Right To Try Law

S. 204 signed into law May 30, 2018

The Act defines eligible patients, diseases and drugs; and establishes National standards for the implementation of treatments under the act.

An act to authorize the use of unapproved medical products (drugs, devices and or biologics) by patients diagnosed with life threatening diseases.

The Act sits along side of the FDA’s expanded access programs and clinical trials as an alternative path to improve access to experimental drugs and increase the use of Real World Data in drug development.

Clinical trials in the U.S. are an expensive and highly regulated undertaking. On average it takes ten years and costs over a billion dollars from discovery of a drug to approval by the FDA. Even for smaller drug markets and smaller biotech companies trials take 7 to 10 years and cost tens and even hundreds of millions of dollars to get to approval.

The FDA clinical trial process tightly controls who can and can’t be treated in a trial; and approval usually requires at least two “well controlled” trials that demonstrate safety and efficacy. Well controlled in this instance means that some sort of placebo is tested in a group who think they are getting the actual drug, to make sure that any effectiveness seen in the end can be ascribed to the drug itself. While it may seem unethical to “pretend” to give seriously ill or dying patients a placebo instead of the actual experimental drug, this system is the foundation of all FDA approvals.

What is the new Right to Try law, and how will it change the entire drug development system?

In May of 2018, after years of effort on behalf of many patient advocates and others, the Federal Right to Try act was passed by Congress and signed into law by the President. This law was created to give patients with a potentially fatal disease the right to access experimental drugs outside of a clinical trial. These can only be drugs that have not yet been approved but have gone at least beyond the first phase of safety testing by the FDA.

As an incentive to encourage drug developers to increase access to these experimental drugs the law allows drug developers to treat eligible patients without asking for FDA approval; without direct FDA oversight of the treatment(s); and with minimal reporting responsibilities. As an additional incentive, the law grants broad immunity from lawsuits other than for gross negligence or outright fraud. Additionally, under the new law the data created by the sponsor belongs solely to that sponsor who can share the data with the FDA or not, at their discretion. The law specifically prohibits the FDA from using the data in any way to delay or deny the drugs review or approval. There are rare exceptions for extraordinary safety failures.

Additionally the law creates a system where patients know that they are getting the actual experimental drug, never a placebo.

Right To Try Parameters

Eligible drugs must have completed an FDA phase 1 safety trial, and be actively in the FDA clinical trial queue for an efficacy study. If/when a drug drops out of the FDA clinical development pipeline, it is no longer an RTT eligible drug.

So while there is minimal FDA involvement in an RTT program, all RTT eligible drugs are under continuous FDA review, providing continuing comfort on safety.

Off label use of FDA approved drugs are not a permissible RTT use.

Eligible patients must be diagnosed with a disease that, if left untreated, is likely to be fatal; or a disease where survival is an end point in an efficacy trial.

#RightToTry