We celebrate Clinical Trials Day on May 20 to honor a scientific breakthrough from nearly 300 years ago, when James Lind launched what is thought to be the first randomized clinical trial, which successfully tested citrus fruit as a treatment for scurvy among sailors.
Since then, clinical trials have led to countless life-changing treatments. But as far as we’ve come, the rules that govern clinical trials in the U.S. haven’t kept pace with today’s science. These rules, written decades ago, did not foresee and do not support the innovative advances we now have to make studies more efficient, representative, and accessible.
The Food and Drug Administration (FDA) issued a Final Guidance in 2024 to support modern community-based research, bringing clinical trials closer to where patients live, work, and play. But federal regulations remain out of date and don’t align with the Guidance, instead prioritizing traditional study designs, tools, and infrastructure over more innovative and accessible approaches. The result: many patients have limited ability to participate in studies, and access continues to favor those with the resources to take time off work, secure childcare, and cover travel expenses.
Everyone should have the ability to participate in clinical trials, which offer the chance to receive potential new investigational therapies or, at a minimum, specialized care and attention from research staff. Now, Congress can take action in two key areas to modernize clinical trials so that all patients have the ability to participate.
Removing financial obstacles.
Participating in a clinical trial often means taking on additional costs, such as travel and childcare, and may require patients to take time off work. This can prevent critical groups of people from participating, including those in rural areas, people with lower incomes, and caregivers tied to responsibilities at home. Many studies offer compensation, but federal statutes and complex financial rules penalize this support.
Here’s how policymakers can fix this:
- Update federal statutes to ensure patients who receive financial support for nonmedical study costs are not subject to tax liabilities or disqualified from income-based services.
- Create safe harbors to prevent study sponsors who compensate patients for time or expenses from being penalized under the Anti-Kickback Statute or the Civil Monetary Penalty Statute.
- Expand income exclusion for Supplemental Security Income (SSI) eligibility beyond study participants with rare diseases to include clinical trial participants in all treatment areas – and eliminate the $2,000 cap.
To that end, the passage of the bipartisan Clinical Trial Modernization Act and its companion bill in the U.S. Senate, as well as the Harley Jacobsen Clinical Trial Participant Income Exemption Act, would directly address these gaps by updating outmoded statutes, expanding financial support options, and protecting patients from unintended penalties. patients from unintended penalties.
Updating regulations to support today’s patient-centered research.
Regulations that govern how we conduct clinical trials still don’t align with today’s more modern study designs. They were created when all parts of the clinical trial were based at large academic medical centers, and patients had to travel there for all of their appointments. But today’s community-based studies involve networks of local providers across the country and can include digital tools that allow patients to provide some health data from the convenience of their homes. Federal regulations must be updated to allow more oversight flexibility and to accommodate new technologies. This includes:
- Giving study leaders more flexibility in how they provide training and oversight. The Federal Food, Drug, and Cosmetic Act must be amended to clarify that an investigator’s responsibilities to supervise local providers and vendors are satisfied if providers have previous training and experience that qualify them as appropriate experts, and if they are trained on relevant elements of the protocol, investigational drug, and the investigator’s brochure.
- Protecting community healthcare providers from heavy administrative burden. The Final Guidance provides this protection by stating that local providers should not be classified as sub-investigators if they are simply providing care that is considered “routine clinical practice.” But that protection has not been written into regulation.
- Promoting broader FDA acceptance of digital health technologies (DHTs). FDA support for these innovative tools has been inconsistent, limiting their widespread use despite their convenience and efficiency for both patients and researchers. To better support these tools, Congress can direct the FDA to provide more guidance on the evidence needed to validate DHTs and to ensure the agency’s internal divisions are aligned on how they review DHT data.
The informed-consent process has also become so complex that it risks no longer serving its core purpose: ensuring patients understand what they are agreeing to by electing to participate in the trial. Congress should direct the FDA to work with sponsors, patient advocates, and research sites to establish a common consent template, one that is plain-language, standardized, and written for the patients it is meant to protect.
With those updates in place, patient-centric research can become the norm, not the exception. Congress must pass the Clinical Trial Modernization Act and the Harley Jacobsen Clinical Trial Participant Income Exemption Act this session and commit to modernizing the regulatory framework governing clinical research in America. The science is ready and patients are waiting. The regulations must now catch up.
Kristi Robertson, Esq. serves as Senior Director, Clinical Trial Foundations, Community-Based Research at Eli Lilly and Company, where she leads Clinical Capabilities’ policy efforts to modernize clinical trials and expand patient access to innovation worldwide.
