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Charities Commission Registration CC22512

NZORD’s request to the Health select committee – May 2012

Dr Paul Hutchison
Chairperson
Health Select Committee
Parliament Buildings
Wellington

Dear Dr Hutchison,

This letter follows the discussion at the Health select committee on Wednesday 23 May 2012, hearing submissions on the Medicines Amendment Bill and your suggestion to us at the end of our submission that we should write to the committee. This letter outlines the issues we raised and which we consider the committee should ask to be considered by both Pharmac and the Ministry of Health, with their findings reported back to the committee.

Overview:

The most significant problem associated with rare disorders is the generally low priority they attract in health service planning and delivery. Rarity often means limited expertise to manage clinical care needs, difficulties getting appropriate clinical care across District Health Board boundaries, and novel therapies that are expensive to develop. A number of rare disorders now have therapies available that have high per-patient costs because of small patient numbers. This often leads to poor cost effectiveness analysis in a tightly budgeted health system and a reluctance by health administrators to fund these medicines.

However patients with rare disorders have the same rights to healthcare, including the right to fair consideration in access to health services and medicines, as any other patients in New Zealand. There are a number of contradictions in medicine policies that impact on the right of patients with rare diseases to receive equitable access to health care.

Specific contradictions in policies:

• Policy frameworks that provide for open consultation on many aspects of prioritisation in health, yet effectively exclude a point of consultation about the total allocation of medicine funding, because of the closed information system related to medicine funding and procurement.
• Broader government policies that encourage research into high value products for commercialisation are negated by other policies that are unwilling to pay the cost of such products for New Zealand patients in need of specialised medicines. We mentioned to the committee the example of one specialised medicine in development which has potential enhancements over existing therapies as a result of technology commercialised through a New Zealand Crown Research Institute.
• Policy frameworks that continue to assess all medicines under the same cost-effectiveness framework, when such an approach cannot provide equitable access for orphan drugs with high unit costs because of very small patient numbers. We note that the UK and Australia both specifically address the inherent and unacceptable discrimination in such an approach, by providing a separate system that effectively compensates for the disadvantage of rarity.
• A medicine strategy that proposes a fine framework for access, fairness and equity in decisions regarding medicines, contradicted by internal policies of Pharmac that adopt a narrow pharmacoeconomic approach that effectively ignores and negates the strategy’s guidance.
• Policy that sees close involvement by Health Ministers and the Ministry of Health in setting targets and access criteria for a range of health services such as cancer treatment times, vaccination uptake, waiting time in emergency departments, and time for specialist appointments for elective surgery, but contrasted with a completely hands-off approach in relation to access to medicines.
  

Particular problems with recent reviews of policy relating to highly specialised medicines:

These examples relate specifically to enzyme replacement therapies for Lysosomal diseases. The examples are likely to be relevant to other specialised medicines.

• PTAC advice to Pharmac in 2009 that rather than a general listing on the schedule for these therapies, consideration would be better made through the exceptional circumstances system including the use of expert advisors/panels, implying a case by case process of consideration.
• The McCormack Panel report recommended using the same decision processes for all medicines but explicit in their reasoning was their assumption that ethics and community values would be taken into consideration alongside cost-effectiveness assessment, when considering highly specialised medicines.
• When the Medicine Strategy was published with its emphasis on access, fairness and community values (among other things) as guidance in medicine decision making, a concurrent review by Pharmac of their Operational Policies and Procedures removed all reference to ethics and community values from their OPP.
• The review of the exceptional circumstances scheme to develop the Named Patient Pharmaceutical Assessment scheme, was presented as a mechanism by which highly specialised medicines could be considered on a case by case basis, yet the application of the completed NPPA policy means any prior consideration of the therapy for a schedule listing for all patients will exclude any subsequent case by case consideration.
• During the 2011 election campaign the Health Minster insisted that the new NPPA scheme met their 2008 campaign pledge on access to specialised medicines and was also a mechanism available to specific patients with lysosomal diseases, despite our analysis and a Pharmac briefing to us at the time, indicating the contrary. Subsequent written information, and additional oral advice to various people by Pharmac, makes it clear these patients are not eligible for consideration under the NPPA scheme.
• If the Minister has been briefed by Pharmac that their review has dealt with the campaign pledge and provided an opportunity for case by case review for these patients, he has obviously received different advice from Pharmac than they have given us.
  

We ask the select committee to consider the policy process that has been followed throughout the past approximately 10 years regarding highly specialised medicines, to seek a report from Pharmac and Ministry officials on these matters, to consider if the policy framework provides equitable access to medicines for those with rare disorders, and to consider how an equitable access scheme to specialised medicines for rare disease patients could be delivered.

Yours sincerely,

John Forman
Executive Director