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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...
  • 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 ...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    FDA: What's Slowing Progress in Some Disease Areas?

    In a post to FDA Voice – the US Food and Drug Administration's blog – recently appointed Deputy Commissioner for Medical Products and Tobacco Robert Califf says progress toward curing some diseases, such as Alzheimer's, is being slowed by a lack of understanding of disease biology. Why are There Effective Treatments for Some Diseases and not Others? Califf raises this question, and points to some diseases like HIV/AIDS, where advances in scientific understanding hav...
  • Regulatory NewsRegulatory News

    FDA's 'Temperance Movement' Guidance Focuses on Alcoholism Endpoints

    A new draft policy issued by the US Food and Drug Administration (FDA) aims to make it easier for drug companies to develop new treatments for alcoholism by allowing them to focus on patients who give up "heavy" drinking, and not drinking entirely. Background The policy, Alcoholism: Developing Drugs for Treatment , is a draft guidance document intended to address some of the clinical development hurdles which might otherwise complicate the process of developing a dru...
  • Regulatory NewsRegulatory News

    FDA Finalizes Guidance Aimed at Accelerating Approval for Some Breast Cancer Treatments

    A new final guidance document issued by the US Food and Drug Administration (FDA) outlines the processes by which the regulator will accept surrogate endpoints to support the accelerated approval of treatments intended for high-risk early-stage breast cancer. Background Under normal circumstances, FDA requires "substantial evidence"—generally two Phase III clinical trials—indicating that a product is safe and effective at treating, preventing or curing a given condition...
  • New FDA Guidance Could See Patients Obtaining Quicker Access to Experimental Therapies

    The US Food and Drug Administration's (FDA) newly released draft guidance on neoadjuvant breast cancer treatments has the potential to radically change the existing pathway through which patients with aggressive early-stage breast cancer can obtain treatment, say experts. The guidance, released on 28 May,  is "intended to assist applicants in designing trials to support marketing approval of drugs to treat breast cancer in the neoadjuvant setting using pathologic co...
  • FDA Releases Draft Guidance for Accelerated Approval of Breast Cancer Treatments

    The US Food and Drug Administration (FDA) released new draft guidance for industry, providing an intended outline for sponsors hoping to utilize an accelerated approval pathway for drug-based preoperative breast cancer treatments. FDA's 29 May issuance of the guidance is "intended to assist applicants in designing trials to support marketing approval of drugs to treat breast cancer in the neoadjuvant setting using pathologic complete response (pCR) as a surrogate endpoin...
  • IOM Releases Report on Biomarkers and Surrogate Endpoints

    The Institute of Medicine (IOM) released a workshop summary on 14 February that explores the evaluation process for biomarkers used to detect surrogate endpoints in chronic diseases. An initial report, Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease , was requested by the US Food and Drug Administration (FDA), and released by IOM in May 2010. Biomarkers are "indicators of normal biological processes, pathogenic processes or pharmacologic response...