• Regulatory NewsRegulatory News

    Five Years of Cancer Drug Approvals: Pricing, Novelty and Improvement

    Mark Shapiro, vice president of clinical development at contract research organization Clinipace, considers a recent JAMA study on cancer drug pricing in a guest post for  Focus . A rebuttal to the post by one of the authors of the JAMA study, Dr. Vinay Prasad, can be found here . The last five years have been exceptionally busy in terms of oncology drug development. Two interesting articles that reflect back on this period of time were recently published. The firs...
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    precisionFDA Launches to Help With NGS Assay Validation, Data Sharing

    In the spirit of sharing, the US Food and Drug Administration (FDA) on Tuesday launched precisionFDA, a new online, cloud-based platform where genomics experts from industry, academia, government and elsewhere can come together and securely share data on next-generation sequencing (NGS). The beta-launch of the site opens the door for accessing and sharing datasets, analysis pipelines, bioinformatics tools and other approaches that also could advance regulatory science. ...
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    FDA Officials Review Algorithms Used for NGS Analysis

    A group of scientists from the US Food and Drug Administration's (FDA) Division of Bioinformatics and Biostatistics have reviewed the algorithms used to align the data generated by next-generation sequencing (NGS) tests to reference genomes. NGS tests hold great promise for precision medicine due to their ability to quickly sequence the human genome and identify thousands of genetic variants. By identifying genetic variants that are associated with different diseases, r...
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    Evaluating Next-Generation Sequencing: From Design Concept to Performance Standards

    Discussions are underway on how the US Food and Drug Administration (FDA) plans to develop new ways of regulating next-generation sequencing (NGS) diagnostics. Kicking off a two-day public workshop today, FDA Commissioner nominee Robert Califf said that NGS tests hold great promise for patients, but still present "tremendous uncertainty." The issue, he said, is determining "how accurate [and] how useful can we make the predictions that come from these tests." Califf als...
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    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...
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    FDA Science Board Calls on Agency to Find New Ways to Stimulate Biomedical Innovation

    The US Food and Drug Administration (FDA) has made “significant strides” in strengthening its scientific capabilities, though there is still some concern about the agency’s ability to support and stimulate biomedical innovation, according to a new report from a subcommittee of FDA’s Science Board. Background Six years after a 2007 report from the Science Board -- which, among other findings, called attention to FDA’s “weak scientific base and inadequate scientific wor...
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    FDA Accepts for Review First NDA for a 'Digital' Medicine

    The US Food and Drug Administration (FDA) has accepted for review what is being considered the first new drug application (NDA) for a combination product to include a sensor embedded into the tablet. The developer of the sensor – Proteus Digital Health – said it could help improve patient medication adherence and better inform physician decision-making to tailor treatments to the patient's needs. In the case of this new drug application, the sensor is paired with Otsuka...
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    FDA Looks to Develop Regulatory Strategies for Genomic Testing

    To further the president's Precision Medicine Initiative (PMI), the US Food and Drug Administration (FDA) is working to develop regulatory systems for diagnostics using next generation sequencing (NGS) technologies. Background Since 2014, FDA has sought input from the public on how it could develop a regulatory system appropriate for reviewing and regulating NGS technologies. The agency says it believes such a system "could potentially be applied to many other types o...
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    precisionFDA: A Crowd-Sourced, Cloud-Based Platform for Precision Medicine

    The US Food and Drug Administration (FDA) has announced the launch of a new crowd-sourced, cloud-based platform, precisionFDA, to enable developers of genomic sequencing diagnostics to share data and methodologies. precisionFDA In a post to FDA Voice Wednesday, FDA Chief Health Informatics Officer Taha Kass-Hout and Policy Advisor for the Office of In Vitro Diagnostics and Radiological Health David Litwack laid out the plan and goals for precisionFDA. The new platfo...
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    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...
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    DSHEA: Preserving Legal Access to Chinese Herbal Medicine or Researching new Remedies for Malaria and Other Tropical Diseases

    This article explores the importance of continued access by researchers to Chinese medicinal herbs via DSHEA, in light of a potential increase in tropical diseases in historically non-tropical regions. Preserving access to traditional Chinese medicine will become important as tropical diseases gradually become endemic to the United States. As the climate warms 1 and mosquitos proliferate, 2 diseases formerly confined to the tropics are moving north. 3 Si...
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    Leverage Purchasing Power to Save Billions on Medicines and Devices, NHS Urged

    According to an independent report commissioned by the UK's Department of Health, the National Health Service (NHS) could save as much as £5 billion (GBP) per year by 2020 through medicines optimization and leveraging NHS buying power to save on medical devices and consumables. Improving Efficiency Last year, Health Secretary Jeremy Hunt appointed Lord Carter, member of the House of Lords (Labour) to chair the NHS Procurement and Efficiency Board. In his capacity as cha...