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Global Health Econ Sustain Fast-track drug approvals in Brazil

data. Manufacturers are required to report any negative current expedited approval processes may not consistently
results of which they become aware, but there is no align with the expected therapeutic benefits for patients
requirement that they actively seek out negative results. (Lexchin, 2015b).
As clinical reporting will remain voluntary, it is likely that These studies highlight the importance of critically
adverse reactions to drugs with accelerated approval will evaluating the therapeutic value of drugs approved through
be significantly underreported, although through hospital fast-track pathways. The discrepancy between expedited
notification systems, adverse drug events for specialist-only approvals and independent assessments of therapeutic value
medications, such as immunotherapies, may be detected raises concerns about the potential compromise in rigorous
and reported at a higher rate (Linger & Martin, 2018). evaluation during expedited review processes. Therefore, it
Concerns about the safety of drugs registered through is crucial for regulatory agencies to establish clear guidelines
fast track have been raised after the provisional approval and post-marketing surveillance to monitor the real-world
process was implemented in Canada. When compared to therapeutic value and long-term benefits of drugs approved
drugs approved through a standard registration process, through expedited pathways. By doing so, regulatory
there is a statistically significant risk that these drugs will agencies can ensure that expedited approvals truly deliver
generate more serious adverse events or even be removed meaningful clinical advances to patients in need.
from the market (Lexchin, 2015a). Accelerated access to new drugs can represent a way of
A study by Mostaghim et al. (2017) reported an increase adapting the regulatory landscape as current regulations
in safety-related changes in drug labels and highlights the do not adequately deal with advanced therapies. Due to
importance of active safety monitoring for all drugs after a deeper understanding of the causes of diseases, these
initial registration, particularly those approved through drugs are generally tested in small clinical trials with
accelerated routes. The FDA receives voluntary reports patients whose genetic profiles are known. On the other
of adverse events related to drugs, which can lead to hand, this approach can put patient at risk and increase
changes in the market authorizations. Since 2007, the financial risks for individuals and society. In addition, the
FDA’s electronic monitoring of health data and other process of accepting regulatory decisions made in other
sources through the Sentinel system has provided a critical countries is often complicated by variable evaluation
mechanism to conduct active observational studies to criteria and differences in clinical practices, which can
complement the voluntary reporting system. Compared to make the extrapolation of these decisions dangerous in
non-accelerated drugs, accelerated drugs had a 48% higher other countries (Kubler, 2018).
rate of changes in warnings and contraindications on their Managed entry agreements are a set of tools used to
market authorization. A qualitative review of changes in mitigate the impact of uncertainty and financial burden
the warning section of the label revealed that < 5% were when registering a new drug. They can be subdivided into
changed to account for a reduction of risk to patients. payments that are tied to health outcomes or financial
The therapeutic value of drugs approved through fast- considerations, where the price is defined based on
track pathways has become a topic of interest and concern quantitative measures such as estimated consumption of
within the medical and regulatory communities. A 2020 the technology in question. These agreements can enable
study examined drugs approved by the US FDA and EMA health systems to reduce their expenditure on advanced
between 2007 and 2017 and found that less than a third therapies, especially where there is significant uncertainty
of these drugs were rated as having high therapeutic value around safety and efficacy, as well as around the value of a
by independent organizations. The study also revealed that new drug. Managed entry agreements can be potentially
expedited drugs were more likely to receive high ratings, important tools for improving scientific capability and
but a significant number of expedited drugs approved by knowledge within health systems, whether public or
the FDA were considered to have low therapeutic value private, while providing access to innovative and high-cost
(Hwang et al., 2020). In the similar vein, a cohort study new drugs and seeking to minimize the opportunity costs
conducted on Health Canada’s priority review process for of decisions (Zampirolli Dias et al., 2020).
new drugs, published in 2014, found that the assignment
of priority approval by Health Canada was only a fair 5. Conclusion
predictor of a drug’s therapeutic value once it entered the With the advancements in drug development, regulatory
market. The study emphasized the need for Health Canada agencies must reorganize to meet the increasing demands
to review its criteria for using priority reviews to ensure for registration, considering the questionable levels of
accurate identification of drugs that represent significant evidence, and ensuring the rapid access of the population
therapeutic advancements. These findings indicate that the to new safe and effective therapies. Considering the

Volume 1 Issue 2 (2023) 9 https://doi.org/10.36922/ghes.0995