OBJECTIVES:

Innovative cancer therapies that target specific genes and proteins to slow or prevent carcinogenesis and tumour proliferation are being increasingly used in clinical practice. In this analysis we examine the drivers for targeted therapies to replace traditional chemotherapy as most appropriate comparator for EU3 health technology assessments (HTAs) in oncology, as well as the impact of this switch on HTA outcomes for subsequent assessments.

METHODS:

Single drug assessments in melanoma, breast, colorectal, kidney, lung, ovarian, and prostate cancer by G-BA, HAS and NICE were analysed (January 2011 – June 2018).

RESULTS:

Targeted cancer therapies have been increasingly listed as appropriate comparator (from 28% [n=5] in 2011 to 74% [n=14] in 2018) albeit varying by tumour type. Drivers for targeted therapies to become relevant comparators extend beyond demonstrating a clear benefit versus traditional chemotherapy, since even targeted treatments without marketing authorisation, or having received a negative HTA outcome were found to be considered an appropriate comparator. Remarkably, in some cases this switch was actually triggered by the submitting manufacturer.

Targeted therapy replaced traditional chemotherapy as the appropriate comparator in three different therapeutic areas: prostate cancer, melanoma, and lung cancer. The speed at which this happened varied by agency and depends on launch of another (targeted) therapy (range: 8 months to 29 months). The impact of the change in comparator was most notable in Germany where in 75% of cases this resulted in a worse benefit rating.

CONCLUSIONS:

The number of assessments, indications, drivers and time by which targeted therapy replaced traditional chemotherapy differs across agencies.

Our analysis shows that targeted therapies are increasingly regarded as relevant comparators for new product launches, despite being relatively new to the market. The rapidity of change can impact HTA outcomes. Early engagement with payers is vital to ensure that appropriate evidence can be generated to meet HTA requirements.