False Hope: Congress Failed to Protect Your Right to Safe, Effective Medicines

On May 30, 2018, President Trump signed “Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, or S.204,” into law, after the House followed the Senate in caving to libertarian de-regulation pressure. The new law creates a second pathway for pre-approval access, redundant given the Food and Drug Administration (FDA)’s existing and effective Expanded Access Program. Under this new pathway, pharmaceutical manufacturers can provide experimental medicines outside of research without no FDA or investigational review board approval. TAG is deeply disappointed by this failure to protect consumer rights and safety. Below is information on TAG’s work to oppose this legislation as well as a summary of TAG’s objections to it. We also recommend reading NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access June 2018 statement.

Many patients need experimental medicines before they are approved. The U.S. Food and Drug Administration (FDA) already has a very effective mechanism for allowing for early access. Yet Trump and his cronies want to take away the FDA’s power to protect your safety with “Right to Try” legislation. Despite the catchy name, “Right to Try” offers only False Hope. It fails to give patients any right to access drugs or devices, and allows companies to sell potentially ineffective or harmful investigational products to the public without price caps.

The Senate already passed a False Hope bill (The Right to Try Act of 2017, or S.204). As of early February 2018, we anticipate a similar bill will go to the House soon.

LEARN THE FACTS

The FDA is not a barrier for patients accessing drugs pre-approval—thanks in large part to HIV activists at TAG and elsewhere—the FDA already has a strong mechanism for allowing patients to access investigational drugs. In fact, the FDA okays the vast majority (99.7%) of the over 1,000 expanded access received each year.
The proposed legislation does nothing to make or help companies make their products available—because FDA approves nearly all requests for pre-approval access, the major barrier to pre-approval access is opaque decisions from companies about who can access their experimental drugs and when. Right to Try legislation does nothing to ensure companies provide access. Some companies do have legitimate constraints to being able to provide pre-approval access (e.g. limited resources to manufacture enough drug, or to develop and staff a pre-approval access program)—but they get no help from this legislation, either.
Too early is as bad as too late—allowing companies to sell experimental drugs and devices after they’ve been in just one small trial in humans (meaning no evidence of efficacy, limited understanding of safety or side effects, or knowledge of the right dose) could unleash ineffective or harmful drug candidates on the public. The majority of experimental drugs fail over the course of clinical development either due to lack of safety or efficacy, which is why clinical trials and the FDA exist.
Companies could charge whatever they want for an unproven product—Right to Try legislation would allow companies to charge any price for their experimental medicines, sidestepping the FDA approval process and potentially ripping off desperate patients and their families.

Learn more about expanded access and why False Hope legislation is so dangerous: