Enabling Diversity in Clinical Trials: New US Law Opens Door to Clinical Research for Millions of Low-Income Patients
Mary Stober Murray
National Minority Quality Forum

n 2020, the United States Congress passed the Clinical Treatment Act, a law “to promote access to life-saving therapies for Medicaid enrollees by ensuring coverage of routine patient costs for items and services furnished in connection with participation in qualifying clinical trials, and for other purposes.” The law became effective in January 2022, yet a 2021 diversity and inclusion survey of eight major pharmaceutical sponsors reported that none of the respondents indicated that they are actively encouraging “sites to recruit Medicaid beneficiaries to participate in our active cancer clinical trials.”

What is Medicaid and whom does it serve? Why does the Clinical Treatment Act matter for increasing representation in clinical trials? How can clinical development professionals learn to engage the Medicaid population and facilitate clinical trial participation?

This article aims to answer each of these questions, outline stakeholder perspectives for clinical development, and recommend next steps for organizations and individuals.

Key Takeaways

  1. The Clinical Treatment Act removes a financial barrier for Medicaid beneficiaries to participate in qualifying clinical trials across disease areas, including cancer.
  2. Medicaid is a public health insurance program covering about 1 in 5 people in the United States, including about 40% of America’s children, as well as low-income adults and disabled individuals.
  3. Racial and ethnic minorities represent a disproportionately high fraction of Medicaid beneficiaries, and nearly two-thirds are living with one or more chronic conditions.
  4. Organizations can continue to transform their culture toward patient-centricity by learning how to reflect the clinical experience and social needs of the Medicaid population in medicines development processes and operations.
  5. Individuals can take the lead by educating themselves and advocating internally within their organizations for collaborative, community-based strategies that reach the Medicaid population and other patients where they live.

Fundamentals of Medicaid

Medicaid is the government-run health insurance program for low-income Americans. More than 83 million people are covered by Medicaid (approx. 77 million) and CHIP (Children’s Health Insurance Program; approx. 7 million), including nearly 40 million children total. Each of the 50 individual states and Puerto Rico administers its own Medicaid program, overseen by the federal Centers for Medicare and Medicaid Services (CMS) and with funding shared by the state and federal governments. Racial and ethnic minorities are over-represented among Medicaid beneficiaries compared to the general US population, with more than half (61.1%) identifying “as Black, Hispanic, Asian American, or another non-white race or ethnicity.”

State-to-state variability in Medicaid programs affects populations locally regarding diagnosis and access to care. Initial trends associated with Medicaid expansion across individual states indicate increases in positive health outcomes, particularly for early-stage diagnosis and survival increase in some cancers, while disparities associated with race, ethnicity, and socioeconomic factors decrease.

CMS collects Medicaid data from individual states and makes it available to qualified nonprofit partners for analysis of patient populations and primary care and specialty providers at the zip code level. Some limitations include variations in state Medicaid eligibility requirements and also in state reporting.

The Clinical Treatment Act

The Clinical Treatment Act addresses a gap in Medicaid coverage related to clinical trial participation. Until its passage, Medicaid beneficiaries were uniquely vulnerable to out-of-pocket costs for “routine care” if they were participating in clinical trials. These costs include non-experimental costs of care for a patient who is also participating in a clinical trial, such as the physician visits or lab tests that are part of the standard of care. Clinical trial sponsors continue to cover the costs of any device or drug under investigation.

Medicare (the US public health insurance program for senior citizens) and private insurers and health plans were required by federal mandate to cover these costs for their beneficiaries, but Medicaid coverage was left to the discretion of the individual states; only 16 states guaranteed coverage of these costs in 2020.

In December 2021, CMS issued a State Medicaid Director letter informing states of the new federally mandated Medicaid state plan requirements, outlining covered services, defining a qualified clinical trial, detailing coverage determinations, and describing state exceptions for compliance.

CMS requires states to file a state plan amendment (SPA) that describes the state’s coverage and benefit policies as well as payment policies for service providers. Implementation of the CTA requirements will vary from state to state because some states need formal legislative action. State-to-state variability in implementing the Clinical Treatment Act may also affect access to cancer clinical trials for Medicaid beneficiaries closer to home.

Health systems and treatment centers that care for Medicaid patients publish patient testimonials, like Laura’s story, that emphasize the life-saving significance of state Medicaid coverage to patients, especially when they are faced with a catastrophic diagnosis and costly treatment plan, such as cancer. Continuing Medicaid’s coverage of these standard of care costs is critical to keeping patients in care and able to consider available clinical trials when these are options.

Stakeholder Perspectives

Social determinants of health (SDOH) include basic needs, such as food, clothing, and shelter, as well as other factors including dependent care, transportation, job schedules, and language support needs. While SDOH affect all people in terms of managing healthcare access and treatment options, Medicaid beneficiaries are particularly vulnerable to SDOH resulting in exclusion from options related to care and research participation.

Estimates suggest that about half to two-thirds (55.7%-62.1%) of Medicaid beneficiaries, about 50 million people, live with one or more chronic conditions, including cardiovascular disease (heart disease, hypertension, hyperlipidemia), diabetes, cancer, asthma, and depression. For example, more than two million children and adults under 65 with a history of cancer rely on Medicaid; children aged 1-4 have the highest incidence of childhood cancers.

