From big volumes to precision medicine: Why pharma’s future is personalised

Targeted therapies have improved treatment outcomes for specific disease subsets, with HER2-targeted therapies in breast cancer being a prime example (1). These advancements have emerged due to improved understanding of the molecular characteristics of specific disease states. The move towards greater personalisation in medicine is following a similar trajectory. As our knowledge of diseases grows, we gain a deeper understanding of the individuality of each disease and the promise of personalised treatments.

Advances in areas like genomics and biomarker research have significantly increased the availability of high-quality data, playing a key role in enabling more personalised treatment approaches (2). AI has also played a part, enabling the integration of increasingly large and diverse datasets and helping to spot patterns (3). The rise of personalised medicine is leading to improved patient outcomes and is emerging as the norm for the treatment of many diseases, especially rare diseases (4,5). Market and regulatory bodies are adapting to this trend, placing the onus on organisations to adjust their production and compliance processes to remain competitive and provide life-changing therapies to patients. This article discusses the shift towards more personalised treatment approaches, the adaptations this transition demands, and how NIRAS can help through guidance on facility design, compliance and sustainability.

The rise of customisation

The high volume era of drug development saw the use of single therapies across a broad range of disease indications. For example, Adalimumab (Humira) is an anti-inflammatory drug used to treat a variety of conditions, including psoriasis, arthritis, Crohn’s disease, and ulcerative colitis by targeting TNF, a molecule common to inflammatory responses (6,7). Pembrolizumab (Keytruda) is a targeted immunotherapy that is used to treat a diverse set of cancer types, including breast cancer, melanoma, and non-small cell lung cancer (NSCLC), and illustrates the shift towards more personalised approaches, while maintaining broad applicability (8). These therapies achieved global sales of $9 billion and $29.5 billion in 2024, respectively, demonstrating a continued importance in the market (9,10).

While high volume drugs continue to have an important role in healthcare, this approach to therapy is becoming increasingly outdated, as patient-specific diseases become more clearly defined, and tailored treatment opportunities arise as a result. Furthermore, many drugs, including Humira, are losing patent protection and market exclusivity, requiring companies to develop new production lines to maintain revenue (11). High volume drugs often fail to work effectively for all patients within certain subgroups. By contrast, personalised medicine offers greater potential to improve patient outcomes and achieve a stronger market impact. Sponsors have an easier time demonstrating the value of personalised medicines to regulators, and they require smaller, more focused clinical trials (12).

What is personalised medicine?

Personalised medicine involves tailoring treatments to patients based on their specific health characteristics (13). These characteristics include genetics, molecular markers, and environmental and lifestyle factors, which can play a part in a patient's susceptibility to disease and the responsiveness to treatment. The push towards morestreatment approaches is driven by greater insights into disease, which are made possible through technological advances in areas like genome sequencing and AI. Cell and gene therapies (CGTs) have emerged as frontrunners in enabling the translation of patient-specific data into personalised medicines (14). Cell therapies are a core technology driving personalised medicine, as a patient's own cells can be used as a treatment, while gene therapies can be used to correct or replace genes rendered defective by mutation.

Cancers are highly individualised, and even patients within a molecular subclass can have vastly different responses to therapy (15,16). Genetic sequencing allows for patient-specific markers to be identified, and new therapy modes like CGTs can be used to exploit those markers to improve outcomes. These developments have a significant impact on pharmaceutical production as long-term production of one-size-fits-all high volume therapies is phased out, including: 

• Smaller batch sizes
• Faster development
• Faster production line turnover 

Shifting investment requires proactive planning

There is growing investment in CGTs, with investments in 2024 reaching $15.2 billion, a 30% increase compared to 2023 (17). Large pharma companies are directly investing in this market and acquiring smaller companies with dedicated CGT development lines. For instance, Eli Lilly recently completed the acquisition of Verve Therapeutics for a possible $1.3 billion. Verve focuses on CRISPR-mediated gene editing, and a proprietary therapeutic targeting the PCSK9 gene has passed Phase 1 clinical trials for reducing low-density lipoprotein levels (18).

To adapt to this shift in the market, pharma companies must rethink production facility dynamics. Mega-factories with ultra-large-scale production lines for single therapies are not suitable for the needs of personalised treatments. Instead, modular, flexible production sites are viewed as the optimal way for companies to adapt to market dynamics and technological advancements, while achieving the necessary scale for personalised treatments. These developments are accompanied by renewed regulatory oversight, especially for gene therapies, which can introduce changes into a patient's genetic makeup (19). Data integrity and traceability tools must co-evolve with new production plant layouts, especially as production lines are turned over and novel therapies introduce unforeseen challenges.

NIRAS is home to pharma industry experts with their fingers on the pulse of the shifting market. As a result, NIRAS has a strong understanding of the shift toward personalised therapies and helps pharmaceutical companies identify and implement production strategies that support adaptability in an evolving market.

●    Facility Design: NIRAS supports industry leaders in the design of modern production facilities, including process plants, classified production areas, and laboratories. The push towards smaller scale and flexible manufacturing requires companies to understand their goals, the available instrumentation and infrastructure solutions, and to build accordingly. NIRAS assists in layout design, equipment selection, and the design of pipes, components, and tanks to help companies achieve seamless transition to the personalised medicine market.

●    Compliance: Regulatory requirements are in flux, and the CGT market is subject to increased regulatory scrutiny. NIRAS regulatory experts help companies align with cGMP standards and safety compliance, while offering robust regulatory guidance to support product classification, regulatory submissions, and the review and updating of regulatory documentation.

