December 2023
December 2023 Global Forum

Table of Contents


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Editorial Board

Content stream editors

Translational science
Gary Kelloff US National Institutes of Health
Ilan Kirsch Adaptive Biotechnologies Corp.

regulatory science
Isaac Rodriguez-Chavez Independent Consultant

Patient engagement
Chi Pakarinen MediPaCe
Natasha Ratcliffe Patient Engagement Specialist
Thomas Smith Independent Patient Consultant

Data and Digital
Lisa Barbadora Barbadora Ink

Young Professionals Editor

Kaley Lugo Daiichi Sankyo

Editorial Staff

Alberto Grignolo, Editor-in-Chief Parexel International

Sandra Blumenrath, Managing Editor, Scientific Publications DIA Scientific Communications

Chris M. Slawecki, Managing Editor, Global Forum DIA Scientific Communications

Linda Felaco, Copy Editor and Proofreader

Regional Editors

David Mukanga Bill and Melinda Gates Foundation

Jin Shun Sandoz

Richard Day University of New South Wales, Medicine, St. Vincent’s Hospital

Ling Su Shenyang Pharmaceutical University, Lilly Asia Ventures

Julie O’Brien Pfizer

J. Vijay Venkatraman Oviya MedSafe

Ozawa Goshi Real Discovery Outdoors Co,. Ltd.

Cammilla Gomes Roche

Ebony Dashiell-Aje BioMarin

DIA Membership

Bringing together stakeholders for the betterment of global health care.

Autumn’s Story: A Pediatric Patient and Caregiver Journey

here are few things that a parent dreads more than having a sick child. But the journey to diagnosis and recovery for what could be a child’s fatal illness can be even more difficult.

When her 8-year-old daughter Autumn was diagnosed with Stage 2 T-cell acute lymphoblastic leukemia (T-ALL), Ebony Dashiell-Aje knew that their family was about to venture down a challenging road. But she didn’t anticipate how Autumn’s outlook would help lead their family through their darkest days, or help encourage others through a documentary of their experience titled Autumn’s Story.

In the following interview, Ebony shares three perspectives on Autumn’s road to diagnosis, treatment regimen, and recovery: as a pharmaceutical industry professional leading patient-centered outcomes research, as a pediatric oncology patient caregiver, and as Autumn’s mom. The video documentary and our audio interview with Autumn are embedded within this interview.

Working with Patient Councils to Mitigate Drug-Related Risks

Cristiane Carvalho, Lais Ribeiro, Raphael Pareschi
Roche Brazil


very authorized medicine has potential benefits and potential risks; its benefits must outweigh its risks for it to be licensed and remain in the market. We can improve the balance between benefit and risk by minimizing the risks, especially those that are serious and may substantially impact patient safety. Risk mitigation can be substantially improved by engaging patients in co-creating educational materials on the medicines they use.

A Novel Approach: Leveraging the Affective Trust Framework
Developing a Sponsor-Specific Clinical Trial Diversity Playbook
Camille Pope, Shayla Wilson, Tiffany Whitlow, Del Smith
Acclinate, Inc.

here is increasing agreement that aiming for health equity through inclusive clinical research is in the best interest of patients. This renewed focus from research sponsors on diversifying their clinical trials has also been driven by new laws (i.e., the December 2022 Food and Drug Omnibus Reform Act – FDORA, sections 3601-3604) and regulatory guidance (i.e., the April 2022 FDA Draft Guidance to Industry on Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials) that require submission of diversity plans to FDA prior to the start of pivotal studies.

Regardless of the governmental catalyst or motivational factors, research sponsors are reassessing their approach for including historically underrepresented patient populations, particularly from communities of color, in their clinical trials. The key question they should ask themselves is whether their strategies are truly positioned for success when it comes to engaging communities that they have previously excluded or ignored.
Predictive Biology Moves Towards Predictive Medicine

Edison T. Liu
The Jackson Laboratory


he major truism accepted by all is that biology is complex. Yet, since the 19th century, the major experimental focus in biology has been reductionist, which is to isolate component parts, divine their individual function, and then use this knowledge to extrapolate the behavior of the larger system. With the advent of comprehensive genomic, proteomic, and metabolomic technologies powered by advanced computational approaches, all possible genes have been mined, and each resultant mutant molecule or pathway can be inferred.

