With the implementation of technology rapidly increasing, stricter regulatory standards, and growing demand for innovative treatments, the trends within the industry continue to evolve, impacting the future of clinical trials both positively and negatively. In our article, we cover the key trends that have been shaping clinical trials so far in 2024, and those that are predicted to continue into 2025.
Patient-Centric Trials Become Essential
One of the most significant shifts in recent years has been the rapid adoption of Decentralised Clinical Trials (DCTs), a trend accelerated by the COVID-19 pandemic. DCTs have quickly become a central trial model, enabling remote participation through medical technologies like wearable devices and mobile health platforms. Decentralised Trials not only expand the breadth of data collected but also enhance patient-centricity by making trials more convenient and accessible. As a result, sponsors are increasingly partnering with Contract Research Organisations (CROs) that provide full DCT support, including remote monitoring, home-based care, and seamless digital platforms for real-time data capture.
In parallel, the industry’s focus on patient-centricity continues to grow, with more emphasis on designing studies that align with patients' needs and preferences. This trend, coupled with the anticipated rise in DCTs throughout the remainder of 2024 and into 2025, broadens the demographic diversity of trial populations and produces outcomes more representative of the general population. Moreover, the upcoming ICH E6(R3) guidelines revision, set to take effect in 2025, will reinforce these shifts. The new guidelines will encourage flexible, risk-based trial designs, integrating decentralised approaches and prioritising patient input. By actively engaging patients in trial design and decision-making, studies will become more aligned with patient priorities, potentially speeding the acceptance and adoption of new therapies while improving trial efficiency and data integrity.
Integration of Artificial Intelligence (ML) and Machine Learning (ML)
There has been a growing trend in the industry where companies are exploring various ways to adopt and integrate AI and ML technologies into different phases of clinical trials. For example, in Phase I oncology trials AI is being used to identify biomarkers that predict patient response to treatment, enabling more precise and targeted therapies. In Phase III cardiology trials, ML models are being applied to analyse large-scale patient data, identifying patterns that can improve the accuracy of efficacy results. The goal is to enhance data analysis capabilities and improve both the efficiency and accuracy of the trials.
AI and ML are more commonly being used to streamline trial design, recruitment, and operational robustness. By 2024, AI tools can already analyse thousands of previous protocols and results, providing real-time, evidence-based advice during study design. AI-driven platforms such as PhaseV are optimising patient selection, data integration, adaptive trial design and protocols, significantly reducing trial timelines.
Machine Learning algorithms have advanced clinical trial outcomes to a whole new level, as it provides the ability to predict patient outcomes, identify potential adverse events more quickly, and optimise dosing regimens. This not only enhances trial efficiency but also enables more personalised medicine, allowing treatments to be tailored to each patient's unique characteristics. Both AI and ML tools hold the potential to reduce costs and shorten timelines, making clinical trials more accessible and scalable.
The Growing Role of Real-World Evidence in Modern Clinical Trials
Real-World Evidence (RWE) has developed from a novel approach to a core element of clinical trials in 2024. By utilising health data from sources like electronic health records (EHRs), insurance claims, wearable devices, patient registries, and observational studies, RWE provides valuable insights into drug effectiveness, safety, and treatment patterns across diverse populations. With rising trial costs and the growing number of rare disease studies, RWE offers sponsors an essential methodology to complement randomised controlled trials (RCTs). Regulatory bodies such as the FDA are becoming more accepting of Real-World Data (RWD) to support drug approvals, particularly in challenging areas like oncology and rare diseases, where traditional patient recruitment is difficult.
In 2025, we expect regulatory authorities to further embrace RWE to complement conventional clinical trial data, streamlining the approval process and enhancing post-market surveillance. CROs are now expected to have advanced RWE capabilities, incorporating EHRs, patient registries, and other RWD sources into trial design and data collection. This shift will also fostering stronger collaborations between Sponsors, CROs, medical device companies, and healthcare providers, promoting a more integrated approach to both research and patient care.
More Novel Trial Designs
The increases in the use of RWE, observational studies and advancements in technology to capture more real-time data, is driving the need for more flexible and adaptive trial designs. The industry is moving away from the gold standard of RCTs in favour of more bespoke trial designs that can be better suited to specific situations.
One significant limitation of RCT designs is the inability to adjust treatment allocation probabilities, whereas adaptive clinical trial designs, and especially response adaptive randomisation (RAR) designs, offer the flexibility to change treatment allocation based on observed performance. If one treatment is seen to outperform others, this type of trial design can allow patients to be allocated to the better performing treatment halfway through trial. This can only be beneficial for the patient and often for the drug company too.
These adaptive designs are often more efficient, informative, and ethical than traditional fixed designs, making better use of resources, enhancing patient outcomes, and potentially requiring fewer participants.
