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PHARMAC’s updated guidelines for
cost-effectiveness analyses, with new discount rate
Further to inviting feedback in the Journal last
year,1 PHARMAC has now released its revised
Prescription for Pharmacoeconomic Analysis
(PFPA).2 We are very grateful for the interest
expressed in the document across the health sector (and more widely), and the
quality of input we received. This input has helped us finalise revised
guidelines that we believe both reflects and can enhance international best
practice and will help generate significant benefits for the Health Sector in
New Zealand over the coming years.
The PFPA describes the approach that PHARMAC takes when
undertaking cost-utility analysis (CUA), and also guides pharmaceutical
suppliers when undertaking their own economic analyses to support new funding
applications. This type of analysis provides information on which
pharmaceuticals offer the most health gains from a limited budget (i.e. the
relative cost-effectiveness of a pharmaceutical). Cost-effectiveness is clearly
an important decision factor, although but one of the nine Decision Criteria
that the PHARMAC Board considers when making funding
decisions.3 Knowledge of PHARMAC’s
approach helps understanding of decisions made and provides comfort that a
robust framework is being applied—including that different funding
applications are assessed in a fair and consistent way.
Following the publication of the first version of the PFPA
in 1999, the revised guidelines for CUA (PFPA version 2) have followed an
extensive review that has included expert advice from New Zealand and overseas
and wide consultation. This process has generated considerable interest both
nationally and internationally in the revised document.
Consultation responses were received from a range of
organisations and individuals including clinicians, District Health Boards,
health economists, pharmaceutical suppliers, and other government agencies.
Consultation responses were broadly supportive overall of PHARMAC’s
revised CUA methodology, including the risk-free discount
rate1 and direct patient healthcare
costs.1 Responses were wide-ranging, and
commented on the perspective of analyses, treatment comparators, statistically
non-significant events, measuring quality of life, indirect patient costs, and
generic pharmaceutical prices.
All consultation responses were considered by PHARMAC staff
and the PHARMAC Board, and a number of changes to the document were subsequently
made. Key amendments to the PFPA from the first version include:
- Lowering
the discount rate PHARMAC uses to assess the future value of funding decisions
to 3.5%;1,4-6
- The
inclusion of direct patient healthcare costs;*
- The
use of statistically and/or clinically significant treatment effects;
- The
use of the Graphic Appraisal Tool for Epidemiology (GATE) to critically appraise
clinical trials (www.epiq.co.nz);7
and
- The
inclusion of future generic pharmaceutical
prices.
In addition, version 2 of the PFPA contains
substantially more information on appropriate data sources for deriving relative
clinical efficacy and recommendations for obtaining and assessing clinical data.
The new risk-free discount rate of 3.5% is the most
significant change in the revised PFPA. Compared with the previous risk-adjusted
rate of 8%, the reduced rate in effect means that medicines with high up-front
costs but enduring benefits will be more cost effective than with the higher
discount rate.† The overall impact of the change is likely to be
relatively small, but may affect the relative cost-effectiveness ranking of new
funding proposals. We reiterate that PHARMAC however also takes into account
other factors (including patient need, total cost and government health
priorities) when making funding decisions (i.e. not just
cost-effectiveness).3
Further details of the key amendments to the PFPA, the
content and discussion of the consultation responses, and the revised PFPA
itself (version 2), 2 can be found on the
PHARMAC website at http://pharmac.govt.nz/pharmo_economic.asp
The PFPA provides important insights into the detailed
structure that PHARMAC uses for its economic analyses of pharmaceuticals. We
think that such transparency is important and benefits everyone interested in
the detail on how PHARMAC performs these
analyses.4–6,8–25
While not everyone will agree with PHARMAC’s funding
decisions every time, we hope it is apparent that PHARMAC has an established and
rigorous approach to making the necessary difficult
trade-offs,8,26–38 helped by
well-researched and well-documented policies in the PFPA.
Rachel Grocott Health Economist / Team Leader,
Assessment
Scott Metcalfe Chief Advisor Population Medicine
Footnotes:
* The revised PFPA states that direct patient
healthcare costs in CUAs should be restricted to healthcare costs that
government partially subsidises, and should be based on the cost to government
plus the additional cost to the patient. These costs include general
practitioner visits, pharmaceutical co-payments, and home or continuing
care.
