Will 20th Century patient safeguards be reversed in the 21st century?

This article summarizes a commentary by the same name by Gregg Gonsalves and Diana Zuckerman, published in The BMJ on March 25, 2015.  The published article is available at: http://www.bmj.com/content/350/bmj.h1500

Will 20th Century Patient Safeguards be Reversed in the 21st Century?

Most physicians and patients assume that medications are proved safe and effective. Those standards from the US Food and Drug Administration (FDA) were born out of a series of 20th century tragedies that resulted in stronger laws to protect patients for unsafe and ineffective treatments.  The first effective challenge to the FDA’s growing authority came in the 1980s, as people with AIDS faced certain death and demanded that the FDA’s drug approval process be more responsive to their needs. AIDS activists pressed for faster drug approval and making experimental therapies widely available, and their successes helped shape FDA reforms.

AIDS activists soon realized, however, that speeding up the approval process had risks as well as benefits, as the first generation of antiretroviral agents were found to have no long-term clinical benefits. Fortunately, a new generation of AIDS drugs, protease inhibitors, were used in combination with the older medicines, and this resulted in dramatic reductions in illness and death.

Starting in the early 1990s, drug industry lobbyists sought to further weaken FDA standards, often citing the legacy of AIDS activists.  AIDS activists opposed those industry efforts, but Congress responding to industry pressure by gradually eroding drug approval and safeguards for patients: reducing the number of studies required to get new drugs on the market from at least two to one and reducing restrictions on the advertising and promotion of medical products.

This erosion has continued in the 21st century.  The FDA now offers four pathways to speed the approval process for many drugs and biologics as well as an easier approval pathway for drugs for orphan diseases (defined as affecting fewer than 200 000 patients in the US). The lower standards for most drugs approved through expedited pathways include smaller and shorter term studies; one study found an average of about 100 patients tested with new drugs that were approved through expedited pathways, compared with almost 600 for standard approvals.

As a result, patients today are relying on many new medications that are not clearly proven to be safe or effective.  Although additional research is often required after approval, it takes an average of 11 years after a drug is on the market for the FDA to institute new warnings, rescind approval, or require new risk information or contraindications be made public.

Medical devices are subject to even weaker approval criteria, with only 1% of devices reviewed through a process that requires clinical trials. With those lower standards, 90% of the thousands of new medical devices are cleared by the FDA for market within 90 days.  Even so, FDA has responded to political pressure by proposing a new faster approval process for devices.

Congressional efforts to reverse the patient safeguards of the 20th century continue. The 21st Century Cures draft legislation released by Republicans on the House health committee in January 2015 would dismantle the major components of the drug approval process in place for the last 50 years. While proponents say patients need these changes, they ignore the fact that patients need answers about the drugs and devices they put in their bodies, not just access to new ones.

If these legislative efforts are successful, it would roll back patient safeguards, and leave an FDA that looks more like the one that existed in the mid-20th century, not one worthy of the 21st.

The published article is by Gregg Gonsalves, lecturer in law at Yale University and a patient living with HIV, and Dr. Diana Zuckerman, president of the National Center for Health Research.