June 2021
June 2021 Global Forum

Table of Contents


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Editorial Board

Content stream editors

Translational science
Gary Kelloff US National Institutes of Health
David Parkinson ESSA Pharma, Inc.

regulatory science
Isaac Rodriguez-Chavez PRA Health Sciences

Patient engagement
Trishna Bharadia Patient Advocate and Media Contributor
Mary Stober Murray National Minority Quality Forum

Monika Schneider Shionogi

Editorial Staff

Alberto Grignolo, Editor-in-Chief Parexel International

Sandra Blumenrath, Managing Editor, Scientific Publications DIA Scientific Communications

Chris M. Slawecki, Senior Digital Copyeditor DIA Scientific Communications

Regional Editors

David Mukanga Bill and Melinda Gates Foundation

Jin Shun Sandoz

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

Judith Glennie JL Glennie Consulting, Inc.

Ling Su Shenyang Pharmaceutical University, Lilly Asia Ventures

Thomas Kühler Sanofi R&D

J. Vijay Venkatraman Oviya MedSafe

Ozawa Goshi Real World Data Co. Ltd.

Cammilla Gomes Roche

Ebony Dashiell-Aje BioMarin

Young Professionals Editors

Kaley Lugo Daiichi Sankyo
Saloni Patel Acorda Therapeutics

DIA Membership

Bringing together stakeholders for the betterment of global health care.

DIA 2021 Global Annual Meeting Advertisement
The pandemic has underscored the connectedness of all people.
It has brought to light disparities and inequities, the spread of misinformation, and challenges in value and access. It has highlighted diversity, recognizing that geographical, racial, and ethnic differences are part of the grand puzzle we all seek to solve.

With this at the front of our minds, the DIA 2021 Global Annual Meeting celebrates our Honorary Co-Chairs, whose dedication to international collaboration and patient engagement has been unparalleled, and welcomes their remarks in our Opening Plenary Session. As leaders of the founding regulatory agencies of the ICH, they lead organizations that have made significant advances in global convergence and harmonization of regulatory science and regulation for more than 30 years.

Changing Landscape: The Role of Patient Influencers in the Healthcare Ecosystem
Trishna Bharadia
Patient Leader
Richelle Horn
WEGO Health

he discussion around patient influencers is becoming increasingly prominent within the healthcare ecosystem. Who are they? What role do they play in developing better patient outcomes and healthcare improvements? How do they positively impact health decisions? This article aims to explore these questions and provide key insights into how the biopharmaceutical industry can and should be collaborating with this important stakeholder group to create positive change in healthcare.

Patient-Focused Initiatives in Oncology: How the US Government Paved the Way
Vishal Bhatnagar
Oncology Center of Excellence
Lori Minasian
Division of Cancer Prevention
US National Cancer Institute
Paul G. Kluetz
Oncology Center of Excellence

ver the past decade, a series of government-lead events set in motion a sea change in how patient-reported outcomes (PRO) in cancer clinical trials are assessed and reported. PRO measures have long provided an opportunity for the patient’s voice to be heard, and when rigorously implemented, PRO assessments can add quantitative symptom and functional data to help characterize risks and benefits of cancer treatments. An important example is the use of PRO to provide a direct report of symptoms and side effects from the person experiencing them.

The US National Cancer Institute (NCI) and the US Food and Drug Administration (FDA) have created publicly available tools to advance the collection, measurement, analysis, and reporting of patient-reported symptomatic adverse events in cancer trials. These initiatives have had ripple effects, expanding the definition of tolerability, developing guidelines to manage symptoms, providing new tools for industry to enhance dose finding in early-phase trials, and providing PRO data to enhance the patient-clinician interaction at the point of care.

In this article, we discuss:

  • NCI’s development of the PRO version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE), the FDA’s work to identify analytic and visualization methods to describe PRO-CTCAE data culminating in Project Patient Voice;
  • future uses of PRO-CTCAE and other PRO data to inform early- and late-stage drug development; and
  • the history of sustained scientific collaboration necessary to advance the rigorous use of PRO data to help characterize tolerability of cancer drugs.
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Modernizing the Digital Evidence Generation Ecosystem
Kai Langel
Janssen Clinical Innovation

igital health and patient-centricity are all the rage these days, with these topics appearing as key headlines across the industry. What does it mean, what’s the value, and why are we not there yet?

White Paper Podcast

Jerome Premmereur smiling

White Paper Podcast by Covance Patient Safety

Virus Variants No Threat to Vaccine Safety
In early 2021, millions of people began receiving vaccinations to prevent COVID-19 infection. This global rollout of vaccinations in such a relatively short timeframe, complicated by virus variants emerging in many countries, presents enormous and previously unseen challenges to professionals working to ensure the safety of these vaccines. “The virus is still adapting itself to be sure that it can still reproduce although there are many people that are vaccinated,” explains Jerome Premmereur, Head of Global Drug Safety and Pharmacovigilance, Covance Patient Safety Solutions. “So far, so good. When you look at the British one and the British numbers, we identify quickly that when there is nearly only one variant, it is really impacted by the vaccine.” Similar stories in different countries and different vaccines (i.e., Israel and Pfizer vaccine) have also been observed.

