NZORD – the New Zealand Organisation for Rare Disorders

Questions and Answers on the Ombudsman’s report into a complaint from Freda Evans against Pharmac’s decision to decline funding for treatment of her rare disease.

The New Zealand Organisation for Rare Disorders (NZORD), Lysosomal Diseases New Zealand (LDNZ), Muscular Dystrophy Association of New Zealand (MDANZ), and other patient groups have for many years been urging Pharmac to reconsider its decision-making processes for applications for funding for highly specialised medicines. Patients needing highly specialised medicines are left abandoned by an unfair system which doesn’t take into account issues unique to very rare diseases.

Following a decision in 2012 to decline funding for treatment with Myozyme, a brave patient took her case to the Office of the Ombudsman. The Ombudsman decided to investigate and early in July 2013 published Ombudsman Dr David McGee’s opinion. NZORD has prepared the following Questions and Answers to help you understand that opinion.

Q. What was the Office of the Ombudsman asked to do?

A. Freda Evans, a patient suffering from Pompe disease, asked the Ombudsman to investigate the fairness and reasonableness of decisions made by Pharmac to decline the funding of Myozyme for the treatment of Freda’s condition. Without treatment Freda will continue to deteriorate and may die prematurely. There are no other alternative medicines available to treat Freda’s condition.

Q. Did the Ombudsman decide to investigate Freda’s case?

A. Yes. Also in addition to considering whether Pharmac’s decision-making was unfair or unreasonable in relation to Freda’s decision, Ombudsman Dr David McGee noted more fundamental issues with the way that Pharmac makes decisions under its Named Patient Pharmaceutical Assessment (“NPPA”) Policy. Dr McGee chose to investigate more fully aspects of the NPPA Policy, Pharmac’s decision-making criteria and issues surrounding the availability of funding for medicines to treat patients with rare diseases.

Q. What did Ombudsman McGee say about the question of whetherthe decisions in respect of Freda were unreasonable?

A.Dr McGee ultimately said:

“I do not find matters so clear-cut that one can categorise the actions that have been taken in Ms Evans’ case as “unreasonable” or “contrary to law”, nor do I consider that it would be helpful for me to do so.”

It is notable that Dr McGee was unable to determine with any certainty the unreasonableness or otherwise of the decision. The Office of the Ombudsman has broad powers to request information. Yet even with complete access to Pharmac’s files and after an investigation lasting more than 6 months,he did not find matters “clear cut”. This leaves NZORD wondering what hope patients (who are given a fraction of the information) have of understanding the decisions that affect them!

Also, as discussed below, Dr McGee’s findings indicated shortcomings in Pharmac’s processes and a need for Pharmac to give further consideration to points that NZORD and others have been raising for many years.

Q. Did Ombudsman McGee find fault with Pharmac’s decision making practices?

A. Yes, the opinion notes a number of issues with Pharmac’s processes and he made a number of suggestions which Pharmac was asked to consider seriously.

Q. What issues did the Ombudsman ask Pharmac to consider?

A. The Ombudsman suggested that Pharmac address the following things:

  • Pharmac’s Named Patient Pharmaceutical Assessment (NPPA) Policy does not record the decision criteria to be used for decisions made under that policy. Dr McGee considered the decision criteria under the NPPA Policy ought to be clearly differentiated from those under the Pharmaceutical Schedule.
  • He described Pharmac’s documentation describing the decision criteria as “unduly complicated”. He also noted that Pharmac has a general discretion to consider applications despite the tight eligibility criteria in the NPPA scheme, but noted that the information on the scheme did not explain this.
  • Dr McGee considered that the NPPA Policy needs to provide that prioritisation will not be a pre-requisite if substantial new information of a clinical or commercial nature becomes available. He stressed that “it is reasonable to expect Pharmac to reassess a medicine if new information on [it] has become available”.
  • Pharmac didn’t specifically consider the patient’s suggestion for a trial period of the medicine. The Ombudsman said Pharmac should consider any further comments on this suggestion the patient wishes to make.
  • Pharmac should reconsider the use of its term “prioritisation”. Dr McGee thought this term was unclear and a different term would give a clearer indication of the meaning and consequences of the prioritisation process. To him prioritisation conveyed the impression of some priority (early consideration or importance) for inclusion of the medicine on Pharmac’s schedule, but it appears that all Pharmac means by “prioritisation” is that a medicine has been assessed and a priority assigned to it (either low or high).
  • Dr McGee noted prioritisation has significant consequences for both schedule listings and NPPA applications. Given this, he thought it important that Pharmac shouldn’t make this decision alone and said it was desirable that Pharmac build an external element into the prioritisation decision. He suggested Pharmac could further involve the Pharmacology and Therapeutic Advisory Committee (PTAC) in the prioritisation process.

These comments were not written as formal recommendations under section 22 of the Ombudsman Act but were recorded “for Pharmac’s attention” as Dr David McGee’s view of what he considered “should happen next”. He stated “I am confident that Pharmac as an agency of goodwill will consider my suggestions seriously, indeed Pharmac in its response to my provisional view indicated that it would do so”.

