Investigational New Drug (IND) Applications: What You Need to Know for Successful Interactions with the FDA

An educational program of the NIH Clinical Center-FDA CDER Joint Task Force emphasizing regulatory requirements for clinical trials of new and repurposed drugs, with a special focus on rare diseases. Target Audience: Clinical investigators, clinical research study coordinators, clinical and translational research scientists and trainees. Objectives: • To provide training in FDA regulations for NIH investigators involved in phase I and phase II clinical trials of investigational drugs, emphasizing safety considerations in the early phases of drug development. • To describe the IND-enabling elements of non-clinical research that are required to implement a first-in-human clinical trial, including chemistry, manufacturing, and controls (CMC) conducted under good manufacturing practice (GMP) conditions, and non-clinical pharmacology and toxicology studies. • To address special considerations applicable to drug development for rare diseases, including limited number of patients and the need for well-designed, efficient, and informative phase I-II clinical trials to support further development of novel therapies for rare diseases. • To inform the NIH clinical research community about new web-based tools that will facilitate IND submissions. Air date: 12/12/2013 8:00:00 AM
Source: Videocast - All Events - Category: Journals (General) Tags: Upcoming Events Source Type: video

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Authors: Liu TY, Zhu YX, Ke PQ, He M, Liang YC, Yao SZ Abstract Unicornuate uterus is a rare disease characterized with reduced fertility, and ovarian tumor diagnosed during pregnancy is uncommon as well. These two diseases have been reported separately. However, patient suffering from both diseases has never been reported before. The authors herein report a case of a 32-year-old Chinese woman presenting with a unicornuate uterus with no horn, who suffered from acute abdominal pain and intra-abdominal hemorrhage at 26 weeks gestation. Incidentally, a borderline ovarian tumor (BOT) and rupture of uterus were found d...
Source: European Journal of Gynaecological Oncology - Category: OBGYN Tags: Eur J Gynaecol Oncol Source Type: research
by Manuel A. Rivas, Brandon E. Avila, Jukka Koskela, Hailiang Huang, Christine Stevens, Matti Pirinen, Talin Haritunians, Benjamin M. Neale, Mitja Kurki, Andrea Ganna, Daniel Graham, Benjamin Glaser, Inga Peter, Gil Atzmon, Nir Barzilai, Adam P. Levine, Elena Schiff, Nikolas Pontikos, Ben Weisburd, Monkol Lek, Konrad J. Karczewski, Jonathan Bloom, Eric V. Minikel, Britt-Sabina Petersen, Laurent Beaugerie, Philippe Seksik, Jacques Cosnes, Stefan Schreiber, Bernd Bokemeyer, Johannes Bethge, International IBD Genetics Consortium , NIDDK IBD Genetics Consortium , T2D-GENES Consortium , Graham Heap, Tariq Ahmad, Vincent Plagnol...
Source: PLoS Genetics - Category: Genetics & Stem Cells Authors: Source Type: research
Source: Internal and Emergency Medicine - Category: Emergency Medicine Source Type: research
Conclusion: Both cyanoacrylate injection and hemoclip placement are effective in treating duodenal DL, and neither of them causes significant side effects. PMID: 29780411 [PubMed]
Source: Gastroenterology Research and Practice - Category: Gastroenterology Tags: Gastroenterol Res Pract Source Type: research
Authors: Amano M, Shimizu T Abstract Mondor's disease (MD) is a rare disease that manifests with a palpable cord-like induration on the body surface. In general, MD is a self-limited, benign thrombophlebitis that resolves in four to eight weeks without any specific treatment. Cases of MD can be roughly categorized into three different groups based on the site of the lesion as follows: original MD of the anterolateral thoracoabdominal wall, penile MD with dorsum and dorsolateral aspects of the penis, and axillary web syndrome with mid-upper arm after axillary surgery. The diagnosis of MD is rather straightforward an...
Source: Internal Medicine - Category: Internal Medicine Tags: Intern Med Source Type: research
AbstractA gastric subepithelial tumor is a common finding during upper gastric endoscopy. The differential diagnosis of such lesions is broad, and sometimes a rare disease can be diagnosed. A calcifying fibrous tumor (CFT) is a rare, benign mesenchymal tumor that usually affects children and young adults. To our knowledge, a CFT of the tubular gastrointestinal tract is very rare with fewer than 30 cases of gastric CFTs reported in the literature.
Source: Journal of Gastrointestinal Surgery - Category: Surgery Source Type: research
NDA Partners Chairman Carl Peck, MD announced today that Bruce Green, D.Ph, an internationally recognized expert in the field of pharmacometrics, rare diseases, pediatric drug development, and dosing...(PRWeb May 23, 2018)Read the full story at http://www.prweb.com/releases/2018/05/prweb15499347.htm
Source: PRWeb: Medical Pharmaceuticals - Category: Pharmaceuticals Source Type: news
AbstractPurpose of ReviewAlthough Beh çet’s syndrome (BS) is classified as a rare disease in European countries and the USA, its neurologic involvement “neuro-Behçet’s syndrome (NBS)” is commonly included in the differential diagnosis of many inflammatory and vascular central nervous system (CNS) disorders. Clinical and neuroi maging findings support two major forms of NBS: parenchymal NBS (p-NBS) and an extra-parenchymal form that presents with cerebral venous sinus thrombosis (CVST). The present review summarizes recent findings on the etiopathogenesis and clinico-radiological featur...
Source: Current Neurology and Neuroscience Reports - Category: Neuroscience Source Type: research
The Undiagnosed Diseases Program (UDP), an initiative launched by NHGRI, the National Institutes of Health (NIH) Clinical Center and the NIH Office of Rare Disease Research, is celebrating 10 years of tackling the most challenging medical cases. The program kicked off in May 2008 with the mission of offering patients with perplexing conditions the hope of a diagnosis and the therapeutic treatments. Its unique strategy applies advanced genomic medicine technologies and multi-disciplinary medical expertise to unsolved health conditions. Happy 10th Birthday, UDP!
Source: NHGRI Homepage Highlights - Category: Genetics & Stem Cells Source Type: news
Publication date: June 2018 Source:Trends in Pharmacological Sciences, Volume 39, Issue 6 Author(s): Misty M. Attwood, Mathias Rask-Andersen, Helgi B. Schiöth High levels of productivity, with an increasing number of approvals for new molecular entities (NMEs) by the FDA during the past decade, have coincided with the emergence of innovative drugs for treatments of rare diseases that have utilized the FDA orphan drug program. Since 2000, NMEs with orphan designation encompass a significant portion of approved drugs and constitute about 80% of the approved drugs that have established novel human genome-encoded product...
Source: Trends in Pharmacological Sciences - Category: Drugs & Pharmacology Source Type: research
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