Access to rare medicines
RDNZ rare medicine advocacy position
We will advocate for health entities to provide equitable access to modern rare disorder medicines through a specific assessment pathway including:
- Future-proofing a pathway for new and innovative modern medicine for those with a rare disorder, for example gene therapy.
- A change in Pharmac's statutory objectives to add the objective to secure pharmaceuticals in ways that have regard to the resulting wider societal and economic benefits, allowing the factors considered in cost-benefit analyses to include the wider health system, social support system, and society costs and benefits.
- Funding PHARMAC so that it is able to fund new and innovative modern medicines in a way that is consistent with best international practice.
- Developing and implementing a medicines strategy or similar to include rare disorders, gene therapy and innovative modern medicine.
- Developing and implementing policy that supports timely and equitable access to new, emerging and best available health technologies.
- Ensure people with a rare disorder have equitable access to medicines with demonstrated clinical benefit for a rare disorder.
- The rare voice is routinely heard and appropriately valued and incorporated within the assessment pathway.
- Appropriate clinical expertise is sought and incorporated into the assessment pathway, including utilisation of international experts when there is not a New Zealand clinician who is an expert in the rare disorder.
This aligns with the following points in the Rare Disorders Strategy
- An early goal of the work will be to improve and clarify how and when people with exceptional health needs or circumstances can access tests, advice, medicines, devices and other treatments that are not generally available. They may be highly expensive or require travel to other parts of the country or the world.
- A programme of work across health entities will be concerned with improving the quality and timeliness of decision-making on access to diagnostic, preventive and treatment services, products and infrastructure. This work will include a particular focus on rare disorders.
- We will actively support international efforts to make it easier, faster, safer and more affordable to use new diagnostics, treatments and technologies for rare disorders.
- Over time, this programme of work will consider how to improve aspects of care such as....fairness across different types of investment (such as in medicines, new services, overseas treatments) [and] fairness across different types of need (such as rare or common, prevention or treatment, ongoing or once-only, urgent or non-urgent, providing a cure or relieving symptoms).
- Aotearoa New Zealand’s regulatory stewardship programme will review relevant legislation and regulatory frameworks to ensure they support rare disorders outcomes. Regulation of new technologies, digital capabilities, medicines, devices and other therapeutic products can support safe and timely access to diagnosis and effective treatments. Flexibility will be required for the appropriate level of scrutiny and safety precautions for a range of different rare disorder scenarios. Aligning Aotearoa New Zealand’s regulatory approaches with those of like-minded countries will reduce timeframes for assessment.
Action plan
2024
- Attended 2-day event with Pharmac board and other charities
- Attended 2-day event with Med NZ/PVA
- Pharmac Board chair attended RDNZ round table meeting Nov 2024
- RDNZ quarterly Pharmac meetings
- Met with Todd Stephenson MP
- Talked to Dr Ayesha Verrall MP
Upcoming 2025
- Community MP visit ask
- MP cross-party group on rare disorders
- RTC MP roundtable meeting
- RDS implementation to be a standing item on quarterly Pharmac meetings
- Bid for increased budgetary provision to be made in submission on Budget Policy Statement 2025
- Rare Disorders specific issues to be articulated in Kerry Prendergast’s report to Paula Bennett
