publication date: Aug. 4, 2017

Guest Editorial

Senate passes a “right to try” bill; Harm to patients comes next

By Kelly McBride Folkers, Lisa Kearns & Alison Bateman-House

Patients for whom there are no existing treatments watch with desperation as a potentially helpful new drug spends years working its way from a lab bench, through clinical trials, and finally to the FDA, where reviewers consider it for approval.

To help those who don’t qualify for clinical trials but wish to try experimental drugs as a last treatment option, the FDA allows patients’ physicians to apply for “compassionate use” of experimental medications through its expanded access program, which allows seriously ill patients without other treatment options to access investigational, unapproved drugs and devices outside of a clinical trial.

But even with the possibility of expanded access, many seriously ill patients remain dissatisfied with the status quo, arguing that applying for compassionate use is confusing and online platforms are hard to navigate. Doctors, who typically receive no training in expanded access, may not know how to make these requests. And unfortunately, companies do not always make their expanded access policies public.

A report from March 2017 found that, of a sample of 100 pharmaceutical companies of all sizes, fewer than 50 percent have done so. And even if physicians know how to make a request, pharmaceutical companies are free to say no. Companies may say no if a drug-in-development is in scarce supply; likewise, they may be fearful of giving their unproven products to patients outside of clinical trials, especially in early stages of research, when basic safety data is scant and efficacy data does not yet exist.

In an attempt to solve these issues, the Goldwater Institute, a libertarian think-tank that works to reduce government … Continue reading Senate passes a “right to try” bill; Harm to patients comes next

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