• Regulatory NewsRegulatory News

    Working group: More guidance needed on MRD in myeloma trials

    A working group formed to consider the use of minimal residual disease (MRD) in the treatment of multiple myeloma and the development of new therapies has issued a call for additional regulatory guidance on the use of MRD as a marker in clinical trials.   The working group, a collaboration of advocacy organizations, patients, research foundations, academic, government agencies and industry, was initially convened in 2016 by the Foundation for the National Institutes of...
  • Regulatory NewsRegulatory News

    CTIS set to launch in EU; sponsor guide now online

    The European Commission (EC) has affirmed that it plans to launch the Clinical Trials Information System by the end of January 2022, as part of its broader efforts to modernize and streamline the conduct and oversight of clinical trials in the EU.   “The pandemic has clearly shown the importance of efficient clinical trials that do not compromise on rigorous safety and efficacy standards,” said Stella Kyriakides, EC commissioner for health and food safety, speaking o...
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    Another drug firm faces potential fines for research non-reporting

    A small Georgia pharmaceutical company has received a notice of noncompliance from the US Food and Drug Administration (FDA) for not registering clinical trial results in a central government registry, as required by federal law.   The 26 July letter is the second issued by FDA for noncompliance with federal research reporting requirements outlined in Section 801 of the Food and Drug Administration Amendments Act of 2007 (FDAAA). If results are not reported within on...
  • Feature ArticlesFeature Articles

    RWD, RWE, and improved patient outcomes: From FDA's Mini-Sentinel to real-world examples

    Uses of real-world data and evidence (RWD and RWE) have proliferated in recent years, driven by advances in data-gathering tools and methods and enhanced understanding of the value of such data and evidence. Under the 21st Century Cures Act of 2016, Congress codified RWD and RWE in the drug development and approval process. There are many opportunities for the use of RWD and RWE in the approval, labeling, clinical use, and marketing of medical products to enhance individu...
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    FDA seeks global shift to include pregnant women in medical research

    The COVID-19 pandemic has put a spotlight on the paradox surrounding the inclusion of pregnant and breastfeeding women in medical research. Even when these women are at higher risk for severe illness and hospitalization, they are unlikely to be included in clinical trials for medical products and vaccines. In a recent article, officials at the US Food and Drug Administration (FDA) signaled their commitment to work with industry and other regulators to support the inclus...
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    MHRA announces streamlined clinical trial review process

    In the wake of the systemic changes flowing from the UK’s exit from the EU, the country’s Medicines and Healthcare products Regulatory Agency (MHRA) plans to streamline review of clinical trials for medicinal products.   Beginning in January 2022, new Clinical Trials of Investigational Medicinal Products (CTIMPs) will receive a combined review from MHRA and the UK Research Ethics Services, with collaboration from the UK’s Health Research Authority (HRA). The process is...
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    FDA finalizes guidance on studying CNS metastases in cancer trials

    A final guidance document from the US Food and Drug Association (FDA) provides recommendations for drug developers on how best to evaluate the safety and efficacy of cancer drugs in patients with central nervous system (CNS) metastases. The document finalizes a draft guidance issued in August 2020.   "There are few effective treatments for patients with central nervous system (CNS) metastases – a type of cancer that has spread from the original tumor to the CNS and is ...
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    PIC/S finalizes GMP data integrity guidance

    This week, the Pharmaceutical Inspection Co-operation Scheme (PIC/S) announced that its new guidance on good practices for data management and integrity for pharmaceutical manufacturers and distributors has come into effect.   When authorities inspect facilities of manufacturers and distributors of active pharmaceutical ingredients to ensure good manufacturing practice and good distribution practice (GMP/GDP) compliance, “The effectiveness of these inspection processes...
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    The transition to ICH M10 and its impact on global submissions

    With the expanding globalization of drug development, regulations put forth under the International Council for Harmonisation’s (ICH’s) M10 guidance support the transition to a more harmonized industry. The draft guidance marks another step of integrating international expectations, in this case, as it applies to bioanalytical evaluations. This article examines the key elements of the M10 standard and recommends strategies for successful new drug or clinical trial applicat...
  • RF Quarterly

    Synthetic data and the innovation, assessment, and regulation of AI medical devices

    Synthetic data are artificial data that mimic the properties of and relationships in real data. They show promise for facilitating data access, validation, and benchmarking, addressing missing data and under-sampling, sample boosting, and the creation of control arms in clinical trials. The UK Medicines and Healthcare products Regulatory Agency (MHRA) is using its current research into the development of high-fidelity synthetic data to develop its regulatory position on ar...
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    Acceleron, under threat of fines, reports out a negative study

    The day after receiving a first-ever threat of civil penalties for failure to report clinical trial results to ClinicalTrials.gov, Acceleron Pharma fulfilled a legal mandate by posting summary results to the federal trials website.   Though the negative results for its trial of dalantercept and axitinib in patients with advanced renal cell carcinoma had been published elsewhere years earlier, Massachusetts-based Acceleron had not previously complied with federal report...
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    FDA updates guidance on covariate treatment in clinical trials

    In a revised draft guidance, the US Food and Drug Administration (FDA) has clarified how drug developers should adjust for covariates in certain clinical trials.   The revision provides “more detailed recommendations for the use of linear models for covariate adjustment and also includes recommendations for covariate adjustment using nonlinear models,” according to FDA’s Federal Register notice of the newly revised draft.   The updates to the April 2019 draft l...