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Right to try legislation well intentioned, but misguided

A few months before his hospitalization with an aggressive tumor, Mr. B was the picture of health, enjoying semi-retirement after an accomplished career. He would have accepted any treatment if it could have provided meaningful time with his family. I would have done anything to help him.

Unfortunately, right-to-try legislation, which President Donald Trump highlighted at the beginning of his recent drug pricing speech, is a step in the wrong direction to help individuals like Mr. B.

Federal right-to-try legislation would provide a pathway for terminally ill patients to obtain experimental drugs, bypassing the Food and Drug Administration (FDA). This week, the House plans to vote on a right-to-try bill that passed in the Senate by unanimous consent last August.

Right-to-try legislation may seem appealing and intuitive at first glance. After all, some may ask, what is there to lose from giving terminally ill patients a last chance at a longer life? However, this legislation is unlikely to meaningfully improve their access to treatment options, and may diminish the safety of their treatment and degrade their quality of life in their final days.

What right-to-try advocates do not emphasize is that our current regulatory system already gives terminally ill patients safer and more effective avenues for trying experimental treatments. Indeed, when patients have exhausted approved medications and are unable to enroll in clinical trials, they can obtain investigational drugs through the FDA’s expanded access program. Right-to-try supporters claim that the FDA is a barrier in this process. Yet according to testimony by FDA Commissioner Scott Gottlieb, his agency authorizes 99 percent of the over 1,000 expanded access applications that it receives annually. An application takes 45 minutes to complete and typically receives authorization within a few days (or immediately by phone, in emergencies).

Instead of being a barrier, the FDA plays an instrumental role in giving terminally ill patients a chance at better outcomes. Whereas the FDA may have confidential information about ongoing, blinded trials of investigational drugs, a patient’s physician would not have access to this information. In about 10 percent of cases, the FDA modifies an expanded access application for safety reasons, for instance, changing the dose of a medication.

Rather than prioritizing what’s best for the terminally ill, some leaders of the right-to-try movement may be more invested in chipping away at the FDA’s authority. The movement’s champions, including Americans for Prosperity and the Goldwater Institute, have worked to reduce federal regulatory authority on other fronts. Indeed, the Goldwater Institute is concurrently campaigning to diminish FDA authority by advocating for legislation to rollback restrictions on how companies communicate about off-label uses of drugs.

As a witness to the day-in, day-out suffering that terminal illness can cause, I believe that policies relating to the terminally ill must honor their needs first and foremost, rather than serving a larger legislative agenda. Moreover, the FDA’s regulatory authority today has been shaped by past tragedies caused by once-promising drugs with unintended consequences. Given my commitment to the Hippocratic oath of “do no harm,” I believe that a strong FDA is vital.

Regrettably, advocacy for right-to-try may already be causing harm through statements that imply investigational drugs are always beneficial. Darcy Olsen, former president of the Goldwater Institute, entitled her book on the topic “The Right to Try: How Federal Government Prevents Americans from Getting the Life-Saving Treatments They Need.” By the very definition of the word “investigational,” we don’t yet know whether a given drug is effective, or whether its effectiveness is sufficient to justify potential harms. In one study, only 43 percent of the drugs given to patients under expanded access received FDA approval within the following five years.

Notably, investigational drugs may cause suffering or hasten death. Some individuals feel that these risks are worthwhile, but for many, they are inconsistent with how they would like to spend what may be their last days with family. The decision is personal. The right-to-try movement’s false promise of benefit and insufficient attention to the risks of investigational drugs may mislead individuals who are trying to make important decisions about their remaining time.

Before passing away, Mr. B returned home. I hope his family’s last days together were peaceful. As much as I would have wanted to offer him an investigational drug that had a chance of prolonging his life, I would not have wanted to worsen his suffering, at least not without a well-informed consideration of the risks and benefits.

Helen Knight (hpknight@gmail.com) will graduate this week with a Doctor of Medicine degree from the Johns Hopkins University School of Medicine.

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