What is a ‘special’?
‘Specials’ is a term used for a range of medicines that do not hold a marketing authorisation in the UK, and which are offered to a patient when the physician has identified a clinical need that cannot be met by any available licensed alternative. It covers the following:
- Medicines manufactured by a holder of a Manufacturer’s Specials (MS) licence from the MHRA in multiple quantities with end-product analytical testing
- A bespoke medicine produced by a MS Licence holder that has not undergone end-product analytical testing
- An extemporaneously prepared medicine made in a pharmacy under a pharmacist’s direct supervision (a MS licence is not required for this)
- A licensed medicine imported from another country which does not have a UK licence
- An unlicensed medicine imported from another country
EU Directive 2001/83sets out the legislative framework for medicines and its purpose to protect public health. It requires that only medicines with a positive risk-benefit ratio, determined through an evaluation of evidence of their safety, efficacy and manufacturing quality, may be granted a marketing authorisation (product licence) and approved for use in patients.
In the UK, an unlicensed medicine may only be supplied in accordance with the provisions of Schedule 1 of The Medicines for Human Use (Marketing Authorisations etc.) Regulations 1994.
Schedule 1 provides an exemption from the need for a marketing authorisation for some medicines, because some patients may have special clinical needs that cannot be met by licensed medicines. To meet these special needs, the law allows the manufacture and supply of specials where:
If a special is manufactured in the UK, the manufacturer must hold a manufacturer's (specials) licence issued by the MHRA. A special may not be advertised and may not be supplied if an equivalent licensed product is available which could meet the patient's needs. Good Manufacturing Practice must be followed, essential records kept, and suspected adverse reactions reported to the MHRA.
Professional guidance supports the legal framework. The General Medical Council (GMC) advises  that before prescribers use medicines outside of their licence they must be satisfied:
“… that it would better serve the patient's needs than an appropriately licensed alternative”
“… that there is a sufficient evidence base and/or experience of using the medicine to demonstrate its safety and efficacy…”
The GMC Good practice in prescribing and managing medicines and devices (2013) provides guidance in a section for prescribing of unlicensed medicines which states unlicensed medicines may be prescribed on the basis of an assessment of the individual patient, where concluded, for medical reasons, that it is necessary to do so to meet the specific needs of the patient.
The RPS gives similar advice  to pharmacists dispensing specials, saying that appropriate standard operating procedures should be in place for dispensing services, which include the supply of a product with a marketing authorisation, where such a product exists in a suitable formulation and is available, in preference to an unlicensed product.
There is a guidance by the RPS for prescribers of specials  which is based around principles that can be used to guide prescribing decisions and includes case studies that illustrate the challenges that are met to ensure that patients receive optimal treatment.
There are circumstances when NHS Trusts recommend the use of specials in preference to a licensed medicine for the same condition. In some cases, this is on the basis of relative cost.
A judgment in 2012 in the European Court of Justice 8 has helped to clarify the legal position on government-sponsored organisations advocating the use of unlicensed medicines on cost grounds by finding that exceptions relating to special needs apply to therapeutic and not financial considerations.
Similarly, in 2018 is the case  specifically around the choice to prescribe an unlicensed medicine based on the grounds of cost, as evident in the scenario of the Judicial Review decision on access to medicines for wet Age-related Macular Degeneration (AMD). There are potentially significant implications for the regulation of medicines and patient safety arising from this judgment.
The ABPI position on the promotion of off-label or unlicensed use of medicines by healthcare bodies highlights the following:
Based on the above points, any approach that is in favour of off-label or unlicensed supply of a medicine for economic reasons only (that is, in the case where alternative medicines licensed for that indication are available) consequently puts the patient at a potentially higher level of risk and this stands in contrast with existing regulatory guidance in the UK and is not keeping with the system for the regulatory approval of medicines in Europe.
Therefore, it raises concerns as it relates to patient safety, and the role of the regulatory systems in protecting patients and ensuring safe use of medicines as well as risk management plans and ongoing pharmacovigilance after a medicine is licensed.
 Directive 2001/83, pages 67-128. (European Union, Nov 2004)
 Schedule 1 of The Medicines for Human Use (Marketing Authorisations Etc.) Regulations 1994 [SI 1994/3144]
 Guidance: Supply unlicensed medicinal products (specials) (HM Government, MHRA, Oct 2018)
 Good Practice in Prescribing Medicines (GMC, Sep 2008)
 Good Practice Guidance on: The Procurement and Supply of Pharmaceutical Specials (RPS, Jun 2011)
 Prescribing Specials: Guidance for the prescribers of Specials (RPS, Apr 2016)
 Judgment in Case C-185/10 (Court of Justice of the European Union, Mar 2012)
 Judgment in Case No: CO/5288/2017 (Royal Courts of Justice, Sep 2018)