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    FDA Issues Disease-Specific Draft Guidance for Pediatric GERD

    The US Food and Drug Administration (FDA) on Thursday issued draft guidance detailing clinical considerations for drugmakers looking to develop products to treat pediatric gastroesophageal reflux disease (GERD). FDA says the draft is meant to be a starting point for discussions between the agency, drugmakers, academia and the public and sets out the agency's current thinking on potential study populations, endpoints and clinical pharmacology issues related to dosing. Sp...
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    EU Pediatric Regulation’s 10-Year Anniversary Report Highlights Shortcomings

    Ten years ago, the European Commission created a new regulation as part of an effort to try to encourage more pediatric research. But in the years since, companies have been slow to increase their development of treatments for pediatric populations, particularly in oncology, where many of the medicines used were developed in the 1990s, “if they exist at all,” an EC report released Thursday said. The report highlighted that the number of completed pediatric investig...
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    EMA Revises Reflection Paper on Pediatric Extrapolation

    The European Medicines Agency (EMA) on Friday released an updated draft reflection paper for public consultation detailing the agency's framework for pediatric extrapolation in drug development. EMA defines extrapolation as extending information and conclusions from studies of a particular subgroup (source population), condition or drug to make inferences for another subgroup (target population), condition or drug. By extrapolating data from adult or other pediatric popu...
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    European Commission Adopts Two Acts on GMPs for Medicines

    Last month, the European Commission adopted two legal acts seeking to improve patient safety through good manufacturing practices (GMP) that ensure the highest quality of medicines for investigational and human use. One of the acts is an  implementing directive  that sets out principles and guidelines of GMP in medicines where the manufacture or import is subject to a manufacturing authorization ( see Article 40 of the Community code Directive (2001/83/EC) ). The ...
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    ICH Plans Work on Clinical Trials Guideline Revision, Pediatric Extrapolation

    The International Council for Harmonisation (ICH) is planning to take up two new topics, according to the minutes released Thursday from the group's meeting in Montreal in May and June. The two topics include the first revision to ICH's 1997 E8 General Considerations for Clinical Trials and a new guideline on pediatric extrapolation in clinical trials, both proposed by the US Food and Drug Administration (FDA). The ICH Assembly also adopted concept papers outlines f...
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    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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    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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    Regulating CRISPR: FDA and Industry Offer Perspective

    Although the US market is likely more than a year or two away from seeing any commercialized medical products that rely on CRISPR-Cas9 technology, the rapidly developing field has already grabbed the attention of the US Food and Drug Administration (FDA) and other drug regulators. What is CRISPR? According to the Broad Institute , CRISPR (pronounced "crisper") stands for Clustered Regularly Interspaced Short Palindromic Repeats, which form the basis for a genome ...
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    Application of EU Clinical Trial Regulation Delayed to 2019

    The European Medicines Agency’s management board said that due to technical difficulties with the development of IT systems, the application of the clinical trial regulation has to be postponed. "Due to these delays, the EU  Clinical Trial Regulation  will now come into application in 2019 instead of October 2018, as previously scheduled," EMA said Friday. The agency says it will provide an update at the next meeting of the Management Board in October 2017, followi...
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    6 Things You Need to Do to Prepare for the New EU Medical Devices Regulation

    When the EU’s new Medical Devices Regulation ( MDR ) entered into force last month, it set in motion a three-year countdown to the new rules’ full application in 2020. For companies marketing devices in the EU that wish to continue to do so, there is a lot to do in that relatively short time, so it is critical to begin as soon as possible. Early preparation is key. Following are six things you should be doing to get ready. 1. Plan Ahead In order to figure out wh...