• Regulatory NewsRegulatory News

    FDA to Close Loophole Allowing Companies to Skirt Pediatric Study Requirements

    FDA commissioner Scott Gottlieb announced Tuesday that the agency will release guidance to close a loophole that allows companies to avoid their obligation to study pharmaceuticals in pediatric populations. The situation, according to Gottlieb, arises if sponsors received an orphan designation for a pediatric subtype of an otherwise common and non-orphaned adult disease. For example, with a condition like inflammatory bowel disease (IBD), a drug may be approved to...
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    FDA Reports on FY2016 Pediatric Device Approvals

    In a report to Congress released this week, the US Food and Drug Administration (FDA) says it approved 13 high-risk medical devices for pediatric patients in FY2016, the highest number since 2011 when 15 were approved. Under the Food and Drug Administration Amendments Act of 2007 (FDAAA), device makers are required to include information in premarket approval (PMA) and humanitarian device exemption (HDE) applications about pediatric subpopulations that are affected by ...
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    $500m Over 9 Years: FDA Details Plan for New 'Cures' Funds

    The US Food and Drug Administration (FDA) on Friday released a plan submitted to Congress for how it will use $500 million in potential new funding from the 21st Century Cures Act over the next nine fiscal years. The plan, similar to a proposal released in May, would see about $185 million of the total go to updates and modernizing FDA’s regulation of combination products, developing FDA standards and consensus definitions to support the development and revie...
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    10 Years in, EU Pediatric-Use Marketing Authorization Called a 'Disappointment'

    Biopharmaceutical companies and regulators across Europe have said that in the 10 years since the Pediatric Regulation took effect, some pediatric therapeutic areas have seen progress, though the pediatric-use marketing authorization (PUMA) has not lived up to expectations. Overall, according to a summary of the comments received from 75 stakeholders on the consultation from last November , the European Commission noted that the reward system built into the regul...
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    EMA and FDA Look to Facilitate Development of Gaucher Disease Treatments

    The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) on Monday released a new strategy document to aid in the development of new treatments for Gaucher disease, a rare lysosomal storage disorder. Given the limited number of patients worldwide with Gaucher disease, EMA and FDA put forth two possible approaches to better facilitate investigations of such products: Extrapolation across age groups within a single drug development program...
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    MHRA Plans for the Future of Drug, Device Regulation Post-Brexit

    The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on Friday released a business plan for the upcoming year, noting its primary focus on developing a consensus around a proposed model for the future of the country’s regulation of medicines and medical devices following its departure from the EU. MHRA is pushing for a more international strategy that focuses on collaboration and engagement with key partners and stakeholders and continuing to partici...
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    WHO Seeks Additional Birth Control API Manufacturers as US FDA Bans China Site

    A Chinese manufacturer of the active pharmaceutical ingredient (API) for the birth control drug levonorgestrel has been placed on the US Food and Drug Administration’s (FDA) import alert list, causing the World Health Organization (WHO) to scramble to find a replacement. "At the moment, there is no alternative levonorgestrel API which has been prequalified," WHO said Wednesday. When an API is prequalified by WHO, it can be used for the manufacture of finished pharmaceuti...
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    Pediatric Cancers: New Bill Seeks to Close Clinical Trial Requirement Loopholes

    A new bipartisan bill reintroduced in the House and Senate in late February, which could ultimately be attached to the reauthorized Prescription Drug User Fee Act (PDUFA), seeks to close the loopholes biopharmaceutical companies use to skirt around clinical trial requirements for cancer treatments in pediatric populations. Background Pediatric study requirements are included in both the Pediatric Research Equity Act (PREA), which requires biopharma companies to ...
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    CTTI Offers Recommendations for Pediatric Clinical Trials for Antibiotics

    The Clinical Trials Transformation Initiative (CTTI) has released a set of recommendations it says will improve the use of antibiotics in children. According to CTTI, a public-private partnership aimed at improving the quality and efficacy of clinical trials, challenges in enrolling and completing pediatric clinical trials for antibiotics leads to a situation where such drugs are used in children despite lacking adequate labeling for pediatric populations. Furthermore, ...
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    FDA Officials Call for Including Adolescents in Adult Oncology Trials

    A team of officials at the US Food and Drug Administration (FDA) are calling for a "culture shift" by drugmakers, regulators and clinical investigators to encourage enrolling more adolescent patients in relevant "adult-type" cancer clinical trials. The officials made the call in a recent article in Clinical Cancer Research , where they argue that excluding older adolescents, ages 12 to 17, from adult oncology trials leads to delayed access to new treatments for those pa...
  • Feature ArticlesFeature Articles

    A Summary of 10 Years of Paediatric Legislation in the European Union

    This article summarizes 10 years of experience with paediatric regulations in the EU. Introduction Almost 10 years ago, on 27 January 2007, new Paediatric Legislation (PL) came into force that reshaped the regulatory landscape for paediatric medicines in Europe. 1, 2 The objective of this Paediatric Regulation (PR) is to improve the health of children in the European Union (EU) by: facilitating the development and availability of medicines for children from birth ...
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    FDA Begins Consultation on Update to ICH Pediatric Clinical Trials Guidance

    The US Food and Drug Administration (FDA) on Monday launched a three-month public consultation on an addendum to the International Council for Harmonisation's (ICH) guidance on pediatric clinical trials. The addendum, E11(R1) Addendum: Clinical Investigation of Medicinal Products in the Pediatric Population , is intended to supplement ICH's E11 guidance by expanding  the discussion on issues related to pediatric clinical trials in light of scientific and regulatory ...