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  • 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

    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

    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...
  • Regulatory NewsRegulatory News

    EMA consults on ICH S12 guideline, shares comments on PFDD reflection paper

    The European Medicines Agency (EMA) on Thursday opened a public consultation on the International Council for Harmonisation’s (ICH) S12 guideline on nonclinical biodistribution (BD) considerations for gene therapies.   The S12 guideline recently reached Step 2b of the ICH process after being endorsed at the ICH Assembly virtual meeting earlier this month. (RELATED: ICH reports ‘significant milestones’ reached on guidelines ranging from impurity testing to eCTD standa...
  • Regulatory NewsRegulatory News

    FDA pilots program to encourage new drug development tools

    The US Food and Drug Administration (FDA) on Monday announced it will pilot a new program it hopes will spur the development of novel drug development tools (DDTs) that do not fit within the agency’s existing DDT qualification programs.   The qualification process for DDTs was revamped under the 21 st Century Cures Act and FDA currently offers specific qualification programs for biomarkers, clinical outcome assessments (COAs) and animal models for use under the Anim...
  • Regulatory NewsRegulatory News

    Study: Few new drugs have high therapeutic value

    A new study published in The BMJ  finds that only a third of new drugs approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the decade from 2007-2017 have high therapeutic value, according to appraisal by one of five independent organizations.   However, the analysis found that drugs approved through expedited programs – and especially EMA’s programs – were more likely to have high therapeutic value.   “Most of the incre...
  • Regulatory NewsRegulatory News

    Updated: FDA delays collection of drug manufacturing volume data

    The US Food and Drug Administration (FDA) on Monday announced it will not begin collecting drug manufacturing volume data next month as required by the Coronavirus Aid, Relief, and Economic Security Act (CARES Act).   Section 3112 of the CARES Act establishes new reporting requirements for drug manufacturers intended to improve the response to drug shortages, including expanding shortage reporting requirements for life-saving drugs to include drugs that are “critical...
  • RoundupsRoundups

    FDA Approvals Roundup: Fintepla, Dojolvi, Phesgo

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New approvals Fintepla gets the go-ahead for Dravet syndrome Zogenix’s Fintepla (fenfluramine) has been approved for the treatment of seizures associated with Dravet syndrome in patients aged 2 years or older. The syndrome is a rare, life-threatening form of epilepsy.   The approval was based on findings from two clinical studies in 202 participants aged ...
  • RoundupsRoundups

    FDA Approvals Roundup: Zepzelca, Ilaris, Lyumjev

    A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).   New approvals Zepzelca handed accelerated approval for metastatic SCLC in adults Jazz Pharmaceuticals and PharmaMar’s Zepzelca (lurbinectedin) has been granted accelerated approval for adult patients with metastatic small cell lung cancer (SCLC) whose disease has progressed during or after platinum-based chemotherapy.   The indication was approved based o...
  • Feature ArticlesFeature Articles

    Risk management, drug shortages and the EU portal for clinical trials

    Feature articles during May focused on risk management and mitigation in dealing with contractors and vendors and included articles on best practices in good vendor management, use of risk management to support outsourcing activities, testing in-house versus outsourcing, and outsourcing in regulatory operations. Also included were in-depth examinations of challenges and opportunities in “bespoke” therapies, a critical appraisal of drug shortages in Germany and an update on...