Most drugs have data only from clinical trials focused on a specific population at the time of first registration, so their indications for use are restricted to this population. In Brazil, the prices of new drugs for clinical conditions with no therapeutic alternatives are relatively high. When these drugs expand to other indications their prices are not reviewed, which can have a major financial impact. This study aimed to evaluate the financial impact of expanding the indication for trastuzumab deruxtecan.

We calculated the annual cost to treat all Brazilian patients with the indications listed for trastuzumab deruxtecan at first registration, and then all additional indications. Populations were estimated from epidemiological data from National Committee for Health Technology Incorporation reimbursement documents and a clinical trial comparing trastuzumab deruxtecan with trastuzumab emtansine. Costs were calculated using the ANVISA Câmara de Regulação do Mercado de Medicamentos price value and a patient weight of 70 kg.

In January 2022, trastuzumab deruxtecan was introduced in Brazil for patients with human epidermal growth factor receptor (HER2) positive metastatic or unresectable breast cancer who had received two or more anti-HER2 treatment regimens. In June 2022, the indication was expanded to patients with HER2-positive metastatic or unresectable breast cancer who had received one anti-HER2 treatment regimen. In November 2022, the indication was further expanded to patients with metastatic or unresectable low-expression HER2 breast cancer who had received prior systemic therapy. The number of patients estimated to be eligible for the drug increased from 383 to 23,000, with an increased total cost from BRL467,970,786 (USD90,621,763) to BRL26,048,234,160 (USD5,044,197,164).

The expansion of indications for trastuzumab deruxtecan may substantially increase its financial impact and compromise the sustainability of health systems. In Brazil, the lack of monitoring of drug prices means that the only change in prices occurs due to regulated annual inflation adjustments. Regulation is needed to reduce drug prices according to new indications, changes in therapeutic options for the same condition, and obsolescence.

Aortic stenosis is an insidious disease that has a high mortality rate when it becomes symptomatic. Surgical valve replacement is the treatment of choice and has predictable risks. Transcatheter aortic valve replacement (TAVR) is a less invasive alternative to surgery, which is indicated for high-risk patients.

Complications after TAVR include paravalvular leak, cerebrovascular events, and the need for pacemaker implantation. A health technology assessment report carried out by the Health Technology Assessment Unimed-BH group in 2018, two years before it became part of the National Supplementary Health Agency, recommended the introduction of TAVR with the following criteria: indications provided by a group of specialists; forwarding of a report with detailed clinical data; results of imaging exams; and follow-up results for up to one year after the procedure. After the introduction of TAVR with the agreed criteria, it was possible to access TAVR results from the private healthcare system of Unimed-BH.

Administrative data were collected from the Unimed-BH database. All patients who received a TAVR implant from 2013 to 2017 were included by virtue of a court injunction, and after 2018 by operator concession and within agreed criteria.

From July 2013 to June 2019, 83 patients underwent TAVR implantation by Unimed BH. The median age of patients was 83.4 years (interquartile range 66.5 to 97.9), most of whom were women (56%). There was a predominance of patients in New York Heart Association classification III (50%) and IV (29%). There were 36 patients who underwent TAVR before 2018 and 47 patients within the agreed criteria. In the period prior to the agreed criteria, 28 percent needed a pacemaker, compared with 23 percent after 2018. During the follow-up period, 39 patients died: 18 (50%) before 2018 and 11 (23%) after 2018.

The agreement made with the providers, which included the obligation of having a team of specialists responsible for the indication and access to clinical data through the report, improved patient outcomes. This may be due to having a better indication for the procedure or to the greater experience of the professionals involved in its delivery.

The Corona Virus Disease 2019 (COVID-19) pandemic is the biggest public health crisis of all time. Private health care plays a major role in health globally. We conducted a study to document the engagement of a Brazilian private health care organization to influence decisions to private and public health emergencies of COVID-19.

This retrospective study evaluated the outputs of the health technology assessment (HTA) group of Unimed-BH, a private Health Maintenance Organization (HMO) with 1.5 million participants in Belo Horizonte, Minas Gerais state. The study evaluated the impacts on the overview of health local decisions in the municipality and the national supplementary health agency (ANS) during the period from March 2020 to December 2022.

During the pandemic, Unimed-BH made all its sanitary decisions based on scientific research, such as the use of masks and appropriate medications. Even though some medicines, such as regdanvimab, were authorized for emergency use by the Brazilian Health Regulatory Agency (ANVISA), Unimed-BH did not recommend their use due to uncertain evidence, and months later, ANVISA withdrew the registration.

Unimed-BH also conducted a systematic review of ivermectin for COVID-19 treatment, which showed no effect, and therefore did not recommend its use. Additionally, Unimed-BH provided weekly updates on COVID-19 data, including suspected and confirmed cases, hospitalizations, and deaths in their customer portfolio. The organization also actively supported the decisions made by ANS and municipal managers using evidence and statistics on the pandemic. The Unimed-BH HTA group produced a total of 167 reports from March 2020 to December 2022.