●    Sustainability: Changes in the pharmaceutical market coincide with the push for more sustainable development practices. As companies adapt to personalised therapies, NIRAS can help ensure they meet their sustainability goals. This includes the design and implementation of ventilation and cooling systems for pharmaceutical facilities, helping to achieve greater energy efficiency while reducing the risk of production downtime.

Conclusion

The medical field has gradually moved towards greater levels of treatment personalisation. While targeting markers like PD1 and HER2 has helped many patients, there remains scope for further personalisation to meet the specific needs of individual patients. This shift is made possible by technological advances in areas like CGTs and genomic sequencing and is having a profound impact on therapy production. Success in the increasingly niche-driven market requires adaptation to more flexible production layouts while adhering to strict regulatory requirements. NIRAS helps forward-thinking pharmaceutical companies navigate this transition, offering expert guidance on facility design, compliance, data protection, and sustainability. The gap between the high volume drug era and personalised therapies continues to widen, and timely, expert-led investment in personalised treatments is set to drive long-term value for decades to come.

Contact a NIRAS expert today to learn more about how you can make the transition to flexible production and excel in a shifting marketplace.

Request more information:

References

1.    Swain SM, Shastry M, Hamilton E. Targeting HER2-positive breast cancer: advances and future directions. Nat Rev Drug Discov. 2023;22(2):101-126. doi:10.1038/s41573-022-00579-0
2.    Khan A, Barapatre AR, Babar N, et al. Genomic medicine and personalized treatment: a narrative review. Ann Med Surg (Lond). 2025;87(3):1406-1414. doi:10.1097/MS9.0000000000002965
3.    Johnson KB, Wei WQ, Weeraratne D, et al. Precision Medicine, AI, and the Future of Personalized Health Care. Clin Transl Sci. 2021;14(1):86-93. doi:10.1111/cts.12884
4.    Passaro A, Al Bakir M, Hamilton EG, et al. Cancer biomarkers: Emerging trends and clinical implications for personalized treatment. Cell. 2024;187(7):1617-1635. doi:10.1016/j.cell.2024.02.041
5.    Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment | National Institutes of Health (NIH). Accessed July 10, 2025. https://www.nih.gov/news-events/news-releases/infant-rare-incurable-disease-first-successfully-receive-personalized-gene-therapy-treatment
6.    Humira | European Medicines Agency (EMA). July 26, 2018. Accessed July 10, 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/humira
7.    van Loo G, Bertrand MJM. Death by TNF: a road to inflammation. Nat Rev Immunol. 2023;23(5):289-303. doi:10.1038/s41577-022-00792-3
8.    Keytruda | European Medicines Agency (EMA). June 21, 2018. Accessed July 10, 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/keytruda
9.    Merck Announces Fourth-Quarter and Full-Year 2024 Financial Results. Merck.com. Accessed July 10, 2025. https://www.merck.com/news/merck-announces-fourth-quarter-and-full-year-2024-financial-results/
10.    AbbVie Reports Full-Year and Fourth-Quarter 2024 Financial Results | AbbVie. Accessed July 10, 2025. https://investors.abbvie.com/news-releases/news-release-details/abbvie-reports-full-year-and-fourth-quarter-2024-financial
11.    Kakkar AK. Patent cliff mitigation strategies: giving new life to high volumes. Expert Opin Ther Pat. 2015;25(12):1353-1359. doi:10.1517/13543776.2015.1088833
12.    Gibson S, Raziee HR, Lemmens T. Why the Shift? Taking a Closer Look at the Growing Interest in Niche Markets and Personalized Medicine. World Med Health Policy. 2015;7(1):3-27. doi:10.1002/wmh3.131
13.    Personalised medicine - European Commission. July 7, 2025. Accessed July 10, 2025. https://health.ec.europa.eu/medicinal-products/personalised-medicine_en
14.    Chancellor D, Barrett D, Nguyen-Jatkoe L, Millington S, Eckhardt F. The state of cell and gene therapy in 2023. Mol Ther. 2023;31(12):3376-3388. doi:10.1016/j.ymthe.2023.11.001
15.    Wu B, Zhang B, Li B, Wu H, Jiang M. Cold and hot tumors: from molecular mechanisms to targeted therapy. Sig Transduct Target Ther. 2024;9(1). doi:10.1038/s41392-024-01979-x
16.    Garcia-Recio S, Zagami P, Felsheim BM, et al. Understanding metastasis mixed-treatment responses through genomic analyses. npj Breast Cancer. 2025;11(1). doi:10.1038/s41523-025-00724-z
17.    Insights 5 min read | BioSpace. Cell and Gene Therapy Sector Sees 30% Investment Surge Despite Market Challenges. BioSpace. March 31, 2025. Accessed July 10, 2025. https://www.biospace.com/career-events/cell-and-gene-therapy-sector-sees-30-investment-surge-despite-market-challenges
18.    Temple-West P, Barnes O. Eli Lilly strikes deal to buy Crispr gene-editing biotech Verve. Financial Times. https://www.ft.com/content/53d021c9-d15f-4471-86ef-e9abfc3ae7db. June 17, 2025. Accessed July 10, 2025.
19.    Drago D, Foss-Campbell B, Wonnacott K, Barrett D, Ndu A. Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs. Mol Ther Methods Clin Dev. 2021;21:524-529. doi:10.1016/j.omtm.2021.04.001