Opportunities in clinical trial innovation and entrepreneurship

A friend of mine is considering entering a clinical trial. She is confused, apprehensive, and desperate to find a ray of hope for her deteriorating condition. She doesn't know which questions to ask and is scared.

Clinical trials answer questions about how well a treatment or therapy works and/or how safe it is by monitoring its effect on people (often through bloodwork and other tests, as well as checking for symptoms and side effects). The people in clinical trials are called clinical trial participants and are volunteers who may be healthy or have a specific illness or condition.

Clinical trials use different design features, which are in place to help reduce the influence of external factors (such as bias, assumptions, or expectations) on the clinical trial results.

The DISRUPT-DS roundtable, which brings together data science leaders from large pharmaceutical companies, aims to be a forum for sharing experiences and networking, for shaping industry-level topics and for amplifying the role of data science across pharmaceutical R&D.

Some of the problems with clinical trials are:

• Slower patient recruitment than required, which can delay the start and completion of trials.
• Compliance with several rules and regulations, which can be complex and costly to follow.
• Managing multiple sites, which can pose challenges in communication, coordination, and quality control.
• Preventing professional patients, who enroll in multiple trials for financial gain and may compromise the validity of the results.
• The risk of minor or major discomfort, complications, or harm to the participants, which may require medical attention or intervention.

PPD, Thermo Fisher Scientific’s clinical research business, surveyed more than 150 decision-making leaders at pharmaceutical and biotech companies around the globe to collect key insights on the state of the evolving drug development industry. Drug developers shared the disruptive impact of the COVID-19 pandemic and ever-changing industry landscape, which resulted in increased challenges in clinical trial execution.

Respondents shared sentiments around current challenges within their organizations and the broader industry. The top challenge varied depending on company size: for biopharma companies, the top challenge was trial complexity, while for biotech companies, the top challenges were funding and elongated study startup times.

Clinical trial obstacles are different for medical devices, in -vitro diagnostics, vaccines, and the emerging use of AI as a medical device.

Reforming clinical research is “a big topic of interest in the industry”, says Lisa Moneymaker, the chief technology officer and chief product officer at Saama, a software company in Campbell, California, that uses AI to help organizations automate parts of clinical trials. “In terms of applications,” she says, “it’s like a kid in a candy store.”

A recent survey conducted by Advanced Clinical and StuffThatWorks sheds light on the positive inclination of respondents towards participating in clinical trials. The primary reason cited for non-participation was lack of awareness, which underscores the need for improved communication strategies to both patients and healthcare providers. The survey, completed by 679 respondents, of whom 98% were patients and 2% were caregivers, provided insights across a diverse range of diseases and geographical locations, emphasizing the broad applicability of patient-centered approaches.

Here are six things to know before entering a clinical trial.

The National Institutes of Health (NIH) is investing approximately $30 million in total over fiscal years 2024 and 2025 to pilot a national primary care research network that integrates clinical research with community-based primary care. The new initiative called Communities Advancing Research Equity for Health – or CARE for Health – seeks to improve access to clinical research to inform medical care, particularly for those in communities historically underrepresented in clinical research or underserved in health care. Informed by the health needs of these communities, CARE for Health will help to grow an evidence base that contributes to improved patient outcomes, provide communities access to the best available scientific research and expand opportunities to participate in clinical trials and studies. NIH Director Monica M. Bertagnolli, M.D., lays out her vision for CARE for Health in a Science Editorial(link is external).

There is growing evidence that a substantial proportion of trials do not generate informative results due to suboptimal design or underrecruitment.1 For every funded study that is uninformative due to poor design, the opportunity is lost to fund an alternative study. This leads to fiscal waste but, more importantly, it represents an ethical issue: every uninformative study has put undue burden on patients who willingly contributed for zero benefits, leading to a negative benefit-risk ratio. Patients and healthcare professionals should be more involved in the design and execution of clinical trials.

