Rejection of Vimizim at March 2016 PBAC Meeting

Page last updated: 23 August 2016

At its March 2016 meeting the Pharmaceutical Benefits Advisory Committee (PBAC) considered a resubmission from Biomarin Pharmaceuticals Australia Pty Ltd for Pharmaceutical Benefits Scheme (PBS) listing for elosulfase alfa 5mg/5mL injection (Vimizim). The submission requested listing for the treatment of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

By law, the Australian Government is not permitted to list a medicine on the PBS without a positive recommendation from the PBAC. The PBAC is an independent, expert advisory body, established to make recommendations to the Government about PBS listings. When considering requests for a recommendation to list on the PBS, the PBAC is required to have regard to safety, clinical effectiveness and value-for-money of the treatment. Clear and reliable scientific evidence is required to show that the medicine works for the intended treatment, is unlikely to cause more harm than good, and that the benefit it gives warrants the money expected to be expended across the Australian community. The same evaluation requirements are applied in all cases to ensure consistency, fairness and value.

In March 2016 the PBAC recommended against listing of this medicine at this time on the PBS. The PBAC considered that while there was sufficient basis to conclude that treatment with elosulfase alfa results in a small but meaningful clinical improvement, it was not satisfied that the drug was good value-for-money for the benefit it provides.

The PBAC would accept a resubmission from the sponsor addressing the PBAC’s concerns at any time, for instance, by seeking a lower asking price.

A submission requesting inclusion of Vimizim on the Life Saving Drugs Program (LSDP), which is separate from the PBS, has also been received. Following receipt of such a request the Chief Medical Officer provides advice to the Minister for Health on potential inclusion of the drug on the LSDP. This advice involves consideration of the criteria for inclusion on the LSDP against the submission and evidence about the disease and treatment with the drug.

The criteria for funding a drug for a particular disease under the LSDP are:

  1. There is a rare but clinically definable disease for which the drug is regarded as a proven therapeutic modality, i.e. approved for that indication by the Therapeutic Goods Administration.
  2. The disease is identifiable with reasonable diagnostic precision.
  3. The disease causes a significant reduction in age-specific life expectancy for those suffering from the disease.
  4. A patient’s lifespan will be substantially extended as a direct consequence of the use of the drug.
  5. The drug must be clinically effective, but rejected by the PBS listing because it fails to meet the required cost effectiveness (value-for-money) criteria.
  6. There is no alternative drug listed on the PBS, or available for public in-hospital patients, which can be used as lifesaving treatment for the disease. However, the availability of an alternative drug under the LSDP does not disqualify the proposed drug from consideration for the LSDP.
  7. There is no alternative non-drug therapeutic modality (e.g. surgery, radiotherapy) which is recognised by medical authorities as a suitable and cost effective treatment for this condition.
  8. The cost of the drug, defined as the cost per dose multiplied by the expected number of doses in one year for the patient, would constitute an unreasonable financial burden on the patient or his/her guardian.
Once a decision has been made, and any appropriate consultation has occurred with the sponsor company, that decision can be made public. An exact date for this is not known, but this process is progressing.

BioMarin has stated that it is committed to working with the PBAC and the Department of Health to make elosulfase alfa available either on the PBS or the LSDP for patients with MPS IVA.

Subsidising clinical treatments is not solely the responsibility of the Australian Government. The states and territories, through the public hospital systems, also have an important role in providing assistance with the cost of pharmaceuticals, including high cost treatments. The Australian Government provides substantial funding to the states and territories to assist with the costs of providing public hospital services. Doctors treating patients can make an application to the drug and therapeutics committee of a local public hospital for assistance with the cost of non-PBS listed medicines.

Alternatively, the sponsor of this medicine may have a compassionate use or expanded access program to enable access to the medicine at a reduced cost. It must be noted that any compassionate use decision is one made by the sponsor alone. The sponsor details are:

BioMarin Pharmaceutical Australia Pty Ltd
119 Willoughby Road
CROWS NEST NSW 2065

Telephone: 02 9956 3809


Reviewed: 27 March 2017