Quality, safety and efficacy

Any medicine that meets the definition of a medicine but does not have a Marketing Authorisation valid in the UK, is an unlicensed medicine.

Licensed medicines undergo full evaluation by the MHRA as part of the licensing process. Pharmaceutical quality, safety and efficacy are assured. Unlicensed medicines are outside the MHRA medicines licensing process, so pharmacists and prescribers must take additional steps to assure themselves of the quality, safety and efficacy of all the unlicensed medicines that they prescribe, purchase, make and supply.

The most common types of unlicensed medicines are described below.

Specials

Specials are medicines manufactured in the UK by the holder of a “Specials” Manufacturing Authorisation (MS) licence, or imported medicines that do not have a GB Marketing Authorisation.

Specials may only be prepared and supplied (often referred to as ‘placed on the market’) under an exemption from the Human Medicines Regulations 2012 (SI 2012/1916). This is further explained in MHRA’s Guidance Note 14.

Specials may only be supplied where there is no licensed medicine available to meet the special clinical needs of individual patients. They must be supplied only in response to a bona fide unsolicited order from an authorised person.

Special clinical need

Some patients have special clinical needs that cannot be met by the administration of medicines with a UK Marketing Authorisation.

Cost saving, convenience or operational need are not special clinical needs and Specials cannot be supplied for these reasons. Manufacturers, importers and distributors must obtain documentary evidence that there is a genuine special clinical need before they supply unlicensed medicines.

Individual needs

Individual patients sometimes need custom preparations, for example those with an intolerance or allergy to a particular ingredient.

Group needs

There may be whole patient cohorts for which a suitable licensed formulation is not available. Examples include:

• children who cannot swallow a tablet or capsule and therefore require an oral liquid
• those receiving oral medicines via a nasogastric tube
• the need for a smaller dose than is achievable using the required licensed medicine

Medicines supply disruption

In the case of disruption to supply of a licensed medicine, there may be a temporary special clinical need. For example, a patient group whose usual licensed medicine becomes temporarily unavailable, and where there is no suitable licensed alternative.

The Medicines Supply Notifications (MSNs) published on the medicines supply tool may include information about availability of unlicensed medicines. Unless specifically stated otherwise, these have not been evaluated for pharmaceutical quality or suitability to meet patients’ needs, so organisations must follow their local unlicensed medicines policies in the usual way.

UK manufactured specials

UK manufactured Specials are manufactured in the UK by the holder of a “Specials” Manufacturing Authorisation (MS) licence. UK manufactured Specials may either be supplied directly by the manufacturer or via the holder of a Wholesale Distribution Authorisation (WDA).

Although the MS holder is regulated and inspected by the MHRA, the products themselves are not independently evaluated for quality, safety and efficacy.

Our Purchasing Specials and Making unlicensed medicines pages provide more information.

Imported Specials

Unlicensed medicines may be imported by:

• the holder of a Wholsesale Distribution Authorisation that allows imports of medicines. Such importers are permitted to import medicines from specified authorised countries, such as those within the EEA
• the holder of a MS licence that allows imports of medicines. Such importers are permitted to import medicines from the rest of the world

Imported medicines may have a marketing authorisation from another country, or in some cases they may be unlicensed medicines in their country of manufacture.

The importer must notify the MHRA of the intention to import a medicine. The MHRA may object to any importation, for example, if there is concern it has not been manufactured following standards equivalent to Good Manufacturing Practice, or if they are not satisfied there is a genuine special clinical need. However, the lack of objection is not equivalent to approval of the medicine by the MHRA.

Our Purchasing Specials page provides more information.

Clinical trials IMPs

Investigational Medicinal Products (IMPs) are a pharmaceutical form of an active substance or placebo being tested or used as a reference in a clinical trial. These may be novel products, or may be medicines already with a UK marketing authorisation but:

• assembled (formulated or packaged) in a different way from the authorised (licensed) form
• used or administered in a different way from the authorised form
• used for an unauthorised indication
• used to gain further information about the authorised form

Although many IMPs are not licensed medicines they do undergo a formal assessment by the MHRA as part of their acceptance into the trial. This means that pharmaceutical quality is assured.

Our clinical trials pages provide more information.

Early Access to Medicines schemes (EAMS)

Some medicines made by the holder of a Manufacturing Authorisation (MIA or MIA(IMP)) are made available to patients as part of the Early Access to Medicines scheme.

EAMS medicines, although not yet licensed (authorised), do undergo a formal assessment of quality, safety and efficacy by the MHRA as part of their acceptance onto the scheme. This means that pharmaceutical quality is assured.

All medicines with a current positive EAMS scientific opinion are listed on the MHRA’s website.

Regulation 174

Regulation 174 of the Human Medicines Regulations enables the MHRA to temporarily authorise the sale or supply of a medicinal product in response to the suspected or confirmed spread of pathogenic agents, toxins, chemical agents or nuclear radiation which may cause harm to human beings. To date, the only example of this is the supply of the first COVID-19 vaccines before they were granted marketing authorisations.

Medicines made under Section 10 exemption

Pharmacists are exempt from licensing requirements pertaining to the provision of medicines if the medicines are prepared:

• in a registered pharmacy, hospital or healthcare premises
• by or under the supervision of a pharmacist
• in response to a prescription from an appropriate practitioner

Aseptically prepared medicines

Many hospital pharmacies prepare ready to administer injectable medicines for supply to their wards and clinics under Section 10 exemption from the Medicines Act 1968.

Aseptic preparation is a critical activity undertaken according to national quality standards. The governance and regulatory arrangements are explained by NHS England’s Assurance of Aseptic Preparation of Medicines

Our Making unlicensed medicines page provides more information.

Extemporaneously prepared medicines

Some hospital pharmacies prepare non-sterile oral and topical preparations for supply to their patients. These are commonly referred to as “Extemps”.

Our Making unlicensed medicines page provides more information.

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