Q. Did the Ombudsman make any other observations that may result in changes to Pharmac’s policies or practices?

A The Ombudsman’s final opinion included other observations NZORD hopes will result in changes to Pharmac’s current policies and practices. In particular:

  • There were a number of comments in the opinion which highlighted shortcomings in Pharmac’s documentation and the need for clearer information in Pharmac’s written reports. For example, the Ombudsman agreed the Government objectives to improve access to highly specialised medicines were relevant under Pharmac’s criterion 8 (Crown objectives) but yet these were not stated in key Pharmac documents. The Ombudsman said “Pharmac’s report on criterion 8 in Ms Evans’ case is fairly brusque. This criterion should not become just a box to tick. If that was the case it should not be there.”
  • In relation to the patient’s arguments that social and ethical factors should be explicitly recognised in the Decision Criteria, Dr McGee’s opinion noted:
  • “There should not be such a high consultation threshold for invoking other factors when reaching decisions under the NPPA Policy as there must be when deciding on Pharmaceutical Schedule listings”.
  • The principle of states not abandoning their citizens is of obvious relevance where there is no known alternative method of treatment.
  • Pharmac’s position is that the values are recognised, at least implicitly, in the legislation. Pharmac does not see the criteria as a means of importing such factors into consideration. The patient and NZORD take a different view on the legislation. The Ombudsman observed that he cannot make the definitive decision on the legal dispute, but that even if the legislative framework does reflect the values urged by Ms Evans that does not preclude them being referred to in policies that Pharmac develops.
  • The lack of evidence in regard to rare diseases and treatments inevitably puts their assessment at a potential disadvantage.

Q. What does the Ombudsman’s opinion illustrate about the effectiveness of Pharmac’s NPPA Policy?

A. NZORD believes the Ombudsman’s opinion highlights that the NPPA is a poor fit for the consideration of funding for medicines for rare diseases. The opinion highlights Pharmac’s views that it cannot target the pre-requisites for individual assessment so as to cover rare diseases. The Ombudsman noted that Pharmac believes “that there is no robust rationale for treating pharmaceuticals for rare diseases differently from other medicines” and that “to attempt a specific recognition for rare diseases in the NPPA Policy would significantly undermine the Pharmaceutical Schedule process”.

NZORD thinks this suggests a significant policy failure, since appropriate consideration of rare diseases was a key policy intent of Pharmac’s development of the NPPA Policy, and the review that resulted in that scheme was presented as having dealt with the issue of improved access to specialised medicines.

We also believe that the Ombudsman’s support for an opportunity for patient views to be submitted and considered as part of the application process, has important implications for all decisions that Pharmac makes. We believe that such an opportunity is a right widely held in all parts of society when public bodies are making decisions, and that the Ombudsman’s noting of a deficiency in Pharmac’s NPPA process has direct implications for all their decisions.

NZORD, LDNZ, patients and academics have suggested to Pharmac that there are inherent inequalities of access that result for patients with rare diseases as a result of the “one size fits all” decision-making criteria applied by Pharmac. Consistent with this, NZORD, the patient and other groups have suggested that Pharmac should consider under its Criterion 9 (“other criteria”) various fairness, equity, social and ethical considerations and values, in order to give effect to the right to health that all New Zealanders have.

The Ombudsman’s opinion highlights that Pharmac does not see criterion 9 as a means of importing social and ethical factors into consideration. Alarmingly the opinion noted that “Pharmac remarked that it had been unable to identify an instance when this criterion [9] had been used. It considered that if such a factor had been accepted this would probably have led to a permanent amendment to the Decision Criteria”.

The Ombudsman’s opinion notes the disagreement in policy between Pharmac and patients, but does not go as far as suggesting that such factors must be taken into account in Pharmac’s decision-making. The opinion does however note “… it would be unreasonable if, in making a decision on an NPPA Policy application, Pharmac would not even consider factors (criteria) suggested to it that fell outside of those identified in criteria 1 to 8 without first engaging in a full round of consultation as if an amendment to the schedule were under consideration”.

Q. Does the Ombudsman’s opinion identify issues relevant to other health stakeholders?

A. Yes. The Ombudsman’s opinion notes issues that the Minister of Health, the Ministry of Health and all District Health Boards should further consider.

In terms of the issues that the Minister and Ministry should consider, the opinion notes some “high policy” issues regarding the factors that should be considered in the context of making decisions about the funding of highly-specialised medicines. The opinion highlights some fundamental disagreements about the extent to which the right to health as embodied in social and ethical goals should be considered in the decision-making process. NZORD intends to discuss the issues raised with the Minister of Health and Ministry of Health.

In terms of the issues that DHBs should consider, DHBs should further consider their ability to purchase medicines not included on the Schedule where exceptional circumstances justify it and also consider what decision-making criteria Pharmac should apply when making purchasing decisions as their purchasing agent. The Ombudsman noted “Pharmac’s function is to maintain a schedule. It is then for DHBs to ensure that they do not act inconsistently with it. Pharmac may legitimately have its own view on what is inconsistent conduct, but I am not convinced that [Pharmac] is authorised to impose that view [on DHBs] by a provision in the schedule”. The Ombudsman noted that he has seen no statutory authority which allows Pharmac to give directions to DHBs, whether through the schedule or otherwise and questioned whether Pharmac is competent to include in the Schedule a provision that limits DHBs ability to directly purchase medicines not on the schedule.

July 2013