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Drug evaluation in children
Published in Evelyne Jacqz-Aigrain, Imti Choonara, Paediatric Clinical Pharmacology, 2021
Evelyne Jacqz-Aigrain, Imti Choonara
The US drug regulations and procedures are federal, and thus procedures may correspond broadly to the centralised EU procedures. However, the number of countries involved in the EU and different backgrounds of drug regulation still constitute a major difference between the two systems. Although the ICH process has led to the harmonisation of many drug development requirements (see Chapter 1.4), significant differences still exist, like the existence of special regulations for paediatric medicinal products at FDA level since 1997 (see Chapter 1.3). In the US a new medicinal product must enter the regulatory arena earlier in the form of an Investigational New Drug application (IND), and can be developed in a more binding collaboration between the sponsor and the regulatory authorities. It is outside the scope of this review to go to any detail, for more information see: http://www.fda.gov/cder/index.html.
Legislators and Legislation
Published in Mickey C. Smith, E.M. (Mick) Kolassa, Walter Steven Pray, Government, Big Pharma, and the People, 2020
Mickey C. Smith, E.M. (Mick) Kolassa, Walter Steven Pray
Incentives provided to the Drug company under the Orphan Drug Act were these: Exclusive marketing rights for 7 years, after approval of the Drug, and this applies whether or not the Drug is patented.Tax credits for clinical testing.Grants and contracts to support orphan Drug research.Flexibility and assistance in the regulating processes. This includes authorization to physicians to administer the Drug prior to approval to patients even if they are not involved in clinical trials – a “compassionate” IND.
Role of the FDA in the Clinical Research Process
Published in Gary M. Matoren, The Clinical Research Process in the Pharmaceutical Industry, 2020
Currently, pharmaceutical company sponsors must submit an IND prior to the first Phase I study. The INDs must contain detailed information on animal pharmacology and toxicology and the proposed Phase I clinical trials, as well as early information on manufacturing controls. As the studies progress, each new clinical protocol must be submitted prior to inception of the study.
COR388 (atuzaginstat): an investigational gingipain inhibitor for the treatment of Alzheimer disease
Published in Expert Opinion on Investigational Drugs, 2022
Marwan N. Sabbagh, Boris Decourt
Exposures based on animal models and mechanism of action suggest that the optimal dosing of COR388 in humans is twice daily. Investigational new drug–enabled phase 1 and phase 2/3 clinical trials have been completed. In the phase 1 trial (ClinicalTrials.gov: NCT03418688), COR388 was well tolerated without severe adverse events in the single ascending dose, in the 10-day multiple ascending dose in healthy elderly patients, and in the 28-day study in AD patients. Drug-related treatment-emergent adverse event rates were similar between placebo recipients (20%) and COR388-treated patients (14%); the only drug-related adverse event that occurred was dizziness (twice, once at 50 mg and once at 100 mg). No dose-limiting toxicity was identified. No clinically significant trends were observed on laboratory tests or electrocardiograms, blood pressure, heart rate, or temperature [49].
Pharmaceutical cocrystal: a game changing approach for the administration of old drugs in new crystalline form
Published in Drug Development and Industrial Pharmacy, 2020
Prabhakar S. Panzade, Giridhar R. Shendarkar
United Food and Drug Administration (USFDA) and European Medicines Agency (EMA) had issued regulatory guidance to the pharmaceutical industry on pharmaceutical cocrystal. Recent definition and other comparative parameters of cocrystal given by USFDA and EMA are summarized in Table 1. Prior to any clinical trial of cocrystal based products in humans, the drug developer must first obtain preauthorization from the FDA for the use of the cocrystal APIs in clinical trials. This process begins by submitting an investigational new drug (IND) application with the FDA. The IND application provides the safety profile of the product and manufacturing information. The application process also comprises an in-depth protocol for the conduct of every proposed clinical trial. The FDA reviews the IND application to make sure the safety and effectiveness of the proposed use of the drug and therefore the adequacy of the manufacturing method. The USFDA accepts the new drug applications on cocrystal if applicants provide evidence on the dissociation of a drug from cocrystal before reaching the site of action. Nangia and coworkers revealed the dissociation of temozolamide from succinic acid cocrystal [18].
Balancing Scientific Progress With Pediatric Protections: No Direct Benefit Now, But Potential Novel Therapy in the Future
Published in The American Journal of Bioethics, 2020
Susannah W. Lee, Jessica C. Ginsberg
Generally, investigators who study new drugs must submit an IND application and receive approval from the U.S. Food and Drug Administration (Center for Drug Evaluation and Research 2015). However, clinical investigations of already FDA approved drugs may be exempt from IND requirements if all criteria for an exemption per 21 CFR §312.2(b) are met, which are (1) the drug is lawfully marketed in the U.S.; (2) the investigator does not intend to report to FDA for a new indication or significant change in labeling, or (3) advertising; (4) the investigation does not involve a route of administration, dose, patient population, or other factor that significantly increases the associated risk; (5) the investigation complies with informed consent requirements and the IRB process; and (6) the investigation does not promote the drug as safe or effective for the purposes for which it is under investigation (Food and Drug Administration 2019, 21 CFR §312.2(b)).