Key considerations in reimbursement decision-making for multiple sclerosis drugs in Australia

In Australia, the Pharmaceutical Benefits Advisory Committee (PBAC) advises on the reimbursement of drugs to be subsidised through the Pharmaceutical Benefits Scheme (PBS). This study aims to provide insights into the PBAC process and key considerations regarding the reimbursement of MS drugs in Australia.

The factors considered by the PBAC and its advice on whether to reimburse a drug are documented in public summary documents (PSDs). Qualitative content analysis of PSDs was conducted for all MS drugs considered by the PBAC between January 2006 and January 2018. Key issues identified by the PBAC were extracted and categorised. Common issues were identified and compared between drugs indicated for MS.

A total of 23 submissions were evaluated relating to 13 MS drugs. Eight were recommended for reimbursement; an approval rate of 35% per submission and 62% per drug. Approval rates were higher for disease modifying treatments (73% per drug) than for symptomatic drugs (0% for nabiximols and fampridine submissions). The most frequently discussed issues in PSDs, irrespective of PBAC decision, were: (1) the validity of the indirect comparisons formed (n = 11); (2) the validity of the approach to obtain utilities (n = 6); (3) the lack of appropriate/long-term safety data (n = 8); and (4) the time horizon used in the economic models (n = 3).

A small but important number of issues have been consistently identified by the PBAC in relation to submissions for reimbursement of MS drugs. Drug developers and clinical trial investigators who are aware of these issues will be able to anticipate data requirements for reimbursement decision-making and thus potentially improve the evidence submitted for listing of MS drugs in Australia.