Background: Hospitals are usually the entry point of new technologies. Facing the challenge posed by the growing costs of cancer drugs, there is a need for healthcare providers to articulate systematic and timely formulary decision-making processes considering not only the efficacy and safety of these treatments, but also their economic impact and efficiency.
Objectives: To develop a method for the rapid assessment of high-cost cancer drug indications (HCCDI) for formulary decision-making in a hospital setting.
Methods: We identified and structured decision criteria from previous formulary decisions regarding HCCDI, and a literature review of decision frameworks in oncology and the Chilean health reimbursement system. We generated a criterion/indication combination score for two samples of HCCDI (solid tumors and hematologic oncology) by measuring their performance and translating it to a common 0-100 scale. From the consensus among clinicians, managers, pharmacists, and health economics researchers of the institution, we assigned weights to each criterion to calculate the aggregate scores. Finally, we validated each step of the process with the decision-makers.
Results: The identified criteria were (1) absolute and relative difference in overall survival and (2) progression-free survival of the area under the Kaplan-Meier curves; (3) difference in the proportion of patients with severe adverse events; (4) improvement of quality of life; (5) incremental expected costs; and (6) efficiency. The inputs for the assessment of each HCCDI were high-quality studies identified through rapid reviews of electronic databases and health technology assessment reports. Aggregation of criteria 1-4 was named “benefit index”. The quotient of the benefit index and the HCCDI costs was called “efficiency index”. The costs were then used to calculate the indication’s budget impact.
Conclusions: We developed a formulary decision-making tool for the rapid assessment of HCCDI validated by decision-makers. The method has allowed us to assess 79 indications for solid tumors and 21 for hematologic oncology since 2019, facilitating the reproducibility of the decision criteria. Improvements in the principles regarding HCCDI formulary decisions contribute to a more egalitarian use of resources and patients’ access to healthcare. Patient, public, and/or healthcare consumer involvement: No patients, public, or healthcare consumers were involved in this project.