Publication:Therapeutic Innovation and Regulatory Science
Date Published: June 18, 2026
Description: “Expertise of European Clinical Trial Units in Conducting and Managing Cross-Border Pediatric Clinical Trials for Rare Diseases,” published in Therapeutic Innovation & Regulatory Science and led by Begonya Nafria of the Institut de Recerca Sant Joan de Déu with co-author Barbara Bierer, surveyed 43 clinical trial units across 17 European countries on their experience managing international participants in pediatric trials for rare diseases. Those units had received patients from 81 different countries, and the good practice they reported most often was providing written and verbal translation of informed consent documents, patient-reported outcome measures, and quality of life scales when these were not available in a family’s native language. The authors also documented 20 cases of discrimination, most stemming from language requirements written into eligibility criteria, and conclude that routine translation, professional interpretation, and dedicated support structures are needed for equitable access regardless of a child’s native language or nationality.
Publication:Therapeutic Innovation and Regulatory Science
Date Published: June 10, 2026
Description: Participants are rarely informed of the possibility that a trial may stop early. Here, Nora Hutchinson, Luke Gelinas, and Barbara Bierer argue that disclosure during the informed consent process would support participant decision-making, temper concerns if termination does occur, and promote transparency and trust in the research enterprise.
Description: The MRCT Center submitted a public comment to FDA on the request for information on the proposed “Expedited Investigational New Drug Pilot Program.” The MRCT Center comments support FDA’s goal of accelerating first-in-human studies but caution that the proposed pilot could unintentionally add complexity, cost, and potential conflicts of interest unless QRI roles, standards, accountability, and interactions with FDA are clearly defined and standardized. The submission recommends a more flexible and inclusive QRI model, stronger safeguards for independence and confidentiality, a centralized review platform, transparent performance metrics, and evaluation based on end-to-end development timelines and participant safety rather than speed alone.
Comments provided to: Office of Inspector General (OIG) and Department of Health and Human Services (HHS)
Description:August 24: The MRCT Center submitted a public comment to the Office of Inspector General (OIG) and the Department of Health and Human Services (HHS) in response to their request for information on examining whether existing fraud-and-abuse regulations appropriately balance the commitment to program integrity with advancing biomedical research and ensuring access to clinical trials. The MRCT Center fully endorsed the adoption of an AKS safe harbor that permits clinical trial sponsors and other funders to provide funds to compensate and cover costs incurred by clinical trial participants without raising AKS or Beneficiary Inducement CMP concerns.
Comments provided to: Office of Management and Budget (OMB)
Description: The MRCT Center submitted a public comment on the Office of Management and Budget’s proposed rule revising the Uniform Guidance for federal financial assistance (2 C.F.R. Part 200), Docket No. OMB-2026-0034. The comment addresses the two provisions that would most affect international clinical research. It agrees that competent U.S. entities should have priority when research can be done well here, but cautions that the proposed “domestic-first framework” would bar the subawards, procurements, and other international elements on which many trials depend for enrollment, biomaterials, equipment, and specialized expertise. It further argues that the proposed prohibition on collaborations with “covered foreign countries” and “covered foreign entities” is undefined and unworkable in practice. Both provisions rest on amorphous terms such as “national interest,” duplicate existing scientific review, and would chill legitimate international collaboration to the detriment of U.S. science and clinical medicine. The MRCT Center urges OMB to give considerably more consideration to the meaning, interpretation, and implementation of any rule of this kind.
Comments provided to: TEHDAS2, coordinated by the Finnish Innovation Fund SITRA
Description: The MRCT Center submitted responses to several TEHDAS2 draft guidelines to Health Data Access Bodies offered for public consultation, including one on international and third country access and transfer of electronic health data; on linkage of health data sets; on informing citizens about the use of health data; and on handling research outcomes. We continue our efforts to engage the stakeholders and guide policy development towards a harmonized data-sharing system for secondary use purposes related to the European Health Data Space (EHDS).
Comments provided to: European Data Protection Board
Description: The MRCT Center submitted a public comment in support of the European Data Protection Board’s Guidelines 1/2026 on the processing of personal data for scientific research purposes. The comment welcomes the Guidelines as a meaningful step toward clearer interpretation of the GDPR’s research provisions, endorsing the presumption of compatibility for secondary research, broad consent, and a research-specific approach to controller and processor roles. It also asks the EDPB to clarify how the Guidelines interact with the European Health Data Space, to allow GDPR data-processing consent to be included in the clinical trial consent form, to provide clearer examples of when erasure may compromise a trial, and to provide a workable pathway for sharing pseudonymized data.
Publication:Therapeutic Innovation and Regulatory Science
Date Published: March 18, 2026
Description: “Parents’ Perspectives on Access to Pediatric Rare Disease Cross-Border Clinical Trials in Europe: Experiences of Language Inclusion and Preferences,” published in Therapeutic Innovation & Regulatory Science and led by Begonya Nafria of the Institut de Recerca Sant Joan de Déu with co-author Barbara Bierer, surveyed parents of children living with a disease about access to cross-border trials in Europe. Of 1,436 responses, 10% of families had participated in a clinical trial, and 30.1% of those had traveled abroad to do so. Among parents whose children were excluded from cross-border trials, roughly one-third cited language or country barriers as the reason.
Comments provided to: NIH National Library of Medicine
Description: The MRCT Center submitted a public comment in response to the NIH National Library of Medicine’s request for information, “Future Directions for the NLM Information Resource Grants (G08) Program.” The comment affirms strong support for NLM’s mission and the trustworthy resources it offers through PubMed, ClinicalTrials.gov, GenBank, and MedlinePlus, and proposes several ways to expand the G08 program with a focus on clinical trials and research. It recommends deploying libraries and librarians as trusted access points that help the public find and interpret research information; extending these resources to rural and historically underserved areas as entry points for digital trial methods and study recruitment; improving the utility of ClinicalTrials.gov through electronic health record integration for point-of-care trial matching, more complete eligibility criteria, plain-language trial summaries, and machine-readable protocol templates; and providing educational resources on the responsible and practical use of AI technologies. The comment urges NLM to foster an AI-ready research information ecosystem grounded in data interoperability standards, computable metadata, benchmark datasets, FAIR research outputs, and systems that protect personal health information.
