The Associations welcome the European Commission’s decision to publish the results of its study on off-label use in the EU as an effort to offer clarity and transparency in relation to this practice.

The report provides useful information on the drivers of off-label use. In particular, it acknowledges that, beyond purely therapeutically motivated decisions, off-label use of medicines at the healthcare system level can also be driven by financial reasons. Whilst we accept that off-label medicines can be prescribed by physicians to address a patient’s concrete medical needs with his or her consent, we strictly oppose the promotion of off-label use for economic reasons which contradicts the EU principle that the protection of public health must unquestionably take precedence over economic considerations.

The report points out that the lack of incentives for pharmaceutical companies to extend the labelling of their existing products is one of the drivers of off-label use. It is important to note that, from our point of view, this trend is reinforced by the promotion of off-label use for economic reasons by Governments. Indeed, pharmaceutical companies may be less ready to invest in costly and lengthy clinical development and authorisation processes for a given indication if public authorities promote the use of cheaper off-label medicines that have not been subject to the same stringent safety and efficacy assessments as existing on-label medicines, for financial reasons.

The report highlights the negative impact of off-label use for the regulatory system mentioning that it creates uncertainties regarding the liability of healthcare professionals, undermines the EU market authorisation process and discourages R&D. However, we regret the lack of emphasis in the report on the fact that off-label use poses additional risks for patient safety which appear all the more unacceptable when alternative on-label treatments are available and not used for financial reasons.

The clear consequence of not controlling the promotion of economic off-label use is the institutionalisation of double standards regarding the documented risks and benefits of medicines. On the one hand, the regulatory process imposes strict regulations to ensure the safety and efficacy of medicines available on the market; on the other hand, off-label use for economic reasons overrides the regulatory approval process by promoting medicines, which have not been subject to those stringent requirements. 

The report argues that one of the advantages of off-label use for healthcare systems is that “budget control at Member States level and the sustainability of healthcare systems can be reached by prescribing the cheaper off-label option”[1]. Whilst we fully recognise the affordability challenges faced by healthcare systems under pressure from rising healthcare demand and are working with national stakeholders to find appropriate solutions, we cannot accept measures that could compromise public health. 

The last chapter of the study describes policy options that could be used at EU or Member states level to address off-label use. In light of some of the measures presented in the report, we would like to emphasise the fact that the reimbursement of off-label medicines for economic reasons when on-label products exist is not an acceptable policy option. We therefore call on decisions makers to put patients’ safety and the integrity of the regulatory regime first and not to support measures that would set double standards in terms of regulatory requirements for on-label medicines and off-label medicines.