Proposal Submission

Applicant
(Person in charge) 		Name: Dr Phillip Nguyen
Position: Director, USP-APEC Center of Excellence and Director, Intl Regulatory Policy & Programs, Global Ext
Organization: United States Pharmacopeia USP
Country: United States
E-mail: ppn@usporg
Topic Pilot CoE Workshop on Ensuring the Quality of Raw Materials in Advanced Therapies in APEC Economies
Proposal Content
(Describe your proposal, including potential areas of collaboration, purpose, and expected impact.) 		[Summary]

Cell, tissue and gene therapies are increasingly being used to treat and potentially to cure human diseases that are not addressed using existing therapeutic products The number and complexity of these products is increasing dramatically while the timelines for development are shortening The manufacturing of these therapies requires the use of diverse and complex raw and starting materials that can be inherently variable and difficult to characterize completely Some materials are available only in research grade and others are from human or animal sources The APEC Life Science Innovation Forum promotes the use the international standards as an important aspect of achieving prospective regulatory convergence, and as such has co-developed workshops in collaboration with the Health Sciences Authority HSA Singapore that provided a platform for global experts to share experiences in regulatory approaches as they relate to advanced therapies A recent workshop under the Pilot CoE in Advanced Therapies for Northeastern University covered a broad overview of these therapies as well as the technical and regulatory challenges This application for a CoE pilot with a focus on one of the major challenges for developers; the identification, sourcing, and qualification of raw materials used in the manufacturing of advanced therapies This workshop will provide opportunities to develop training materials to support building strong qualification programs and enhance the quality of raw materials used in manufacturing Some of the raw materials used in advanced therapies eg cell culture media are also used in the production of biotherapeutics, creating opportunities to leverage work and lessons learned between two APEC RHSC Priority Work Areas

[Objective and Outcomes]

The purpose of this pilot is to bring together APEC representatives from regulatory agencies together with global academic and industry experts for a 2-day workshop, to share experiences on how to address qualification of raw materials used in the manufacturing of advanced therapies The participants will specifically learn and practice how to address the following:
1 Diversity of starting and raw materials used in manufacturing of advanced therapies – cells, plasmids, media, reagents, viral vectors
2 Identification and selection of critical raw materials used to produce these therapies
3 Risk- based approaches and categories of raw material used in manufacturing, and use of risk assessment tools such as Failure Modes and Effects Analysis
4 Lessons learned from biotherapeutics and other biological medicines
5 Type of quality systems to have in place to assure the quality of raw materials
6 Clarification of responsibilities between suppliers and end-users of raw materials

Participants to practice how to assess qualification of raw materials through case studies using materials associated with trends in cell and gene therapy products
Expected date 2020-07-01 to 2020-07-10 (Total 2 Days)
Expected Venue (Economy) Republic of Korea or Singapore
Expected No. of Guests 60 Open
Expected Total Cost of the Event USD 105,000

Amount requested from AHC: USD 75,000
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