December 2021


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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 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.

Developing Digitally Derived Endpoints that Measure what Matters to Patients: The Case for Collaboration
Elektra J. Papadopoulos
Center for Drug Evaluation and Research (CDER)
US Food and Drug Administration
Sonya Eremenco
Critical Path Institute
Michelle Campbell
Center for Drug Evaluation and Research (CDER)
US Food and Drug Administration

n recent years, FDA has undertaken multiple efforts to better incorporate the patient’s voice in drug development and evaluation through adoption of clinical outcome assessments (COAs) that measure what matters to patients. Digital health technology (DHT) tools, such as sensor-based assessments of activity or sleep, hold potential to play a pivotal role in modernizing patient-centered outcome measurement by reducing barriers to trial participation and capturing outcomes that matter to patients in daily life. FDA regulations require well-defined and reliable assessment methods, a standard that applies to any outcome assessment intended to support claims of drug effectiveness. A COA is considered fit-for-purpose when the level of validation associated with it is sufficient to support its context of use.

Development of novel, fit-for-purpose digitally derived COA endpoints intended to support regulatory decisions is a complex and potentially lengthy undertaking necessitating a multistakeholder approach, which makes it difficult to achieve within individual sponsors’ drug development programs. We do not know of any regulatory precedent for US FDA labeling claims based on a novel digitally derived endpoint to date.

Special Section: Pharmacovigilance

Special Section: Pharmacovigilance

Special Section: Pharmacovigilance

Preamble and Introduction
Dr. J. Vijay Venkatraman standing behind a proctor and giving a speech

ear Global Forum Readers,

As a pharmacovigilance aficionado and an active contributor to a variety of DIA initiatives over the past 15 years, I consider it one of my greatest honors to have been invited to serve as the Editor for this Special Section of Global Forum on Pharmacovigilance. I take this opportunity to thank Dr. Alberto Grignolo, Global Forum Editor-in-Chief, and Chris Slawecki, Senior Digital Copy Editor, DIA Scientific Communications, for this invitation to contribute to this vital topic.

Special Section: Pharmacovigilance

scientist pointing to graphics of scientific icons
How Valuable is Patient Direct Reporting in Pharmacovigilance?
Paola Kruger

he past decade has seen the impressive growth of patient involvement in healthcare, to the point that the term “patient centricity” has been introduced steadily in all patient-related activities. This is particularly true in clinical research, where patients have gone from being just passive recruited subjects to active participants thanks to the realization that if something is meant to benefit the patient, then the patient must be included in the process and his/her voice must be as heard and relevant as other stakeholder voices to develop better quality medicines that truly improve their lives. The importance and impact of the patient perspective in drug safety reporting is now emerging.

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Special Section: Pharmacovigilance

scientist pointing to graphics of scientific icons
Smart Safety Surveillance (3S): Proven Approach to Building Functional LMIC Pharmacovigilance Systems
Shyam Bhaskaran, Charles Preston, Raj Long
Bill & Melinda Gates Foundation

mart Safety Surveillance (3S) is a product-focused, principles-based approach to building functional LMIC (low- and middle-income countries) pharmacovigilance (PV) systems that has been validated across multiple products and geographies. The lessons learned and key success factors enabling this work have been shared with interested country regulators and public health decision makers tasked with protecting the safety of their populations, funders, and multilateral organizations partnering with these countries, and to product developers developing global product launch plans.


Three Good Reasons to Use Artificial Intelligence in Pharmacovigilance
“There are good reasons to explore the use of artificial intelligence in pharmacovigilance. There are an increasing number of sources of drug safety information, and efficient screening, processing, or evaluation of them would benefit from accurate automated methods,” explains Gerald Dal Pan, Director, Office of Surveillance and Epidemiology (OSE), CDER. “First, there’s the potential application of AI to the processing of case reports. Second, there’s the potential application of AI for assessing causality in a case report. Third, we’re exploring the use of AI in real-world data as part of the Sentinel Initiative.”

Special Section: Pharmacovigilance

scientist pointing to graphics of scientific icons
Advanced Analytics Drives Innovation in Pharmacovigilance Risk Management Systems
Francois Audibert

Mariette Boerstoel

Courtney Granville

Jeremy Jokinen
Bristol Myers Squibb Company


rapid acceleration of automation sees artificial intelligence (AI) driving efficiency across the safety continuum. Although cost, unfamiliarity, and hesitancy to explore new ways of working were (and to some extent remain) barriers to rapid integration of AI, the demands of increasing case volumes and the consequent need to reduce human workload are shifting priorities and may promote further adoption of AI. This comes with many benefits, including reduction in errors and overreporting, and improvement in timelines and transparency. It is also enabling a shift in workforce needs and new ways of working. Finally, evidence generation in the COVID era has shifted mindsets and could stimulate a future in which post-approval safety assessment shifts from exploratory to confirmatory.