Another promising development in early-phase clinical trials is the introduction of Bayesian Optimal Interval (BOIN) designs. BOIN designs provide a more efficient framework for dose-finding trials by allowing for flexible decision-making, ensuring that the optimal dose is found more quickly and with fewer patients.
In addition, regulatory initiatives like Project Optimus are encouraging a shift away from traditional dose-escalation designs that rely on identifying the Maximum Tolerated Dose (MTD). This approach, historically used in oncology trials, often prioritises higher doses that maximise efficacy, but also come with significant toxicity risks. Project Optimus is pushing for dose optimisation strategies that put patient safety and long-term outcomes at the forefront, which could change the way dose-finding studies are designed.
RAR, BOIN designs, and initiatives like Project Optimus represent just a few of the novel approaches that move beyond the RCT gold standard. As trial methodologies continue to evolve, we can expect to see more of these novel designs in the near future.
The Expansion from Risk-Based Monitoring (RBM) to Risk-Based Quality Management (RBQM)
Over the past few years, we’ve seen a notable shift in the industry, moving on from Risk-Based Monitoring (RBM) to Risk-Based Quality Management (RBQM), which is a more comprehensive approach. The ICH GCP E6(R2) Addendum initially drove the adoption of a risk-based approach to clinical trials, and while RBM was already gaining traction prior to the release of these guidelines in 2017, the industry has since progressed to apply these risk-based principles across all aspects of trial management, not just monitoring.
What Does a Risk-Based Approach Look Like Today?
A modern risk-based approach includes several key components:
- Risk Assessment: Conducted during pre-study start-up and continuously reassessed throughout the trial to identify and mitigate potential risks.
- Protocol and Investigational Plan: A well-designed and detailed protocol, based on the risk assessment, ensures the trial is designed to minimise risks and optimise data quality.
- Risk-Based Monitoring Plan: While monitoring remains important, it is now integrated within a broader RBQM framework, focusing on overall trial quality, patient safety, and regulatory compliance.
These components work in tandem to implement risk controls and take corrective actions as required during the trial, all of which are documented in an auditable log. This broader remit has led the industry to develop on from RBM to RBQM, which incorporates a more holistic view of trial oversight.
Since 2017, the role of RBQM has continued to expand due to several factors:
- ICH E6 (R3) Guidelines: Once finalised in 2025, these new guidelines will further highlight quality by design (QbD) and risk-based principles at every stage of the clinical trial process, strengthening the integration of risk management throughout trial planning and execution.
- Increased Use of Technology and Data Analytics: Advanced analytics, machine learning (ML), and real-time data collection tools are now essential for identifying risks earlier and more accurately. These technologies enable continuous monitoring and decision-making throughout the trial, improving both efficiency and data quality.
- Decentralised and Hybrid Trials: The acceleration of decentralised and hybrid trial models has necessitated new risk assessment strategies. RBQM frameworks now account for the complexities of remote data collection, trial oversight, and patient engagement, ensuring high data quality in diverse trial environments.
- Vendor and Supplier Oversight: As clinical data is increasingly collected from multiple external sources, such as CROs and other third-party vendors, maintaining control over data quality has become critical. RBQM places a strong focus on ensuring that sponsors remain accountable for the quality of the data, even when outsourced, and that vendors comply with risk management protocols.
The ICH GCP E6(R2) Addendum, along with the anticipated updates in E6(R3), puts onerous on sponsors, vendors, and suppliers to work together to implement risk-based strategies across all phases of trial management. By adopting RBQM, organisations are not only better equipped to manage modern trials, but are also positioned to enhance efficiency, reduce costs, and ensure that patient safety and data integrity remain at the forefront of clinical trials.
Rare Diseases and Gene Therapy: Accelerating Innovation
The rare disease sector is experiencing significant growth, with over 700 gene and cell therapy applications under review by the FDA as of 2024. Advances in gene editing technologies, such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) are driving the development of new, targeted therapies that hold the potential to treat rare and previously untreatable conditions.
The global gene delivery technologies market, estimated at USD 4.91 billion in 2023, is anticipated to expand at a compound annual growth rate (CAGR) of 11.4% between 2024 and 2030. This growth is largely fueled by the rising incidence of genetic disorders and cancers, creating a demand for effective gene therapies. Moreover, advancements in biotechnology and molecular biology have introduced new gene delivery methods, further accelerating the market’s expansion.
Gene therapy trials, particularly in the rare disease space, often require innovative regulatory pathways and specialised trial designs due to the small patient populations and the complexity of the treatments. These trials also face significant challenges in patient recruitment and the logistics of gene therapy manufacturing.
By March 2023, there were 100 approved gene, cell, and RNA therapies available globally, with an additional 3,700 therapies undergoing clinical and preclinical trials. This strong pipeline of therapies indicates promising prospects for the market’s future. The continuous innovation and application of these treatments are expected to drive significant growth in the gene delivery technologies sector in the years ahead.
