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Global Health Econ Sustain Fast-track drug approvals in Brazil

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1. Introduction exorbitant costs, exemplified by Zolgensma employed in
spinal muscular atrophy (SMA) treatment and Hemgenix
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Due to the emergence of novel technologies that provides administered for hemophilia B intervention. These
a more in-depth understanding of molecular intricacy, therapies currently hold the foremost position in terms
the health-care sector is currently undergoing substantial of price, commanding a value of 3.5 million dollars. In
transformations, which could affect how therapeutic Brazil, exclusively Zolgensma has obtained registration,
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targets are selected. The industry has exhibited a specific signifying its status as the costliest available medication
inclination toward the advancement of technologies within the nation, amounting to 1.3 million dollars
tailored to addressing rare diseases and exploring niche
drugs. This inclination extends to prevalent diseases (equivalent to 6.5 million BRL, based on the 2022 exchange
as well, due to their ability to target niche markets and rate from the Organization for Economic Co-operation and
command exorbitant prices within the market. Moreover, Development [OECD]). Our research endeavors involved
adjusting the monetary figures to reflect a conversion rate
these studies tend to be more cost-effective due to the
involvement of a smaller patient cohort, while the costs of 5.164 BRL = 1 USD (OECD, 2023). In December 2022,
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stemming from advertising and business development Zolgensma was granted reimbursement approval by the
are lesser as they are typically managed by a select group National Health System (SUS) for pediatric patients up to
of specialists. The considerable and escalating disparity 6 months of age diagnosed with SMA type I (Ministério
between the therapeutic efficacy of numerous novel niche da Saúde, 2022). CAR-T cell therapies represent a recent
drugs and their associated price elucidates the rationale advancement in innovative treatments, accompanied by
behind the pharmaceutical industry’s adoption of a new new regulatory challenges. This approach involves the
business model centered around these drugs (Gagnon, genetic modification of the patient’s own T cells for the
2015). purpose of treating specific hematological cancers. Several
CAR-T cell products have already obtained approval from
According to epidemiological data retrieved from the the Food and Drug Administration (FDA) (Amini et al.,
Orphanet database (Nguengang Wakap et al., 2020), a total 2022). In 2022, Kymriah (tisagenlecleucel) became the
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of 6172 rare diseases have been identified, with 71.9% of first CAR-T therapy approved by Brazil’s National Health
them being of genetic origin and 69.9% affecting children. Surveillance Agency (Anvisa), with authorization for the
The prevalence of these diseases in the general population treatment of acute lymphoblastic leukemia and diffuse
ranges from 3.5% to 5.9%, equivalent to an estimated 263 to large B-cell lymphoma (Archanjo, 2022). In addition, in
446 million affected individuals (Nguengang Wakap et al., 2022, Anvisa granted approval for the first clinical trial
2020). In Brazil alone, there are approximately 13 million aimed at the national development of a CAR-T product for
individuals with rare diseases. Furthermore, the National cancer treatment (ANVISA, 2022a).
Cancer Institute (INCA) projects that by 2025, there will
be approximately 704,000 new cases of cancer per year in The randomized, controlled, and clinical trial is the
Brazil (INCA, 2022). These conditions present significant optimal study design for assessing the efficacy and safety
challenges to the provision of universal healthcare as of novel pharmaceutical agents. However, challenges arise
mandated by the national constitution, not only in Brazil when working with limited populations that meet the
but also in other countries. Governments worldwide are eligibility criteria, thereby imposing inherent constraints
currently grappling with ethical considerations, access to on conducting such ideal evaluations. The credibility of
healthcare, sustainability, and the regulatory frameworks the resultant evidence is compromised by studies featuring
governing the registration of new drugs, which may or may small sample sizes, lack of blinding and randomization, and
not enhance patients’ quality of life and overall survival. the absence of direct comparators, leading to a heightened
risk of bias (Fogel, 2018; Zeng et al., 2015).
The utilization of precision medicine in studies holds
a prominent position in the current medical landscape. Considering the evolving health market landscape,
Recent advancements in genomics technology, coupled along with mounting pressures from the pharmaceutical
with reduced costs, play a crucial role in enabling the industry and patients, as well as the constraints imposed
swift identification and selection of therapeutic targets by limited and substandard available evidence, a host of
within human cells. The pursuit of drugs incorporating novel challenges has emerged in relation to technology
innovative technologies, such as monoclonal antibodies, registration, pricing, and evidence-based recommendations
targeted therapies, oligonucleotides, as well as gene and for appraisal. These challenges pose a universal defiance
cell therapies, has emerged as a primary objective in the for regulatory agencies, which must make deliberations
registration of new clinical trials (Shahryari et al., 2019). based on the restricted evidence concerning the efficacy
Gene therapies represent a subset of pharmaceuticals with and safety of these innovative therapies, while ensuring the

Volume 1 Issue 2 (2023) 2 https://doi.org/10.36922/ghes.0995