Clinical Trials: Bringing Cancer Research to All Possible Participants
The COVID-19 pandemic accelerated the use of virtual interactions between health care providers and patients. Researchers conducting clinical trials also increased their adoption of telemedicine, which helped NCI-supported clinical trial enrollment rebound from a 40% drop early in the pandemic to 80% of prepandemic levels. Researchers now hope to use the telemedicine experience gained during the pandemic to improve cancer clinical trials moving forward.
NCI supports clinical trials at over 2,200 sites throughout the United States and around the world through networks such as NCI’s National Clinical Trials Network (NCTN), the NCI Community Oncology Research Program (NCORP), and others. These networks provide patients with access to highly trained scientists and physicians who study how to better prevent and treat cancer through carefully designed research studies. In addition, NCI funds clinical trials conducted by individual investigators or investigative teams.
While many companies and institutions around the world conduct cancer clinical trials to test new treatments, NCI also focuses on studies that industry does not. These include cancer prevention trials, trials testing behavioral interventions for cancer risk factors like smoking and obesity, de-escalation trials testing the effectiveness of using less treatment, and complex combination therapy trials using novel adaptive designs.
Clinical trials advance cancer prevention, screening, treatment, and survivorship, but challenges in planning and conducting clinical trials hinder even more progress for patients. For example, only a small percentage of all adults with cancer participate in clinical trials, and trials often do not adequately represent minority and underserved populations. This may lead to study results that fail to reflect health outcomes for the broad population with cancer. There are additional hurdles for pediatric patients due to the rarity of childhood cancers and the additional support children and families need throughout the trial process.
Added challenges include the exponential growth in complexity and expense associated with cancer clinical trials. Extensive and complex data collection imposes burdens on everyone involved in a clinical trial and is a major contributor to the cost of conducting clinical trials.
Clinical trial innovations that break down barriers to participation and simplify study design are on the horizon. In 2020, NCI released a strategic vision for clinical trials that conceives of a future in which clinical trials are flexible, faster, simpler, less expensive, more equitable, and higher impact. With additional research investments, the clinical trial enterprise will be able to:
- expand the use of telemedicine in clinical trials
- increase trial access for minority and underserved communities
- incorporate new enrollment and data collection approaches using modern, digital technology
- determine more quickly if a new intervention provides benefits to patients
Ending cancer as we know it includes reimagining the clinical trial enterprise so that clinical research is available to participants wherever they are. Participating in a clinical trial should bring hope for the future, not additional burdens to bear. Imagine joining a trial from your home or having your health monitored for a trial via wearable technology. There would be no need to take time off from work or travel to distant cities. With additional resources and collaboration, NCI has an opportunity to lead changes that will take advantage of the most compelling new scientific opportunities while assuring that the research benefits will be made available to all.
Leveraging the Power of Technology to Reach Patients Where They Live
Reaching participants in their communities will increase clinical trial access. Integrating telemedicine into clinical trials allows participants to talk to trial physicians or their health care providers without the added time and costs of traveling to a clinical trial site. To ease additional burdens of clinical trial participation, investments in telemedicine infrastructure will enable participants to sign consent forms online, receive medications in the mail or at local health care facilities, and undergo monitoring from afar. With support from the Cancer Moonshot℠, NCI is also funding research to enhance the engagement of patients in clinical trials and other research studies through technology and online networks.
A survey conducted at Sidney Kimmel Cancer Center at Thomas Jefferson University revealed that 99% of adult patients with cancer who responded were satisfied with their telemedicine visits, and 87% of them felt telemedicine provided the same care as an in-person visit. In addition to learning from real world experiences, NCI has awarded grants to further study how telemedicine has impacted cancer care during the COVID-19 pandemic.
Telemedicine is only one way modern technology is being used to bring clinical trials to more people. With the widespread use of computers and smartphones, society has embraced digital technology in many aspects of life, including health care. Digital tools, such as health-related mobile apps, patient portals, wearable devices, and electronic health record, provide a rich source of information for clinical trials and open new avenues of access.
