Meeting Highlights: DIA Japan 2021

DIA Japan 2021 conference logo of red and white plus signs

Japan and the Pandemic: New Challenges, New Solutions

Rie Matsui
Pfizer R&D Japan

Stewart Geary
Eisai Co., Ltd.

T

he theme of DIA Japan 2021, “New Challenges, New Solutions: To Overcome With People All Over the World,” reflected the current background of marshaling resources from regulatory authorities, industry, and academia to develop innovative new therapies in the midst of an unexpected public health emergency while simultaneously transforming daily work practices during lockdowns and limited public access to hospitals.

New Society and Healthcare Ahead of the Digital Revolution

In remarks titled A New Society and Healthcare Ahead of the Digital Revolution, Keynote Presenter Hiroaki Miyata (Keio University) challenged the clinical research and therapeutic product development community in Japan to shift its focus from GDP (gross domestic product) to GDW (gross domestic well-being). He further described this as shifting focus from the “Greatest Happiness of the Greatest Number” to the “Greatest Happiness of the Greatest Diversity.” While it has been difficult to customize and individualize therapies, this is now increasingly possible using artificial intelligence and great growth in available data sources to gain deeper insights into and more empathy toward patients, and digital power can connect people to create greater individualization and inclusion. The increased ability to tailor therapies to an individual’s circumstances, sense of values, and personal biology allows a diversification of therapy to meet the needs of people of all ages and backgrounds, across different medical and social conditions, as we increasingly recognize the essential diversity within all societies in our pursuit of the “Greatest Happiness.” He concluded that it is important for all stakeholders in Japan to work toward “better co-being, to connect lives and make lives shine.”

Current and Future State of Drug Development Under COVID-19

Drugs and vaccines for COVID-19 have been developed at an unprecedented pace, with Emergency Use Authorizations, Special Approvals for Emergency, or equivalent authorizations issued across the globe. The pandemic introduced many changes to drug development, clinical operations, and regulatory evaluations. Decentralized clinical trials have been accelerating in drug development, and real-world data (RWD) is now utilized at nearly every stage of the drug lifecycle. Other changes made in response to COVID-19 include increasing use of smart watches and other digital health technology in clinical trials, remote instead of onsite inspections, and hybrid (a combination of remote and face-to-face) monitoring.

PMDA in Japan, as well as the EMA and FDA, issued regulatory guidance for sponsors in changing trial protocols to adapt to the pandemic and yet ensure essential GCP compliance. Other impacts highlighted the need for further collaboration on GMP inspections in response to sudden shifts in supply chains. Globally, the role of the International Coalition of Medicines Regulatory Agencies (ICMRA) has become more important during the pandemic, as it has held several workshops on the development of vaccines and therapeutics and issued official statements on clinical trials, regulatory flexibility, remote inspections, emergency authorizations, and a host of issues related to public health and confidence in vaccines.

Collaboration and transparency between regulators, industry, healthcare professionals, and patients will remain essential to enabling rapid adoption of new processes and tools. The drug and vaccine regulatory evaluation system must continue to earn and sustain the public’s trust. Transparency in policy making is as important as transparency in science, or “open science,” which we’ve seen demonstrated by several sponsors publishing their COVID-19 vaccine study protocols for broader use. The multiplication of media sources providing sometimes incomplete or inaccurate information presents the public with a wide range of both useful and misleading information, making it difficult for people to know which source they can trust. What are the public’s most pressing questions about the pandemic, and what would they like to learn more about? Answering these questions is essential to transparent public communication.

The current pandemic has also helped identify important principles and tools to help prepare for the next one, including agility and sustainability; variety of platforms for clinical trials; availability of manufacturing sites; international collaboration; and capacity building and governance for developing vaccines and medicines. Additional principles and tools identified by the speakers as especially important for Japan include collaboration between government and industry; continuous government investment in technology; secure funding for infectious disease researchers, epidemiologists, and similar professionals; continued research into emerging infectious diseases; and development of government tools for emergencies such as national shutdown orders and other legislation.
Current and Future Status of Drug Development in Japan, the US, and Europe Under COVID-19 Pandemic

Session Chairs: Junko Sato, PMDA; Hironobu Saito, Daiichi Sankyo.

Panelists: Emer Cooke, EMA; Yasuhiro Fujiwara, PMDA; Satoshi Iwata, National Cancer Center Hospital; Theresa M. Mullin, FDA; George Nakayama, Daiichi Sankyo.

PMDA Town Hall

The government of Japan has recently established a new Digital Agency. PMDA has already experienced challenges with digital data standardization and reliability and looks forward to working with this agency on these and related issues to help advance the digitalization of healthcare in Japan.
Real-world evidence/data (RWE/RWD) continue to grow in importance in regulatory decision making in Japan. However, several sponsors have expressed various difficulties with new drug applications utilizing such evidence/data. One speaker shared three examples in which FDA did not consider RWD used as an external control to be evidentiary and recommended getting prior regulatory agreement before initiating the study, identifying potential sources of bias in selection of RWD, and doing a feasibility assessment of the sample size for RWD that can be achieved using the study’s inclusion/exclusion criteria. Data integrity has also been raised as an issue in decentralized clinical trials (DCTs) in Japan. These and related experiences have highlighted the importance of large, robust, high-quality RWE data sets and prior, advance regulatory consultation about use of these data.
PMDA Town Hall

Session Chairs: Toshihiko Doi, National Cancer Center Hospital East; Takuko Sawada, Shionogi & Co. Ltd.

Panelists: Mie Ikeda, Office of Safety I; Kiyomi Ueno, Office of Safety I; Yasuhiro Araki, Deputy Director; Kenichi Tamiya, Associate Executive Director; Kensuke Ishii, Office of Medical Devices I; Yoshiaki Uyama, Office of Medical Informatics and Epidemiology.

PMDA has also recently issued guidance to promote patient participation in the PMDA’s work. This guidance builds upon the input PMDA already receives from patients in areas of high unmet medical need, calling for patient participation in certain PMDA and MHLW subcommittee meetings, information exchange meetings with patient groups, and seeking patient feedback about the content and methods of communication currently provided to patients.