October 2021


Love Global Forum’s new online format? Subscribe today and never miss an issue.

Editorial Board

Content stream editors

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

regulatory science
Isaac Rodriguez-Chavez ICON plc

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

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.

Tackling Prostate Cancer with Increasing Precision
William G. Nelson
Johns Hopkins Kimmel Comprehensive Cancer Center

uring his long tenure as the head of urology at what is now the Memorial Sloan-Kettering Cancer Center, Willet F. Whitmore presciently asked whether a cure for prostate cancer was necessary for those for whom it was possible, and whether a cure was possible in those for whom it was necessary. Decades later, ongoing improvements in prostate cancer screening, detection, diagnosis, and treatment appear finally on the cusp of delivering precision medicine answers to the Whitmore query, more and more often steering the right men to the right treatment at the right time.

The introduction of serum prostate-specific antigen (PSA) testing in the 1980s revolutionized the care of prostate diseases, including prostate cancer. Still the most widely used tumor biomarker, PSA was rapidly adopted for prostate cancer screening and risk stratification, for assessing response to treatment, and for monitoring disease recurrence and progression. Now, a growing portfolio of new molecular biomarkers and imaging technologies have begun to impact the routine care of men with prostate cancer. From prostate cancer screening and early detection to treatment of life-threatening disease, these new tools increasingly stratify men into subsets that drive clinical decision-making, underpinning precision medicine approaches to prostate cancer care.

Globalizing the Pharmacovigilance System Master File: Challenges and Opportunities
Clare Lavery
AstraZeneca Limited
Joanne Emmott
Merck Sharpe & Dohme (UK) Limited
Sabine Jeck-Thole
Boehringer Ingelheim GmbH
Pascale Rouben
Bristol Myers Squibb International Corporation, Belgium
Clare Lavery
AstraZeneca Limited
Joanne Emmott
Merck Sharpe & Dohme (UK) Limited
Sabine Jeck-Thole
Boehringer Ingelheim GmbH
Pascale Rouben
Bristol Myers Squibb International Corporation, Belgium
Dionne Usher
Merck Sharpe & Dohme (UK) Limited
Willemijn van der Spuij
Bristol Myers Squibb SA, Switzerland
Louise Woodward
Roche Products Ltd, UK

he requirement for a Pharmacovigilance System Master File (PSMF) was introduced in the European Union (EU) legislation in 2010. The PSMF provides the description of the European Marketing Authorisation Holder’s pharmacovigilance (PV) system, and according to the legislation, supports their compliance with the legislative requirements, e.g., by fulfilment of QPPV supervision and verification by national regulatory agencies (NRA). The Implementing Regulation, and in 2012 the EU Good Pharmacovigilance Practices (GVP) Module II guidance, provides a framework allowing a flexible accommodation of the different organizational structures of marketing authorization holders (MAHs). Since 2012, the concept of the PSMF has evolved in countries/territories outside of the EU, creating challenges for industry related to maintenance and production.

Protecting the Integrity of Online Patient Engagement Surveys
Julia B. Kim
National Minority Quality Forum
George Washington University School of Public Health

urveys are an important tool for gathering diverse patient insights and engaging patients in healthcare decisions. With the ubiquitous use of the internet, there are greater opportunities for patients to engage with researchers, but also greater vulnerability for scammers to interfere with response collection and results. This article describes the importance of protecting survey instruments as a tool for patient engagement along with best practices for reducing vulnerability to fraudulent online activity.

MasterControl webinar Ad
Digital Biomarkers to Improve Quality of Life in Cancer Patients
Moonshot-Funded Decentralized Clinical Research Aims to Better Capture Effects of Cancer Therapy
Ingrid Oakley-Girvan
Reem Yunis

he National Cancer Institute’s (NCI) Cancer Moonshot program was launched five years ago with the goal of generating new cancer research via an additional $1.8 billion investment into the 21st Century Cures Act. The main goals of the program are “to accelerate scientific discovery in cancer, foster greater collaboration, and improve the sharing of data.” To date, 240 research projects across more than 70 initiatives have been supported by Moonshot funding, with more to come in the next seven years. One such project is Digital Biomarkers for Clinical Impact (DigiBioMarC). This study aims to understand and improve the quality of life for patients receiving cancer therapy, which is critical, because many patients undergoing treatment for serious conditions like cancer often struggle to tolerate their aggressive treatments.

Emergency Use Authorization to Full Approval—Charting a Course in Unexplored Territory
Part 2: Demystifying the 505(b)(2) Route for Drug Approval
David L. Rosen
Foley & Lardner LLP
J. Shawn Roach
Halloran Consulting Group

n Part 1 of this two-part series on “Emergency Use Authorization to Full Approval—Charting a Course in Unexplored Territory,” we outlined the pathway to bring a novel therapeutic from Emergency Use Authorization (EUA) to Full Approval. In Part 2, we demystify the 505(b)(2) route for drug approvals and provide key considerations if your organization is considering this pathway to develop a therapeutic to respond to the current public health crisis.

