Extension of Indication for Authorised Oncology Products in the European Union: A Joint Effort of Multiple Stakeholders.

Jorn Mulder,Hubert G M Leufkens,Anna M G Pasmooij,Anthonius De Boer,Violeta V Stoyanova-Beninska,Francesco Pignatti,Ciska Verbaanderd,Elias Pean,Just Weemers,Robin Verjans,Emile E Voest

doi:10.3389/fmed.2021.790782

Abstract

After marketing authorisation, the development of a medicinal product often continues with studies investigating new therapeutic indications. Positive results can potentially lead to changes to the terms of the marketing authorisation, such as an extension of therapeutic indication(s). These studies can be initiated and sponsored by the marketing authorisation holder (MAH) or by others. When results from an investigator-initiated trial suggest that an authorised medicinal product is safe and effective for a new therapeutic indication, physicians may want to treat their patients with this medicinal product. In such a situation, it is desirable to extend the therapeutic indication(s) via the regulatory approval process, as this can facilitate patient access within the European Union. There may however be challenges when the MAH did not conduct the study and might not have access to the data. In this perspective, we focus on the possibilities to extend the therapeutic indication(s) of an already authorised medicinal product based on results from investigator-initiated trials. We address: (1) the advantages of an extension of indication; (2) the regulatory requirements for a variation application; (3) investigator-initiated trials as a basis for regulatory approval; (4) the role of the MAH in extending the indication. With this article, we want to emphasize the importance of a collaborative approach and dialogue between stakeholders with the aim to facilitate access to effective medicinal products.

Highlights

After marketing authorisation, the development of a medicinal product often continues with studies investigating new therapeutic indications
When results from an investigator-initiated trial suggest that an authorised medicinal product is safe and effective for new therapeutic indications, physicians may want to treat their patients with this medicinal product
When results from well-conducted investigator-initiated trials establish that an authorised medicinal product can be used outside the terms of the marketing authorisation, patients should be given the opportunity to be treated with such a medicinal product

Summary

INTRODUCTION

The development of a medicinal product often continues with studies investigating new therapeutic indications. It is desirable to apply for an extension of the therapeutic indication(s) via the regulatory approval process, as this can facilitate patient access within the EU To initiate this process for (anti-cancer) medicinal products authorised via the centralised procedure, the MAH needs to submit a variation application to the European Medicines Agency (EMA). We consider it of relevance to further discuss the possibilities concerning the addition of a new therapeutic indication to an already authorised medicinal product based on results from investigator-initiated trials. Before submitting a variation application, the MAH could consider to request scientific advice from regulatory authorities to discuss the use of results from an investigator-initiated trial to support the extension of indication. There is a risk that the price of a medicinal product decreases following the extension of indication [22], which may represent a barrier for MAHs when considering the addition of a new therapeutic indication

DISCUSSION

DATA AVAILABILITY STATEMENT