Concerns Over Format Of EU GMP Guideline For ATMPs Finally Resolved; Publication Due

EU biopharmaceutical industry group EBE says it is awaiting keenly the publication of good manufacturing practice guide for advanced therapy medicinal products, after learning that the European Commission is making changes to address concerns it had over the appropriate format of the document.

The European Commission's much-awaited guideline on good manufacturing practices (GMP) for advanced therapy medicinal products (ATMPs) is likely to be published in the first half of 2017 as a stand-alone document. The document has been in the making for over a year and has undergone two rounds of stakeholder consultation.

Initial concerns that the industry had regarding the format of the final document – i.e., whether it should be a stand-alone guide or an annex to the existing EU GMP guideline for human and veterinary drugs (EudraLex Volume 4) – are no longer an issue, according to the European Biopharmaceutical Enterprises (EBE), which represents biopharmaceutical companies of all sizes.

Following a dialogue with stakeholders in November 2016, the commission put forward a path to resolve the concerns, said EBE executive director Barbara Freischem. "What is most important for us is that there is going to be a GMP guideline for ATMPs," Freischem said.

The format of regulatory guidelines is not usually a contentious issue, but in this case it drew the attention of most stakeholders, including industry, during the two rounds of consultations that took place for the document. Specifically, stakeholder concerns related to the legal uncertainties that were feared might arise if a clear relationship were not established between the proposed GMP guideline for ATMPs and EudraLex Volume 4. (Also see "Not There Yet: Concerns Persist On Appropriate Format Of EU GMP Guide For ATMPs" - Pink Sheet, 14 Dec, 2016.)

The first draft that was issued for consultation in July 2015, was "sitting in the middle," Freischem told the Pink Sheet. If the commission were to go down the annex route, then it would have had to add more references about the general requirements and explain how they applied to ATMPs. If it went for a stand-alone document, then it needed to repeat a lot of basic information from EudraLex Volume 4 to make the guideline self-sufficient, Freischem explained. She said the EBE would have been happy to accept either a stand-alone document or an annex to EudraLex Volume 4.

The commission then issued for consultation, in June 2016, a second draft of the guideline that included more details from Eudralex Volume 4 to establish it as a stand-alone document. (Also see "Second Time Around: EU GMP Guideline For ATMPs Out For Consultation Again" - Pink Sheet, 10 Jul, 2016.) After reviewing the second draft, the EBE, along with EU pharmaceutical industry federation EFPIA, said it believed that ATMP developers "would be best served by a specific ATMP Annex to EudraLex Volume 4".

Freischem said the EBE and EFPIA were concerned that the second draft guideline "did not go far enough" in repeating the basic requirements. "We also thought that it might be easier to administer the guideline as an annex [to EudraLex Volume 4]".

The EBE has since reconciled its position following the November stakeholder meeting with the commission. The commission explained what further changes it planned to make to the document so that the final version would be detailed enough to be a self-sufficient, stand-alone guideline.

Freischem explained that the industry's concerns regarding the second draft were "pretty much driven by the format of the document that we commented on" and a way has been proposed to address these concerns. She added that it was also necessary "to consider the background and the context of where our comments come from", i.e. from an industry that already has GMP requirements implemented for non-ATMP pharmaceuticals and vaccines.

The EBE executive director is of the view that that a stand-alone guideline would be easier to handle for people who have less experience in implementing GMP requirements, such as those dealing with ATMPs covered by the hospital exemption scheme. Freischem expects the commission to finalize the GMP guideline for ATMPs by the end of January, and to publish the document, at the very latest, by the first quarter of this year.

From the editors of Scrip Regulatory Affairs.