Related Experiment Video
Updated: Jan 23, 2026
15:48
ALS - Motor Neuron Disease: Mechanism and Development of New Therapies
Published on: July 29, 2007
10.0K
ALS drug development guidances and trial guidelines: Consensus and opportunities for alignment
Jinsy A Andrews1, Lucie I Bruijn2, Jeremy M Shefner2
1From the Eleanor and Lou Gehrig ALS Center (J.A.A.), Columbia University, New York, NY; ALS Association (L.I.B.), Washington, DC; and Department of Neurology (J.M.S.), Barrow Neurological Institute, University of Arizona College of Medicine, Phoenix. Dr. Bruijn is currently at Bruijn-Yard Neuroscience R&D Consultant. Ja2289@cumc.columbia.edu.
Neurology
|June 8, 2019
Summary
The US Food and Drug Administration (FDA) issued draft guidance for amyotrophic lateral sclerosis (ALS) drug development, incorporating community input to improve clinical trial efficiency and accelerate new treatments.
Area of Science:
- Neurology
- Drug Development
- Clinical Research
Background:
- The US Food and Drug Administration (FDA) released draft guidance for amyotrophic lateral sclerosis (ALS) drug development in February 2018.
- This guidance incorporated extensive input from the ALS community, including researchers, industry, advocacy groups, patients, and caregivers.
Purpose of the Study:
- To analyze the alignment between the FDA's draft guidance, community recommendations, and revised ALS Clinical Trial Guidelines.
- To identify opportunities for enhancing the efficiency of drug development in ALS.
Main Methods:
- Comparative analysis of three key documents: FDA draft guidance, ALS community recommendations, and updated ALS Clinical Trial Guidelines.
- Discussion of areas of agreement and divergence among the documents.
Main Results:
- Identified areas of consensus and opportunities for improving ALS drug development efficiency.
- Highlighted the need for further research in biomarkers, efficacy endpoints, and predictive algorithms.
Conclusions:
- Greater alignment among stakeholders can be achieved through continued research and patient engagement.
- Incorporating patient experience data throughout the drug development process is crucial for facilitating the approval of new ALS treatments.
Related Concept Videos
01:16Clinical Trials
10.2K
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
There are four phases in a clinical trial. A phase one...
10.2K
01:11Clinical Trials: Overview
4.7K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
4.7K
01:12Trial and Error and Algorithm
403
A problem-solving strategy is a plan of action used to find a solution. Different strategies have distinct action plans. Trial and error involves trying different solutions until one works. For instance, to fix a broken printer, you might check ink levels, ensure the paper tray isn't jammed, and verify the printer's connection to your laptop. This method can be time-consuming but is commonly used. Thomas Edison, for example, used trial and error to find a suitable filament for the light...
403
01:26Guidelines for Nursing Documentation II
1.6K
Effective documentation is an integral part of nursing practice. Here are some essential guidelines to follow when documenting patient care:
Timely documentation is crucial to ensure continuity of care for patients. Any delays in recording or reporting medical information can result in medical errors and even adverse patient outcomes. From medication administration to diagnostic test results, every detail must be accurately and promptly documented to provide the best possible care for patients.
Timely documentation is crucial to ensure continuity of care for patients. Any delays in recording or reporting medical information can result in medical errors and even adverse patient outcomes. From medication administration to diagnostic test results, every detail must be accurately and promptly documented to provide the best possible care for patients.
1.6K
01:06Legal Guidelines for Documentation
2.1K
The legal guidelines for nursing documentation are essential for ensuring accurate, professional, and ethical recording of patient care. The guidelines are discussed here:
2.1K
01:23Guidelines for Sketching a Curve
110
Curve sketching is a systematic method for understanding the overall behavior of a function by analyzing its key mathematical features. A function defines a curve on the coordinate plane, where the horizontal axis represents the input variable and the vertical axis represents the output. The process begins by determining the domain, which specifies the set of input values for which the function is defined and establishes the horizontal extent of the graph.Intercepts with the horizontal and...
110