einstein (São Paulo). 21/Dec/2021;19:eED6840.
Assessment of novel technologies in healthcare – off-label use of drugs and the ethics of implementation and distribution of COVID-19 vaccines
DOI: 10.31744/einstein_journal/2021ED6840
Assessment of new health technologies in the context of off-label use and repurposing of drugs for treatment and prevention of COVID-19
Some medications are used for purposes that are not described in the package insert; therefore, they are not analyzed in controlled studies, neither submitted to evaluation by regulatory processes, such as HTA, or official agencies, such as CONITEC, the National Health Agency (ANS – Agência Nacional de Saúde) and Anvisa. This practice is generally known as off-label use.() The off-label use of medications or other medical technologies must be differentiated from repurposed or repositioned use, in which medications that are already approved are submitted to a rigorous and systematic process of analysis, aiming to identify compounds that might be applied to other conditions, such as emerging or rare diseases, for which no specific treatment is available.() Drug repurposing has advantages relatively to the traditional development of new medicines, since repurposed drugs have often undergone safety studies, and can be implemented faster, with lower financial investments. The initial identification of candidate compounds for repurposing can be conducted by experimental or computational methods, which identify molecules more likely to act in targets of interest. Another option is to select candidate compounds that have phenotypical or functional characteristics similar to the drugs already used for that specific purpose.()
While repurposing of drugs implies the demonstration of efficacy in clinical trials, off-label use can be indicated by a physician, who simply believes the patient will benefit from that medication. In the case of medicines sold over the counter, off-label use can be decided by the patients themselves, not requiring medical prescriptions. Off-label use of medications may imply risks not only for patients but also for physicians, due to the lack of clinical studies or approval of the new purpose of the drug by Anvisa, leaving them with no legal support. Moreover, off-label prescription exempts the pharmaceutical industry from legal and judicial responsibilities if adverse reactions occur, since this use is not described in the package insert.()
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