Leveraging Natural History Data in One- and Two-Arm Hierarchical Bayesian Studies of Rare Disease Progression

2021 ◽  
Author(s):  
Arnaud Monseur ◽  
Bradley P. Carlin ◽  
Bruno Boulanger ◽  
Andreea Seferian ◽  
Laurent Servais ◽  
...  
Keyword(s):  
Natural History ◽  
Disease Progression ◽  
Rare Disease ◽  
Hierarchical Bayesian ◽  
History Data

P24—Prognostic Indicators of Renal Disease Progression: Natural History Data From the Fabry Registry

2012 ◽  
Vol 34 (4) ◽  
pp. e27
Author(s):  
C. Wanner ◽  
J.P. Oliveira ◽  
A. Ortiz ◽  
M. Mauer ◽  
D.P. Germain ◽  
...  
Keyword(s):  
Natural History ◽  
Renal Disease ◽  
Disease Progression ◽  
Prognostic Indicators ◽  
Renal Disease Progression ◽  
History Data

scholarly journals Prognostic Indicators of Renal Disease Progression in Adults with Fabry Disease: Natural History Data from the Fabry Registry

2010 ◽  
Vol 5 (12) ◽  
pp. 2220-2228 ◽  
Author(s):  
Christoph Wanner ◽  
João P. Oliveira ◽  
Alberto Ortiz ◽  
Michael Mauer ◽  
Dominique P. Germain ◽  
...  
Keyword(s):  
Natural History ◽  
Renal Disease ◽  
Fabry Disease ◽  
Disease Progression ◽  
Prognostic Indicators ◽  
Renal Disease Progression ◽  
History Data

Clinical Outcome Study for Dysferlinopathy: Three years of natural history data for clinical trial readiness

2019 ◽  
Author(s):  
V Straub ◽  
MK James ◽  
AG Mayhew ◽  
S Spuler ◽  
JW Day ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Natural History ◽  
Clinical Outcome ◽  
Outcome Study ◽  
History Data ◽  
Clinical Outcome Study

scholarly journals Natural History of NAFLD

2021 ◽  
Vol 10 (6) ◽  
pp. 1161
Author(s):  
Raluca Pais ◽  
Thomas Maurel
Keyword(s):  
Liver Disease ◽  
Natural History ◽  
Disease Progression ◽  
Specific Treatment ◽  
Alcoholic Fatty Liver ◽  
Individual Level ◽  
Close Relationship ◽  
History Of ◽  
The Individual ◽  
The Impact

The epidemiology and the current burden of chronic liver disease are changing globally, with non-alcoholic fatty liver disease (NAFLD) becoming the most frequent cause of liver disease in close relationship with the global epidemics of obesity, type 2 diabetes and metabolic syndrome. The clinical phenotypes of NAFLD are very heterogeneous in relationship with multiple pathways involved in the disease progression. In the absence of a specific treatment for non-alcoholic steatohepatitis (NASH), it is important to understand the natural history of the disease, to identify and to optimize the control of factors that are involved in disease progression. In this paper we propose a critical analysis of factors that are involved in the progression of the liver damage and the occurrence of extra-hepatic complications (cardiovascular diseases, extra hepatic cancer) in patients with NAFLD. We also briefly discuss the impact of the heterogeneity of the clinical phenotype of NAFLD on the clinical practice globally and at the individual level.

scholarly journals The Use of External Controls in FDA Regulatory Decision Making

2021 ◽  
Author(s):  
Mahta Jahanshahi ◽  
Keith Gregg ◽  
Gillian Davis ◽  
Adora Ndu ◽  
Veronica Miller ◽  
...  
Keyword(s):  
Decision Making ◽  
Natural History ◽  
Product Development ◽  
Rare Diseases ◽  
The United States ◽  
External Control ◽  
Common Source ◽  
Published Data ◽  
History Data ◽  
Benefit Risk Assessment

AbstractThe regulatory standards of the United States Food and Drug Administration (FDA) require substantial evidence of effectiveness from adequate and well-controlled trials that typically use a valid comparison to an internal concurrent control. However, when it is not feasible or ethical to use an internal control, particularly in rare disease populations, relying on external controls may be acceptable. To better understand the use of external controls to support product development and approval, we reviewed FDA regulatory approval decisions between 2000 and 2019 for drug and biologic products to identify pivotal studies that leveraged external controls, with a focus on select therapeutic areas. Forty-five approvals were identified where FDA accepted external control data in their benefit/risk assessment; they did so for many reasons including the rare nature of the disease, ethical concerns regarding use of a placebo or no-treatment arm, the seriousness of the condition, and the high unmet medical need. Retrospective natural history data, including retrospective reviews of patient records, was the most common source of external control (44%). Other types of external control were baseline control (33%); published data (11%); and data from a previous clinical study (11%). To gain further insights, a comprehensive evaluation of selected approvals utilizing different types of external control is provided to highlight the variety of approaches used by sponsors and the challenges encountered in supporting product development and FDA decision making; particularly, the value and use of retrospective natural history in the development of products for rare diseases. Education on the use of external controls based on FDA regulatory precedent will allow for continued use and broader application of innovative approaches to clinical trial design, while avoiding delays in product development for rare diseases. Learnings from this review also highlight the need to update regulatory guidance to acknowledge the utility of external controls, particularly retrospective natural history data.

scholarly journals Developing a vocabulary and ontology for modeling insect natural history data: example data, use cases, and competency questions

2019 ◽  
Vol 7 ◽  
Author(s):  
Brian Stucky ◽  
James Balhoff ◽  
Narayani Barve ◽  
Vijay Barve ◽  
Laura Brenskelle ◽  
...  
Keyword(s):  
Natural History ◽  
Large Scale ◽  
Use Cases ◽  
Data Systems ◽  
Multiple Sources ◽  
History Data ◽  
Insect Ecology ◽  
Many Sources ◽  
Multicellular Organisms ◽  
Initial Results

Insects are possibly the most taxonomically and ecologically diverse class of multicellular organisms on Earth. Consequently, they provide nearly unlimited opportunities to develop and test ecological and evolutionary hypotheses. Currently, however, large-scale studies of insect ecology, behavior, and trait evolution are impeded by the difficulty in obtaining and analyzing data derived from natural history observations of insects. These data are typically highly heterogeneous and widely scattered among many sources, which makes developing robust information systems to aggregate and disseminate them a significant challenge. As a step towards this goal, we report initial results of a new effort to develop a standardized vocabulary and ontology for insect natural history data. In particular, we describe a new database of representative insect natural history data derived from multiple sources (but focused on data from specimens in biological collections), an analysis of the abstract conceptual areas required for a comprehensive ontology of insect natural history data, and a database of use cases and competency questions to guide the development of data systems for insect natural history data. We also discuss data modeling and technology-related challenges that must be overcome to implement robust integration of insect natural history data.

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