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    FDA Lists 205 Molecular Targets for Pediatric Cancer Research

     To help with anti-cancer drug development, the US Food and Drug Administration (FDA) has developed two new lists of molecular targets to guide submissions for pediatric study plans.   The two lists, posted Tuesday by FDA’s Oncology Center of Excellence, are aimed at fostering the development of new oncology drugs or biologics for pediatric populations. They also fulfill a commitment the agency made under the FDA Reauthorization Act of 2017 (FDARA).   One list po...
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    CDRH Drafts New Framework to Create a Pediatric Device Safety Network

    A draft framework is in the works at the US Food and Drug Administration (FDA) to create an innovative safety net for pediatric medical devices, Vasum Peiris, Chief Medical Officer for pediatrics and special populations at the Center for Devices and Radiological Health (CDRH), told Focus . The new framework is “centered around the fundamental issue of safety for children,” Peiris said, posing the question “where can we actually innovate safely?” Pediatric academic medic...
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    FDA Closes Loophole Companies Used to Skirt Pediatric Study Requirements

    The US Food and Drug Administration (FDA) on Tuesday released draft guidance indicating that it no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases except for under certain conditions. Those conditions are if the use of the drug in the pediatric subpopulation are for a valid orphan subset, meaning "that use of the drug in a subset of persons with a non-rare disease or condition may be appropriate but use of th...
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    European Regulatory Roundup: Critical Moment for EMA (15 December 2017)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. Commission Creates Roadmap for Evaluation of Pediatric Rare Disease Legislation The European Commission has created a roadmap for evaluating legislation covering pediatric rare diseases. Officials are embarking on the nearly two-year evaluation to understand why regulations on pediatric and orphan medicines have failed to translate into the hoped-for medical advances. ...
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    Have PRVs Incentivized New Rare or Neglected Disease Research? Experts Discuss

    The priority review voucher (PRV) programs, created by Congress with an eye to incentivizing the development of new rare pediatric and neglected tropical disease drugs, have so far rewarded a wide range of small and large biopharma companies and products, though whether PRVs have actually spurred new research remains a question. Andrew Robertson, head of regulatory science and policy at Sanofi, and Rachel Cohen, regional executive director for the Drugs for Neglect...
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    More Efficient Rare Pediatric Drug Development: FDA Drafts Guidance

    The US Food and Drug Administration (FDA) on Wednesday released draft guidance that could help speed the development of treatments for rare pediatric disease drugs. Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, said the draft guidance "could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would receive a placebo instead of a potentially helpful...
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    Senate Tax Plan Seeks to Reform, Rather Than Repeal, Orphan Drug Tax Credit

    The Senate’s tax plan, running counter to the House’s call to eliminate the orphan drug tax credit for research on drugs to treat rare diseases, seeks to modify the credit instead. The proposal would limit what level of expenses will qualify and limit qualified clinical expenses if they are related to the use of a drug which has previously been approved for diseases or conditions affecting more than 200,000 persons in the US. The tax credit is part of a law from...
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    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...