This represents another opportunity to practice primary care entrepreneurship and is another missing piece of medical education and training.

Patient participation in clinical trials has been underestimated. The overall estimated patient participation rate to cancer treatment trials was 7.1%. Patients with cancer participated in a wide variety of other studies, including biorepository (12.9%), registry (7.3%), genetic (3.6%), QOL (2.8%), diagnostic (2.5%), and economic (2.4%) studies. Treatment trial enrollment was 21.6% at NCI-designated comprehensive cancer centers, 5.4% at academic (non–NCI-designated) comprehensive cancer programs, 5.7% at integrated network cancer programs, and 4.1% at community programs. One in five patients (21.9%) participated in one or more cancer clinical research studies.

2024 has been a year of exciting developments for wearables. More than two in five Americans (44%) now own wearable devices. Smart rings are on the rise and stretchable biosensors are opening doors for new form factors. Wearables are increasingly relevant to behavior change, clinical trials, and patient safety. Clinicians are warming up to their roles in patient health conversations, and regulators are expanding consumer guidance and oversight.

Clinical trial innovation and entrepreneurship are rapidly evolving fields that present numerous opportunities for individuals and organizations. As the healthcare industry advances, new technologies, methodologies, and approaches are transforming clinical research. Here are some key areas where innovation and entrepreneurship are flourishing:

1. Decentralized and Remote Trials

• Opportunity: The shift toward decentralized clinical trials (DCTs) enables patients to participate remotely, improving access and reducing costs.
• Entrepreneurial Potential: Entrepreneurs can develop platforms that enable remote monitoring, data collection, virtual visits, and patient engagement.
• Innovation Focus: Technologies like wearable devices, telemedicine, and mobile apps can be integrated to collect real-time data and enhance patient adherence.

2. Artificial Intelligence (AI) and Machine Learning (ML) in Clinical Trials

• Opportunity: AI and ML can optimize trial design, patient recruitment, and data analysis.
• Entrepreneurial Potential: Companies can create AI-powered tools to predict patient outcomes, identify optimal trial designs, and detect adverse events faster.
• Innovation Focus: Predictive analytics for patient recruitment, personalized medicine approaches, and automating routine administrative tasks.

3. Data Integration and Real-Time Analytics

• Opportunity: Combining diverse datasets (e.g., genomic, electronic health records, and trial data) allows for better decision-making and faster results.
• Entrepreneurial Potential: Developing platforms that integrate various data sources and provide real-time analytics to researchers, sponsors, and regulators.
• Innovation Focus: Streamlining data pipelines, ensuring data privacy and security, and creating dashboards for efficient monitoring.

4. Patient-Centric Trial Design

• Opportunity: Trials that consider the needs, preferences, and experiences of patients can improve recruitment and retention.
• Entrepreneurial Potential: Creating tools or services that enhance patient recruitment by focusing on patient engagement, education, and retention strategies.
• Innovation Focus: Personalized patient journeys, patient-reported outcomes, and tools to simplify trial participation.

5. Biomarker and Companion Diagnostics Development

• Opportunity: Incorporating biomarkers into trials allows for better-targeted treatments, leading to more efficient trials and more accurate results.
• Entrepreneurial Potential: Developing novel biomarkers or companion diagnostics, which can help tailor treatments to specific patient subgroups.
• Innovation Focus: Advancements in genomic, proteomic, and other “omics” technologies, as well as regulatory pathways for biomarker approval.

6. Clinical Trial Supply Chain Innovation

• Opportunity: Optimizing the clinical trial supply chain, including drug distribution, temperature-sensitive logistics, and inventory management, can reduce costs and enhance efficiency.
• Entrepreneurial Potential: Developing innovative supply chain solutions using blockchain, AI, and other technologies to improve transparency and reduce inefficiencies.
• Innovation Focus: AI-powered logistics management, real-time tracking of supplies, and automation of manufacturing and distribution processes.

