DIA 2025 Global Annual Meeting

Component Type: Tutorial
CE: ACPE 2.75 Knowledge UAN: 0286-0000-25-501-L04-P; CME 2.75; IACET 2.75; RN 2.75

Pre-registration required and is an additional fee.

Already registered? Log in to DIA's My Account My Events.

This Short Course will be offered virtually – Join from anywhere! This short course will provide an overview of the current use of real-world data (RWD) and real-world evidence (RWE) for evidence generation in major markets, with a primary focus on regulatory use and how it can inform clinical development planning. Following the major points from recently released guidance documents from around the world, the course will explain how the fitness of evidence from RWD are assessed for any use case, key elements of design and analysis, and the current status for when real-world studies will be sufficient to meet regulatory evidence requirements. Specifically, the course will discuss (a) the evolving regulatory landscape; (b) the evolution of RWD, how to utilize it, and when RWD is fit-for-purpose; (c) the evolution from safety evaluation to label extensions; and (d) how to ensure design transparency and data feasibility when proposing external comparator arms. The course will feature interactive elements such as polling questions, and participants are encouraged to present their own scenarios for open discussion.

Receive $50 off  your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart.

Registered attendees for this virtual Short Course will receive access to the course recording for 2 full months post-course! This allows you to remain flexible with your schedule and not worry if you need to step out momentarily. Have a conflict with the dates of the course, but are interested in the content? Register anyway and you will receive access to the recording!

Who should attend?

Professionals involved in or interested in learning about real-world evidence, real-world data, epidemiology, clinical research, regulatory and technology development.

Learning Objectives

• Discuss how regulators are using RWE for regulatory decision support and how RWD and RWE are being used to inform clinical development planning
• Describe the concept of RWD being “fit-for-purpose” including the importance of relevant data and methodologic rigor
• Identify how RWE can be assembled and integrated to generate a compelling message

Component Type: Tutorial
CE: ACPE 2.75 Application UAN: 0286-0000-25-504-L04-P; CME 2.75; IACET 2.75; RN 2.75

Pre-registration required and is an additional fee. *Please note: Short Courses are stand-alone events. Registration for the main conference, forum, etc. are not mandatory. Already registered? Log in to DIA's My Account My Events. This Short Course will be offered virtually – Join from anywhere! Cell and Gene Therapies (CGTs) are a rapidly expanding area in the pharmaceutical sector, offering the potential to address serious unmet medical needs in many indications. Yet some of the conceptual frameworks in this field differ dramatically from those of traditional pharmaceutical medicines, specifically with regard to CMC, Non-clinical and Clinical requirements and Health Authority expectations. This short course will help those who have pharmaceutical sector experience but are newcomers to the CGT field ‘get into the right frame of mind’ to think about CGT development. Participants will be introduced to requirements for each of the major disciplines for regulatory submissions: CMC, Non-clinical and Clinical. The course will also explain the rationale behind the CMC approach to CGT manufacture and basic CMC regulatory expectations for CGT products, including process and product characterization, comparability and process validation. Biological mechanisms for CGTs will also be discussed, with a focus on how mechanistic understanding can inform the design of in vitro and animal studies to provide non-clinical evidence of safety and efficacy. Key principles for clinical development of CGTs will be presented, with an emphasis on clinical study design elements that are critical for Health Authority evaluation of benefit-risk. Case studies of recent FDA approvals will be showcased to highlight key clinical considerations and changes in the regulatory environment for CGTs. The course features ex-Health Authority Regulators presenters who have a combined 20 years of experience at the FDA and EMA. Receive $50 off your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart. Registered attendees for this virtual Short Course will receive access to the course recording for 2 full months post-course! This allows you to remain flexible with your schedule and not worry if you need to step out momentarily. Have a conflict with the dates of the course, but are interested in the content? Register anyway and you will receive access to the recording!

Who should attend?

Professionals involved in or interested in gaining an understanding of the basics of cell and gene therapy development, including regulatory personnel, clinical operations personnel, researchers and investigators, medical writers, and commercial specialists.

Learning Objectives

• Demonstrate why cell and gene therapies (CGT) require a different and very specialized regulatory approach compared to small molecules and biologics
• Develop knowledge of CGT-specific considerations and requirements for CMC, Non-Clinical, and Clinical development
• Analyze important regulatory considerations for CGT development programs

Component Type: Tutorial
CE: ACPE 2.75 Application UAN: 0286-0000-25-507-L04-P; CME 2.75; IACET 2.75; RN 2.75

Pre-registration required and is an additional fee. *Please note: Short Courses are stand-alone events. Registration for the main conference, forum, etc. are not mandatory. Already registered? Log in to DIA's My Account My Events. This Short Course will be offered virtually – Join from anywhere! Combination products are emerging as innovative medical products due to their contribution to advancing medical care and are thus expected to have major impact in the coming years. Future technologies are most appealing to patients with ongoing medical conditions that require consistent treatment with daily injections or weekly procedures and unmet medical needs. Overall, the successful development of combination products will require great collaboration within the industry to overcome regulatory, clinical, and technical challenges. Implementation of a strategy includes the compilation of the plans and/or documents pertaining to strategic partnerships, intellectual property (IP) initiatives, product development approaches, quality management systems (QMS) optimizations, and regulatory submission plans into the integrated regulatory strategy for the complete “asset” as the traditional “ways of working” and/or processes will be challenged when developing an innovative combination product. This short course will explore key challenges and opportunities, offering practical exercises and a structured framework for success. It will focus on mitigating regulatory risk(s) in submissions timeliness and addressing issues such as excessive queries from health authorities, additional testing needs, and delays that impact time to market. Receive $50 off your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart. Registered attendees for this virtual Short Course will receive access to the course recording for 2 full months post-course! This allows you to remain flexible with your schedule and not worry if you need to step out momentarily. Have a conflict with the dates of the course, but are interested in the content? Register anyway and you will receive access to the recording!

Who should attend?

Entry/Intermediate professionals in quality, product development, and risk management who are suppliers/CDMOs or patent attorneys.

Learning Objectives

• Analyze the latest regulatory expectations and challenges for complex devices and combination products
• Explain potential future innovation platforms
• Identify opportunities to integrate regulatory strategies across functional areas including risk management activities and content between Sponsors, Suppliers and CDMOs

Component Type: Session
Level: Advanced

Pragmatism in clinical trials advances modernization of clinical research. Key stakeholders including experts from the FDA will review advantages and challenges of pragmatism in clinical trials including trials in support of new labeling claims.

Learning Objectives

Assess the strengths and limitations of pragmatic trials and the reasons for uneven adoption among industry and regulators; Discuss the regulatory framework and FDA initiatives supporting the use of pragmatic elements in clinical trials; Determine the appropriate context-of-use for successfully deploying pragmatic trials in clinical research.

Chair

Zhanna Jumadilova, DrMed, MBA

Speaker

Guidances at a Glance: Modernizing Oncology Clinical Trials through Decentralized and Pragmatic Approaches
Timil Patel, MD

Component Type: Forum
Level: Intermediate

In 2019, FDA’s OCE initiated Project Orbis to provide a framework for concurrent submission and review of oncology drug products. Project Orbis Partners will provide an update on their recent collaboration and answer questions from the audience.

Learning Objectives

Provide an international regulatory update on Project Orbis; Discuss recent collaboration efforts beyond Project Orbis that were prompted by the initiative; Explain how sponsors can benefit from participating in Project Orbis.

Component Type: Forum
Level: Intermediate

Among the largest areas of concern with the Inflation Reduction Act (IRA) are potential unintended consequences to pharmaceutical innovation. This forum will convene industry experts to explore the impact of and potential adjustments to the IRA.

Learning Objectives

Discuss the unintended consequences of the IRA on innovation; Examine potential legislative solutions.

Chair

SARAH K Martin, PhD, MS

Component Type: Forum
Level: Intermediate

The forum will explore key legislative proposals affecting FDA regulation of drugs and devices, while FDA and industry prepare to negotiate the next round of user fees and performance goals.

Learning Objectives

Describe key legislative initiatives that could alter FDA's regulation of drugs and medical devices; Understand the interplay between current FDA legislative proposals and the upcoming FDA user fee negotiations and legislation; Consider the impact of legislative proposals on FDA and regulated industries.

Chair

Jeffrey Francer, JD, MPA

Component Type: Session
Level: Intermediate

In this session, leaders from Health Canada will provide an update on regulatory priorities for Canada. The audience will be invited to submit questions of general interest.

Learning Objectives

Describe Health Canada’s regulatory initiatives and strategic priorities; Identify opportunities for engagement with the Canadian regulator.

