The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has given its first opinion on the compassionate use of a medicine. Based on a request from Finland, it relates to an intravenous formulation of Roche’s flu prevention and treatment product Tamiflu IV (oseltamivir).
In its opinion, the CHMP states that compassionate-use Tamiflu IV should be considered only to treat critically-ill adults and children older than one year of age having a life-threatening condition due to suspected or confirmed pandemic (H1N1) infection or infection due to seasonal influenza A or B virus and answering to the following criteria: – patients not responding to either oral or inhaled authorised antiviral medicinal products; or – patients for whom drug delivery by a route other than IV (eg, oral oseltamivir or inhaled zanamivir) is not expected to be dependable or is not feasible.
For infants below one year of age, no recommendation can be given at this stage due to the absence of pharmacokinetic and safety data on the use of Tamiflu IV in this very young population, it says and, should a physician decide to treat an infant aged less than one year, the decision should be taken based on the assessment of the benefit and risk for the individual.
Oseltamivir is already authorised in the European Union (EU) as Tamiflu for oral use and, usually, authorised medicines are excluded from compassionate use programmes. However Tamiflu IV, which is not authorised, is a new development for intravenous use and for a new target population.
Compassionate-use programmes seek to give patients with a life-threatening, long-lasting or seriously disabling disease who have no available treatment options, access to treatments that are still under development and that have not yet been authorised. In the EU, the conditions of such programmes are covered by Article 83 of Regulation (EC) No 726/2004.
They are coordinated and implemented by the EU member states, which decide independently how and when they should be opened. Doctors who wish enroll a patient on such a programme must contact the relevant national authority in their respective country and follow the procedure that has been set up. The national authority keeps a register of patients treated with the medicine within the programme.
The CHMP can provide recommendations to all member states on how to administer, distribute and use medicines for compassionate use, and identify which patients may benefit from such programmes. It can provide these recommendations at the request of a member state, and also when it becomes aware that compassionate-use programmes with a given medicine are being set up in a number of member states.
However, the EMA emphasizes that these recommendations are optional and complement national legislation – they do not replace it, nor do they create any legal framework in the member states.
The Agency also cautions against confusing compassionate-use programmes with treatment on a “named-patent basis,” under which doctors can request a supply of a medicine from the manufacturer, to be used for a patient under the physician’s direct responsibility. In these cases, manufacturers record what they supply, but there is no central register of the group of patients being treated in this way, it notes.
The EMA website will provide a list of compassionate-use opinions adopted by the CHMP.
