The Pharmaceutical Benefits Advisory Committee (PBAC) considered four blood cancer treatments at its March 2020 meeting including a drug for myeloma – plitidepsin (Aplidin®).

Plitidepsin was given a subsequent decision not to recommend. This is the second time the drug was not recommended for listing on the Pharmaceutical Benefits Scheme (PBS) for this particular indication, having been previously considered by the PBAC at its July 2019 meeting.

The submission from Specialised Therapeutics was for plitidepsin in combination with dexamethasone for people with relapsed or refractory myeloma who have had three prior treatment regimens*.

Summary clinical trial information indicated that plitidepsin, which is administered in hospital as an IV therapy, was well tolerated and may reduce the risk of disease progression and death.

However, in its decision not to recommend plitidepsin for listing on the PBS in this indication, the PBAC noted that it considered the claim of non-inferiority versus the comparator drug (pomalidomide) was not demonstrated in the third-line setting and that the cost-effectiveness of plitidepsin was not demonstrated in the fourth-line setting.

The PBAC also considered the treatment comparison against dexamethasone as a monotherapy demonstrated only a marginal benefit in terms of progression free survival (PFS) without progressive disease (PD) confirmation, and the clinical benefit attributed to PFS with PD confirmation was not reliable.

The Leukaemia Foundation provided consumer comments to the PBAC in February 2020. These comments reflect the everyday experiences of people living with myeloma, sourced from our disease-specific community groups on Facebook.

A Victorian patient made the following comment:

“Very excited to hear of new drugs coming from nature – powerful bio-active agents have proven to wildly useful in medicine to date, and have only heard good things about the prospects of this drug for myeloma, which is known to be strongly relapsing to treatment. So while I’m not using it as yet, to have it in the arsenal of available drugs in the future is very encouraging.”

In its submission, the Leukaemia Foundation said: “the benefits of more effective and well tolerated treatments on the lives of individuals with myeloma cannot be underestimated. We assist myeloma patients over the course of their experience with this blood cancer and we know they would welcome the availability of a new treatment option”.

“The impact of this incurable cancer and the cytotoxic effects of treatment have an immeasurable impact on people’s lives and the lives of their families and carers.

“Giving clinicians more options in their ‘toolbox’ in the fight against myeloma, and removing barriers so patients can commence treatments tailored to their disease without any undue delays, is vitally important.”

The Leukaemia Foundation also made comments to the July 2019 PBAC meeting for a previous submission for plitidepsin for myeloma.

*   At least three treatments including both a proteasome inhibitor (PI) and an immunomodulator (IMiD), or in patients who have received two prior lines of therapy, if the patient is refractory and/or intolerant to both a PI and an IMiD.