Who Should Decide Drugs Are Safe And Effective?

Would you want a plumber piloting the next plane you board? A chef to give a second opinion on the advisability of surgery? Perhaps a grocer to assess the structural integrity of the bridge you are about to cross?  No? Then why ask legislators or neighbors to decide a drug is safe and effective for patient use? Citizens and legislators in 29 states have done this with marijuana. Advocacy groups successfully lobbied several state legislatures to approve the sale of a non-intoxicating cannabis oil for treatment of certain types of epilepsy. Forty states and the US Congress have decided to override federal regulators and open patient access to experimental drugs through right-to-try legislation. Such actions are an unwise foray into activities we should leave to the Food and Drug Administration (FDA).

First, legislators and the average citizen are not qualified to assess if a treatment is safe and effective—anymore than they are qualified to assess the safety of a plane to fly. These decisions require substantial expertise and access to critical data regarding the drug product. The esteemed members of our state and federal legislatures possess neither.

Major legislation shaping the authority of the FDA has repeatedly arisen in response to unsafe products on the market. One early example is the Massengill Massacre of 1937, in which about 100 people died from a liquid preparation of a sulfa drug. The drug was both safe and effective—but because of the use of a toxic solvent to dissolve the drug to create a liquid form, the product was lethal. Consequently, the Food, Drug and Cosmetic Act was passed in 1938 requiring the FDA to assess the safety of drug products. This requires careful evaluation of the total product, not just the active ingredient. Our legislators lack the information and expertise to make such evaluations.

Second, history shows the error of past decisions by legislators approving medical therapies in response to advocacy groups. By the mid-1980s, 27 states legalized the sale of the dubious cancer treatment laetrile. Subsequent studies demonstrated the ineffectiveness of laetrile beyond reasonable doubt. In the meantime, state legislative action approving this therapy encouraged patients to forgo proven treatments for an unregulated remedy of unknown quality. Federal legislators made similar missteps. Under pressure from Congress in 1994, the office controlling federal worker health benefits mandated insurance plans cover bone marrow transplant and high dose chemotherapy for breast cancer—at the time an unproven experimental treatment. Subsequent studies demonstrated the treatment did not improve patient survival.

Third, we jeopardize the health of our citizens by asking legislators to take on a responsibility for which they are not able to provide appropriate safeguards. Despite a rigorous evaluation system before marketing, the FDA regularly oversees recalls of lots of approved drugs due to inadvertent contamination with unsafe components. This contamination most commonly occurs through problems with the manufacturing process. No such safety net exists to detect and respond to these inadvertent contaminants in a product when state legislators approve the sale of medical therapies not sanctioned by the FDA.

The heart breaking experience of patients with ailments having no approved effective treatments deserves a compassionate response. But opening the door to products of unknown quality which lack sufficient evidence of safety or effectiveness places the health of the public at risk. The federal drug approval process is imperfect, but it protects us from unsafe products. Congress created the FDA and authorized the agency to assemble experts to fulfill this task. Our elected officials are in no position to evaluate medical treatments and we should not pressure them to do so.