Off-Label treatments or drugs are used in a way that does not strictly adhere to or follow FDA guidelines. Normally, you would find prescribed drugs in the U.S. with an FDA-approved label. If an FDA-approved drug is used outside of its permitted labeling, it falls under ‘off-label treatment.’ Let’s discuss how off-Label treatments are researched via patient registries in the US.
Many people are surprised to learn that many off-label treatments are prescribed if a physician considers it important for any medical treatment or function. In fact, off-label is commonly used under one circumstance or the other. Despite this widespread use, many people are still unaware that they are using off-label drugs.
Some commonly used off-label drugs include:
- Prazosin, which is prescribed for hypertension, but is also used for nightmares
- Memantine for OCD
- Quetiapine to treat insomnia
Use of Patient Registries in Off-Label Treatments
Many people wonder that if an off-label drug is so efficacious, why is it not able to procure government approval? The answer is simple. Acquiring FDA approval is a long and costly process. Moreover, several generic products do not have the necessary funds to complete FDA-approval studies. Due to these financial constraints, many drugs are not able to covert from off-label to on-label.
However, this doesn’t mean there is no accountability or evidence-based research behind the prescription of off-label treatments. Patient registries are often used to gain more information about off-label treatments.
· Efficacy of Off-Label Treatments for Disease-Specific Conditions
The primary purpose is to examine the effects of off-label drugs on a larger patient population. This helps to identify patient or disease-specific conditions where off-label treatments could be more beneficial in comparison to traditional methods. This holds especially true for rare diseases and cancer, where off-label treatments produce better results than the standard procedures.
· Product Safety
Research about off-label treatments can establish product safety, which is perhaps the most important consideration. Patient registries that test a specific condition will help assess if any negative outcomes were reported on patients that were given off-label treatments. This can help make a case for their future use and also secure funding to obtain an FDA approval.
· Discover New Uses
A product probably gained FDA approval to treat a certain condition. However, patient registries can perhaps test their effectiveness (or lack thereof) on other treatments. If a drug is approved for the treatment of condition A doesn’t mean it is also approved for condition B. For that, you will need to assess patient reported outcomes recorded in patient registries. For example, a chemotherapy drug used to treat one type of cancer may report positive outcomes in treating another type of cancer.
Most physicians and medical experts argue that the benefits of off-label drugs largely outweigh the risks, if any. Off-label treatments have considerable scope, especially for treating conditions like cancer and rare disease where all other conventional options have been exhausted. Perhaps the only major risk of off-label treatments is the threat of a lawsuit from an unaware patient. With patient registries, off-label treatments can gain a vote of confidence regarding their safety, their multiple uses, and dosage.