The delayed Clinical Trials Regulation (CTR) is set to take effect on Monday, 31 January, marking a milestone in the European Union’s attempt to improve transparency and boost the amount of human research that takes place in the region.   Currently, sponsors must file clinical trial applications to national regulators and ethics committees in each country that they want to include in their studies, then cope with multiple processes for registering their studies and pos...

Industry, medical societies and other stakeholders have weighed in on draft guidance from the US Food and Drug Administration (FDA) detailing how sponsors that want to study multiple versions of cell and gene therapies could combine them in a single “umbrella” trial.   The draft guidance, released in September 2021, outlined FDA’s proposed thinking for how this process would work. For cell and gene therapies intended to treat a single disease, sponsors would typically ...

The US Food and Drug Administration, as part of an end-of-year spate of guidance documents, has issued a draft guidance for sponsors who wish to use digital tech to aid in remote clinical data acquisition.   Digital health technologies (DHTs) stand to benefit clinical investigations in a multitude of ways, but regulatory considerations have to be weighed as the technologies are employed, noted the agency in setting out the background for the draft guidance, issued in l...

This article discusses pediatric drug development in the EU and US, with an emphasis on overcoming regulatory obstacles. The authors present the commonalities and differences between US and EU regulations and guidelines and examine the legislative acts and subsequent regulatory requirements, while outlining the necessary steps for successful implementation of pediatric drug clinical trials. They conclude with a discussion of three case studies that highlight strategic init...

The US Food and Drug Administration (FDA) hasn’t been notifying most clinical trial sponsors they are in violation of reporting requirements.   That’s according to results of a Freedom of Information Act (FOIA) investigation by Reshma Ramachandran, of the Yale School of Medicine in New Haven, CT, and colleagues in a recent Viewpoint published in JAMA .   In January 2017, a final rule issued by the National Institutes of Health (NIH) determining ClinicalTrials.go...

For drugs and biologics that receive accelerated approval, could information drawn from real-world data sources supplant traditional postapproval clinical trials to confirm that the therapies have benefit? Real-world data (RWD) are not yet robust enough to confirm the benefit of drugs awarded accelerated approval based on surrogate endpoints, answered the authors of a recent study.   “The findings of this cross-sectional study suggest that none of the 50 [Food and Drug...

Friends of Cancer Research is calling on all stakeholders in the cancer community to move away from the idea of “more is better” in drug dosing and begin to incorporate dose-finding studies into the cancer trial process.   Dose optimization is the focus of a new white paper that the group released at its 25 th annual meeting. The paper offers recommendations for performing adequate dose optimization studies in oncology, with a focus on the pre-market setting. “These...

A new electronic information system for clinical trial reporting in the EU is one step closer to launch with the European Medicines Agency’s (EMA’s) Thursday publication of a go-live planning document.   The 12-page document covers key steps remaining before the launch of the Clinical Trial Information System (CTIS), planned for 31 January 2022, and follows an April 2021 audit by EMA’s management board that confirmed that CTIS is “fully functional and meets the agreed ...

The International Council for Harmonization (ICH) recently announced that the E8(R1) guideline on general considerations for clinical studies had reached Step 4 and is awaiting sign-off by regulators. The widely anticipated guideline modernizes the design, conduct and reporting of clinical trials by adopting quality by design principles. The guideline incorporates “current concepts achieving fit-for-purpose data quality.”   ICH announced that “the modernisation of IC...

The European Commission (EC) has laid out recommendations on best practices for preparation, translation and dissemination of lay summaries of clinical trial information, intended both for trial participants and the general public, in a newly published document.   The EC’s Clinical Trials Expert Group (CTEG) adopted the guidelines in July 2021; the 85-page document walks users through the lay summary (LS) process from the planning and scoping stage, through the develop...

The U.S. Food and Drug Administration has released draft guidance for clinical trial investigators outlining their safety reporting duties for investigational drugs and devices.   The draft , published on 29 September 2021, is focused exclusively on the role of investigators – not sponsors – and merges past final guidance from 2009 and 2012 on safety reporting that was previously mixed in with advice to sponsors. The prior guidance will remain in effect until the new ...

In mid-2021, a survey was launched to evaluate where European medical device manufacturers and pharmaceutical companies see the greatest challenges in clinical trials and which trends they envision in this area in coming years. The top challenges included lack of personnel, time investments, complexity of the study protocol, the identification of suitable subjects, and data analysis and publication. Although most participants had not yet conducted decentralized trials, mor...