The past year has marked a turning point for New Approach Methodologies (NAMs) in preclinical drug development. No longer experimental curiosities, these human-relevant approaches have rapidly gained traction, driven by a growing evidence pool and major regulatory reforms. There is rising pressure on regulators to support drug developers to look beyond in vivo animal testing. As a result, NAM adoption is evolving into a global movement, recognizing that these advanced, human-relevant, animal-free approaches can bring new treatment options to patients faster and with greater predictive accuracy.
The status quo of drug discovery and development
Animal research has historically been central to drug discovery, enabling researchers to evaluate how drugs behave in a whole living system prior to human trials. However, with only one in ten drugs entering phase I trials going on to receive regulatory approval, the disconnect between the preclinical lab and the clinic is clear. These drug failures are not only costly, but can result in unnecessary animal use – all driven by the inherent, inter-species differences that limit the predictive value of animal models.
Now more than ever, NAMs need to be considered a core aspect of modern drug development – and scientists, decision-makers, regulators and governments must keep pace with this evolving landscape to ensure they are equipped to deliver the next-generation of therapies.
Regulatory shifts are increasing adoption of NAMs but there’s still a way to go
2025 saw unprecedented regulatory shifts shine a spotlight on the NAMs sector. Announcements from the US FDA, as well as the UK Government’s animal testing roadmap, have led to a clear acceleration in the market, reminiscent of the early days of organ-on-a-chip (OOC) technology, which saw growth rates of up to 40%. These moves prompted a sharp uptick in scientists across academia and industry showing interest in these technologies. Researchers who previously overlooked NAMs began exploring its potential, and those that were already considering it made the leap to purchase and now have the data and confidence to invest more deeply.
There is, however, still work to be done, particularly in educating researchers about where NAMs deliver the most value. Many organizations want to integrate these methods as an alternative to animal models, but are less certain about how to deploy them effectively. There is an important role for NAMs providers to play in education and awareness – showing people what is possible, the applications, and what sort of data they can generate, will help to drive broader adoption of these technologies.
New initiatives, such as the FDA’s ISTAND (Innovative Science and Technology Approaches for New Drugs) program are extremely valuable to address the awareness and understanding of these bottlenecks. The program accepts submissions for qualification of drug development tools (DDTs) that are out of scope for existing DDT qualification programs, allowing NAMs developers to build the evidence base needed to strengthen regulatory confidence in emerging technologies.
It is also important to note that there is still much discussion between pharma and regulators in terms of what sort of NAMs data they will accept in IND filings. Until there is clarity on this, adoption is going to happen slowly, despite evidence demonstrating superior performance over traditional approaches. Added to that, a lot of the guidance is just that – guidance – and without more compelling reasons to make the switch, there are many who are happy to remain in the status quo. The next few years will be crucial in that regard, as we begin to see more IPs come through. Once people see the sort of datasets used in regulatory filings from OOC technologies and other NAMs, it will make it a lot easier for others to follow.
Integrating AI and OOC technologies to drive more accurate preclinical insights
AI-driven approaches have quickly become one of the hottest topics in drug discovery, and almost everyone working in the space has likely considered if and how to embed AI within their discovery pipelines. Technologies like OOC offer significant advantages here as their data outputs naturally feed into machine-learning algorithms. In turn, these algorithms can be harnessed to help refine models; helping to produce better results, generate potential new leads, and aid with validation.
For NAMs providers, the next step now is asking how we can make the most of AI – how do you take data from a microphysiological system (MPS) or advanced in vitro model, and then use computational modelling to translate that data more broadly to predict outcomes across broader patient populations? We’re already seeing advancements here to some extent, but it’s likely we’ll continue to see more of this in the coming years.
Accelerating research for new modalities
NAMs are providing invaluable insights across the new modalities space; from gene and cell therapies, to ADCs and PROTACs. One of the main bottlenecks when developing these therapeutics is that each molecule class requires new, highly specific assays, largely due to difficulties in validation because there are no pre-existing tests. Ultimately, a lot of these modalities are being held back because they are harder to develop, relatively few exist in clinical development and even fewer have received regulatory approval. These challenges create a lot of uncertainty in the advanced therapies space – with less existing data validating safety, many therapeutic developers are reluctant to progress therapeutic candidates without extensive preclinical data.
In many cases, animal models are unsuitable for these modalities, as they often exploit human-specific targets or pathways. Perhaps as expected, this is also where we are seeing NAMs become more and more prevalent in regulatory submissions, and likely the first area where we’ll see NAMs garner broad acceptance.
NAMs providers excel in a changing biotech market
Amongst years of market turbulence, we continue to see biotech companies unfortunately struggle, and it’s becoming increasingly apparent how challenging it is to sell an early-stage asset without a strong, validated base of evidence that those assets will be successful. Added to this is the limited funding available to most small biotechs, which leaves many needing to find new and more affordable ways to generate earlier data providing clear proof of the asset or company’s value. This is where NAMs are poised to play a major role, because they are generally more cost effective for a lot of assays. OOC in particular has already proven its value in several patent filings and early regulatory submissions – all that remains is for more researchers to make the jump and use it as a springboard to push their pipelines toward commercial success.NAMs are already demonstrating their value in overcoming the in vitro to in vivo translation gap, supporting accurate decision-making earlier in the pipeline, ultimately reducing our reliance on animal studies where their use is not suited. At a time when the life sciences sector faces economic and scientific pressures, NAMs providers are uniquely positioned to work alongside regulators, industry and academia to accelerate adoption. If this momentum continues, the coming years could see a fundamental shift in how preclinical research is conducted – creating a new drug development landscape with a greater focus on more predictive, human-relevant science.
Photo: Petmal, Getty Images
Dr. Paul Brooks joined CN Bio Innovations in 2022. He has over 25 years of experience in building businesses and leading high-performance research, product, marketing, and sales teams to develop and commercialize new biotechnology technologies globally for drug discovery, bioproduction and diagnostics.
Dr. Brooks has held senior leadership positions in the USA and the UK, including Head of Business Operations and Managing Director of Horizon Discovery Ltd; Chief Commercial Officer and Executive Board member of Oxford Genetics Ltd; Head of Discovery Research Services at MilliporeSigma (Merck KGaA); and Global Marketing Manager at Sigma-Aldrich Corp. Dr Brooks has a BSc in Biochemistry from the University of Wales, a PhD in Molecular Biology from the University of Manchester Institute of Science and Technology (UMIST), and an MBA from the University of Nottingham Business School.
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