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A Patient Perspective on ALS Clinical Trials and Timely Access

When time matters, how much evidence is enough?

How much evidence should be required before people living with ALS can access a promising new therapy?

In a newly published Reader Response in Neurology, Dr. Andrew Darke, an ALS Action Canada Advocacy Committee Member, responds to a recent article examining what its authors describe as a “credibility gap” in ALS clinical trials, due to what it describes as a “Clinical Trial Industrial Complex” involving ALS neurologists and manufacturers of new drugs for ALS.

Andrew offers an important patient perspective on the role of Phase 2 clinical trial evidence in regulatory decision-making. Using the development and subsequent withdrawal of Albrioza (sodium phenylbutyrate–taurursodiol) as an example, he argues that adequately powered Phase 2 studies, supported by robust statistical analysis, can have an appropriate role in expedited or conditional approval pathways — particularly for ALS and other rare diseases where time matters profoundly to patients.

Andrew’s response represents his individual perspective and contributes to a broader and important discussion about how we balance scientific rigour, uncertainty and the urgent need for new ALS treatments.

Read Dr. Andrew Darke’s Reader Response in Neurology

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