CMC Strategy Forum Prague Enda Moran EBE Satellite Session, 06 May 2013

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1 CMC Strategy Forum Prague 2013 Enda Moran EBE Satellite Session, 06 May 2013

2 European Biopharmaceutical Enterprises EBE s VISION: fostering innovation, and promote favorable business and regulatory conditions for biopharmaceutical enterprises of all sizes in Europe. EBE s MISSION: advocating the benefits of a healthy biopharmaceutical industry vis-à-vis all key stakeholders and to nurture a healthy membership base by: Increasing awareness of the tangible benefits of biopharmaceutical innovation to patients and society Promoting connectivity between big and small pharma and biopharma Helping the development of policies at European level that support biopharmaceutical innovation, regulatory and entrepreneurial conditions Delivering resources and access to technical & regulatory expertise, research and funding partnership opportunities, education & training on all aspects of the biopharmaceutical sector Through collaboration within EFPIA and its working groups, strengthen representation on specific issues relating to biotech and small & medium size enterprises in biopharmaceutical sector. 2

3 European Biopharmaceutical Enterprises European Biopharmaceutical Trade Association Non-profit organisation, governed by a board of directors and annual general assembly. Managed by an executive team based in Brussels. Established in 2000 as a specialised group of EFPIA, European Federation of Pharmaceutical Industries and Associations. 53 members to date. 9 working groups on variety of key topics related to biopharmaceutical industry. 3

4 European Biopharmaceutical Enterprises Members ( April 2013 ) 4

5 EBE Structure EBE Board Strategy Setting EBE TEAM Execution and Management of Working Groups Biosimilars Rare Diseases and OMP Advanced Therapies BioManufacturing Pharmacovigilance Personalised Medicines Models for innovation and funding Public Affairs & Regulatory Technical expertise Value Add to EBE Members Connectivity Pharma - Biotech European Policy Development Biotech Sector Awareness Resources: Information &Training 5

6 European Biopharmaceutical Enterprises ( Our website is currently under re-construction. We apologise for any inconvenience and look forward to welcoming to our new EBE website as from July ) 6

7 Reflection 2012 Concept papers discussed at CMC Forum Berlin 2012 Satellite session at the CMC Strategy Forum, Berlin. Concept Papers - Setting specifications - Platform manufacturing CMC Strategy Forum Europe 2012 Monday, April 23, Wednesday, April 25,

8 Concept papers on Setting Specifications & Platform Manufacturing now available Will be accessible via July2013: site being updated.

9 Industry representatives & BWP Discussed Process Validation at EMA, London on 09 Apr 2013 Industry / BWP workshop on Process Validation A valuable check-in before furthering the forthcoming PV guideline for biotechnology-derived drug substances More later... 9

10 New Concept Paper. I Forced Degradation Studies Describe approaches and best practices amongst biopharmaceutical companies in the design and execution of Forced Degradation Studies (FDS). Rationale & drivers: Very few regulatory guidances on FDS Lack of clarity on what should be done at each development phase, both from a regulatory and a scientific perspective. New topic group lead by Annick Gervais, UCB and involving 6 other EBE Member Companies Janssen Biologics MedImmune Merck-Serono Novo Nordisk Sanofi Swedish Orphan BioVitrum

11 New Concept Paper. I Forced Degradation Studies Expected Content Why a FDS? When to initiate a FDS? Which clinical Phase? Which Stressing Agents? What are the relevant stressing conditions? In which case? How to design a FDS? What are the limitations? What is the extent of degradation to achieve? How to define the acceptance criteria for comparability? Temperature Excursions Storage / freeze-thaw Method Development Stability Indicating Methods Stability Predictor Possible Degradation Pathways Understand CQA Forced Degradation Studies Comparability Product Related Species Characterisation Candidate Selection Manufacturing Process Development Formulation Development

12 New Concept Paper. I Forced Degradation Studies Describe approaches and best practices amongst biopharmaceutical companies in the design and execution of Forced Degradation Studies (FDS) Plans: Kick-off meeting 3 rd May 2013 Shape and structure of paper laid out Table of Contents, subject matter to be discussed, types of study examples to be described, data to be included. Q4 2013

13 New Concept Paper. II Visible Particles Problem statement - By reference to Ph. Eur. Monograph # 2031 Monoclonal antibodies for human use, EU Health Authorities can request to define as acceptance criteria without visible particles for the Drug Product appearance during assessment of late stage Clinical Trial Application or Marketing application Practically free of visible particles is challenged as a QC acceptance criterion - Even with best formulation optimization effort supported by excellent long term stability studies, stress stability studies (e.g. agitation), without (zero) Visible Particles is an unrealistic requirement for QC release/shelf life testing the baseline should be practically free of visible particles - Setting acceptance criteria corresponding to practically free of visible particulates or setting acceptance criteria when visible proteinaceous particles are unavoidable is a topic that require some discussion/ harmonization in the industry 13

14 New Concept Paper. II Visible Particles Topic Group Lead: Serge Mathonet, Sanofi, Global Reg Aff R&D - Biotherapeutics CMC + Experts from Sanofi, Roche, Novo Nordisk, MedImmune, Abbvie, J&J Deliverable: Concept paper: available for consideration by industry, BWP, EDQM etc. - Scope: Monoclonals - Filled vials, syringes and pens (clinical and commercial supplies) - Content Problem statement Best practices in the industry in term of visual inspection process and associated operator training, QC sampling, testing and setting acceptance criteria corresponding to practically free of visible particles or settting acceptance criteria when visible proteinaceous particles are unavoidable. Consideration for particles ID, characterization and qualification - Timelines Development of position paper in 2013 Kick off 20 Feb nd draft being discussed 14

15 New Concept Paper. II Visible Particles: On-going discussion items Categorisation of extrinsic/intrinsic particles defects into minor, major or critical 100 % visual inspection: Number of units to be re-inspected if one defect is found Operator certification program (manual/semi-automated 100 %inspection and AQL verification): Non destructive versus destructive particle testing and influence on sample size - vials, pre-filled syringe and pre-filled pens Acceptance criteria: AQL and QC testing (release/stability) Role of Quality investigation into the cause of an initial failure for decision making (re-inspection or lot release) Pre-Clinical/Clinical qualification of intrinsic particles 15

16 EBE Satellite Session Agenda 09:00 09:15 Welcome and Introduction to the European Biopharmaceutical Enterprises (EBE) Ongoing Activities and Initiatives. Enda Moran, Pfizer Ltd., Grange Castle, Ireland 09:15 09:30 EBE Interest Topic Bioburden Control at the Sterile Filtration Step: A risk-based approach Ray Field, MedImmune, UK