Special Section: Cancer Moonshot – Five Years and Rising

image of a cancer cell
Representative Trials are Rocket Fuel for the Cancer Moonshot
Sans Thakur

n 2021, there were an estimated 1.9 million new cancer cases diagnosed in the US and 608,570 cancer deaths, or more than 166 deaths per day. The 2022 reignition of the Cancer Moonshot aims to cut that number by at least half over the next 25 years.

The US National Cancer Institute has already spent more than half ($1 billion) of its allocated $1.8 billion since the Moonshot was launched in 2016. Progress has been slow. FDA Commissioner Dr. Robert Califf, who also founded the Oncology Center of Excellence in 2017, has frequently noted that technological innovation should play a critical role in overcoming the barriers that continue to hinder progress–especially the lack of population representation in clinical research, including (but not limited to) racial minorities, women, and the elderly. In April 2022, FDA issued draft guidance outlining recommendations on developing plans to enroll more diverse participants, facilitate the development of better treatments, and better ways to fight diseases that often disproportionately impact underrepresented communities.

“This guidance also further demonstrates how we support the Administration’s Cancer Moonshot goal of addressing inequities in cancer care, helping to ensure that every community in America has access to cutting-edge cancer diagnostics, therapeutics, and clinical trials,” Califf explained.

Not only are technological innovation and adequate patient representation both essential ingredients in the Moonshot’s rocket fuel; they are inextricably linked. Decentralized clinical trial (DCT) technologies, including remote, digital tools, are proving the key to reaching a broad spectrum of patients and developing universally effective therapies. Simply put, if we bring in the right patients, we make the best medicine for all populations. We are at a medical crossroads, where the discovery of new cancer therapies and their effectiveness are at stake if we do not change how research gets done.

Three Major Barriers to Equitable Oncology Research

The reignited Cancer Moonshot program is up against three major barriers that discourage widespread trial participation by the populations most in need: economic, awareness, and cultural distrust.

For many people, trial participation is often unpleasant, costly, and time-consuming. Time off work, transportation costs, and childcare are out of reach for many in need–sadly, often for those populations more predisposed to the very conditions that researchers are attempting to treat. Although more than 40% of the U.S. population is made up of different ethnic minority groups, they make up only 5% to 10% of clinical trial participants.

Awareness is another common barrier. Earlier awareness of clinical research is important to the advancement of medicine, as is greater awareness of one’s own health needs. Most patients (particularly members of minority groups) are unaware of clinical research as a care option (CRAACO). Clinical research is often confined to large teaching institutions, which means that sites such as local hospitals or community centers servicing these minority populations are not asked to participate in clinical trials. Further, CRAACO is often unrealistic and therefore not encouraged by doctors because they recognize that the barriers to participate are prohibitive.

There are also cultural and historical issues. Some populations harbor understandable distrust of the scientific community due to past abuses, while language, customs, and religious differences bar other groups from participating in research. For instance, women are often hesitant to take experimental medicine due to fears of harming their fertility or future children’s health–possibly stemming from historical fears around certain investigative drugs (most notably thalidomide) causing serious birth defects. Elderly populations are similarly wary of being “guinea pigs,” as far fewer adults age 65 and older participate in trials than younger people, despite the fact that 50% of new cancer cases in the US occur in this age group. A cancer diagnosis can exacerbate reservations, as the severity of the disease can make patients anxious about doing anything that might further jeopardize their vulnerable health.

Underrepresentation in clinical trials has grave implications, especially in oncology, where treatment involves so many complexities. Age, genetics, gender, weight, ethnicity, environment, and more often lead to different responses to the same medication, including higher mortality and lower quality of life. For instance, one study found notable variations in how people from different ethnic groups reacted to nearly 20% of new drugs. And this is not just because of inherent biological differences but also because of lived experience–e.g., people of color tend to have higher rates of stress and higher exposure to environmental toxins.

Could the inherent biases unintentionally characteristic of the traditional clinical trial model, plus cancer’s complexities, be thwarting Moonshot goals? Consider patients diagnosed with acute myeloid leukemia—a rapidly progressing blood and bone marrow cancer—who fight physical and psychological side effects throughout treatment. The nausea and fatigue make in-person visits particularly challenging, transportation to sites on top of doctor’s visits is costly, and visits including invasive procedures such as blood transfusions are time-consuming. Tacking on research assessments before patients can go home or requiring additional site visits hours from home may be more than they can bear or afford.

DCTs are a game-changer, not just to accelerate cancer research but also to accelerate the development of better cancer treatments that help patients of every biology, not only white males.

Moving the Needle with Decentralized Trials

DCTs are becoming a preferred research model across many therapeutic areas. By easing patient burden, DCTs improve patient recruitment and retention and (most critically) increase opportunity for balanced representation. DCTs, coupled with an emerging community pharmacy site model that brings sites to within five miles of 90% of all Americans, can drive real Cancer Moonshot progress.

DCTs avoid lengthy travel to and from sites that can be too taxing on or otherwise prohibitive for cancer patients undergoing harsh treatments. In fact, the distribution of traditional trial sites partly explains why rural populations and minorities are poorly represented in oncology trials, according to a study in the Journal of the American Medical Association. A DCT can eliminate most, if not all, site visits.

Patients are already seeing the value of remote solutions. “Now, I do video visits with my oncologist, which is much less burdensome,” said one survivor of acute lymphoblastic leukemia who has entered a three-year maintenance and monitoring phase. “You probably can’t replace in-person visits completely in the early stages of an oncology trial, but virtual technologies can ease the burden long-term.”

Endless paperwork can also be tackled remotely in a DCT. Patients coming to a clinic for regular infusions are usually asked to complete a questionnaire on how they have been feeling. DCT solutions can leverage mobile devices to measure this data more frequently, providing improved quality-of-life insights about health impacts. Whereas traditional trials use a blunt methodology to assess symptoms, researchers can now include remote, real-time data capture with wearable devices for out-of-clinic insight on symptom development in direct correlation to treatment timing.

These noninvasive sensors are already being applied to cancer research in terms of prevention, risk, detection, diagnosis, treatment, and survivorship. Digital Biomarkers for Care is one Cancer Moonshot study that uses smart watches to continuously collect, measure, and analyze data. It aims to measure and understand the quality of life for patients receiving cancer therapy who may experience a host of long-term or late-onset side effects. In another study, sensors are being used to track changes in a patient’s voice, which may alert researchers to growths of lesions in the throat.

Fuel to Speed Cancer Treatments for All

With more than 600,000 lives and billions of dollars–the national patient economic burden associated with cancer care in the US was $21.09 billion in 2019–at stake, the Moonshot program needs a power boost. DCTs and associated technologies are the rocket fuel it needs. DCT technologies can save 35% and potentially add 50% of a $150 million to $450 million gain in revenue by accelerating the trial processes.

Smartphones are already replacing in-person visits for everything from patient enrollment to wellness checks. Wearable devices are detecting symptoms earlier than ever before and enabling preventative, more effective care. Clinicians and researchers are reacting in real time to patients’ response to treatments. In all, DCTs are easing the heavy burden oncology patients bear.

These results are better for patients and researchers alike, reducing care costs while simultaneously boosting clinical trial retention. The research community must drive a concerted, coordinated effort to integrate and adopt these solutions into routine clinical care–facilitating more representative study participation, faster approvals, fewer product failures in market, and better health outcomes for all.