DIA 2025 Global Annual Meeting: Full Schedule

2:15pm EDT

Expanding Innovative Master Protocol Trial Designs to Cell and Gene Therapies for Rare Diseases

Monday June 16, 2025 2:15pm - 3:15pm EDT

Component Type: Forum
Level: Intermediate

Master Protocols, including basket trials and umbrella trials, enable efficiencies in investigating multiple conditions or multiple treatments, respectively, under a single trial design. Applying Master Protocol trial designs to Cell & Gene therapies (CGTs) can streamline clinical and regulatory processes to speed the delivery of treatments to patients with rare diseases.

Learning Objectives

Identify barriers to and solutions for leveraging Master Protocol clinical trial designs to study Cell & Gene Therapies for rare diseases.

Chair

Nancy Myers, JD

Speaker

Academic Perspective
Kiran Musunuru, MD, PhD, MPH

Researcher Perspective
Jeff Allen, PhD

Speakers

Jeff Allen

President and Chief Executive Officer, Friends of Cancer Research, United States

Jeff Allen, Ph.D. serves as the President and CEO of Friends of Cancer Research (Friends). During the past 25 years, Friends has been instrumental in the creation and implementation of policies ensuring patients receive the best treatments in the fastest and safest way possible. As... Read More →

Kiran Musunuru

Professor of Medicine, University of Pennsylvania, United States

An actively practicing cardiologist and committed teacher, Kiran Musunuru, MD, PhD, MPH, ML, MRA, is Professor of Cardiovascular Medicine, Genetics, and Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. His research focuses on the development of novel... Read More →

Nancy Myers

CEO and Founder, Catalyst Healthcare Consulting, United States

Nancy Bradish Myers, JD, CEO & Founder, Catalyst Healthcare Consulting, is a sought-after leader with deep expertise in health law, regulation and policy. She has led a successful strategic regulatory policy consultancy for 19 years and has been active in the DC policy arena for three... Read More →

Monday June 16, 2025 2:15pm - 3:15pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA

02: ClinTrials-Ops, Forum

Level Intermediate
Keyword Rare Disease, Clinical Trial Design, Cell and Gene Therapy
Level Intermediate
format csv
Feature Topics Rare Disease,Clinical Trial Design,Cell and Gene Therapy
Tags Forum

3:45pm EDT

Enhancing Evidence Generation Across Pharmaceutical Life Cycle: The Importance of Integrating the Patient Lived Experience

Monday June 16, 2025 3:45pm - 4:45pm EDT

TBD

Component Type: Session
Level: Intermediate

Addressing data gaps in drug development, especially capturing patient experiences in both rare and non-rare diseases, is crucial. From Proof of Concept to Proof of Value, integrated evidence planning and incorporating patient voices in Target Product Profile (TPP) creation drive comprehensive evidence generation.

Learning Objectives

Discuss the utility in global drug development context, including post-marketing efforts; Identify strategies to create a patient informed comprehensive Target Product Profile; Recognize strategies outlined in the interactive guide to align PAG and industry efforts in creating a comprehensive Target Product Profile.

Chair

Agathe Le Lay, PhD, MSc

Speaker

What are we Missing: Potential Data Gaps in Drug Development and the Importance of Capturing Lived Experience in rare and non-rare Diseases
Kristin Hatcher

From Proof of Concept to Proof of Value, an industry approach to integrated evidence planning throughout the product lifecycle
Agathe Le Lay, PhD, MSc

New Strategies and Practices to Incorporate the Patient Voice in TPP Creation
Patricia Roselle

New Strategies and Practices to Incorporate the Patient Voice in TPP Creation
Bruce E. Miller, PhD

Panelist
Martine Zimmermann, PharmD

Speakers

Kristin Hatcher

Director of Pediatric and Rare Disease/ Alpha One Lived Experience Advocate, Global Liver Institute/Alpha One Patient, United States

Agathe Le Lay

Global Head of Value Evidence & Patient Insights, H. Lundbeck A/S, Denmark

Agathe Le Lay is the Global Head of Value Evidence and Patient Insights at H. Lundbeck A/S, and is responsible for leading a global team responsible for the strategic planning and delivery of scientific evidence supporting R&D assets as well in-line brands. Agathe is an expert in... Read More →

Bruce Miller

Chief Scientific Officer, COPD Foundation, United States

Dr. Miller is the Chief Scientific Officer of the COPD Foundation overseeing its patient-focused research programs. Inclusion of patient partners is a requirement for any research activity the foundation supports. Dr. Miller has been a participant in the Paladin Consortium since its... Read More →

Patricia Roselle

Head of Patient Stakeholder Engagement, Sanofi, United States

Martine Zimmermann

Senior Vice President, Head of Global Regulatory Affairs, Ipsen , France

Dr Zimmermann joined Ipsen in January 2023 as SVP, Head of Regulatory & Quality R&D, based in France and since January 2024, SVP, Head of Global Regulatory Affairs. . She has over 25 years of combined R&D and global regulatory strategy experience. She joined Alexion Pharmaceuticals... Read More →

Monday June 16, 2025 3:45pm - 4:45pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA

05: Patient-Impact-Product-Dev, Session

Level Intermediate
Keyword Rare Disease, Patient Experience
Level Intermediate
format csv
Feature Topics Rare Disease,Patient Experience
Tags Session

8:30am EDT

Diversity Action Plans in Rare Genetic Disease Drug Development: Obstacles and Opportunities

Tuesday June 17, 2025 8:30am - 9:30am EDT

TBD

Component Type: Forum
Level: Advanced

This forum will provide information regarding new requirements for Diversity Action Plans. A panel of experts will describe details of these requirements, implementation challenges for rare genetic diseases, and strategies to address these challenges.

Learning Objectives

Describe how regulatory requirements for Diversity Action Plans might apply to orphan drug development; Plan regulatory strategy for inclusive and representative rare disease drug development; Identify challenges regarding compliance to Diversity Action Plan requirements in rare disease settings.

Chair

Mark Tobolowsky, JD

Tuesday June 17, 2025 8:30am - 9:30am EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA

02: ClinTrials-Ops, Forum

Level Advanced
Keyword Rare Disease
Level Advanced
format csv
Feature Topics Rare Disease
Tags Forum

10:30am EDT

US and EU Orphan Drug Regulations: Shaping the Ecosystem for Rare Diseases

Tuesday June 17, 2025 10:30am - 11:30am EDT

TBD

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

Discuss 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; Explain the importance of collaboration between regulatory agencies, industry stakeholders, and patient advocacy groups.

Chair

Imran Shah

Speakers

Imran Shah

SVP & Head, Global Regulatory, R&D Quality and Safety (RQS), EMD Serono, United States

Tuesday June 17, 2025 10:30am - 11:30am EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA

09: Regulatory, Session

Level Intermediate
Keyword Rare Disease
Level Intermediate
format csv
Feature Topics Rare Disease
Tags Session

1:45pm EDT

How Medical Writing Can Support Accelerating the Development of Rare Disease Therapies: Insights from the FDA’s START Program Pilot

Tuesday June 17, 2025 1:45pm - 2:45pm EDT

TBD

Component Type: Session
Level: Intermediate

This session will explore the FDA START program for accelerating rare disease therapies. We will invite companies who participated in the START pilot and a FDA representative.

Learning Objectives

Explain the objectives of the FDA START Program and its role in supporting rare disease development; Discuss how medical writers can support companies applying for and participating in the START program.

Chair

Robin Whitsell

Speaker

Panelist
Madiha Khalid, PhD

Panelist
Jhon Cores, PhD

Speakers

Jhon Cores

Associate Director, Medical Writing, Moderna, Inc, United States

Jhon is an Associate Director of Medical Writing at Moderna Inc., where he serves as the Medical Writing lead for various rare disease an oncology programs. He has 10 years of experience in the field working in different sectors, including university startups and contract research... Read More →

Madiha Khalid

Senior Director, Medical Writing, Larimar Therapeutics, United States

Madiha is a pharmaceutical industry professional with a background in fundamental bench research. Her experience as a regulatory medical writer working with small, mid-size, and large pharmaceutical companies over the past 10 years has given her an in-depth understanding of all phases... Read More →

