In This Section

ACORI Research Review: October 2022


Improving Access to Transgender Patients in Clinical Trials


Each year between November 13-19, people and organizations around the country participate in Transgender Awareness Week to help raise the visibility about transgender people and address issues members of the community face. In recognition of this important event, issue of Research Review looks at ways to increase representation of transgender patients in clinical trials.

Read Full Article

Improving diversity, inclusion, and health equity in clinical trials applies not only to racial and ethnic minorities, but also to groups of individuals with varied socioeconomic and cultural backgrounds, health profiles, and gender identities, among others. To characterize the disparities among transgender patients, Parexel performed a thorough search of the PubMed database to identify articles reporting results from randomized, interventional clinical trials that included transgender and non-binary patients. Between July 2018 and February 2022, 116,057 articles were published reporting clinical trial results. Among these, 78 studies (0.06%) reported participation of transgender patients.

In the U.S., it is estimated that currently 1.6% of adults are transgender or nonbinary, meaning that their gender identity differs from their sex assigned at birth or that they do not identify exclusively as male or female.1 Equitable representation of the transgender population in research is needed to understand the effects of different cancer (and non-cancer) treatments in the setting of long-term gender-affirming hormone therapy. Transgender patients are not well represented in clinical trials largely because they are not being asked to participate, they are not willing to participate, or the data being collected does not include this population. Increasing representation of transgender patients in clinical research can be achieved by gaining an understanding of the key barriers and ways to overcome them.

Lack of inclusive practices is one of the primary reasons that discourages transgender patients from participating in trials, among other barriers such as stigmatization, discrimination, and lack of trust in the medical profession and pharmaceutical industry. Transgender individuals can feel excluded in some environments, even if the organization is not intentionally trying to ostracize the individual. In the healthcare field, medical documentation is typically binary in nature, and a transgender patient may feel uncomfortable having to choose between male and female on a form. The two-step gender measure is becoming an increasingly common and validated way to identify people who are transgender, allowing patients the opportunity to note their sex assigned at birth along with their current gender identity.

Liam Paschall, Senior Consultant of Management Development at Parexel and one of LinkedIn’s 2022 Top LGBTQIA Voices, recommends the two-step as “a starting point,” but reiterates that it does not encompass everything that medical providers and the pharma industry can do to create a more inclusive and supportive environment for transgender patients. The two-step approach would give transgender individuals the opportunity to denote their identification without making them feel uneasy. Additionally, another important practical consideration for institutions is the provision of gender-neutral bathrooms. It can be distressing for transgender individuals to use a restroom in which they do not feel welcome. The best way to circumvent this obstacle is to provide single-unit, gender-neutral restrooms that a transgender individual could use without drawing unwanted attention to themselves.

Of course, it goes without saying that inclusivity extends beyond bathrooms and documents; inclusivity is an attitude and a mindset that governs how we view and interact with others. Paschall shares that an example of practicing inclusivity is asking patients their pronouns and chosen names. These queries, while seemingly simple, can make patients feel safe and respected and, thus, open the door to more meaningful relationships with their providers. Inclusivity, however, is important not only for medical providers to practice, but for all individuals. An inclusive environment should be evident from the second the patient steps into the clinic or hospital, well before they meet with their provider. Education around cultural competency and gender identity are critical to ensuring that all institutional staff treat patients and visitors with an open mind and compassionate care. This type of environment fosters trust and understanding and can empower transgender patients to take a greater role in their care.

Aside from inclusivity, transgender patients also experience other barriers to research, such as various financial and socioeconomic barriers (i.e., lack of adequate insurance coverage [uninsured or underinsured] or reliable transportation) and lack of access to providers with the education and training to treat and care for transgender patients. Previous research has indicated that few physicians have sufficient knowledge or comfort in managing transgender patients and the care of these patients is rarely a topic in medical school curricula.2 In 2016, the University of Cincinnati (UC) College of Medicine established a transgender medicine program that integrated transgender medicine into the basic science curriculum so that medical students would learn about gender diversity while learning about hormone pathways and sexual development. Sarah Pickle, MD, Founder of the Transgender Medicine Program at the UC College of Medicine, notes that “the idea was [to] give students the tools, like understanding the physiology of gender affirming hormones, and also prepare them for inclusive conversations with our patients as partners in their gender journeys.”3 As with inclusivity, education and ongoing training, preferably by a member of the community, is the key to equipping all providers with the knowledge and confidence that they need to sufficiently care for these patients. Moreover, education also helps fight the stigmatization that commonly plagues transgender individuals.

