April 2023


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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 COUCH Health
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, 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

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.

Five Building Blocks to Achieving Diversity, Equity, and Inclusion in Clinical Research
Maimah Karmo
Tigerlily Foundation
Amy Sitnick
Del Smith
Stephen Walker
CSL Behring
Kate Wilson

linical trials rely on volunteers, and it’s important that they come from diverse backgrounds. However, it’s been documented that 85% of those who participate in clinical research are Caucasian.

While the desire to increase diversity in clinical trials isn’t new, the industry has renewed its commitment to make a difference. In fact, 84% of sponsors and CROs who responded to a recent Greenphire survey said that to “increase diversity of underrepresented populations (including ethnicity, socioeconomic, urban/rural, gender and more) among clinical trial participants” is a goal.
Immunotherapies Merit Unique Patient-Reported Outcomes to Inform Treatment Tolerability
Matthew Reaney
Catherine Coulouvrat
Mark Stewart
Friends of Cancer Research
Courtney Granville,
Maria Paula Bautista Acelas
Drug Information Association

mmuno-oncology (I-O) therapies provide a unique alternative for treating different cancer types. By enhancing the individual’s immune system to target and fight cancerous cells, these therapies can increase the survivorship rate for many patients. But they can also lead to a wide experience of symptoms that affect treatment tolerability.

Understanding tolerability as part of the development and delivery of I-O therapies is important. New FDA guidance advocates for collecting patient-reported outcomes (PROs) to better assess tolerability and optimize dosage in trials. However, using PROs to inform tolerability is not without its challenges.
Use Plain Language to Increase Understanding: The MRCT Center Clinical Research Glossary
Sylvia Baedorf Kassis
Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard
Rhonda Facile
Kayleigh To
Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard
Sarah White
Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard
Barbara E. Bierer
Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard

Harvard Medical School


edical and research information and terminology can be challenging to understand, even at the best of times. But for patients and their loved ones, facing a difficult health situation and considering possible research options makes sifting through technical study information an even more daunting task. As an industry, we must work toward using harmonized plain language definitions to explain clinical research. The MRCT Center together with CDISC is bringing us one step closer to that goal, with the MRCT Center’s patient-centered Clinical Research Glossary becoming a global standard.


ANVISA Explains New Online Optimized Assessment Project
For regulators in Brazil, the pandemic’s aftermath includes a backlog of post-approval changes, delayed by emergency development of COVID-19 vaccines and therapies, related to biologic product quality. ANVISA’s new Online Optimized Assessment Project has significantly resolved these pending requests. “The pandemic had already enabled use of online tools for remote assessment of applications and virtual meetings between ANVISA and applicants, and also helped us be closer to other reference regulatory authorities in the process of building trust,” explains project architect Elkiane Macedo Rama, an Adviser of ANVISA’s Third Directorate. “The Online Optimized Assessment Project was a way to gather all these tools in a strategy to speed up the review process of these applications.”
The Rights and Ways for Children to be Involved in Clinical Trial Decisions
Begonya Nafria Escalera
Sant Joan de Déu Research Institute, Spain

hildren and young people have been historically excluded from participating in clinical trials to protect them from any potential harm. Considering that there are many conditions that only affect the pediatric population, the only way to study them and to investigate novel potential treatments is by designing clinical trials addressed to this targeted population.

In the near future, in Europe, new strategies will be implemented to design and conduct pediatric clinical trials based on the revision of the Pharmaceutical Legislation that will have an impact on Pediatric & Orphan Drug Development.
Complex Biomarkers Create New Opportunities for Advancing Cancer Medicine
Gary J. Kelloff
National Cancer Institute, NIH
Caroline C. Sigman
CCS Associates

uccesses in understanding disease biology lead to the discovery and use of complex biomarkers in drug development, characterizing patient disease and making patient care more effective. High-technology methods help address the opportunities and challenges of using complex biomarkers, most often in clinical cancer research geared toward precision medicine. These technologies are less invasive and/or provide more data than standard tissue biopsy and imaging.

