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PHARMAC responds on long-acting inhalers for COPD
Dr David Jones has recently written in the Journal ( http://www.nzma.org.nz/journal/118-1222/1669)1
about access to long-acting inhaled bronchodilators for patients with chronic
obstructive pulmonary disease (COPD). We make some observations:
Tiotropium is already available but is under-used
Tiotropium has been funded for patients with severe COPD
(FEV1 < 40% predicted) since February this year. Details of Special Authority
criteria can be found in Appendix 1.
The Pharmacology and Therapeutics Advisory Committee (PTAC)
in August 2004 considered tiotropium to be beneficial2,3 and cost-effective, and
recommended that tiotropium be listed with a high priority ( http://www.pharmac.govt.nz/latest_PTAC_minutes.asp).
For patients with severe COPD, tiotropium appears to be cost
effective compared with other new medicines, with a cost per QALY of $8,400.4
This includes major reductions in hospitalisations for COPD exacerbations
(although there is an overall cost to the health sector from using tiotropium
over ipratropium).
However, patients with severe COPD are not yet getting this
effective treatment. The uptake of tiotropium since the February listing has
been low:
- The
prevalence of COPD—and numbers of patients eligible for tiotropium
treatment—can be difficult to quantify,5,6 with wide-ranging estimates
according to the definitions of COPD used.7 A possible range is between 65,000
and 85,000 patients with COPD,8 with perhaps one-quarter having severe COPD at
the level of FEV1 < 40% predicted.9 Hence there may be 15,000 to 21,000
patients with severe COPD (FEV1 < 40% predicted).
- PHARMAC
had estimated actual usage (numbers of patients using tiotropium) would be 5,900
by June 2005 (28–39% of the 15–21,000 eligible), reaching perhaps
11,200 patients by June 2007 (53–75%).
- However,
HealthPAC data indicate there were 1,690 dispensings for tiotropium in June
2005, being 29% of what had been predicted.
- Currently
(September 2005) there are perhaps 2,200 patients using tiotropium (2,146
dispensings), being 10–14% of eligible patients and 7% of users of both
short-acting anticholinergics and long-acting beta agonists (LABAs)10—see
Figure 1 below:

The uptake of tiotropium in New Zealand has been one-quarter
that of Australia over the comparable time period since listing11—see Appendix 2 for further details.
Extending access to tiotropium for less severe COPD
PTAC has previously considered, at its August 2004 meeting,
the clinical benefits of a commercial proposal that included widening tiotropium
access to FEV1 < 60% predicted. This access was more than had been
recommended by PTAC’s Respiratory subcommittee,12 possibly doubling the
number of users.13 PTAC was unenthusiastic about this proposal, noting a lack of
evidence at the FEV1 40–59% level and commenting on the likely extra
patient numbers.
PTAC has now received a reapplication from the supplier to
extend tiotropium access to FEV1 < 60% predicted. This reapplication has been
placed on the agenda for PTAC’s next quarterly meeting.
Long-acting beta agonists (LABAs)
International guidelines/guidance (COPDX/TSANZ,14 GOLD,15
NICE16) to date do not differentiate between LABAs and long-acting
anticholinergics in the treatment of COPD. However, as Dr Jones suggests,
tiotropium may be better than LABAs for treating COPD,17 with some head-to-head
trials showing significant FEV1 improvements with tiotropium,18–20 others
a trend to tiotropium but no significant differences,21–24 and possible
tolerance to salmeterol.25 Some trials show little improvement with LABAs
compared with placebo in some measures.20,26 This area requires a more
systematic analysis.
PTAC in the past has recommended against funding LABAs for
COPD. PTAC considered an application for eformoterol (as Foradil) for COPD in
August 2001, which it recommended be declined because of the absence of good
evidence of meaningful effects. PTAC considered the evidence for Symbicort for
COPD in May 2004, which PTAC considered to be weak and did not show any clear
benefit, and recommended that this application be declined.
The Cochrane review of LABAs in COPD27 was less than
enthusiastic, concluding “In the few studies that could be included in
this review, treatment of patients with COPD with long acting beta-2 agonists
produces only small increases in FEV1. The improvement in airways function does
not seem to be associated with a consistent effect on other outcomes such as
health related quality of life or reductions in breathlessness.”
