The Evolution and Future of Clinical Trials
Clinical trials are one of the most critical steps in the regulatory approval process for biopharma treatments and therapies as well as medical devices and procedures. Like most breakthroughs in science and healthcare, advances in technology have dramatically changed clinical trials and provided solutions for some of the most pressing problems.
Participants in clinical trials used to have to live near research sites or be willing to travel. That led to participant groups that often included a disproportionate number of white patients. In addition, trials testing treatments for rare diseases could be challenging to complete because there were not enough eligible participants near trial sites to conduct meaningful trials.
New advances make it possible for the clinical research industry to solve those problems. Trial volunteers can now participate remotely, using digital technology to collect and report trial data.
Read on to learn more about how technology is shaping the future of clinical trials.
What is a Clinical Trial?
Clinical trials are controlled tests on human subjects designed to ensure that new drugs or treatments work as intended and do not cause harmful side effects. Researchers conduct clinical trials for several reasons, including:
- To determine if a new therapy, treatment, device, or procedure is safe and effective for people to use
- To study different ways to use standard or approved treatments so they can be more effective, easier to use, or cause fewer side effects
- To learn how to safely use an approved treatment in a population not included in the initial clinical trial, such as children
Are there different types of clinical trials?
There are two types of clinical trials, observational and interventional. In observational trials, researchers collect real-world data to track and measure specific outcomes. Observational trials are often used to test the effectiveness of medical devices, such as remote monitoring or wearable technology.
In interventional trials, participants are randomly assigned specific interventions—the treatment being studied, a placebo, or no intervention—so researchers can compare the results. Interventional trials are often part of drug development.
Clinical trial design and protocols
Protocols describe how a clinical trial should be conducted to help ensure that trials are accurate, reliable, and can be duplicated. Clinical trials are structured according to protocols that describe:
- Patient participation and who is eligible for the study
- The number of people included
- How to eliminate research bias with control groups or other techniques
- Schedules of tests and procedures
- The therapies involved and their dosages
- The data collected and how it will be used
- The length of the study
- What researchers hope to learn
Protocols benefit trial operators because they detail precisely how a clinical trial should go from start to finish.
What are the phases of a clinical trial?
According to the FDA, most clinical trials have four phases:
- Phase I includes 20 to 80 participants and studies the medication’s effects on people with no underlying health conditions. Phase I helps determine the highest dose humans can take without serious side effects and how their bodies react.
- Phase II involves several hundred participants with the condition the new medication is meant to treat. Investigators monitor participants for months or years to measure the effectiveness and identify possible side effects.
- Phase III usually involves up to 3,000 trial participants and can last several years. This phase helps researchers evaluate how the new therapy works compared to existing medications.
Phase III trials are usually double-blind to help eliminate bias. Double-blind means neither the investigator nor participants know which medication each is taking. The FDA usually requires a phase III clinical trial and will usually approve a new therapy if trial results show that the treatment is at least as safe and effective as others already on the market.
- Phase IV takes place after the FDA has approved a new medication and allows investigators to gather more information about the medication’s long-term safety, effectiveness, and other benefits.
Are there international standards for clinical trials?
The International Conference on Harmonisation is a group of regulatory agencies from the European Union, Japan, and the U.S. that issue guidelines establishing clinical research standards.
In addition, the World Health Organization (WHO) issues international guidelines that regulate clinical trial registries. Registries are databases that let patients, healthcare professionals, researchers, and the public easily search for information about clinical studies. One such registry is ClinicalTrials.gov, a registry maintained by the U.S. National Institutes of Health (NIH).
How many clinical trials are conducted each year?
According to a WHO report, nearly 60,000 clinical trials were conducted worldwide in 2021. More than 12,900 of those took place in the United States. More than 671,000 clinical trials have been performed worldwide since the WHO started tracking them in 1999.
What are the advantages and disadvantages of participating in clinical trials?
Most clinical trials have guidelines about who can volunteer to participate. In some cases, participants may need to have a specific disease or medical condition and must qualify for the study. “Inclusion criteria” allow volunteers to participate in a clinical trial.
“Exclusion criteria” are factors that disqualify volunteers from participating. Among those criteria are age, gender, the type and stage of a disease, previous treatment, or other medical conditions.
What are the advantages of participating in a clinical trial?
People who volunteer to take part in clinical trials can see several advantages, including:
- They may receive medications or treatment that is only available through the trial
- The medication or treatment they get may be more effective than currently approved treatments or therapies
- They can help improve future medical treatments
- They may receive more frequent blood tests, scans, or other tests than normal
- They may have check-ups with a physician more often than usual
What are the disadvantages of participating in a clinical trial?
Participating in a clinical trial offers advantages but can also bring disadvantages for volunteers. They include:
- Medications or treatments they receive could cause severe or unexpected side effects
- There is a risk that experimental treatments or therapies could cause harm
- Participants may have to complete extra paperwork
- They may have blood tests, scans, or other cancer tests
- They may require more frequent check-ups, and each may take longer than usual
- Extra appointments could cost time and money for transportation or parking
How has technology impacted clinical trials?
Not long ago, participants had to be near the research sites if they wanted to participate and get access to treatments that could save their lives. That often led to a lack of diversity among patient populations represented in the trial.
A 2021 report by the Journal of the American Medical Association (JAMA) showed that in vaccine trials conducted between 2011 and 2020, nearly 79% of all recipients were white, compared to just 21% for all other races and ethnic groups combined.
