Результаты поиска

Reshaping study design for faster extrapolation-based drug approval in pediatric inflammatory bowel diseases: An ESPGHAN-NASPGHAN position paper.

Children with inflammatory bowel diseases (IBD) have limited access to the available advanced therapies, given the lengthy gap between adult and pediatric approval. We aimed to review key hurdles for pediatric trials and recommend practical solutions. This position paper was developed jointly by the European and North American Societies for Pediatric Gastroenterology Hepatology and Nutrition (ESPGHAN and NASPGHAN), in consultation with patient representatives. A systematic review was performed for identified topics, and two voting rounds with two group meetings led to agreement on 24 statement

Model-Informed Drug Development-Based Bridging from Subcutaneous to Intravenous Secukinumab Dosing: Approval in Psoriatic Arthritis and Axial Spondyloarthritis.

The objective of this modeling and simulation analysis was to determine an intravenous (IV) secukinumab dosing regimen with steady-state exposure within the ranges of the approved subcutaneous (SC) regimens (300 and 150 mg every 4 weeks [q4w]) and to predict the efficacy and safety of this IV regimen for patients with psoriatic arthritis (PsA) or axial spondyloarthritis (axSpA). This approach was suggested by the US Food and Drug Administration (FDA) following review of the primary endpoint analysis results of secukinumab 3 mg/kg q4w IV in patients with PsA (INVIGORATE-2 study). Noting the hig

Drug Review and Approval Policies Based on Real-world Evidence in China and the United States: A Comparative Study.

PURPOSE: The use of real-world evidence (RWE) in regulatory reviews and approvals is currently experiencing significant changes amid increasingly active discussions, primarily reflected in relevant policies, regulations, and guidance documents. However, disparities persist between China and the United States regarding the acceptance and formulation of policies for incorporating real-world data/evidence (RWD/E) in regulatory evaluation and authorization. Furthermore, the current policies lack specific operational details necessary for effective implementation and widespread adoption. METHODS:

Eyes on the Prize: Decoding the Ophthalmic Product Regulations and Intricacies of the U.S. Food and Drug Administration Approval.

The dynamic and continuously evolving field of ophthalmology necessitates rigorous regulatory oversight in the United States. This review outlines the multifaceted Food and Drug Administration's (FDA) approval process for ophthalmic products, detailing the classifications, pathways, and regulatory compliance for devices, drugs, biologics, and combination products. Particular emphasis is placed on distinct frameworks for Class I, II, and III devices, as well as regulations for drugs, biologics, and combination products. The organizational structure of the FDA is detailed, with highlights on spe

U.S. Food and Drug Administration Approval: Neratinib for the Extended Adjuvant Treatment of Early-Stage HER2-Positive Breast Cancer.

On July 17, 2017, the FDA approved neratinib (NERLYNX; Puma Biotechnology, Inc.) for the extended adjuvant treatment of adult patients with early-stage HER2-overexpressed/amplified breast cancer, to follow adjuvant trastuzumab-based therapy. Approval was based on data from ExteNET, a randomized, double-blind, placebo-controlled multicenter trial. Women with early-stage HER2-positive breast cancer and within 2 years of completing adjuvant trastuzumab were randomized to neratinib (n = 1,420) or placebo (n = 1,420) for 1 year. The primary endpoint was invasive disease-free survival (iDFS), define

Food and Drug Administration and Institutional Review Board Approval of a Novel Prehospital Informed Consent Process for Emergency Research.

Research on the management of acute pain in the prehospital setting is fraught with challenges. The prehospital setting is complex due to constrained time, resources, and training. Research activities must not interfere with the underlying clinical priorities of immediate patient stabilization and rapid transport to an appropriate hospital. The patient's pain, fear, and anxiety immediately after a traumatic event may interfere with undertaking an adequate informed consent process.Pain management trials do not satisfy the criteria for application of the U.S. Food and Drug Administration (FDA) 2

Model-Informed Drug Development Approach Supporting Approval of Adalimumab (HUMIRA) in Adolescent Patients with Hidradenitis Suppurativa: a Regulatory Perspective.

