News Feature | November 25, 2014

Spreading "Right To Try" Laws Present Challenges For Pharma Industry

By Suzanne Hodsden

Legislatures in Arizona, Colorado, Louisiana, Michigan, and Missouri have all passed laws this year which attempt to give terminal patients faster access to experimental medicines, and there are additional initiatives starting in more states. Critics fear that these laws will do more harm than good both in terms of patient care and the overall drug development process.

These “right to try” laws bypass existing FDA measures which grant expanded access and compassionate use permission to experimental drugs on a case-by-case basis. Regulatory Affairs Professionals Society (RAPS) reports that of the nearly 1,000 applications the FDA received between 2009 and 2013, nearly all of them were approved.

According to RAPS, the FDA process includes application by physician, FDA review and approval, and extensive evaluation of safety and the patient’s understanding of risk.

But for some, the FDA’s four to five month application process does not move fast enough. The new laws are an initiative of the Goldwater Institute, a conservative non-profit.

Victor Riches, VP of external affairs at Goldwater, explains the project. “For people with terminal illness, for whom nothing else has proved effective, they don’t have the luxury of waiting four to five months to get through the FDA’s compassionate-use program.”

The new laws allow patient access to a drug after it has passed phase 1 of clinical trials, but they do not require the company to provide the drug.

Critics say that greatest risk of these laws is that the safety and efficacy of drugs is unproven so early in the development process and could expose patients to serious side-effects.

What’s more, Arthur Caplan, bioethicist at NYU Langone Medical Center, warns that the laws could give false hope to patients and shoulder them with potentially astronomical costs to cover a drug that doesn’t even work.

Scientists speculate that the risk to the drug development process could be equally problematic. Brenda Huneycutt, director of regulatory strategy and FDA policy at Avalere Health told RAPS, “The concern is that these laws don’t really address the risks to the companies.”

NPR’s report warns that adverse or dangerous side-effects could set back a drug’s approval process significantly and hinder it’s delivery to market and a much wider population. Furthermore, experimental access could make patients less likely to participate in controlled clinical trials where the drug could be carefully monitored, studied, and potentially improved.

R. Alta Charo, law and bioethics professor at University of Wisconsin Madison, commented to U.S. News and World Report, “The laws are a simplistic way of going after a much more complicated issue.”