• Regulatory NewsRegulatory News

    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 The document finalizes a draft guidance issued in August 2020.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...
  • Feature ArticlesFeature Articles

    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 The draft guidance marks another step of integrating international expectations, in this case, as it applies to bioanalytical evaluations.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...
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    New guidance for developers of CINV prophylaxis shifts endpoints

    New draft guidance from the US Food and Drug Administration (FDA) updates which endpoint assessments and evidence are needed to show efficacy for medicines that treat chemotherapy-induced nausea and vomiting (CINV).   The guidance shifts away from the previous approach, where sponsors would choose a primary efficacy endpoint of complete response, which was defined as no vomiting and no use of rescue antiemetic medication. Direct evaluation of nausea severity and freque...
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    Euro Convergence: EU pediatric medicines regulatory framework needs simplification, optimization

    Industry would like to see some “pragmatic” changes to the regulatory process for pediatric medicines in the EU, according to a recent presentation at RAPS Euro Convergence 2021.   These changes include incorporating scientific discussion throughout a product’s life cycle, comparable reporting requirements for pediatric trials to those for adult trials, and optimization and simplification of pediatric investigation plan (PIP) procedures and compliance checks, said Thom...
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    Global regulators issue call for clinical trial data transparency

    World regulatory authorities are calling for increased transparency from the pharmaceutical industry in how they report and give access to clinical trial data. In a joint statement, the World Health Organization (WHO) and the International Coalition of Medicines Regulatory Authorities (ICMRA) cited need for “wide access to clinical data for all new medicines and vaccines.”   Data related to a therapy or vaccine “must be published at the time of finalization of the regu...
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    FDA threatens drugmaker with fines for failing to report trial results

    Acceleron Pharma became the first drugmaker to face potential fines from the US Food and Drug Administration (FDA) for failing to report clinical trial results to ClinicalTrials.gov.   In a notice of noncompliance sent to Acceleron on 27 April, FDA notified the Cambridge, MA-based drugmaker that it has 30 days to correct the issue or face up to $10,000 per day in civil monetary penalties or other regulatory action including injunction or criminal prosecution.   Mat...
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    Older adults, Black patients underrepresented in post-marketing studies

    In postmarketing studies of cancer therapies approved by the US Food and Drug Administration (FDA), representation of older adults and Black patients does not appear to be improved compared with premarketing studies, a recent investigation in JAMA Network Open suggests.   In addition, the sex and race of participants were identified less frequently in postmarketing studies than premarketing studies, according to a cross-sectional analysis of 77 premarketing and 56 po...
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    EU clinical trial portal and database declared functional

    The European Medicines Agency (EMA) announced that its long-delayed EU clinical trial portal and database are fully functional, paving the way for the launch of the EU Clinical Trial Information System (CTIS) and the application of the EU Clinical Trial Regulation early next year.   The CTIS was originally expected in September 2018; however, a series of delays have pushed back the launch date for the system, and the EU Clinical Trial Regulation it is intended to suppo...