Navigating Labelling Exemptions: Strategies for Market Access (Part I)

European market is very granular in terms of national languages. In order to label all medicinal products in national languages and still fulfil commercial needs such as meeting MOQs (minimal order quantity), companies have to strategize their product supply well. When it comes to smaller markets, multilingual packages become the “go to”. But you can only fit as much information on the given pack size, thus how to ensure that patients receive medicinal products important to them, if their condition is not as prevalent and there might be no business case to have product with national language in their country?

There are several pathways which can be taken to introduce products to markets without meeting the standard labelling requirements.

Orphan medicine

In order to emphasize the importance of applying the labelling exception, it is important to start with orphan drugs, which are used to treat rare disease. Orphan medicinal products are intended for the rare diseases prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union. Medical and scientific knowledge about rare diseases is lacking, over 6,000 distinct rare diseases exist, affecting around 26 million people in the EU. To date, the European Commission has already authorized more than 200 orphan medicines for the benefit of patients suffering from rare diseases. The supply of orphan medicines in the market is low, corresponding to the need (market demand) for the proportional number of patients.

The assignment of the marketing authorization does not imply the immediate availability of the drug in all countries of the European Union. The owners of the marketing authorization must decide in advance how the drug is marketed in each country and the drug will then have to follow a specific procedure, in order to establish the methods of reimbursement and usually also its cost. Despite joint efforts, the heterogeneity of approach in different countries still makes access to orphan drugs by patients problematic.

In order to ensure that the product reaches the market and patients successfully, labelling exemptions apply to medicinal products in this area. Labelling can be one of the main obstacles preventing marketing of medicines in small markets.

Exemptions to the orphan labelling and package leaflet obligations in the centralized procedure

The request for a translation exemption affecting outer packaging should be submitted and reviewed by the QRD group. In case the QRD group grants the exemption, it will only be applicable for the printed materials. Labelling or package leaflet will still be translated in all EU official languages and published on the EMA website. The outcome of the QRD group decision will be reflected in the CHMP assessment report and in the EPAR published on EMA website (for requests made as part of a regulatory procedure) and in the QRD table of decisions. The validity of the exemption granted in the pre-authorisation phase is subject to the COMP confirmation of the maintenance of orphan designation after CHMP opinion and endorsement by the European Commission.

Applicants are advised to provide the following information:

• A detailed and comprehensive request justifying why it is not possible to market the product with the labelling and package leaflet information in the language(s) of the Member State(s) concerned or with the full particulars of the labelling and package leaflet. This should include detailed figures for aspects such as manufacturing issues, production volumes/forecasts, estimated number of patients treated per country (prevalence of the disease), distribution vs. cost implications for the applicant, information on language(s) to be used, handling of the medicine by the users etc.
• To specify exactly which components of the labelling will be affected. In addition, the applicant should explain how, in their view, the product will still be able to be distributed, stored, administered/used correctly and safely, when certain information is not to be provided at all or will be provided in a language(s) different than the one of the Member State(s) concerned.
• To submit the proposed mock-ups for the labelling components to be placed on the markets concerned, when applicable.
• In addition to the above, for translation exemption requests as per Art. 63(3) handled at national level, the latest version of the product information available, as well as the outcome of any other exemption request handled by QRD Group, should be provided. Please liaise with the relevant Member State(s) for the full list of documents to provide for this national request.

Timing

• For new marketing authorisation applications, all exemption requests handled by the QRD group should be submitted at the earliest at the time of submission of the Marketing Authorisation Application and at the latest when submitting the responses to Day 180 LoOIs or D90 LoQs in case of an accelerated procedure.
• For the review of requests at the QRD plenary meetings, the QRD group should receive the information at least 4 weeks prior to the meeting. For the review of the requests to be handled by written procedure, the QRD group will need 28 calendar days (4 weeks) to review them. This timetable might need to be revised in case of urgent requests, where the availability problem could lead to an out of stock situation, e.g. in small Member States.
• The MAH should liaise with the relevant Member State(s) for the timing for submission of exemption requests handled at national level.

Part II

In part I we provided an overview of labelling exemptions at European level. In part II we will provide outlook on labelling exemption regulation on national level and share experience and examples of some of EU member states approach.

Partner with Experts for Seamless Labelling Exemptions in the EU

Navigating the complex landscape of labelling exemptions for medicinal products in the European Union requires specialized knowledge and a strategic approach. Our team, with its profound expertise in regulatory affairs, stands ready to assist you in overcoming these challenges. Whether you’re dealing with orphan drugs or seeking solutions for small market distribution, we have the insights and experience to guide you through the process effectively.

Contact us to leverage our deep understanding of EU regulations and to ensure your products reach those who need them most, without delay. Let us be your partner in navigating the intricacies of labelling exemptions, demonstrating our commitment to advancing patient access and care across diverse markets.