7. Regulatory and Compliance Technology

• Opportunity: As regulatory requirements evolve, companies need technology solutions to streamline compliance with regulations like FDA, EMA, and GxP.
• Entrepreneurial Potential: Entrepreneurs can create software tools to manage regulatory submissions, clinical trial documentation, and audit trails.
• Innovation Focus: AI and machine learning for regulatory intelligence, automated document management, and enhancing regulatory submission workflows.

8. Wearable Devices and Digital Biomarkers

• Opportunity: Wearables are becoming more integral in monitoring patients in real-time, providing data on heart rate, movement, sleep, and other vital signs.
• Entrepreneurial Potential: Developing wearable devices that capture digital biomarkers for chronic disease management, safety monitoring, or efficacy analysis.
• Innovation Focus: Wearables for continuous monitoring of physiological parameters, real-time health data analysis, and integration with clinical trial data platforms.

9. Blockchain for Clinical Trials

• Opportunity: Blockchain can enhance data security, transparency, and traceability in clinical trials, ensuring the integrity of clinical data.
• Entrepreneurial Potential: Startups can explore the use of blockchain for secure patient data storage, trial data sharing, and audit trails.
• Innovation Focus: Decentralized data storage, smart contracts, and secure data exchange platforms.

10. Global Trial Expansion and Access

• Opportunity: Expanding clinical trials to underrepresented global regions can help diversify patient populations and accelerate drug development.
• Entrepreneurial Potential: Developing platforms to facilitate global trial management, addressing local regulatory requirements, and overcoming logistical barriers.
• Innovation Focus: Cross-border trial coordination, local partnerships for recruitment, and ensuring compliance with global standards.

11. Trial Recruitment and Retention Technologies

• Opportunity: Efficiently recruiting and retaining patients in clinical trials is a major challenge. New technologies can help.
• Entrepreneurial Potential: Startups can create patient recruitment platforms that use AI and social media tools to identify and engage eligible patients.
• Innovation Focus: AI-driven patient matching, social media recruitment strategies, and virtual trial management to improve retention.

12. Sustainability in Clinical Trials

• Opportunity: As the industry looks to reduce its environmental impact, sustainable trial design and operations are becoming a priority.
• Entrepreneurial Potential: Developing environmentally friendly solutions, such as reducing waste, minimizing travel for clinical visits, or offering virtual participation.
• Innovation Focus: Green logistics, virtual visits, and sustainable trial infrastructure.

13. Patient-Reported Outcome Measures (PROMs)

• Opportunity: The growing importance of capturing real-world outcomes through patient self-reports offers a novel approach to clinical trials.
• Entrepreneurial Potential: Creating innovative tools for patients to report symptoms, side effects, and quality of life in real-time.
• Innovation Focus: Mobile apps, user-friendly interfaces, and real-time analytics of PROM data.

14. Adaptive Trial Design

• Opportunity: Adaptive trial designs allow for modifications during the trial based on interim results, increasing flexibility and efficiency.
• Entrepreneurial Potential: Developing platforms and statistical tools to support adaptive trial methodologies, ensuring that trials are conducted with maximum efficiency.
• Innovation Focus: Advanced statistical models, trial simulation tools, and automated decision-making support.

Conclusion:

Clinical trial innovation and entrepreneurship offer a wealth of opportunities in fields like data integration, patient engagement, AI, decentralized trials, and supply chain management. As the healthcare landscape continues to evolve, the demand for new solutions to make clinical trials more efficient, patient-centric, and cost-effective is growing, making this an exciting space for innovation and entrepreneurial ventures.

If you are considering entering a clinical trial, be sure you understand the risks and benefits before making this difficult decision and take advantage of information and educational resources like the NIH database of clinical trials.

Arlen Meyers, MD, MBA is the President and CEO of the Society of Physician Entrepreneurs on Substack