MANAGING UNCERTAINTY IN ONCOLOGY DRUG ASSESSMENTS: APPLICATIONS IN MULTIPLE MYELOMA

Abstract

The surge in novel oncology drugs forced healthcare payers to prioritize cost-effective options within limited budgets. Health technology assessment (HTA) is vital in evaluating treatments based on clinical effectiveness, safety, and cost-effectiveness, yet, challenges persist: the amplified uncertainty in oncology and the need for financing decisions in cases of high unmet clinical need and financial burden to patients.

This thesis tackled these issues in two parts through a series of mixed-method studies. First, it explored strategies to manage uncertainty in evaluating oncology drugs, applied to multiple myeloma – a disease area characterized by high costs and substantial uncertainty. The initial phase assessed a myeloma drug regimen's effectiveness and cost-effectiveness. Second, it investigated financing mechanisms to mitigate risk in the real-world. This phase evaluated the feasibility of risk-sharing schemes through a systematic review, a qualitative inquiry, and a simulation of schemes in Singapore context. This thesis advocates for improved uncertainty communication, aligning assessment results with payer strategies. It offers insights for health systems navigating the evolving landscape of high-cost treatments.