One would think that having a full understanding of a drug’s profile would be crucial to have before unleashing such a compound to patients around the world. Surprisingly, however, some are espousing the view that the U.S economy is being shackled by overregulation and that the Obama administration could stimulate economic growth by “lowering unnecessary barriers facing innovative U.S companies that are trying to bring new products to market.” For potential new drugs, the overregulation in question is the requirement to conduct late stage clinical trials to show the risk-benefit profile of the new medicine.
Critics of the pharmaceutical industry would deem such a proposal as another example of this industry’s attempt to exploit patients for profits. Ironically, it is not the pharmaceutical industry pushing this view. It is being brought forth by Dr. Andrew von Eschenbach, a former commissioner of the FDA and Dr. Tomas Philipson of the University of Chicago.
Von Eschenbach and Philipson have recently proposed a system in which a “drug could come to market after promising early-stage research in targeted patients, with appropriate post-marketing studies required”. In other words, if a drug works in 500 – 1000 patients, the FDA should allow this compound to be marketed to the patients who could benefit from it – which could be anywhere from 10,000 to millions of people. The company would then run surveillance studies to be certain that no unusual side effects arise during its use.
What are the benefits of this newly proposed system? This would eliminate the costly late stage studies (phase 3) that are now required by the FDA. These studies make up at least 25% of the overall R&D spending for innovative companies. Furthermore, drugs would be approved at least 3 – 4 years sooner, thus extending the amount of time a drug can be marketed on its existing patent. Presumably, these saving and added profits would be ploughed back into funding new R&D programs, thereby generating greater medical innovation.
This all sounds great, but what about unforeseen side effects? The authors handle this in the following way.
“Companies would still be liable for unforeseen side effects, but patients and doctors would be warned – through the drug’s labeling – that the product had been approved based on promising but provisional research.”
This is not the first time that von Eschenbach has made this proposal. He first discussed this in a Wall Street Journal op-ed last year . He based his arguments back then on the need for the U.S. to remain a leader in medical innovation and that the FDA’s clinical trial requirements were restricting this potential. Philipson and von Eschenbach frame their arguments this time on the belief that their proposal would help to stimulate the U.S. economy via the biopharmaceutical industry’s enhanced success.
This proposal was flawed last year and it is even more flawed today. I have no doubt that their system will result in reducing the money spent in getting new drugs to market. But phase 3 trials are crucially important for a number of reasons.
1) Phase 3 is a not a glorified efficacy trial. Rather, it exists to help to define the risk and benefits of a drug. You really can’t do such an evaluation solely on the results of a small subpopulation of patients.
2) Only with a full risk-benefit profile can the FDA judge whether a drug merits approval. A significant side effect for an obesity drug, which potentially would be taken by millions, would likely make its use untenable, whereas this same side effect for a new pancreatic cancer treatment may be perfectly acceptable given the life saving nature of the latter medicine.
3) Phase 3 studies are also needed to compare the efficacy of a new drug with existing therapies. How are doctors and patients to know whether a new drug should be used without knowing this?
The von Eschenbach/Philipson proposal, if adopted, would likely result in more drug recalls and more liability costs, resulting in a decrease of confidence in the work of pharmaceutical companies on the part of physicians and patients. Critics of the industry already challenge (wrongly, in my view) that new medicines offer little advantage to older products and may even be less safe. Eliminating phase 3 studies would erode the attempts that the industry is making to restore its image.
I, for one, feel that the FDA does a good job in evaluating the risks and benefits for new drugs under the current paradigm and I don’t believe that they are being restrictive. Hopefully, they will not pay heed to the advice of their former colleague. I also think that the pharmaceutical industry would benefit by disassociating itself from this proposal. Silence on this issue may cause people to assume pharma support.
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Source: Forbes (LaMattina, 3/5)
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