July 2024

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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 4Biosolutions Consulting

Patient engagement
Natasha Ratcliffe Patient Engagement Specialist
Thomas Smith Independent Patient Consultant

Data and Digital
Lisa Barbadora Barbadora Ink

VALUE AND ACCESS
Wyatt Gotbetter Parexel

Editorial Staff

Alberto Grignolo, Editor-in-Chief

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

AFRICA
David Mukanga Bill and Melinda Gates Foundation

ASEAN
Jin Shun Belief BioMed

AUSTRALIA/NEW ZEALAND
Richard Day University of New South Wales, Medicine, St. Vincent’s Hospital

CHINA
Ling Su Shenyang Pharmaceutical University, Lilly Asia Ventures

EUROPE
Emma Du Four Independent R&D/Regulatory Policy Professional

EUROPE
Isabelle Stoeckert Bayer AG Pharma

INDIA
J. Vijay Venkatraman Oviya MedSafe

JAPAN
Toshiyoshi Tominaga Keio University Hospital, Clinical and Translational Research Center

LATIN AMERICA
Cammilla Gomes Roche

US
Ebony Dashiell-Aje BioMarin

DIA Membership

Bringing together stakeholders for the betterment of global health care.

Editor-in-Chief Message
Divergent Trajectories
Alberto Grignolo
DIA Global Forum Editor-in-Chief
S

ince at least the birth of ICH in 1990, regulatory harmonization and trade globalization have followed parallel trajectories, synergizing successfully, exceeding expectations, and surprising those who may have initially been skeptical of its prospects. These trajectories are now diverging. This is not good news for patients.

Survey: Patient Attitudes Toward Participation and Use of Digital Technologies in Clinical Research
Melva T. Covington
AGAPE Strategic Solutions of Maryland, LLC
People Empowering People for Inclusion Now, LLC
Jeannine Cain, Tamara R. Allen
People Empowering People for Inclusion Now, LLC
Cheryl Beal Anderson
ACE Regulatory Affairs Consulting, LLC
T

here is no single solution to solve the challenge of recruiting and retaining participants in clinical research, and especially for those who have been underrepresented or historically excluded from participation. The need is great because such underrepresentation can affect drug utilization and patient experience in terms of product safety, efficacy, and quality. It can also materially impact the applicability of results to broader patient populations and the cost of research innovation. This challenge will not be effectively addressed or solved by incremental adjustments to recruitment approaches, particularly for individuals or groups most impacted by life-threatening health conditions in the real world. More diverse and inclusive representation would benefit everyone in the clinical research and product development process, including patients themselves.

Implementing Patient-Reported Outcomes in Cancer Clinical Trials

A Closer Look at FDA’s Draft Guidance and a Call to Action
Anthony T. Everhart
Signant Health
T

he absence of labeling related to Patient-Reported Outcome Measures (PROM) has been striking: Between 2010 and 2020, out of the 108 oncology drugs approved by the US Food and Drug Administration (FDA), only nine (8.3%) included PROM-related labeling. In June 2021, the FDA released a draft guidance document titled “Core Patient-Reported Outcomes in Cancer Clinical Trials.” This guidance aims to enhance the quality and consistency of data collection related to PROMs in cancer trials. However, despite its potential benefits, pharmaceutical companies have been slow to fully embrace these recommendations.

This article examines the challenges pharmaceutical companies face in implementing patient-reported outcomes (PROs) in cancer clinical trials as outlined in the FDA’s draft guidance. It highlights the value of PROs, outlines the key points from the FDA’s guidance, and proposes ways forward through collaboration to overcome the challenges and fully embrace the use of PROs in cancer trials.
Enabling Platform Trials with Master Protocols: How EU-PEARL and TransCelerate BioPharma Created a Global Template
Madhavi Gidh-Jain
Sanofi
Mary Lynn Mercado
Novartis
A

one-size-fits-all approach to biopharma R&D no longer fits the technology-savvy, diversified world of drug development. Limited resources, patient recruitment challenges, and other barriers have forced the biopharma R&D industry to explore alternative trial designs, which this article illustrates along with some recently developed tools to enable their implementation.

