by Naomi Lopez Bauman
December 3, 2018
The Right to Try Act represents an important step toward helping dying patients with little recourse.
The recently enacted Right to Try law marks a new day for the process of approving medicines that sick and dying Americans desperately need. The law—which came about through a bipartisan grassroots effort across the United States—provides that patients have the right to seek medicines that have received basic safety approval by the U.S. Food and Drug Administration (FDA) but have not yet been fully approved for sale.
A forthcoming journal article, “Regenerative Medicine and the Right to Try,” by Christine Coughlin, Nancy King, and Melissa McKinney addresses patient safety and treatment access in the context of the right-to-try movement, but it is important to point out some of the shortcomings of the forthcoming article. Coughlin and her coauthors base their forthcoming article on faulty understandings of the text of the right-to-try law and the reality of the FDA process for accessing investigational treatments before the movement to adopt right-to-try laws. Perhaps most importantly, Coughlin and her coauthors disregard the important patient protections built into the Right to Try Act.
These errors are clear in Coughlin and her coauthors’ rhetorical question: “Have we moved so far toward relaxing protective regulations that we have begun to enable fraud, exploitation, and injustice toward the sickest among us?” The authors give the impression that the Right to Try law is a dramatic departure from the status quo.
In reality, the Right to Try Act works in tandem with the current FDA approval process and its safety rules. For example, the Right to Try Act only applies to patients who have been diagnosed with a life-threatening disease or condition, have exhausted approved treatment options, and are under the care of a doctor in good standing who recommends the treatment and has provided written consent regarding the risks involved. And the law applies only to treatments that FDA has deemed safe enough to be administered in ongoing, FDA-approved clinical trials. If a medicine proves so unsafe that FDA withdraws it from clinical trials, it also gets withdrawn from right-to-try patients.
In other words, the treatments allowed under the Right to Try Act are the same treatments being given to patients with the same disease who are lucky enough to qualify for a clinical trial. By removing the federal red tape, the Right to Try Act makes “compassionate use” more accessible for everyone.
The Right to Try Act’s approach is a significant step forward—and does not sacrifice existing safety protocols—because the “compassionate use” program bars untold numbers of terminal patients at the door, thanks to an overly complex application process that can take a physician dozens of hours to navigate and complete, if it can be done at all. Coughlin and her coauthors claim that the application process now takes only one hour, but this claim reflects a misunderstanding of the application itself, as well as the information the FDA requires as part of the process.
Claiming the application takes a physician just an hour to complete is like claiming it takes one hour to complete a federal tax form. Such a statement might be technically true, but it ignores the fact that one must also read and understand the instruction and gather documents, among other preparations. As with a tax form, completing the “compassionate use” application requiresburdensome information gathering, which presumably takes roughly 30 hours—time a physician could spend caring for other patients.
As the Goldwater Institute’s Mark Flatten explains, the “compassionate use” system is inaccessible to most patients for other reasons, too: Patients are frequently too sick to qualify, and often, the information FDA demands from applicants is not available. As a result, it is impossible to tell how many people are denied “compassionate use” because they cannot fill out the form to begin with. FDA approves fewer than 2,000 applications annually. This year alone, 600,000 people will die of cancer. The idea that fewer than 2,000 terminal patients from all diseases per year would seek potentially lifesaving treatment is implausible. In fact, it suggests how unattainable “compassionate use” really is.
Unattainable for ordinary patients, that is. Affluent patients are already free to travel the world seeking treatments. It is those without the means who are left without options under “compassionate use.”
The Right to Try Act’s expense rules are also the same as “compassionate use.” Federal law already prohibits companies from profiting from any drug or treatment not approved by FDA, but companies can recoup the costs of treating an individual. This rule applies to patients who qualify under both compassionate use and Right to Try Act standards: Federal law is the same for any investigational treatment.
But the Right to Try Act’s most important contribution is that it recognizes and protects the fundamental principle of patient autonomy. Indeed, the law explicitly intends to “expands the scope of individual liberty and agency among patients, in limited circumstances.” It does so by recognizing that these deeply personal decisions belong in the hands of patients and their physicians.
No one else owns a patient’s life—especially not the government. Coughlin and her coauthors explicitly acknowledge this tension: “The goal of right to try legislation is to provide individual patients with the right to request direct and rapid access to what they regard as potentially life-saving or life-prolonging interventions,” which “may simply be at odds with the goal of ensuring that enough data can be gathered to prove that a new drug or biologic is sufficiently safe and effective to be provided to patients as a treatment.”
The idea that the government or researchers’ interests in “data” takes precedence over the patient’s right to make her own medical decisions is absurd and runs counter to Americans’ deeply held personal beliefs and constitutional protections. Patient safety, of course, is a critical priority—but the Right to Try Act retains existing protections while increasing access to medical treatment.
The Right to Try Act has been a bottom-up, bipartisan, national reform driven by terminally ill patients, advocates, and their loved ones. And it marks a new era in medical regulation. It is important to approach that new era armed with the facts.
Naomi Lopez Bauman is the Director of Healthcare Policy at the Goldwater Institute.
First appeared at The Regulatory Review.