• 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...
  • 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...
  • Regulatory NewsRegulatory News

    FDA reiterates support for semi-distributed tracing model under DSCSA

    The US Food and Drug Administration (FDA) recommends that trading partners use the semi-distributed model to trace drug products though the drug distribution chain because of this model’s flexibility and advantages over other data architecture models. The Drug Supply Chain Security Act (DSCSA) requires that trading partners have these systems in place by November 2023.   Connie Jung, acting associate director for policy and compliance in FDA’s Office of Drug Security, ...
  • Regulatory NewsRegulatory News

    Should the US government share the Moderna COVID vaccine recipe?

    As much of the developing world struggles to gain access to COVID-19 vaccine doses, some legal experts say the key lies in having the US government share the step-by-step instructions for manufacturing and testing the Moderna mRNA vaccine with other manufacturers.   In a recent Health Affairs blog , Christopher J. Morten, Zain Rizvi, and Ameet Sarpatwari made the case that the US government, through its contract with Moderna, has both access to the details of how the ...
  • Regulatory NewsRegulatory News

    CBO: Proposed Medicare pricing plan could curb drug development

    A new drug development model released by the Congressional Budget Office (CBO) estimates a Medicare drug pricing bill like the one proposed by Democrats in the US House of Representatives could result in between 21 and 59 fewer drugs brought to market over the next three decades.   The Elijah E. Cummings Lower Drug Costs Now Act ( H.R. 3 ), introduced in the 116th Congress during the 2019-2020 legislative session, would require the Secretary of Health and Human Servi...
  • Regulatory NewsRegulatory News

    Researchers: France could teach the US lessons in biosimilar pricing

    The US should look to the French healthcare system for lessons in reducing biosimilar pricing, according to a recent paper published in Health Affairs .   James C. Robinson, of the Division of Health Policy and Management, School of Public Health at the University of California Berkeley, and colleagues said the US “lacks consistent incentives for physicians to prescribe low-price biosimilars and for manufacturers to compete using price reductions” and that the slow up...
  • Regulatory NewsRegulatory News

    WHO revises guidance on GMPs for investigational products, R&D facilities

    The World Health Organization (WHO) recently issued a revised draft guidance to industry addressing good manufacturing practices (GMPs) for investigational drug products as well a new draft guideline addressing GMP principles for research and development facilities in the context of the COVID-19 pandemic.   The draft guidance on GMPs for investigational products was prompted by WHO’s Prequalification Team Inspections Services (PQT), which last year expressed the need...
  • Regulatory NewsRegulatory News

    FDA seeks comments on regulation of kratom, 6 other drugs

    Kratom, a popular but unregulated psychotropic herbal extract, is one of seven currently unscheduled psychotropic drugs that will be reviewed by the World Health Organization (WHO) under the provisions of the international psychotropic convention to which the US is a party.   WHO notified the US and other international parties to the 1971 convention that the seven new psychotropic drugs may be added to its list of scheduled drugs. By law, that notification triggers a r...
  • ReconRecon

    Recon: Biden and Democrats face dilemma on vaccine mandates; EU to discuss joint recognition of vaccine certificates with Russia

    Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US U.S. FDA narrows patient group for Biogen's Alzheimer's drug ( Reuters ) Alzheimer antibody rush begins, as efficacy concerns remain ( Nature ) AstraZeneca-Amgen drug gets FDA speedy review as asthma treatment ( Reuters ) Death rates are declining for many common cancers in the U.S., report finds ( STAT ) HHS head: 'Absolutely the government's business' to kn...
  • Regulatory NewsRegulatory News

    FDA focuses on efforts made to avert drug shortages in 2020

    New drug shortages in the US decreased in 2020, in part because of the US Food and Drug Administration’s (FDA’s) use of enforcement discretion to ensure an uninterrupted supply of critical medical products during the COVID-19 public health crisis.   Yet, the agency’s new drug shortages report to Congress said that while these actions helped avert shortages in the short-term, more work is needed to improve supply chain resiliency.   This report summarizes the agency...