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.
Conclusion
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.