Finally, we are aware of at least two RCTs comparing the
LABA eformoterol with ipratropium (a short-acting anticholinergic).28,29 These
trials show no significance between the two drugs—in contrast to the large
effects seen between tiotropium and ipratropium.2 One-third of publicly-funded
LABA use in New Zealand is by patients aged 65 years and over.31–33
Scott Metcalfe Public
Health
Physician Wellington
Sean
Dougherty Therapeutic Group
Manager PHARMAC Wellington
Conflict
of interest: Scott Metcalfe is externally
contracted to work with PHARMAC for public health advice. Sean Dougherty
declares no conflicts.
- Jones
D. Long-acting inhaled bronchodilators for COPD—lack of logic continues. N
Z Med J. 2005;118(1222). URL: http://www.nzma.org.nz/journal/118-1222/1669/
- Vincken
W, van Noord JA, Greefhorst AP, Bantje TA, Kesten S, Korducki L, Cornelissen PJ;
Dutch/Belgian Tiotropium Study Group. Improved health outcomes in patients with
COPD during 1 year's treatment with tiotropium. Eur Respir J. 2002
Feb;19(2):209-16.
- Casaburi
R, Mahler DA, Jones PW, Wanner A, San PG, ZuWallack RL, Menjoge SS, Serby CW,
Witek T Jr. A long-term evaluation of once-daily inhaled tiotropium in chronic
obstructive pulmonary disease. Eur Respir J. 2002 Feb;19(2):217-24.
- Listing
of tiotropium on the Pharmaceutical Schedule for COPD. PHARMAC Technology
Assessment Report No. 59, November 2004.
- COPD
prevalence. Bandolier July 2003. http://www.jr2.ox.ac.uk/bandolier/band113/b113-3.html
- Halbert
RJ, Isonaka S, George D, Iqbal A. Interpreting COPD prevalence estimates: what
is the true burden of disease? Chest. 2003 May;123(5):1684-92.
- Celli
BR, Halbert RJ, Isonaka S, Schau B. Population impact of different definitions
of airway obstruction. Eur Respir J. 2003 Aug;22(2):268-73.
- Based
on various extrapolations from empirical survey data and mathematical models
from both New Zealand and elsewhere, numbers of patients with COPD in New
Zealand appear to ranging anything from 40,000 to 200,000 depending on the
source, but likely numbers might be 66,000 to 84,000 (M Tobias MoH personal
communication; ABDS COPD data at http://www.aihw.gov.au/bod/bod_yld_by_disease/m_respiratory/m3_othresp.xls;
Yan K, Salome CM, Woolcock AJ. Prevalence and nature of bronchial
hyperresponsiveness in subjects with chronic obstructive pulmonary disease. Am
Rev Respir Dis. 1985 Jul;132(1):25-9.; Media release 'Huge Under Diagnosis of
COPD in New Zealand' 15 March 2004 http://www.otago.ac.nz/news/news/2004/15b-03-04_press_release.html).
- Patients
with more severe disease with FEV1 <40% will comprise less than one quarter
of patients with COPD – where in an Australian study, FEV1 <50%
comprised 24% of all patients with COPD (FEV1 <70% predicted without
dr-diagnosed asthma). (PHARMAC analysis of Busselton data 1994/95 from ABDS at
http://www.aihw.gov.au/bod/bod_yld_by_disease/m_respiratory/m3_othresp.xls
– original 1981 study from Yan K, Salome CM, Woolcock AJ. Prevalence and
nature of bronchial hyperresponsiveness in subjects with chronic obstructive
pulmonary disease. Am Rev Respir Dis. 1985 Jul;132(1):25-9)
- 30,691
dispensings of short-acting anticholinergic agents during September 2005, with
31,853 dispensings of LABAs including Symbicort.
