Pile of colorful pills in blister packs

Expanding The Right to Try Unproven Treatments: A Dangerous, Deregulatory Proposal

By Richard Klein, Kenneth I. Moch, and Arthur L. Caplan

A new proposal out of the Goldwater Institute (GI), a libertarian think tank, advances an oversimplified critique of the U.S. regulatory process for approving medicines for COVID-19 and other diseases, with the ultimate goal of weakening the U.S. Food and Drug Administration.

You may remember the Goldwater Institute as the architect of the initial state “Right to Try” (RtT) legislation from a few years ago. The idea, marketed as increasing access to experimental medicines, was actually calculated to circumvent FDA oversight so that individuals could try still-unproven experimental medicines without what Goldwater viewed as pointless bureaucratic paternalism. RtT legislation was adopted by 41 states and ultimately by the U.S. Congress.

When former President Trump signed the Right to Try bill into federal law with great fanfare on May 20, 2018, he stated that “countless American lives will ultimately be saved.” Three years later, the promise proved to be meaningless, as evidenced by the difficulty in identifying more than a handful of individuals who have even pursued the RtT pathway, much less finding data to show that it has saved lives.

And the Institute’s latest proposal, “Putting Patients First: Unleashing Innovation in America Healthcare,” is as poorly conceived as RtT.

In this proposal, Goldwater has grossly oversimplified the response to COVID-19, incorrectly blaming the slow response in the U.S. to the emphasis on understanding the safety and efficacy of treatments and experimental vaccines. The goal of blunting what GI sees as the heavy hand of bureaucracy: accelerated access to unproven treatments, presumably including such miracles as hydrochloroquinine, leronlimab, and former President Trump’s favorite, bleach.

But developing new medicines is not a simple process. As has been proven time and again, “gut feeling” and wishful thinking have never been shown to replace controlled clinical testing.

If Goldwater truly believes that “it is time for the U.S. to recognize that the current FDA approval process no longer meets the realities of 21st century medicine and instead stifles treatment options for doctors and their patients,” then the best way to achieve this would be to support FDA with the resources, personnel, and systems to make sure that these new therapies are provided with the highest quality evidence of safety and efficacy.

Instead, they offer a formula for stepping backward, allowing highly profitable quackery by those who seek to profit off the sickness of others — an outcome which seems the exact opposite from what Goldwater says it wants.

Ask yourself this: if a medicine could not be provided under the FDA’s regulations, would you want, or want your loved one, to receive that experimental medicine without FDA oversight? Especially where the company specifically avoided FDA involvement? And when they charge huge fees for access? If you said no, you are not alone.

Another focus of the Goldwater proposal is to “Bring the world’s leading medical treatments to Americans.” Goldwater states that, “an estimated 30% of the newest advances in medicine are first available overseas.” They could, of course, have stated that 70% of the newest advances in medicines are first available in the United States, by far the best record globally for new and innovative medical products.

It is difficult to argue that Americans don’t have access to the world’s leading treatments. The pathways available for new medicines — Accelerated Approval, Breakthrough Therapy Designation, Fast Track Designation, Priority Review, Subpart E of the IND regulations — are all successful ways for expediting approval of important and effective medical treatments.

Yes, the U.S. has high standards for approval. If people want to have the best medical products as rapidly as possible, then it is necessary to maintain high standards. New does not necessarily go hand-in-hand with improved, nor does the “right to try” mean that a person should be subjected to unproven therapies and unknown risks. Other countries have different pathways to approval, and most people do not consider or understand those differences, or the considerable differences in how health care is accessed, delivered, and paid for around the world.

Goldwater talks too about “Remov[ing] the secrecy from the U.S. drug approval process” so that “Americans aren’t left in the dark about how the agency makes life-or-death decisions.” But in fact, the U.S. has a highly transparent drug approval process, as observers of the Emergency Use Authorization process for COVID-19 vaccines have readily seen.

Let’s be clear: most people are ill-equipped to make their own evaluation of complex scientific data. At the same time, Goldwater proposes to “End ’expert panels‘ that keep treatments from the most vulnerable.” Who then, should make decisions about the safety and efficacy of new medicines? Experts are essential to making decisions based on understanding and analysis of facts, details, and statistics when it comes to medical products. Drug development and decision-making about approvals are not akin “do-it-yourself brain surgery.”

Goldwater’s plan for accessing treatments without adequate and professional oversight harkens back to a time when there was no regulatory oversight. And it is as inadequate and dangerous today as was in 1860, if not more dangerous, as drugs today are often used worldwide and can pose much greater risks.

Weakening the FDA, the hallmark of Goldwater’s efforts, is not a prudent path to access or approval of new, complex medicines. Accusations of “mounds of red tape” may stimulate the public’s imagination and ire, but such unfounded statements make it harder for people who actually need treatment to navigate through the clutter of misinformation to make informed decisions. They may also deter patients from joining trials, which are the foundation of evidence necessary to really understand the safety and efficacy of medical products.

This proposal will not advance the interests of patients, but rather will push back decades of regulatory policies that demonstrably protect individuals and create pathways for developing new and better medicines.

Richard Klein, an internationally recognized expert in preapproval access to therapeutic agents, is the director of Expanded Access Programs and Policy for the GE2P2 Global Foundation. He worked at the FDA for more than 40 years before leaving the agency in 2017. 

Kenneth I. Moch, MBA has been CEO or co-founder of five companies that pioneered novel therapies for life-threatening diseases: Cognition Therapeutics; Chimerix; Alteon; Biocyte; and The Liposome Company. He has served as chair of the Biotechnology Innovation Organization’s bioethics committee and co-chair of its Emerging Companies Section Strategy and Policy Committee, which is focused on issues surrounding the development of new medicines.

Arthur L. Caplan, PhD is the Drs. William F. and Virginia Connolly Mitty Professor and founding head of the Division of Medical Ethics. He co-chairs the Compassionate Use Advisory Committees (CompAC) and is the author or editor of 32 books and more than 750 papers in peer-reviewed journals.

The authors are all members of the NYU Working Group on Compassionate Use and Preapproval Access (CUPA), whose mission is to advance research, policy, and education regarding the ethical issues surrounding access to investigational medical products. Arthur Caplan’s disclosures are available here. Richard Klein and Kenneth Moch have no disclosures.

The Petrie-Flom Center Staff

The Petrie-Flom Center staff often posts updates, announcements, and guests posts on behalf of others.

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