Meeting Highlights: DIA Europe 2021

Industry Survey on Implementation of ICH E17 Guideline
General Principles on Planning and Designing Multi-Regional Clinical Trials
Solange Corriol-Rohou
Dorothee Grimald
Anette Hjelmsmark
Novo Nordisk
Carolyn Hynes
Henrik Nielsen
Novo Nordisk
Maria Cristina Mota
Julie O’Brien
On behalf of the European Federation of Pharmaceutical Industry Associations (EFPIA)
DIA Europe

ow does industry perceive the impact of ICH E17 Multi-Regional Clinical Trial (MRCT) guideline in some key international countries? Are the guideline recommendations fully utilized, and how are regulatory authorities being perceived in implementing them? This article shares the results from a recent industry survey about how implementation of the E17 guideline is progressing in seven countries.

Meeting Highlights: DIA Europe 2021
ICH E17 Guideline on Multi-Regional Clinical Trials
Catalyst for Simultaneous Global Development and Registration
Julie O’Brien
On behalf of the EFPIA International Regulatory Expert Group
DIA Europe

ulti-Regional Clinical Trials (MRCT) is not a new term and has been in use for many decades. It refers to a clinical trial conducted in more than one region under a single protocol and allows data from one country or region to help gain approval in another country or region. This is a critical component of successful and timely development and registration of medicines globally. The ICH E17 guideline on General Principles on Planning and Design of MRCTs was finalized in 2017. It describes how, with careful planning, new medicines can be developed globally from the outset.

White Paper Podcast

Dr. Qi Li headshot
Stannard headshot

White Paper Podcast by InterSystems

Learning Health Systems Creating Virtuous Circles of Care
With so much real-world data available, how can pharmaceutical and healthcare delivery organizations achieve alignment to maximize its value? “Sponsors definitely recognize the value of real-world data, real-world evidence, and they’re getting more comfortable working with health systems to find meaningful use cases using the health system data. The desire is there, the capability is there, and people are willing to generate evidence together and help each other on a global scale,” explains InterSystems Physician Executive Dr. Qi Li in this interview with healthcare and technology writer Jack Murtha. “A learning health system is a system that seeks to continuously generate and apply evidence innovation, quality, and value in health care, and the aim of the learning health system is to really enable that continuous, efficient, and rapid cycle of study feedback and then subsequent practice change,” concurs Matthew Stannard, InterSystems Life Sciences Advisor. “This concept of a virtuous circle that provides care, collects data, and then feeds those data back into the care process and continuously improves insights and outcomes is where we see the future headed.”
Meeting Highlights: Diversity, Equity, and Inclusion in the Drug Development Lifecycle
Diversity and Inclusion in Clinical Trials
An Insider’s View
Tamei Elliott
Courtney Granville
DIA Diversity circular icon

ustained integration of diversity and inclusion in clinical trials and drug development will require more than just outreach to marginalized and underrepresented communities when their participation is needed by the scientific community. To achieve genuine equity and inclusion, we must make long-term structural changes in the science of medicine and drug development. In highly heterogeneous communities, it is important to ensure similar diversity is included and represented in the populations tested within each clinical trial.

In a fireside chat at DIA’s Diversity, Equity, and Inclusion in the Drug Development Lifecycle Meeting in April 2021, Robert Fullilove, Associate Dean for Community and Minority Affairs, and Professor of Clinical Sociomedical Sciences at Columbia University, provided insight into the changes that are needed to build a fully inclusive process from the perspective of someone who knows underrepresented communities from the inside.

Meeting Highlights: Diversity, Equity, and Inclusion in the Drug Development Lifecycle
Building Healthcare Equity: FDA Frameworks and Community Foundations
Chris M. Slawecki
DIA Diversity circular icon

atients of different genders, ages, races, or ethnicities can react differently to certain medical products, so it is essential that clinical trials study the safety and efficacy of these products in these different groups to reasonably predict how these products will perform in the various, diverse populations that will ultimately use them.

Historically, however, this has sometimes seemed to work more as theory than practice. In 2017, for example, the International Myeloma Foundation’s African American Initiative estimated that African-Americans comprised 20% of the myeloma patient population but accounted for only 6% of patients in myeloma clinical trials.

The global COVID-19 pandemic shone a bright light on the need for healthcare systems and communities to work for the benefit of all.

What steps are being taken by clinical researchers, the industry sponsors of that research, and the Agency regulating that research to ensure more equitable healthcare in the US, from bench to bedside?

White Paper
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Document Quality Control Emerges as a Discipline in Its Own Right
Compiling a high quality regulatory submission is a basic requirement for biopharmaceutical companies. These submissions are highly technical and complex and, without the right approach, producing these to the required level of quality can be challenging and resource intensive.

Click through to read our white paper and find out more about how we can help you to innovate and improve your documents’ consistency and integrity.