According to the latest Health Information National Trends Survey (2020), 85% of US adults use health or wellness apps, and 80% would be willing to share health data from a wearable device with their health care providers. Overcoming the logistical and data quality challenges of extracting clinical trial data from electronic health records and remote technology devices would greatly improve clinical trials.
In the first fully remote clinical trial led by the NCI Intramural Research Program, researchers will use a variety of digital tools to study the effects of a dietary and exercise intervention on health outcomes in melanoma patients being treated with immunotherapy. Participants will interact remotely with physicians, dieticians, and psychologists during telemedicine appointments. And with wearable technologies, fitness apps, and online databases, researchers will monitor how participants are adhering to their assigned interventions.
Part of NCI’s efforts to incorporate telemedicine and digital technologies into clinical trials includes understanding telemedicine’s limitations. Investments in digital research studies will address the ethical implications of remote clinical trials, issues of equal access to digital tools in underserved communities, challenges of making all electronic health records compatible between clinical trial sites, and study designs that create safe and secure ways for patients to contribute medical information to a health record.
Breaking Down Barriers to Clinical Trial Participation
Widespread patient participation in clinical trials improves the clinical trial process in many ways. Without it, the eventual research findings may not apply to all populations, and robust participation also improves the speed and efficiency of trials. Researchers are studying how to remove barriers to clinical trial participation, especially from underserved communities.
An NCI-funded study led by researchers at Fred Hutchinson Cancer Research Center examined why people with cancer do not participate in clinical trials. The researchers looked at trial availability, patient eligibility, and physical barriers. They found that more than three out of four patients could not enroll in a clinical trial because none were available in the area, or the patient was ineligible to enroll. This study suggests that limited enrollment is not due to patient unwillingness. NCI is working to address challenges to trial availability and eligibility.
Minority and underserved populations encounter additional barriers, including but not limited to distrust of clinical trials, lack of information about the process, time and resource constraints, and unawareness that trials exist.
Reaching Minority and Underserved Participants
To establish a treatment’s safety and effectiveness in the general population, minority representation in clinical trials is critical. Yet, there is an imbalance in representation of minorities in clinical trials. For example, in the clinical trials that led to Food and Drug Administration approval of new drugs in 2020, only 8% of trial participants were Black or African American even though they represent 13% of the US population.
NCI is actively working to reach, recruit, and retain clinical trial participants from minority and underserved populations. NCI-funded studies are making progress toward full representation, but there is room to improve.
Over the last two decades, the proportion of racial and ethnic minority patients enrolled in NCI-funded NCTN and NCORP clinical trials has nearly doubled—from 14% in 1999 to 25% in 2019. A large NCI-funded analysis found that 9% of trial participants in NCI’s SWOG Cancer Research Network were Black, compared with less than 3% of participants in pharmaceutical company–sponsored trials. In the NCI Intramural Research Program, 13% of prostate and bladder cancer trial participants are Black, which is in line with the percentage of Black people with cancer in the US population.
Different populations of patients may also respond differently to therapies, and so clinical trials focused on those populations are important. For example, Black patients with cancer who are treated with some chemotherapies may have more neuropathy—weakness, numbness, or pain in the extremities caused by nerve damage. One ongoing NCI-funded study aims to reduce neuropathy side effects in Black women treated for breast cancer.
Increasing Eligibility and Reducing Cost
To reach all possible participants, including patients with coexisting health conditions, broadening eligibility criteria and reducing financial costs and other barriers to participation are critical factors.
In the last several years, NCI updated eligibility guidelines for NCI-sponsored cancer clinical trials. The changes aim to extend clinical trial access to more people, including those with coexisting health conditions. Many NCTN studies are now using more inclusive eligibility criteria.
With increased eligibility, clinical trials must ensure that participants are able to join the study without detriment to their families, employment, and finances. NCI has found that there is a lack of information about how clinical trial costs affect a patient’s decision to join a study. Patient costs can include expenses to care for dependent family members; travel-related expenses to reach the clinical trial site; and, in the case of pediatric clinical trials, additional out-of-state medical costs for a guardian’s own health conditions. More research is needed to understand how clinical trial study designs can ease financial burdens on patients.