White Paper

eCTD 4.0 Implementation Including Understanding of Regional Differences and Benefits

White Paper

IQVIA: eCTD 4.0 Implementation: Including Understanding of Regional Differences and Benefits
The Electronic Common Technical Document (eCTD) is the standard format for submitting applications, amendments, supplements, and other regulatory reports to health authorities around the world. eCTD v3.2 has been the default version for more than 10 years since its release in 2008. The initial draft implementation guidelines for eCTD 4.0 were developed between 2015 and 2016 to improve robustness, flexibility, long-term stability, and a more advanced lifecycle management process. After many years of collaboration with regulatory bodies and industry sponsors, eCTD version 4.0 is finally ready for implementation.

Special Section: AI in Clinical Research

Introduction: Defining Intelligence Shaped by Subjective Perceptions
Sridevi Nagarajan

efining intelligence, whether natural or artificial, remains a realm shaped by subjective perceptions. Intelligence is often measured by the yardstick of reasoning, with mathematics serving as the benchmark for such logical processes. While deep learning artificial intelligence (AI) language models excel at pattern recognition, they currently struggle with reliably solving even eighth-grade mathematical problems. Two quotes worth considering from a human utility standpoint are George Box’s “All models are wrong, but some are useful” and Lord Kelvin’s “To measure is to know. If you cannot measure it, you cannot improve it.”

Special Section: AI in Clinical Research Part 1

Catalyzing AI In Clinical Research for New Cures

Michelle Longmire

hat does the path to eradicating all human disease look like?

Almost two years ago, I asked this question in a blog titled “Accelerating the path from possibility to proof in the development of new medicines.” I wrote that leveraging our most advanced tools would drive new synergies at the intersection of safety, science, and speed and usher in a new era of drug development to save and improve countless lives. Today, we are here. Today, we stand at the threshold of a new era of scientific development fueled by artificial intelligence and machine learning (AI and ML).

Special Section: AI in Clinical Research Part 2
3 Essential Characteristics for AI Impact in Clinical Research
Rohit Nambisan

rtificial intelligence/machine learning (AI/ML) has been leveraged in various industries for decades, but generative AI (GenAI) has recently emerged to offer new use cases in life sciences. Ultimately, GenAI—or any other form of AI—is not going to meet today’s lofty expectations if it does not provide a tangible return on investment (ROI).

Special Section: AI in Clinical Research Part 3
How AI Can Finally Address Notorious Site Burdens
Wendy Tate

ecent years have seen a dramatic increase in new clinical trial technologies. These tools, layered onto sites’ daily workflows, are meant to capture more high-quality data as well as alleviate the traditional burdens of trial operations.

White Paper

Adaptive clinical trial success via a unified data management and acquisition platform

White Paper

Adaptive Clinical Trial Success via a Unified Data Management and Acquisition Platform
Adaptive trials—clinical trials whose design allows for planned modifications to one or more trial aspects based on accumulated data—can range from early-phase studies and exploratory trials to studies conducted to satisfy post-marketing commitments. Using adaptive design in an exploratory setting can allow, for example, evaluation of a broad range of doses, regimens, and populations, giving investigators the opportunity to continue evaluating only the most promising possibilities. In short, adaptive designs maximize the trial’s potential and utility based on data gleaned through the study.
Meeting Highlights: Advancing Solutions for ALS
Advancing Solutions for ALS
Part 1: Biomarkers, Surrogate Endpoints, and Patient Preferences
Mark Albers
Harvard Medical School
Steven Kowalski
ALS Patient Advocate
Peter Sorger
Harvard Medical School
Nazem Atassi
Lahar Mehta
Sandra Blumenrath
James Berry
Massachusetts General Hospital
Stacy Rudnicki
Courtney Granville

t’s not just about survival, it’s about increasing or maintaining function as a patient. While you’re still able to, you know, give a hug to a child, or take a sip of water, … the focus seems to be on survival. And I agree, that is the end game. But functionality until you get to the cure is equally important.” —Steven Kowalski, ALS patient and patient advocate.

Meeting Highlights: Advancing Solutions for ALS
Advancing Solutions for ALS
Part 2: A Framework for Prioritizing Treatment R&D
Mark Albers
Harvard Medical School
Sandra Blumenrath
Peter Sorger
Harvard Medical School
Courtney Granville

he range of treatment modalities now available is promising for amyotrophic lateral sclerosis (ALS) patients, but frameworks to guide prioritization of development are lacking. The development of efficient solutions for patients is impeded by a multitude of challenges that require collective efforts from researchers, policymakers, and other stakeholders.

