Federal Right-To-Try Law Gains Strength: Hope or False Hype
​​[Posted on: Thursday, March 8, 2018]

It seems that after about 5 years of attempts by supporters where Right-To-Try (RTT) laws were passed in about 40 states, the US Congress is getting ready to pass a Federal law that would, on paper, make experimental unproven drugs available to patients more easily. Even FDA, that has been a strong opponent of the RTT laws in the past, seems to be succumbing to the political pressure. The goal of the RTT laws was always to get FDA out of the negotiation between a needy patient and a company with an experimental drug. Currently, a company willing to give an experimental drug to a patient has to get permission from the FDA under the expanded access program by filing an IND and submit the results of the expanded access use to FDA. In this process, FDA maintains oversight of the research. FDA allows more than 99% of the expanded access requests, but many companies do not file for it. Estimates are that only about a third of the companies apply for the expanded access program for logistical, liability and financial reasons. With the RTT law, a patient can directly approach a company and negotiate to get the experimental drug without either party ever going to FDA for permission or even informing FDA. The biggest difference between the expanded access program and RTT law is that in the expanded access program there are strict limitations on what a company can charge the patient for the experimental drug with emphasis being on not profiting from the sale of such drug. Such limitations are not present in the RTT laws, so practically a company can charge as much as the patient is willing to pay. Insurance companies still don’t need to cover the experimental treatment, just like they don’t cover drugs provided under the expanded access program. And the company does not need to tell FDA if there are undesired effects, i.e., the drug doesn’t work or there are new adverse effects.  And it is this non-accountability that worries the opponents of the RTT law. Another concern is that this could create a back-channel for companies to profit from sales of unapproved medications. However, such concerns have not been seen in the states where RTT laws already exist. The reason is simple, drug companies exist to create FDA-approved products that can be covered by insurance. It does not benefit the drug company over long term to provide their experimental drugs to a few patients for a fee, while jeopardizing their ongoing clinical trials and FDA approval process. Since drugs companies still cannot be forced to provide a given experimental drug under the RTT law, for the companies, the conditions change marginally. In the states where RTT laws exist, there is little data to show that patients got more access to experimental drugs than they had under the expanded access program. But hope is very powerful, and politics of appearing to help needy patients is very appealing. So we will have a federal RTT law soon, whether it will have any practical impact on drug access, no one wants to discuss.