Cargando…

Randomized controlled trial data for successful new drug application for rare diseases in the United States

BACKGROUND: Randomized controlled trial (RCT) data have important implications in drug development. However, the feasibility and cost of conducting RCTs lower the motivation for drug development, especially for rare diseases. We investigated the potential factors associated with the need for RCTs in...

Descripción completa

Detalles Bibliográficos
Autores principales:	Kubota, Yosuke, Narukawa, Mamoru
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2023
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10114466/ https://www.ncbi.nlm.nih.gov/pubmed/37076897 http://dx.doi.org/10.1186/s13023-023-02702-9

Ejemplares similares

A Comparison of Safety Information in Drug Labeling at the Initial Approval of New Drugs Approved Both in Japan and the United States
por: Hoshino, Yuko, et al.
Publicado: (2022)

Approval success rates of drug candidates based on target, action, modality, application, and their combinations
por: Yamaguchi, Shingo, et al.
Publicado: (2021)

Response rate of anticancer drugs approved by the Food and Drug Administration based on a single-arm trial
por: Oda, Yoshihiro, et al.
Publicado: (2022)

Factors associated with successful phase III trials for solid tumors: A systematic review
por: Otsuka, Yasushi, et al.
Publicado: (2021)

Impact of the Economic Status of the Patient’s Country of Residence on the Outcome of Oncology Clinical Trials
por: Nishiyama, Saki, et al.
Publicado: (2022)

Anti-Tumor Efficacy of Anti-PD-1/PD-L1 Antibodies in Combination With Other Anticancer Drugs in Solid Tumors: A Systematic Review and Meta-Analysis
por: Inoue, Takashi, et al.
Publicado: (2022)

Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs
por: Maeda, Kojiro, et al.
Publicado: (2017)

Factors affecting the association between overall survival and progression-free survival in clinical trials of first-line treatment for patients with advanced non-small cell lung cancer
por: Aboshi, Masayuki, et al.
Publicado: (2014)

Characteristics of safety information obtained from postmarketing observational studies for re-examination in Japan
por: Watanabe, Tatsuya, et al.
Publicado: (2016)

Current state of therapeutic development for rare cancers in Japan, and proposals for improvement
por: Kawai, Akira, et al.
Publicado: (2018)

Publication of Clinical Trials Supporting Successful New Drug Applications: A Literature Analysis
por: Lee, Kirby, et al.
Publicado: (2008)

Lower-dose prescriptions in the post-marketing situation and the influencing factors thereon
por: Ogata, Akiko, et al.
Publicado: (2019)

Factors Affecting Success of New Drug Clinical Trials
por: Kim, Eungdo, et al.
Publicado: (2023)

Consumer Understanding, Preferences, and Responses to Different Versions of Drug Safety Messages in the United States: A Randomized Controlled Trial
por: McCormack, Lauren, et al.
Publicado: (2015)

Comparing different models to forecast the number of mass shootings in the United States: An application of forecasting rare event time series data
por: Lei, Xue, et al.
Publicado: (2023)

Successful nutritional control of scratching and clinical signs associated with adverse food reaction: A randomized controlled COSCAD'18 adherent clinical trial in dogs in the United States
por: Weemhoff, James L., et al.
Publicado: (2021)

Perspectives on Anaphylaxis Epidemiology in the United States with New Data and Analyses
por: Mulla, Zuber D., et al.
Publicado: (2010)

Success of High Tibial Osteotomy in the United States Military
por: Waterman, Brian R., et al.
Publicado: (2015)

A comparison of success rates of introduced passeriform birds in New Zealand, Australia and the United States
por: Moulton, Michael P., et al.
Publicado: (2014)

Response to letter regarding “Successful nutritional control of scratching and clinical signs associated with adverse food reaction: A randomized controlled COSCAD'18 adherent clinical trial in the United States” and “Successful nutritional control of scratching and clinical signs associated with adverse food reaction: A randomized controlled COSCAD'18 adherent clinical trial in the United Kingdom”
por: Weemhoff, James L., et al.
Publicado: (2021)

Non-additive measures theory and applications
por: Torra, Vicenc, et al.
Publicado: (2014)

Exploratory Analysis of the Factors Associated With Success Rates of Confirmatory Randomized Controlled Trials in Cancer Drug Development
por: Wu, Can, et al.
Publicado: (2020)

Data on breastfeeding and state policies in the United States
por: Chen, Chun, et al.
Publicado: (2021)

New Population of the Rare Dragonfly Ophiogomphus howei (Odonata: Gomphidae) in Southern Michigan, United States
por: Craves, Julie A, et al.
Publicado: (2020)

Drug reimportation practices in the United States
por: Bhosle, Monali J, et al.
Publicado: (2007)

A Rare Manifestation of Tuberculosis Presenting in the United States
por: Bhatty, Osman, et al.
Publicado: (2016)

Application of Inertial Measurement Units and Machine Learning Classification in Cerebral Palsy: Randomized Controlled Trial
por: Khaksar, Siavash, et al.
Publicado: (2021)

Social support for exercise from pregnancy to postpartum and the potential impact of a mobile application: A randomized control pilot trial in Southern United States
por: Perera, Madhawa, et al.
Publicado: (2023)

Randomized controlled comparative study on effect of training to improve lower limb motor paralysis in convalescent patients with post-stroke hemiplegia
por: Kawakami, Kenji, et al.
Publicado: (2015)

Adjusting health spending for the presence of comorbidities: an application to United States national inpatient data
por: Dieleman, Joseph L., et al.
Publicado: (2017)

A Bayesian Hierarchical Spatial Model to Correct for Misreporting in Count Data: Application to State-Level COVID-19 Data in the United States
por: Chen, Jinjie, et al.
Publicado: (2022)

Randomized experimental testing of new survey approaches to improve abortion reporting in the United States
por: Lindberg, Laura D., et al.
Publicado: (2022)

The United States Food and Drug Administration and Prescription Drug Promotion
por: Kalola, Ankur S., et al.
Publicado: (2019)

Francisella tularensis in the United States
por: Farlow, Jason, et al.
Publicado: (2005)

Comparing Methods of Recruiting Spanish-Preferring Smokers in the United States: Findings from a Randomized Controlled Trial
por: Medina-Ramirez, Patricia, et al.
Publicado: (2020)

Scientific opportunities with a rare-isotope facility in the United States
por: Council, National Research, et al.
Publicado: (2007)

The national economic burden of rare disease in the United States in 2019
por: Yang, Grace, et al.
Publicado: (2022)

Burden of Prescription Drug Costs in the United States
por: Curtiss, Frederic R.
Publicado: (2003)

Assessment of the Level of Satisfaction and Unmet Data Needs for Specialty Drug Formulary Decisions in the United States
por: Choi, Yoonyoung, et al.
Publicado: (2016)

Prevention of Traumatic Brain Injury in the United States: Significance, New Findings, and Practical Applications
por: Fatuki, Tolulope A, et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_mgh68ekh3jc2pq1vb1mbk5r6b8