Belo Horizonte had the lowest in-hospital mortality rates with COVID-19 in Brazil. Unimed-BH’s HTA reports provided evidence-based assessments for decision-making, proposed partnership with policymakers, fomented information transparency, and strict follow-up on pandemic numbers, which may have contributed to the lower fatality rate in our city. These findings underscore the importance of private healthcare organizations in responding to COVID-19 emergencies, and their potential to support evidence-based decision-making and minimize the impact of the pandemic.

Most new drugs have only clinical studies focused on a single population at the time of first registration, hence their indications for use are restricted to this population. For clinical conditions when there are no other treatments available, new drugs have higher costs in Brazil. There is no review of prices when these medications broaden their therapeutic areas, and this can have a significant financial impact. This study’s objective is to assess the financial implications of pembrolizumab’s incremental indication after its initial registration.

We calculated the annual cost to treat all Brazilian patients with indications for use in the first registration and all incremental indications of pembrolizumb. Populations were estimated by epidemiological data from the pembrolizumab clinical trials called, KEYNOTE studies, and the INCA 2023 cancer estimate for the Brazilian population. Costs were calculated by CMED-ANVISA price value and considering the dosing of 200mg every 3 weeks.

In 2016, pembrolizumab was granted registration in Brazil was restricted to patients with advanced melanoma. In 2022 the indication was expanded to more than 20 new indications, with several studies in progress that potentially will lead to further inclusions. The estimate of patients eligible for indications increase of 1,796 to 99,544 patients with an increased total cost from BRL625,802,837 to BRL34,685,366,192 (USD121,185,677.4 to USD6,716,763,399.04).

The financial burden of pembrolizumab’s expanded uses after it was first approved could significantly rise, endangering the long-term viability of healthcare systems. In Brazil, where medicine costs are not regularly monitored, the annual inflation adjustment is the only factor that causes prices to change. In order to lower medicine prices in response to the addition of new indications, the expansion of therapeutic options for the same condition, or even obsolescence, regulations are required.

The fast track in drug registration by the Brazilian Health Regulatory Agency, ANVISA, began in 2017 and is intended to prioritize analysis related to drugs relevant to public health. This process is appropriate in situations where there are no therapeutic alternative available or for technologies show significant improvement in safety, efficacy, or adherence to treatment.

The Brazilian public administration has a tool for accessing information called Transparency Portal. Thus, data on the number of drugs approved by fast track between 2018 and 2021 were requested through this tool and evaluated.

The data received by the Brazilian transparency portal shows that the number of requests for fast track had an increase from one in 2018 to 32 in 2021. There is an important increase of registrations, being drugs with phase II trials and with single-arm clinical trials. With registration based on a phase II trial, these patients are in fact receiving drugs in a context similar to clinical trials, but with funding from the healthcare system. Given that phase II studies are conducted in a limited population, there has been an increase in the registration of drugs with suboptimal efficacy and safety concerns. This scenario of uncertainty leads to non-adherence to treatment and a discrepancy in real-world outcomes in comparison to the clinical trial. About 70 percent of phase II trials, show no benefit, and only 30 percent proceed to later phases. It is noteworthy that about 50 percent of the studies that move on to later phases fail to show benefits. Nowadays the number of drugs approved by the fast track has increased, many probably with phase II studies and no comparator group.

Given the uncertainties in the efficacy and safety of a drug registered via fast track, often based on phase II studies, implementing provisional registration with real-world evaluation of outcomes, and coupled with financing based on risk-sharing agreements, may be a sustainable alternative for health systems.

The Corona Virus Disease 2019 (COVID-19) pandemic has impacted the functioning of health systems, imposing the need for adaptations. Elective surgeries also needed to adapt, and research has shown higher mortality in newly infected surgical patients after or during procedures. Thus, was recommended the suspension of elective surgeries during the pandemic. Early studies evaluating the effect of COVID-19 pandemic on bariatric surgery have reported a substantial reduction in procedures performed.

This retrospective study evaluated the impact of the suspension of bariatric surgeries for a Brazilian Health Maintenance Organization: UNIMED-BH, based on the analysis of data from before and during the pandemic of COVID-19.

There were 2,641 bariatric procedures conducted in 2019 with a 14.1 percent reduction in volume to 2,314 procedures in 2020. In 2021, there were 2,813 bariatric procedures and 1,700 procedures were observed from January to August 2022. Therefore, it appears that in 2022 the demand for bariatric procedures will be similar to the year 2019, which was before the COVID-19 pandemic.

From the analysis of the data, a decrease in bariatric surgical volume was evidenced during the year 2020 when compared to 2019. Post-pandemic, monitoring is necessary to assess whether the system was able to meet the demand for bariatric surgical procedures.