The Pharmaceutical Benefits Advisory Committee’s decision not to recommend the listing of nusinersen (Spinraza™) on the Pharmaceutical Benefits Scheme in November 2017

Page last updated: 16 January 2018

Nusinersen (trade name Spinraza™) is marketed by the pharmaceutical sponsor Biogen Australia Pty Ltd (Biogen) as a treatment for spinal muscular atrophy (SMA), a rare genetic neuromuscular disorder.

Biogen submitted a request to subsidise Spinraza through the Australian Government’s Pharmaceutical Benefits Scheme (PBS) for the treatment of infantile onset (Type I) and childhood onset (Types II and III) SMA.

The request was considered by the Pharmaceutical Benefits Advisory Committee (PBAC) in November 2017. The PBAC, an independent, expert group, makes recommendations to the Australian Government about PBS listings.

At the November 2017 meeting, the PBAC did not recommend the listing of Spinraza on the PBS for the proposed treatments. Although the PBAC acknowledged the high and urgent clinical need for SMA treatments, the Committee was concerned with the uncertainty of the clinical effectiveness of Spinraza across the different onset stages.

The PBAC considered that further information on the cost-effectiveness of the treatment is needed to determine an appropriate PBS subsidy price. Based on the information available at the November meeting, the PBAC determined that a substantial reduction in the price proposed by Biogen will be required.

The PBAC’s decision can be found on the PBS website. The outcome for Spinraza is found in the table titled 'November 2017 – 1st Time decisions not to recommend'.

A more detailed summary of the PBAC’s decision, known as a Public Summary Document, will be published on 16 March 2018 on the PBS website.

Biogen has also made a submission to the PBAC to list Spinraza on the PBS for the treatment of Type I SMA. The PBAC will consider the resubmission at its meeting in March 2018, and the meeting outcomes will be published on the PBS website on
20 April 2018.

The PBAC would welcome a further submission for the listing of Spinraza on the PBS for the treatment of Type II & III (childhood onset) SMA at any time. The timing of any submission is a matter for Biogen.

In the meantime, there may be alternative options for patients to access Spinraza at a reduced cost outside of PBS arrangements.

The treating specialist of a patient may wish to contact Biogen to find out whether the medicine can be supplied through an expanded access or compassionate access program. However, decisions to grant individual access to medicines through these pathways occur at the sole discretion of the sponsor concerned.

Furthermore, the Australian Government provides funds to the states and territories to assist with the costs of providing public hospital services. A treating specialist might consider making an application on their patient’s behalf to the drug and therapeutics committee of a local public hospital to request assistance with the cost of treatment.

A decision not to list a medicine on the PBS does not represent the final view of the PBAC regarding the merits of the product. A company can resubmit to the PBAC at any time with new information that addresses the concerns highlighted by the PBAC.

The PBS listing process provides an opportunity for consumers to make comments on submissions before the PBAC. Consumer comments on the medicines to be considered at the March 2018 PBAC meeting, such as Spinraza, can be provided via the PBS website.