May. 10. 2023Blog
How to unlock Japan’s RM sector opportunity: the role of Japanese CDMOs
The cell and gene therapies and regenerative medicines (CGT&RMs) market is booming. In 2021 alone, the sector received almost $23 billion of investment.1
Accordingly, countries and companies across the world are seeking ways to accelerate the route to market for these transformative therapies. And Japan has become the go-to location for such expedited development.
But fully unlocking Japan’s CGT&RM potential can be a challenge for companies ill-prepared and unfamiliar with the region.
In the third blog of this series, we’ll reiterate the immense opportunity Japan presents for CGT&RM developers, the potential challenges these developers might face, and most importantly, how they can overcome these to best ensure success.
Japan: a global hotspot for CGT&RM innovation
In our blog highlighting Japan’s CGT&RM opportunity, we described just how well-equipped and forward-thinking Japan is when it comes to CGT&RM development, manufacture, and commercialization: the market is rapidly expanding, the country’s aging population is set to drive continuing demand for novel therapies such as CGT&RMs, and a unique set of CGT&RM-centric regulations have carved an expedited route-to-market for these promising products.
Such benefits, along with a host of other capabilities ( find out more about them in our eBook.), are what make Japan one of the most attractive regions for CGT&RM development.
Navigating regulatory differences
While the benefits Japan offers are immense, the country’s unique regulatory landscape can catch unprepared and ill-informed companies by surprise, potentially barring the path to success.
For example, the Standards for Biological Ingredients (SBI) — which help ensure products manufactured using biologically derived materials have the required quality, safety, and efficacy — means manufacturers must prove they meet a host of requirements before clinical trials can start. Similarly, the Cartagena Act (CA) demands a potentially complex and time-consuming environmental risk assessment ahead of clinical trials. (For more information about these unique regulations, read our blog highlighting the key considerations for CGT&RM success in Japan.)
The Japanese CDMO: your key to CGT&RM success in Japan
Navigating regulatory differences, and other unique aspects of CGT&RM development in Japan, can be daunting.
Fortunately, companies have a tried and tested approach to help them fast-track their path to commercialization in Japan: partnering with a knowledgeable, experienced Japan-based contract development and manufacturing organization (CDMO). For smaller foreign pharmaceutical and biotechnology companies, this is often the only truly cost-effective route to CGT&RM commercialization in Japan.
Selecting the right partner, however, is crucial — the range of capabilities and experience on offer varies significantly, and this can be the difference between CGT&RM program success and failure.
Below we’ve shared some of the critical capabilities to look for in a success-enabling CDMO:
Deep regulatory know-how is a priority
Japan’s regulatory environment has the potential to significantly accelerate the delivery of CGT&RMs to market. Indeed, under the Pharmaceutical and Medical Devices (PMD) act, CGT&RM developers that can demonstrate safety and presumable clinical benefit, can bring their product to market much faster under conditional approval. To ensure foreign companies aren’t caught off guard by this new landscape (by regulations such as the SBI), they must source deep regulatory expertise as early as possible, ensuring they can grasp regulatory expectations quickly, and better plan a risk-mitigating development strategy.
For similar reasons, companies need to engage with Japan’s regulatory authorities early and frequently. Such early consultation can also bring to light important financial privileges, helping developers avoid unnecessary costs.
Given the above, it is vital that a CDMO partner has broad and deep regulatory knowledge, as well as established relationships with regulatory bodies to smooth the consultation process. In fact, this is perhaps one of the most important capabilities to look for when selecting a CDMO partner.
CGT&RM-specific manufacturing process expertise
Since CGT&RMs rely on sensitive and variable biological raw materials and/or living cells, manufacturing them is uniquely challenging. Creating scalable, compliant manufacturing processes may not even be possible without significant manufacturing process development know-how and a range of appropriate advanced technologies.
Minimizing the risk of out-of-specification (OOS) products is also inherently more difficult for cell and tissue-based therapies. Doing so successfully requires skills and experience in continuous improvement of manufacturing methods and specifications, and the ability to rapidly correct the cause of OOS results.
These capabilities, therefore, should be high up on your list when it comes to selecting your ideal CDMO.
Deeper considerations to unlock success in Japan
Ensuring your CDMO partner meets the above two requirements will go a long way to helping you make the most of Japan’s CGT&RM opportunity.
However, many other capabilities are key for a smooth path to commercialization, too.
Get a comprehensive CDMO capability check-list — and discover more about Japan’s unparalleled CGT&RM opportunity — in our FREE, insight-packed eBook: “Tapping into Japan’s Opportunity in Regenerative Medicine and Cell & Gene Therapy”
References (Link to external sites) ：
1) Alliance for Regenerative Medicine (ARM) Regenerative Medicine: The Pipeline Momentum Builds H1 2022
