OBJECTIVES:

Medicines to treat rare cancers (prevalence <1 in 10,000) often have high cost and an insufficient evidence base to inform their registration and reimbursement decisions. Various risk sharing arrangements (RSA) have been proposed to improve patient access to cancer medicines in Australia. We aimed to examine the Pharmaceutical Benefits Advisory Committee recommendations on submissions made to list rare cancer medicines on the Pharmaceutical Benefits Scheme (PBS).

METHODS:

We reviewed publicly available PBS documents from March 2010 until July 2017 for antineoplastic and immune-modulating agents designated as orphan drugs by the Therapeutic Goods Administration. Data extracted included medicine name, indication, type of supporting evidence provided, source of uncertainty, reason for rejection or deferral, and the special RSA applied to medicines with a positive recommendation. The arrangements were categorised into non-outcome based (i.e., price reductions or rebate), outcome-based (i.e., clinical continuation rule), or data provision (i.e., coverage with evidence development) arrangements.

RESULTS:

We identified 70 submissions for 30 rare cancer indications. Positive recommendations were made in 26 (37%) submissions with an average of 2.2 submissions to approval (range: 1-5). Uncertain clinical evidence was reported in 80% of the rejected/deferred submissions, predominantly due to uncertain overall survival benefit. Other reasons for rejection/deferral included high and/or uncertain cost-effectiveness ratios (75%) and inappropriate comparator (5%). Of the indications with positive recommendation, twenty (77%) had price reduction and/or rebate arrangements and 24 (92%) had a clinical continuation rule; however, only 2 indications (8%) were listed conditional on collecting more data.

CONCLUSIONS:

The majority of RSAs have focused on price reductions and/or rebates and clinical continuation rules; nevertheless, there is limited utilisation of coverage with evidence development arrangements. Provisional PBS listing conditional on collecting additional fit-for purpose evidence is a potential solution to mitigate decision uncertainty and improve patient access to medicines for rare cancers in Australia.