost-utility analysis)
CostsTreatment – CostsStandard care
QALYsTreatment – QALYsStandard care
= ICER
Key concept: ICER
CostsTreatment – CostsStandard care
QALYsTreatment – QALYsStandard care
Total costs
Treatment
Standard Care
Incremental
cost, Treatment vs
Std. Care
$10,000
$20,000
$10,000
Life-years
gained (LYG)
7
5
= ICER
Utility for LYG
QALYs
0.5
0.6
3.5
3.0
Incremental
life-years
Incremental
cost/LYG
Incremental
QALYs
ICER
cost/QALY
2
$5000/year
0.5
$20,000/
QALY
Economic analysis: “willingness to
pay” threshold
• Some countries/health care systems use an
official or unofficial threshold of acceptable ICERs
for new technology assessments
Cost difference (+)
WTP threshold
Reject
Effect
difference
(−)
SC
Accept
Cost difference (−)
Effect
difference
(+)
Example – new class of drug to treat
rheumatoid arthritis
• An economic model determines that the ICER for the
new drug is $40,000/QALY
• If the WTP was $50,000/QALY, this drug would fall into
the range of acceptable cost/QALY
Cost difference (+)
X
Reject
Effect
difference
(−)
WTP threshold
SC
Accept
Cost difference (−)
Effect
difference
(+)
Cost-utility: sensitivity analysis
• Calculating costs per QALY requires estimation of
several parameters



utility values
cost of treatment
duration of treatment/patient lifespan (“time horizon”)
• Base case = best estimate of all parameters
• Sensitivity analyses test alternative parameter
estimates to assess the range of possible results
One-way sensitivity analysis
• Each parameter varied one-at-a-time over
plausible range
base case
Tornado
diagram
ICER ($thousand/QALY)
Figure: Reynolds MR et al. Circ-Arrhythmia Elec 2009;2:362–69.
Example – new class of drug to treat
rheumatoid arthritis
• Sensitivity analysis shows that the new drug may
rise above the WTP threshold in certain
circumstances
Cost difference (+)
X
Reject
Effect
difference
(−)
X
X XX
X X
SC
Accept
Cost difference (−)
WTP threshold
Effect
difference
(+)
Other sensitivity analyses used in
HTA
• Scenario analysis
 Similar 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
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
plan
Desirable qualities in HTA medical
writers
• Well-versed in literature searching
 Experience 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 models
• Excellent writing skills