Presentation (CTI)

OBJECTIVES: Early drug development decisions rarely include formal assessment of the relationships between potential target product profile (TPP) characteristics and cost effectiveness (CE). This study investigated those relationships for a hypothetical DrugX (DX) in atopic dermatitis (AD) relative to a reference treatment, dupilumab (DU).
METHODS: A pharmacometric (PM) - pharmacoeconomic (PE) model was developed to describe the PM-PE relationship for DU. The PM-PE model for DX was based on the same structure, with modifications of PM model parameters, relative to DU. Literature meta-data were digitized from published studies and the PM model described longitudinal eczema area and severity index (EASI) score as a fractional decrease from baseline EASI score, including effects for placebo response, topical corticosteroids (TCS), and drug effects. The PE model was derived from a published PE analysis of dupilumab in AD and was characterized as a Markov model with transition probabilities between health states: non-responder, EASI response, and death, linked to state-specific quality-adjusted life years (QALYs). Monte Carlo simulations varied DX properties relative to DU parameters: maximum fractional efficacy (Emax), inter-individual variability, onset time (T50), and persistence of therapy (POT).
RESULTS: Improvements in Emax and T50 for DX relative to DU improved mean efficacy, but did not impact QALYs or probability of CE at any WTP level. Reductions in IIV did not provide a CE benefit. Improvements in POT resulted in an increase of approximately 1 QALY and improved probability of CE for DX relative to DU (30% vs 15%, respectively, at a WTP of $100,000). To achieve a similar probability of cost effectiveness without the effect on POT, a decrease in DX pricing of approximately 10% relative to DU would be necessary.
CONCLUSIONS: TPP factors improving efficacy do not necessarily translate to increased QALYs or probability of CE for DX relative to DU.