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The New Zealand Medical Journal

 Journal of the New Zealand Medical Association, 20-July-2007, Vol 120 No 1258

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
PHARMAC
Wellington
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:
  1. 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/
  2. PHARMAC. A prescription for pharmacoeconomic analysis (version 2). May 2007. http://www.pharmac.govt.nz/pdf/PFPAFinal.pdf
  3. 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.
  4. Milne 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/
  5. Metcalfe 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/
  6. Milne 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/
  7. Jackson 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.
  8. Metcalfe S, Dougherty S, Brougham M, Moodie P. PHARMAC measures savings elsewhere to the health sector. N Z Med J. 2003;116(1170). http://www.nzma.org.nz/journal/116-1170/362/
  9. Holt S, Harwood M, Aldington S, Beasley R. PHARMAC and tobacco control in New 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
  10. 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
  11. Bloomfield 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/
  12. 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/
  13. Krebs 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
  14. Jones D. Long-acting inhaled bronchodilators for COPD—lack of logic continues. N Z Med J. 2005 Sep 16;118(1222). http://www.nzma.org.nz/journal/118-1222/1669/
  15. Grainger 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/
  16. Simpson 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/
  17. Whyte 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
  18. Hamilton 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/
  19. Gilchrist N. Antiresorptive agents, raloxifene, and PHARMAC. N Z Med J. 2006 Mar 10;119(1230). http://www.nzma.org.nz/journal/119-1230/1885/
  20. Ellis 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/
  21. McNaughton 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
  22. Rawson 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/
  23. 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/
  24. 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/
  25. Rosevear 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/
  26. 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/
  27. 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/
  28. Metcalfe 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/
  29. 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/
  30. 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/
  31. 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/
  32. 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/
  33. 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/
  34. 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/
  35. 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/
  36. 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/
  37. 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/
  38. 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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