The world is full of what-ifs. And while some ‘what if’ scenarios may be inconsequential, there are other circumstances where in ‘what could have been’ may have meant the difference between life and death. The time it takes for the Food and Drug Administration (FDA) to approve drugs might be one of those that fall under this life-or-death category.
Patients need help. And oftentimes, it’s the delay in providing that help which turns the situation from hopeful into hopeless.
There are many newly developed drugs that can’t be administered yet because the FDA hasn’t given the go-ahead to make these drugs available to the public. This delay in approval — it takes an average on “only” 12 years to get a new drug from the laboratory onto the pharmacy shelf — is done under the premise of making sure that the drugs are safe for patients and consumers to use. The question however is: can the FDA really guarantee that the drugs it approves are 100% safe to use? And when approval process is finally given, will it be done in time to help those it intends to help? If it takes more than a decade for a drug – if it makes it to human testing – to hit the market, the question needs no answering.
On the other hand, the FDA’s position is quite understandable because their number one priority is public safety. But then again, so is their obligation to issue timely and credible evaluations. Also, what about those patients who are literally at the end of their rope? The ones who are willing to try anything because they’ve already tried everything else and are at the point where they feel they no longer have anything to lose? If the drugs are already, say in an investigative state, shouldn’t these patients be given the chance to decide for themselves whether to take them or not at their own risk? Should the power to decide which drugs should or shouldn’t be commercially available really rest entirely on the hands of the FDA?
In an unpredictable move that is typical of President Donald Trump, the possibility that FDA regulations may soon experience a major shakeup looks imminent. First, Trump promised the country’s top drug manufacturers that FDA regulations will be slashed to make it easier for them to make their products in the U.S. Then he followed it up by saying that the FDA commissioner he plans to nominate — one name mentioned as a potential FDA pick is that of Silicon Valley investor and managing director at Mithril Capital Management, a venture capital firm run by Peter Thiel — is a ‘fantastic person’ who will support this endeavor, ultimately accelerating drug approval decisions.
Whether this will pan out or not remains to be seen. Because even if this results in faster drug approvals, it still means that it is the FDA that will make the decision. And those who are desperate to try drugs that are stuck in the approval process will still have no hope of finding out whether those drugs would have worked had they taken them considering that by the time the drug gets approved, it might be already too late. So the question ‘what if the patient was given the drug sooner, and would it have helped keep him/her alive’ will never be answered.
This might be the rationale behind some of those who are suggesting that instead of the FDA, why not let the consumer market, a Yelp for drugs type approach, make their own decisions about them? Just like other products, the only thing the manufacturers have to guarantee is that the product is safe to use. As for everything else including its effectiveness, there’s no guarantee, which means any person who takes the drug does so at his/her own risk.
But then, this might be just like giving the drug makers the license to concoct whatever they want. And even if their product isn’t beneficial in any way, as long as they are able to market it well, they’ll rake in the profits they’re aiming for. So where will that leave the sick? Probably in worse shape than before because in their desperation to get better, they might take in drugs that make them sicker.
Which brings us back to the FDA. Maybe their rules just need to change so drug approval can be expedited. And maybe Trump’s decision to make a major FDA overhaul might be the solution that’s needed after all.
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This is total horse manure. Here are some non-alternative facts.
1. More than 60% of all new drugs are first approved in the U.S. The “drug lag” is a 25 year old myth.
2. There are already procedures in place to allow patients in dire circumstances to use drugs that have not gone through the full FDA approval process.
3. Using a drug without proven efficacy is not a “victimless” crime. If the situation is, indeed, serious than it means _not_ using a drug in which there is some known benefit. So it means the harm of not treating a condition for which there is treatment.
4. For that and other reasons, safety and efficacy are inseparable. One of the other reasons is because, as pharma never stops reminding us, there are no drugs without dangers. That is true. So to use one with no known benefit is a polite form of Russian roulette.
“1. More than 60% of all new drugs are first approved in the U.S. The “drug lag” is a 25 year old myth.”
Despite a slightly decreased NDA/BLA filing count, the approval rate in 2016 is the lowest since 2010. This is on Califf and his “safety” push.
“2. There are already procedures in place to allow patients in dire circumstances to use drugs that have not gone through the full FDA approval process.”
Indeed. However, that does not make the drug commercially viable. No one develops drugs for this procedure.
“3. Using a drug without proven efficacy is not a “victimless” crime. If the situation is, indeed, serious than it means _not_ using a drug in which there is some known benefit. So it means the harm of not treating a condition for which there is treatment.”
Proven? By whom? There has to be evidence of efficacy and that level of evidence needs to be clearly communicated. They almost canned Sarepta’s drug, and the company itself, by refusing accelerated approval. The drug is now making real life changes.
“4. For that and other reasons, safety and efficacy are inseparable. One of the other reasons is because, as pharma never stops reminding us, there are no drugs without dangers. That is true. So to use one with no known benefit is a polite form of Russian roulette.”
But the FDA’s current practice is far from this extreme.
I wrote an article (41 Seeking Alpha pages) about solithromycin, a safe and effective drug which should have been approved. But it wasn’t, because of the FDA’s incompetence: http://seekingalpha.com/article/4044221-cempras-solithromycin-great-fda-echo-chamber
I read that Biogen has a drug that stops maybe reverses Alzheimer’s. Is it possible I could try it on my wife? She in later stage.
The manufacturer must always be in pursuit of improving his game. It is a high heads up game. I think, but I do not know, this investigation team is getting better about planning and implementing its game. The investigator almost all of the time does not work or report to the owner of the molecule/product under investigation. I am too far from the process. Whoever owns the horse in the race must be a very good analytical visionary in order to keep the road clean to prevent the horse from stumbling and to never ever let the horse fall. The horse must be “fresh” at all times. Collection of any and all data must be gathered and tabulated daily. Computer technology provides immediate awareness of all aspects of clinical trials. If the data is collected daily, tabulating & reporting takes more than 24 hours, that is an administrative issue. Every delay compounds itself by the end of the investigation. It appears every product in clinical trial should have its own FDA jockey. A successful clinical trial I would think would be a completed one. Novartis cardiac clinical trial was stopped early because of the very early successful results. This is a rare find. They must be looking for possible unplanned events whatever they may be. The public has demanded and received all of these rules and regulations. Events that occurred the same or less than placebo were not reported in the package insert. Today all events are reported. Even if one in 10,000 with drug and 10 in 10,000 with placebo. This has proven helpful to the physician in managing the patient. There have been a few molecules to make it to the market with delayed side effects, toxicity especially death, but this is rare for the FDA to allow today.