DIA Japan Annual Meeting

Innovation: From Japan to the World…and Back Again

Innovation: From Japan to the World…and Back Again

Kazuhiro Kanmuri
Founder & CEO
Inter-Professional, Inc.

Alberto Grignolo
Corporate Vice President
PAREXEL International

J

apanese scientists have won the Nobel Prize for Physiology or Medicine in four of the past seven years (2012, 2015, 2016, and 2018). Japan is no stranger to innovation.

The November 2018 DIA Japan Annual Meeting made it clear once more that today’s cutting-edge innovations and ideas in the healthcare environment know no national boundaries and are only constrained by our ability and willingness to flex, adapt, and improve patients’ lives.

Diverse discussions, driven by a common desire to make progress in developing new medical products effectively and efficiently through global collaboration, ranged from the costs of R&D to clinical trials, real world evidence, accelerated pathways such as sakigake, AI applications in drug development, patient centricity and engagement, translational and precision medicine, Health Technology Assessment, and more. These are indeed the “hot topics” of the day around the world and in Japan.

Cost of R&D

Rising R&D costs are a worldwide issue. Although technological cooperation and business alliances such as open innovation have been promoted globally and aggressively, we still struggle to depart significantly from the current high-cost R&D model. Innovative technology is contributing not only to R&D but also to healthcare improvement. For example, the idea of enabling telemedicine has become possible by combining Information Technology with technological innovations in medical equipment, an approach that could also be applied to clinical development. The idea of utilizing existing data (such as medical big data) and developing a common platform to increase R&D efficiency can be one of its drivers.

Clinical Trials

There are great expectations in the world of clinical trial operations for the application of Information and Communication Technology (ICT) through mobile health devices, and using existing data collected from many sources, such as electronic health/medical records and mobile devices, was discussed extensively. Japan’s Medical Information Database Network (MID-NET) is leading the way. Novel use of emerging technologies and large bodies of data could help reduce the cost of R&D, provided that the necessary collaborations are established and function well.

The Investigator Initiated Clinical Trials for Registration (IICT) framework, which is fully compliant with the J-GCP standard, has been available in Japan since 2003. Many additional indications contributed by the IICT have been approved by the Japanese health authority and this framework has become important within the Japanese healthcare environment. Currently, from a speed, quality and cost perspective, the efficiencies applied in this IICT environment stimulates interest in early stage clinical trials run by small biotech companies as well as by venture companies in Japan.

Key components of ICH-E6 (R2), such as clinical trial design, data integrity and computer/IT system validation, clinical trial operations/oversight, SOP refinement, and appropriate training, are closely linked to both clinical trial efficiency and patient safety. Many stakeholders have struggled to adopt an appropriate approach to the coming ICH-E6(R2) implementation (risk-based monitoring, QMS, centralized monitoring strategy, CRO oversight etc.). These challenges are still in their initial stages.

Kazuhiko Mori, the representative from MHLW and Senior Councilor for Pharmaceutical Affairs, repeatedly emphasized the importance of applying new technologies (such as the Internet of Things [IoT], artificial intelligence, and medical big data) and new methodologies (fully using existing data such as patient registries in new approaches to clinical trials and post-marketing safety) in regulatory science, and PMDA’s readiness to collaborate in such R&D advancement.

Sakigake and PMDA Workload

The goal of the accelerated development pathway known as sakigake does not simply imply fast approval but rather the opportunity to engage PMDA in early and regular consultations to deeply understand the nature of the proposed innovation, Mr. Mori explained. PMDA stated that the sakigake workload created “a lot of overtime” for PMDA reviewers, and there are plans to hire 100 additional staff to cope with the workload as a result.

Health Literacy and Patient Centricity

While “literacy” is defined as the ability to read and write. “health literacy” has broader meaning. As information sources have diversified, improvements in health information literacy have introduced global challenges in both the proper use of pharmaceutical products and patient centricity. Healthcare providers and professionals often struggle to convey health information to patients as a risk minimization tool. It is already difficult to help patients better understand the effectiveness and safety information of their medical products. Medical economics touch another area of health information that experts must explain when patients have multiple treatment options at varying costs from which they can select. Debate continues on how to address varying levels of literacy in patients and what experts must do to improve health literacy. Furthermore, medical information professionals and other information providers must consider ethical and compliance implications. Momentum towards patient centricity is rapidly growing; patients’ health literacy and information providers’ awareness/efforts/behaviors are expected to improve significantly as one fundamental pillar of patient centricity in Japan.

ICMRA

The International Coalition of Medicines Regulatory Authorities (ICMRA) met in parallel with the DIA Japan Annual Meeting and presented some elements of its ongoing work. The ICMRA Innovation Project is composed of three workstreams: 1) to investigate and research each Agency’s horizon scanning methodology; 2) to share and leverage the outcomes of horizon scanning; and 3) to discuss the latest trends in novel pathways to licensing. The final report of these workstreams will be available in March 2019.

It is now very clear, and was stated openly, that 21st century regulators see a new role for themselves as “enablers” who connect the relevant stakeholders and facilitate the development and approval of innovative products for patients – engaging in such fields as genome editing, RWD/RWE, AI and “bedside manufacturing” of cell therapy products. Their message was that disruptive technologies call for disruptive (expedited and facilitated) regulatory processes: Regulators are ready for change.