February 2019

February 2019


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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
Yoshiaki Uyama Pharmaceuticals and Medical Devices Agency (PMDA)
Adora Ndu BioMarin Pharmaceutical, Inc.

Patient engagement
Deborah Collyar Patient Advocates In Research (PAIR)
Lode Dewulf Servier

Value & access
Edith Frénoy European Federation of Pharmaceutical Industries and Associations (EFPIA)

Editorial Staff

Alberto Grignolo, Editor-in-Chief PAREXEL International

Ranjini Prithviraj, Global Associate Director, Content Collaboration DIA Publications

Sandra Blumenrath, Science Writer DIA Publications

Chris M. Slawecki, Senior Digital Copyeditor DIA Publications

Regional Editors

David Mukanga Bill and Melinda Gates Foundation

Silke Vogel Duke-National University of Singapore Medical School

Richard Day University of New South Wales, Medicine, St. Vincent’s Hospital

Judith Glennie JL Glennie Consulting, Inc.
Megan Bettle Health Canada

Ling Su Shenyang Pharmaceutical University, Lilly Asia Ventures

J. Vijay Venkatraman Oviya MedSafe

Kazuhiro Kanmuri Inter-Professional, Inc.

Ebony Dashiell-Aje FDA

Young Professionals Editor

Kaley Weintraub Rutgers Post-Doctoral Fellow, Acorda Therapeutics, Inc.

DIA Membership

Bringing together stakeholders for the betterment of global health care.

Minimal Residual Disease in Leukemia:
The Case for Drug Development Tools
J. Milburn Jessup
Scientific Director
Precision Cancer Care Program

ecent history has shown that pathologic complete response (pCR) in breast cancer is a critical drug development tool (DDT). Particularly, the I-SPY 2 trial has supported development of agents for FDA accelerated approval based on pCR data obtained in the neoadjuvant setting in breast cancer. Leukemias have similar characteristics in that tumor burden can be measured directly in the bone marrow as well as in the blood after treatment. As discussed below, the key concept of Minimal Residual Disease (MRD) is that there is a threshold of disease below which the likelihood of relapse is low. It is somewhat easier to identify leukemia cells than to measure pCR, because usually (not always) the leukemic cells express antigens that are distinct or in a pathological profile that distinguishes leukemic cells from normal cells.

How Continuous Publishing Speeds Regulatory Submissions
John Lawrie
Vice President, Vault RIM
Veeva Systems

cross the life sciences industry, companies are increasingly focused on how they can improve the speed and efficiency of their operations. This is especially true now with greater attention being paid to drug prices. From the clinical through the commercial phases, companies are seeking unique ways to reduce costs to maintain their margins; regulatory operations is no exception.

One new approach being explored is “continuous publishing,” which reduces steps in a traditionally lengthy process. Continuous publishing replaces serial publishing with a parallel process where regulatory teams identify issues in real time rather than waiting until the end. This allows regulatory teams to resolve validation errors faster and eliminate cumbersome manual hand-offs to significantly reduce time from planning to submission.


Separate But Equal: Regulatory & Pricing Assessments in the EU

Around The Globe

A National, Personally Controlled Health Record
Australia Getting Privacy Right?

Richard Day
Professor of Clinical Pharmacology
University of New South Wales


personally controlled health record that summarizes an individual’s health issues, medical interventions, investigations, medicines, allergies and adverse reactions and is legitimately accessible to healthcare providers anywhere has immense appeal.

Such a record is highly likely to reduce the duplication of already prescribed medicines, prescriptions written despite previous allergic or adverse reactions, duplication of costly and sometimes risky investigations, and the significant time spent in attempting to access these data by practitioners as their patients navigate through various healthcare “silos.”

Around The Globe

Drugs for Rare Diseases Receive Overdue Policy Attention in Canada

Judith Glennie
JL Glennie Consulting, Inc.

espite significant efforts over the past decade–and the fact that the world’s major regulatory bodies long ago developed tailored regulatory frameworks for orphan and/or drugs for rare diseases, or DRDs–Canada remains one of the few developed countries without a specific orphan drug regulatory framework.

Around The Globe

China Enters 2019:
Encouraging Innovation, Broadening Access
to Medicines

Minhua Chu
VP, Research Intelligence

018 marks a banner year of marketing approvals of new drugs and implementation of important regulatory reform measures in China. On the other hand, the vaccine scandal sounded a significant alert that a stringent regulatory system is very much needed to safeguard the quality of medicinal products and ensure patient safety. Reform of the Drug Administration Law and pricing/reimbursement pressures will drive more changes in 2019 to benefit patients as well as innovative pharmaceutical companies.