On October 16, 2018, FDA expanded the adalimumab dosing regimen to adolescent hidradenitis suppurativa (HS) patients 12 years and older, weighing at least 30 kg without new clinical data. This approval was mainly supported by the model-informed drug development approach. Population pharmacokinetic simulations suggest body weight-tiered dosing regimens in adolescent patients will achieve similar exposure to adult patients across all weight range. Adalimumab has a well-established, 16-year long-term safety profile in various diseases in adult and pediatric populations. Current data of adalimumab

U.S. Food and Drug Administration approval summary: Eltrombopag for the treatment of pediatric patients with chronic immune (idiopathic) thrombocytopenia.

The U.S. Food and Drug Administration (FDA) approved eltrombopag for pediatric patients with chronic immune (idiopathic) thrombocytopenia (ITP) ages ≥6 on June 11, 2015, and ages ≥1 on August 24, 2015. Approval was based on the FDA review of two randomized trials that included 159 pediatric patients with chronic ITP who had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. This manuscript describes the basis for approval of these applications. The FDA concluded that eltrombopag has shown efficacy and a favorable benefit to risk profile for pediatric patients with ch

Clinical benefit, price and epidemiology of orphan drugs approved in China: a cross-sectional analysis of orphan drug policy (ODP) in China.

OBJECTIVES: To evaluate the regulatory approval, epidemiology, clinical benefits and pricing of orphan drugs approved by the National Medical Products Administration (NMPA) in China. DESIGN: Cross-sectional analysis. STUDY SAMPLE: Orphan drugs approved by the NMPA for rare disease indications between 1 January 2005 and 31 December 2022. OUTCOME MEASURES: The outcomes included regulatory approval lag time, National Reimbursement Drug List (NRDL) coverage, monthly treatment costs, clinical benefit (SMR, Service Médical Rendu) and additional therapeutic value (ASMR, Amélioration du Service Méd

Computational prognostic evaluation of Alzheimer's drugs from FDA-approved database through structural conformational dynamics and drug repositioning approaches.

Drug designing is high-priced and time taking process with low success rate. To overcome this obligation, computational drug repositioning technique is being promptly used to predict the possible therapeutic effects of FDA approved drugs against multiple diseases. In this computational study, protein modeling, shape-based screening, molecular docking, pharmacogenomics, and molecular dynamic simulation approaches have been utilized to retrieve the FDA approved drugs against AD. The predicted MADD protein structure was designed by homology modeling and characterized through different computation

Assessing adverse drug reaction reports for antidiabetic medications approved by the food and drug administration between 2012 and 2017: a pharmacovigilance study.

OBJECTIVE: Between 2012 and 2017, the U.S. Food and Drug Administration (FDA) approved 10 antidiabetic indicated therapies. Due to the limited literature on voluntarily reported safety outcomes for recently approved antidiabetic drugs, this study investigated adverse drug reactions (ADRs) reported in the FDA Adverse Event Reporting System (FAERS). RESEARCH DESIGN AND METHODS: A disproportionality analysis of spontaneously reported ADRs was conducted. FAERS reports from January 1, 2012 to March 31, 2022 were compiled, allowing a 5-year buffer following drug approval in 2017. Reporting odds rat…

The Food and Drug Administration-approved antipsychotic drug trifluoperazine, a calmodulin antagonist, inhibits viral replication through PERK-eIF2α axis.

Virus-related diseases are seriously threatening human health, but there are currently only 10 viruses with clinically approved antiviral drugs available. As non-cellular organisms, viruses parasitize in living cells and rely on the protein synthesis mechanism of the host cells. In this study, we found that the antipsychotic drug trifluoperazine (TFP), a dual dopamine receptor D2 (DRD2)/calmodulin (CALM) antagonist, increases the phosphorylation of eukaryotic initiation factor 2α (eIF2α), a key factor in the regulation of protein synthesis and significantly inhibits vesicular stomatitis virus

Evaluating Food and Drug Administration approved gastrointestinal cancer drugs: clinical benefit and trial endpoints over the past two decades.