Talking Statistics: Why Clear Communications and Close Collaborations are Important in Cross-Functional Teams Engaged in Clinical Research
Stephen Corson
Phastar
C

lear, effective communication and close collaborations between statisticians and other functions at all stages of the clinical trial pipeline are key to ensuring that clinical trials are informative (regardless of the outcome). Unfortunately, clinical and statistical colleagues within the industry sometimes find this challenging. Statistical jargon often results in inefficient, confusing discussions between statisticians and the team at large, and study teams, particularly those from smaller organizations, often seek statistical support too late in the process. Both can compromise the study design and the data and their analysis. These challenges can lead to inefficiency and inconsistency in messaging to senior leaders who are keen to see how assets are progressing so that they can determine the next steps in terms of resourcing, prioritization, and regulatory discussions.

How Did We Get Here? A History of eCTD and Prospects for eCTD 4.0
Karl-Heinz Loebel
Cencora PharmaLex

Overview

T

he electronic Common Technical Document (eCTD) is 16 years old, approved by the International Council on Harmonisation (ICH) to Step 4 in 2008. It’s old enough to drive a car in the US. It’s had some small changes over the years but is still pretty much the same as it was in the George W. Bush era. It’s had a huge impact on the speed and accuracy of regulatory submissions for pharma … but it could be better. That same year, the US Food and Drug Administration (FDA) started looking at a next-generation submission format, which would become eCTD 4.0. That standard was approved to Step 4 at ICH in 2015, and nine years later, FDA is still not using it. Let’s look at how we got to this point, and what’s holding us back.

How to Communicate Unapproved Uses Without Breaking the Law
Darshan Kulkarni
Kulkarni Law Firm
M

edical product companies are entrusted to engage with healthcare providers and payers in a compliant manner. However, the emerging laws, the FDA’s evolving guidance landscape, and interpreting court decisions make compliance a complex process. This article discusses the new laws and FDA guidance documents that enable off-label communications with payers and providers and the differences between them. Specifically, focus will be placed on the Pre-Approval Information Exchange (PIE) Act, the Scientific Information on Unapproved Uses (SIUU) guidance, and the FDA’s guidance on medical product communications that are consistent with FDA-required labeling.

eBook

Embrace the Opportunities in a Changing FDA Advisory Committee Landscape cover
eBook
New eBook: Achieving Excellence in Regulatory Information Management
Members of the DIA Regulatory Affairs Community working in Regulatory Information Management (RIM) have continued to update the RIM consensus white paper to bring you a complete publication in the form of best practices across the spectrum of RIM capabilities and functional interfaces: Achieving Excellence in Regulatory Information Management.
Around the Globe
Making Global Reliance a Reality for Post-Approval Changes of Medicines and Vaccines
Susanne Ausborn
Roche
Andrew Deavin
GSK
W

hile it is widely recognized that Reliance should be a fundamental part of a modern 21st century regulatory framework to efficiently use resources and avoid duplication of efforts, many open questions on how to operationalize its use on a global scale still remain. This is especially relevant for handling Post-Approval Changes (PAC), which represent the majority of regulatory submissions. Considering this, senior regulatory experts representing Health Authorities from all around the world joined industry representatives at a DIA Europe 2024 Pre-Conference Workshop to discuss the practical aspects of applying reliance approaches to PACs more widely.

Around the Globe: Africa
The African Medicines Agency: Building a New Gold Standard in Regulatory Excellence
Insights from DIA Europe 2024
Sarah Adam
International Federation of Pharmaceutical Manufacturers and Associations (IFPMA)
T

he African Medicines Agency (AMA) is paving the way for Africa to achieve regulatory self-sufficiency and reliance. By aligning with global standards, this continent-wide regulatory ecosystem is expected to revolutionize patient care and access to medicinal products across Africa in new and unprecedented ways.

During DIA Europe 2024, the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) organized an Africa Townhall session in collaboration with the European Medicines Agency (EMA) which brought together various regulatory experts from the African Union Development Agency-NEPAD, the World Health Organization (WHO), the Bill and Melinda Gates Foundation, the Economic Community of West African States (ECOWAS), and industry. It revolved around the progress of different partners to enrich Africa’s regulatory ecosystem by implementing a robust and modern AMA.
Around the Globe: Australia
New TGA Head Welcomes Emerging Science and Therapies to Australia
I

n mid-2023, Professor Tony Lawler succeeded Professor John Skerrit as Deputy Secretary, Health Products Regulation Group (HPRG), Australian Government Department of Health and Aged Care, leaving his position of Chief Medical Officer of the State of Tasmania, Australia.