Promoting Patient-Centric Business: Medical Communication in Latin America

White Paper

RIM Framework Interim Consensus White Paper
Sheila Mahoney-Jewels
Joel Finkle
Director, Regulatory Innovation & IDMP Strategy
V. “Bala” Balasubramanian
Cabeus, Inc.
Cary Smithson
Director, Digital Transformation & Management
Grant Thornton, LLP
Vahe Ghahraman
Senior Director, Global Regulatory Operations
Alexion Pharmaceuticals, Inc.
Bernie Coney
Head, Assay Content Development & Product Labeling
Siemens Healthineers

DIA Regulatory Affairs Community Regulatory Information Management (RIM) Working Group


ife Sciences organizations are increasingly facing several business challenges: an expanding global footprint, evolving regulatory requirements, dynamic partnership models, and rapidly evolving technology. Many organizations are realizing the importance of regulatory information as a corporate asset and hence improving and innovating how regulatory information is captured and utilized across the regulatory and pharmaceutical value chains, commonly referred as regulatory information management (RIM). Organizations, both sponsors and the vendors that serve them, have come up with organic solutions that are fit for specific purposes; however, the resulting systems are often not harmonized in approach or terminology which compounds the complexity associated with ownership, flow of data across the enterprise, interoperability, quality responsibility, and governance of regulatory data, or any form of collaboration/integration.

The Regulatory Information Management Working Group (RIMWG) of DIA’s Regulatory Affairs Community has identified potential benefits that could be captured from the development of a conceptual framework for Regulatory Information Management (RIM). Such a framework would aid organizations in structuring the complex organizational, informational, and technological issues germane to RIM’s myriad of challenges related to cross-functional global data identification, ownership, quality management, and maintenance. The ultimate goal is to enable RIM as a strategic corporate asset that will have direct impact on a sponsor’s profitable operations and, ultimately, on expediting delivery of needed treatments to patients.

Over a two-year period, the RIMWG has authored an Interim Consensus White Paper describing the evolution of the RIMWG’s discussions, debates, feedback, and decisions made by various global RIM stakeholders during framework development. The framework itself comprises the identification of RIM capabilities according to common themes, or “buckets”; an “RIM Elements” catalog (in process); and a data map that identifies data elements and determines how data flows through the organization and related systems (also in process). The key objective of this initiative is to define an “Ideal RIM State” along with a RIM Reference Model which collects process, data, and organizational constructs that both sponsors and vendors could use as a benchmark to compare their respective capabilities and offerings. Together, the collection of data and process flow recommends not a binding regulation, but rather a reference-able framework for that “Ideal RIM State” upon which organizations can begin to model and benchmark against. The intention here is to create a framework which could benefit sponsor organizations, as well as technology and service providers, in their effort to improve how we manage regulatory information as a corporate asset.

While RIM has broader reach and scope, this interim consensus white paper starts by addressing key RIM components that are traditionally understood as “in-scope” for RIM. Many topics such as correspondence and commitments, labeling, and advertising and promotion, are not included in this version of the white paper. However, as the paper evolves, we will be adding more comprehensive definitions and other components. We expect this to be a “living document” that will continue to live until it covers all components included within the scope of RIM. We welcome broader industry participation and the expertise of individuals willing to join our efforts as volunteers to further the cause of this working group and contribute to the creation of artifacts to benefit industry.

Key stakeholders from this RIMWG will be conducting a round table at the Regulatory Submissions, Information, and Document Management (RSIDM) Forum from 7:30 – 8:30AM on February 12 in Bethesda, Maryland. We welcome participation from the regulatory community at large: Join us at RSIDM and make your voice heard by providing input into the RIM Elements Catalog, data map, and the evolving white paper.


HSA Harmonizing Culture and Science in Singapore

Young Professionals’ Corner

Lead Out Loud: The Art of Public Speaking

Lyndsay Golden
Rutgers Post-Doctoral Fellow
Medical Affairs


our heart is pounding at what feels like 100 beats per minute. Your palms are sweaty, and your anxious nerves start to kick in. Immediately before you stand in front of an audience, whether it is five or five hundred people, your heart sinks into your stomach.

These are common feelings many people experience moments before delivering a speech or presentation, and I can certainly relate. Though public speaking can be a nightmare for some and come very naturally for others, fear of public speaking, with differing degrees of intensity, can affect everyone from the most accomplished and experienced to the novice speaker. That said, there are ways we can overcome fears of public speaking and ultimately give the best presentation possible.

Thanks for reading our February 2019 Issue!