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Regulatory Focus™ > News Articles > 2019 > 9 > FDA Finalizes ALS Drug Development Guidance

FDA Finalizes ALS Drug Development Guidance

Posted 23 September 2019 | By Zachary Brennan 

FDA Finalizes ALS Drug Development Guidance

The US Food and Drug Administration (FDA) on Monday finalized guidance to assist in the clinical development of treatments for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

The 11-page guidance, which finalizes a draft version from February 2018, includes information on general development, safety and effectiveness considerations, as well as specific effectiveness trial considerations.

The guidance incorporates parts of a guidance put together by the ALS Association, known for its viral "ice bucket challenge."

Changes made between the draft and final versions, according to FDA, also took into consideration written and verbal comments on the draft.

“Information was added about strategies to expedite clinical trials in ALS and minimize exposure to placebo, about the importance of broad inclusion criteria in ALS clinical trials, encouraging the use of patient input and experience in the development of new outcome measures that are capable of measuring clinically meaningful effects in patients, and encouraging the incorporation of exploratory biomarkers in ALS development programs,” FDA said.

Acting FDA Commissioner Ned Sharpless added in a statement: “Specific to clinical trial design, this final guidance provides clarity around some of the questions raised by stakeholders. It emphasizes that all patients in ALS trials should receive the best standard of care, and no patient should be denied effective therapies in order to be randomized to a placebo-only arm. While exposure to placebo may be the best way to determine the effectiveness of some products, the guidance also explains various strategies that can be applied to minimize unnecessary exposure to placebo and expedite trials, such as master protocols, adaptive designs and enrichment strategies.”

The agency also noted additional language to provide greater context on the use of historical control data in ALS clinical trials, to suggest approaches to minimize patient burden and to clarify safety data expectations for new ALS treatments.

“Finally, additions emphasize FDA’s willingness to exercise flexibility in applying the statutory standards for approval of drugs for the treatment of serious diseases with unmet medical needs, while preserving appropriate assurances for safety and effectiveness,” the agency added.

ALS Developing Drugs for Treatment: Guidance for Industry


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Tags: ALS, FDA, guidance

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