Tony’s responsibilities include the Therapeutic Goods Administration (TGA), the Office of Drug Control, the Office of the Gene Technology Regulator, and the Australian Industrial Chemicals Introduction Scheme.

Around the Globe: China
Evolving Policies and Use of RWE in Regulatory Decisions in China
Mingcan DU, Fengyun (Vicky) HAN
Johnson & Johnson Global Regulatory Policy and Intelligence
I

n recent years, real-world evidence (RWE) developed from real-world data (RWD) has become one of the hottest topics worldwide in the practice of drug and medical device registration and supervision. China’s regulatory authorities have also shown positive attitudes toward the role of RWE in drug R&D and registration review.

Around the Globe: Europe
Clinical Trials in Wartime: One Patient’s Journey to Survival
Anna Titkova
Kharkiv National Medical University
Pratia Clinic Ukraine
O

n February 24, 2022, many aspects of life in Ukraine were profoundly changed or altogether stopped. This included the clinical research industry: On this very day, the approximately 600 clinical trials in Ukraine were stopped. Subsequently, the State Expert Center of the Ministry of Health of Ukraine, the primary regulator of clinical research in the country, received 132 letters of premature clinical trial termination: 108 due to the invasion and 24 due to financial, safety, and efficacy issues. The State Expert Center also received 223 letters from sponsors and CROs regarding the transfer of clinical trial patients to other Ukrainian clinical trial sites or sites in other countries. Most of these were oncologic, neurologic, rheumatologic, gastroenterologic, or cardiologic patients.

Here is the story of one of those patients.

White Paper

Embrace the Opportunities in a Changing FDA Advisory Committee Landscape cover

White Paper

ProEd Regulatory, The HCG Regulatory Powerhouse, Healthcare Consultancy Group: Embrace the Opportunities in a Changing FDA Advisory Committee Landscape
Over the past four years, we’ve seen seismic shifts in the conduct of FDA Advisory Committee (AdCom) meetings. Changes already in place and those on the horizon are creating new challenges for sponsors. The biggest challenge has been adapting to the virtual format, which then morphed into a hybrid format, and is now transitioning back to in-person meetings. In addition, sponsors have had to deal with tighter timelines for point-counterpoint briefing documents and shared Q&A with the FDA. These challenges also create opportunities for a productive and transparent dialog with clinicians and regulators.
We Are DIA
DIA Congratulates New 2024 Fellows and Global Inspire Award Winners

New Fellows of DIA

Portrait headshot close-up photograph view of Cynthia Verst smiling
Cynthia Verst
(Chair)
Immediate Past Chair, DIA Board of Directors
President, R&DS Design & Delivery Innovation, IQVIA
Portrait headshot close-up photograph view of Antonio Barra Torres grinning
Antonio Barra Torres
President, ANVISA (Brazil)
Portrait headshot close-up photograph view of Peter Marks smiling
Peter Marks
Director, Center for Biologics Evaluation and Research (CBER), US FDA

DIA 2024 Global Inspire Award Winners

Global Antibiotic Research & Development Partnership (GARDP) logo
GLOBAL AWARD FOR OUTSTANDING CONTRIBUTION TO HEALTH
Global Antibiotic R&D Partnership (GARDP)
Portrait headshot close-up photograph view of Nimita Limaye smiling
GLOBAL CONNECTOR

Nimita Limaye
Research VP, Life Sciences R&D Strategy and Technology, IDC

Portrait headshot close-up photograph view of Shun Lu grinning
EXCELLENCE IN SERVICE

Shun Lu
Director, Center for Clinical Medicine of Lung Cancer
Shanghai Chest Hospital, Shanghai Jiao Tong University, China

Portrait headshot close-up photograph view of Kamila A. Novak smiling
COMMUNITY ENGAGEMENT

Kamila A. Novak
KAN Consulting

Thanks for reading our July 2024 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.