OIA response: information regarding Named Patient Pharmaceutical Assessment (NPPA) applications for ustekinumab

29 September 2020

Dear [name and contact details redacted]

REQUEST FOR INFORMATION

Thank you for your request dated 1 September 2020 under the Official Information Act 1982 (OIA) for information relating to Named Patient Pharmaceutical Assessment (NPPA) applications for ustekinumab. Your questions and our corresponding responses are detailed below.

Please note that PHARMAC approaches its assessment of requests for information under the OIA on the basis that, once released, the information becomes publicly available - in other words once we release the information to you it becomes available to any other party in that exact form (whether by you distributing it to others or by virtue of us receiving the same request from a different third party).

PHARMAC uses its NPPA process to consider whether to fund a treatment for an individual patient whose clinical circumstances are exceptional. Please refer to our webpage Making a NPPA application for details regarding the NPPA application process.

1.  The number of NPPA applications that have been made for ustekinumab.
2.  The number of rejections of those applications and the number of approvals.

To date, PHARMAC has received a total of 48 NPPA applications for ustekinumab for various indications. Of these applications:

  • 3 applications have been approved
  • 1 application has been withdrawn by the applicant
  • 44 applications have been declined due to the NPPA principles not being met.

3. The number of appeals to those applications that were rejected and the number of approvals and rejections of those appeals.

Declined applications can be resubmitted at any time for consideration if relevant new clinical circumstances arise or new evidence becomes available.

PHARMAC has a review process for applicants not satisfied with decisions made under the NPPA Policy. This review process is available for all NPPA applications, including Rapid Hospital Assessments.

To date, no reviews have been initiated for NPPA applications for ustekinumab.

4.  The person or committee who makes the initial determination of the NPPA application.
5.  The credentials of the person who initially screens the applications and how often there is a final determination that is different from that person's initial recommendation.
6.  Whether a gastroenterologist is involved in that initial determination.

Every NPPA application received by PHARMAC is initially reviewed by the relevant Funding Coordinator.

There are two steps in the assessment of NPPA applications. First the application must meet the principles of the NPPA Policy (Principles). If the Principles are met, we then consider the application against our Factors for Consideration (Factors). Staff that may be involved in either of these steps are:

  • Therapeutic Group Managers; or
  • Members of PHARMAC’s Medical Directorate; or
  • Health Economists.

The credentials of staff in these positions is considered when they are appointed to their role at PHARMAC as part of PHARMAC’s recruitment process and as such, we are not providing these details to protect their privacy (section 9(2)(a)).

We may also seek external clinical advice to help us to consider an application. Clinical advice may be sought from the NPPA Advisory Panel or members of the Pharmacology and Therapeutics Advisory Committee (PTAC) or any of its subcommittees (eg Gastrointestinal Subcommittee).

The credentials of our external clinical advisors are available via the above links.

In the assessment of every application, the outcome is reviewed and considered by multiple people.

7.  Whether Pharmac monitors the number of patients who are receiving infliximab at higher than the initial dosage specified on the Special Authority application (i.e. 5mg/kg every eight weeks)
8.  If Pharmac does monitor the information requested in question 7, what is that number?

When monitoring medicine usage, PHARMAC focuses primarily on overall usage from a budget management perspective and so we do not actively monitor infliximab usage for individual patients.

The Pharmaceutical Schedule allows dosing up to 10mg/kg for 3 doses every 16 weeks as part of a re-induction regime.

We trust that this information answers your queries. We are making our information more freely available, so we will now publish selected OIA responses (excluding personal details) on our website. Please get in touch with us if you have any questions about this.

Yours sincerely 

Rachel Read
Manager, Policy and Government Services