Global Forum for Qualified Persons for Pharmacovigilance (QPPV) Ad

Around the Globe

AVAREF Workshops Accelerating COVID-19 Vaccine Access in Africa
Africa Medicines Agency (AMA) Treaty Update
by David Mukanga
Bill and Melinda Gates Foundation

s part of the African Union guidance on emergency regulatory authorization of COVID vaccines, the Africa Vaccine Regulatory Forum (AVAREF) convened several workshops in 2021 with Africa member states (see below). These workshops were intended to enable regulators to rely on the World Health Organization (WHO) Emergency Use Listing (EUL) without having to undertake their own reviews, and in turn accelerate national emergency use authorization and access to these vaccines.

The regulators accessed the full dossiers and WHO Prequalification (PQ) assessment reports for these vaccines through a WHO portal, after signing a confidentiality agreement. The workshops were facilitated by members of the WHO PQ team followed by extensive Q&A sessions. The facilitated workshops covered six vaccines produced by AstraZeneca-Serum Institute of India and other organizations.

Around the Globe

Changing Landscape of Evidence Charting New Assessment Pathways
Q&A with Canadian Agency for Drugs and Technologies in Health (CADTH) CEO Suzanne McGurn

ealth technology assessment (HTA) is the evaluation of the clinical, economic, and humanistic value of a new product compared to existing approaches to care. In Canada, as in many other jurisdictions (such as the UK, Australia, France, and Germany), health technology reviews are used to support decisions by provincial payers about drug funding and appropriate use.

Judith Glennie, Global Forum regional editor for Canada, discusses the concept of HTA and its place in the therapeutic development of pipeline products—first on a global basis and then within the context of Canadian and other international jurisdictions—with Suzanne McGurn, president and chief executive officer of the Canadian Agency for Drugs and Technologies in Health (CADTH), in the below Q&A. CADTH is an independent not-for-profit organization responsible for providing Canada’s healthcare decision makers with objective evidence about the optimal use of drugs and medical devices, where Suzanne has served as president and CEO since July 2020.
Suzanne McGurn headshot

Suzanne McGurn
Canadian Agency for Drugs and Technologies in Health (CADTH)

Privacy Analytics Ad


Telehealth Emerging in Japan’s Clinical Research and Care

E. Stewart Geary

How are clinical research and clinical care industries in Japan responding to the new challenges emerging in the post-pandemic world? “During 2020, it became a very difficult time to initiate a clinical trial, a difficult time to recruit patients to clinical trials which were ongoing, especially if those were clinical trials in patients who were elderly or in cancer therapies. Patients were a little bit reluctant to go to hospitals, even though we had very few cases in Japan,” explains E. Stewart Geary, global safety officer and senior vice president, Eisai Company Limited. “The healthcare system in Japan has one big challenge in that a lot of people are concentrated in large cities, and that’s also where healthcare professionals are concentrated. But you have a large elderly population which is still in the countryside in towns which are kind of dying off, are very small, and are probably underserved by healthcare professionals. Because of that, there had been ongoing debate about telehealth.”

Around the Globe

Regulatory Utilization of Real-World Data and Real-World Evidence in Japan
Pharmaceuticals and Medical Devices Agency (PMDA) Chief Executive Perspective
Yasuhiro Fujiwara
PMDA Chief Executive

eal-world data (RWD) and real-world evidence (RWE) have been actively discussed worldwide in terms of utilization for regulatory decision-making on the benefit-risk assessment of drugs. In Japan, the Ministry of Health, Labour and Welfare (MHLW) and the Pharmaceuticals and Medical Devices Agency (PMDA) have worked to promote the utilization of RWD and RWE throughout a medical product’s lifecycle, from pre-approval through development to the post-marketing phase.

In clinical trials, randomization is critical because it ensures comparability between groups and thereby minimizes potential bias in the allocation of test treatments. For this reason, a pivotal study supporting the efficacy and safety for marketing approval should be conducted in a randomized controlled manner, as long as such a study is feasible for the target disease or investigational intervention, as the basic foundation of generating quality evidence.
Yashuhiro Fujiwara against a blue background
PMDA Chief Executive
Yasuhiro Fujiwara
18th DIA Japan Annual Meeting 2021 Ad


Aligned Review Timelines Key to Co-Developing Companion Diagnostics
Megan Doyle and Tiffany Levin
“We’re moving toward an era of truly personalized medicine where research is uncovering the genetic basis for disease as well as mutations and biomarkers that can be targeted with drug and biologic therapies. We need to continue to identify the right patients for these innovative therapies, and this is where companion diagnostics has a foothold,” explains Tiffany Levin (55th Parallel). “One of the key challenges for drug sponsors is the timing of the development and review of companion diagnostics when they’re intended to accompany an accelerated therapeutic development program,” continues Megan Doyle (Amgen). “We know regulators have developed or are developing accelerated regulatory pathways for drugs that are intended to address areas of high unmet need. But we don’t have a similar accelerated pathway in most jurisdictions for the companion diagnostic.”
Real-World Evidence Conference Ad
Thanks for reading our October 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.