Here’s how to structure successful right-to-try laws

Thirty eight States have enacted right-to-try (RTT) laws, the intent is to increase the availability of experimental medicines to individuals battling life-threatening conditions A federal version has support at the highest levels of the Trump administration including the president himself. Yet the Goldwater Institute, which created and has strongly championed these laws, cannot provide clear examples of patients who have gained access to potentially life-saving treatments through state RTT laws that they otherwise wouldn’t have received under the FDA’s current Expanded Access Program (EAP).  Furthermore, federal RTT law will not increase access either.

Why is this the case? First, both the enacted state RTT laws and the pending federal RTT legislation do not address the risk to the drug development process by increasing the availability of experimental medicines.

{mosads}In fact, the proposed federal RTT law increases risk to patients and the drug development process by creating a path to circumvent the Food and Drug Administration (FDA) regulatory and oversight process by permitting access to experimental medicines directly from drug developers.

No ethical biopharmaceutical company would allow its experimental medicine to be used without FDA oversight and involvement. Developers of new medicines have the prime responsibility for deciding if an experimental medicine should be made available based on the evolving safety and efficacy profile as well as the medicine’s stage of development and overall development pathway.  

The companies bear the risk of providing an experimental medicine  —whose safety and efficacy may not be well understood — with consequences that could slow down or jeopardize the drug development process and the experimental medicine’s ultimate availability to both current and future patients.

Second, providing pre-approval access to an experimental medicine is costly — from the costs of providing the medical care to a patient in need to the costs of managing the process and providing the experimental medicine itself. In Europe, companies may be able to charge under Named Patient Programs, which have not garnered any support in the United States

Finally, given that the FDA approves 99 percent of the 1000 or so applications they receive, FDA oversight is not the issue. The number of applications submitted is due in large part to many physicians not knowing about EA programs or that their patients may qualify for these programs, and hopefully this number will grow in the future. The recently enacted 21st Century Cures Act mandated that companies state their specific EA policies once they progress into the later phases of clinical development.

All this being said, one cannot ignore the desire to increase the access of critically and terminally ill individuals to experimental medicines. The question then becomes how to accomplish this in a way that meets the needs and requirements of regulators, companies and the current and future individuals in need?

One way to meld the intent of RTT laws with the existing EA process would be to create a more explicit regulatory pathway. This allows EA safety and efficacy data to be incorporated into the label of a new medicine once it is formally approved for its primary indication via “traditional,” highly controlled clinical trials.

In this way, companies could more fully balance the risks and benefits of an EAP to the overall development process of the experimental medicine. Companies would be incented to undertake these broader EA programs because they could determine how the EA program would potentially lead to the ability to treat a larger future patient population.

This would require legislative and regulatory changes to existing guidelines for intermediate- or large-scale EAPs, but would still be within the framework and intent of the 21st Century Cures Act.  

Rather than circumventing the FDA oversight process, companies would have to reach agreement with the FDA as to the parameters under which “real-world evidence” (evidence regarding the potential benefits and risks of a product) collected from patients enrolled in expanded access trials could be used to support additional label claims for a new medicine. These programs would be overseen as if they were formal clinical trials designed to provide full input into the drug development process.

All of us who are committed to creating life-saving medicines would like to see medicines made available to as many people as possible and as quickly as possible.

However, we also recognize that the process of conducting rigorous clinical trials to demonstrate both efficacy and safety is lengthy and complex, and that there are patients in need who cannot wait for the approval process to be completed.

Rather than skirting regulatory oversight through RTT legislation that will not change the landscape in terms of access, we should seek to rework existing legislation to create the potential for broader label claims, more appropriate and successful reimbursement pathways, and the collection of data from larger expanded access programs. Only then will patients in dire need gain access to potentially life-saving medicines.

Kenneth I. Moch is CEO of Cognition Therapeutics, a company developing new medicines for Alzheimer’s disease, and has been the co-founder or CEO of five companies developing therapies for life-threatening diseases. Andrew McFadyen is executive director of the Isaac Foundation. Arthur Caplan, Ph.D. is a professor and founding head of the Division of Bioethics at New York University Langone Medical Center.