A sim­ple pa­tient sur­vey rev­o­lu­tion­ized ALS drug de­vel­op­ment in the 1990s. Its cre­ator says it may be time for an up­grade

It’s been an­oth­er tough year for ALS ther­a­pies. New tri­als that sparked pa­tient hopes ei­ther fell short of sta­tis­ti­cal sig­nif­i­cance or failed to pro­vide enough ev­i­dence for the FDA to rec­om­mend mov­ing for­ward with­out an­oth­er look. The end re­sult is large­ly more of the same for pa­tients — lit­tle progress in a fa­tal dis­ease with on­ly two ap­proved treat­ments.

That frus­trat­ing slog has stirred pas­sion­ate de­bate with­in the pa­tient and ad­vo­ca­cy com­mu­ni­ties, large­ly around things like tri­al de­sign, com­pas­sion­ate use pro­grams or even what the bar for ap­proval should be. Long over­looked in those dis­cus­sions, how­ev­er, is a tool used to eval­u­ate every ALS ther­a­py that’s draw­ing in­creas­ing scruti­ny.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER