Innovation and Regulation: A Cautionary Tale

The photographs in the loud, glossy magazines of the era were brutal, shocking, unforgettable.  We were shown babies with disturbing birth defects: severely shortened or absent limbs; flipper feet and hands; malformed bodies, heads and faces.  It was the early 1960’s – we had seen the vast human toll of nuclear explosions, remember, we had seen untold injured soldiers and prisoners and concentration camp survivors – but this was truly different.  This was the aftermath of what has been called ‘one of the biggest medical tragedies of modern times’.  This was the era of thalidomide.

Thalidomide was a promising sedative drug  in the 1950s.  Developed by a German pharmaceutical company, it was an equally effective painkiller and a treatment for insomnia, coughs and colds and headaches.  It was also thought to be a great solution to the problem of morning sickness – for some women, a very real, debilitating side effect of pregnancy.  At the time, most scientists believed that any drug taken by a pregnant woman would not pass the placental barrier or harm a developing fetus.  Thalidomide was thus prescribed to thousands of pregnant women to relieve their morning sickness symptoms.

It didn’t take long for the horror to unfold.  By the early 1960’s, more than 10,000 children (in 46 separate countries) had been born with the telltale deformities.  An Australian obstetrician and a German pediatrician both suspected a connection and the link between the drug and the birth defects was proved in 1961.  The drug was withdrawn.  But so much damage had already been done.

There were 2,500 thalidomide babies born in Germany.  In the UK, where the drug was approved and licensed in 1958, of the some 2,000  children born with defects, only 466 survived.  After a long and passionate campaign led by The Sunday Times newspaper, a compensation settlement to be distributed by the Thalidomide Trust was established in 1968.  These funds were increased in 2005 and again in 2009.

Here is the US, the story was different, but there were still victims.  Then, as today, drugs under investigation were distributed to physicians during a clinical testing program.  Millions of thalidomide tablets were offered to American doctors; patients also bought and took the drug while traveling outside the country.  The drug had to be acquired beyond US borders because it was never approved here.  And it was never approved here because of one smart, thoughtful and vigilant FDA medical officer: Dr. Frances Kelsey.

It was fifty years ago that pharmacologist and physician Frances Kelsey did her homework.  An application from the Richardson-Merrell company to market thalidomide in the US came to her attention.  As she investigated the drug, the reports of severe birth defects and deformity were  coming in from women all over the world who had taken the sedative.  She found out that the drug had never been tested on pregnant animals.  Dr. Kelsey then refused the application, stating that further studies and trials were needed.

What Dr. Kelsey did was honor both her training and education and the FDA’s real mission: she used science at its best to protect the American public while still promoting health and research.  In 1962, thanks to public demand, Congress enacted the Kefauver-Harris amendments to the Federal Food, Drug and Cosmetic Acts.  Now manufacturers  had to prove that any new drug was both safe and effective.  Safety standards were raised and good manufacturing practices emerged as further protection for the public.  The scientific standards themselves were raised, bringing the whole health care industry with it.

Regulation is not very popular today in some quarters.  It is deemed old-fashioned, reactionary, a hindrance to innovation, a drag on economic growth, even a violation of personal rights and values.  And maybe the wrong sort of regulation is indeed all of these things.  But not the right kind.

Regulation done right – with flexibility and knowledge and courage – means less litigation, enhanced consumer trust and the promotion of real science and achievement.  It doesn’t close doors, it opens them.  What happened when the thalidomide disaster was properly understood and analyzed gave us the way to bring to market medical treatments and drugs scrupulously tested for quality, performance, safety and efficacy.  Not a bad deal.

And one more thing.  Thalidomide has come back.  It has proven itself to be a safe and effective treatment for a complication of leprosy.  It was approved in 1998 in the US – after very rigorous testing and with a very, very strict safety monitoring and dispensing protocol – for use in that disease.


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