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| Journal of the New Zealand Medical Association,
16-December-2005, Vol 118 No 1227
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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.
Treatments for PAHSubsidised 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
Prioritisation of very high cost medicinesIn 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.
Scott Metcalfe
Public Health Physician Wellington Dilky Rasiah
Acting Medical Director PHARMAC Wellington Sean
Dougherty
Therapeutic Group Manager PHARMAC Wellington 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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