NZORD’s submission on Pharmac’s NPPA review

29 May 2014
Thank you for the opportunity to submit on this important policy. First we refer you back to our submissions and commentary on the 2011 final version of the NPPA policy which can be found at this link. We think the weaknesses and contradictions that we identified in the policy back then are still valid concerns today, and we ask that you assess the key points made back then as relevant to this current review.
The essential summary of our current views of the NPPA scheme is:
Positives -
* We note that it has produced some intended outcomes such as shifting some medicines from NPPA consideration to the schedule. That is a positive step.
* We understand from informal conversations with a limited number of clinicians that there seems to be an improvement in access to off-label use of medicines, or where special authority requirements might initially exclude some patients accessing the medicines they need. These steps, if more widely applicable, are also positive and are supported.
* We also note with pleasure the now standard access to enzyme replacement therapies for those for whom a transplant procedure is planned. We think this access should be formalised on the schedule, subject to appropriate eligibility criteria.
* We are also aware of what seems to be improved access to some very specialised medicines for rare inborn errors of metabolism, but we note that success seems to be limited to those usually less than $100,000 per patient per annum.
Negatives -
* Clearly the policy has failed, and continues to fail, in relation to access for specialised medicines for rare disorders where the per annum cost is high and where ongoing treatment is the standard of care for those diseases. This is particularly concerning when the original brief from Ministers included an intention to improve such access.
* The NPPA policy has a number of artificial constraints built into it, including the pre-requisites which we believe are unnecessarily restrictive. Pharmac has a general discretion to consider applications that do not meet the pre-requisites and they should make this clear and explicit in their policy, thus shifting the “pre-requisites” to a lower level status of “guidance points”.
* Another artificial constraint is the exclusion of consideration under NPPA where a medicine has already been considered for possible schedule listing, but not yet listed. Pharmac’s rationale about this is understood, in relation to your stated wish to “protect the schedule”, but we believe this is a contrived rationale which is not an obligatory conclusion about how exceptional circumstances could operate appropriately. Allowing such consideration would not necessarily undermine the schedule process, though it could possibly modify it at times. Pharmac’s fear of such an outcomes does not seem adequate justification for unreasonably excluding such consideration.
* Also artificially contrived is your emphasis in the NPPA policy on individuals only, when it is widely acknowledged that tiny groups also have particular issues that need particular consideration. In fact your intention to do an RFP later this year for medicines for rare disorders, is evidence that you see the need for additional consideration for those in this sort of exceptional circumstance, even though your proposed decision criteria recently consulted on, suggests there is no rationale for this. These contradictions are glaring.
* We restate here our frequently made criticism of Pharmac policies that do not explicitly acknowledge and incorporate the human rights dimension into decision making, nor accept that Pharmac holds more than technical Pharmacoeconomic and budget management responsibilities, such as moral duties, ethical responsibilities, and duties to achieve equity and reduction in disparities in health outcomes.
Other points -
* Your recent decision criteria document went to great lengths to exclude patients as defined by disease, from being considered a population, yet your NPPA policy document refers to patients by disease as populations in a number of places. We believe you have been cynically manipulative of language and logic in order to reach a conclusion that is convenient to you, rather than one that is fair, reasonable and accurate.
* We are acutely aware of the legal provisions regarding Pharmac which make it almost completely autonomous and practically barely accountable to anyone else, including DHBs or Ministers. In these circumstances we believe it is incumbent on Pharmac to regularly undertake serious self-examination of its policy directions in the light of many criticisms frequently levelled at you. External independent advice should also be taken to test the validity of many of the policy points you adopt but which are criticised by an increasing number of commentators as policy points that are suspect in their logic or appropriateness.
* We consider that an inevitable consequence of the path that Pharmac has chosen over recent years, including the intransigence it has shown in relation to moral factors such as equity, fairness and community values, is the emergence of a scenario that is as frightening and inappropriate as the late 19th and early 20th century situations when those with almost unbridled power made decisions in health and related domains that were focussed on their own narrow interests and perspectives and seriously devoid of moral justification. There were endless scandals and reviews into medical and scientific abuse of patient interests. Strong moral and ethical guidance was put in place in regulations and professional ethics to prevent this occurring. We see the policy directions and behaviours of Pharmac as moving parallel to those abusive and unethical situations, where Pharmac’s obsession is with pharmacoeconomic analysis and budget management, to the exclusion of morality.
John Forman
Executive Director