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The Study Population:
Published in Lynne M. Bianchi, Research during Medical Residency, 2022
Lynne M. Bianchi, Luke J. Rosielle
Note: Define inclusion and exclusion criteria for every study. Investigators usually know to consider inclusion and exclusion criteria for prospective studies but sometimes overlook their importance in retrospective designs, such as chart reviews.
External Control Using RWE and Historical Data in Clinical Development
Published in Harry Yang, Binbing Yu, Real-World Evidence in Drug Development and Evaluation, 2021
Qing Li, Guang Chen, Jianchang Lin, Andy Chi, Simon Davies
This chapter presents an overview of how to adopt external control using RWD and historical control in clinical development. Randomized clinical trials are considered the gold standard for clinical development. However, recruiting a large number of subjects into a randomized clinical trial can be challenging, especially for orphan drug enrollment, certain subtype diseases, and rare diseases. Moreover, it is sometimes unethical or not feasible to conduct such a randomized clinical trial with a parallel control arm. When there are no approved drugs for a certain life-threating rare disease, randomizing patients into a placebo group may raise ethical concerns (FDA 2014b). Despite the challenges of conducting randomized clinical trials, there are also common limitations of traditional clinical research, including generalizability of clinical research findings and rapidly rising cost of both time and money. Due to the strict inclusion and exclusion criteria in clinical trials, patients in clinical trials are restricted and may not reflect the real clinical practice. Using RWD in clinical development can reflect the actual treatment effect in the real-world setting. Therefore, many researchers have become interested in integrating RWD with traditional clinical research in more diverse scenarios.
Postmarketing Surveillance of Drugs*
Published in Gary M. Matoren, The Clinical Research Process in the Pharmaceutical Industry, 2020
Before a drug is marketed, it is investigated intensively in experimental animals and human subjects to determine its safety and efficacy. During the Phase III development period it is tested in carefully designed prospective randomized controlled clinical trials in as many as 3,000 subjects. These clinical trials have a rigorous design in which the participants must satisfy specific inclusion and exclusion criteria. For instance, children and pregnant women (or women who might become pregnant) are likely to be excluded. Patients with a broad range of comorbid conditions of varying severity are specifically excluded. Dosing and length of prescription are carefully predetermined. The result is that the observations and conclusions made under rigorous scientific conditions may not specifically apply to the broad subsequently treated population. Nor are events that may occur when much larger populations are treated likely to be observed among the limited-sized population which is studied.
Correlation between oculometric measures and clinical assessment in ALS patients participating in a phase IIb clinical drug trial
Published in Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, 2023
Eitan Raveh, Assaf Ben-Shimon, Vova Anisimov, Rivka Kreitman, Edmund Ben-Ami, Elisheva Nechushtan, Nurit Birman, Vivian E Drory
A cohort of 32 ALS patients (mean age 60.75 ± 10.36 years, 13 females), all participants of a phase IIb randomized prospective double-blind placebo-controlled study, with an open label extension, conducted at Tel Aviv Sourasky Medical Center (TASMC), Israel, were enrolled. The study protocol was approved by the Tel-Aviv Sourasky Medical Center Helsinki Committee for Human Rights (trial number 0421-22) and was publicly registered with clinicaltrials.gov (Identifier NCT05407428). All participants provided written informed consent at screening. Inclusion and exclusion criteria were based on the protocol of the clinical drug trial. The main inclusion criteria were age ≤75 years, a disease duration of less than 30 months, ALSFRS-R score ≥25 and a slow vital capacity ≥60% of predicted. Further exclusion criteria for the OM study were markedly poor vision and advanced cognitive impairment, that would render the examination impossible. All patients underwent a prior neurological examination including basic eye movements testing, and an evaluation including a standard muscle testing and the Revised ALS Functional Rating Scale (ALSFRS-R) (16).
Clinical trials for neuroregenerative therapies for spinal cord injury: what have we learnt so far?
Published in Expert Review of Neurotherapeutics, 2023
Raymond Wong, Nader Hejrati, Michael G. Fehlings
The design of randomized clinical trials (RCTs) in SCI must consider the limited pool of individuals with SCI. Furthermore, depending on the mechanism of the experimental therapy, only a subset of the SCI population would typically be eligible for enrollment. For instance, certain neuroregenerative therapies may only be relevant to chronic, acute, or subacute stages of SCI [3]. In general, to minimize potential confounding variables in clinical trials, the cohort of participating patients is controlled via strict definitions of inclusion and exclusion criteria, where the former are factors that permit someone to enroll in a study while the latter disqualifies the person from enrolling [5]. Thus, there is typically insufficient patient recruitment within a single trial site. Nonetheless, it should be noted that the sample size necessary to reach significant statistical power depends on the nature of the clinical trial. For example, a trial examining the chronic stage of injury would require fewer participants to detect potential significant effects from treatment in a trial; this is due to the fact that spontaneous neurological improvements would be minimal in patients with SCI over a chronic period of time, thus establishing a greater static functional baseline compared to the acute or subacute stages [5].
Lessons learned from the SARS-CoV-2 pandemic; from nucleic acid nanomedicines, to clinical trials, herd immunity, and the vaccination divide
Published in Expert Opinion on Drug Delivery, 2023
Hiba Hussain, Aishwarya Ganesh, Lara Milane, Mansoor Amiji
The clinical trials conducted by Pfizer/BioNTech on their vaccine candidates against SARS-CoV-2 followed an expedited process to qualify for emergency use authorization (EUA). Phase 1 trials were focused toward identifying the preferred vaccine candidate and dose levels, followed by Phase 2/3 trials that expanded the cohort and evaluated the vaccine efficacy. The vaccine candidate, BNT162b2 (30 µg), was selected for efficacy evaluation in Phase 2/3 trials. The primary outcome of the clinical trials focused on local/systemic/adverse events observed with varying frequency of doses and time frames. The secondary outcome focused on the antibody levels before and after vaccination with BNT162b2. Inclusion and exclusion criteria are part of all clinical trials, and include factors such as age, gender, comorbidities, and willingness to comply with interventional methods [53].