Agenda

1. Senior Leaders stand to refine their leadership ethos and embrace novel strategies to nurture a thriving workplace ambiance.
2. Mid-level executives can unlock invaluable insights, enhance team synergy and communication, and hone their unique leadership persona, priming themselves for the next career milestone.
3. Emerging Leaders on the brink of their journey can craft the foundational pillars of their leadership identity, mastering the art of adaptation and interpersonal communication amidst the diverse tapestry of the workforce.

·         Gene therapy offers tremendous potential and is continually evolving.

o   Today the industry is tackling incurable diseases, providing durable cures and modifying diseases for relatively small patient populations.

o   In the not-too-distant future gene therapy will provide permanent cures for much larger patient populations, enhancing existing treatments with precision medicine and preventing disease altogether.

·         Almost by definition, the evolving nature of gene therapy drives the need for continual changes in technology, talent, platforms and development – all with the requirement for inherent flexibility.

·         Spark Therapeutics developed and launched the very first gene therapy for a genetic disease approved in both the US and EU.

·         As a leader and driving force in gene therapy, Spark is continually evolving ahead of the industry and working to achieve its vision of a world where no life is limited by disease.

·         Join Chris Stevens, Spark&rs

This presentation will focus on transformative initiatives within Office of Quality Surveillance (OQS) in FDA's Center for Drug Evaluation and Research (CDER) aimed at enhancing and modernizing the agency's approach to drug quality oversight. OQS plays a pivotal role in safeguarding dug quality by leveraging various data sources and analytical methods to monitor, assess, and report on the state of drug product quality and manufacturing facilities. The OQS speaker will discuss key advancements and implementation of innovative programs that enhance FDA’s oversight of drug quality. These programs include the New Inspection Protocol Project which streamlines inspection processes; the Drug Quality Sampling and Testing Program which ensures that the drugs sold in the US meet rigorous standards; and the Quality Management Maturity Program aimed at elevating industry quality oversight. Attendees will gain insights on how these programs enhance FDA’s quality oversight and encourage continual improvement from manufacturers.

The guidelines ICH Q12 (2019) and ICH Q2(R2)/Q14 (2023) embody a science- and risk-based approach to lifecycle management to ensure the continued quality of the quality of drug substances and drug products.  ICH Q12 describes the tools that can be utilized for effective post approval change management and regulatory reporting, while ICH Q2(R2)/Q14 focus on development, validation, and lifecycle of analytical procedures; and covers concepts such as the Analytical Target Profile (ATP) and the applicability of enhanced knowledge to manage Q12 concepts such as Established Conditions.  There are many expected benefits which include harmonizing scientific approaches and terminology, enabling effective knowledge and risk management to facilitate continual improvement, and improving communication between health authorities and industry.  This session will talk about real-life examples from industry and the challenges encountered when implementing these guidelines both from the sponsor and regulator perspectives.  Topics presented from industry (the ISPE PQLI ICH Q2(R2)/Q14 and Q12 teams) will include platform analytical procedures, enhanced approaches to development practices, change management utilizing the principles of ICH Q12 and Q2(R2)/Q14 and how ECs can work, and the status of guideline adoption across various companies and health authorities.  These presentations from four major companies will be followed by an extended panel discussion including regulators to provide a format for a candid exchange of experiences and ideas to address the perceived challenges. 

A Regulatory Town Hall Meeting focused on the challenges and opportunities to expand innovation, improve product quality assurance and patient access to medicines relies on global regulatory harmonization and collaborative approaches in establishing effective policies.  This paradigm shift is predicated on transformation that only a future-looking workforce can deliver.

The pharmaceutical industry is advancing through an unprecedented innovative transformation, driven by the development of new product modalities, the emergence of innovative technologies, the evolution of alternative and adaptive regulatory frameworks, and the expeditious approaches introduced and leveraged in response to the COVID-19 pandemic experience. 

The Regulatory Town Hall will cultivate a compelling discussion among several panelists on the merits, opportunities and challenges associated with the introduction of innovative technologies, the use of modelling, artificial intelligence, and digital platforms that are shaping the future of pharmaceutical development and commercialization.  The key messages from this event will provide a template for the attributes and qualities necessary to enlist and engage a skilled and competent workforce capable of adapting to  the fluid demands and expectations of patients and health care providers globally.

The panel will include six representatives from industry and regulatory authorities who will address the following evolving topics:

1. Mutual reliance: How can the industry and regulators leverage the existing frameworks and initiatives, such as the Pharmaceutical Inspection Co-operation Scheme (PIC/S), the Mutual Recognition Agreement (MRA), and the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), to enhance the mutual recognition and reliance of inspections, assessments, and standards across different jurisdictions?
2. Information sharing: How can the industry and regulators enhance the information sharing and communication between and among themselves, as well as with other stakeholders, such as patients, health care professionals, and the public, using the available and emerging digital platforms and tools?
3. Modelling: How can the industry and regulators increase the global acceptability and use of modelling and simulation techniques, such as mechanistic models, physiologically based pharmacokinetic (PBPK) models to support the design, development, and evaluation of medicines and vaccines?
4. AI algorithms: How can the industry and regulators ensure the quality, reliability, and validity of the artificial intelligence (AI) algorithms and systems that are increasingly used in the pharmaceutical sector, such as for drug discovery, clinical trials, pharmacovigilance, and manufacturing?
5. Inspection harmonization: How can the industry and regulators harmonize the implementation and interpretation of the PIC/S Guide to Good Manufacturing Practice for Medicinal Products (GMP), especially the Annex 1 on the manufacture of sterile medicinal products?
6. Supply chain management: How can the industry and regulators improve the resilience, transparency, and security of the global pharmaceutical supply chain, especially in the post-pandemic context, where the demand and supply of medicines and vaccines are subject to unprecedented fluctuations and disruptions?
7. Structuring the data: How to address the terminology when submitting data to different regulatory agencies? How to harmonize the data? What kind of standard should be used?
 
We invite all the participants to share their questions on these topics and engage in a constructive and productive dialogue on charting the direction for innovation in the industry to embrace the future that is now!