September 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

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.

Emergency Use Authorization to Full Approval–Charting a Course in Unexplored Territory
Part 1: Pathway for a Novel Therapeutic
J. Shawn Roach
Halloran Consulting Group

he COVID-19 pandemic has put the FDA guidance on Emergency Use Authorization to a new test. Both sponsor companies developing new therapeutics and the FDA itself have had to find new and innovative ways to rapidly make therapeutics, vaccines, diagnostics, and devices available to combat the public health emergency. Even though speed has been of the essence, no sacrifices were made to maintain solid, data-based standards to ensure both the safety and efficacy of these novel treatments. This two-part series describes how therapeutics (drugs or biologics) have navigated this novel path to bring aid to respond to the current public health crisis.

Implication of Cancer Growth Kinetics for the Further Development of Antibody-Drug Conjugates
Joshua Z. Drago
Larry Norton
Memorial Sloan Kettering, New York

compelling but underappreciated hallmark of cancer is its pattern of growth. Cancers do not grow exponentially as they would were growth cellular autonomous. Rather, their growth follows sigmoid (“S” shaped) curves. As described in our article published in the August 2021 issue of Global Forum, this fact has been shown to be helpful in the design of effective anticancer chemotherapy regimens, as illustrated by the treatment of primary breast cancer. But what is the etiology of sigmoid growth? And can explorations of this question be used to improve the application of novel anticancer therapeutics, especially the antibody-drug conjugates (ADCs), which differ mechanistically from chemotherapy in several key respects?

White Paper

Medrio White Paper

White Paper

The Rise of Hybrid Trials
Although decentralized trials (DCT) existed prior to the pandemic, COVID-19 undoubtedly had a large impact on the rapid adoption of DCT technologies. As global regulators become more accepting of these novel technologies, patients become accustomed to using them, and DCT enters the mainstream lexicon, it’s important to distinguish how and where it will benefit clinical research the most.

This paper defines what a hybrid trial is, how it utilizes decentralized technologies, and the reason for mainstream adoption of hybrid clinical trials.

C.O.V.I.D.: Five Steps Toward Patient Engagement During and After the Pandemic
Trishna Bharadia
The Spark Global

atient engagement can come in different forms and serve different purposes. The COVID-19 pandemic has highlighted just how important it is to bring patients into the medicines development lifecycle, not only as clinical trial participants but as active collaborators in how the lifecycle is shaped. While many factors for effective engagement are common both during normal times and during a pandemic, the pandemic has brought to light some specifics. This article offers practical advice and tips from the perspective of patients as to how stakeholders involved in clinical research, specifically the pharmaceutical industry, academic institutions, and public bodies, like the National Institute for Health Research (NIHR) in the United Kingdom, can continue to embed the patient voice in the medicines development lifecycle despite the challenges posed by the pandemic.

Public Consultation: New Guidance for Smarter Randomized Controlled Trials
Aisha Mazhar
Nick Medhurst
Martin Landray
Good Clinical Trials Collaborative

ood randomized controlled trials (RCTs) play a central role in generating the evidence needed to inform the development and implementation of health interventions. However, useful evidence from good RCTs is often lacking. This can be because the RCTs were never done, because those that were done failed to produce scientifically robust and clinically relevant answers, or because the results were never published.

The pandemic has particularly highlighted the need to be better at generating good-quality evidence more quickly. A prominent study, Trends in COVID-19 therapeutic clinical trials, estimated that only 5% of clinical trials for COVID-19 therapies were designed to be capable of producing robust results. It demonstrates the wasted effort and missed opportunity, resulting in delays or outright failure to identify effective interventions.

The Good Clinical Trials Collaborative (GCTC) has developed a new guidance to promote and enable good RCTs. The inclusive and broad-based guidance, which is available in five languages, will help researchers make RCTs smarter and better inform healthcare strategies. The collaborative is currently seeking input to shape the guidance through a public consultation until September 30. We invite you to get involved and share this opportunity with any organizations or individuals with an interest in RCTs.

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Commentary: The Impact of FDA’s Draft Guidance for Oncology Patient-Reported Outcomes on Future Trial Design
Bill Byrom
Jill Platko
Anthony Everhart
Signant Health

n recent years, we have seen enhanced emphasis on the importance of patient-reported outcome measures (PROM) collected in oncology clinical trials reflected by the FDA. Project Patient Voice, for example, was established to provide a mechanism to share patient-reported symptom data from cancer clinical trials with approved treatments to provide more information to patients and healthcare providers in treatment decision making.

Decentralized Clinical Trials Across Europe: Regulatory and Other Practical Considerations
Maya Zlatanova
FindMeCure Ltd.

ecentralized Clinical Trials (DCTs) can be an accelerator to increase patient diversity, recruitment, and engagement. The COVID-19 pandemic made decentralization not only a preference but also a necessity for many clinical trials globally whose operations were disrupted due to the complicated healthcare and logistical situation the pandemic caused.

