Submission to the Health Select Committee on the Medicines Amendment Bill
To the Health Select Committee,
This submission is from John Forman, 228 Tinakori Rd, Thorndon, executive director of
NZORD, the New Zealand Organisation for Rare Disorders.
I wish to appear before the committee to present this submission.
I can be contacted at 04 471 2226 or at exec.director@nzord.org.nz
Introduction
NZORD supports the provisions in the Bill aimed at improving the approval processes for
new medicines, and other measures to streamline the prescribing framework and make it
more responsive to innovative practice. We believe many of these measures are long
overdue.
However, we consider that the regulatory approval of medicines and medical devices, and
the process for prescribing of them needs to be seen as just one aspect of the whole
medicines system. These changes need to be seen in that context, and not in isolation. We
are concerned that the Bill and associated commentary does not indicate how all matters
relating to medicines in New Zealand are going to be effectively “joined up” for a
comprehensive solution.
We note in particular the problems that are ongoing in relation to access to specialised
medicines for rare “orphan” diseases, the failure of the medicine strategy to be effectively
implemented to ensure fairness and equity of access for patients in need of such medicines,
and a number of contradictions in government policies relating to the discovery and
development of new medicines, versus access to them by those New Zealand patients who
need them.
Our organisation
NZORD is an umbrella group set up to provide information, support and advocacy for
patients and families affected by rare disorders, and to assist with the development of
support groups for them. Rare diseases affect about 8% of the entire population.
NZORD builds collaborative relationships with health professionals, health planners and
researchers to improve knowledge about rare diseases, improve clinical care, and develop
treatments and cures for rare diseases.
Our network includes over 150 rare disease support groups, and we work very closely with
more than 60 support groups for common disorders on a range of health and disability sector
improvement initiatives.
Commentary
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 referrals across District Health
Board boundaries, and that novel therapies are expensive to develop, resulting in high
treatment costs.
A number of rare disorders now have therapies available that have high unit 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.
The Bill does not change any of the provisions in legislation or policy that is likely to make a
real difference to the significant relative neglect of patients with rare disorders.
In particular there are contradictions between:
 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, negated by other policies that are unwilling to pay the cost of such
products for New Zealand patients in need of specialised medicines.
 Policy frameworks that continue to assess all medicines under the same costeffectiveness 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.
We ask the select committee to note there is a lot of ongoing work required to address
many inequities in health services and medicine access for rare disorders, and to
explore how its report on this Bill, or any other initiatives it might take, can spur action by
government and officials to address these serious outstanding problems.
Yours sincerely,
John Forman
Executive Director