- PHARMAC
analysis of: HealthPAC dispensing data; PBS data at http://www.medicareaustralia.gov.au/statistics/dyn_pbs/forms/pbs_tab1.shtml,
using PBS codes (http://www1.health.gov.au/pbs/scripts/listtherlvl1.cfm?sched=GA)
1540C for tiotropium, 8135E, 8279R, 1541D, 1542E, 8238N, 1543F for ipratropium);
population data at http://www.stats.govt.nz/ and http://www.abs.gov.au/Ausstats/
- The
supplier had requested the Special Authority criteria be amended to remove the
requirement for ipratropium and to increase the FEV1 threshold. The proposed
criteria from Boehringer Ingelheim would remove the need for a patient to try
ipratropium initially, and also would allow access to tiotropium to those
patients with less (i.e. moderately) severe COPD (with FEV1s between 40% and 60%
of predicted). This would be in addition to the patients included in the
criteria recommended by the Respiratory Subcommittee of PTAC.
- The
likely impact of extending access criteria on patient numbers is difficult to
quantify, because of a lack of good epidemiological data. However, suggests
numbers of eligible patients could at least double (analysis of Australian
survey data), but how this translates to uptake is not
known.
PHARMAC
staff have only been able to identify epidemiological data from Australia
(Busselton), which was restricted to stratifying FEV1s only as low as < 50%
of predicted. Analysis suggests that of those patients with COPD (FEV1 < 70%
of predicted AND absence of doctor-diagnosed asthma) – who accounted for
6.2% of adults – one quarter had an FEV1 < 50% of predicted (1.5% of
adults), and one half had had an FEV1 < 60% (2.8% of adults – RR
<60%/<50% = 1.92). Although the Busselton data do not extend as low as
FEV1s < 40%, the above data would suggest that, under Boehringer
Ingelheim’s proposed extended criteria, numbers of eligible patients could
at least double.
PHARMAC analysis of
Busselton data 1994/95 from ABDS at http://www.aihw.gov.au/bod/bod_yld_by_disease/m_respiratory/m3_othresp.xls
- original 1981 study from Yan K, Salome CM, Woolcock AJ. Prevalence and nature
of bronchial hyperresponsiveness in subjects with chronic obstructive pulmonary
disease. Am Rev Respir Dis. 1985 Jul;132(1):25-9.
- McKenzie
DK, Frith PA, Burdon JGW, Town GI. The COPDX Plan: Australian and New Zealand
Guidelines for the management of Chronic Obstructive Pulmonary Disease 2003. Med
J Australia 2003 178(6 Suppl 17 Mar): S1-S40. http://www.mja.com.au/public/issues/178_06_170303/tho10508_all.html;
Chronic Obstructive Pulmonary Disease (COPD). Australian and New Zealand
Management Guidelines and the COPD Handbook, http://www.nzgg.org.nz/guidelines/0073/COPDHandbook.pdf
(November 2002)
- Global
Initiative for Chronic Obstructive Lung disease. National Heart, Lung and Blood
Institute and WHO. 2004 Update. http://www.goldcopd.com/
- National
Collaborating Centre for Chronic Conditions. Chronic Obstructive Pulmonary
Disease: Management of adults with Chronic Obstructive Pulmonary Disease in
Primary and Secondary Care. Draft for second stakeholder consultation, November
2003 http://www.nice.org.uk/pdf/COPD_Fullguideline_2ndconsultation.pdf
- PubMed
search keywords tiotropium (salmeterol or eformoterol or formoterol) RCTs, date
30 September 2005
- Briggs
DD Jr, Covelli H, Lapidus R, Bhattycharya S, Kesten S, Cassino C. Improved
daytime spirometric efficacy of tiotropium compared with salmeterol in patients
with COPD. Pulm Pharmacol Ther. 2005;18(6):397-404. Epub 2005 Apr 25.
- van
Noord JA, Aumann JL, Janssens E, Smeets JJ, Verhaert J, Disse B, Mueller A,
Cornelissen PJ. Comparison of tiotropium once daily, formoterol twice daily and
both combined once daily in patients with COPD. Eur Respir J. 2005
Aug;26(2):214-22.
- Donohue
JF, van Noord JA, Bateman ED, Langley SJ, Lee A, Witek TJ Jr, Kesten S, Towse L.
