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  • Regulatory NewsRegulatory News

    Amgen Challenge of FDA Decision on Pediatric Exclusivity Could Have Wider Implications

    Back in May, the US Food and Drug Administration (FDA) denied a six-month extension of market exclusivity for Amgen's blockbuster Sensipar (cinacalcet), resulting in lawsuit filed by the drugmaker. A JAMA viewpoint published Wednesday argues that a decision in Amgen's favor could end up diminishing FDA's ability to encourage clinically meaningful pediatric studies. The case centers on the interpretation of how companies "fairly respond" to FDA's written requests ...
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    FDA Issues Disease-Specific Draft Guidance for Pediatric GERD

    The US Food and Drug Administration (FDA) on Thursday issued draft guidance detailing clinical considerations for drugmakers looking to develop products to treat pediatric gastroesophageal reflux disease (GERD).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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    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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    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...