Documents relating to NPPA applications and supply issues

OIA response

Thank you for your request dated 8 June 2025 under the Official Information Act 1982 (the Act) for information relating to Named Patient Pharmaceutical Assessment (NPPA) applications, and supply issues.

Please find a response to each part of your request below.

1. Internal Guidance on NPPA and Exceptional Circumstances Decisions

a. All current internal guidelines, policy documents, standard operating procedures (SOPs), or internal memoranda used to assess Named Patient Pharmaceutical Assessment (NPPA) applications, including where these relate to:

  • Off-label use of a Pharmac-funded medicine under the Exceptional Circumstances Framework;
  • Special Authority waivers for medicines not listed on the Pharmaceutical Schedule;
  • Requests made under compassionate, exceptional, or non-standard clinical grounds.

Pharmac’s Exceptional Circumstances Framework can be found on Pharmac’s website 

Pharmac’s Exceptional Circumstances Framework

Pharmac uses the Exceptional Circumstances Framework to consider whether to fund pharmaceutical treatments for people in exceptional circumstances when those treatments are not currently available for them on the Pharmaceutical Schedule.

Regarding your specific questions, applications can be submitted through the Named Patient Pharmaceutical Assessment (NPPA) process for treatments that do not have Medsafe approval.

A medicine that is not on the Pharmaceutical Schedule does not have Special Authority criteria. Special Authority criteria are created when a medicine is put onto the Schedule to dictate the circumstances in which funded access can be given.

Pharmac does not assess requests for compassionate access. This is a process that is generally engaged in by medicine suppliers. Requests for exceptional clinical scenarios are managed through the NPPA process. There should be some degree of clinical evidence or theoretical plausibility before a NPPA will be approved. We assess this using our Factors for Consideration.

b) Any tools or frameworks used to assess these applications, including but not limited to:

  • Decision templates;
  • Clinical threshold or scoring rubrics;
  • Risk–benefit evaluation matrices;
  • Internal criteria or workflow decision aids.

I refer you again to the Exceptional Circumstances Framework on Pharmac’s website, which contains details on the NPPA principles and questions we consider when assessing a NPPA application. The templates that are used for a Named Patient Pharmaceutical Assessment (NPPA) application are included in Appendix 1 as documents 1 – 5.

You may also be interested in information regarding how Pharmac engages in pharmacoeconomic analysis. 

Prescription for pharmacoeconomic analysis: methods for cost utility analysis

2. Pharmac's Internal Response Protocols for Medicine Shortages

a) Any internal guidance, protocols, policy documents, or communications (including internal emails or briefing notes) outlining how Pharmac approaches funding or substitution decisions where a currently funded and approved medicine is:

  • Temporarily or permanently unavailable (e.g. Pegasys / pegylated interferon alfa-2a), or
  • In short supply nationally or internationally.

Please find an extract from a desk file for Pharmac’s Contract Managers regarding supply issues in Appendix 1 as document 6.

While there is no formalised process documentation for managing supply issues, a generally accepted approach is followed. This process may vary depending on the specific circumstances, as each supply issue is unique. Certain steps may be adapted or omitted based on the urgency and nature of the situation.

The general process followed includes:

  1. Engaging with the supplier to help them resolve the supply issue directly. This may involve support with regulatory processes, permits, or freight solutions to reduce supply risks.
  2. Encouraging the supplier to use their network to source an alternative product, ideally a medicine already registered in New Zealand. Where necessary, suppliers may be asked to work with Medsafe to supply an unregistered brand under exemption.
  3. If no alternatives are available, Pharmac will approach other suppliers, starting with those who have New Zealand registered products. If none are available, we work with suppliers experienced in sourcing medicines from overseas markets.
  4. Seeking clinical advice from the relevant committee, including the implications of a stock-out, potential brand change, suitability of other funded alternatives, and the appropriateness of an unapproved medicine under Section 29 of the Medicines Act.
  5. In cases of global shortages (such as Pegasys), Pharmac utilises information from suppliers on which products being used in other markets as well as advice from clinical committees on suitable alternatives and may include requests for those options alongside the affected medicine.
  6. Once a suitable alternative is identified, we work with the supplier to agree on supply terms, which may involve listing on the Schedule or establishing a short-term contract.
  7. Communicating with stakeholders, including patient advocacy groups, general practitioners, and in some cases pharmacies depending on the medicine and affected patient population.

Further Information regarding Pharmac’s management of supply issues can be found on Pharmac’s website at:

Managing medicine supply