Introduction
The United States have always been known to have one of the strictest regulations when it comes to protecting it's citizens from medical drug usage. The Food and Drug Administration (FDA) provides a barrier to market entry as well as strict guidelines with the usage of these products. Prescription drugs in particular require abundant evidence proving that it is safe to consume in clinical situations. However, in recent years, these regulations have been loosened, particularly when it comes to off label drug usage. Off-label drug use is the use of a specific medicine for unapproved symptoms, ages, dosages, or administrations. The drug can be administered to patients when the doctor believes it might help them with their symptoms beyond the officially approved list. [1]
Usually, when using a prescription drug for its approved use, one can expect that the FDA has conducted an evaluation of its benefits and risks for that use and that there is instructions how the drug should be used to maximize effectiveness. The drug labeling will include information in regards to the specific diseases and conditions that the drug is approved to treat, how to use the drug to treat specific diseases, and risks associated with the drug. [2]
[6]
[7]
Approved Drug Used For Unapproved Use [2]
According to the FDA, once the drug has been certified by the FDA, a doctor may prescribe the drug to a patient for uses other than the ones that have been approved and tested by the FDA when they see it medically appropriate to do so. This sort of behavior would be acceptable in cases such as:
1) There exists no drug approved for the specific medical condition
2) Patient has not seen any benefits of the existing and approved drugs
Benefits Of Off-Label Drug Use
Some examples of common off-label drug uses are:
Chemotherapy
A common example of this would be a chemotherapy used to treat one type of cancer can be used to try and treat another. One question to ask if given an off-label drug to treat a condition would be what are the benefits and risks associated with treating the medical condition with this drug.
Asthma
Asthma medications can be very helpful for people with lung conditions with similar symptoms to asthma.
Heart Attack
Most heart attacks happen when when cholesterol builds up in the coronary arteries rupture. When they do they attract platelets to their surface, which can trigger blood clotting. Aspiring is used to inhibit the platelets.
Chronic Pain
Neurological drugs can be used to treat people that have chronic pain
Adult Vs Children Medication
Prescribing a drug to a patient which the drug was not originally meant for. An example of this would be a medication approved for adult use which is prescribed for a child. This is very common practice as lots of drugs are not able to be tested on adults due to the fact that testing on children is very hard to achieve due to the very strict laws that govern it. However this does not mean that it will not also treat a child patient.
[8]
Regulations In Europe [4][5]
Off-label uses cannot be avoided for drugs as there will always be situations where it is needed. Although EU legislation does not regulate off-label usage, it is well aware that it exists and as a result states that marketing authorization holders (MAHs) are responsible to provide all available information on their products including the results of clinical trials or other studies.
The level of evidence to prescribe and use a product off-label use may differ. Sometimes, evidence of efficacy and safety is available, but the pharmaceutical company does not take steps to extend the market authorization for cases such as in children. [4][5]
The European Medicines Agency (EMA) makes clear that medication errors should be clearly distinguished from patient misuse of a drug. EMA officials made clear of the distinctions between the off-label use of a medicine that results in harm to a patient and such off-label use that does not result in harm. The obligations of MAHs in relation to the collection and reporting of information related to the off-label use of drugs resulting in harm includes:
- Reporting of individual cases of off-label use associated with suspected adverse reactions
- Periodic reporting of the clinical importance of risks related to the off-label use of a medicinal product
- Risk management planning based on the quantification of off-label use in the context of particular risks and concerns.
[9]
Questions To Consider [2]
It is important that doctors discuss off-label drug options with their patients before prescribing it to them. Some questions that should be thought of are:
What is the drug approved for?
Are there other drugs or therapies that are approved to treat my disease or medical condition?
What scientific studies are available to support the use of this drug to treat my disease or medical condition?
Is it likely that this drug will work better to treat my disease or medical condition than using an approved treatment?
What are the potential benefits and risks of treating my disease or medical condition with this drug?
Will my health insurance cover treatment of my disease or medical condition with this drug?
Are there any clinical trials studying the use of this drug for my disease or medical condition that I could enroll in?
[10]
Bibliography
1) http://www.nejm.org/doi/full/10.1056/NEJMp0802107
2) https://www.fda.gov/forpatients/other/offlabel/default.htm
3) http://www.rotlaw.com/legal-library/what-is-off-label-use/
6) http://www.stus.com/stus-cartoon.php?cartoon=hea0039
7) https://i0.wp.com/www.chrisbeatcancer.com/wp-content/uploads/2013/02/not-approved-text1.jpg
8) http://news.stanford.edu/news/2008/december3/gifs/label_600web.jpg
9) http://elliho.com/featured/off-lable-drug-usage-the-voodoo-of-science/