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Special Section: Pharmacovigilance

scientist pointing to graphics of scientific icons
Using Community Reporters as a Strategic Source for Evaluating Vaccine Effectiveness
Nancy A. Dreyer
Matthew W. Reynolds

IQVIA Real World Solutions

OVID-19 has highlighted the need for new methods of pharmacovigilance. During the pandemic, people stayed home as much as possible, avoiding public gatherings, delaying going to the doctor for routine medical care, and even holding off seeking medical care for COVID symptoms. This situation was further complicated by social media reports encouraging use of medications outside of their labeled indications, as well as anxiety about using vaccines that were developed quickly and first authorized under emergency use authorization.

Special Section: Pharmacovigilance

scientist pointing to graphics of scientific icons
Monitoring Safety Signals for Vaccines: Current Strategies and Future Directions
Kausik Maiti
Neeraj Parmar

he role of vaccines in preventing infectious diseases globally has become more important than ever in the COVID-19 pandemic. Safety has always been a key focus in vaccine development, but vaccines (like all medical products) are not without risks of adverse effects. Along with meticulously conducted clinical trials, a robust post-market safety surveillance system is essential to ensure safety throughout the vaccine lifecycle. Efficient and scientifically sound signal detection systems and responsible communication also play a significant role in vaccine safety.

Special Section: Pharmacovigilance
scientist pointing to graphics of scientific icons
Six Key Considerations to Scale Pharmacovigilance with Modern Technologies
Saba Darvesh
Veeva Systems

ore than 16 million adverse events have been reported to the FDA over the last 10 years, nearly four times the volume from the prior decade. This growth is driving industry-wide action to improve intake and case processing. Many companies are scaling operations and increasing efficiency with modern tools for more strategic product development. This presents both opportunities and challenges and requires fresh thinking and more nimble processes.

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Around the Globe ASEAN

Key Features of Orphan Drug Registration in Singapore, Malaysia, Philippines, and Vietnam
Agnes Ho
Cindy Zen
Tan Kwee Lan
Boehringer Ingelheim

egulatory authorities such as US FDA, EMA, and PMDA have had regulatory frameworks for orphan drugs (OD) in place for decades. Upon recognizing that their existing drug development and regulatory frameworks may not be suitable for rare disease (RD) therapeutic interventions, regulators in Singapore, Malaysia, Philippines, and Vietnam have also started to initiate regulatory reforms for OD registrations. This table illustrates the current state of these reforms.

Around the Globe CANADA

Funding Established for Canadian Drug Agency Transition Office
Judith Glennie
JL Glennie Consulting Inc.

he September 2021 federal election represented a second trip to the polls for Canadians in the last two years, resulting in another minority government for the incumbent Liberal Party. From a pharmaceutical policy perspective, the party platform mirrored the federal budget tabled in April 2021 and affirmed previous direction on files related to this sector. In the context of this renewed mandate, it is worth reviewing recent developments in the Canadian pharmaceutical policy space and how they might move forward in the future.

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Around the Globe EUROPE

EUnetHTA 21 Launches Joint Scientific Consultations for Regulators, HTAs, and Sponsors
Thomas Kühler
Sanofi R&D

lobal Forum recently published a piece on Health Technology Assessment (HTA) in the EU and how a regulation that had been the subject of protracted negotiations for close to three years eventually seemed to have been cleared for final adoption. The aim of this regulation is to secure timely patient access to novel therapies, to avoid duplicative HTA-assessment work at the Member State level, and to provide the sector with a predictable and consistent HTA process. A few formal steps remained, the most important one being the adoption of the regulation at a Council Meeting, which happened in a Council vote on November 9, 2021.

We Are DIA
In Memoriam: Dr. J. Rick Turner: “A regulatory scientist of the highest order”
Dr. J. Rick Turner Headshot

IA honors the life and work of J. Rick Turner, expert consultant at DRT Strategies Inc. and editor emeritus of Therapeutic Innovation & Regulatory Science (TIRS), who died in October 2021. He was a leader, friend, and mentor whose energy, enthusiasm, and inspirational nature will be missed by many.

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Thanks for reading our December 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.