† PHARMAC’s new risk-free (3.5%) discount
rate applies solely to the measurement of costs and benefits in CUAs. It does
not apply to budget impact analyses, which still use the risk-adjusted discount
rate (currently 8%).
References:
- Grocott
R, Moodie P. PHARMAC seeks clinical feedback on its cost-utility analysis
methodology. N Z Med J. 2005 August 4;119(1239). http://www.nzma.org.nz/journal/119-1239/2113/
- PHARMAC.
A prescription for pharmacoeconomic analysis (version 2). May 2007. http://www.pharmac.govt.nz/pdf/PFPAFinal.pdf
- PHARMAC.
Operating policies and procedures of the Pharmaceutical Management Agency.
(“PHARMAC”), 2nd edition. January 2001. http://www.pharmac.govt.nz/pdf/opps.pdf.
Section 2.2 Decision Criteria.
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R. Valuing prevention: discounting health benefits and costs in New Zealand. N Z
Med J. 2005 May 6;118(1214). http://www.nzma.org.nz/journal/118-1214/1443/
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S, Brougham M, Moodie P, Grocott R. PHARMAC responds to Richard Milne on
discounting health benefits and costs. N Z Med J. 2005 Jul 29;118(1219). http://www.nzma.org.nz/journal/118-1219/1601/
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R. Richard Milne responds to PHARMAC on discounting future health benefits and
costs. N Z Med J. 2005 Aug 12;118(1220). http://www.nzma.org.nz/journal/118-1220/1620/
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R, Ameratunga S, Broad J, et al. The GATE frame: critical appraisal with
pictures. Evid Based Med. 2006 Apr;11(2):35-8. http://ebm.bmj.com/cgi/content/full/11/2/35.
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health sector. N Z Med J. 2003;116(1170). http://www.nzma.org.nz/journal/116-1170/362/
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Zealand: government policy 'up in smoke'. N Z Med J. 2005 Jun 3;118(1216). http://www.nzma.org.nz/journal/118-1216/1502/
PMID: 15937536
- Metcalfe
S, Moodie P, McNee W. PHARMAC and tobacco control in New Zealand: two licensed
funded options are already available (with responses by Holt et al and the
Editor). N Z Med J. 2005 Jun 24;118(1217):U1544; author reply U1544; discussion
U1544. http://www.nzma.org.nz/journal/118-1217/1544/
PMID: 15980915
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A. Buproprion, public funding, and smoking cessation. N Z Med J. 2005 Jul
15;118(1218). http://www.nzma.org.nz/journal/118-1218/1576/
- Metcalfe
S, Moodie P. More on PHARMAC and tobacco control in New Zealand N Z Med J. 2006
Jan 27;119(1228). http://www.nzma.org.nz/journal/119-1228/1837/
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J. PHARMAC and long-acting insulin analogues: a poor man's insulin pump--but not
available to the poor man. N Z Med J. 2005 Aug 26;118(1221). http://www.nzma.org.nz/journal/118-1221/1641/
PMID: 16138177
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D. Long-acting inhaled bronchodilators for COPD—lack of logic continues. N
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R, Harrison A. TNF inhibitors for inflammatory arthritis in New Zealand. N Z Med
J. 2005 Oct 28;118(1224). http://www.nzma.org.nz/journal/118-1224/1706/
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A. What's happening in PHARMAC--where do all the submissions go? On the trail of
gemcitabine. N Z Med J. 2005 Nov 11;118(1225). http://www.nzma.org.nz/journal/118-1225/1733/
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K. PHARMAC not funding some treatments for rare, life-threatening diseases:
bosentan as an example. N Z Med J. 2005 Nov 25;118(1226). http://www.nzma.org.nz/journal/118-1226/1759
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D. Evidence, economics, and emotions: the case for temozolomide. N Z Med J. 2005
Dec 16;118(1227). http://www.nzma.org.nz/journal/118-1227/1774/
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N. Antiresorptive agents, raloxifene, and PHARMAC. N Z Med J. 2006 Mar