As a result, Contract Research Organisations (CROs) with expertise in rare disease trials are becoming indispensable. Their ability to handle complex regulations, implement adaptive trial designs, manage the intricacies of manufacturing, and recruit hard-to-find patient populations makes them essential partners for the likes of biotech companies.
Industry Emphasis on Sustainability and Diversity
Sustainability is becoming an important focus for both sponsors and CROs as the clinical trials industry evolves. The healthcare industry is responsible for generating 5.2% of global greenhouse gas (GHG) emissions, equating to approximately 2.7 billion metric tons of CO2. This amount exceeds the emissions produced by the aviation sector, which stands at 2.5%, and is comparable to the emissions of 720 coal-powered plants. Clinical trials alone contribute up to 100 million tons of CO2, which is roughly equivalent to the emissions from 27 coal-fired power plants or the annual electricity usage of 19.5 million U.S. households. The World Health Organisation (WHO) emphasises that to safeguard public health, healthcare systems must not only strengthen climate resilience but also work to lower their own carbon emissions.
The shift toward digital and decentralised trials is helping reduce the environmental footprint of research by minimising patient travel, cutting down on paper use, and lowering energy consumption in clinical operations. CROs that provide environmentally friendly trial solutions such as EDC’s for paperless data capture, remote patient monitoring, and virtual site visits, are expected to see rising demand as companies focus more on sustainability. These practices not only address environmental concerns, but also reflect a broader push for corporate responsibility within the industry.
Diversity in clinical trials is equally a top priority for 2024 and beyond. Regulatory bodies like the FDA have introduced new guidelines aimed at ensuring clinical trials better represent diverse populations. Historically, trials have often lacked participation from racial minorities, women, older adults, and other underrepresented groups, leading to gaps in the understanding of how therapies perform across different demographics. Sponsors and CROs are now tasked with implementing targeted strategies to improve diversity in patient recruitment, ensuring that trial data is reflective of the real-world populations these therapies will serve. Greater diversity in clinical trials will not only improve the accuracy of results but also foster equity in healthcare by ensuring therapies are safe and effective for all patient groups.
Together, the emphasis on sustainability and diversity is shaping a future where clinical trials are not only more efficient and eco-conscious but also more inclusive and representative of the global population.
The Progression of Functional Service Provision (FSP) and Hybrid Outsourcing Models
Looking back to 2023, Functional Service Provision (FSP) remained a key outsourcing model between sponsors and service providers. Traditionally, FSP involved vendors providing dedicated functional teams to support specific services, offering sponsors flexibility while maintaining some internal control over trial management. The 2023 trend showed that larger sponsors split their outsourcing between FSP and full-service models at a ratio of 48% to 52%, while smaller sponsors leaned more towards full-service outsourcing, with only 36% opting for FSP. At the time, it was anticipated that smaller companies would begin adopting a more balanced, 50/50 approach between FSP and full-service outsourcing, creating more opportunities for CROs to position themselves as FSP specialists.
As we reflect on 2024 so far, we’ve seen this prediction materialise, with smaller biotech companies increasingly adopting FSP models. This shift has been driven by the need for flexibility, cost control, and the ability to scale services quickly, which FSP models can provide. At the same time, hybrid models—which combine elements of both FSP and full-service outsourcing—have gained popularity. These hybrid approaches allow sponsors to tailor their outsourcing strategies to specific needs, giving them the agility of FSP while still benefiting from the comprehensive support of full-service partnerships. As a result, CROs are modifying their service offerings to cater to these dual demands, becoming experts in hybrid models that can scale between FSP and full-service as needed.
Looking ahead to 2025, it is expected that this hybrid outsourcing model will continue to grow in adoption, especially among smaller biotech and mid-sized pharmaceutical companies. These organisations will likely seek long-term relationships with CROs that can offer both specialised functional services and full-service capabilities, enabling them to optimise their clinical trial strategies as their needs evolve. For CROs, this presents an opportunity for larger, more integrated contracts and ongoing, repeat business, as sponsors increasingly centralise their clinical operations with trusted partners who understand their processes and long-term goals.
In terms of revenue distribution, while FSP continues to be a significant portion of CROs' revenue, the lines between FSP and full-service outsourcing are blurring. In 2024, we anticipate that the balance will remain relatively stable, but with hybrid models taking a larger share of the market. As smaller providers move towards the anticipated 50/50 split between FSP and full-service models, CROs that can offer expertise in both areas, as well as flexible hybrid solutions will be well-positioned.