To help advance the care of transgender patients, Parexel has conducted a significant amount of research to understand the disparities, determine the gaps in care, and provide guidance on tackling the various issues. This is a new topic in the industry, and Parexel is taking the time to listen, learn, and understand. From extensive conversations with transgender patients to the production of sex and gender identity best practice guidelines, Parexel is paving the way toward more inclusive care for the transgender population. However, the issues surrounding representation of transgender patients in clinical research can only be overcome when addressed from a variety of angles. Government agencies, sponsors and research organizations, academic institutions, hospitals, clinics, and more, can work together to promote inclusivity and accessibility for this underrepresented group of patients. Ultimately, it will require people getting comfortable being uncomfortable and acknowledging what they do not know to truly make a difference in this community.


  1. Brown A. About 5% of Young Adults in the U.S. Say Their Gender is Different from Their Sex Assigned at Birth.” Pew Research Center, Washington, D.C. Published June 7, 2022. Accessed September 9, 2022.
  2. Safer JD, Coleman E, Feldman J, et al. Barriers to Health Care for Transgender Individuals. Curr Opin Endocrinol Diabetes Obes. 2016;23(2):168-71. doi: 10.1097/MED.0000000000000227
  3. Bangert B. Students, faculty and staff embrace transgender medicine curriculum in the College of Medicine. University of Cincinnati News. Published March 29, 2022. Accessed Aug 19, 2022. 

New Global Cancer Coalition Calls for a "Reimagining" of Patient-Centric Clinical Trials

In 2021, the Bloomberg New Economy International Cancer Coalition was formed to promote international collaboration and regulatory harmonization in clinical research around the world. Earlier this year, the Coalition published a statement urging for a paradigm shift in the way patients are cared for in clinical trials as a result of changes brought about by the pandemic. Although the pandemic disrupted many processes in cancer care and clinical trial delivery, it also taught us to be more creative with our resources, giving meaning to the phrase “where there’s a will, there’s a way.” Use of telemedicine and remote monitoring has risen drastically during the pandemic out of necessity, allowing more patients to be seen for routine care. The Coalition emphasizes that digital technology efforts should continue to be harnessed for clinical trials to expand access and increase diverse participation in research.

Read Full Article

A significant barrier to trial diversification is the identification and enrollment of patients who are potentially eligible for trials. Since most trials are limited to academic medical centers, patients who do not live near academic centers may not have access to many trials. Likewise, community providers who are not highly involved in trials may lack information to adequately discuss trial options with patients. Digital tools can help resolve some of these issues, such as building an international database in which patients can input their cancer data or implementing a “hub-and-spoke” network where academic centers serve as a hub for community research sites.

Once patients are on trial, they are tasked with countless study visits that can take a significant amount of time, especially for those who live farther away from sites. The pandemic forced many clinical trials to adopt digital monitoring, local laboratory and imaging studies, and remote shipment of study medications to patients’ homes. These adjustments can widen the scope of trials to include more patients who would not typically be able to commit to frequent on-site study appointments. Lastly, the Coalition strongly recommends increased harmonization among international regulatory agencies to standardize cancer molecular profiling, international cancer genetics data sharing, and remote monitoring.

Virtual Clinical Trials in Oncology

The COVID-19 pandemic has led to innumerable changes in the provision of medical care, most notably in the way that patients are managed in the outpatient setting. The need to reduce in-person visits forced medical providers to embrace telemedicine and develop creative ways to monitor and treat patients virtually. Although the shift toward virtual medicine has not been without challenges, it has triggered many positive developments, particularly in the field of clinical research. Despite the strong recommendations for clinical trials in oncologic treatment guidelines, only 2% to 8% of all cancer patients participate in oncology trials. These low rates of participation can be attributed to a variety of barriers from patient recruitment to data collection. Telemedicine and digital tools can help overcome some of these barriers to expand access to trials and minimize disparities in research.

