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Focus on Regulation

EMA updates guidance concerning medicinal products intended exclusively for non-EU countries

The European Medicines Agency (“EMA”) has updated its guidance (Guidance) regarding procedural advice for medicinal products intended exclusively for markets outside the European Union (“EU”). The Guidance addresses several questions which applicants requesting a scientific opinion as provided in Article 58 of Regulation (EC) No 726/2004 (“Article 58”, the “EMA Regulation”) may have. Article 58 of the EMA Regulation provides that EMA, in co-operation with the Word Health Organisation (WHO), may provide a scientific opinion concerning certain medicinal products for human use. These medicinal products are intended exclusively for markets outside the EU in which population is largely low and middle income.

The Guidance also provides information regarding issues that are commonly discussed during pre-submission meetings related to requests for scientific opinions. Pre-submission meetings provide applicants with the opportunity to obtain regulatory and procedural advice regarding their application in accordance with Article 58 of EMA Regulation. Accordingly, EMA explicitly encourages the establishment of pre-submission meetings with the applicants concerning the evaluation of medicinal products that are intended for non-EU markets.

Evaluation under Article 58 of Regulation (EC) No 726/2004

The main purpose of the scientific opinions, for which Article 58 of the EMA Regulation provides, is to assist in the promotion and protection of public health throughout the world. The Article 58 procedure, as provided in the EMA Regulation, is intended to facilitate the access of non-EU countries in which population is largely low and middle income to medicinal products which are intended to prevent or treat diseases of major public interest.

EMA encourages developers of medicinal products that target an unmet medical need or which are of major public health interest to seek the scientific advice provided by Article 58 of the EMA Regulation. A scientific opinion based on Article 58 of the EMA Regulation will provide applicants with the EMA’s views, within the context of the co-operation with WHO, concerning related scientific and regulatory questions concerning the targeted population and market. Following this procedure will, therefore, facilitate the development of the medicinal product.

In particular the EMA’s Committee for Medicinal Products for Human Use (CHMP) may co-operate with the WHO on matters related to medicinal products intended exclusively for non-EU markets. The CHMP, in the context of the co-operation with WHO, may provide its scientific opinion for the evaluation of the applications submitted to the EMA pursuant to Article 58 of the EMA Regulation. Consequently, the CHMP’s scientific opinion assessment report contains data concerning quality, safety and efficacy of such medicinal products. It further attains the benefit-risk balance of the concerned medicinal products applied for in the context of the targeted population and market.

The Guidance

The Guidance addresses a number of questions regarding the procedural steps that should be followed for applications submitted in accordance with Article 58 of the EMA Regulation. The Guidance provides answers to questions concerning the types of applications that may be submitted, the way in which the dossiers are submitted and related costs. The questions addressed in the Guidance can also be discussed during the pre-submission meetings with EMA.

The Guidance further mentions the eligibility criteria that should be fulfilled in relation to an application for scientific opinion based on Article 58 of the EMA Regulation. In particular, it provides that medicinal products eligible for evaluation on the basis of Article 58 of the EMA Regulation are those intending to prevent or treat diseases of significant public health interest. Such medicinal products, which are intended exclusively for the markets of non-EU countries include among others:

  • “vaccines that are or could be used in the WHO Expanded Program on Immunization (EPI);
  • Vaccines for protection against a WHO “public health priority disease”; and
  • Vaccines that part of a WHO-managed stockpile for emergency response”.

The Guidance provides that the eligibility of a medicinal product for evaluation under Article 58 of the EMA Regulation is assessed on a case-by-case basis.

The Guidance also mentions that the fact that an application has been submitted on the basis of Article 58 of the EMA Regulation does not mean that a future application for a marketing authorisation in the EU cannot be submitted.