Clinical trials are the gold standard for testing the effectiveness of treatment interventions for both common and rare conditions, yet the time and financial investment required to find and recruit eligible patients can be cumbersome. That’s why it’s important for clinical researchers to become familiar with some of the latest trends in research that can provide feasible alternative methods to answer some of the field’s most pressing questions, according to Sagar Nigwekar, MD, MMSc, co-director of the Kidney Research Center at Massachusetts General Hospital and co-director of Harvard Medical School’s Global Clinical Scholars Research Training program. Thanks to advances in technology and artificial intelligence, he points out that a host of new research methods and tools are worth exploring. 

“Investigators need to think about creative ways to still get to the same answer that a clinical trial may provide without having to go through all of the burdensome processes,” says Nigwekar. Below, he shares an overview of some of the latest clinical research methods that can be used to increase the understanding of how well different treatments work for different populations.

Clinical Research Trends

Trial emulation: “Trial emulation is a systematic approach to designing an observational or simulated study to quantify the effect of a treatment method on a clinical outcome,” Nigwekar says. This means that, rather than conducting a full clinical trial, investigators can collect existing data and apply it to measure the effectiveness of a medicine. 

For example, when looking at the causal effect of a kidney treatment, utilizing trial emulation would require comparing the outcomes of two groups of patients—one group that uses the treatment and one that doesn’t—to identify the differences between these two groups that could be attributed to the medication use. The findings could be used to supplement the results of traditional clinical trials.

“Instead of having to do five trials, maybe we can do only two or three and then use the simulation for the remaining two. This allows us to take the collective evidence to see how it reinforces the overall clinical trial findings,” he adds.

Nigwekar explains that trial emulation provides a valuable approach for newer researchers who don’t have the resources to conduct a large, randomized, controlled trial but who still have a research question to answer. “When a full-scale clinical trial is not feasible for a variety of reasons, alternative approaches like this can still give them a reasonably accurate answer,” he says.

Decentralized trials: Decentralized clinical trials enable patients to complete study requirements remotely, often from the convenience of their own home, instead of having to visit a centralized clinical trial site. This model, which became an accepted practice during the pandemic, continues to make it possible for investigators to recruit a more diverse group of participants, including patients who are juggling careers and families, people living in remote areas where they don’t have access to clinical trial sites, and the elderly and home-bound populations who might be unable to travel to take part. 

Nigwekar notes that providing such flexibility in clinical trials can be essential in understanding the effect of medications on different populations and in addressing some of the inequities that exist today in clinical trial participation.

Digital health technologies: Technology has been advancing in leaps and bounds recently, leading to an array of new digital health tools that can make clinical trials easier and more effective. Some of the latest tools include at-home blood pressure measurements, activity trackers, glucose monitors, and other sensors that enable people to monitor their own vital signs and other health measurements in the course of their daily activities and to share the results remotely with clinical trial staff. 

“Improvements in technologies are enabling people to collect and transfer data more seamlessly, making it easier for them to take part in clinical trials,” Nigwekar stresses. Such technology also helps to facilitate decentralized clinical trials.

“These tools also provide a way for clinical trial staff to get a more realistic picture of participants’ daily lives and to see how treatments work, or don’t work, in the real world,” he adds. This can provide a clearer picture of a treatment’s effectiveness while reducing time and minimizing costs at the same time.

Adaptive trials: While clinical trials have traditionally been organized into three steps (design, implementation, and analysis of findings), this framework doesn’t allow for adjustments during the process if conditions change. Nigwekar explains that adaptive trials incorporate flexibility into the plan by having potential changes already written in that they can activate if needed. Investigators periodically review the data and can make pre-specified changes as needed without compromising the validity and integrity of the clinical trial. He also stresses that this is different from unplanned changes that researchers might request during a traditional clinical trial, as an adaptive trial anticipates potential needs and how the design might need to be adapted and has them approved and ready to implement in advance.

Rare disease research: It’s particularly difficult to recruit people with rare diseases for clinical trials, as finding and addressing these populations can be especially challenging. Artificial intelligence is providing new ways to identify people with rare diseases, and is making it possible to engage them in order to better understand their condition and to help find targeted treatments. 

Staying Focused on Research Goals

For clinical researchers looking to advance their careers in new directions or to take on leadership roles, Nigwekar says that staying on top of these and other trends in clinical research can be essential to position them for success, both now and in the future. With any research efforts, he notes, it’s also important for investigators to keep their eye on the end goal.

“Most people who want to go into clinical research want a chance to not just help their patients, but also answer pressing questions about how to improve the health of populations,” he says. The latest tools and methodologies can help them take questions from the bedside and look at them from a broader perspective to glean insights that they can take back to the patient.

However, Nigwekar says that no matter what methods and tools investigators use, it’s important to remember that clinical research is an ongoing process. “Most researchers are not going to just go and invent a new, magical treatment. They will face many stumbling blocks and disappointments on their way to success, so it’s important to keep looking to the future and taking small steps toward bigger goals,” he stresses.