The European Commission wants to systematically promote the use of tried and tested medicines for new indications.

UM – 11/2021

On 28 October, the European Medicines Agency (EMA) and the heads of the competent national medicines authorities launched a pilot project to support the reuse of medicines. A suitable framework is being sought for the search for new fields of indication for existing active pharmaceutical ingredients, so-called "drug repurposing". The pilot project is based on a June 2019 proposal by an expert group of the European Commission on safe and timely access of patients to medicines (STAMP) and was announced in the Commission's Pharmaceutical Strategy Notification of 25 November 2020. A working group ("RepOG") has been set up for implementation.

No COVID-19 therapies sought

Drug repurposing has become a major issue in the context of the COVID-19 pandemic. Since the beginning of the pandemic, researchers the world over have been screening substance databases for their antiviral potential. However, the framework in which data is to be generated via the launched pilot project explicitly excludes COVID-19. Instead, the initiative addresses non-profit organisations (NPOs), having an interest in ensuring approval of a medicine, which is on the market and no longer protected by patent or other protection, for a new indication and thus, if necessary, also prescribed and reimbursed. The non-profit organisations can come from the scientific community, but they can also be patient organisations.

Target: supply at favourable prices

According to the STAMP expert group, it is important to provide interested and suitable organisations - so-called "champions" - with the necessary knowledge of regulatory requirements and processes, existing data and, where appropriate, data still to be collected, as well as access to information from clinical trials. In addition, help will be provided to find cooperation partners in the pharmaceutical industry who can ultimately market the product with a new indication. In brief: A systematic approach is needed to find new indications and to bring together suitable partners so that medicines that become cheaper after patent expiry remain available for a broader supply – just with a different indication.

Not every NPO is eligible

High expectations are placed on non-profit organisations. They must be able to shoulder a programme of research into the suitability of an active pharmaceutical ingredient for a new indication in order to generate new scientific knowledge. They must also be able to coordinate cooperation between medicines regulators, the industry and patient groups and provide them with new information on a regular basis. This is not an easy task and certainly not for small self-help organisations.

Not to be confused: Off-label use

The repurposing of active pharmaceutical ingredients is, in principle, nothing unusual, but common practice. One well-known variation is off-label use, but this is quite different from "drug repurposing". On the one hand, in the case of off-label use, medicines that are still patent-protected can be used beyond the scope of the marketing authorisation. Examples are often found in paediatrics or oncology. On the other hand, other liability regulations apply for the prescribing physician and additional requirements for patient information. In addition, the criteria for eligibility