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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A clinical-stage pharmaceutical firm with Duke University roots has filed paperwork to go public in an $86.3 million offering – one that could mean a windfall for a Triangle investor. PhaseBio Pharmaceuticals, which spun out of Duke University in 2002 and counts Durham's Hatteras Venture Partners as a backer, relocated its headquarters from Durham to Pennsylvania in 2010. Late Friday, the company, which focuses on trea ting rare diseases – particularly in the cardiopulmonary space – filed…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
Authors: Di Prete M, Ferlosio A, Mazzilli S, Bianchi L, Orlandi A, Campione E PMID: 30229639 [PubMed - as supplied by publisher]
Source: Giornale Italiano di Dermatologia e Venereologia - Category: Dermatology Tags: G Ital Dermatol Venereol Source Type: research
With advances in gene technology helping to diagnose very rare diseases, has the new era of personalised medicine finally arrived?Evie Walker sits on her mother ’s lap, playing a game she never grows tired of: turning her mother’s hand over and over, stroking and examining it. When she takes a break and looks around, it is with the open-mouthed look of curiosity and awe that you see in many infants. Evie’s vocabulary currently consists of a repertoire of squawks and “mmm” sounds. In the past few months, she has begun to stand unaided for short periods – even taking a few steps in her wal...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Genetics Health Society Politics Children Cancer Cancer research Medical research Science Biology Source Type: news
IJERPH, Vol. 15, Pages 2072: Meeting Patients’ Right to the Correct Diagnosis: Ongoing International Initiatives on Undiagnosed Rare Diseases and Ethical and Social Issues International Journal of Environmental Research and Public Health doi: 10.3390/ijerph15102072 Authors: Sabina Gainotti Deborah Mascalzoni Virginie Bros-Facer Carlo Petrini Giovanna Floridia Marco Roos Marco Salvatore Domenica Taruscio The time required to reach a correct diagnosis is a key concern for rare disease (RD) patients. Diagnostic delay can be intolerably long, often described as an “odyssey” and, ...
Source: International Journal of Environmental Research and Public Health - Category: Environmental Health Authors: Tags: Review Source Type: research
Hinman Syndrome is a rare disease with urodynamic findings and clinical course that resemble neuropathic bladder and no neuropathic etiology. Diffusion Tensor Imaging (DTI) is a special magnetic resonance imaging (MRI) technique that has recently been used for peripheral nerves but shown to be applicable for evaluation of lumbosacral plexus. Our aim was to evaluate the lumbosacral plexus using DTI, which was not previously performed in Hinman Syndrome.
Source: The Journal of Urology - Category: Urology & Nephrology Authors: Source Type: research
Authors: Marchioni M, Berardinelli F, De Nunzio C, Spiess P, Porpiglia F, Schips L, Cindolo L Abstract Penile cancer is a rare disease. Most of penile cancer are squamous cell carcinoma. Diagnosis is based on self-examination, clinical examination and confirmatory biopsy. Several imaging technique could be used for staging purpose. However, the best modality for staging in intermediate and high risk patients is by surgical evaluation and the use of inguinal lymph node dissection, that has also a therapeutic effect. Unfortunately, inguinal lymph node dissection is underused. Penile cancer treatment may have a major ...
Source: Minerva Urologica e Nefrologica - Category: Urology & Nephrology Tags: Minerva Urol Nefrol Source Type: research
Hug shirts, smellphones, virtual tastes, bionic eyes and hearing aids doing translations – just a few keywords showing how technology will take human perception to a higher level in the future. Innovative healthcare solutions will go way beyond improving our senses when we experience problems, they will augment our capabilities and open new horizons for humanity. Let’s jump into the pool of details. How humans perceive the sensory cacophony called the world Car. Flower. Smartphone. Leaf. Shadow. Ponytail. Red Sweater. Monitor. Water. Coffee. Beeps. Sidney Bechet tunes. Bicycle. Laugh. Light breeze. Holiday mem...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Biotechnology Cyborgization Health Sensors & Trackers Medical Professionals Patients body augmentation future Healthcare hearing human human perception Medicine sense sensing smell taste touch vision Source Type: blogs
Contributors : Chen Weng ; Fulai Jin ; Leina LuSeries Type : Expression profiling by high throughput sequencing ; Genome binding/occupancy profiling by high throughput sequencingOrganism : Homo sapiens ; Mus musculusPancreatic endocrine cells orchestrate the precise control of blood glucose levels, but the contribution of each cell type to diabetes or obesity remains elusive. Here we used a massively parallel single-cell RNA-seq technology (Drop-Seq) to analyze the transcriptome of 26,677 pancreatic islets cells from both healthy and type II diabetic (T2D) donors. We have analyzed cell type-specific gene signatures, and de...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Genome binding/occupancy profiling by high throughput sequencing Homo sapiens Mus musculus Source Type: research
Cherubism is a rare autosomal dominant disorder of the jaws caused by mutation of the SH3BP2 gene. The bone is replaced by a fibrous granuloma containing multinucleated giant cells. Cells of the cherubism granulo...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Noonan syndrome-like disorder with loose anagen hair-2 (NSLH2) is an extremely rare disease caused by a heterozygous mutation in the PPP1CB gene on chromosome 2p23. The syndrome causes not only numerous dysmor...
Source: BMC Neurology - Category: Neurology Authors: Tags: Case report Source Type: research
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