COST-EFFECTIVENESS OF PERSONALIZED THERAPY IN A RARE CANCER CASE AT END-OF-LIFE: A REAL-WORLD EXPERIENCE
European Statement
Clinical Pharmacy Services
Author(s)
Rossella Centola, Elisabetta D’Amico, Stefano Morabito, Maria Alfieri, Maria Giovanna Elberti, Lucilla Grisi, Giorgio Lilla, Amelia Filippelli, Francesco Sabbatino
Why was it done?
Precision Medicine (PM) in oncology remains a highly debated topic within Italian healthcare institutions. Currently, Next-Generation Sequencing (NGS) tests are not included in the Essential Levels of Care (LEA), and reimbursement policies vary across regions. Moreover, most targeted therapies based on mutational profiling are prescribed off-label.
What was done?
This study aimed to conduct a cost-effectiveness analysis of a personalized cancer therapy administered in an end-of-life setting.
How was it done?
Clinical and prescription data were collected for a targeted treatment with olaparib. Data were presented as a case report, and median progression-free survival (PFS) and overall survival (OS) were calculated in months. A cost-effectiveness analysis was performed comparing standard care with NGS-driven therapy, using company management software to estimate healthcare expenditure. The Incremental Cost-Effectiveness Ratio (ICER) was calculated and compared against a willingness-to-pay (WTP) threshold of €60,000, considering the rarity of the tumor.
What has been achieved?
The case involved a 56-year-old male diagnosed with cholangiocarcinoma in 2017. After multiple treatments, an NGS test revealed a BAP1 gene mutation associated with a BRCA-like phenotype. Off-label treatment with olaparib 300 mg BID began in June 2019. The personalized therapy resulted in a PFS of 21 months compared to approximately 2 months with best supportive care (standard treatment), and an OS of 48 months versus 29 months, respectively. The cost-effectiveness analysis, conducted from the Italian National Health Service perspective, considered direct healthcare costs including disease management, drug administration, and management of serious adverse events. Personalized therapy showed a survival gain of 2.5 life-years (LY) compared to 1 LY with standard care. The resulting ICER was approximately €45,000 per LY gained—well below the €60,000 WTP threshold.
What next?
This case illustrates that applying PM in end-of-life care for a rare cancer can yield significant survival and economic benefits. These findings highlight the need for greater integration of PM into clinical trials, structured data-sharing networks, and the establishment of solid evidence-based and pharmacoeconomic frameworks.
THE ADDITION OF A COST ANALYSIS CHANGES THE OUTCOME OF A TENDER (submitted in 2019)
Pdf
European Statement
Introductory Statements and Governance
Author(s)
Camilla Munk Mikkelsen
Why was it done?
Including a CA in the evaluation is time-consuming and I wanted to evaluate whether the obtained drug recommendation was different from the result we could have achieved without the inclusion of a CA. The CA process includes data collection from clinicians within resource consumption per drug, including the costs of time usage of physician, nurse and patient, transportation expenses, monitoring costs, blood tests, co-medicine, utensils, shipping and hospital facilities. When a CA is included it is possible to take the derived costs associated with treatment of different drug dispensing forms and specific costs of treatment with various analogue drugs into account to achieve a recommendation upon the lowest total price including the tender price and the derived costs associated with the treatment.
What was done?
Tenders are made on ATC-level 5, but clinically equivalent therapeutic areas are evaluated on ATC-level 4. The analogue competition is an important strategic tool when conducting tenders and elaborating national recommendations on therapeutic areas (TA). Since 2017 the evaluation of TA has been based on a clinical evaluation, an economic evaluation and a tender. Previously the call for tenders was based on clinical evidence only. To evaluate whether the addition of a cost analysis (CA) to a tender evaluation would alter the drug recommendation of TA, a re-evaluation of the processed TA, evaluated from October 2018 until October 2019, was made on multiple sclerosis, rheumatoid arthritis and severe asthma.
How was it done?
The drug recommendations on TA made in the period was re-evaluated. Results from the cases with multiple sclerosis, rheumatoid arthritis and severe asthma were evaluated on clinical evaluation, tender price and finally with or without the CA.
What has been achieved?
From October 2018 to October 2019 three TA have ended the evaluation process. The recommendation of severe asthma had a similar outcome regardless of the process used. For multiple sclerosis and rheumatoid arthritis, the CA altered the drug recommendations.
What next?
In order to balance resource consumption on performing CA and the economic impact on the outcome, the plan is to identify TA where it isn’t meaningful to conduct a cost analysis. In all other areas a CA will be included in the standard procedures.