“Drug repurposing” is finding new therapeutic use of substances no longer protected by patent or regulatory exclusivity. This route is actively pursued by parts of the pharmaceutical industry. But what if there is a high unmet medical need and no commercial interest? This is something that has concerned the European Commission in Brussels.
Let us say that a certain product is being used in an indication for which it is not approved. The market authorisation holder (MAH) has no interest, from a business perspective, to perform studies and seek regulatory approval for the indication. It is difficult, if not impossible, for a non-profit organisation or research institute to develop a new product and take on the legal role as an MAH. So, the likelihood of the indication to be properly documented and approved is small. Why is this a problem? Well, firstly, an approval is a marker of “quality” since the benefit risk balance is assessed and deemed positive, secondly, an approval puts legal demands on the product owner to follow the quality, safety and efficacy over time, ensuring that the benefit risk remains positive.
The European Commission has provided a supportive framework to aid the development of new data. The framework is built on engagement from the organisation generating data, regulatory authorities supporting the organisation with regulatory advice and finally MAH compiling the data into a dossier and handling the submission to the relevant regulatory agency.
The targeted indication should be in an area where important public health benefits are likely to be achieved. There should be a valid marketing authorisation granted within EU and the substance should be off patent, with no other protections. To be accepted as a target project, there should be some supportive clinical evidence. To test the proposal, the non-profit organisation Anticancer Fund has identified candidates with interesting clinical data and is now considering approaching the principal investigator(s) of the relevant clinical studies to explore the possibility of jointly championing responsibilities.
Engagement from regulators in EU is key. Regulatory advice (through EMA and national agencies) is one of the main tools to support repurposing projects. At such an event, regulators are expected to give guidance on the regulatory and scientific aspects of the project. There is a risk, however, that regulators may be expected to go beyond the remit of the advice and actively participate as co-developers. This would seriously challenge the regulatory role as independent and unbiased and should be actively avoided from the agencies.
The intention behind the proposal is admirable, reducing off-label use and ensuring proper regulatory management post-approval. Industry representatives have been positive to the proposal. This could be an opportunity when data is derived with limited industry support. Hopefully, in the end, patients will be winners by receiving on-label treatments for which benefit-risk has been properly assessed.