Traceability of Autologous Drug Product from Cell Procurement through Infusion

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1 Traceability of Autologous Drug Product from Cell Procurement through Infusion Ian O Reilly February 22, 2017 Nasdaq : BLUE

2 Contents Highlighting and exploring the intricate autologous supply chain and ensuring traceability between patients and their cells Identifying key areas of improvement and how they were implemented over time Discussing the importance of right first time and how that overlays with supply chain Starting in a paper-based system and progressing to an electronic traceability system

3 General High Level Pathway There must be a two tiered system which exists for the purpose of original patient cells (starting material) and drug product through infusion to patient At the tissue establishment there must be a link between donor and donation At the manufacturing site there must be a link between donation and product At the infusion site (hospital, etc) there must be a link between product and recipient The system must allow for full traceability between donor from receipt via an unique identifier and coding system This means from beginning of the patient (donor) going through apheresis (donation), to shipping of donation to the drug product manufacturer there must be seamless traceability via chain of identify

4 What Does this Mean The drug product manufacturer must have a system to take the minimum of two unique identifiers coding system and turn that into drug product lot number(s). Some scenarios may be different however a linkage between the two is critical The drug product lot number(s) must then still be traceable when sent to the hospital and be linked back to the original donation (apheresis) and patient (donor) to receive their final product It s all about linking arm to arm via identifiers which ensure traceability

5 Cell Therapy Supply Chain Confidential - 4 Patient safety is at the center of our traceability supply chain process to ensure tracking of autologous cells and drug product

6 Apheresis Process What is Apheresis from a Cell and Gene Therapy (CGT) perspective? Starting material to manufacturing process Apheresis (mobilized), stem cell collection, or leukaphersis (non-mobilized) Autologous or Allogeneic Confidential - 5

7 Challenges with lack of traditional regulations Guidance for Industry cgmp for Phase 1 Investigational Drugs As it relates to phase 1 clinical trials, distribution includes the transport of a phase 1 investigational drug covered by this guidance to clinical investigators. You should handle phase 1 investigational drugs in accordance with labeled conditions (e.g., temperature) to ensure retention of the quality of the product. A distribution record of each batch of phase 1 investigational drug must be sufficiently detailed to allow traceability and facilitate recall of the phase 1 investigational drug if necessary ( (a)).

8 Some Clarity Exists REGULATION (EC) No 1394/2007 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 Article 15 Traceability shall establish and maintain a system ensuring that the individual product and its starting and raw materials, including all substances coming into contact with the cells or tissues it may contain, can be traced through the sourcing, manufacturing, packaging, storage, transport and delivery to the hospital, institution or private practice where the product is used

9 Continuing REGULATION (EC) No 1394/2007 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 Article 15 Traceability The hospital, institution or private practice where the advanced therapy medicinal product is used shall establish and maintain a system for patient and product traceability. That system shall contain sufficient detail to allow linking of each product to the patient who received it and vice versa

10 Key Areas for Improvement in General Better defined regulatory guidance would improve industry consistency in tackling the autologous world Harmonization between FDA, EMA, ICH and FACT/JACIE guidance to ensure all documents are consistent Larger consortium for industry (ATMP) leaders to discuss the current and upcoming guidance documents as they are drafted so as to better inform and work with regulatory bodies on draft guidance.

11 Key Areas for Improvement Internally As the manufacturing and collection (apheresis, etc) footprint expands, ensuring that the training is provided via sponsor requirements. Periodic confirmation is important to determine if original requirements are maintained at each site. Not each country is equal in terms of regulations. Some EU countries have different country-based regulations which are more strict than EMA regulations it is important to not only navigate the broad but also the local regulations Training is extremely important each company has a different system for traceability. There are many players at each apheresis and infusion site and learning your system is one of many for them.

12 Single European Code

13 Single European Code Intended to translate to a detailed description of the material Based on the tissue code used by the collection center Specified by EC coding rules

14 Right First Time Autologous therapy is just that personalized medicine One error in the traceability chain will have a potential domino effect resulting in potential loss of product or worse There is no room for a patient receiving another patient s cells Rejection of material, loss of individual drug product, loss of faith in the ATMP industry

15 Starting with Paper and moving Electronic With the potential pitfalls and damage that errors in traceability can inflict, logic dictates a move away from paper systems to electronic systems. There are some very good electronic solutions currently available These present an improvement away from human error, provide consistency across languages, sites, manufacturers and error proofs against mistakes Additionally these provide cost reduction over time and allow a footprint to expand with less effort

16 Regulations for Electronic Traceability Good Manufacturing Practice (GMP) FDA 21 CFR Part 11 FDA 21 CFR Part 1271 Eudralex Volume 4 Annex 11, Volume 4 Chapter 4 EU Tissue and Cells Directives Directive 2004/24/EC Directive 2006/17/EC Directive 2006/86/EC Directive 2002/98/EC Good Clinical Practice (GCP) PII (Personal Identifiable Information) ICH E6 Good Clinical Practice HIPAA Health Insurance Portability and Accountability Act Confidential - 15

17 Moving towards Electronic Systems Confidential - 16

18 Thank you Questions?