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Global Health Econ Sustain Impact of new drug indications in Brazil

provided information on the specific inclusion criteria for patient populations, informing decision-making processes
the drugs and estimated patient populations for different and resource allocation strategies.
indications.
2.4. Ethical considerations
2.2. Calculation of treatment costs This study involved the analysis of publicly available data
The annual costs for treating all eligible Brazilian patients and did not involve any human subjects or personally
for trastuzumab deruxtecan and pembrolizumab were identifiable information. Therefore, ethical approval was
calculated based on the current prices regulated by CMED not required for this study.
and Anvisa.
2.5. Statistical analysis
For trastuzumab deruxtecan, the costs were calculated
by considering the CMED/Anvisa price at the time of Descriptive statistics were used to summarize the estimated
registration and expanded use. The calculations were based patient populations and associated costs for trastuzumab
on patients with an average weight of 70 kg. deruxtecan and pembrolizumab. Graphs and tables were
created to present the results effectively using Microsoft
For pembrolizumab, the annual cost of treatment Excel 2021.
for all eligible Brazilian patients was calculated for the
first indication considered for registration in 2016 and 3. Results
all incremental indications up to November 2022. The 3.1. Trastuzumab deruxtecan
populations were estimated based on epidemiological
data from the pembrolizumab clinical trials (KEYNOTE Trastuzumab deruxtecan’s indications in Brazil underwent
studies) and the 2023 INCA cancer estimates for the several expansions. In January 2022, it was initially
Brazilian population. The costs were calculated using indicated for patients with HER2-positive unresectable
the current updated CMED/Anvisa price value and or metastatic breast cancer who had received two or more
considering a 200mg dose every 3 weeks. anti-HER2 treatment regimens, representing the third
line of treatment for metastatic disease. By June 2022,
Monetary values were adjusted using the 2022 exchange the indication was expanded to include patients who had
rate (USD to BRL) from the Organization for Economic received only one anti-HER2-based treatment regimen. In
Co-operation and Development (OECD). The cost values November 2022, the indication was further expanded to
were converted to USD using the exchange rate of 5.164 include patients with low HER2-expressing or low HER
BRL = 1 USD (OECD, 2023). (IHC 1+ or IHC 2+/ISH-) unresectable or metastatic

2.3. Limitations breast cancer who had received previous systemic therapy.
It is important to acknowledge certain limitations The expansion of indications for trastuzumab
and uncertainties inherent in the data sources and deruxtecan resulted in a significant increase in the
methodology utilized in this study. The estimates of eligible estimated number of eligible patients. The eligible patient
patient populations and cost projections are based on population increased from 383 for the initial indication
various data sources, which may introduce variations and to 23,000 for the expanded indications (Table 1). This
expansion had a substantial impact on the total costs of
potential inaccuracies. Unaccounted variations in patient treatment, with the total cost for trastuzumab deruxtecan
characteristics, treatment patterns, and disease prevalence increasing from USD 90.6 million to USD 5.0 billion.
across different regions of Brazil could influence the
accuracy of the estimates. Table 1 presents the incremental indications for
trastuzumab deruxtecan, including the estimate of eligible
In addition, the analysis did not account for potential
changes in clinical practice or treatment guidelines that patients, annual treatment cost per patient, and total cost.
For the initial indication of HER2-positive breast cancer in
may arise from the expanded indications of pembrolizumab the third line of treatment, there were 383 eligible patients
and trastuzumab deruxtecan. Such changes in clinical with an annual treatment cost of USD 236,610.35 per
practice patterns could significantly impact the number of patient, resulting in a total cost of USD 90,621,763.50. The
eligible patients and associated costs, potentially affecting expansion to HER2-positive breast cancer in the second line
the overall findings of the study.
of treatment increased the number of eligible patients to
Furthermore, the study did not assess the comparative 581, while the annual treatment cost per patient remained
effectiveness or cost-effectiveness of the expanded the same. Consequently, the total cost for this indication
indications. Such an assessment could provide valuable reached USD 127,420,806.64. The largest expansion in
insights into the value of these treatments in different indications occurred for HER2-low-expressing breast

Volume 1 Issue 1 (2023) 4 https://doi.org/10.36922/ghes.0994