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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.

IMMPACT: A THERAPEUTIC EDUCATION PROGRAM TO SUPPORT SEAMLESS CARE FOR PATIENTS LIVING WITH CANCER

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European Statement

Clinical Pharmacy Services

Author(s)

A. Decottignies, D. Bouchoucha, S. Lévêque, S. Barthier, J.F. Morere, A. Marfaing, A. Rieutord

Why was it done?

Cancer has become a chronic disease. However, the impact of the disease and related treatments on patients’ everyday life remain poorly addressed all along the patient clinical pathway. The objective was to customise support to the patient to be able to better understand and control the disease and treatment consequences.

What was done?

An integrated care model based on a therapeutic education programme (TEP) was designed to better support patients living with cancer in their everyday life. The hospital pharmacy led the project, launched in October 2013 and implemented for patients in March 2015. It is called IMMPACT (impact of illness and medication in patients undergoing chemotherapy). This TEP was accredited by the National Authority (ref No ETP 13/30 in August 2013).

How was it done?

A transprofessional working group was formed. Our concern was to propose a smooth and safe transition from hospital to home. To do so, the hospital set up a partnership with the health network to facilitate seamless care. A prospective survey was conducted in October 2013 including 41 ambulatory patients. Three priority themes were identified: tiredness, haematological problems and digestive disorders management. According to the results, educational sessions have been collaboratively designed and implemented.

What has been achieved?

The patients group workshops began in January 2014. To date, 31 group workshops have been run. These group workshops are facilitated by trained healthcare professionals using educational tools such as Barrows Cards, mosaic characters or metaplan. 83 patients have already experienced this programme. Patient global satisfaction is >92%.

What next?

Given the success of this programme and to fulfil the growing expectations of patients, new sequences are being designed about pain, chemotherapy related neurotoxicity, socio-aesthetics and lymphoedema management as well as developing a serious game for the patient. Finally, IMMPACT extension to community pharmacists is being considered to further sustain patients empowerment and continuity of care.

IMPLEMENTING CHEMOTHERAPY DOSE-BANDING USING RETROSPECTIVE DATA ANALYSIS AND EXPONENTIAL CALCULUS

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European Statement

Production and Compounding

Why was it done?

Chemotherapies are generally prescribed and produced as a function of Body Surface Area (BSA). The most recent literature recommends that marketed drugs continue to use BSA-based dosing supported by clinical evidence. If not, it recommends DB with adjustments for other important parameters.

What was done?

Determine which of the drugs compounded in our centralised chemotherapy production unit were potential candidates for dose banding (DB) for adults, whilst guaranteeing patient safety and meeting the needs of physicians, pharmacists and nurses.

How was it done?

The database of chemotherapy doses produced between 2010 and 2013 was analysed to define a Top 10 chart of the most common protocols and compounds. Dosage patterns were analysed and new bands were modelled using exponential calculus in order to aid in DB decision-making. Discussions with interdisciplinary teams and senior physicians took place in order to promote acceptance of the project and its deployment.

What has been achieved?

Oncology professionals requested an integration of bands into the electronic prescription system, the possibility to prescribe doses above those suggested using BSA and a maximum 5% margin of difference to the usual prescribed dose. They highlighted the necessity of maintaining “ready for administration” doses. For example, in 2013, 613 infusion bags of gemcitabine were produced in 111 different doses, ranging from 266 to 2900 mg. Following the new specifications, just two bands (2000 mg and 1805 mg) already fulfil 50% of annual production needs; producing five band doses streamlines 90% of annual production needs.

What next?

Chemotherapeutic doses can now be prepared in bands and the pharmacy activity can be rationalised by producing doses in batches. The imminent introduction of automation should ensure accuracy of the doses delivered. Future studies should examine product stability so that chemotherapy production planning becomes highly efficient.

DEVELOPMENT AND IMPLEMENTATION OF GUIDELINES FOR THE SAFETY MANAGEMENT OF INTRATHECAL CHEMOTHERAPY IN PATIENTS WITH HEMATOLOGICAL MALIGNANCIES

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European Statement

Patient Safety and Quality Assurance

Why was it done?

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What was done?

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