What evidence are funding decisions for new cancer drugs based on?
CHE RESEARCH BITES
By Kah-Ling Sia and Jing Jing Li
Published: 15 January, 2024
New cancer drugs can be lifesaving. However, funding decisions are often based on limited evidence about their long-term effectiveness and cost-effectiveness.
Such evidence includes surrogate outcomes: early outcomes of a therapeutic treatment that can help predict the final treatment outcome. For example, time to cancer progression can be an early predictor for the long-term effectiveness of new cancer drugs.
Surrogate outcomes have become increasingly important to support the registration and subsidy of new cancer drugs, with growing pressure for earlier access to potentially lifesaving treatments. Yet, few surrogate outcomes used in clinical trials accurately predict overall survival (or time to death).
In Australia, more than two thirds of the funding decisions for cancer drug submissions made between 2012 and 2022 relied at least partially on surrogate outcomes, according to a recent review conducted by Monash and CHE researchers.
Examining all relevant Pharmaceutical Benefits Advisory Committee (PBAC) Public Summary Documents during that period, they found that more than half of the funding decisions relying on surrogate outcomes presented survival data based on interim trial results. Some trials have since published final survival results. Validations studies – which compare surrogate and final outcomes, revealed that surrogate outcomes and overall survival were moderately related at best.
This research suggests that surrogate outcomes offer a useful but limited measure of the lifesaving effect of new cancer drugs. Better evidence on the relationship between the surrogate and final outcome is required to support its use in funding decisions.
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