Robin Whitsell

President, Whitsell Innovations, Inc., United States

Founder and president of Whitsell Innovations, Inc., a medical writing firm headquartered in Chapel Hill, NC, Ms. Whitsell has over 25 years of experience, specializing in global submission strategy, regulatory medical writing, emerging technologies, and data visualization. Prior... Read More →

Tuesday June 17, 2025 1:45pm - 2:45pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA

04: MedAffairs-SciComm, Session

Level Intermediate
Keyword Rare Disease
Level Intermediate
format csv
Feature Topics Rare Disease
Tags Session

10:30am EDT

Why Does Drug Loss Occur? Challenges to be Addressed for Patients, and Japan's Contribution to Globalized Drug Development

Wednesday June 18, 2025 10:30am - 11:30am EDT

TBD

Component Type: Forum
Level: Intermediate

This forum will address need for a global development strategy aimed at enhancing patient access to new drugs and maximizing business opportunities in Japan. The panelists will consist of members from pharma industry associations, agency and academia

Learning Objectives

Discuss maximizing value of innovative products for the global market including Japan; Apply learnings to clinical development strategy to expand target patients and market effectively; Describe need to enhance access to innovative drugs for children and patients with rare diseases; Discuss influencing regulators, industry, and trial sites to improve the local environment and reduce burdens in global simultaneous development.

Chair

Toshiharu Sano, RPh

Speaker

Changes in Japan's Pharmaceutical Regulations and Clinical Environment
Akihiro Ishiguro, PhD

Initiatives of Medical Institutions - Introduction to High-Quality Trials Using Networks
Kenichi Nakamura, DrMed

What is Drug Lag and Loss: Challenges for Globalization in Japan
Nobutaka Kobayashi, MPharm

Speakers

Akihiro Ishiguro

Deputy Review Director, Office of New Drug III, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Dr. Akihiro Ishiguro is the Director of the Review Planning Division, Office of Review Management, Pharmaceuticals and Medical Devices Agency (PMDA). His professional experience includes new drug review and post marketing drug safety. He had the opportunity to join projects to develop... Read More →

Nobutaka Kobayashi

Deputy Director, Mitsubishi Tanabe Pharma Corporation, Japan

Kobayashi was engaged in medicinal chemistry as a research scientist in the pharmaceutical company for 13 years. After that, he was engaged in medical policy, drug price application, and management planning for 13 years.

Kenichi Nakamura

Director, Department of International Clinical Development, National Cancer Center Hospital, Japan

Dr. Nakamura is the Director of the Department of International Clinical Development at the National Cancer Center Hospital. He completed his degree at Kyoto University in 1999. After spending seven years training as a general surgeon, he joined the National Cancer Center to participate... Read More →

Toshiharu Sano

Associate Vice President, MSD, Japan

Toshiharu Sano has over 30 years of experience in pharmaceutical industry and has broad experiences in Clinical Research, R&D planning and Business Operations for Clinical Development in Japan. He also worked as several initiative’s leads and change manager through his career in... Read More →

Wednesday June 18, 2025 10:30am - 11:30am EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA

07: ProjectManagement-StrategicPlanning, Forum

Level Intermediate
Keyword Rare Disease, Global Perspectives
Level Intermediate
format csv
Feature Topics Rare Disease,Global Perspectives
Tags Forum

4:00pm EDT

FDA Rare Disease Town Hall

Wednesday June 18, 2025 4:00pm - 5:00pm EDT

TBD

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

Speakers

James Valentine

Director, Hyman, Phelps & McNamara, PC, United States

James Valentine, JD, MHS is an attorney at Hyman, Phelps & McNamara where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine has been central... Read More →

Wednesday June 18, 2025 4:00pm - 5:00pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA

09: Regulatory, Forum | 02: ClinTrials-Ops, Forum

Level Intermediate
Keyword Rare Disease, Student Programming
Level Intermediate
format csv
Feature Topics Rare Disease,Student Programming
Tags Forum

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