Read Full Article

Recruitment is typically the highest cost burden for clinical trials and can be a substantial obstacle due to the disproportionate availability of trials within academic medical centers compared to non-academic centers. Since most trials are started in academic centers, patients who have access to such centers are exposed to more research opportunities. Digital tools can play a significant role in reaching more potential trial candidates by collecting patient information at non-academic centers, determining eligibility for certain trials, and matching the patient to a trial. Use of artificial intelligence for patient-trial matching can also benefit providers in non-academic settings who may lack awareness on the range of available trials. Once patients are identified for trials, many elect not to enroll due to logistical factors and misconceptions about clinical research. For the medical team, the enrollment process also poses a considerable administrative burden on staff. Digital tools can be utilized to obtain electronic consent from patients, saving patients and providers time and resources.

The prospect of frequent in-person appointments can deter patients from participating in clinical trials, especially those who lack access to reliable transportation or have childcare concerns. During the peak of the pandemic, many trial processes were adjusted to leverage the use of telemedicine and alternative study locations for patients, minimizing the need for in-person visits or reducing congestion in certain study sites. The silver lining to these changes is that patients who indicate geographical location as a hindrance to trial participation can now be monitored primarily at home or closer to home. Moreover, many investigators started to ship oral medications to patients’ homes, further enhancing the convenience of participating in trials for those who are geographically disadvantaged.

As the hub of research transitions to the home, it is important to consider how data will be collected if patients are not coming into the clinic throughout the week. Perhaps the greatest impact of virtual medicine in the research field is the ability to monitor patients remotely, allowing patients more freedom without jeopardizing the quality of the trial. Noninvasive medical devices and home-based blood draws can be implemented to collect quantitative data, while digital tools can be developed to assess quality of life and other subjective endpoints. Remote monitoring not only enhances convenience for patients, but it also shifts data collection from an episodic to a more continuous approach.

Although the virtualization of clinical trials has many advantages, virtualization should not be generalized to all trials without deliberate consideration of the risks and benefits. Early-phase trials (Phase I/II) are not easily amenable to a virtual platform due to the need for frequent pharmacokinetic studies and specialized specimen handling. In addition to the importance of a highly controlled setting, the use of a completely new medication undergoing dose-finding studies also warrants in-person monitoring. Where digital technology could be integrated into early-phase studies would be helping with identification and recruitment of patients, as well as data analysis. Clinical trials that are more amenable to virtualization include those testing approved drugs for new indications, combinations, or sequencing. Virtual monitoring can also be implemented in later-phase studies to assess secondary endpoints, such as quality-of-life.

As the use of virtual clinical trials grows, organizations and regulatory agencies must consider revising policies to accommodate the changes brought on by the pandemic. Virtual clinical trials may offer more convenience for patients, but we, as a medical society, must ensure that virtualization does not come at the price of patients’ privacy. For instance, medical devices, such as Apple Watches, can be used for both consumer and research purposes, but how do we leverage these tools without blurring the lines of research and privacy? Lastly, policymakers must also consider the need for technical infrastructure investments as clinical trials continue to shift toward virtual platforms. While digital tools can expand access to trials overall, they can also widen disparities between patients who do and do not have access to reliable internet and electronics. Without proper infrastructure, the digital divide will continue to exist, even if we resolve other disparities.

Despite the challenges that arose with COVID-19, the transition of clinical trials into the virtual space is regarded as one of the more productive outcomes of this unfortunate global crisis. Virtual clinical trials can increase participation in oncology research by streamlining recruitment and enrollment processes, enhancing convenience for patients, and optimizing data collection and analysis. However, not all trials are suited for virtual operations, and investigators must consider the advantages and disadvantages of trial virtualization before implementation.

Can Digital Health Increase Diversity in Clinical Trials?

Although African Americans comprise about 12.5% of the U.S. population, they represent only 5% of patients enrolled in clinical trials.1,2 Likewise, Hispanic/Latinx individuals account for approximately 18.5% of the U.S. population but represent only 2.3% to 3.9% of cancer trial participants.3 A quick literature search on “diversity in cancer clinical trials” reveal that these statistics highlight only a few examples of the disparities that exist in cancer research. Aside from race and ethnicity, disparities in clinical trials are also found among patients of different socioeconomic classes, education levels, genders, and more. Today, a significant focus of clinical trial development revolves around strategies to enhance diversity and ensure health equity.

