OBJECTIVES

Oncology drugs are often approved for multiple indications, for which their clinical benefit varies. Aligning a price to this differing value remains a challenge. This study investigates the effect of clinical trial and other drug characteristics on launch sequencing at marketing authorisation (MA) and health technology assessment (HTA) level across 7 countries.

METHODS

25 multi-indication drugs were identified with FDA approvals between 2009-2019. The FDA approval date was taken as a measure of ‘global launch’. Data on clinical trial characteristics, regulatory approval and HTA recommendations were extracted from regulatory agency websites, clinicaltrials.gov and HTA agency websites for all indications across England, Scotland, France, Germany, Canada, Australia and the USA. Logistic regression models were used to assess the association of variables with launch sequence and HTA outcomes.

RESULTS

Preliminary results suggest that first launched indications, relative to subsequent ones, are statistically and substantially more likely to have both conditional approval (OR, 2.91, p<0.05) and orphan designation (OR, 3.50, p<0.001), a higher phase clinical supporting trial (OR per trial phase, 1.62, p<0.01), and receive priority review (OR, 3.24, p<0.01). At HTA level, indications with positive recommendations are more likely to be first in FDA sequence (OR, 2.17, p<0.05), first line treatments (OR, 2.23, p<0.05), and have more patients enrolled in the pivotal trial (OR per 100 patients, 1.14, p<0.001) relative to indications with negative recommendations. Products with better clinical endpoint performance (OR per MCBS point, 1.32, p<0.01) and stronger clinical supporting trial designs (OR per trial phase, 1.49, p<0.001) are statistically more likely to receive a positive recommendation at HTA level.

CONCLUSIONS

Although there is considerable variability, manufacturers show a tendency to first launch drugs in orphan indications in the USA with accelerated and/or conditional approval. They then expand to other markets through indications with higher disease prevalence.