Statement from the Chair of the Pharmaceutical Benefits Advisory Committee on eculizumab (Soliris®)

Statement from the Chair of the Pharmaceutical Benefits Advisory Committee Associate Professor Suzanne Hill on eculizumab (Soliris®)

Page last updated: 19 September 2014

PDF printable version of Statement from the Chair of the Pharmaceutical Benefits Advisory Committee Associate Professor Suzanne Hill on eculizumab (Soliris®) (PDF 234 KB)

19 September 2014

As PBAC Chair I am deeply concerned by Alexion’s response to the Minister’s approval of funding for Soliris for the treatment of aHUS. The PBAC recognises the value of this drug in the treatment of aHUS, which is why we recommended it at the full price requested by the company, Alexion. Unfortunately this positive recommendation has been misrepresented, patients have been unduly alarmed and the listing of the product has been further delayed at the expense of very ill patients.

Let me first be clear about what PBAC recommended. We have NOT recommended that all patients must stop receiving treatment after 12 months.

Atypical HUS is a severe and in some patients life-threatening disease. However, it is not a single disease. There are a mix of causes and the scientists studying this disease still do not fully understand it. Treatment with Soliris works in some patients – but in some patients it does not.

On this basis and the basis of the evidence presented by the company to the Committee in March and August this year, PBAC has recommended that:

  • All patients meeting the starting criteria, that is patients with active, progressive disease should commence treatment as soon as diagnosed.
  • These patients should be reviewed at six months to ensure they are responding to the treatment.
  • Then at 12 months, patients will be assessed again. If a patient is responding well but does not have the disease fully under control, they will continue to receive subsidised treatment as long as it is needed to maintain their health and as long as they continue to improve.
  • However, if the tests show that a patient has achieved remission of the disease, based on criteria developed with experts in this field, the PBAC has advised that the treatment should stop and the patient’s condition monitored. This is good standard clinical practice. You do not give people drugs unless you absolutely have to as all drugs have side effects.
  • If at any time that standard clinical monitoring shows the slightest reduction in health and therefore a patient is starting to relapse, even just an abnormal blood test, then the PBAC has recommended that the patient be reinitiated on treatment immediately, under simplified criteria.
Unfortunately the evidence available right now does not tell doctors how to choose which patients will respond. That is why PBAC recommended a managed entry scheme for this drug so we can collect the data and use that data to help inform better clinical practice and support further submissions when required. It is about using the clinical data from patients being treated today, to help the patients that might need treatment tomorrow.

The PBAC, most of whom are practicing doctors, has balanced all these factors in making this recommendation to Government. These criteria make it possible to give patients full subsidised access to this drug immediately, while data is collected on the longer term use of this drug, and work to identify which patients will be able to recover fully and live a normal life without long-term treatment and those that will need life-long treatment.

Media Enquiries: Kay McNiece 0412 132 585

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