Regulatory issues in ALS clinical trials
Abstract
ALS and other motor neuron disorders 2004 5(Suppl 1), 36â41 # 2004 ALS and other motor neuron disorders. All rights reserved. ISSN 1466-0822 36 Issues in Clinical Trials Regulatory issues in ALS clinical trials Wilson W Bryan US Food and Drug Administration, Maryland Drug development should be an orderly process, with each stage building upon the information obtained in the previous stage(s). Each clinical trial should investigate speciï¬c questions related to safety and/or efï¬cacy and attempt to provide information critical to future drug development. The design of each clinical trial should reï¬ect these speciï¬c goals. This presentation discusses several topics relevant to clinical trial design for the orderly development of drugsa to treat amyotrophic lateral sclerosis (ALS): 1) Common problems in clinical trial design at each stage of drug development; 2) Food and Drug Administration (FDA) requirements for demonstration of effectiveness of a drug or biologic; 3) Selection of endpoints; 4) Accelerated approval; 5) Regulatory issues in drug combination trials. who are deemed to be at high risk for developing the anticipated toxicity. Clinical trials typically include monitoring programs that are directed at assessing the anticipated toxicity in humans. For example, if the study drug is associated with