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  • Regulatory NewsRegulatory News

    Studies Raise Questions on Trial Designs for New Drugs, Devices Sped to Market

    Two new articles and an accompanying editorial from former US Food and Drug Administration (FDA) commissioner Robert Califf published Tuesday in JAMA raise questions about the risks and clinical trial designs of new drugs and medical devices sped to market without enough preliminary evidence that they are effective. In one of the articles, which evaluated the use of FDA’s accelerated approval pathway, questions were raised on the use of surrogate measures as outc...
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    Novel Trial Endpoints Generated by Mobile Tech: CTTI Offers Recommendations

    The Clinical Trials Transformation Initiative (CTTI), a public-private partnership established by Duke University and the US Food and Drug Administration (FDA), on Monday released new recommendations on developing novel clinical trial endpoints generated by mobile technology. Such mobile technology can offer new ways to collect objective measurements of clinical trial participants as they go about their daily lives. And though the use of such novel endpoints, whi...
  • Regulatory NewsRegulatory News

    FDA Drug Approvals on Limited Evidence: Follow-Ups Lacking, Study Finds

    For drugs approved by the US Food and Drug Administration (FDA) based on limited evidence, a recent study published in the British Medical Journal found “noticeable variability” in the degree to which novel drugs were studied in the postmarket period. The authors, including Yale University professors Harlan Krumholz and Joe Ross, urged FDA to take a cautious approach in increasing its reliance on surrogate markers, smaller and shorter trials, and evidence derived ...
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    European Regulatory Roundup: EMA Drafts Guidelines on Controlling False Positives in Clinical Trials (6 April 2017)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. EMA Drafts Guidelines on Controlling Against False Positives in Clinical Trials The European Medicines Agency (EMA) has released draft guidelines about multiplicity in clinical trials. The text deals with how to mitigate the risk of false positives arising when clinical trials look at multiple treatment groups and endpoints. EMA’s guideline is applicable to most clini...
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    Multiple Endpoints in Clinical Trials: Biopharma Companies Seek More From FDA Draft Guidance

    Biopharmaceutical heavyweights – from Novartis to GlaxoSmithKline to Regeneron – are seeking additional clarification from the US Food and Drug Administration (FDA) on draft guidance on multiple endpoints in clinical trials, according to comments posted to the docket on Thursday. Background On 12 January, FDA issued a 54-page draft guidance to provide sponsors and review staff with the agency’s thinking on the problems posed by multiple endpoints in the analysis and i...
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    Multiple Endpoints in Clinical Trials: FDA Issues Draft Guidance

    The US Food and Drug Administration (FDA) on Thursday released draft guidance for industry on the problems posed by multiple endpoints in the analysis and interpretation of study results and how these problems can be managed in drug and biologic clinical trials. The 54-page draft guidance looks to provide greater detail than ICH’s E9 Statistical Principles for Clinical Trials and clarify when and how multiplicity due to multiple endpoints should be managed to avoid re...
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    Ulcerative Colitis Drugs: FDA Offers Draft Guidance on Clinical Development

    Pharmaceutical companies looking to understand FDA’s current thinking on efficacy endpoints for clinical trials to develop new ulcerative colitis (UC) treatments will be interested in new draft guidance released Friday. And though the guidance spells out what primary endpoints should be used, the agency also bemoans the fact that there are not well-defined and reliable clinician-reported, patient-reported and observer-reported outcome instruments for clinical trials. Ba...
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    Researchers Propose Framework to Validate Surrogate Endpoints

    A team of researchers and health officials, including European Medicines Agency (EMA) Executive Director Guido Rasi, are proposing a three-step validation process to enhance the use of surrogate endpoints in regulatory and reimbursement decision-making. The issue, they say, is that poorly validated surrogate endpoints can lead to products being approved or reimbursed that have little or no benefit, and in some cases even harm patients. In recent years, drugmakers have i...
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    FDA Revises Draft Guidance on COPD Drug Development

    The US Food and Drug Administration (FDA) on Thursday released a revised version of its draft guidance intended to help pharmaceutical companies develop products to treat the lung disease known as chronic obstructive pulmonary disease (COPD). Specifically, FDA says the guidance is meant to assist sponsors in determining the efficacy of new drugs to treat the disease in Phase III clinical trials, and includes new information on using health-related quality-of-life instrum...
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    Pharma Companies Call on FDA to Make Clinical Outcome Assessment Pilot More Useful

    More than a dozen drug developers, including Pfizer, Genentech, Sanofi and GlaxoSmithKline, and industry groups are calling on the US Food and Drug Administration (FDA) to ensure that a clinical outcome assessment (COA) pilot program includes more useful information for researchers. Background The pilot project, first proposed by FDA in January , is intended to help industry with a starting point for considering how certain COAs might be used in clinical trials and ear...
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    Researchers Question Consistency of FDA’s Use of Surrogate Endpoints for Drug Approvals

    The US Food and Drug Administration (FDA) is not consistent with its use of surrogate endpoints in assessing the risks and benefits of treatments for some diseases, making it difficult to assess the treatments' actual clinical effects on outcomes important to patients, according to researchers from Johns Hopkins. The researchers came to this conclusion after evaluating 58 FDA drugs approved between 2003 and 2012 for chronic obstructive pulmonary disease (COPD), Type 1 an...
  • Regulatory NewsRegulatory News

    Researchers Question FDA's Use of Surrogate Endpoints for Cancer Drug Approvals

    Two researchers are questioning the US Food and Drug Administration's (FDA) use of surrogate endpoints in approving new oncology drugs and the agency's failure to more strictly enforce postmarketing study requirements for these drugs, according to a letter appearing in JAMA Internal Medicine . Drugmakers have increasingly relied on surrogate endpoints to support the approval for new cancer drugs. Instead of demonstrating improvements in overall survival, often called "t...