Component Type: Forum
Level: Intermediate

Regulatory and Industry experts will reflect on progress on global regulatory harmonization of requirements for the development of medicines since the founding of ICH and it’s expanded global footprint following the reforms in 2015.

Learning Objectives

Assess progress in global harmonization of regulatory requirements for the development of medicines since ICH reforms in 2015; Evaluate impact of specific ICH guidelines in global drug development such as E6 Good Clinical Practice, E17 Multi-Regional Clinical Trials and Q9 Quality Risk Management; Identify opportunities for greater regulatory harmonization to advance global drug development.

Chair

Amanda Marie Roache, MS

Component Type: Session
Level: Intermediate

This session will focus on assessing the effectiveness of current regulatory collaboration efforts and identifying how those principles can be applied to chronic disease areas outside of oncology, such as metabolic disease and obesity.

Learning Objectives

Describe the impact of global regulatory collaboration on patient outcomes; Understand risks associated with chronic disease and its impact on society as a whole; Identify innovative ways to address chronic disease through regulatory innovation and collaboration.

Chair

Brad Jordan, PhD

Component Type: Session
Level: Intermediate

FDA's Accelerated Approval Program has been criticized for delays in withdrawing approved therapies when clinical benefit is not confirmed. This is a small percentage of drugs, however, and the reasons need to be understood in overall context.

Learning Objectives

Examine the current fragmentation in RWD/RWE data quality standards and its impact on research and regulatory decisions; Identify the key stakeholders and their varying data quality criteria within the pharmaceutical landscape; Explore potential strategies for developing a unified data quality framework to enhance consistency and reliability in data-driven research.

Chair

Bridget Doherty, MPH, MS

Component Type: Forum
Level: Basic

Explore the transformative potential of generative AI (genAI) in Regulatory Affairs. This session will share insights and experiences from industry, focusing on education, culture change, and impactful use cases.

Learning Objectives

Understand the value and potential of generative Artificial Intelligence (genAI) in Regulatory Affairs (RA); Outline how use cases are selected and implemented by Industry in their RA departments; Identify examples of application of genAI in RA; Adapt to and practice genAI within your own RA organization.

Chair

Paul Bolot, PharmD, MSc

Component Type: Session
Level: Intermediate

Explore WHO-led initiatives and global collaborations driving regulatory harmonization and innovation to ensure equitable access to medical products. This session highlights strategic efforts to address shared challenges and advance global health.

Learning Objectives

Analyze the progress and challenges in achieving regulatory convergence and harmonization; Understand the role of regulatory reliance and collaboration in facilitating access to medical products; Discuss international partner’s perspective on navigating regulatory landscapes to enhance global health equity.

Chair

Hiiti Sillo, MSc, RPh

Component Type: Session
Level: Intermediate

This session will present on recent work to harmonize RWE guidelines globally. Ongoing efforts in international collaboration on ICH M14 and the ICH reflection paper will be highlighted.

Learning Objectives

Explain recent developments in international guidelines on RWE; Discuss challenges to harmonization efforts in RWE; Evaluate gaps in RWE harmonization needs

Chair

Stephanie Choi, PhD

Component Type: Session
Level: Intermediate

This session will focus on the attractiveness of developing and obtaining marketing approval of pharmaceutical products in Japan from regulatory and industry perspectives, aiming to get rid of old-fashioned ideas and views about Japan.

Learning Objectives

Debunk outdated misconceptions and “urban legends” about Japan when considering Research and Development (R&D) on new medicines in Japan; Examine the attractiveness of performing R&D in Japan, both from regulatory and industrial perspectives: attractive market, flexibility/predictability, designation for each product characters; Analyze the Roles and Prospects of the PMDA’s Overseas Offices.

Component Type: Session
Level: Intermediate

In this forum, leaders from ANVISA will provide an update on regulatory priorities for Brazil, and inform about local trends, regulatory convergence, and collaboration initiatives. The discussion will also invite questions of general interest from the audience.

Learning Objectives

Describe ANVISA’s regulatory initiatives and strategic priorities; Discuss regulatory convergence of Brazilian regulations for medicines and medical devices to international guidelines and standards; Identify trends and opportunities for engagement with the Brazilian regulator

Chair

Bianca Zimon, LLM

Component Type: Session
Level: Intermediate

This session will explore engagement pathways with global regulators to obtain feedback on AI technologies in drug development. It will also identify effective evaluation strategies to support regulatory acceptance of novel methodologies.

