Currently a multitude of pharmaceutical companies and research institutions are working feverishly to develop an effective and safe antidote for the viral pandemic sweeping the globe, especially in the United States. Research information is released in anticipation of relief from the ravages of COVID-19 and its personal, community and economic impact on all aspects of our daily lives.
Although many are poised to applaud and promote an immediate and “miraculous” fix, scientists are trained to draw conclusions from repeatable observations that demand a multitude of trials and long-term studies to separate fact from hype and fiction. It is difficult to be patient and watch this process unfold amid personal and economic distress when the stakes are so high.
There are many instances – past and present – of quackery and fraudulent claims in the health market. When Smallpox was prevalent, the discovery of a vaccine by Dr. Edward Jenner in 1776 was a miraculous event. An attempt by Congress in 1813, which passed the Vaccine Act, to monitor the purity of the vaccine was a step in the right direction, but ultimately not successful and later repealed.
In 1883, Harvey Wiley was appointed chief chemist at the USDA's Division of Chemistry and investigated tainted food products. This led to the Food and Drug Act of 1906 and regulation of pharmaceuticals and food products. Pushback from legal and political opponents to the law weakened it, but ensuing harmful products including a cosmetic called “Lash Lure” and an antibiotic sulfa drug, tipped the balance, resulting in the Food, Drug and Cosmetic Act of 1938, giving FDA pre-market review and control of efficacy claims.
Even as medical knowledge and expertise has changed dramatically in recent decades, lessons from the past can still give guidance and confirm the validity of a reasoned method to confirm the efficacy of something that will be widely disseminated for public use. The account of Thalidomide is a cautionary reminder that comprehensive study of the effect of any substance for our health requires extensive and thorough review. Introduced in 1957 as a safe sedative, by 1960 Thalidomide even rivaled aspirin in sales and was prescribed “off-label” for morning sickness in pregnant women.
As pressure mounted on the U.S. Food and Drug Administration (FDA) to approve use of this drug in the United States, doubts arose about its safety. FDA inspector Frances Kelsey was not convinced that data was sufficient and requested additional study before approval would be granted. With subsequent discovery of severe birth defects associated with Thalidomide, restrictions were tightened and the approval process for new drugs was extended under the Kefauver-Harris Drug Amendments Act of 1962.
In contrast, success of an injected polio vaccine, developed in the 1950s by Jonas Salk and a subsequent oral vaccine by Albert Sabin, has served to suppress that dreaded disease to near extinction.
Normally the testing and approval process can take up to 12 years. An unintended consequence of this process is the high cost and pricing of pharmaceuticals. The 21st Century Cures Act (December 13, 2016) relaxes requirements to accommodate an urgent need for weapons to combat new pathogens. Again, the public is faced with an existential dilemma. Cautious optimism about the advances of science and technology is warranted, but a careful examination of results and caveats is equally important.
The tragedy of Thalidomide is not the end of that story. It serves as a warning of how to utilize an important discovery. Despite its harmful side effects, thalidomide is FDA-approved for two uses today—the treatment of inflammation associated with Hansen’s disease (leprosy) and as a chemotherapeutic agent for patients with multiple myeloma, purposes for which it was originally prescribed off-label. Due to its suppression of inflammatory response, use of Thalidomide in the battle with COVID-19 is under review and holds some promise.
The question to be answered may not be the ultimate usefulness of a scientific discovery, but under which circumstances it should be applied. Let’s hope the rush to safety – our hope for an effective vaccine – is a reasoned and careful approach with adequate safeguards for our protection.