Advanced Therapy Medicinal Products

United Kingdom 14 Oct 2021 - 14 Oct 2021

Organizer: Xtalks

Event Details

  • 14 Oct 2021 - 14 Oct 2021
  • -
  • BST (UK) / 4pm CEST (EU-Central),BST (UK) / 4pm CEST (EU-Central)

Organized By

  • Xtalks
  • [email protected]
  • Xtalks, powered by Honeycomb Worldwide Inc. 1235 Bay Street Suite 802 Toronto, ON M5R 3K4 Canada

About Advanced Therapy Medicinal Products

  1. Donor variability: Among the many aspects that may impact on the quality of the final gene therapy are thevariability of donor starting material and how the disease status affects the quality of the starting material. Due to variability, a wide but acceptable product specification is required.
  2. Batch variability: Naturally, the donor variability leads to thepotential variability of the final drug product. In addition to the batch variability, the limited stability of an ATMP requires great attention to the storage, transport and handling conditions of the finished product which can prove to be very challenging.
  3. Investment: Transitioning from lab-based production to large scale production can require significant investment. Capital investment for large scale equipment and suitable facilities will be required. ATMPs, like any other medicinal product, must demonstrate quality, safety and efficacy in order to obtain its Marketing Authorisation and therefore significant investment goes into meeting these quality, safety, and efficacy standards set by the regulatory authorities.
  4. Small patient population: A big challenge for cell and gene therapies is that they often target conditions that affect a very small number of patients.And the reality is, it’s not always commercially viable for the company to go into large scale manufacture.
  5. Regulatory complexity: The development of ATMPs requires significant effort from the regulatory agencies to make available all guidance to support the development of such treatments and support them get to market.

The European Medicines Agency (EMA) has published several new guidelines discussing different aspects of the development of ATMPs. In addition, the EMA has also issued the final version of its new Regulatory Strategy to 2025 along with Analysis and summaries of public consultation results

  1. QP certification challenges: It is imperative that the QP is experienced in ATMP manufacture and release. Typically, ATMPs have short shelf lives and therefore a particular strategy is required to justify release without full test results being available. Your QP may need to be comfortable with releasing the product quickly and without all release testing being completed, in order to meet a short treatment window. Therefore, a thorough understanding of biological processes and the variability this brings is required. This understanding coupled with a flexible and pragmatic approach are invaluable. To help support the QP in the complex issues of QP certification of ATMPs, the EMA published a Q&A on Exemptions on batch controls carried out on ATMPs providing guidance for the QP on when exemptions from batch controls are acceptable for ATMPs imported into the European Union from a third country.
Advanced Therapy Medicinal Products

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