President Trump recently signed legislation permitting terminally ill patients to obtain experimental drugs directly from manufacturers, without having to wait for full approval. The medicines in question have completed only the most basic safety testing—Phase 1, usually performed on a small number of healthy volunteers. About three-quarters of drugs that pass Phase 1 will ultimately fail to be approved because of insufficient safety or efficacy; and the ones that do get approved may turn out to have quite different uses than originally intended.

In allowing the use of lightly tested drugs, the Right to Try Act takes an important step on behalf of seriously ill patients’ rights to make choices about their own care. Yet these same patients cannot use thoroughly evaluated, safe, and effective medicines approved by foreign regulators but not yet by the Food and Drug Administration.

This paradox could be remedied by congressional authorization of drug-approval reciprocity among select foreign counterparts, giving patients rapid access to drugs that have been already proven to work in countries whose testing regimens are similar to our own. Reciprocal approval would benefit patients directly: the negative effects of FDA delays in approving certain new drugs already available in other industrialized countries are well documented. Meningitis B, for example, is a devastating infectious disease that can become debilitating so quickly that by the time it is recognized, the patient may be too sick for effective treatment. The European Union, Australia, and Canada approved the first MenB vaccine, Bexsero, in January 2013. The FDA did not follow suit until February 2015. Meningitis B outbreaks resulted in deaths and limb amputations during that interval, when federal agencies had to resort to a cumbersome process in order to approve limited usage of Bexsero. The Centers for Disease Control applied to the FDA for permission to acquire and distribute the European version of the vaccine.

Reciprocity would alleviate shortages of critical drugs in the U.S. Many of the drugs in short supply include generic injectable medications commonly used by EMTs and in hospitals: analgesics, cancer drugs, anesthetics, antipsychotics for psychiatric emergencies, and electrolytes needed for patients on IV supplementation. Though the FDA has improvised procedures for importing drugs approved and marketed abroad that have not been approved in the U.S., this “enforcement discretion” approach—a kind of ad hoc reciprocity—is legally questionable. In a recent court decision, the FDA was blocked from using enforcement discretion to permit the importation of an unapproved drug for capital punishment, because the law is clear that an unapproved drug cannot come through U.S. Customs. That’s why Congress must step in.

With the Right to Try Act, politicians reaffirmed patient access to investigational drugs, even if these drugs offer only questionable health benefits. It would be far more compassionate and productive to enact legislation that creates reciprocity of approvals, offering patients access to medicines already shown to work.

Photo: MechanismDigital/iStock


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