Submission on Pharmac’s proposal to decline funding for eculizumab

1 August 2013
This submission is from the NZ Organisation for Rare Disorders (NZORD). We are a charitable trust set up in 2000 to improve information, support, clinical care, treatment and research for rare disorders. We work closely with wider networks of 160 rare disease support groups, and 60 larger groups advocating for those with common disorders.
NZORD held a one-day seminar on Thursday 1 August with a range of speakers covering academic and professional experience, patient advocacy, basic science research, media and industry representatives, plus a political panel. Attendance, though varying at different times of the day, was an average of about 65 to 70 people present for all sessions.
This submission reflects presentations and discussions at the seminar, as well as many themes which have emerged from discussions and networking NZORD has done with a variety of patient advocacy groups and other stakeholders (including professional groups, industry, regulators, and payers, both nationally and internationally) over many years relating to development, registration and funding of medicines, and specialised medicines in particular. It is also informed by discussions at several of Pharmac’s recent forums on its decision criteria consultation, and advice received from other professional advisors.
We ask Pharmac to consider this submission in the context of a previous request we sent to Pharmac on 29 July 2013, asking Pharmac to withdraw this consultation because of errors and misleading information contained in it. That request should also be considered a submission from NZORD to you on this consultation, and is attached as Appendix 1.
Given your concurrent consultation on your decision criteria, you should also consider this submission to be relevant to and forming a submission on that consultation as well, even though we are likely to submit further on that particular consultation closer to the close of those submissions.
Summary of submission:
  • The consultation document contains several significant errors and has other misleading information in it. Pharmac should withdraw the document, alert all stakeholders, and publish an explanation of this.
  • In deciding on this matter and any other decisions, Pharmac must act consistently with the human rights framework that exists in New Zealand, including acting equitably and incorporating community values to give practical effect to the right to life and the right to health. There is no indication in the document that any of these issues have been addressed.
  • Pharmac must also comply with the principles of NZ’s legal and constitutional system, and in this context this means when acting as an agent of the DHBs, placing greater emphasis of the purposes of the Act, the objectives of DHBs, Minister’s expectations (specifically about access to specialised medicines), its MoU with DHBs, and policy and decision criteria guidance in the health sector. The document does not address any of these important factors nor discuss how the proposal addresses these objectives and requirements.
  • The role of ethics in decisions is an important one but poorly addressed by Pharmac over many years, and not addressed at all in this consultation. Appropriate and proper decision-making in health requires that these issues are more robustly addressed in a way that is consistent with the widely accepted role of ethics in healthcare in NZ.
  • Consumer engagement has not been adequately undertaken by Pharmac in respect of this particular decision, and in general Pharmac’s consumer engagement falls far short of a standard expected across all areas of life in NZ.
  • There are several weaknesses in the economic evaluation done by Pharmac on eculizumab, and failures by PTAC and its subcommittee to adequately address several important decision criteria set out in your operational policies and procedures. Pharmac itself also fails to address several of these criteria in its document.
  • There are contested views of Pharmac’s interpretation of several key phrases in its legislative brief, including the meaning of “best health outcomes” and “reasonably achievable”. We propose alternative perspectives which Pharmac should take into account and which we believe would lead to a different decision on this consultation.
Read the full text of our submission at this link.