10;119(1230). http://www.nzma.org.nz/journal/119-1230/1885/
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P, Mulder R, Porter R. PHARMAC and treatment of bipolar depression—the
limits of utilitarianism. N Z Med J. 2006 Mar 31;119(1231). http://www.nzma.org.nz/journal/119-1231/1924/
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H, Kayes N, McPherson K. Interferon beta, PHARMAC, and political directives: in
the best interests of people with multiple sclerosis? N Z Med J. 2006 Apr
21;119(1232). http://www.nzma.org.nz/journal/119-1232/1939
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N. Response to McNaughton and colleagues regarding their
article—interferon beta, PHARMAC, and political directives: in the best
interests of people with multiple sclerosis? N Z Med J. 2006 Jun 2;119(1235). http://www.nzma.org.nz/journal/119-1235/2018/
- Fitzharris
P, Empson M, Ameratunga R, et al. Anaphylaxis management: the essential role of
adrenaline (epinephrine) auto-injectors. Should PHARMAC fund them in New
Zealand? N Z Med J. 2006 May 5;119(1233). http://www.nzma.org.nz/journal/119-1233/1965/
- Carter
J, Clay J. New Zealand cancer patients should have access to erythropoietin
treatment. N Z Med J. 2006 May 19;119(1234):U1989 http://www.nzma.org.nz/journal/119-1234/1989/
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M. PHARMAC and Herceptin for early-stage breast cancer in New Zealand: Herceptin
or deception? N Z Med J. 2006 Jun 2;119(1235). http://www.nzma.org.nz/journal/119-1235/2014/
- Moodie
P, Metcalfe S, McNee W. Response from PHARMAC: difficult choices. N Z Med J.
2003 Mar 14;116(1170). http://www.nzma.org.nz/journal/116-1170/361/
- Metcalfe
S, Evans J, Moodie P. PHARMAC responds on long-acting insulin analogues. N Z Med
J. 2005 Oct 28;118(1224). http://www.nzma.org.nz/journal/118-1224/1716/
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S, Dougherty S. PHARMAC responds on long-acting inhalers for COPD. N Z Med J.
2005 Nov 11;118(1225). http://www.nzma.org.nz/journal/118-1225/1743/
- Grocott
R, Metcalfe S. Going against the flow: the impact of PHARMAC not funding COX-2
inhibitors for chronic arthritis. N Z Med J. 2005 Oct 7;118(1223). http://www.nzma.org.nz/journal/118-1223/1690/
- Metcalfe
S, Moodie P, Grocott R, Wilkinson T. PHARMAC responds on TNF inhibitors for
inflammatory arthritis. N Z Med J. 2005 Dec 16;118(1227). http://www.nzma.org.nz/journal/118-1227/1799/
- Crausaz
S, Metcalfe S. PHARMAC's response on gemcitabine and transparency. N Z Med J.
2005 Nov 11;118(1225). http://www.nzma.org.nz/journal/118-1225/1741/
- Metcalfe
S, Rasiah D, Dougherty S. PHARMAC responds on treatments for pulmonary arterial
hypertension. N Z Med J. 2005 Dec 16;118(1227). http://www.nzma.org.nz/journal/118-1227/1805/
- Metcalfe
S, Crausaz S, Moodie P, McNee W. PHARMAC's response on temozolomide and funding
costly medicines that prolong life shortly. N Z Med J. 2005 Dec 16;118(1227). http://www.nzma.org.nz/journal/118-1227/1806/
- Metcalfe
S, Wilkinson T, Rasiah D. PHARMAC responds on agents to prevent osteoporotic
fractures. N Z Med J. 2006 Mar 10;119(1230). http://www.nzma.org.nz/journal/119-1230/1895/
- Moodie
P, Metcalfe S, Dougherty S. PHARMAC and EpiPen for anaphylaxis. N Z Med J. 2006
Jun 23;119(1236). http://www.nzma.org.nz/journal/119-1236/2038/
- Grocott
R, Metcalfe S, Moodie P. PHARMAC and erythropoietin for cancer patients. N Z Med
J. 2006 Jun 23;119(1236). http://www.nzma.org.nz/journal/119-1236/2039/
- Metcalfe
S, Moodie P. PHARMAC and statins--correction is needed. N Z Med J. 2007 Mar
2;120(1250). http://www.nzma.org.nz/journal/120-1250/2453/
- Metcalfe
S, Evans J, Priest G. PHARMAC funding of 9-week concurrent trastuzumab
(Herceptin) for HER2-positive early breast cancer. N Z Med J. 2007 Jun
15;120(1256). http://www.nzma.org.nz/journal/120-1256/2593/
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