Biomarkers are used in oncology for early detection, as predictive and prognostic signals, and for response monitoring. Next-generation sequencing (NGS), liquid biopsies (LBx), multiplex immunohistochemistry (IHC), digital biopsy, and real-world data (e.g., from electronic medical records or wearables) have expanded the possible uses. Challenges to developing effective biomarkers include factors in the tumor microenvironment (TME), the large amount of data generated leading to the need for standards development to cover sample acquisition and analysis, the small amounts of analyte available, and the issues around FDA clearance, use of laboratory-developed tests, insurance coverage, and IP.
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Modern HCP Portal Strategies for Pharma Leaders: An Industry Assessment

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Think Company: An Assessment of HCP Portals in 2022: Modern HCP Portal Strategies for Pharma Leaders
Today’s top US biopharmaceutical companies are working to improve the digital experience of their HCP portals and implement modern HCP engagement strategies—but few leaders have the industry data they need to understand their opportunities in the larger landscape. In this report, Think Company compiled data on 39 HCP portals from the top 20 US pharmaceutical companies to gather insight into who is leading the way in digital maturity, the trends we’re observing in updates over time, and the modern best practices for pharmaceutical leaders who want to better engage with HCPs, earn trust, and build their brand reputation.
Special Section: Clinical Research in Ukraine
The Lessons of War Inform Good Clinical Practice Guidelines
Viktoriia Dobrova
National University of Pharmacy, Ukraine
Mark Turner
University of Liverpool (UK)
Katrina A. Bramstedt
Roche, Switzerland
Sandor Kerpel-Fronius
Semmelweis University, Hungary
Beate Aurich
conect4children, France
Francis P. Crawley
Good Clinical Practice Alliance – Europe (GCPA)

Strategic Initiative for Developing Capacity in Ethical Review (SIDCER)


he ongoing war in Ukraine is teaching us invaluable lessons about the resilience of people and the impact of such events on clinical trials and research. It also reveals opportunities to improve guidance to protect people and ensure research quality during disruptions which can also include pandemics and natural disasters. One such opportunity arises from the ongoing revision of the ICH E6 Good Clinical Practice (GCP) Guideline as this document shapes clinical trial operations.

Clinical trials are a critical element of the development of medicines and medical technologies that are both effective and safe. Ukraine actively participates in research in the fields of cardiovascular disease, oncology, and rare/orphan disease, led by drug and device companies based in Japan, the EU, the US, and other countries in the modern, globalized world. Participation of patients and healthy volunteers in clinical trials in Ukraine contributes to research that potentially benefits future patients around the world.

Protecting research participants and the quality of research data are fundamental to global GCP and are key priorities for regulatory authorities, including research ethics committees. The local regulatory authority in Ukraine is collaborating with regulatory and health authorities such as the European Medicines Agency and the World Health Organization to identify solutions to the challenges imposed by the war. These learnings may provide useful lessons for similar situations elsewhere and contribute to the evolution of regulatory and clinical trial practice.

Special Section: Clinical Research in Ukraine
Three Perspectives: Maintaining Good Clinical Practice (GCP) During War
Igor Bondarenko
Dnipro State Medical University
Evgeny Levenko
ARENSIA Exploratory Medicine GmbH
Olena Popova

aintaining quality and ensuring participant protection in clinical trials during wartime takes incredible effort and coordination. The ongoing war places unprecedented pressure on the healthcare system and clinical trials in Western Ukraine, and on patients, who need to find ways to continue treatment in their own country or find treatment in another country and then travel there. Investigators and sites coordinated activities to maintain good clinical practice (GCP) as the war in Ukraine began, ensuring continuity of research and care and providing lessons for future clinical trial and research conduct during disruptions such as war.

Special Section: Clinical Research in Ukraine
GCP in the Context of Global Regulatory Reliance and the Situation in Ukraine
Samvel Azatyan
World Health Organization

t is clear that good health is not possible without access to medical products, which are in this context considered to be instruments for public health, and that clinical trials are an integral and essential part of the overall product development process.

