OFF-LABEL

Dona Josefa approaches the counter where I stand and, with a fluid gesture, places a medicine bottle in front of her as she gives me a “good morning.”

“Look, I was here this morning to pick up the prescription the doctor gave me, but this medication must have been mistaken.”

I look down at the packaging that I immediately recognize as tamsulosin. Although not properly dignified, I cannot help but smile.

First of all, it is important to confirm everything well. I search and find the lady’s prescription, I confirm the name of the patient and the name of the doctor, as well as the diagnosis that had been made: “kidney stones”.

“I’m glad I opened it before I took it … This says here it’s for the prostate!” She says as she takes the package leaflet out of the box and shows it as posing irrefutable evidence in the face of a criminal.

First of all, this situation should have happened soon after the medicine was given out. It is at this point that clarifications and possible misunderstandings must be clarified. Because of my failure, I apologized to the lady. Sometimes it happens that we do not even think that the person may not have been instructed by the doctor, and we forget to ask.

But as soon as that was settled, I was finally able to explain to the lady what an off-label prescription was.

Placing a medicine on the market is complicated. The duration and cost of the required clinical trials is a significant undertaking. This is why the medicines are so expensive, especially in the beginning, when the company is trying to recover the money invested in all studies and developments. These clinical studies are designed to prove the effect on a particular disease and not on all possible ones because the higher the ambitions of a clinical trial, the greater the accuracy required, and the greater the cost involved.

But medicines have side effects: literally, effects that occur beyond what is usually desired, and which demonstrate that the molecule acts at various levels in our body and that despite being recorded as side effects, have not been actively tested as treatments for other diseases.

I did not want to talk only about economic problems in this health issue … but the truth is that the two things cannot be separated and to be hiding the influence that the economy has (and indeed has!) On these subjects is also to be to oversimplify the subject and close the eyes to the real problems that have to be addressed.

Despite this, I will hardly be the best person to give a good perspective of what goes on in the pharmaceutical economy…

But I’m already rambling.

The point I wanted to get is simple:
  • Sometimes it is discovered that some medicines have uses beyond what was expected of them.
  • For one reason or another, these uses are not exploited by the company which has the rights of that medicinal product.
  • Despite this, some studies are done in one way or another, either by health professionals in their regular practice or by some entity that may be interested.
  • There is a certain degree of safety and efficacy in the use of the drug in cases not directly described in the package leaflet.

“I’m not aware of anything!”, exclaims Dona Josefa, clearly tired of such long explanation and so little relevant information.

When the doctor prescribed the medicine to the prostate, he had full notion that it was not part of the usual use. Despite this, based on some evidence and his clinical practice, he chose the drug specifically for his situation because he knows that it will help to improve his particular problem.

This has many advantages!
  • Reuse of molecules that are already tested for their safety, and cut some development costs.
  • They are alternatives that people sometimes need! For example, imagine that Dona Josefa was allergic to the medicine that has the desired indication or cannot take it for any other reason. Here is an alternative!
  • That is what makes medical research possible. Thanks to some off-label use these new applications are found, and new, very important knowledge is created to advance medicine and develop new therapies.
However, not everything is positive…
  • Every doctor, even though within the legality, is a little “left to his/her own devices” when prescribing off-label. Sometimes you can rely on good quality evidence, but not everything is perfect and, on the one hand, you have to build good studies that you support, on the other hand, we need to rely on good studies to prescribe safely and effectively. This catch 22 situation has to be well measured for each doctor and for each case.
  • The advantage of the approved uses is that they pass the very tight screening of the drug regulatory agencies (FDA, EMA, Infarmed, etc.). Using off-label studies may be good, but we do not always have this confirmation made by the official bodies.
  • During the dispensing by the pharmacist, sometimes we may not recognize the use the doctor wants to give the drug, and this may lead to not know how to give the best advice without contacting the doctor.
  • It is sad to see that certain off-label uses of some medicines do not acquire “official” status only because of economic interests. When what could be an opportunity to decrease treatment costs, increase therapeutic options and still advance scientific knowledge is not pursued because it involves costs without return.

Doctors are the most appropriate professionals to make these decisions, and have the legal possibility of doing so, with proper authorization. As we have seen, it is important that they do so, both for medicine in general and for the particular patient. But it is also something that has to be properly regulated to be done in the safest way. Usually this is done by reporting the uses of off-label medication to pharmacovigilance units that record and compile information to produce new and better knowledge based on practice.

“So what my pharmacist is telling me …” concludes Dona Josefa. “I just grew a prostate, and now I have to take it because it’s the prostate that’s blocking my kidney stones?” “…

Yes,” I say, giving up. “Yes, that’s right. Take the medicine and it will get better. If you want more clarification, you already know that you can consult your doctor or pharmacist.”

Writen by João.

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