• 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 Willing to Accept Different Endpoints When Assessing Lung Cancer Drugs

    Companies will be permitted to use several types of clinical trial endpoints to show the efficacy of drugs intended to treat non-small cell lung cancer (NSCLC), the US Food and Drug Administration (FDA) confirmed in a new guidance document released this week. The final guidance document, Clinical Trial Endpoints for the Approval of Non-Small Cell Lung Cancer Drugs and Biologics , is intended to clarify the evidence FDA is willing to accept in the review of new NSCLC d...
  • 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 Background The policy, Alcoholism: Developing Drugs for Treatment , is a draft guidance document intended to address some of the clinical development hurdles which might otherwise complicate the process of developing a dru...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...
  • FDA to Assess New Regulatory Endpoints for Four Disease Areas

    The US Food and Drug Administration (FDA) has announced a four-day meeting to discuss clinical endpoints potentially capable of being used to support future drug development in four therapeutic areas. The 19-24 September workshop, entitled " Gastroenterology Regulatory Endpoints and the Advancement of Therapeutics (GREAT) ," is intended to discuss issues related to endpoints for eosinophilic esophagitis, pediatric and adult inflammatory bowel disease (IBD) and parenteral...
  • Feature ArticlesFeature Articles

    Approval of Jakafi (ruxolitinib) Based on a Home-Grown, Patient-Reported Outcome Instrument: A Case Study

    Jakafi® (ruxolitinib) was approved 1 by the US Food and Drug Administration (FDA) on 16 November 2011 for the treatment of intermediate or high-risk myelofibrosis (MF) including primary MF, post-polycythemia vera MF (PPV-MF) and post-essential thrombocythemia MF (PET-MF). Prior to this time, there were no approved products, and therefore no precedent for regulatory endpoints in this orphan disease. MF is a highly symptomatic 2 myeloproliferative neoplasm (MPN), cha...
  • 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...