Pharma associations take action over national European off-label authorisation laws

They could be a disincentive to pharma companies developing drugs for rare diseases

National laws within the European Union (EU) that promote the off-label use of medicines are illegal under EU law and could disincentivise pharmaceutical companies from developing certain medicines, the European Federation of Pharmaceutical Industries and Associations (EFPIA) has claimed.

Faraz Kermani, EFPIA External Affairs Communications Manager warned that medicines used for off-label applications have not gone through the same quality control as approved drugs, such as rigorous testing and reviews by the European Medicines Agency (EMA) and the European Commission.

'So you’ve done all that work, put in all that investment in order to come out with an end product that you know has the quality, efficacy, and ultimately the safety to be used in that particular condition. Then someone comes along and says… ‘this drug seems to be doing the same thing’ – that’s not good enough,' he added.

Medicines used for off-label applications have not gone through the same quality control as approved drugs

The EFPIA, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) and the European Association for Bioindustries (EuropaBio) filed a complaint on 31 August against a French law allowing the country’s medicines regulator (ANSM – Agence Nationale de Sécurité du Médicament et des Produits de Santé) to issue a temporary authorisation for a medicine to be used for an unapproved purpose for economic reasons, even if there is an authorised alternative treatment. The ANSM did not respond to requests for comment.

This follows another complaint filed on 28 January by the organisations to the Commission against a similar Italian law.

Kermani added that off-label use could jeopardise patient safety, disincentivise companies particularly from developing orphan drugs (for rare medical conditions), and could lead to confusion over which party is held legally responsible for the off-label use.

He argued that the new French and Italian laws were particularly aimed at allowing the use of Avastin – a cancer drug – for macular degeneration (wet AMD), although the more expensive Lucentis is approved for that use.

The EFPIA and its partners are awaiting a response from the European Commission.

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