Registering clinical trials and submitting them for review by the appropriate regulatory and ethical oversight bodies ensures that their healthcare strategies are informed by scientific evidence. Article 35 of the Declaration of Helsinki states that every clinical trial must be registered in a publicly accessible database before recruiting the first subject. This registry allows scientists in different countries to know if they are duplicating research already being conducted elsewhere.
Special Section: Clinical Research in Ukraine
Clinical Trials Continuity: Ukraine State Expert Center Perspective and Recommendations
Taisa Herasymchuk
State Expert Center, Ministry of Health, Ukraine
Sergii Rasputniak
State Expert Center, Ministry of Health, Ukraine
Victoriia Dobrova
National University of Pharmacy, Ukraine

efore the war, the field of clinical trials had developed steadily in Ukraine, framed in a strict legal framework oriented to the legislation of the European Union and based on the Declaration of Helsinki, ICH GCP, and EU Directive 2001/20 (the Clinical Trials Directive). The State Expert Center regularly conducted clinical trials inspections, and its inspectors took part in EU GCP Inspectors Working Group Workshops and participated as observers in EMA, FDA, and PMDA inspections.

The number of approved clinical trials had been increasing annually in Ukraine. These were mostly phase 2 and 3 clinical trials in oncology, neurology, psychiatry, gastroenterology, and orphan diseases. The number of subjects involved in clinical trials had also been increasing.
Special Section: Clinical Research in Ukraine
Maintaining Safety and Continuity While Relocating Ukrainian Clinical Trial Participants
Agata Bloswick
Labcorp Drug Development, Poland
Bettina Ryll
Lindsay McNair
Equipoise Consulting
Stefan Strasser
Austrian Agency for Health and Food Safety
Serhiy Mykhaylov
Kateryna Orlovska

MSD, Ukraine
Anna Titkova
Pratia/Pratia Clinic, Ukraine

nsuring the safety of research participants during times of war is unique to the situation of each individual participant and must consider multiple aspects of each individual and the situation they are in.

A UNHRC agency estimated that 6 million Ukrainians left Ukraine and about 7.7 million were displaced internally (about a third of the population) by June of 2022.


Australian Clinical Trials Alliance Part 1 – Current Perceptions
“The pandemic had a substantial impact on community understanding of clinical trials. Having said that, the sort of ambient level of community knowledge of clinical trials is still substantially lower than it needs to be,” explains Australian Clinical Trials Alliance Board Chair Steve Webb. “Community awareness of the difference between evaluation of experimental interventions, about which relatively little is known regarding safety and effectiveness, compared with comparative effectiveness research, is an important distinction often with limited understanding in the community, in government, and amongst policymakers.”


Australian Clinical Trials Alliance Part 2 – Future Projections
“Healthcare systems obviously have as their primary role the delivery of healthcare,” says Steve Webb, Australian Clinical Trials Alliance board chair. “But we can see big improvements in health outcomes, combined with substantially better financial performance of the healthcare system, if the healthcare system has the dual role of treating patients as well as generating evidence about the most effective treatment options to guide that care.”
Around the Globe
Middle East Regulatory Landscape:
Collaboration, Agility, and Adaptation
Amira Younes
AbbVie, UAE

he COVID-19 pandemic highlighted the need for new ways of thinking about delivering regulatory effectiveness and efficiency, and for flexibility from all stakeholders to ensure that therapies continued to reach patients in a timely manner.

Most countries in the Middle East already had priority or expedited pathways prior to the pandemic. These pathways delivered shorter assessment timelines and demonstrated the usefulness of regulatory reliance during the global emergency. Jordan, Saudi Arabia, Bahrain, and Egypt created guidelines for Emergency Use Authorizations (EUA) for vaccine or therapeutic product use, as well.

Take This Survey: Your Chance to Contribute to Meaningful Change

IA’s Study Endpoints, Statistics & Data Science, and Clinical Research Communities have formed a Meaningful Change Working Group which aims to help sponsors calculate “meaningful change thresholds” for their clinical trial endpoints, to supplement traditional statistical tests and improve the interpretability of trial data.

Thanks for reading our April 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.