Learning Objectives

Identify the appropriate regulatory pathways and mechanisms for engaging with health authorities to discuss AI technologies in drug development applications; Discuss regulatory strategies, use cases, and considerations for AI model development based on context of use; Evaluate lessons learned and experiences from manufacturers, developers and health authorities.

Chair

Ryan Hoshi, PhD, MBA, MSc

Component Type: Session
Level: Intermediate

The US Orphan Drug Act and the European regulatory framework have accelerated orphan medicine development, boosting investments in rare disease treatments. This session will gather key stakeholders to discuss successes a and challenges

Learning Objectives

Explore the impact of regulatory policies on the rare disease ecosystem; Discuss the challenges and opportunities associated with orphan drug regulations; Examine the role of regulatory frameworks in fostering innovation and access to therapies for rare diseases; Highlight the importance of collaboration between regulatory agencies, industry stakeholders, and patient advocacy groups.

Chair

Pedro Franco, SR, PharmD, PhD, MS, MSc

Component Type: Session
Level: Intermediate

In this forum, speakers from the National Medical Products Administration (NMPA) and the Center for Drug Evaluation (CDE) will present and discuss the latest information on regulation, regulatory science, and drug review and approval. Speakers will also discuss recent development in clinical research in China. Attendees will have an opportunity to discuss the issues related to drug regulation with NMPA officials.

Learning Objectives

Summarize the latest information on the National Medical Products Administration’s activities in drug regulation; Describe the recent update on the progress in drug review, approval and clinical research in China; Identify challenges in drug development and regulation in China.

Chair

Ling Su, PhD

Component Type: Session
Level: Intermediate

This session will explore the ways in which the data quality criteria within the EMA, FDA and ICH M14 guidelines converge or diverge, and propose some definitive actions for consideration to achieve harmonization of terminology.

Learning Objectives

Examine the current fragmentation in RWD/RWE data quality standards and its impact on research and regulatory decisions; Identify the key stakeholders and their varying data quality criteria within the pharmaceutical landscape; Explore potential strategies for developing a unified data quality framework to enhance consistency and reliability in data-driven research.

Chair

Elena Popa, MPharm

Component Type: Forum
Level: Intermediate

Representatives from pediatric cluster health authorities will describe the common deficiencies they see in global pediatric development programs and describe best practices and what to expect for those companies that seek a common commentary.

Learning Objectives

Describe common deficiencies in global pediatric development plans; Explain the process for obtaining a common commentary; Recognize topics that are discussed at monthly pediatric cluster calls.

Chair

Melodi J McNeil, MS, RPh

Component Type: Session
Level: Intermediate

Global key initiatives towards harmonization, convergence and collaborative efforts in advancing the development and accessibility of advanced therapy medicinal products (ATMPs) will be explored, with a special focus on supporting MICs.

Learning Objectives

Outline global efforts on collaboration and harmonization initiatives for ATMPs; Identify and discuss efforts to build regulatory capacity, provide technical assistance, and facilitate access to ATMPs in middle-income countries (MICs); Discuss strategies for achieving convergence and harmonization for ATMPs.

Chair

Maria Antonieta Tony Roman, MPharm

Component Type: Session
Level: Intermediate

With several regulatory and ethics authority initiatives to encourage patient input to trial design, how can authorities provide aligned review, guidance and expectations towards these activities and how should sponsors communicate patient engagement?

Learning Objectives

Discuss US (FDA) and EU (EUCTR and country ethics committees) initiatives for systematic conduct and documentation of patient input to trial design; Explain learnings from sponsor survey on sponsor submission/advice experience and guidance needs; Understand gaps and opportunities for authority (regulatory and ethics committee) harmonization in expectations and guidance; Describe emerging markets.

Chair

Melissa Pauline Herman, MSc

Component Type: Forum
Level: Intermediate

This forum will address the implementation of innovative clinical trial designs. Specific examples will be discussed and regulatory efforts to foster innovation will be examined to identify opportunities to increase stakeholder understanding.

Learning Objectives

Identify precedents and opportunities to adopt innovative trial designs in severe, chronic diseases; Describe and assess regulatory efforts to foster clinical trial innovation; Identify mechanisms to enhance public awareness regarding implementation of clinical trial innovation in drug development.