BACKGROUND: The increasing reliance on accelerated approvals and surrogate endpoints for Food and Drug Administration (FDA) approvals of gas … roportion meeting ESMO-MCBS substantial benefit (score ≥ 4). RESULTS: The FDA granted 60 GI cancer drug approvals from 67 trials. Approvals rose from 15 (25%) in 2006-2014 to 45 (75%) in 2015-2025. Single-arm trials increased to 24%, and surrogate endpoints were used in 41.8% (ORR 20.9%, PFS 19.4%). Median OS improvement was 2.1 months (IQR: 1.6-2.65). Only 24 …

Identification of approved drugs with ALDH1A1 inhibitory potential aimed at enhancing chemotherapy sensitivity in cancer cells: an in-silico drug repurposing approach.

The aldehyde dehydrogenase 1A1 (ALDH1A1) also known as retinal dehydrogenase, is an enzyme normally involved in the cellular metabolism, development and detoxification processes in healthy cells. However, it's also considered a cancer stem cell marker and its high levels of expression in several cancers, including breast, lung, ovarian, and colon cancer have been associated with poor prognosis and resistance to chemotherapy. Given its crucial role in chemotherapy resistance by detoxification of chemotherapeutic drugs, ALDH1A1 has attracted significant research interest as a potential therapeut

Ex vivo and In vitro antiplasmodial activities of approved drugs predicted to have antimalarial activities using chemogenomics and drug repositioning approach.

High malaria mortality coupled with increased emergence of resistant multi-drug resistant strains of Plasmodium parasite, warrants the development of new and effective antimalarial drugs. However, drug design and discovery are costly and time-consuming with many active antimalarial compounds failing to get approved due to safety reasons. To address these challenges, the current study aimed at testing the antiplasmodial activities of approved drugs that were predicted using a target-similarity approach. This approach is based on the fact that if an approved drug used to treat another disease ta

Repurposing of FDA-approved drugs against active site and potential allosteric drug-binding sites of COVID-19 main protease.

The novel coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) still has serious negative effects on health, social life, and economics. Recently, vaccines from various companies have been urgently approved to control SARS-CoV-2 infections. However, any specific antiviral drug has not been confirmed so far for regular treatment. An important target is the main protease (Mpro ), which plays a major role in replication of the virus. In this study, Gaussian and residue network models are employed to reveal two distinct potential allosteric sit

Racial/Ethnic Disparities in Ophthalmology Clinical Trials Resulting in US Food and Drug Administration Drug Approvals From 2000 to 2020.

IMPORTANCE: Diverse, representative enrollment in pivotal clinical trials is vital to sufficiently power subgroup analyses and ensure equity and validity of trial results. OBJECTIVE: To evaluate the racial/ethnic representation, trends, and disparities in clinical trials leading to US Food and Drug Administration (FDA) ophthalmology drug approvals from 2000 to 2020. DESIGN, SETTING, AND PARTICIPANTS: This cohort study used data from participants in clinical trials of drugs for neovascular age-related macular degeneration (AMD), open-angle glaucoma (OAG), and expanded indications for diabetic…

Impact of Japanese Subpopulation Data and Overall Survival in Pivotal Trials on Oncology Drug Approvals in Japan.

Globalization of oncology drug development has accelerated the adoption of multiregional clinical trials (MRCTs) as pivotal evidence for regulatory approvals in major markets, including Japan. While MRCTs enhance efficiency and generalizability, they introduce challenges related to inter-ethnic variability and data extrapolation. Japan's regulatory framework, guided by pharmaceuticals medical devices agency (PMDA) principles and the "Guideline for Clinical Evaluation Methods of Anticancer Drugs," emphasizes patient efficacy, safety and relevance, historically requiring local data to supplement

Совпадение в полном тексте источника
… Inclusion Criteria for Research The scope of this research encompassed all oncology drugs approved by the PMDA from FY 2020 to FY 2024 (i.e., April 2020–March 2025). … Fig. 1 Flowchart of new oncology drug approval identification in Japan between FY 2020 and FY 2024. …