The Disasters That Built the FDA and Where We Go From Here

The US Food and Drug Administration, as it exists today, was formed in reaction to public health disasters. The devastation wrought by elixir sulfanilamide, thalidomide, and the AIDS epidemic, among other tragic events, prompted popular outrage and demands for change; in turn, the public’s response to these tragedies helped establish a robust federal agency that evaluates the safety and efficacy of prescription drugs and high-risk medical devices before they are widely sold. To safeguard the integrity of the FDA and protect the health of people in this country for generations to come, we have a responsibility to ensure that the current pandemic reshapes the Food and Drug Administration for the better.

The Elixir Sulfanilamide Tragedy and the FDA’s Premarket Oversight of Drug Safety

Before public outrage erupted over the deaths of more than 100 people that were traced back to a new and untested drug,  Congress was not able to pass proposed legislation to empower regulators to verify the safety of new pharmaceuticals. Supported by their lobbyists, manufacturers prevailed with their arguments that federal oversight of drug safety was “dangerous and un-American.” For more than five years after it was introduced in the Senate, the original bill that would become the Food, Drug, and Cosmetic Act received little attention from lawmakers.

It was only after the 1937 elixir sulfanilamide tragedy, in which a drug containing a poisonous solvent killed scores of people, that demands for reform prompted Congress to take action. In 1938, President Franklin Delano Roosevelt passed the landmark Food, Drug, and Cosmetic Act into law, authorizing the FDA to test the safety of new pharmaceuticals before those products could be sold and marketed.

The Thalidomide Disaster and FDA’s Premarket Oversight of Efficacy and Manufacturing

Though the Food, Drug, and Cosmetic Act accorded the FDA some authority to monitor the safety of new drugs, the legislation had some major limitations; in particular, the agency still had no real power to ensure that new drugs were effective, it could not enforce safe manufacturing standards, and manufacturers could still sell new products without FDA approval after 60-180 days if the agency did not prevent such action.

Those flaws were made abundantly clear in the case of thalidomide, a drug intended by its manufacturer to be used by pregnant women to relieve nausea, but which instead vastly increased the risk of phocomelia, a birth defect. The drug was widely sold in Europe but had a muted effect in the US due to the heroic efforts of FDA scientist Frances Kelsey. The drug still caused some damage in the US because the drug’s manufacturer distributed thalidomide to physicians across the country as part of an unregulated “trial” while Dr. Kelsey did everything in her power to prevent full FDA authorization.

In response to this international incident and the public’s increased awareness of the risks of widespread use of drugs before adequate testing, Congress passed the Kefauver-Harris Amendment in 1962. This legislation required that manufacturers seek the FDA’s affirmative approval before new drug products are sold to the public, mandated that manufacturers demonstrate with substantial evidence the efficacy of new drugs prior to approval, and gave the agency the power to regulate manufacturing standards and facilitates, among other new powers.

HIV/AIDS, the AIDS Coalition to Unleash Power, and Patient Communities’ Input at FDA

Facing a federal government that willfully ignored and trivialized the deaths of people with AIDS, the trailblazing activism of ACT UP (AIDS Coalition to Unleash Power) demanded that government officials pay attention to an emerging national epidemic that threatened the health of gay people and others with AIDS. Activists went to heroic lengths to fight for more patient input in the FDA drug approval process, leading the FDA to formalize opportunities for expanded access to investigational drugs during clinical trials and to develop a number of expedited development programs for new drugs treating serious or life-threatening diseases.

AIDS activists’ protests and campaigns overcame discrimination, stigma, and blatant disregard for human life to leave a lasting imprint on the FDA. Their brave actions helped promote the role of patient voices and advocates in the drug development process.

Leaving It Better Than We Found It: The COVID-19 Pandemic’s Impact on the FDA

In the middle of a global pandemic that has taken hundreds of thousands of lives in this country, the FDA is facing the gravest crisis of faith in its history. The sitting president of the United States recently seemed to accuse “deep state” actors at the FDA of impeding the development of vaccines and therapeutics. In August, at a press conference organized to tout the administration’s achievements in addressing a pandemic that is killing thousands of Americans each day, Commissioner Stephen Hahn incorrectly overstated the potential benefits of convalescent plasma as a treatment for Covid-19 (the agency fired its recently-installed chief spokesperson afterward).

Perhaps most alarmingly, Commissioner Hahn has issued emergency use authorizations for Covid-19 treatments with virtually no evidence supporting them and appears willing to do the same for a Covid-19 vaccine candidate before Phase III trials are complete. This is hard to square with the commissioner’s prior commitment to sign off on widespread distribution of a Covid-19 vaccine only “once studies have demonstrated the safety and effectiveness of the vaccine.

In spite of the damage that the last few months have done to the agency’s integrity, the novel coronavirus pandemic can still leave a positive legacy at the FDA. As I put it in a blog post in June, the FDA needs to get back to basics. Transparency, integrity, and rigorous evaluation of drug safety and efficacy are the bedrock of the FDA. Diverging from these tried-and-true principles, such as by issuing an emergency use authorization for a Covid-19 vaccine candidate without clear emerging evidence of its benefits outweighing its risks, would jeopardize both the public health and the public’s trust in the agency.

Research for this piece was supported by the Laura and John Arnold Foundation.


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