Chair

Samantha Roberts, PhD

Component Type: Session
Level: Intermediate

Hear directly from the regulators and industry about the challenges and opportunities in applying the ICH E17 guidelines. Some cases based on ICH E17 guidelines will be presented in the panel. Panelists will be asked to make comments for each case regarding points to consider in planning or evaluating results (including acceptability for regulatory submission) for each case.

Learning Objectives

Explore how ICH E17 guidelines are being implemented in the conduct of multiregional clinical trials; Evaluate the limitations, gaps, and opportunities for applying the guideline globally.

Speaker

Panelist
William Wang, PhD

Component Type: Session
Level: Intermediate

Dialogue between key stakeholders on AMA operationalization, proposals on improved collaboration, regulatory harmonization and convergence, reliance pathways and other strategies for an efficient and sustainable regulatory system in the continent.

Learning Objectives

Evaluate progress made on AMA operationalization including achievements so far with the support of partners; Discuss key insights and lessons from the Continental Pilot; Identify the way forward for successful implementation of revised AU Model Law; Cultivating a sustainable regulatory ecosystem in Africa employing good regulatory practices and agilities as well as prioritizing capacity building.

Chair

Saba Berhane, BSN, MPharm

Component Type: Session
Level: Intermediate

In this session, leaders from the EMA will provide an update on regulatory priorities. The audience will be invited to submit questions of general interest.

Learning Objectives

Describe EMAs regulatory initiatives and strategic priorities; Identify opportunities for engagement with the regulators.

Component Type: Session
Level: Advanced

Operating models and company’s culture can affect an organization’s value and efficiency. This session will delve into Regulatory Affairs operating models, including opportunities and challenges, providing best practices for your organization.

Learning Objectives

Compare and contrast operating models being used by regulatory affairs organizations that are driving greater value, efficiency, and career empowerment; Understand successes and challenges with different operating models.

Chair

Alison Maloney, PhD, MBA, MS

Component Type: Forum
Level: Intermediate

This session will provide an opportunity for key stakeholders, including regulators, product developers and patients to discuss best practices around patient experience data, including planning, collecting, and submitting such data to regulators.

Learning Objectives

Become familiar with current expectations for regulator/sponsor interactions related to patient experience data that will be used in regulatory submissions; Understand common pitfalls for submission of patient experience data in regulatory applications, and how to avoid them.

Component Type: Forum
Level: Intermediate

This forum on obesity drug development helps the audience understand the present landscape, participate in discussions on the prevention and treatment of comorbidities, and assess various strategies in enhancing clinical trial efficiency.

Learning Objectives

Understand the present landscape, opportunities, and challenges of obesity drug development; Delve into discussions on the prevention and treatment of comorbidities associated with obesity; Assess various considerations and strategies in enhancing the efficiency of obesity drug development.

Chair

Jingyu (Julia) Luan, PhD

Component Type: Forum
Level: Intermediate

This annual town hall provides a forum for senior FDA officials to reflect on key trends and discuss opportunities in rare disease drug development and review. Officials answer moderated and audience burning questions facing the work that they do.

Learning Objectives

Describe new FDA programs and initiatives intended to help facilitate development of drugs for rare diseases; Evaluate opportunities for sponsors and patient stakeholders to engage with regulators; Identify current trends in challenges and opportunities facing orphan drug development.

Chair

James Valentine, JD, MHS

Component Type: Forum
Level: Intermediate

Regulators will showcase how working together to advance regulatory science is key for new approaches. Participants will discuss how to efficiently translate drug development tools into practice, how industry and not-for-profits can drive innovation.

Learning Objectives

Learn about the usefulness of engaging in advancing regulatory science tools and subsequent translation into research practice, product development and evaluation; learn about global regulatory convergence and activities towards new regulatory science approaches to enable new products.

Component Type: Forum
Level: Intermediate

In this interactive forum experts from EMA and FDA will address questions from the audience and share their experiences of collaboration in specific areas as well as on how both Agencies collaborate in addressing current regulatory, scientific, and communication challenges.

Learning Objectives

Identify an overall understanding of the regulatory and scientific collaboration between EMA and FDA; Describe experience and explore specific areas of collaboration between the two Agencies; Examine how EMA and FDA are addressing regulatory and scientific challenges in new areas.

Component Type: Forum
Level: Intermediate

The FDA Town Hall is the forum to hear from FDA leadership about Center priorities. The audience will be invited to submit questions of general interest.

Learning Objectives

Discuss FDA Center priorities.

Chair

Amy Bertha