The Medicines and Healthcare products Regulatory Agency is planning to reform the UK arrangements permitting healthcare professionals to commission an unlicensed medicine to meet the special needs of an individual patient.

The current provisions have been in existence for a considerable time and, given that much else has changed over the years, there is a clear case for reviewing how well they are suited to today’s healthcare context, says the MHRA, which is holding informal consultations on the issue and also calling for stakeholders’ views on its proposals.

The existing arrangements are set out in European Union Directive 2001/83/EC, which states that a medicine placed on the market must have received a marketing authorisation following demonstration of its safety, quality and efficacy. However, Article 5.1 of the Directive also permits member states to put in place national arrangements to allow an authorised healthcare professional to commission the manufacture of an unlicensed medicine to meet an individual patient’s special needs, under the health professional’s personal responsibility.

“Special needs” may arise for a number of reasons, such as: - no equivalent licensed medicine is available in the UK to meet the need; - a licensed medicine is available, but not in a formulation or presentation suitable for the patient; - the equivalent licensed product is likely to be unavailable for a significant period because, for example, the manufacturer is experiencing difficulties; and – a formerly licensed product is no longer available because of commercial considerations.

Among the issues the MHRA wants to tackle is the role played by medicines regulation in safeguarding patients who receive unlicensed products. The level of protection will clearly be lower than for licensed medicines but, it asks: how far should such protection extend?

“Known issues” with the current regulations include inconsistencies in the mechanisms that provide safeguards, in the regulatory approach to safety and in the feasibility of scrutiny as to whether a patient’s special need actually exists, it says. There is also, overall, a lack of clarity as what constitutes appropriate use of Article 5.1 arrangements and, therefore, the extent and type of monitoring that might be required.

Evidence that there may be important issues associated with this information gap is illustrated by the "volatility" in recent years in the number of import notifications received by the MHRA, says the agency; while these were running at well under 20,000 in 2000, they had risen to around 180,000 in 2006 but, following the introduction of fees for notifications in April 2007, the annual total has now dropped to around 60,000.

The review will not cover off-label prescribing of authorised medicines, and it is beyond its scope to make recommendations concerning prescribing practice in the use of unlicensed medicines, says the MHRA. However, it adds: “it is important that the review should be informed about the direction of developments on good prescribing practice. Any proposed reforms in medicines regulatory arrangements should facilitate rather than hinder good prescribing practice.”

The review will also need to be aware of the drivers that can significantly affect the need for and use of unlicensed medicines, says the agency. For example, industry rationalisation trends can have a strong effect on whether a company considers it commercially viable to seek or maintain a marketing authorisation for products for which there is a lower demand. Similarly, major changes in the regulatory burden relating to unlicensed medicines may have the effect, intended or otherwise, of shifting the balance of demand between licensed and unlicensed medicines. “It will be important to ensure that any proposed changes recommended by this review do not undermine the medicines licensing system and the safeguards it gives to patients on safety, quality and efficacy,” says the MHRA.