Journal of the New Zealand Medical Association, 16-December-2005, Vol 118 No 1227
PHARMAC responds on treatments for pulmonary arterial hypertension
Dr Ken Whyte recently wrote about the funding of medicines for rare life-threatening diseases (high-cost treatments for ‘orphan diseases’), using the example of bosentan for pulmonary arterial hypertension (PAH) (http://www.nzma.org.nz/journal/118-1226/1759).1 He raises difficult issues that need discussion.
Subsidised access to high-cost treatments for PAH (such as iloprost, bosentan and high-dose sildenafil2) had since 2001 been initially provided under the Community Exceptional Circumstances (CEC) scheme (http://www.pharmac.govt.nz/exceptional_circumstances.asp). Over that time applications were relatively rare, no more than a few per year.
However, the CEC scheme requires rarity, i.e. that the prevalence of a condition is limited to no more than 10 cases nationally. During 2004 it became apparent that the rarity limit would be significantly exceeded (28 patients are now funded for high-cost PAH treatments, many on sildenafil2). Under the limits of the CEC scheme, PHARMAC was no longer able to approve applications for high-cost PAH treatments under CEC. PHARMAC therefore moved to find a permanent solution to the funding of PAH treatments.
The Pharmacology and Therapeutics Advisory Committee (PTAC) has noted a lack of information and a number of dilemmas with the management of PAH, and made a high priority recommendation that funding issues be resolved as soon as possible. PHARMAC is actively working on this. The relevant portions of the minutes of the two relevant PTAC meetings can be found in the Appendix to this letter.
Until a permanent solution is found, applications for new patients can still be made for subsidised treatment through the Hospital Exceptional Circumstances (HEC) scheme. Unlike CEC, HEC does not have a rarity criterion, but requires that treatment is cost-saving to the District Health Board (DHB). Since mid-2004, 19 patients have received approval for the use of high-cost PAH treatments under the HEC scheme, with more applications being received and approved every week.
Treatments for PAH are expensive, and annual treatment costs for each patient vary substantially between medications, with $90-180,000 for iloprost, $56,000 for bosentan and $20-30,000 for sildenafil. Current DHB expenditure on high-cost PAH treatments is some $600-700,000 per year, and the number of patients seeking treatment continues to grow.
The evidence for bosentan, iloprost and sildenafil also continues to grow. The recently published SUPER trial,3 referred to by Dr Whyte, and the SERAPH study4 indicate that sildenafil may be as effective as the more expensive bosentan.5 Neither high-dose sildenafil nor nebulised iloprost is registered in New Zealand for use in PAH.
In response to Dr Whyte’s comments about the cost-effectiveness of PAH treatments, we find it difficult to comment on the one published economic analysis on PAH (Highland et al 2003)6 that we7 and he can locate, which did have important limitations.8 Economic analyses for individual PHARMAC CEC funding decisions have indicated that all three treatment options may be cost-effective as a bridge to transplantation, but perhaps the only cost-effective maintenance treatment for patients ineligible for transplant is sildenafil. Funding a medicine in Australia does not necessarily mean convincing cost-effectiveness—for instance, Australia continued to fund COX-2 inhibitors despite dubious cost-effectiveness.9
In general terms, PHARMAC’s prioritisation process tries to allocate scarce resources in a fair way.10 There are very expensive treatments that may offer significant benefits to a small number of people. Such very expensive treatments have to compete for limited funds with less expensive medicines that treat large numbers and achieve greater population health gains11 for the same total costs. The growing number of costly new treatments makes such decisions both more common and more difficult.
PHARMAC is currently reviewing its decision-making process for high-cost medicines—driven in part by having to turn down treatments for small numbers of people who then miss out because there are no alternative treatments. This is where, even after assuming 100% effectiveness with large clinical benefits, the cost of these medicines is very high (at times $250,000 per patient year or more). Funding them would deny treating too many people with other diseases.
PHARMAC’s Board intends to consider the outcome of this review process next year; prior to that, any proposed changes to our decision-making processes would undergo public consultation.
That said, PHARMAC is actively working to permanently solve the funding of PAH treatments, independent of the high-cost review process.
Public Health Physician
Acting Medical Director
Therapeutic Group Manager
Conflict of interest: Scott Metcalfe is externally contracted to work with PHARMAC for public health advice. Dilky Rasiah and Sean Dougherty declare no conflicts.
References and endnotes:
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