Correspondence and information about Pompe disease

OIA response

12 August 2021

Dear [name and contact details withheld]

Request for information

Thank you for your request dated 15 July 2021 under the Official Information Act 1982 (OIA) for information relating to Pompe disease.

You requested the below information, between 1 January 2010 to 25 June 2021.

  1. Correspondence between the Minister of Health and the Ministry of Health and PHARMAC referencing Pompe.
  2. Reasons for declining the medicine Myozyme for Pompe disease.
  3. Any death records or reports where Pompe has been referenced in any capacity.
  4. All information/correspondence regarding newborn screening for Pompe disease. 

In our correspondence to you on 28 July 2021, we advised that we had decided to transfer requests 3 and 4 of your request for information to the Ministry of Health. Please find our response to requests 1 and 2 below. 

Correspondence between the Minister of Health and the Ministry of Health

Please find copies of correspondence between Pharmac and the Minister of Health and/or the Ministry of Health regarding Pompe disease attached with this response letter. 

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). 

In order to ensure we are able to get this information to you promptly, we have not released some of the names of individuals from non-Pharmac parties as this would require further consultation to make a decision on release. If you do wish to request these names (and it is our assumption that you do not require these) please let us know. 

Additionally, we have redacted a small amount of information from the documents where this is out of scope of your request or where we consider this is necessary to:

  • protect the privacy of natural persons (section 9(2)(a))
  • protect information where the making available of the information would be likely to unreasonably prejudice the commercial position of the person who supplied or who is the subject of the information (section 9(2)(b)(ii))
  • protect information which is subject to an obligation of confidence or which any person has been or could be compelled to provide under the authority of any enactment, where the making available of the information would be likely to prejudice the supply of similar information, or information from the same source, and it is in the public interest that such information should continue to be supplied (section 9(2)(ba)(i))
  • maintain legal professional privilege (section 9(2)(g))
  • enable Pharmac to carry on, without prejudice or disadvantage, negotiations, including commercial negotiations (section 9(2)(j)). 

As required under the OIA, we also considered whether, in the circumstances, the withholding of this information was outweighed by other considerations which render it desirable, in the public interest, to make this information available. In this case we did not consider that the public interest outweighed the reasons for withholding the information. 

Please note you have the right, by way of complaint under section 28(3) of the OIA to an Ombudsman, to seek an investigation and review of our decision.

Reasons for declining the medicine Myozyme for Pompe disease

Alglucosidase alfa (Myozyme) first became funded by Pharmac in December 2016. Myozyme is currently funded via Special Authority application. Initial funding applications can be submitted for individuals aged up to 24 months at the time of initial application and who are diagnosed with Infantile Pompe Disease (IPD). 

We currently have two open funding applications for Myozyme which can be found on our Application Tracker: link)

  1. P000988: Late onset Pompe disease
  2. P001452: Pompe's disease 

We have received recommendations from our clinical experts that the funding applications for alglucosidase alfa for the treatment of Pompe disease should be declined on the basis of:

  • uncertainties regarding survival benefit
  • modest clinical benefits with regards to ambulation and pulmonary function
  • a lack of evidence for clinically significant benefit in juvenile and adult-onset Pompe disease
  • high cost of disease-modifying treatment 

For complete details of the clinical advice and funding recommendations given to Pharmac, please refer to copies of the records of the Pharmacology and Therapeutics Advisory Committee (PTAC) and the Rare Disorders Subcommittee meetings found within the Seeking Clinical Advice section of the applications within the Application Tracker. 

We have not yet made a funding decision for either of these funding applications however, they have both been ranked to our Recommended for decline prioritisation list. This means we do not currently intend to fund the medicines for the proposed indications, based on current evidence available. 

To read more about our prioritisation lists, please refer to our website: > Medicine funding and supply > The funding process > Priority lists for funding applications

We trust that this information answers your queries. We are making our information more freely available, so we 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