FDA Reforms for Trial Access, Reduced Regulatory Burdens: Report
The Milken Institute is urging the FDA and other federal agencies to overhaul clinical trial policies, arguing that unnecessarily restrictive eligibility criteria, cumbersome consent requirements and fragmented regulatory oversight are limiting patient participation and slowing medical research.
In a 10/13 report, Enabling Access to Clinical Research: An Action Plan for Policy and Practice Change, the institute’s FasterCures initiative outlined eight priorities for expanding clinical research into community healthcare settings while reducing administrative burdens on sponsors, investigators and patients. They are:
- Expand trial inclusivity: Broaden eligibility criteria, strengthen IRB oversight and incorporate patient input into study design.
- Simplify informed consent: Streamline consent documents and expand electronic informed consent (eConsent).
- Reduce participation barriers: Address travel expenses, participant compensation, insurance coverage and other financial obstacles.
- Return results to participants: Routinely provide study findings and individual health information to trial participants.
- Expand pragmatic trials: Encourage trials integrated into routine care, harmonize regulatory requirements and address reimbursement barriers.
- Streamline data collection: Limit unnecessary data requirements and allow more flexible documentation and remote assessments.
- Improve electronic health records: Strengthen interoperability, trial-matching capabilities and integration of research data.
- Modernize trial execution: Expand decentralized trials, digital health technologies, telehealth flexibility and IRB training.
Among its recommendations, the report calls on FDA to issue additional guidance encouraging sponsors to use real-world data to establish more representative eligibility criteria and justify exclusions based on safety evidence rather than conventional trial-design practices.
It also recommends that FDA and the HHS Office for Human Research Protections clarify institutional review boards’ authority to challenge unnecessarily restrictive enrollment criteria and require greater patient involvement in protocol development.
The institute proposes making electronic informed consent the default for clinical trials, with paper alternatives available when needed. It urges FDA to establish clearer expectations for readable consent documents and promote standardized electronic consent systems.
Another recommendation urges FDA and trial sponsors to “actively develop, evaluate, and scale pragmatic trial approaches that treat regulatory framework modernization — not just demonstration project participation — as the central objective,” according to the report. The agency should publish a structured lessons learned report from the STEP demonstration project “covering what regulatory flexibilities were exercised, what friction points remained, and what protocol or administrative changes most enabled pragmatic design — and use that report as the empirical foundation for updated pragmatic trial guidance,” it says.
To reduce unnecessary data collection, the report calls for FDA to issue guidance that specifies what it expects to see in IND and protocol submissions with respect to identification of “critical-to-quality” factors and justification of data elements, consistent with ICH E6(R3). The agency’s review divisions should also update internal reviewer guidance to operationalize new external expectations at the reviewer level, it adds.
Additionally, the report recommends improving electronic health record interoperability, increasing the use of decentralized trials and digital health technologies, and changes to Medicare and Medicaid reimbursement policies to cover routine trial costs more consistently and reduce financial barriers to participation.
The report notes that many reforms could be implemented under existing regulatory authorities without new legislation, although broader structural changes would require congressional or agency action.