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  • Regulatory NewsRegulatory News

    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 ...
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    Priority Review Voucher Fees to Decline in FY 2017

    The US Food and Drug Administration (FDA) on Thursday unveiled the new user fee rates for the tropical disease and rare pediatric disease priority review voucher (PRV) programs. The additional fees necessary to use the vouchers for both programs are set to decline by about $20,000 when compared to last year. Tropical Disease Priority Review Voucher User Fee Year Voucher Fee FY 2011 $4,582,000 FY 2012 $5,280,000 FY 2013 $3...
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    Congress Signs Off on Short-Term Extension to Rare Pediatric PRV Program

    The House late Tuesday followed its Senate counterparts and sent to President Barack Obama a re-authorization of the rare pediatric disease priority review voucher (PRV) program that will last through the end of December. A spokeswoman for Sen. Bob Casey (D-PA), a sponsor of the bill, told Focus that between now and 30 December, Congress will discuss ways to amend the program and extend it again. “Our policy changes are in S. 1878, which is headed to the president, so...
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    Pediatric Priority Review Vouchers on the Chopping Block as Reauthorization Stalls

    Congress and President Barack Obama have one week to reauthorize the rare pediatric priority review voucher (PRV) program or else it will end on 30 September 2016, after seven pediatric PRVs have been awarded, one of which sold for $350 million last year. The PRV program offers vouchers that companies can win for gaining approval of new rare pediatric treatments, affecting fewer than 200,000 patients under the age of 18. The vouchers can be used to speed FDA’s review ...
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    FDA Finalizes Guidance on Extrapolating Data for Pediatric Medical Devices

    A little more than a year after introducing the draft version, the US Food and Drug Administration (FDA) on Monday finalized guidance on how and when it may be appropriate to leverage existing clinical data to support pediatric medical device indications and labeling. The guidance, which, like the draft from May 2015 , reveals FDA’s concerns with the paucity of scientific evidence substantiating medical device submissions for pediatric indications, outlines ways in whic...
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    European Commission Unveils Four New Public Consultations on Clinical Trials

    The four new consultations, released Thursday from the European Commission, deal with ethical considerations for pharmaceutical trials with minors, risk proportionate approaches in trials, summaries of trial results for non-specialists or “laypersons” and definitions of investigational medical products (IMPs) and use of auxiliary medicinal products (AMPs). The consultations offer respondents a chance to weigh in (comments on the four consultations are due by 31 August) o...
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    EMA Drafts Framework for Extrapolating Adult Patient Data for Pediatric Indications

    The European Medicines Agency (EMA) released a reflection paper on Monday with the hope of helping drugmakers avoid unnecessary pediatric research by establishing a framework for extrapolating clinical data from adult patients to support pediatric indications for new drugs. EMA says the framework will help "optimize" clinical trial participation for pediatric patients by using existing data from other populations, including adults and other pediatric subgroups, to predic...
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    FDA Shares Views on Juvenile Animal Studies for Oncology Drugs

    In a recently published article, officials from the US Food and Drug Administration (FDA) say that studies comparing the effects of drugs in juvenile animal populations against their effects in adult animals are generally not useful in guiding pediatric clinical development, especially if such studies delay the launch of a clinical trial. The authors of the article, John Leighton, Haleh Saber, Gregory Reaman and Richard Pazdur, of FDA's Office of Hematology and Oncology ...
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    Senate HELP Advances Bill to Reauthorize Pediatric PRV Program and Six Other Bills

    The Senate Health, Education, Labor & Pensions committee on Wednesday advanced seven bills, including one that would reauthorize the rare pediatric priority review voucher (PRV) program through 2022, and one bill that would add the Zika virus to the tropical disease PRV program. The advancement of the bill sponsored by Sen. Bob Casey (D-PA) to extend the pediatric PRV program comes as the US Food and Drug Administration (FDA) told the Government Accountability Office ...
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    Pediatric Study Plans: FDA Unveils Revised Draft Guidance

    The US Food and Drug Administration (FDA) on Tuesday released updated draft guidance intended to help pharmaceutical sponsors submit pediatric study plans, including what should be included in the plans and how they should be submitted. This revision, which FDA says was based largely on public comments, includes additional clarifications on the 2013 draft guidance, such as new information on what constitutes a materially incomplete initial pediatric study plan (iPSP), th...
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    FDA Calls for End to Priority Review Vouchers as GAO Says Too Early to Gauge Effectiveness

    The US Government Accountability Office (GAO) said Wednesday in a new report that it’s still too early to assess whether the Food and Drug Administration’s (FDA) three-year-old pediatric priority review voucher (PRV) program has stimulated the development of drugs to treat or prevent rare pediatric diseases. The PRV program offers vouchers that companies can win for gaining approval of new rare pediatric treatments affecting fewer than 200,000 people, more than half o...
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    Sanofi Redeems $245M Priority Review Voucher for Type 2 Diabetes Treatment

    The US Food and Drug Administration (FDA) accepted for review the New Drug Application (NDA) for Sanofi’s investigational type 2 diabetes treatment on Monday and granted the use of another Priority Review Voucher (PRV), which speeds up FDA’s decision by four months. In redeeming the voucher, Sanofi had to pay a $2.7 million fee in addition to the standard new drug filing fee of $2.4 million. Retrophin sold the PRV to Sanofi last May for one of the highest amounts eve...