An ALS drug fails, again

Regulatory Failure vs. System Working as Designed

• Many see the ALS drug’s approval as a clear regulatory failure: weak Phase II efficacy data, initial FDA advisors against approval, then a second committee reversing course under pressure.
• Others stress that Phase III failure and subsequent market withdrawal show the broader system still works, though this case exposes loopholes in “conditional” early approvals.

Role of Patient Advocacy and Political Pressure

• Strong concern that ALS and Alzheimer’s advocacy groups, driven by desperation, pushed the FDA and Congress to shortcut safeguards, harming the very patients they aim to help.
• Some argue unmet medical need alone shouldn’t lower evidentiary standards; motivated reasoning (from patients, advocates, or companies) conflicts with truth-finding.

Right-to-Try and Proposed Legislation (e.g., S.1906)

• One camp favors “right to try” for terminal diseases: if risks and uncertainty are clearly disclosed, patients should access promising but unproven drugs.
• Critics counter this enables high-priced “snake oil,” shifts costs to insurers/taxpayers, and exploits desperation.
• Supporters of the bill claim it is targeted, requires prior safety and scientific plausibility, and is not a blank check for quackery; skeptics say lowering thresholds for efficacy inevitably invites abuse.

Harm, False Hope, and Resource Allocation

• Some minimize harm here since the drug appeared safe; others highlight:
  • Enormous cost to patients (~$158k/year list) for a combination of cheap generics.
  • Opportunity cost: money, research effort, and competing therapies displaced.
  • Psychological and practical damage from false hope and time lost on ineffective treatments.

Clinical Trials, Incentives, and Pharma Behavior

• Debate over trial phases: some frame Phase II as mainly safety, others insist it is also about efficacy and should prevent weak candidates from reaching Phase III.
• Discussion of perverse incentives: early approval plus high prices rewards companies even when data are thin, encourages “bootstrapping” on marginal results, and may bias researchers and executives.
• Others note that late-stage failure can bankrupt companies and is part of the intended risk of drug development.

Patient and Caregiver Experiences

• Personal accounts of ALS trials describe burdensome procedures with unclear benefit and frustration at how trial slots are allocated.
• The thread also includes anecdotal claims of dramatic improvement from Ayurvedic/herbal treatments, which other comments contextualize amid concerns about quack remedies and exploitation of the desperate.