• Regulatory NewsRegulatory News

    FDA draft guidance addresses clinical development of drugs to treat early Lyme disease

    The US Food and Drug Administration (FDA) Center for Drug Evaluation Research (CDER) has issued a draft guidance to help sponsors develop new drugs to treat the early stages of Lyme disease.   The draft addresses criteria for clinical trial enrollment, efficacy endpoints, and clinical microbiology considerations for treating early stages of the disease when manifested by erythema migrans (EM), a rash that is often one of the first symptoms.   In North America, Lyme...
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    Industry calls for flexibility in FDA’s pediatric measurement guidance

    A pharmaceutical trade group and several drugmakers urged the US Food and Drug Administration (FDA) to take a more flexible approach in its guidance on pediatric measurements in clinical trials and questioned whether 12 months is sufficient to provide meaningful information for growth and development.   These comments were made in response to the agency’s call for feedback on the draft guidance, which outlines ways sponsors can measure pediatric growth consistently in ...
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    ICH E11A: Pharma groups want more information on data extrapolation, pediatric biomarkers

    Two US pharmaceutical industry groups note that they are generally supportive of the International Council for Harmonization (ICH) guideline on pediatric extrapolation in drug development but suggest that the guidance should address a broader subset of the population and not just focus on extrapolating data from adults. The groups called for more information on using biomarkers for the pediatric population and for including the estimands concept in the final guidance.   ...
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    FDA draft guidance aims to ensure accurate measurements of pediatric growth

    Joint draft guidance from two centers at the US Food and Drug Administration (FDA) outlines ways sponsors can measure pediatric growth consistently in clinical trials for drugs used to treat both rare and common diseases.   “If an investigational drug or biological product may affect growth or pubertal development, then accurate, serial measurement and recording of growth parameters are essential for data interpretation in pediatric clinical trials,” the agency announc...
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    Neonatal drug development addressed in FDA final guidance

    The US Food and Drug Administration (FDA) issued final guidance this week to assist sponsors in developing clinical pharmacology studies for neonatal populations. The guidance is meant to address gaps in neonatal labeling and encourage the development of therapies that are “unique to neonates.”   The guidance is tailored to sponsors developing these studies for investigational new drug applications (INDs), new drug applications (NDAs), biologics license applications ...
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    RWD in clinical trials: External control arms take the lead

    The US Food and Drug Administration (FDA) has now published four draft guidances spelling out how it will consider real-world data (RWD) in regulatory decision-making, from the use of electronic health records and registries to RWD in non-interventional studies. More guidance documents are planned, including details on study designs that incorporate RWD for external control arms.   External control arms, which use data collected outside of the current trial to provide ...
  • 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...
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    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...
  • Regulatory NewsRegulatory NewsRAPSRAPS' Latest

    Orphan drug experts discuss new book on developing rare disease treatments

    Developing therapies for rare diseases involves complex incentives, unique requirements, and often extensive patient engagement. The authors of RAPS’ Orphan Drug Development for Rare Diseases , Sundar Ramanan, PhD, MBA, and Sunny Kamlesh Dave, MPharm, recently took time to discuss with RAPS Senior Editor Gloria Hall the importance of orphan drugs, the peculiarities and challenges of obtaining an orphan drug designation (ODD), and what is covered in the book. During a “m...
  • RF QuarterlyFeature 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 QuarterlyFeature 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...