• Feature ArticlesFeature Articles

    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. ...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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 ...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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 ...
  • Feature ArticlesFeature Articles

    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...
  • Feature ArticlesFeature Articles

    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 ...
  • Feature ArticlesFeature Articles

    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 ...