Australia: Growing Clinical Trials Even in Pandemic’s Wake

White Paper Podcast

Charlotte Moser headshot
Kinisha Gala headshot

White Paper Podcast by LARVOL

Emerging Trends in Immuno-Oncology Trial Design
Recent approvals of multiple immune checkpoint inhibitors, which help the body target and combat cancer cells, has both changed the standard of care in different cancers and brought cancer research to a critical juncture. “When you want to design a clinical trial and observe benefit for a new drug, you have to take into account all the other treatments given as standard of care for that type of cancer. Overall, we are working in more specific subgroups of the disease, which means automatically smaller cohorts, and the challenge is to monitor not only the treatment you add but all the other treatments given as well,” explains Charlotte Moser, Vice President, Clinical Development, Galera Therapeutics, Inc. “The longer timeline makes clinical trial design in immunotherapy especially difficult, and we still have a lot of work to do in terms of predicting response and how to best use biomarkers,” says Kinisha Gala, Project Owner of Oncology, LARVOL.

Around the Globe

Pharmaceutical Policy Highlights in the Canadian Federal Budget
Judith Glennie
JL Glennie Consulting Inc.

n addition to obvious investments to support various sectors suffering as a result of the pandemic, Canada’s 2021 Federal budget continues its support of previous initiatives related to pharmaceutical policy.

The October 2019 federal election resulted in a minority government for the incumbent Liberal Party. The budget put forth in March 2019 served as the basis of the government’s platform for that election, and the pharmaceutical policy components of that platform (i.e., patented medicines pricing regulations, National Pharmacare, and a rare disease strategy) were highlighted in the October 2019 edition of Global Forum.
As with most countries, the COVID-19 pandemic has fully occupied all levels of government since early 2020. Thus, the federal budget tabled on April 19, 2021 was the government’s first full budget in almost two years. It was a critical document, both in terms of its content as well as in the context of a minority government. In a parliamentary system, budget votes are considered “confidence votes” and–if voted down–can result in the fall of a minority government. The plan that was tabled represents—for all intents and purposes—the governing Liberal party’s election platform for the foreseeable future. (Note: The budget was passed by parliament on April 26, 2021.)

With the view towards rebuilding, the budget also touches on investments in innovation and improving domestic capacity that may benefit the pharmaceutical sector in the long run.

White Paper

Patient Engagement Guide for Sponsors and CROs

White Paper

Medrio’s Patient Engagement Guide for Sponsors and CROs
In light of the past year, sponsors and CROs must focus now, more than ever, on building patient engagement into their study builds from the ground up. This involves applying the patients’ perspective into recruitment and outreach strategies, removing barriers for entry and retention, and creating healthy two-way information exchanges between patients and their clinical teams. But effective, sustainable patient engagement goes beyond single-focused solutions for recruitment and retention. This white paper focuses on how sponsors and CROs can put themselves into the mindsets of their patients and find strategic ways to incorporate patient engagement into the entire trial lifecycle.

Around the Globe

China’s New Era of Reform Transforming Regulatory Professionals
Fang Zhou
Harbour BioMed

he Chinese pharmaceutical industry, through the first five years of China’s National Medical Products Administration (NMPA) regulatory reform which began in 2015, has experienced groundbreaking changes and stepped into a new era full of blooming innovation and intense competition. Today, the regulatory professionals are facing unprecedented opportunities as well as challenges in this diversified environment.

White Paper Podcast

Dale Usner headshot
Jim Townsend headshot

White Paper Podcast by SDC

Data Science & Automation Driving Research Science Forward
In clinical research, only one thing is guarded as carefully as the patient – and that is the data that patients help generate. For biometrics clinical research organizations, developing and integrating a data science and automation department into operations is a critical component of modern trial management. How are advances in data science changing research science? “We all recognize clinical trial data hosts a wealth of information, and data science allows us to analyze and present this information enabling us to make more efficient decisions around our protocol development and study execution,” explains Dale Usner, Statistics & Data Corporation (SDC) Chief Scientific Officer and Senior VP of Strategic Scientific Consulting, in this Q&A with SDC Senior VP of Business Development Jim Townsend. “Analyzing historic and current information will be a real benefit towards enabling a robust protocol to be built and executed more expeditiously, while retaining high quality.”

Around the Globe

Impact of COVID-19 on US Clinical Studies Conducted by Japan-Based Pharmaceutical Companies
Daisuke Tasaki
Kazumi Taguchi
Takaaki Nakamura
Yuriko Fujita
Hidetoshi Takeda
Masaya Tanaka
Takahiro Mitsuyoshi
Yu Tanaka
Hiroko Neely
Shunsuke Kobayashi
Takahiro Yoshida
NY Chapter, Project Planning & Management (PP&M) Forum

he spread of COVID-19 and the resulting social activity restrictions have severely impacted drug development and threatened the continuity of clinical trials in the US. Japanese pharmaceutical companies that develop drugs in the US were affected by these unprecedented circumstances as severely as US companies.

Moreover, due to their relatively limited financial and human resources (compared to the US pharmaceutical giants), these Japanese companies were forced to be more cautious in their decisions to start, postpone, or suspend clinical trials. How did these expatriates of Japanese companies in the US handle this pandemic?
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Thanks for reading our June 2021 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.