Most of us think of the FDA as our protector, screening Big Pharma and all of their corporate research studies on every new drug and every new medical device for possible approval. FDA approval means something very important to us: an unquestionable level of safety, efficacy, and legitimacy. We feel sorry for people in countries that do not have such a high-level system of well-trained and well-trained critical assessors and inspectors. We fear for the public health and welfare when money and profits seem to take priority. From time to time, those of us in the medical community will hear complaining that the price for all this security is too high. That the approval process takes too long (a year or more?) Or costs too much to go through (often close to a million dollars for a single device or drug?) And that this inhibits manufacturers from producing things that, Although beneficial, they do not win. Generate enough to recoup all that time and money in a timely manner. Is it possible that these complaints and other public and political pressures have affected the FDA in ways that could make us seriously question to what extent we can trust the FDA these days? The answer is, maybe yes.
In August 2015, Forbes magazine commissioned Biomed Tracker to analyze data on FDA approvals; This has obvious big implications for corporate leadership and success. Biomed Tracker is a recognized company in the pharmaceutical industry and among large pharmaceutical investors who rely on Biomed for pharmaceutical business data tracking and reporting. Forbes was looking at the industry from a business performance standpoint, but some pretty surprising information about the FDA came to light.
In 2008, the FDA rejection rate was generally around 50%. But in August 2015, the rejection rate was about 4.0%! How rigorous, demanding, and comprehensive do you think a final exam is for a college course with a 96% pass rate?
Many manufacturers are looking to generate more revenue for a drug or device by obtaining expanded FDA approval for additional applications or indications. These “new marketing claims” had an approval rate of approximately 50% in 2008. But by 2015, the approval rate had increased to almost 90%!
The odds maker would say this is almost like insider trading: a company with a new drug or new directions for approval has a high chance of getting it. Do you want FDA approval? The stock price is likely to go up just based on the odds of approval, is it time to invest?
What’s going on? It seems that the approval process has become more of a quick “rubber stamp” than it should be. Or could it be that the FDA is simply doing its job better?
There are those who say that the FDA is doing much better advising Big Pharma on what to expect from the research presented, the testing requirements, and so on. It’s like telling him what to expect to get a good grade on that class project or what to study because “he’ll be on the test.” But there is a fine line between this kind of supportive help and the actual and frankly training to manipulate the game, like telling you the two questions and the right vs. wrong answers.
The FDA has come under a lot of political pressure about meeting deadlines and generally looks less “negative” in its approach to its important work. Has this resulted in more efficient team, better protocols, and improved streamlined efforts, or has this pressure swayed the powers to cut the entire process, trim some corners, and err more on the approval side whenever possible? What do you think is the easiest, least expensive, and time-consuming way? What do we expect to see commonly when similar problems arise with other government agencies? A recently proposed bill, the “21st Century Cures Act,” is designed to streamline the speed of FDA approval and cut red tape, but who does this really benefit? Is public safety still priority one?
There has also been not-so-subtle public pressure: the public perception that a drug with even marginal benefit to those who need it, but which carries serious side effects, might be better than nothing. It is scary to think where this type of logic could lead those who decide on the fate of the approval of drug X. Not all of us would agree on what a true situation of “need” represents, and the same could be said regarding what (barring death) a “serious” side effect actually is.
Should approval standards be lower for a drug that treats a rarer or more serious health problem? The MIT researchers say yes. They say the FDA is “too risk averse” for the deadliest diseases. The researchers proposed creating a “risk threshold” for each disease that would lower regulatory standards based on prevalence, severity, lethality, and limitations of available treatment. But do we want a new chemotherapy drug that has a 50% death rate and terrible side effects, compared to a 60% death rate without any treatment or side effects? Maybe we want that opportunity, even if it is a long shot. California is considering a “Right to Try Act” that would allow patients with life-threatening conditions to have the right to access experimental drugs (ie, not approved by the FDA in any way). Perhaps it would be helpful in such situations if the FDA at least gives the drug a relative risk / benefit rating based on an analysis of all available data to help patients make a more informed decision.
And if something is not for the treatment of a life-threatening disease, but has very marginal evidence of benefit and absolutely no side effects (maybe some new laser to erase wrinkles, for example), does that warrant lower standards and a easier approval? Should the FDA allow Big Pharma to take hard-earned money for something that doesn’t really work, but “at least doesn’t hurt anybody”? A recent FDA proposal regarding the 510 (k) approval process for medical devices suggested just that. The FDA is considering exempting more than 100 medical devices from the approval requirements stating that these devices are “well enough understood” and the risks are “low enough.” But won’t that create a greater chance that we can all spend our money on nothing more than FDA-approved “snake oil”?
Do these names sound familiar: Vioxx, Accutane, Phen-Fen? These drugs are in a single group: FDA-approved drugs or devices, whose approval is revoked when serious problems arise after public use. But how unique is this group? Not so much! A recent internet search returned a list of 30-40 drugs that are in this “club”! More than half of these approved and then unapproved drugs come from the past ten years. More than half of them were recalled within 5 years of being fully approved by the FDA! How can strict testing and research, conducted at great length and depth by great minds, result in a drug that turns out to be dangerous in such a short time? It could be argued that when used for many years by millions of people, ultimately a very rare but dangerous side effect could be revealed. But a revocation of approval within just 5 years of public use really suggests that something might have been overlooked in the approval process. At least three FDA-approved drugs were withdrawn from the market in less than a year of FDA approval! What does that alone suggest about what the strength of our confidence should be in the approval process?