Meeting Highlights: DIA Real-World Evidence Conference
Beyond Data: Navigating the Real-World Evidence Landscape
Insights, Collaboration, and Global Impact
Sorcha McCrohan

eal-world evidence (RWE) has taken center stage in the biopharma landscape, offering insights into drug applications and therapeutic impacts in real-world settings. As the field evolves, understanding the present use of RWE and anticipating its future trajectory is key. At DIA’s recent RWE Conference, stakeholders and experts from the pharmaceutical industry, regulatory bodies, and academia convened. Various questions surrounding RWE were broached, addressing concerns about data quality and access, compliance, and the application of emerging technologies.

IQVIA RIM Advertisement
We Are DIA
DIA Congratulates Japan 2023 Regional Inspire Award Winners
Portrait photograph headshot of George Nakayama grinning
Outstanding Contribution to Health Award
George Nakayama
Daiichi Sankyo Co., Ltd.
Portrait photograph headshot of Shinzo Hiroi grinning
Excellence in Service Award
Shinzo Hiroi
Shionogi & Co., Ltd.
Portrait photograph headshot of Mika Ogasawara grinning
Excellence in Service Award
Mika Ogasawara
Pfizer R&D Japan G.K.
Portrait photograph headshot of Sho Mizokawa grinning
Leader of Tomorrow Award
Sho Mizokawa
Astellas Pharma Inc.
We Are DIA: Community Report
Bioethics Co-Chairs Review Community Roots and Preview Future Growth

thical behavior in clinical research and therapeutic product development can help demonstrate the trust that transforms patients from research participants into research partners. In the following interview, DIA Bioethics Community Co-Chairs Karla Childers and Lindsay McNair look back at their foundational educational experiences, explain the founding of the Bioethics Community, and outline topics they anticipate this community will focus on in the future. “It’s a real privilege to think about not just what we’re doing but the people we’re doing it for and how we’re doing it,” Karla explains.

Videos | Around the Globe: US
New IRB/EC Toolkit: Streamlining Ethical Review of DCT Applications

s the number of decentralized clinical trials (DCTs) continues to grow, the US FDA, the US Office for Human Research Protections, Harvard’s Multi-Regional Clinical Trials (MRCT) Center, Medable, and other industry stakeholders have collaboratively developed a new toolkit to streamline and accelerate IRB/Ethics Committee review of DCT applications. Medable Chief Science Officer Pam Tenaerts and Harvard MRCT Director Barbara Bierer explore the strategic and practical benefits of this new toolkit with DIA Head of Science and Scientific Strategy Courtney Granville in this DIA Global Annual Meeting 2023 interview.

Around the Globe: US
The Evolution of US FDA Diversity Requirements in Clinical Research
Darshan Kulkarni
Kulkarni Law Firm

he US Food and Drug Administration has increasingly recognized the importance of diversity and representation in clinical trials over the past few decades. This evolution reflects growing awareness across medicine that differences in age, gender, race, and ethnicity can impact disease risk, treatment response, and health outcomes. A review of the relevant legal and regulatory history helps document this growing awareness and points the way to further necessary advances to make clinical research truly available to all and representative of all.

Videos | Around the Globe: US
Women in Health 2023: Curious, Persistent, Resilient

Natalia Ledo Husby, CluePoints

uravit Clinical Research Business Development Manager Natalia Husby explains the skillset and shares advice that has helped her discover her role as a woman in the life sciences industry. “It’s exciting to see more and more women moving into leadership positions in science. Thankfully, I had professors who were women who were mentors to me,” she says. “But on the business side, you’re seeing women CEOs of biotech companies and the opportunity of going into different avenues within science. It doesn’t have to just be in the lab as a research scientist.”

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Thanks for reading our December 2023 Issue!
Views and opinions expressed in Global Forum are those of the authors alone and do not necessarily represent those of DIA or any other agency, organization, employer, or company. DIA does not guarantee the accuracy or completeness of any information published in Global Forum and will not be responsible for any errors, omissions, or claims for damages, including exemplary damages, arising out of use, inability to use, or with regard to the accuracy or sufficiency of the information contained in Global Forum.