Patient Registries: An Underused Resource for Medicines Evaluation

ConclusionsThese are the first explicit proposals, from a regulatory perspective, on operational methods for increasing the use of patient registries in medicines regulation. They apply to registry holders, patients, regulators, marketing authorisation holders/applicants and healthcare stakeholders broadly, and their implementation would greatly facilitate the use of these valuable data sources in regulatory decision-making.
Source: Drug Safety - Category: Drugs & Pharmacology Source Type: research

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This is a rare disease in which the immune system attacks the brain and disrupts normal brain signaling. It first affected her ability to think clearly, but soon she was suffering from relentless seizures.
Source: WebMD Health - Category: Consumer Health News Source Type: news
             Basel, 23 January 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Safety for risdiplam in the FIREFISH study was con...
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news
Authors: Akagi S, Miki T, Sando Y, Fujii N, Sarashina T, Nakamura K, Ito H Abstract Chronic-active Epstein-Barr virus infection (CAEBV) is a rare disease that can lead to pulmonary arterial hypertension (PAH). However, the treatment for CAEBV-associated PAH has not been established. We discuss a case of improved pulmonary hypertension after chemotherapy in a patient with CAEBV-associated PAH. A 44-year old man was admitted to our hospital because of an abnormal electrocardiogram and liver dysfunction detected by annual medical examination. Echocardiography showed a dilated right ventricle and an estimated right ven...
Source: International Heart Journal - Category: Cardiology Tags: Int Heart J Source Type: research
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA) are co-hosting a Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies. Current adeno-associated virus (AAV) manufacturing capacity cannot keep pace with the demand for AAV gene therapy clinical trials. The purpose of the meeting is to bring together thought leaders, key stakeholders and innovators to explore obstacles and identify opportunities for expanding the capacity of AAV manufacturing f...
Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA) are co-hosting a Workshop on Expanding AAV Manufacturing Capacity for Rare Disease Gene Therapies. Current adeno-associated virus (AAV) manufacturing capacity cannot keep pace with the demand for AAV gene therapy clinical trials. The purpose of the meeting is to bring together thought leaders, key stakeholders and innovators to explore obstacles and identify opportunities for expanding the capacity of AAV manufacturing f...
Source: Videocast - All Events - Category: General Medicine Tags: Upcoming Events Source Type: video
Quest Diagnostics said it has acquired Blueprint Genetics in an all-cash equity transaction. Helsinki, Finland-based Blueprint Genetics has deep expertise in gene variant interpretation based on next-generation sequencing (NGS) and proprietary bioinformatics.  Additional financial terms were not disclosed.  Quest said the addition of Blueprint Genetics will help broaden access to actionable insights in genetic and rare diseases, improving patient care and pharmaceutical drug research and development. "The great challenge with genetic testing is generating quality, actionable and broadly accessi...
Source: MDDI - Category: Medical Devices Authors: Tags: IVD Source Type: news
Autoimmune pancreatitis (AIP) is a relatively newly discovered and rare disease that can be challenging to recognize and diagnose.1 Although a case report first described it in 1961, not until 1995 did it begin to be recognized as a clinical entity in a case series by Yoshida et  al.2 Type 1 AIP is the most common worldwide and is associated with IgG4-positive cells and histologic features described as lymphocytoplasmic sclerosing pancreatitis (LPSP) without granulocyte epithelial lesions, which are seen in type 2 AIP.
Source: Gastrointestinal Endoscopy - Category: Gastroenterology Authors: Tags: Original article Source Type: research
The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor.
Source: FDA Center for Drug Evaluation and Research - What's New - Category: Drugs & Pharmacology Authors: Source Type: news
Combined malonic and methylmalonic aciduria (CMAMMA) is an inborn error of metabolism which has been proposed being a benign condition. However, older patients may present with neurological manifestations such...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Letter to the Editor Source Type: research
To compare the similarity of the non-patented T2* and the high cost patented R2 (Ferriscan ®) MRI techniques in the measurement of liver iron concentration (LIC) in heavily transfused patients with thalassaemia...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Letter to the Editor Source Type: research
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