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  • Feature ArticlesFeature Articles

    April’s Regulatory Focus: Specialist vs. generalist, RI, and more

    Feature articles during April examined the roles of the regulatory specialist and generalist and the application of regulatory intelligence (RI) in managing the new EU In Vitro Diagnostic Medical Devices Regulation (IVDR), developing an in-house regulatory database in the oncology setting, and strategic planning. Also included are articles on expedited regulatory pathway options, selecting control groups in pediatric clinical trials, and the role of artificial intelligence...
  • Regulatory NewsRegulatory News

    ICH drafts guideline on pediatric extrapolation in drug development

    The International Council for Harmonization (ICH) on Tuesday released a draft ICH E11A guideline that establishes a framework for extrapolating drug development data from adult trials to the pediatric population.   “This new ICH guideline aims to promote international harmonization of methodologies and strategies to incorporate paediatric extrapolation into overall drug development plans and to improve the speed of access to new drugs for paediatric patients, while l...
  • Feature ArticlesFeature Articles

    Considerations for selecting control groups in pediatric clinical trials

    ICH E10 provides specific guidance on the choice of control groups for clinical trials, but there are additional considerations for choosing a control group for a pediatric trial. In this article, the author looks at different types of control groups and the specific challenges that present when considering the design for a pediatric trial, such as the medical condition being treated, the potential patient population, suitability of comparators, and availability of real-wo...
  • Feature ArticlesFeature Articles

    Expedited pathways: Breakthrough therapy designation, PRIME, and innovative licensing & access

    Abstract Over the last decade, regulators have introduced initiatives to reduce time to approval for medicines that address an unmet medical need. This article compares the US Food and Drug Administration’s (FDA’s) breakthrough therapy designation (BTD), the European Medicine’s Agency’s (EMA’s) PRIority MEdicines (PRIME) scheme, and the innovative licensing and access pathway (ILAP) from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and reviews th...
  • RF Quarterly
    Feature ArticlesFeature Articles

    RF Quarterly, December 2021: Key takeaways from RAPS Convergence 2021

    Welcome to the December issue of RF Quarterly which focuses on key takeaways from RAPS Convergence 2021, including patient-focused approaches in regulatory decision making and drug development, the transition from convergence to harmonization for global pharmaceutical regulatory requirements, pediatric drug development, and regulatory and clinical strategies.   Patient perspective and global harmonization There is increasing recognition of the importance of patients...
  • RF Quarterly
    Feature ArticlesFeature Articles

    Pediatric drug development: Increasing success and overcoming obstacles

    This article discusses pediatric drug development in the EU and US, with an emphasis on overcoming regulatory obstacles. The authors present the commonalities and differences between US and EU regulations and guidelines and examine the legislative acts and subsequent regulatory requirements, while outlining the necessary steps for successful implementation of pediatric drug clinical trials. They conclude with a discussion of three case studies that highlight strategic init...
  • Feature ArticlesFeature Articles

    The RACE is on: Opportunities in pediatric oncology product development

    Pediatric oncology patients continue to have significant unmet medical need. Although the Pediatric Research Equity Act (PREA) mandated evaluation of new drugs for pediatric patients, the rarity and uniquity of pediatric cancers allowed for waivers or exemptions from PREA requirements. The passing of FDA Reauthorization Act amendments in 2017 included the Research Acceleration for Cure and Equity Act, which expands the scope of oncology products subject to PREA. Sponsors d...
  • Feature ArticlesFeature Articles

    Global pediatric development: Challenges, potential solutions, and opportunities

    The US and EU require sponsors to evaluate their drugs for use in children. The low prevalence of many conditions in children means sponsors commonly want to conduct one pediatric development program that satisfies the requirements of both the US Food and Drug Administration (FDA) and European Medicines Agency (EMA). However, multiregional pediatric development is challenging. This article will describe some of the challenges associated with pediatric development, offer po...
  • Feature ArticlesFeature Articles

    Strategies for pediatric clinical trials and drug development

    This article discusses the considerations for designing and conducting pediatric clinical trials from a pediatrician’s perspective. The author covers pediatric subpopulations, issues related to age, endpoints, and pharmacokinetic considerations, while presenting practical solutions and ethical considerations. Patient and family‒centric approach to studies and engagement with patient advocacy groups are also discussed. The author concludes with some recommendations about pe...
  • Feature ArticlesFeature Articles

    Regulation of cell and gene therapy products in Japan

    In recent years, new cell and gene therapies have been developed and approved in a number of countries, including Japan. The Japanese government fully supports academia and the pharmaceutical industry in the research and development of these innovative therapies, and in 2019, revised the Act on Securing Quality, Efficacy, and Safety of Products Including Pharmaceuticals and Medical Devices to enact the conditional early approval system for accelerating the development and ...
  • Regulatory NewsRegulatory News

    ICMRA releases framework for aligning global track and trace systems

    The International Coalition of Medicines Regulatory Authorities (ICMRA) has issued a broad set of recommendations outlining how regulators and industry can set up track and trace systems that are globally aligned to ensure counterfeit or falsified products do not infiltrate supply chains.   The report , which was issued by a joint regulators/industry working group, explores the technical features that would allow for interoperable national and regional track and trace...
  • Regulatory NewsRegulatory News

    FDA panel split on approach to COVID-19 vaccines for younger children

    Members of the US Food and Drug Administration’s (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC) disagreed as to whether the agency should grant emergency use authorizations (EUAs) for COVID-19 vaccines for children at a 10 June meeting.   Some said that EUAs were necessary to get the pediatric population vaccinated quickly to achieve herd immunity as the country emerges from the pandemic and that these vaccines should be available soon as th...