Read Full Article

One question that arises regarding this issue is if digital health can help increase diversity in clinical trials. Recently, a study published by Adams et al. attempted to answer this question by analyzing the association between remote technology and patient likelihood to enroll in cancer clinical trials.4 In this study, an internet-based survey was sent out to cancer patients and survivors of cancer who had been diagnosed with or treated for cancer in the past seven years. Over a two-month period, 1,183 participants responded to the survey: 2% identified as Native Hawaiian/Other Pacific Islander, 2% Asian, 3% American Indian/Alaska Native, 20% Black, 70% White, and 4% declined to answer. Twenty-six percent had a household income of $70,001 to $125,000, while 16% had a household income of $35,000 or less. Forty-nine percent of respondents reported living within 30 minutes of their primary cancer provider. The vast majority (94%) had reliable high-speed internet at home, but 4% did not.

Eighteen percent of respondents had participated in a clinical trial; however, 46% reported that they had never discussed trial participation with a provider. Regarding the logistics of participating in a trial, 77% noted that they would join a trial if it were similar to their regular care in distance and frequency of visits, whereas only 47% would participate if it required additional effort in travel distance or visit frequency. Age and household income were the two main factors that determined if patients would participate in trials requiring additional effort. Those of older age (>55) or in lower-income households (<$70,000/year) were significantly less likely to participate in trials farther from their home or with more frequent visits compared to regular care.

When the survey participants were asked if they would be willing to adopt digital tools to participate in telemedicine, more than 80% reported that they would be willing to adopt most of the remote interventions. In the context of clinical trials, 60% to 85% of participants reported an increase in the likelihood of enrolling in a clinical trial if the trial adopted digital tools. Notably, even those who initially expressed high reluctance to clinical trials reported an increased willingness to participate with digital tools. These results suggest that digital health can help improve diversity in trials by reducing time and travel commitments, thereby providing patients easier access to care.

Last year, the Diversifying Investigations via Equitable Research Studies for Everyone (DIVERSE) Trials Act was introduced in the senate in hopes of improving access to clinical trials, particularly among under-represented populations. This act would require the FDA to issue guidance on decentralized clinical trials to promote meaningful demographic and geographic diversity. The act would also allow the U.S. Department of Health and Human Services to “enter into contractual arrangements to support education, outreach, and recruitment for clinical trials for diseases with disproportionate impact on under-represented populations.” Practically speaking, this act would allow sponsors to reimburse patients for non-medical costs of trials and provide patients with the technology to participate in remote trials.

As decentralized trials grow in popularity, many companies are designing virtual platforms that can be adopted by academic institutions, pharmaceutical companies, and research organizations. One example of a company specializing in virtual clinical research software is Curebase, which provides a full range of tools and services to run clinical studies at home and in community clinics. Curebase works with principal investigators around the world in a variety of therapeutic areas to recruit, enroll, and monitor patients in trials by using virtual consenting, telehealth appointments, mobile nursing/phlebotomy, local healthcare providers, and other strategies.

Ideally, decentralized clinical trials can be used alongside traditional trials in a hybrid system that embodies the best of both worlds. For example, if a large academic center is struggling to recruit patients who live farther away, then the protocol could be revised to allow participants to participate from home via remote platforms and home drug delivery. The principal investigators would then oversee staff and patients at their main facilities in addition to those at remote locations with the help of the decentralized technologies. This type of system has a strong potential to expand the reach of clinical trials, particularly among those who are less able to devote significant time and resources to participating in a trial. If used wisely, digital health could be the answer to improving diversity in clinical trials.


