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The US regulation of off-label uses of medicines
Published in Andrea Parziale, The Law of Off-label Uses of Medicines, 2023
The rationale underlying the existing US drug approval process is that new drugs must show safety and efficacy before selling. In essence, companies must apply to the FDA for the marketing authorisation of new products, providing the FDA with experimental data showing safety and efficacy.30
Clinical Trials
Published in Michael Ljungberg, Handbook of Nuclear Medicine and Molecular Imaging for Physicists, 2022
Clinical trials are performed to obtain scientifically sound and robust data on the safety and efficacy of an intervention, while at the same time respecting the rights and guaranteeing the safety and well-being, of the trial subjects. The prospective nature of a clinical trial reduces the bias inherent to retrospective data collection and permits randomization to different interventions to be able to compare them to each other. Clinical trials are also the basis for obtaining marketing authorization for a new drug, that is, exclusive rights to commercialize a new drug within the geographic area of the relevant regulatory authority – the EMA (The European Medicines Agency. www.ema.europa.eu) for the European Union, the FDA (The U.S. Food & Drug Administration. www.fda.gov) for the United States, and so forth. In order to guarantee the scientific and ethical soundness of a clinical trial, as well as the quality of the data resulting from it, there is a whole system consisting of clinical trials offices, clinical research organizations, regulatory authorities, ethics committees, and others, dedicated to just that. In this chapter, you will get an overview of this system as well as some useful references for further reading.
Translational Challenges
Published in Carla Vitorino, Andreia Jorge, Alberto Pais, Nanoparticles for Brain Drug Delivery, 2021
Bárbara Rocha, Nelson Pacheco Rocha, Bruno Gago
The evidence of clinical effect in humans must then be confirmed in patients, treated in controlled environments during phase IIb/III clinical trials - T2, translation to patients. At the end of this stage researchers will have insights about the clinical application, efficacy and safety. Marketing authorisation can be obtained at this stage.
Immunosenescence, inflammaging, and cancer immunotherapy efficacy
Published in Expert Review of Anticancer Therapy, 2022
Julieta E Rodriguez, Marie Naigeon, Vincent Goldschmidt, Matthieu Roulleaux Dugage, Lauren Seknazi, Francois X Danlos, Stephane Champiat, Aurélien Marabelle, Jean-Marie Michot, Christophe Massard, Benjamin Besse, Roberto Ferrara, Nathalie Chaput, Capucine Baldini
Two bispecific T cell engagers have been approved. Catumaxomab, targeting epithelial cell adhesion molecule (EpCAM) and CD3, was approved by the EMA in 2009 for the treatment of malignant ascites [62]. However, at the request of the marketing authorization holder market authorization was withdrawn in June 2017. Blinatumomab, targeting CD19 and CD3, was approved by the FDA in 2014 and by the EMA in 2015 for the treatment of Philadelphia chromosome negative B cell acute lymphoblastic leukemia [63]. Older patients treated with blinatumomab are expected to have similar benefit from this therapy [1,64,65]. Bispecific T cell engagers that target CD3 activate T cells without the need of engagement of costimulatory molecules such as CD28. However, recent data have shown that expression of CD80 or CD86, which signal through CD28, on cancer cells increases bispecific T cell engager cytotoxicity in vitro [66] as does co-treatment with a monoclonal CD28 antibody [67]. These data suggest the importance of CD28 activation for maximal anti-tumor efficacy of bispecific T cell engagers. Recently, the use of bispecific antibodies targeting PSMAxCD28 and EGFRxCD28 has demonstrated antitumor efficacy in combination with anti-PD-1 antibodies in syngeneic and xenograft models. For these bispecific T cell engagers, the efficacy of this treatment in humans may be limited by loss of CD28 expression on T cells classically observed during lymphocyte senescence [68].
Comparison of the registration process of the medicines control authority of Zimbabwe with Australia, Canada, Singapore, and Switzerland: benchmarking best practices
Published in Expert Review of Clinical Pharmacology, 2022
Tariro Sithole, Sam Salek, Gugu Mahlangu, Stuart Walker
Labeling issues must be addressed before a product is authorized in all five agencies. At the MCAZ, responsibility for the marketing authorization decision lies with the Registration Committee. The Director General makes the decision on registration for Health Canada and HSA, whereas for TGA, the responsibility is delegated to a senior medical officer, and at Swissmedic the decision is made by the case team with the involvement of the Head of Division/Sector. In all five agencies, compliance with GMP is audited during the review process and the outcome informs product authorization. The target time for the overall approval for a full review for the MCAZ is 480 days, inclusive of the applicant’s time and this is comparable to the target times for the comparator countries: TGA, 330 days including the applicant time; Health Canada, 355 days excluding applicant time to respond to an NOD and any other approved pauses, ranging from 5 to 90 days; HSA, 378 calendar days excluding the queue and applicant time; and Swissmedic, 330 days excluding the applicant time (Table 2). The target times are comparable because the 480 days for MCAZ includes the applicant’s time. If the applicant’s time (target 60 days per assessment cycle for 2 cycles) was to be excluded, this would come down to 360 days.
An overview of PLGA in-situ forming implants based on solvent exchange technique: effect of formulation components and characterization
Published in Pharmaceutical Development and Technology, 2021
Tarek Metwally Ibrahim, Nagia Ahmed El-Megrab, Hanan Mohammed El-Nahas
In the case of parenteral preparations, FDA states that the qualitative (Q1) and quantitative (Q2) composition of the test and reference products should be similar and the product copies should satisfy the Q1/Q2 sameness to be authorized with the Abbreviated New Drug Application (ANDA) (Hua et al. 2021). For example, the in-vitro and in-vivo performance of the polymeric PLGA-based products can be significantly influenced by the alterations in the manufacturing process in addition to the changes in the chemical properties of PLGA polymer regarding its composition, molecular weight and end-capping. This can result in major changes in the biopharmaceutical properties and bioavailability of the proposed drug. Given the complexity of this context, FDA requires a complete characterization of PLGA polymer to fulfill the Q1/Q2 requirements by the comparison of polymer composition, molecular weight distribution and polymer architecture (Zhou et al. 2018). After the marketing authorization, the marketed product may introduce some changes or adverse outcomes that clearly may lead to alterations in the quality, safety and efficacy of the drug. Therefore, several procedures including continuous clinical study analyses in patients should be followed by the manufacturer to ensure the maintenance of positive long-term performance and safety of the product (FDA 2016, 2018b).