A scan of clinicaltrials.gov indicates that about 11,000 clinical trials are recruiting participants in these diseases and therapeutic areas (primarily cancer) in the United States in 2022.

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Many patient advocacy organizations, particularly those representing the needs of cancer patients, drove advocacy efforts to achieve the national clinical trial coverage for Medicaid populations mandated in the Clinical Treatment Act.

Medical associations, such as the American Society of Clinical Oncology (ASCO), advocated alongside patient groups for the Clinical Treatment Act and are continuing to track its implementation in 2022 among the individual states.

Changes to the infrastructure for clinical research and healthcare delivery are also needed to include Medicaid populations. The Acting Commissioner of the US Food and Drug Administration (FDA), Janet Woodcock, advocates for integrating community clinicians into the “clinical research enterprise,” contributing to aspects of clinical development, such as informing the establishment of inclusion/exclusion criteria, expanding the pool of clinical trial sites, and cocreating trusted communications with patients in their local communities. Such an approach would aim to make clinical trials available to patients regardless of their proximity to the academic medical centers where most clinical research is conducted today.

Integrating Federally Qualified Health Centers (FQHCs) and their practitioners into the clinical research infrastructure promises to extend clinical research options to Medicaid beneficiaries. Born of the civil rights activism in the United States in the 1960s, FQHCs combine funding from the US Health Resources and Services Administration (HRSA) with community resources to provide primary care in rural as well as urban areas.

In 2020, 1,375 federally funded health centers (FQHCs) served nearly 29 million Americans. Nearly half of patients seen at FQHCs are insured by Medicaid (about 13 million), including more than 5 million children under the age of 17; about 25% of FQHC-treated patients prefer service in a language other than English. HRSA data describes the FQHC patient population and health outcomes.

Initial research is emerging to indicate that pharma sponsors are making strides in overall diversity and inclusion efforts related to clinical research. Findings from a survey of eight major pharmaceutical developers presented in an abstract at the 2021 AACR Science of Cancer Health Disparities meeting reported demonstrable gains, particularly in the areas of site selection, general capabilities, and leadership. Specifically, 95% of the eight major pharma companies responding to the survey reported having a diversity and inclusion officer that focuses exclusively on clinical trials, seeking to select more minority serving sites, developing materials that are health literate and culturally sensitive, covering out-of-pocket costs and including patient organizations as partners to gain trusted community insights. However, none of those sponsors surveyed are yet actively encouraging “sites to recruit Medicaid beneficiaries to participate in our cancer clinical trials.”

These findings indicate that there is an opportunity and a need to prepare sponsors to learn how to expand clinical trial options to the Medicaid populations with medical need, in order to generate research data that represents the diverse populations living with the conditions under study.

Next Steps

Individuals and organizations can consider the following next steps for integrating the Medicaid population into clinical research for medicines development, organized into three areas of priority: Learn the Data, Seek Collaboration, and Reimagine Communication. A starting resource list appears at the end of this article.

Learn the Data

  1. Educate yourself about Medicaid and its health impact among American patients across disease areas. Search published research as well as programs specifically exploring Medicaid populations and services, such as the Robert Wood Johnson Foundation (RWJF).
  2. Brief yourself and your colleagues about the Clinical Treatment Act, and develop internal strategies to stay updated on its implementation among the individual states.
  3. Access/explore data about healthcare among Medicaid and other underserved populations. Start with CMS and HRSA.

Seek Collaboration

  1. Seek internal and external partners to track each state’s legislative calendar and any applications for delay that may impact plans for patient engagement and clinical trial operations.
  2. Support and/or join with patient advocates/nonprofits and community organizations through working groups or other collaborative models to research topics related to serving the Medicaid population.
  3. Invite those experienced with serving the Medicaid population, such as FQHCs and community engagement leaders, to share effective engagement interventions.
  4. Identify areas where insights from the Medicaid community can inform changes to your organization’s workflows and operational strategies, such as protocol development and site selection, considering clinical as well as socioeconomic and cultural characteristics.

Reimagine Communication

  1. Aim for communication strategies that start a mutually informative conversation, allowing you to better understand patients in the context of their lives and communities, rather than to unilaterally direct behavioral change.
  2. Support and/or join with patient advocates/nonprofits and community organizations in collaborative efforts to develop content that is most relevant, and to deliver it in contexts that help you identify the audience most receptive to it and the trusted voices within. Examples include RWJF’s “That’s Medicaid” and Vult Lab’s “COVID Diaries.”
  3. Continuously measure the impact and reach of communications in order to identify, follow, and engage the most trusted voice(s) among patients in their communities, and expand the conversation to achieve better health outcomes and deeper reach. Social media providers can help to build meaningful dashboards.


The Clinical Treatment Act has not only removed a financial barrier for Medicaid beneficiaries to participate in clinical research, it has also created an urgent imperative for all stakeholders in clinical research to learn how to engage the Medicaid population and drive their inclusion in clinical research. These efforts are necessary to ensure that representative clinical research data will lead to meaningful treatments and effective policies that improve the lives of all people living with serious and chronic diseases, regardless of race/ethnicity, income level, or geography.