ACT for ALS

 ALS is a 100% fatal disease. Before it slowly kills you, it paralyzes your entire body. There are promising drugs in clinical trials for the first time in the 150 year history of the disease. We need your help to cosponsor and pass the ACT for ALS – S.1813 -- which empowers the FDA’s existing Expanded Access Program (“EAP”), helping 30,000 Americans fight for their lives.

The ACT for ALS (s.1813) was introduced last session by Senators Coons & Murkowski.  The House companion bill is H.R. 3537.  Importantly, the ACT empowers the FDA’s existing Expanded Access program.  It provides a grant to fund Expanded Access for therapies in Phase 3 clinical trials. The grant fund is only available to small biopharma companies & the therapy can only be administered at the clinical trial sites conducting the trial.  

The ACT ensures humanity by giving dying patients, who were excluded from the clinical trial process, a promising therapeutic option.  Without access, they have a 100% chance of dying.  No drug or therapy increases those foreboding odds.  Additionally, by limiting it to Phase 3 trials, the ACT ensures the drug has passed Phase 1 safety trials & multiple DSMB reviews.  Safety concerns should not block support for the ACT.

The limitation to Phase 3 clinical trial sites accomplishes three goals.  It limits the likelihood of adverse events with EAP participants & assuages manufacturers’ concerns that EAPs will impact drug approval (although the FDA confirmed this has never happened in 11,000 EAPs).  This provision furthers scientific purpose by ensuring the trial’s researchers collect data in the EAP, alongside the trial arm, to enable the FDA to make more informed decisions about a drug’s efficacy.

The ACT levels the playing field with patients for rare underfunded diseases.  In the last decade, the FDA has authorized EAPs over 15,000 times – but most were for cancer & other common conditions developed by well-funded drug sponsors.  ALS drug sponsors are almost exclusively small pharma without funding for Expanded Access.  Thus, the ACT is merely giving people with ALS the same chance to fight for their lives as is afforded to people with cancer, et al.

In sum, the ACT benefits our search for treatments in rare, untreatable, rapidly progressing, lethal neurodegenerative diseases with vast heterogeneity like ALS.  Unlike Right to Try -- which had no scientific purpose -- the ACT increases the  sample size for the FDA’s statistical analysis.  With more data, it makes evaluations of efficacy more informed and, it increases the likelihood of identifying viable treatments for small subsets of the heterogenous ALS population.