On February 4, 2021, the European Commission published the updated, fourth version of the guideline on the management of clinical trials during the COVID-19 pandemic, i.e., the “Guidance on the Management of Clinical Trials during the COVID-19 (Coronavirus) Pandemic.” Although it is clear that these are temporary recommendations to manage clinical trials while the public health emergency lasts globally (e.g., implementing home health visits, shipping investigational medicinal products [IMP] directly to participants, and conducting remote Source Data Verification [rSDV]), these recommendations are key components deployed in DCTs which make for their global adoption under the current conditions and also in the future after the COVID-19 pandemic is resolved.
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Meeting Highlights: DIA Global Annual Meeting 2021

Meeting Highlights from DIA Global Annual Meeting 2021

Meeting Highlights: DIA Global Annual Meeting 2021

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A Year Like No Other: COVID-19 and the US FDA’s Response
Fintan R. Steele

he declaration of a Public Health Emergency on January 31, 2020, by the US Department of Health and Human Services turned out to be only the first small ripple in what would become a global COVID-19 tsunami over the next 18 months. Since then, the US Food and Drug Administration (FDA) has played a central role in the US government’s frontline defense against SARS-CoV-2 and its variants. It has also been challenged in ways never anticipated throughout what Acting Commissioner Janet Woodcock called “a year like no other.”

In June 2021, CBER Director Peter Marks and CDER Director Patrizia Cavazzoni joined Woodcock for the DIA 2021 Virtual Global Annual Meeting FDA Town Hall discussion to look back at whether and how the FDA, as a public health agency at its core, has risen to the many COVID-related challenges it faced. They also looked ahead, not just at the continued pandemic but also at how some learnings from the COVID-19 crisis can and should be applied to other diseases and areas for which FDA is responsible.

Key Takeaways

  • Through challenges of the pandemic, several new approaches emerged that likely should be sustained when the current public health crisis finally abates.
  • FDA’s final guidance released in May 2021 on the design and implementation of COVID-19 master protocols can help get clinical research “out of the ivory tower and into communities,” making clinical trial participation a regular part of medical care.
  • As the complexity and amount of data increase, the Technology Modernization Action Plan (TMAP) set out in late 2019 will help ensure that the FDA has proper and secure tools for data storage, access, and interoperability, as well as a central organization and platform structure to support them.

Meeting Highlights: DIA Global Annual Meeting 2021

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When Can You Trust Real-World Evidence for Decision-Making?
Chris M. Slawecki

ne of the unexpected impacts of the social isolation and distancing policies mandated during the COVID-19 pandemic has been growing interest in the use of real-world evidence (RWE), as an alternative or a complement to evidence provided by traditional randomized clinical trials (RCTs), for regulatory review and approval of therapeutic products.

But RWE, generated from real-world data (RWD), was contributing to many of these decisions long before COVID-19. RWE has played and continues to play a large role in the continuing research and development of vaccines and treatments to fight COVID-19. And other research strongly suggests that RWE will remain part of regulatory review and approval, especially but not exclusively for rare diseases/orphan products, long after the pandemic subsides.
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Around the Globe

Joint Assessment of Marketing Authorization Applications:
Cooperation Among ASEAN Drug Regulatory Authorities
Rosilawati Ahmad
National Pharmaceutical Regulatory Agency, Malaysia
Tharnkamol Chanprapaph
Food and Drug Administration, Thailand
Samvel Azatyan
World Health Organization
Valerio Reggi
World Health Organization
Prapassorn Thanaphollert
World Health Organization

he first steps toward harmonization of ASEAN pharmaceutical regulations started in 1992 through the establishment of the ASEAN Consultative Committee for Standards and Quality (ACCSQ). In 1999, the ACCSQ established a Pharmaceutical Product Working Group (PPWG).

The objective of the PPWG is to develop harmonization of pharmaceutical regulations across ASEAN member countries to complement and facilitate the ASEAN Free Trade Area (AFTA), particularly the elimination of technical barriers to trade posed by these regulations, without compromising on drug quality, safety, and efficacy.


Australia: Early Access Programs Complicating Comparative Data Analyses
The Pharmaceutical Benefits Advisory Committee (PBAC) is an independent expert body appointed by the Australian Government to recommend new medicines for listing on the Pharmaceutical Benefits Scheme (PBS). How have advanced therapies impacted the cost and cost effectiveness of pharmaceuticals, and the work of PBAC, in Australia? “Our decision making is becoming harder because we’re being asked to make decisions with substantially more uncertainty about the benefits and safety and the value propositions because medicines are coming to market earlier,” explains PBAC Chair Andrew Wilson to Ric Day, Global Forum Regional Editor, Australia/New Zealand. “I don’t think the quality of the information has fallen. It’s just that we’re seeing it earlier in the drug development program process to be considered, and so there is more uncertainty associated with it.”

Around the Globe

Concrete Steps Toward National Pharmacare in Canada
Judith Glennie
JL Glennie Consulting Inc.

reation of a National Pharmacare program has been a focus of the Federal Liberal government since their election in 2015. Various committees, reports, and government budget announcements have reiterated a commitment to National Pharmacare, although a specific path forward had yet to be articulated—until recently.

Over the past few months, the government has started to advance some of the recommendations in the final report of the Advisory Council on the Implementation of National Pharmacare. (See October 2019 Global Forum.) Movement has occurred on the proposed Canadian Drug Agency, the national strategy for drugs for rare diseases, and the creation of an essential medicines list, as key developments on the path toward National Pharmacare in Canada.
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Around the Globe

Regulatory Agilities During the COVID-19 Pandemic:
Observations from a Multinational Pharmaceutical Company
Julie O’Brien
Rebecca Lumsden

uch has been written about the experience and learnings from a regulatory point of view during the COVID-19 pandemic. Large multinational innovative pharmaceutical companies developing COVID-19 vaccines and therapeutics, and supplying critical medicines, are experiencing this reality daily. The following practical actions were deployed by regulatory authorities and collectively experienced by industry to ensure the continued smooth functioning of regulatory systems during the pandemic. We propose that there is an imperative to continue to build on many of these actions to accelerate regulatory strengthening even further in international markets and to make some of these practices permanent.

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Thanks for reading our September 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.