You might hear the terms “licensed”, “unlicensed” and “off label” commonly thrown around (by doctors) to describe drugs or medicines. However, these terms can be very confusing for patients and even other medical practitioners who don’t usually prescribe medications. That’s a shame as these terms are actually really important to understand the approval and use of medicines. This is especially important with the rise of telehealth platforms. These often use off label and unlicensed treatments such as compounded skincare and semaglutides. In this article we will discuss the differences between licensed and unlicensed drugs. We’ll also review the uses of unlicensed drugs and regulations surrounding off label prescribing in the UK, EU and USA.
The Difference Between Licensed and Unlicensed Medicines
A licensed medicine is one that has undergone strict clinical trials and assessments to ensure its safety, efficacy, and quality. On that basis it receives approval for its placement on the market for a specific indication. This is what we call marketing authorisation. In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for granting it. The European Medicines Agency (EMA) handles this for EU countries. Lastly, the Food and Drug Administration (FDA) is in charge of this in the USA.
It is really important to remember that licensed medicines only get approval for a specific use or condition based on evidence. Within this the licensing will include the exact dosage and administration route that the approval covers. It is not a blanket authorisation for every possible use of the medicine nor for different routes of use or doses outside of what is in the license. The biggest benefit of using licensed medicines is that they had thorough testing for the safety and effectiveness in the uses they got their license for. They also come with very clear dosage and administration guidelines. Additionally, there is ongoing monitoring and reporting of adverse effects even after the drug has approval for use.
So with all these benefits you might be wondering, why on earth does anyone bother with unlicensed medicines. Especially since these, in contrast, have not undergone the same level of regulatory approval for a specific use in a particular country that licensed drugs have. Unlicensed medications tend to fall into different categories:
- Imported Medications: These are drugs with a license in other countries. Under certain condition, healthcare providers can import them for patients even if they do not yet have approval in the country where they are.
- Compounded Medicines: These are special formulations or versions of medications that meet specific patient needs. This includes custom dosages, combination of drugs in one treatment or alternative forms like topical creams or serums instead of tablets. It essentially allows customisation of a drug to meet a patient’s individual needs.
- Off-label Use: This is probably the most common use of unlicensed medicines. A drug might be licensed for one condition. However, doctors may use it unlicensed for another indication even if it doesn’t officially have approval. So whilst the medicine itself is licensed, its use in a specific context may not be. Examples of off-label prescribing include using a higher or lower dose, administering in a different route or using the drug for a different condition than it has approval for. A good example of this is Tretinoin which is a licensed treatment for acne but is often prescribed off label for anti-ageing and hyperpigmentation.
Regulation of Unlicensed Drugs & Off Label Prescribing in the UK, EU & USA
To be clear, unlicensed does not mean unregulated. In fact there are strict regulations and rules around use of unlicensed medications. In the UK, the MHRA oversees the licensing of medicines but allows prescribing of off-label and unlicensed drugs under certain conditions:
- When no licensed alternative exists: Clinicians may prescribe an unlicensed medicine if no other licensed option is available or if the patient needs it in a different form.
- Off label prescribing in specific situations: For example, a drug licensed for adult use might be prescribed off label use for children if it is thought to be of benefit. Equally a doctor may prescribe a drug for a different disease than it has a license for if it could help treat it. Another example of unlicensed and off label drugs in the UK is Ketamine which is has off label uses in depression and post-traumatic stress disorder.
- Compassionate use: In some cases doctors can use unlicensed medicinal products that are still in development (ie experimental). This is permissable under strict conditions for patients who have a disease where there is no treatment and who cannot enter clinical trials
There are strict rules in the UK on prescribing, manufacturing and supply of unlicensed drugs. This aims to ensure responsible use of unlicensed medicines and prioritise patient safety. Clinicians must inform patients when they are prescribing an off label or unlicensed medication. They also need to explain the potential benefits and risks.
In the EU, similar guidelines apply under the EMA’s regulatory framework. Medications must meet the same high standards as in the UK, but individual member states can make provisions for off label or unlicensed use, particularly in rare diseases or public health emergencies. In the USA, off-label prescribing is legal and widespread, particularly in fields like oncology, psychiatry and paediatrics. The FDA also allows prescribing of unlicensed drugs when there is an official shortage of licensed drugs.
Uses of Unlicensed or Off Label Medicines
Doctors usually prescribe unlicensed medicines in cases where no licensed alternative is available. In these cases, doctors may prescribe unlicensed drugs. This is usually based on wider scientific research that might indicate it could meet the patient’s needs. A famous example off label drugs use is Gabapentin which originally had approval for epilepsy. It is now commonly prescribed off label for chronic pain and migraines. Similarly, Botox was initially licensed for treating eye muscle disorders. It was then widely used off label for cosmetic reasons before it got formal approval for some of these uses. Below are the most common reasons for using unlicensed medicines:
- Rare Conditions: For conditions that affect a small number of patients, pharmaceutical companies may not seek to license a treatment. This is usually due to limited commercial benefit.
- Customised Treatments: These compounded medicines are made by a specialist pharmacist to meet the unique needs of an individual patient. Doctors usually use them when a licensed drug isn’t available in the necessary form, strength or dosage. In dermatology, compounding allows use of a blend of ingredients to better target conditions like rosacea, acne or melasma. Compounded treatments are also crucial in paediatrics, where child friendly doses or alternative forms like liquids may not be commercially available.
- Emerging Treatments: Certain medicines may require use unlicensed due to their newness or lack of formal approval for all potential uses. An example of this is semaglutides like Ozempic. The inital license was for treating Type 2 diabetes because it mimicks the hormone GLP-1 which then regulates insulin and blood sugar. Since then semaglutides have shown benefit in reducing appetite and encouraging weight loss. Although the licence for Ozempic is for diabetes, many doctors now prescribe it off label for weight loss.
Understanding the distinctions between licensed, unlicensed and off label medicines is crucial for both healthcare providers and patients. Whilst licensed medicines undergo strict regulatory scrutiny, there are circumstances where unlicensed or off label use becomes necessary. In general unlicensed drugs have traditionally filled in the gap where commercial treatments may not be available or suitable. In the right circumstances, they allow doctors to provide effective care and better meet a patient’s individual needs. Off-label prescribing and unlicensed drugs are complex but vital to modern medicine and legal in the UK, EU, USA and much of the world. Whilst these unlicensed drugs do not have marketing authorisation, it does not mean that they are not subject to any regulation. In fact the authorities that normally license medicines provide strict rules around the manufacture, supply and prescription of unlicensed medicines and off label drug use.
At City Skin Clinic, we are passionate about personalised skincare. We offer safe and effective custom skincare treatments with prescription-strength ingredients. These include Tretinoin and Hydroquinone where appropriate through our online skin clinic. Our doctors treat a range of conditions such as acne and scars, hyperpigmentation, melasma, skin ageing. To start your personalised skincare plan, book a virtual video consultation or use our online consultation form. The journey towards great skin starts here.
