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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...
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    Expanded Access Programs: Making Compassion Work

    This article discusses issues and efforts related to expanded access programs aimed at providing patients with quicker access to new and experimental drugs, many of which are potentially lifesaving. The perspectives of health authorities, industry and patients are explored as well as the historical legal and ethical issues involved in ensuring expanded access drugs are safe and efficacious. Introduction In the late 1980s and early 1990s, the HIV/AIDS emergency prompted ...
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    Orphan Drug Development Progress

    This article discusses how orphan drug development has benefitted from a decade of collaboration between FDA and EMA and how other stakeholders have continued to help accelerate the progress after FDA and EMA joined forces to establish an "orphan cluster group" aimed at stimulating the development of orphan drugs. The author reviews how the orphan drug designation has expanded since 2008 and how the number of new drugs available to patients with rare diseases has increase...
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    Regulatory and Economic Challenges for Gene Therapy Utilization

    This article addresses pricing and reimbursement challenges associated with clinical utilization of approved gene therapies. The authors recommend potential approaches to resolve these challenges, the implementation of which may help solve issues related to the high cost of these therapies and encourage continued innovation and further development of advanced gene therapies. Introduction Gene therapy is an experimental technique using genes to treat or prevent diseases ...
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    US and EU Veterinary Medical Device Regulation

    This article describes regulatory efforts applied to veterinary medical devices in the US and EU and explores a small, descriptive sample of veterinary medical devices available on the US market to stimulate growth, innovation and dissemination throughout Europe. Introduction The effects of drugs and medical treatment in animals may vary from drug-to-drug and from species-to- species because of physiological and other species differences. In addition, the effectiveness ...
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    Public Availability of Expanded Access Policy: how to Comply With new Requirements

    This article discusses the process for complying with expanded access policy requirements. Introduction The unexpected passage of the 21st Century Cures Act (the act) in December 2016 has become in terms of job security for the Regulatory Intelligence (RI) professional, a gift that keeps on giving. Many companies, even now, continue to grapple with analysis, planning, integration and compliance with various policy and regulatory components of the 966-page piece of leg...
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    Re-Designing Regulatory Capabilities

    Faced with growing regulatory burdens, Bristol Myers Squibb (BMS) and Halozyme each committed time and resources to connect regulatory functions from end-to-end with the goal of improving efficiency and transparency. This article describes how two very different companies made their business case, streamlined processes and managed change. The article also provides elements of what the two companies learned along the way. Introduction Life sciences companies are driving ...
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    US Orphan Drug Legislation: Current Status and Recent Developments

    This article discusses the impact of recent orphan drug legislation and regulatory developments and covers the Food and Drug Administration Reauthorization Act of 2017 (FDARA), the Orphan Drug Modernization Plan and the 21st Century Cures Act. The authors answer questions regarding what drugs qualify as an orphan drug and how an orphan drug gets approval. Introduction While they have had legal existence for close to 35 years and often served as a regulatory strategy for...
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    Regulatory Operations, Resources and eCTD Benefits

    Articles posted throughout March included in-depth coverage on the use of electronic Common Technical Documents (eCTDS), regulatory resources and operations, FDA's citizen petition process and ASEAN harmonization efforts. eCTD Update The deadline after which all Investigational New Drug Applications (INDs), Drug Master Files (DMFs) and their amendments will be required to be both in Common Technical Document (CTD) format and submitted electronically (collectively called...
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    eCTD: A Global Regulatory Overview

    This article outlines the global status of electronic Common Technical Document (eCTD) and its implementation impact on companies. The author provides an international overview and focuses on regulatory eCTD submission requirements and global harmonization for medicinal products for human use in Canada, Europe, Japan and the US. Tips for organizing common technical documents are included. Introduction The electronic Common Technical Document (eCTD) is an international s...