Offering Patients the ‘Right to Try’

Last Wednesday, surrounded by terminally ill patients and their families, President Trump signed a bill allowing people with terminal illnesses for which traditional medicines and procedures have not helped to have smoother access to experimental medical treatments not yet approved by the Food and Drug Administration (FDA).

Dubbed the “Right to Try” law, a play on the name of the movement that advocates for doctor-assisted suicide, or “the right to die,” the new law aims to accomplish that effort’s opposite: lengthening the lives of those facing fatal illnesses.

What it does is allow such people to seek access directly from drug manufacturers to experimental medicines that are in clinical trials but have not yet been fully approved by the Food and Drug Administration.

Until now, in fact since the 1970s, a program known as “compassionate use,” or “expanded access,” has allowed patients with serious diseases or conditions to obtain experimental medicines through the FDA. And the agency says it authorizes 99 percent of the requests for expanded access that it receives. According to FDA Commissioner Dr. Scott Gottlieb, in comments last year, “Emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days.”

More than three dozen states, moreover, already have laws allowing patients and doctors to ask drug companies directly for access to the experimental drugs, rather than wait for approval by the agency. New York and New Jersey are not among them, though, and the new federal law expands the “right-to-try” option nationally.

Most Congressional Democrats and an assortment of public health groups opposed the law when it was being considered in the House and Senate, arguing that it offers “false hope” to terminally ill patients, and could put them in danger. But few hopes can ever be judged as false, and the new law only permits access to drugs that have been deemed safe in what is known as Phase One of the FDA approval process. The drugs at issue are already in the “human trial” phase of testing but just haven’t cleared the substantial hurdles needed to ascertain that they will yield true benefits to patients.

The more daunting issue concerns who will pay for such experimental drugs. Insurance companies, many of which don’t even cover some FDA-approved drugs, are not likely to cover treatments that have not been deemed effective by the FDA, and new drugs are often prohibitively expensive.

As Arthur Caplan, head of medical ethics at New York University’s School of Medicine, observed, “The law gives the right to pay, not the right to try.”

Medical costs, in fact, are already the leading cause of bankruptcy in the U.S. Under “right to try,” a patient could end up paying additional hundreds of thousands of dollars for experimental treatment, on top of their existing costs.

The hope, though — and it is not a false one — is that the drug companies will make requested drugs available at affordable prices, something that could turn out to be to their financial advantage should an experimental drug prove effective in a large number of right-to-try cases, which could fast-track full FDA testing and lead to the agency’s quicker approval of the medicine.

Over time, this law has the potential to save “thousands and thousands” of lives, as President Trump stated at the bill’s signing. The right-to-try movement and the attention that Mr. Trump has drawn to it are undeniably positive steps toward raising patients’ awareness about experimental treatments. Needless to say, care and caution must be exercised by patients and their families, in consultation with their medical professionals, before embarking on an experimental drug regimen.

Fundamentally, though, the new law is designed to expand the choices of patients, and to limit the need for a federal body’s regulatory approval. Federal agencies can play an important role in protecting the public, no doubt. But they can also be impediments to the citizenry’s rights to make choices about their lives. What the “right to try” law permits does not endanger patients, and may well lead to longer lives for at least some patients.

The bottom line is that drugs that have already been deemed sufficiently safe by the FDA for use in trials on humans will now be more easily accessible to Americans. And allowing citizens to make the choice, if they deem it a right one, to try a new medical therapy is a simple matter of freedom, and a most welcome development.