[co-author: Grégoire Paquet]

1. Introduction

On 21 August 2019, the European Medicines Agency (EMA) published questions and answers regarding imported advanced therapy medicinal products (ATMP). The document focuses on the possibility of batch controls exemption for ATMP imported into the European Union from a third country. It provides guidance concerning grant of the exemption, data to be submitted in order to justify such exemption and obligations of the qualified person for batch control.

2. Obligation to carry out batch controls

Advanced therapy medicinal products are medicines for human use that are classified into three types by Regulation (EC) No 1394/007 according to their basis: gene therapy, somatic-cell therapy or tissue-engineered medicines. Article 51 (1) (b) of Directive 2001/83/EC on the Community code provides that ATMPs imported from a third country must be re-tested upon importation by the qualified person for batch control. This qualified person must ensure that each batch has been manufactured in accordance with good manufacturing practice and that the quality is in accordance with the terms of the marketing authorisation.

3. The exemption

Article 52 (2) of Directive 2001/83/EC provides an exemption from the need to carry out re-testing by relying on controls conducted in a third country. The EMA’s Q&A document provides that such exemption may only be granted where the conditions laid down in paragraph 11.17 of the EU Good Manufacturing Practice guideline for ATMPs are met. These conditions are:

• only a limited amount of material is available; or
• the material has a short shelf-life; and
• the testing in the third country is conducted in good manufacturing practice-certified facilities or under conditions equivalent to those in the EU.

Requests for an exemption should be supported by a justification and by data to substantiate the claim made. This includes the following data in the initial marketing authorisation application relating to:

• total batch size and number of units required for batch release testing;
• available stability data and proposed shelf life;
• analytical sampling plan;
• a GMP certificate issued by an EEA Competent Authority relevant to the particular category of testing at the facility located in the third country.

Applicants intending to request an exemption are advised to proactively consult with the EMA early in product development. Technical difficulties in the transfer of analytical methods from third countries to the EU will not be accepted as a valid basis for request for an exemption. Similarly, the EMA provides that changes to the particulars of the marketing authorisation may annul the exemption granted if the ATMP no longer meets the criteria set out in the abovementioned EU good manufacturing practice guideline for ATMP.

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