A 6-month, placebo-controlled study comparing lung function and health status
changes in COPD patients treated with tiotropium or salmeterol. Chest. 2002
Jul;122(1):47-55.
- Cazzola
M, Noschese P, Salzillo A, De Giglio C, D'Amato G, Matera MG. Bronchodilator
response to formoterol after regular tiotropium or to tiotropium after regular
formoterol in COPD patients. Respir Med. 2005 May;99(5):524-8. Epub 2004 Nov 26.
- Baloira
Villar A, Vilarino Pombo C. [Bronchodilator efficacy of combined salmeterol and
tiotropium in patients with chronic obstructive pulmonary disease] Arch
Bronconeumol. 2005 Mar;41(3):130-4.
- Cazzola
M, Centanni S, Santus P, Verga M, Mondoni M, di Marco F, Matera MG. The
functional impact of adding salmeterol and tiotropium in patients with stable
COPD. Respir Med. 2004 Dec;98(12):1214-21.
- Cazzola
M, Di Marco F, Santus P, Boveri B, Verga M, Matera MG, Centanni S. The
pharmacodynamic effects of single inhaled doses of formoterol, tiotropium and
their combination in patients with COPD. Pulm Pharmacol Ther. 2004;17(1):35-9.
- Donohue
JF, Menjoge S, Kesten S. Tolerance to bronchodilating effects of salmeterol in
COPD. Respir Med. 2003 Sep;97(9):1014-20.
- Brusasco
V, Hodder R, Miravitlles M, Korducki L, Towse L, Kesten S. Health outcomes
following treatment for six months with once daily tiotropium compared with
twice daily salmeterol in patients with COPD. Thorax. 2003 May;58(5):399-404.
Erratum in: Thorax. 2005 Feb;60(2):105.
- Appleton
S, Poole P, Smith B, Cates C, Veale A, Bara A. Long-acting beta2-agonists for
chronic obstructive pulmonary disease patients with poorly reversible airflow
limitation (Cochrane Review). In: The Cochrane Library, Issue 4, 2004.
Chichester, UK: John Wiley & Sons, Ltd.
- Wadbo
M, Lofdahl CG, Larsson K, Skoogh BE, Tornling G, Arwestrom E, Bengtsson T, Strom
K; Swedish Society of Respiratory Medicine. Effects of formoterol and
ipratropium bromide in COPD: a 3-month placebo-controlled study. Eur Respir J.
2002 Nov;20(5):1138-46.
- Liesker
JJ, Van De Velde V, Meysman M, Vincken W, Wollmer P, Hansson L, Kerstjens HA,
Qvint U, Pauwels RA. Effects of formoterol (Oxis Turbuhaler) and ipratropium on
exercise capacity in patients with COPD. Respir Med. 2002 Aug;96(8):559-66.
- PHARMAC
analysis of one-way encrypted NHI-annotated dispensing claims data from the
PharmHouse data warehouse for inhaled corticosteroids (ICSs), inhaled
long-acting beta-agonists (LABAs) and inhaled/oral short-acting beta-agonist
relievers (SABAs), for the period July 1999 to June 2004. 36% of dispensings for
LABAs (including Symbicort) during 2003/04 were for patients aged 65 years and
over – being 55,339 LABA dispensings in patients aged 65+ during 2003/04
out of 167,695 total LABA dispensings 2003/04.
- The
endorsement criteria for eformoterol 6mcg (Oxis 6) and the Special Authority
criteria for Oxis 12, salmeterol and eformoterol/budesonide (Symbicort) specify
the use of LABAs for the treatment of asthma alone.
- Selected
morbidity data for publicly funded hospitals 2000/01. Wellington: New Zealand
Health Information Service, Ministry of Health, 2004. Table 1, ICD10 codes J45-6
(asthma), codes J40-44 (COPD). 5% of admissions during 2000/01 for asthma were
for patients aged 65+ (392/8557), whereas 71% of COPD admissions were aged 65+
(6198/8675), and any admission for obstructive respiratory disease in a patient
aged 65+ is 16 times more likely to for COPD than asthma
(6198/392).
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