Making timely decisions on funding cancer drugs is challenging because of their high cost and uncertain evidence base when they enter the market. Various risk sharing arrangements (RSA) have been introduced to mitigate uncertainty and improve patient access to cancer drugs in Australia.
To examine the Pharmaceutical Benefits Advisory Committee recommendations on submissions made to list cancer drugs on the Pharmaceutical Benefits Scheme (PBS).
We reviewed publicly available documents on the PBS website for antineoplastic and immune-modulating agents from March 2010 until March 2017. Data extracted included drug name, indication, type of supporting evidence provided, source of uncertainty, reason for rejection/deferral, and the special RSA applied to medications 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., collecting further evidence) arrangements.
We identified 179 submissions for 64 cancer drugs. Positive recommendations were made in 67 (37%) submissions with an average of 2.1 submissions to approval (range: 1-5). Although the majority of submissions were based on randomised controlled trials, uncertain clinical evidence was reported in 80% of the rejected/deferred submissions, predominantly due to uncertain overall or progression free survival benefit. Other reasons for rejection/deferral included high and/or uncertain cost-effectiveness ratios (77%) and inappropriate comparator (11%). Of the drugs with positive recommendation, forty-eight (75%) had price reduction and/or rebate arrangements and 50 (75%) had a clinical continuation rule; however, only 4 drugs (6%) were recommended conditional on collecting more data.
Uncertainty in the clinical evidence and cost-effectiveness remains the leading cause for the delay in funding new cancer drugs. The majority of RSAs have focused on continuation rules and price reductions and/or rebates. However, there is limited utilisation of flexible arrangements to fund new cancer drugs conditional on collecting further evidence.