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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
Rare Diseases Drug Development
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Shein-Chung Chow, Shutian Zhang, Wei Zhang
In 1992, FDA initiated the FDA Accelerated Approval Program to allow faster approval of drugs for serious conditions that fill an unmet medical need. The faster approval relies on use of surrogate endpoints. Drug approval typically requires clinical trials with endpoints that demonstrate a clinical benefit, such as increased survival for cancer patients. Drugs with accelerated approval can initially be tested in clinical trials that use a surrogate endpoint, or something that is thought to predict clinical benefit. Surrogate endpoints typically require less time, and in the case of a cancer patient, it is much faster to measure a reduction in tumor size, for example, than overall patient survival.
Designing and Running a Clinical Trial
Published in Trevor F. Cox, Medical Statistics for Cancer Studies, 2022
A systematic review looks at relevant publications relating to the intervention in question and summarises their findings, see Chapter 8 – Meta-Analyses. Evidence is based on several studies, whereas for a single RCT, evidence is based on just one study – and, as always with statistical methods, errors do occur such as Type I and Type II errors in hypothesis testing which can lead to wrong conclusions. The US Food and Drug Administration (FDA) usually requires two well-designed clinical trials supporting the use of a new treatment in order to gain drug approval. Case-control studies and cohort studies are described in detail in Chapter 7 – Cancer Epidemiology. A case report gives the details of a particular patient's demographics, diagnosis, treatment and outcome data. A case series is the case reports of several patients who have received similar treatments. Expert opinion can often be wrong!
Pharmacotherapeutic options for cancer cachexia: emerging drugs and recent approvals
Published in Expert Opinion on Pharmacotherapy, 2023
Lorena Garcia-Castillo, Giacomo Rubini, Paola Costelli
The lack of effective approaches against cachexia results from several issues, but particularly relevant are the many limitations that make drug approval slow and difficult, such as cost, complexity, and length of the studies. This has led to a significantly lower number of clinical trials dedicated to investigating new drugs endowed with anti-cachexia activity. The heterogeneity of the trial design and patient population can also contribute to expand the journey to drug approval. Moreover, a deep analysis of the literature dealing with the development of novel strategies for the treatment of cachexia, revealed that a critical limitation still stands in the definition of this syndrome. A shared definition and standardized diagnostic criteria are essential for a homogeneous patient enrollment, which would render the studies much more relatable and reproducible, ultimately improving the research in this field. Additionally, gathering information from clinical trials is a difficult task due to tardiness in study updates and the unavailability of the results.
Machine learning-based prediction of drug approvals using molecular, physicochemical, clinical trial, and patent-related features
Published in Expert Opinion on Drug Discovery, 2022
As the process of drug development, especially clinical trials, is continuously changing in time [30], it is important to observe how the success of modeling drug approval varies over time. For this, we applied a temporal performance analysis. We split clinical trials into six different time frames (Figure S4). For instance, the first training time frame comprises clinical trial data until 2008 (starting from the oldest record), and testing time frame includes the trial data between the years of 2009 and 2020. For the second time frame, the training data is until 2010 whereas the testing data is 2011–2020, etc. The model was trained and tested on each time slice independently. The RF classifier with the hyper-parameter values of; the number of trees: 200, the maximum feature number: square root of the total number of features; maximum depth: 16; minimum samples leaf: 2; minimum samples split: 2 were used. The performances of the models were estimated using F1-score, accuracy, precision, recall, and MCC metrics.
Meropenem-vaborbactam: a critical positioning for the management of infections by Carbapenem-resistant Enterobacteriaceae
Published in Expert Review of Anti-infective Therapy, 2022
Maria Mouktaroudi, Antigone Kotsaki, Evangelos J. Giamarellos-Bourboulis
As mentioned above, the main comparators of meropenem-vaborbactam are ceftazidime-avibactam, imipenem-relebactam, plazomicin and colistin. Table 5 is comparing the main characteristics of the four drugs regarding in vitro activity and clinical efficacy [55–61]. Imipenem-relebactam is not included in Table 5 since the phase 3 program of clinical development is ongoing. What needs to be outscored is that with the exception of the phase 3 trials conducted for drug approval, there are two available randomized clinical studies aiming to the evaluation of the clinical efficacy of the new agents against resistant enterobacteria. REPRISE assessed the clinical efficacy of ceftazidime-avibactam over BAT but enrolled patients were sufferers from infections by ceftazidime-resistant enterobacteria and P.aeruginosa and not isolates resistant to carbapenems [55]. The CARE study compared the efficacy of plazomicin over colistin for bloodstream infections. Although the study showed a trend toward superiority of plazomicin, it remains inconclusive due to premature termination caused by slow enrollment [56]. As a consequence, TANGO II is the only available study showing superiority of meropenem-vaborbactam over BAT for the clinical cure of infections caused by KPC-producers. However, it should always be considered that the number of participants in TANGO II was limited.