Decentralized clinical trials (DCT) use technology to help create more diverse studies by allowing researchers to reach patients where they live. The COVID-19 pandemic helped accelerate the use of DCTs and allowed trials to continue when participants could not travel to a research site.
What are decentralized clinical trials, and how do they work?
Decentralized clinical trials use technology to help make trials more accessible to volunteers who could not otherwise participate in helping make trials accessible to a broader demographic. Decentralized trials can also improve patient recruitment and retention, generate greater engagement, increase diversity among participants, and lower the cost of running trials.
Decentralized clinical trials may be fully remote—a virtual trial—or they can use a hybrid approach that requires some on-site attendance combined with remote monitoring using:
- Remote monitoring and diagnostic technology
- Home health providers
- Local labs
- Electronic consents
- Direct-to-patient drug distribution
Among the advantages of decentralized clinical trials are:
- Allows more diverse and representative participant populations
- Easier participation for volunteers
- Fewer in-person study visits
- Passive data collection
- Removes barriers to participation, such as geographical factors, time, and travel.
In addition, decentralized trials provide more opportunities to conduct tests that cover potential treatments for rare diseases that were impossible because participants are geographically dispersed.
Decentralized clinical trials can present some risks to patient safety. For example, participants could be harmed if they improperly administer trial medications. Complex remote clinical trials could generate inaccurate trial data if participants do not fully understand the trial.
Finally, decentralized trials can burden patients because they must activate, charge, and wear digital sensors for several hours daily. It can also burden trial operators, who may have to provide technical support to participants and learn to use new technology to run the trial.
Hear what Claudia Berrón, Avantor’s senior vice president, clinical services, says about decentralized trials here.
What technologies make the future of clinical trials possible?
Innovative technology has already changed how clinical trials are conducted and will continue to improve trials in the years ahead. Mobile technology, for example, makes it easier for patients to participate in trials than ever before. That single advance means researchers can recruit participants from broader geographic areas, as well as ensure participants make up a representative population of races and ethnic groups.
Artificial intelligence (AI)
Artificial intelligence (AI) can help speed drug development and improve the efficiency of clinical trials. A study by the Massachusetts Institute of Technology (MIT) found that only 13.8% of therapies successfully pass clinical trials. Because clinical trials can cost up to $2 billion each, pharma companies have begun using AI to help increase the chances of approval by predicting results for untested treatments.
AI can help researchers analyze trial data and identify patterns more quickly than in the past.
Mobile health (mHealth)
Mobile and wireless technology—known collectively as mHealth— is one of the most critical factors in decentralizing clinical trials. According to the NIH’s National Library of Medicine, 185 clinical trials used mHealth between 2013 and 2015, and that number more than doubled in tests conducted between 2016 and 2018.
Rutgers University in New Jersey reported it used mHealth during the COVID-19 pandemic to cut the time needed for patient recruitment in half. The university could also recruit lower-income and underrepresented populations who could not previously participate in clinical trials and widen each trial’s geographic coverage to allow more diverse participants.
According to the NIH, mHealth can help clinical trials in several ways, including:
- Reducing the overall cost of trials
- Helping in patient recruitment and retention
- Making it more accessible for trial operators to provide comprehensive data on drug safety and efficacy
- Giving patients access to studies that are specific to their health needs
- Making trials more convenient for participants by reducing study visits
- Using online surveys and assessments
- Helping with patient education and consent requirements
- Encouraging retention and participant engagement through “gamification,” reminders, and online surveys
- Allowing participants to interact with each other for social and emotional support
Internet of medical things (IoMT)
The Alliance of Advanced Biomedical Engineering (AABME) says the internet of medical things (IoMT) is “an amalgamation of medical devices and applications that can connect to health care information technology systems using networking technologies.”
The IoMT can be divided into segments, including:
- The on-body segment includes wearables such as activity trackers, sports watches, and smart garments. Many of the devices are consumer products and not regulated by the FDA.
- The in-home segment includes remote patient monitoring (RPM) devices and telehealth virtual visits.
According to a report by the National Institute of Standards and Technology, big data “consists of extensive datasets—primarily in the characteristics of volume, variety, velocity, and/or variability—that require a scalable architecture for efficient storage, manipulation, and analysis.”
In clinical trials, big data can come from various sources, including wearable devices and the internet of medical things. When trial operators take advantage of big data, they can use it to predict outcomes, get quicker alerts for negative interactions, and interpret trial data more quickly and accurately.
Social media can be helpful in clinical trials, including recruitment, building awareness and increasing communications. However, it can also create issues such as privacy concerns, confidentiality, and the integrity of the trial.
A study published by the American Cancer Society says increasing enrollment in clinical trials can be the most significant benefit of social media. The same research identifies pitfalls related to privacy, confidentiality, and transparency issues that could allow sensitive health information to be made public.
Future trends in clinical trials
New technology is dramatically impacting clinical trials and how they are conducted. Those advances are helping remove geographic barriers for trial participants. That, in turn, allows researchers to increase diversity among trial participants and recruit more patients for trials testing treatments for rare diseases.
Technological advances also benefit participants by making the entire trial process more patient-centric. Operators can use technology to help create a better, more engaging patient experience.
The coming years will undoubtedly bring additional changes to clinical trials as technology advances. It is up to everyone who organizes and operates clinical trials and those who support their research to stay current with the state of the industry.
The growth of decentralized clinical trials is having an impact on every stage of the clinical workflow. Avantor is leading the way in ensuring that supply chain, as well as shipping and logistics, can meet the changing needs of clinical trials.
Find out how Avantor® can help optimize clinical trials. Read here.