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    China Regulatory Reform: An Update on Review Timelines and Drug Lag

    This article providesthree case studies with an assessment ofthe impact ofthe regulatory changes and insight intothe importance of including China in a global strategy for clinical development and drug registration. Introduction China has implemented a series of regulatory changes and improvementsthrough a comprehensive regulatory reform starting in August 2015.the largely shortened new drug review timeline provides one ofthe biggest impacts forthe pharmaceutical indust...
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    Japan's Conditional Approval Pathway for Regenerative Medicines

    This article discusses the history, reasoning, legal decisions and subsequent regulatory requirements behind Japan's bid to become a world leader in regenerative medicine. The authors outline the process for seeking new regenerative product approvals through an accelerated regulatory pathway and spell out the oversight role played by Japan's regulatory authorities to enable new regenerative, cell-based therapies to be made available to the public in Japan as soon as possi...
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    Regenerative Medicine Advanced Therapy Designations

    This article discusses recent legislative action regarding Regenerative Medicine Advanced Therapies (RMAT). The author describes the criteria RMAT candidate treatments must meet to apply for RMAT designation from the US Food and Drug Administration (FDA) and outlines the steps applicants must take to gain RMAT designation from FDA. She compares the processes and success rates for applying for and gaining RMAT designation to the processes and success rates for gaining "bre...
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    Providing Early Access to Innovative Medicines in the UK

    This article discusses the variety of steps and agencies involved in providing patients in the UK with serious and/or life-threatening illness early access to promising innovative medicines. The author reviews and spells out how designations to Promising Innovation Medicines (PIM) are gained and the schemes under which early access may be granted. Introduction In February 2018, the UK's Medicines and Healthcare products Regulatory Agency (MHRA) issued a press release an...
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    PMA Advisory Panels: The Impact of FDA's Change in Policy on Voting Pattern

    This article provides a statistical analysis of Premarket Approval Application (PMA) data illustrating some new and interesting insights into the advisory panel process. Introduction The Food and Drug Administration (FDA) issues numerous guidance documents, policies and similar statements that apply to devices. These documents are not legally binding. Nevertheless, many of these documents are widely cited and incorporated into decision-making, both by FDA and industry. ...
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    FDA Regenerative Medicine Policy Framework and Advanced Therapy Designation

    This article discusses the policy framework established by the US Food and Drug Administration (FDA) for the regulation of regenerative medicine and advanced therapies. The four guidance documents supporting regenerative medicine regulation are summarized against the background of FDA's regulation of human cells, tissues and cellular and tissue-based products. Introduction In November 2017, FDA published a comprehensive Regenerative Medicine Policy Framework to stimulat...
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    Temporary Importation of a Prescription Pharmaceutical Product

    This article offers a hypothetical scenario in which a company manufacturing a drug to treat a rare genetic disease must cease manufacturing because of a serious manufacturing problem. The authors discuss step-by-step how the company should obtain an exemption from FDA to temporarily import the same product approved for use in another country, thereby providing US patients with their drug during the interim drug shortage caused by a manufacturing issue in its US facilitie...
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    Gene Therapy Harmonization Milestones and Challenges

    This article provides a brief history of gene therapy and looks at gene therapy regulatory harmonization efforts worldwide, with an emphasis on regulation in US, EU, Japan and China. It also reviews developments regulatory professionals should look for in the near future and serves as a "call-to-action" for continued engagement in developing transnational gene therapy regulatory harmonization. Introduction Ready or not, gene therapy has arrived. Since the first gene the...
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    Expanded Access Policy Requirements, Orphan Drug Legislation and Gene Therapy Regulatory Challenges

    Articles posted throughout April include a look at how to comply with new expanded access requirements and programs, expanded access programs for medical devices, orphan drugs, regulatory and economic challenges for gene therapies, re-designing regulatory capabilities to improve operational efficiency and a review of US and EU veterinary device regulations. Expanded Access Policy Requirements Regulatory manager, Matt Medlin and regulatory intelligence expert, Meredit...
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    Expanded Access and Rare Diseases: FDA's New Initiatives

    This article highlights developments in providing expanded access to treatments for rare diseases by outlining legislative progress as well as the US Food and Drug Administration's (FDA's) efforts to streamline regulatory requirements. The authors review benefit-risk evaluation, offer step-by-step procedures for physicians and patients seeking expanded access and make recommendations for pharmaceutical companies with drugs in development that may be candidates for expande...
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    Australia's Orphan Drugs Program

    This article discusses recent changes to Australia's Orphan Drug Program, including eligibility criteria as well as the designation process for applications and limits on the designation period. Preliminary review of the first eight months indicates the number of designations has reduced significantly. Background Following the successful introduction of an Orphan Drugs Program in the US, and plans to introduce a similar program in Europe, Australia introduced an Orphan ...
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    Expanded Access Programs for Medical Devices

    Expanded access, also referred to as compassionate use, is a vital pathway for patients with life-threatening diseases to gain early access to unapproved investigational medical devices. This article summarizes expanded access programs for medical devices in the US, Europe, Japan, Canada and Australia. Background The average time from medical device concept to marketing approval has been reported as three to seven years. 1 This long-time lag is problematic for patients...