The pharmaceutical industry is evolving, supported by innovations that are changing the R&D landscape. Among these changes, we can note a growing use of innovative trial designs, the increasing contribution of China to clinical trials with a significant increase in the number of leading research institutions and very good success rates in clinical inspections. Finally, healthcare companies are increasingly leveraging artificial intelligence tools in preclinical and clinical development, thus promising a future where AI could revolutionize the way we discover and develop new treatments. Thus, in this article, Alcimed proposes to explore each of these 3 major trends in pharmaceutical R&D.
The successes obtained during the pandemic offered the pharmaceutical industry a valuable glimpse of how innovative trial designs can disrupt traditional clinical development approaches. Improving trial efficiency, reducing costs, accelerating drug development or improving treatment personalization while minimizing risks for patients, are innovative models that have revived the interest of governments, healthcare providers, regulatory authorities and the industry in optimizing clinical outcomes. The FDA and EMA have both recognized the potential of these approaches, integrating them into their strategic priorities for the coming years.
Since 2020, new clinical trial models have taken a significant place in clinical development, representing on average 18% of trials. Among these models, “Master protocols” (like “Umbrella trials,” “Basket trials”) and “Adaptive designs” have established themselves as key elements in the clinical trial pipeline. Oncology, in particular, has seen notable adoption of these models, with more than 29% of trials in 2023 using these approaches, showing a significant increase compared to the 13% observed in 2014. Although this percentage slightly decreased after reaching a peak of 32% in 2022, it remains nonetheless a strong indication of sustained interest in these approaches. This highlights the industry’s willingness to continue leveraging the benefits of innovative trial designs to improve the accuracy and efficiency of clinical development, particularly in complex therapeutic areas such as oncology.
According to IQVIA data, the contribution of Chinese pharmaceutical companies to global R&D has seen remarkable growth, going from 3% in 2013 to 28% of trials in 2023. This spectacular increase has allowed China to surpass Europe in terms of contribution, now being second only to the United States.
The rise of China in the field of pharmaceutical R&D is supported by a substantial increase in the number of leading research institutions in life sciences and medical research. According to the Nature Index, since 2015 China has seen an increase of 25 in the number of research institutions in life sciences and 31 in medical research institutions, which now also places it second in the world, just behind the United States. This progression reflects the rapid growth of China’s research and development capabilities in advanced technologies, strengthening its position on the international stage.
Alongside this expansion, the quality of R&D in China is receiving increasing attention. In 2022, the Food and Drug Inspection Center of the National Medical Products Administration (NMPA) conducted 215 clinical trial inspections for the registration of new drugs, with an impressive success rate of 99.07%. Moreover, FDA statistics between 2022 and today show that China has a higher success rate than the United States during manufacturing and clinical trial inspections. These results highlight not only the growing scale of R&D in China, but also the quality and rigor of this activity in the pharmaceutical field.
Pharmaceutical laboratories are today increasingly adopting approaches based on artificial intelligence (AI) for the research and development of new drugs.
Indeed, we can see for example in the 2024 report on R&D in the pharmaceutical industry sector from the IQVIA Institute, that more than 12 billion dollars in deals, linked to AI or Machine Learning (ML), were announced in 2023 (like Moderna and Immatics or Shape Therapeutics and Roche), representing more than double the total deals of 2022 and 2021. Furthermore, as proof of the dynamism of this activity, the average value of deals has also greatly increased, reaching 98 million dollars compared to 27 million dollars for the three years 2022, 2021, and 2019.
Clinical development programs leveraging artificial intelligence in the early discovery phase are now reaching a stage of maturity, marked by an increase in the number of programs in advanced phases. Although these programs still need to demonstrate the marketing of a new active substance, they already offer examples of new indications for existing drugs. This evolution is largely driven by companies in the healthcare sector, whether they are startups or already well established. They use AI and ML technology to exploit increasingly large sets of chemical, biological, and patient data, thereby accelerating target selection and drug optimization throughout the discovery process.
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The most common application of AI/ML in this field remains drug design, representing 42% of the products analyzed. These technologies allow the optimization of drug design by analyzing complex data sets, including molecular structure, molecular dynamics, genome, and combinatorial drug screening. According to Clarivate data, between 2013 and 2022, early phase trials (phase 0–2) dominated AI/ML-related clinical development, highlighting the importance of this technology in the early stages of new treatment development.
Finally, in an article published in June 2024, aiming to highlight the clinical potential of AI-discovered molecules, Madura KP Jayatunga et al. found, through an initial analysis of clinical pipelines, that in Phase I, molecules discovered by AI had a success rate of 80 to 90% (compared to 40% to 65% on average for human-discovered molecules). However, in Phase II, the success rate was about 40%, which is comparable to historical averages (30%-40%). These results thus suggest that drug discovery through AI would have a higher success rate than what is done today without it.
In conclusion, the pharmaceutical industry is today at a crossroads, redefining traditional approaches to clinical development. Indeed, the global pandemic has highlighted the effectiveness of new trial designs, which not only accelerate drug development but also reduce costs and improve treatment personalization. At the same time, China is emerging as a major player in global R&D, surpassing Europe and positioning itself just behind the United States, both in terms of contribution and quality. Finally, massive investments in AI have led to an increase in late-stage clinical development programs, although the marketing of new active substances remains to be demonstrated. These trends are shaping a new era of innovation and efficiency in the pharmaceutical industry. Alcimed closely follows innovations in the pharmaceutical sector and can support you in your projects related to drug research and development. Do not hesitate to contact our team!
About the author,
Thibaud, Consultant in Alcimed’s Healthcare team in France.
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