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Pharmaceutical R&D in Japan Needs an Upgrade

Fumitaka Noji


lthough the importance of open innovation in drug research and development has long been recognized in Japan, it cannot be said that the open innovation ecosystem in Japan is functioning sufficiently when many issues remain problematic or unresolved in terms of systems, funding, and human resources compared to Europe, the US, and China. This situation was made even more evident by the COVID-19 pandemic.

As a countermeasure, in addition to regulatory policy measures such as establishing a novel regulatory review/approval system for medical products, it is essential to urgently establish and operate an ecosystem that effectively utilizes open innovation from drug discovery research and development (R&D) to manufacturing and commercialization. This article illustrates several challenges to open innovation in drug research and development in Japan.

In the US, most of the drugs approved by FDA originate in academia and bioventures (Japanese only), demonstrating that these two entities (the former often supported by government funding, e.g., NIH) are the main players in drug research and development. But it is less common in Japan (Japanese only) to see drugs developed collaboratively by academia and industry receive regulatory approval even though 70% of researchers in the life sciences field belong to universities (Japanese only) and are actively conducting basic research. Well-known drugs such as crizotinib, mogamulizumab, trametinib, and nivolumab that target a disease-related molecule discovered by a Japanese researcher* are the few success stories of drugs commercialized as a result of industry-academia collaborations in Japan.

To improve this situation, it is necessary (1) to find excellent “seed” technologies from the perspective of pharmaceutical companies and related scientific interests since they conduct clinical development, manufacturing, and marketing; (2) to promote collaboration between various academic centers and pharmaceutical companies; and (3) to create an ecosystem for the practical application of research results. But the following challenges remain.

Challenges in Strategy and Information

The major platforms for promoting open innovation in Japan focus on providing one-stop opportunities for academia or industry that have novel seeds or technologies to find potential collaboration partners to further their development.

When considering how to pursue the practical application of seeds or technologies in collaboration with pharmaceutical companies, it is essential for academic or industry researchers to consider profiles on pharmacology, pharmacokinetics, safety, and manufacturing/CMC. They must also consider intellectual property rights, feasibility studies, marketing strategy, and regulatory strategy.

However, it is not so easy for academia or bioventure companies to investigate and analyze these points on their own. Conversely, experts in each of the above fields are considered essential in industry. But the expertise to identify and resolve challenges with such issues is rarely if ever sufficiently established in academia or bioventures.

For their part, pharmaceutical companies considering collaboration with academia or bioventures for novel seeds or technologies must clarify their desired target product profile (TPP) during due diligence, in order to minimize the risks normally associated with business investments. In cases where the drug TPP was not clarified, significant gaps arise between the information provided by academia or bioventures and the information required by pharmaceutical companies.

Even when excellent seeds are found, academia and industry sometimes do not agree on the social value of the potential product and do not proceed further in that collaboration.

Challenges in Receiving Investment

The Japanese Agency for Medical Research and Development (AMED), established by the Japanese government in 2015, provides public funding to drive pharmaceutical R&D, taking into account the characteristics and properties of modalities in areas ranging from target identification to clinical research, to promote the practical application of drugs to address high medical needs.

These funds are less than the comparable public funding in Europe and the US. In addition, there are few venture capital firms in Japan. This often causes a shortage of funds for pharmaceutical R&D in Japan and leads to difficulty in gathering information on emerging seeds or proceeding to the next development stage. Some bioventures may receive a large amount of overseas investment. In these cases, management team members who demonstrate expertise in R&D, business development, clinical development, fundraising, legal and government works, etc., are nearly as essential as the target seeds.

Challenges in Finding Qualified Experts

Some expert observers have noted that in Japan (Japanese only) there is a shortage of professionals who can skillfully gather the required information and can identify high-potential seeds or technologies, especially when compared to their numerous counterparts in Europe and the US. They also note a shortage of experts who can act as a bridge between academia/bioventures and pharmaceutical companies by providing knowledge about industry research, development, manufacturing, and marketing practices. Funding constraints often make it impractical for academic or bioventure research organizations to hire or otherwise engage such expertise (e.g., via a third-party contract).

Continued and committed collaboration between academia, bioventures, pharmaceutical companies, and the government are the only way to solve these issues and to promote open innovation that brings true value to patients and society in Japan.

* Crizotinib target EML4-ALK discovered by Hiroyuki Mano.
Mogamulizumab target CCR4 discovered by Ryuzo Ueda.
Trametinib target MEK by RB-reactivator screening discovered by Toshiyuki Sakai.
Nivolumab target PD-1 discovered by Tasuku Honjo.