Outsourcing Trends and the Role of Biotech and Small-to-Mid-Sized Pharma
As the optimisation of clinical trials continues, outsourcing expenditure is predicted to grow, with an increasing focus on strategic partnerships between sponsors and Clinical Research Organisations (CROs). In 2024, the expenditure split between in-house activities and outsourced services highlights the increasing reliance on external expertise. As of 2023, over 49% of clinical trial expenditures were allocated to outsourced services, with larger sponsors still maintaining a portion of trial operations in-house, but gradually leaning more on CRO partnerships.
While the top 20 largest pharmaceutical companies continue to invest heavily in in-house resources, it's the biotech and small-to-mid-sized pharma companies that are expected to drive the most significant growth in the CRO market. These smaller sponsors often benefit from outsourcing due to the added flexibility and access to specialised expertise that it provides, allowing them to efficiently manage clinical trials while focusing on their core capabilities. This sector’s preference for outsourcing is reflected in the double-digit growth rate of CRO services in recent years, particularly among smaller firms.
The push from biotech and smaller pharma companies has also led to a more competitive market for CROs, with these sponsors often seeking long-term partnerships with flexible, scalable services. CROs that can offer a blend of FSP, full-service, and hybrid outsourcing models are poised to capture the largest share of this growth, particularly as smaller sponsors centralise their clinical operations to reduce costs and enhance efficiency.
Looking forward, it is anticipated that biotech and small-to-mid-sized pharmaceutical companies will account for a larger proportion of CRO business compared to the top 20 largest pharma’s, who continue to maintain more robust in-house capabilities. For CROs, this presents a significant growth opportunity as these smaller companies increasingly outsource their clinical operations to trusted, specialised partners.
The Growing Priority of Cybersecurity and Data Privacy
As decentralised trials and wearable technologies become more prevalent in clinical research, the volume of data being collected and transmitted has significantly increased. With this rise in data streams comes heightened concerns around cybersecurity and data privacy. Ensuring the security of sensitive patient information is more critical than ever, particularly as clinical trials rely on digital tools and remote data collection.
Regulatory bodies, such as the GDPR in Europe and HIPAA in the United States, impose strict guidelines on the management and protection of personal health data. It’s important to partner with CROs that have robust data privacy and cybersecurity measures in place, as they’re better equipped to meet these regulatory standards. Sponsors are increasingly seeking CROs with proven in-depth expertise in secure data handling to mitigate risks of data breaches, non-compliance, and reputational damage.
As we move ahead into 2025, the emphasis on data integrity and patient privacy is expected to grow, with the ability to safeguard personal health data while managing decentralised trials being a regulatory requirement.
Automation of Clinical Trials
The automation of routine trial tasks is optimising the way clinical operations are performed, reducing manual workloads and increasing efficiency for both CROs and sponsors. So far in 2024, we have seen a growing adoption of Robotic Process Automation (RPA) and AI-driven solutions to streamline key processes such as site management, patient follow-up, and report generation. These tools are designed to handle repetitive tasks that were traditionally managed by human teams, allowing for faster, more accurate execution and freeing up valuable resources for more strategic activities.
Automation is also being applied to clinical data management, with tools such as Study Data Tabulation Model (SDTM) automating the process of organising and formatting clinical trial data for submission to regulatory authorities. These automation tools ensure that data is collected, standardised, and submitted in compliance with regulatory requirements, reducing the risk of errors and speeding up the submission process.
AI-based solutions are further optimising trial operations by enabling predictive analytics, risk-based monitoring, and adaptive trial designs. For example, AI can analyse patient data in real-time to identify potential issues or deviations, allowing CROs and sponsors to proactively manage trials and make data-driven decisions more quickly.
As 2025 fast approaches, the automation of trial operations is set to reduce operational burdens, lower costs, and improve overall trial efficiency. The need to invest in advanced automation technologies or partner with technology providers is expected to become a necessity for modern clinical trials.
The Future of Clinical Trials
As we have seen over the last few years, the future of clinical trials is evolving rapidly, with trends like decentralised trials, technology integration, Real-World Evidence, and a heightened focus on patient-centricity shaping the way. The adoption of automation in clinical trials is set to become an essential part of the process in modern trials to drive efficiency, reduce manual tasks and enhance data accuracy. Sustainability and diversity we predict will continue to be a key driver in the industry, with new regulations pushing for more eco-conscious and inclusive trials. Not only are these innovations improving patient engagement and streamlining operations, but they are also allowing for more personalised treatment approaches.
As cybersecurity concerns grow, CROs with strong data protection capabilities will be vital. Adapting to these trends will be key for CROs and sponsors looking to lead in the future of clinical trials.
Partner with Quanticate for Specialist Clinical Solutions
At Quanticate we specialise in collecting, analysing and reporting clinical and real world data, across biostatistics, statistical programming, biostatistical consultancy, clinical data management, pharmacovigilance, medical writing and regulatory submission review services. Whatever the data, we’ve got you covered. If you are looking for support on any of the topics highlighted in this blog please Submit a RFI and member of our team will be in touch with you shortly.
