A systematic review and economic model of the effectiveness and ...
A systematic review and economic model of the effectiveness and ...
A systematic review and economic model of the effectiveness and ...
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only level <strong>of</strong> social disability that IHRQoL<br />
includes is slight social disability. This is an <strong>of</strong>tcited<br />
criticism <strong>of</strong> generic indexes <strong>of</strong> HRQoL: that<br />
<strong>the</strong>ir content validity may be weak when applied<br />
in specific disease areas. In practice, <strong>the</strong> IHRQoL<br />
is rarely used today <strong>and</strong> values <strong>of</strong> health states<br />
were gained from a small sample <strong>of</strong> individuals<br />
whose preferences are unlikely to be representative<br />
<strong>of</strong> <strong>the</strong> population <strong>of</strong> Engl<strong>and</strong> <strong>and</strong> Wales as a<br />
whole. The authors undertook multi-way<br />
sensitivity analyses in order to explore <strong>the</strong> effect<br />
that <strong>model</strong>ling plausible variations in quality <strong>of</strong><br />
life improvements pre- <strong>and</strong> post-treatment had on<br />
cost–utility estimates. Overall, <strong>the</strong> effect <strong>of</strong><br />
<strong>model</strong>ling different disability/distress levels had<br />
little impact on <strong>the</strong> estimates. The authors did not<br />
report on any uncertainty associated with <strong>the</strong><br />
QALY estimates.<br />
An alternative approach to employing a generic<br />
preference-based health index <strong>of</strong> HRQoL is to<br />
describe health states specific to <strong>the</strong> disease under<br />
consideration, <strong>and</strong> value <strong>the</strong>m directly using<br />
techniques such as SG or TTO. The validity <strong>of</strong><br />
<strong>the</strong>se measures depends on <strong>the</strong> content <strong>and</strong> style<br />
<strong>of</strong> <strong>the</strong> vignette used to describe each health state.<br />
Matza <strong>and</strong> colleagues 125 published an abstract<br />
including utility information based on <strong>the</strong> SG<br />
technique. Utility information was elicited from 43<br />
parents <strong>of</strong> children with ADHD. Eleven<br />
hypo<strong>the</strong>tical health states were developed based<br />
on <strong>the</strong> opinion <strong>of</strong> doctors <strong>and</strong> informed by<br />
published literature <strong>and</strong> unpublished clinical trial<br />
data. Health states that were valued included<br />
untreated ADHD, stimulant treatment <strong>and</strong> nonstimulant<br />
treatment (e.g. ATX). The actual<br />
vignettes used to describe each health state were<br />
not available to us as <strong>the</strong> study is currently<br />
published only as an abstract. The parents rated<br />
each health state including <strong>the</strong> current health state<br />
<strong>of</strong> <strong>the</strong>ir child (mean parent SG rating = 0.74), <strong>and</strong><br />
<strong>the</strong> SG utility scores varied from severe untreated<br />
ADHD (0.48) to effective, tolerable ATX nonstimulant<br />
treatment (0.88). However, <strong>the</strong> full<br />
range <strong>of</strong> utilities for all 11 health states was not<br />
reported in <strong>the</strong> abstract <strong>and</strong> nei<strong>the</strong>r were any data<br />
on <strong>the</strong> variation around each estimate. The<br />
authors stated that comparisons between health<br />
states found expected differences between<br />
untreated mild, moderate <strong>and</strong> severe ADHD<br />
states. In <strong>the</strong> case where stimulant <strong>and</strong> nonstimulant<br />
medication were both effective <strong>and</strong><br />
tolerable, parents preferred <strong>the</strong> latter (ATX)<br />
(p < 0.03).<br />
A potential advantage <strong>of</strong> this approach, <strong>the</strong>refore,<br />
is that small differences in health states can be<br />
© Queen’s Printer <strong>and</strong> Controller <strong>of</strong> HMSO 2006. All rights reserved.<br />
Health Technology Assessment 2006; Vol. 10: No. 23<br />
estimated in terms <strong>of</strong> utility values. Also, with<br />
reference to this <strong>review</strong>, <strong>the</strong> values are based on<br />
direct patient valuations, ra<strong>the</strong>r than expert<br />
judgement. We note that NICE prefers a generic<br />
<strong>and</strong> validated classification system for <strong>the</strong><br />
estimation <strong>of</strong> health state utilities. However, <strong>the</strong><br />
estimated utilities available for ADHD using<br />
generic instruments are crude, <strong>and</strong> based on<br />
expert opinion. Hence values obtained directly<br />
from patients, using SG methodology, may be<br />
more relevant for this <strong>review</strong>.<br />
Cost-<strong>effectiveness</strong><br />
Five relevant <strong>economic</strong> evaluations were found in<br />
<strong>the</strong> published literature, 4,123,126,128,129 including<br />
Lord <strong>and</strong> Paisley, 4 which was part <strong>of</strong> <strong>the</strong> original<br />
NICE appraisal. (Reference 126 is reported in <strong>the</strong><br />
<strong>economic</strong> evaluation section <strong>of</strong> Miller <strong>and</strong><br />
colleagues 30 <strong>and</strong> is also reported in brief in Shukla<br />
<strong>and</strong> Otten 127 .) The last two studies 128,129 are not<br />
formal <strong>economic</strong> evaluations since costs were not<br />
compared with outcomes for <strong>the</strong> interventions<br />
being assessed. However, <strong>the</strong>y do include <strong>the</strong><br />
benefit <strong>of</strong> response to treatment in terms <strong>of</strong><br />
reduced costs <strong>and</strong> <strong>the</strong>refore provide useful data.<br />
Gilmore <strong>and</strong> Milne 123 assessed <strong>the</strong> cost-utility <strong>of</strong><br />
IR-MPH compared with placebo for children<br />
diagnosed using DSM Criteria for Pervasive<br />
ADHD/ADDH 2 or Barkley’s research criteria 130<br />
who are o<strong>the</strong>rwise normal. Gilmore <strong>and</strong> Milne 123<br />
argue that <strong>the</strong>y chose to use placebo-controlled<br />
trials as placebo effects are important.<br />
The NHS healthcare perspective was adopted <strong>and</strong><br />
utility information was determined as described in<br />
<strong>the</strong> QoL section above. In terms <strong>of</strong> resource use,<br />
<strong>the</strong> dosage <strong>of</strong> MPH <strong>and</strong> <strong>the</strong> average number <strong>of</strong><br />
outpatient clinic attendances over <strong>the</strong> year were<br />
estimated based on <strong>the</strong> opinion <strong>of</strong> five child <strong>and</strong><br />
adolescent psychiatrists (personal communication:<br />
it is likely that paediatricians’ case loads have<br />
different population characteristics <strong>and</strong> that this<br />
will have significant implications for <strong>the</strong> number<br />
<strong>of</strong> attendances. It should be acknowledged that<br />
<strong>the</strong> growing development <strong>of</strong> nurse-led ADHD<br />
services will also have <strong>economic</strong> implications). It<br />
was assumed that all follow-up was hospital-based,<br />
that those who terminated treatment or those who<br />
did not respond were treated for 6 weeks, on<br />
average, <strong>and</strong> that those who were included in <strong>the</strong><br />
analysis for 1 year received five outpatient<br />
appointments. Additional information on drug<br />
dosages was obtained from <strong>the</strong> literature, <strong>and</strong> data<br />
on children’s weights were taken from percentile<br />
charts. The cost <strong>of</strong> <strong>the</strong> drugs was obtained from<br />
MIMS (August 1997) 131 <strong>and</strong> <strong>the</strong> cost <strong>of</strong> child <strong>and</strong><br />
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