  1. U.S. Census Bureau (2021). 2020 Census Illuminates Racial and Ethnic Composition of the Country. Retrieved from [].
  2. Alegria M, Sud S, Steinberg BE, et al. Reporting of Participant Race, Sex, and Socioeconomic Status in Randomized Clinical Trials in General Medical Journals, 2015 vs 2019. JAMA Netw Open. 2021; 4(5):e2111516.
  3. Espinoza-Gutarra MR, Duma N, Aristizabal P, et al. The Problem of Hispanic/Latinx Under-representation in Cancer Clinical Trials. JCO Oncology Practice. 2022; 18: 380-5.
  4. Adams DV, Long S, Fleury ME. Association of remote technology use and other decentralization tools with patient likelihood to enroll in cancer clinical trials. JAMA Netw Open. 2022;1;5(7):e2220053. doi: 10.1001/jamanetworkopen.2022.20053

Just ASK™ Training Seeks to Increase Diversity in Clinical Trial Research

Just ASK™ is a training program developed by ACCC-ASCO to educate cancer research teams on diversity, equity, and implicit biases in clinical research. This 60-minute course features didactic slides, interactive case studies, and interview segments to demonstrate how implicit biases interplay with health disparities and to explore strategies for addressing barriers to trial participation among under-represented populations. The training program commences with a review of the key concepts, using visuals and examples to illustrate definitions. For instance, in discussing equality vs. equity, the segment shows a group of individuals with differing heights, including a wheelchair-bound person, who are trying to reach for apples on a tree; equality in this context would mean giving everyone the same stepping stool to help them reach higher, whereas equity would be giving each individual the customized boost they need to ensure that all hands reach the same height on the tree. Visual learners will appreciate the use of illustrations to portray these key concepts.

Read Full Article

After this conceptual review, the training program incorporates an activity in which the learner designates different real-world scenarios as examples of “equity” or “equality” (i.e., “hosting a health seminar in English and inviting everyone to attend,” “hiring translators to attend a community meeting about health concerns”). In the review of implicit biases, learners click on different buttons to uncover common implicit biases in healthcare (i.e., “women presenting with cardiac disease symptoms are less likely than men to receive diagnosis, referral and treatment, due to misdiagnosis of stress and/or anxiety”). There is also a series of real-world case studies that present patients with cancer of various health and socioeconomic backgrounds who are being evaluated for clinical trials. For each patient, the training program asks learners to identify what they perceive as potential barriers to trial participation (i.e., “history of substance abuse,” “long commute,” “hesitant to enroll,”etc.), and in response, the training program suggests additional questions to ask the patient to gauge their barrier (i.e., “concerns about long-term effects of substance abuse are valid—does the patient have ongoing support for this?”; “long commute may seem like the primary barrier for this patient—but does she think so?”; “what’s behind her reluctance to participate?”). Healthcare providers should find it impactful not only to consider these cases and acknowledge their own implicit biases, but most importantly, to work through their biases and perceived barriers by getting to know how the patient feels and what their concerns are. Overall, this training program encourages research teams to critically assess their current practices and brainstorm improvements or additions that they can make to address systemic barriers and advance health equity.

A Spotlight on Compass HER2-pCR in Recognition of October as Breast Cancer Awareness Month

CompassHER2-pCR is a trial assessing if HER2-positive Stage II-IIIa breast cancer patients can be safely treated with less chemotherapy in the adjuvant setting. Patients enrolled in this study receive a taxane (an established treatment for breast cancer) and two anti-HER2 antibodies for 12 weeks before surgery. At surgery, if patients are found to have pathologic complete response, then they would proceed with adjuvant treatment consisting of only trastuzumab and pertuzumab for one year (along with radiation and anti-estrogens as needed). Patients who have residual disease would undergo standard of care treatment with ado-trastuzumab emtansine.

Read Full Article

The primary endpoint for this study is recurrence-free survival, with secondary endpoints consisting of invasive disease-free survival, distant disease-free survival, distant relapse-free survival, recurrence-free interval, overall survival, event-free survival, and incidence of adverse events. Patients will be followed for up to 15 years. Notable exclusion criteria include prior ipsilateral DCIS; prior invasive breast cancer; T4 and/or N3 disease, including inflammatory breast cancer; prior treatment for current breast cancer; baseline neuropathy > grade 1; concurrent serious medical condition; or pregnant/breastfeeding patients.

The CompassHER2-pCR study launched in early 2020 and is currently active in 902 study sites, with most sites still recruiting. The primary completion date is February 28, 2023. This study will shed light on the potential to minimize chemotherapy exposure for HER2+ Stage II-IIIa breast cancer patients without sacrificing outcomes.