Full Day Comparability Workshop
9:00 – 17:00, Monday 22 January
Change is inevitable and necessary both in development and over the post-approval product lifecycle. Whenever changes are made it is necessary to confirm they do not adversely impact the quality and therefore safety and efficacy of the product; this requires data beyond meeting current specifications. With any biological product this is challenging, for cell, gene and tissue products that cannot be fully characterised the challenges are greater still. Concerns about comparability undertaken during development are common issues at approval with significant comparability concerns raised during 12 of the 17 ATMP submissions to the EMA. Such questions often delay market approval or contribute to failure to get approval. This course explains what comparability is and how to develop a successful comparability protocol.
Workshop Learning Overview:
- What is comparability?
- Why is meeting existing specifications not comparability?
- How do I apply the principles of comparability to highly variable products?
- Case studies: Common mistakes with comparability and their consequences.
- Interactive exercise: Spot the weaknesses and propose improvements to a worked comparability study
Half Day Quality-by-Design
Quality by Design (QbD) is a science- and risk-based approach to pharmaceutical development. Applied to manufacturing processes, it
is intended to ensure a rational and cost-effective approach to product quality. Both the EMA and FDA have systematically assessedQbD elements of regulatory filings and, especially since the FDA’s 2011 Process Validation Guidance and the ICH’s Q8 through Q11, it has become an intrinsic part of regulatory guidance.
However, understanding and adoption of the principles of QbD has
remained relatively limited in the field of cell and gene therapy. We
strongly believe that its methodologies are critical to the successful
development of ATMPs and that they should be incorporated as
early as possible into the science of manufacturing. This workshop
will both demystify the fundamentals of QbD and provide concrete
examples of its application to this field. You will leave with a crystalclear
understanding of P Diagrams, RAMM matrices, the QTPP and
many more tools which you can immediately use to improve your
products and processes.
Half Day Intellectual Property 101
This session is geared towards providing an overview of intellectual
property, including trademarks, design patents and utility patents,
geared especially for those new to the process. The course will include a general overview of different types of IP that a company may employ to develop a robust portfolio to build and protect their brand, as well as their contemplated commercial products. The session will emphasize utility patents (their specifications and claims), different types of applications (provisionals, nonprovisionals, continuations, divisionals, and continuations-in-part), and what patents do and do not allow patentees to do (enforcement versus the ability to practice patents). The session will cover the nuts and bolts of the patenting process from conception of an invention, to filing/claiming strategies, to prosecution, patenting, and considerations for continued filings, with an aim to touch on domestic and international strategies at a high level. Time permitting, an interactive session will be held where filing strategies
are developed for hypothetical inventive entities.
- High level overview of different types of IP
- Distilled overview of utility patents and the patenting process
- Overview of patent application types including considerations on
- when to file
- Interactive session to reinforce the course
Half Day Authoring the Common Technical Document
11:15 – 15:30, Thursday 25 January
The ICH Common Technical Document was developed to standardize the way information is shared with regulators, and is accepted or mandatory by agencies following ICH (e.g. EU, US, Japan, Canada etc). The CTD is used for all types of medicinal product but the purpose of each section may not always be clear for ATMP.
This workshop aims to clarify:
- What the purpose of each section is
- Whether the section is descriptive or data-driven
- What sorts of data are expected
- Tips and considerations for style, use of figures and tables, etc.