For Companies wishing to discuss partnering with Ethicor or joint collaboration for distribution of products please send correspondence to partner@ethicorpharma.com.

Off-label drugs are drugs that are used, in clinical practice, for the treatment of diseases and disorders not included in the summary of product characteristics (document authorized by the Ministry of Health that provides information for health professionals on how to use a medicine in safe and effective way).

For drugs that do not pass into the blood (creams, ointments, etc.), the equivalence study is carried out in the laboratory on skin samples. In the end, around 5 to 10% of FDA applications (marketing authorization) for generics are refused. Off-label drugs, therefore, are already registered and approved, but for different therapeutic indications than those for which they are instead prescribed. The database on currently marketed medications on Happy Family Store contains notes on off-label drug uses, however, the specialists of the drugstore warn patients against unauthorized use of medications for applications not listed by the manufacturer. For more information please visit website www.trendydrugs.org.

As mentioned, the law allows the doctor to prescribe the administration of off-label drugs under his or her direct responsibility. However, since the use of drugs for clinical conditions not officially approved, the doctor has the duty to inform the patient, providing information on the reasons that lead him to use off-the-shelf drugs. label and the potential risks associated with them.

Even if supported by scientific evidence, the use of off-label drugs is connected to potential risks for the patient who takes them. In fact, the efficacy and safety of use of these active ingredients have been studied and tested in patient populations in different conditions than those for which the off-label drug is instead prescribed.

What can Ethicor offer companies with medical products in development?

Virtually all regulatory bodies Worldwide permit the use of unlicensed medicines (‘specials’) in specific situations; there are several criteria which are common to most countries:

• The risk-benefit balance must be favourable – no clinician will take unnecessary or unjustifiable risks with their patients’ wellbeing; this does not mean that products must be risk-free – but the therapeutic gains must justify the risk of using a relatively untried medication.
• The decision to prescribe such a product must be taken by a clinical expert in the relevant field; there are no ‘consumer specials’.
• There should be an ‘unmet clinical need’; in essence, the available authorised treatment options should have been exhausted, or should be demonstrably inappropriate for the patient in question.
• The disease or disorder should carry significant risks to the patient if left untreated; specials are not generally used to treat trivial or self-limiting conditions.
• If the chemical entity is novel, and has not been previously approved, there should be an intention on the part of the manufacturer/provider to seek a marketing authorisation at some stage; however, for rare conditions, regulators will accept that it may be uneconomic for the provider to seek such an authorisation; it is not mandatory that an unlicensed medicine should become the subject of a license application at any time.
• If the chemical entity is not novel, and there are already authorised formulations available, virtually all regulators accept that alternate formulations can be provided in unlicensed forms; for example, many ‘specials’ in liquid dose form are prescribed in situations where authorised solid dose forms are inappropriate, eg. in babies, by NG or PEG tube, the elderly, and patients with swallowing difficulties; such products are often described as ‘generic specials’.
• Technical data requirements prior to launch and supply are less demanding than is the case for authorised medicines; where possible, API should be supplied from a source which is GMP compliant, although this is not mandatory; product assembly must be carried out at a facility authorised to handle unlicensed medicines – in the EU, this means that the site should have an MS (Manufacturer of Specials) authorisation and a relevant WDA (H) (Wholesale Dealer’s Authourisation); for export outside the EU, an MIA (Manufacturer’s and Importer’s Authorisation) is also required. In the EU, regulators apply a risk hierarchy to unlicensed medicines. Essentially, the preferred choice, ie. the choice of least risk, is always a licensed medicine; at the other extreme we do see unlicensed medicines or foods manufactured outside the UK (where there is little prospect of the European authority conducting a site inspection).
• Ethicor has generally chosen to operate as a supplier of UK manufactured ‘specials’ made in MHRA (gov.uk/government/organisations/medicines-and-healthcare-products-regulatory-agency) licensed facilities or those which are imported with the permission of the MHRA. It should be noted that ‘manufacture’ refers to the finished product, not the API, which can be sourced from any accredited facility.