As Dr. Simon pointed out in his first video, it is important to understand the history of the FDA when learning about regulation. One event I would like to highlight is the thalidomide scandal of 1962. Thalidomide was a sedative prescribed to many Europeans, many of whom were pregnant. As a result of the drug, thousands of children were either born dead or deformed. The children that were born deformed had phocomelia, or seal limbs. The chemical reason why this occurred was that the drug became racemic when sent to patients and contained both S and R enantiomers of the compound. The S enantiomer was tetragenic. It sparked several debates, including contraception, abortion, and, most notably, regulation.
Now I specifically said thousands of Europeans were affected. What about Americans? Well, there were only a few dozen cases reported of phocomelia as a result of thalidomide use. So your next question may be, why wasn't America affected as severely? Well, you can thank Frances Kelsey for helping to avoid the same disaster in America. Dr. Kelsey was working for the FDA at the time she received an NDA for thalidomide. She was unconvinced of the safety and efficacy of the drug and required more testing. Other officials at the FDA neglected the potential harm of thalidomide, so if not for her, we would have shared with Europe in one of the biggest drug catastrophes in recent history. After her intervention, she was awarded the Distinguished Civilian Service Medal in 1962. Because of her efforts, there was a huge push in Congress for further regulation. In 1962, John F. Kennedy signed the Kefauver-Harris Act, a law that strengthened the powers of the FDA to evaluate the efficacy and safety of drugs as well as requiring stricter guidelines for drug advertising.
So my question to you is, knowing all of this, what do you think made the Thalidomide disaster in the 1960's so crucial in strengthening the regulating powers of the FDA? How do you think the FDA would look like today if the Thalidomide disaster never happened?
Great question Talha,
The FDA's intervention to prevent the release of Thalidomide into the US market is critical to how important drug trials and long term effects should be considered. If had the drug been released,the US market would have experienced the same tragedies as the European Union. If thalidomide had been released, I believe that the events that followed would have resulted in a major strengthening in regulatory and scientific over view for future drugs. I believe that the outcome would have been the same had the drug been released, but the US market would've experienced the same tragedy as the European Union.
Chris
What the Thalidomide disaster did to the FDA was not only demonstrate the importance of drug trials, as Chris’s post pointed out, but, even more importantly, tightened surveillance on how these trials were conducted, their results, and the efficacy of the finished drug. Basically, it justified the need for rigorous regulation policies and practices. Unfortunately this meant that new drug development required several long years of testing before the drug could be released into the market. My follow up question is, do strict policies stunt medical innovation and economic growth? I am sure manufacturers and scientists confident in their development eager to see it be used think so.
If the Thalidomide disaster never happened some other similar disaster would have occurred eventually and inevitably. However, if no such disaster would have happened to this day then Dr. Kelsey would have been out of a job, there would have been no Kefauver-Harris Act, and there would have been more deaths in America caused by unsafe, poorly tested drugs and devices. This would mean more time and money lost at removing these drugs and devices from the market (then redesign, reintroduction, and convincing consumers that they got it right this time), grievance lawsuits, and reimbursements to the victims. As an answer to my own question, stricter policies actually foster medical innovation and economic growth.
The Thalidomide disaster was talked about in my undergraduate organic chemistry course. It emphasis the interpretation of new discoveries and most importantly allows us to see that not every new discovery works the way we see it at first. This is the reason major testing of any new discovery must be tested with multiple mediums. The drug was given in Europe because the experts lacked the understanding of the basics of science and different forms of it S & R. This disaster would of never had happened if the experts tested the drug more. The fact it was approved shocks us and we should appreciate the FDA rules and regulations and why they are strict. This was an historical event so it was given attention in the west. Regardless of if this event took place or not, many other drugs and discoveries happened and FDA had to strengthen. For example, it is didn't strict the rules and regulations, anyone with the rights skills can launch a product without testing and selling on the market with not so strict FDA rules. So this event does not matter.
I agree with what everyone else has said, that the thalidomide crisis played a pivotal role in shaping the regulatory authority of the FDA and creating more awareness to remain vigilant. Without the thalidomide crisis the FDA may have had a more lax environment in approving drugs that were already approved in other international markets, unless another similar tragedy occurred to shine light on the importance of regulating internationally approved phamarceuticals/medical devices. This goes back to the point that Dr. Simon's mentioned in the lectures as well that the FDA is known to be the most strict in regulation compared to similar international agencies. This crisis helped shape that aspect of the FDA as they still have strict guidelines for outside products even if they are already approved and used in other markets.
Before the implementation of the drug lot of trials must be done. This is the major impact of disaster, which caused in tightening up the FDA's rules. If this disaster would have not happened, the trials would have been performed without any proper measures in a large scale population. This large scale would have not been controlled unless the effect of this disaster affected tremendously. So it's a worth one that prevented us from the huge disasters.
My follow up question is, do strict policies stunt medical innovation and economic growth?
This is a really good follow up question to think about. I believe strict policies do stunt medical innovation and economic growth, but it is necessary. We as a society most definitely want to prevent tragedies like the Thalidomide one if we can do so (which Dr. Kelsey did) using strict policies. Lowering the strictness on policies can only lead to more companies taking advantage of it to push products out faster.
For economic growth, I'm no economist but I believe if we had a less strict system in place, a lot of the standards we have set would be much lower. We have a system in place that is meant to root out the weeds and allow the cream-of-the-crop-products to rise. At least theoretically. Even with our rigorous policies the DePuy scandal with the hip implant recalls still happened, so how strong and strict are our current policies really?
As the previous posts have stated, thalidomide played a big role in increasing the regulatory power of the FDA. However without this incident, the FDA would still most likely have developed strict regulations regarding the sale of over-the-counter drugs that have not been tested. This is because chiral drugs are very common and so if not thalidomide, some other drug would have caused a disaster similar to if not worse than the thalidomide babies. That being said, it was very fortunate that it was Dr. Kelsey who decided to question the effects and safety of thalidomide.
In response to Agnieszka's follow up question, strict policies do stunt medical innovation and economic growth to a degree. For the safety of the consumer this is necessary but it is also entirely possible that this system can make useful, safe drugs almost never reach the market. Being safe in this case opens up the possibility of companies pursuing safer option drugs that they know will get funding and will make them money over riskier drugs that need more clinical trials and may not receive funding.
The thalidomide disaster established a standard of how much testing must be performed before a product must be released. It builds integrity within the safety of the product, making sure that people are not negatively affected by the medicine. The Thalidomide disaster may have changed the course of the medical device regulation, however, there are more events that have happened through history that also changed regulation with the same magnitude as the Thalidomide disaster. If the thalidomide disaster did not happen, surely there would have been other drugs that could have negatively affected the public which the FDA would have regulated. One such event was the distribution of Elixir Sulfanilamide. It was a medicine that is used for streptococcal infections. It was safe in its inherent solid form however, has dangerous side effects when ingested in liquid form. It was distributed in solid form however, there was a demand for its liquid form which caused that population’s condition to worsen. If the Thalidomide disaster did not occur, this could surely be one of the events that would prompt the FDA to create regulations.
