To ensure resiliency in the medical supply chain, the US Food and Drug Administration (FDA) should publicly disclose the location of all manufacturing facilities that supply ingredients and parts for pharmaceuticals and medical devices approved in the US, according to a consensus study report from the National Academies of Science, Engineering and Medicine (NASEM).   This is one of seven recommendations in a 365-page report on building supply chain resiliency. The re...

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...

The US Food and Drug Administration (FDA) has extended its temporary pause on non-mission-critical domestic surveillance inspections through 4 February 2022, an agency spokesperson told Focus on Wednesday.   FDA previously announced it would halt certain inspectional activities out of concern for its employees and those of the companies it regulates amid the wave of COVID-19 infections driven by the omicron variant. At the time, the agency said it would continue to c...

The US Food and Drug Administration (FDA) on Thursday issued a draft guidance to help manufacturers set up inspection testing programs to ensure that their injectable drugs are free of visible particles.   The 18-page draft guidance "addresses the development and implementation of a holistic, risk-based approach to visible particulate control that incorporates product development, manufacturing controls, visual inpsection techniques, particulate identification, inves...

An official with the US Food and Drug Administration (FDA) announced the agency will soon resume unannounced onsite inspections in India and China. Also, prioritized foreign inspections will resume in February.   Elizabeth Miller, assistant commissioner for medical products and tobacco operations in FDA’s Office of Regulatory Affairs (ORA), provided this update on the agency’s inspection activities at a 9 December virtual conference on enforcement sponsored by the Fo...

While there are benefits to conducting remote good clinical practice (GCP) and good manufacturing practice (GMP) inspections and these assessments can continue being used  post pandemic, these methods have their limitations, concludes the International Coalition of Medicines Regulatory Authorities (ICMRA) in a new reflection paper .   Among the topics ICMRA touches on in the reflection paper are the decision-making process to use remote approaches, tools for remote in...

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...

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...

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...

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...

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, ...

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...