Medical regulations aren’t all red tape

The government body in Australia that regulates drugs, the Therapeutic Goods Administration (TGA), exists to make sure the drugs offered in the pharmacy are safe and actually do their job. But not long ago regulatory testing was less demanding than it is now. Unfortunately, it took a widespread scandal to shape medical regulations all around the world into the robust systems they are today.

 

The scandal that started it all

Most, if not all, medical experts and regulators know the story behind the thalidomide scandal.

It’s 1957, a wonder drug from Germany promises to cure insomnia, stress and anxiety, and even help with a mother’s morning sickness. Tests from the manufacturer show no problems in rats nor humans. Excitement for the drug spreads while it’s released in 46 different countries around the world. As the drug’s popularity soars, its manufacturer turns their sights on distribution within the United States. When thalidomide is submitted to the FDA for approval, one scientist, Dr. Frances Kelsey, sticks to her scientific guns and requests more testing data. The disapproval from Dr. Kelsey meant thalidomide was never released to the US public. Only a smaller portion of the population received the drug for clinical testing.

Dr. Kelsey receiving an award from President Kennedy for being a bad ass. Source: Wikimedia

 

Dr. Kelsey’s diligence saved much of the United States from the wide spread disastrous effects of thalidomide. Starting immediately after thalidomide’s release, babies were born with severe deformities. Babies, whose mothers had taken the drug, were not developing limbs properly and were often born with shortened or even absent arms and legs. In July of 1961 an Australian doctor, Dr. William McBride linked thalidomide to these horrific birth defects. Finally, in November of 1961 the drug was pulled from the pharmacy shelf worldwide.

Estimates suggest thalidomide caused 10,000 miscarriages. 8,000 of the 20,000 affected children, known as thalidomide babies, died within their first year of life. Those that survived still live with the consequences of poor medical regulatory procedures today.

 

Response to thalidomide scandal

Before the thalidomide scandal, drug regulations varied widely across the world. In response to the scandal, the United States introduced an amendment which requires a new drug to be tested for both efficacy and safety before approval. Today this requirement seems like a given but before this scandal clinical tests were only concerned with the safety of a drug.

Will a reasonable dose of this drug kill anyone? No? Cool, drug approved.

Now manufacturers must prove their new drugs are both safe and successful at what they claim it can do. With each new approval application, manufacturers must provide a large amount of data including safety, efficacy, dosing information, side effects, how long the drug stays in the body, and any other drugs that should be avoided while taking this new drug. Applications for approval can take months if not years. And they’re not cheap. Not to mention the long process of creating the drug first.

Some people think such an arduous process to get a drug approved stifles scientific research and medical breakthroughs. But, in reality they keep the public safe from dangerous drugs and shady manufacturers. Now, one can argue these demanding testing procedures also help to increase the already large cost of modern medical care but that’s a story for another post.


6 Responses to “Medical regulations aren’t all red tape”

  1. Tharaka Kaluarachchi says:

    I’d known about the thalidomide scandal, but had never heard of Dr. Frances Kelsey and her badassery – thank you for highlighting the work of this incredible scientist!
    We need to tell the stories of women in STEM, without them we would all be poorer.

  2. Alasdair Browning says:

    Thank you for your post, it is great to find out a bit more about the history of such an important regulatory institution. I think it is fascinating that if appropriate testing was in place, not only would thousands of children would’ve been saved but it could’ve led earlier to the realisation that it is useful for cancer treatment. This case reminds me a bit more about both aspirin and heroin: both created well before testing was required and with drastically different effects on health. With Aspirin being a very important pain and fever reliever and heroin causing so many tragic ill-effects on health.

  3. Kellen Lowrie says:

    I find the history of medicine to be absolutely fascinating. Thanks for the info about its enantiomer. That’s not a word I’ve heard since organic chemistry way back when I took it in undergrad. Thalidomide is still used in some cases against cancer which makes sense if its blocking blood supply.

  4. Nathalie Bolton says:

    Good choice of topic, Kellen! I This is a particularly interesting medicinal chemistry topic as it definitely goes to show the importance of drug testing. At the time, the structure of thalidamide and it’s effectiveness on insomnia was known, however the enantiomer (non-superimposable mirror image) of this compound blocks blood supply to organs which at the time was not known. Even if this was known, under physological conditions the enantiomers inter-convert so birth defects still would have occurred had the correct enantiomer been isolated and given. A horrendous example of insufficient knowledge about a drug before being distributed, and inadequate drug testing!

  5. Debbie says:

    I remember learning about thalidomide in high school. It horrified me then, and it still send shivers down my spine. It’s crazy to think we didn’t have rigorous approval processes/standards before this catastrophe. I count myself lucky that I live in this era, so I can be fairly confident the drugs I need will do what they say they do and nothing else!

  6. Sarah Nielsen says:

    Really interesting read, Kellen! It’s a shame that often it takes a huge catastrophe before people stop and think carefully about possible consequences, especially in the medical world where our trust is put in the medical personnel to try to keep us safe