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  • Regulatory NewsRegulatory News

    For Second Time Ever, FDA Awards Special Voucher Meant to Accelerate Drug Reviews

    Drugmaker United Therapeutics has become just the second company in US history to obtain a new type of voucher which allows a company to potentially get its drug approved by the US Food and Drug Administration (FDA) in 40% less time than it normally takes. Background The voucher in question is known as a rare pediatric disease priority review voucher (PRV), and is modeled off a similar program intended to help spur the development of new drugs for so-called "neglected" ...
  • Regulatory NewsRegulatory News

    New FDA Guidance Explains how to Test Drugs on Pediatric Patients

    A new guidance document issued by the US Food and Drug Administration (FDA) established new recommendations for how companies should conduct pediatric studies on new drug and biological products. Testing products in pediatric patients is notoriously difficult. As FDA regulators have reiterated on numerous occasions, children "are not just small adults ," and drug and biological products can behave much differently in pediatric patients compared to how they behave in...
  • Regulatory NewsRegulatory News

    FDA Explains How its New Incentive Program for Rare Pediatric Disease Treatments Works

    The US Food and Drug Administration (FDA) has released a long-anticipated guidance document intended to explain how a new incentive program known as the Rare Pediatric Disease Priority Review Voucher system works. Background Created in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA) , the Rare Pediatric Disease Priority Review Voucher is modeled closely off a similar program known as the Tropical Disease Priority Review Voucher system. T...
  • Regulatory NewsRegulatory News

    NIH Wants Safety, Efficacy of Viagra, Epogen and Other Drugs Tested in Children

    • 25 August 2014
    The National Institutes of Health (NIH) has released a new "Priority List" of pediatric therapeutic areas and medical products it wants the medical community—including the pharmaceutical and biopharmaceutical industries—to focus on. Background Historically, many companies seeking product approval in the US avoided clinical studies involving children, wary of ethical problems, a lack of incentives and the potential consequences if testing uncovered new problems. As a res...
  • Regulatory NewsRegulatory News

    First Pediatric Priority Review Voucher Goes up for Sale, Fetching $67M

    As far as regulatory incentives go, some are pursued more than others. The US Food and Drug Administration's (FDA) orphan drug exclusivity provisions, for example, have brought about a renaissance of therapies meant to treat patients suffering from rare diseases and conditions. But on the other side of the coin are incentives not often sought out by companies. And perhaps no incentive maintained by FDA is used less than its priority review vouchers. Background: Vouchers...
  • Regulatory NewsRegulatory News

    FDA Planning Five Guidance Documents to Help Children With Rare Diseases

    • 10 July 2014
    The US Food and Drug Administration (FDA) has released a new report indicating four core ways it plans to accelerate the development of treatments for pediatric rare diseases (PRD). The report, Complex Issues in Developing Drugs and Biological Products for Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases , was required under Section 510 of the Food and Drug Administration Safety and Innovation Act ( FDASIA ) and based off a...
  • Regulatory NewsRegulatory News

    After Months of Silence, FDA Returns to 'Name and Shame' Strategy on Pediatric Trial Compliance

    In 2013, the US Food and Drug Administration (FDA) launched a new webpage seemingly intended to shame companies into meeting their pediatric study commitments under the  Pediatric Research Equity Act ( PREA ). But after an initial deluge of postings, FDA's updates to the page have all but halted, with just five letters posted to the page after its launch, and none within the last six months. Now FDA has posted its first letter to the page in more than half a year detail...
  • Regulatory NewsRegulatory News

    EMA, FDA Team Up to Propose Guideline on Pediatric Gaucher Disease

    US and EU regulators have come together to release an unusual joint proposal that they say will help speed up the development of new treatments affecting pediatric patients with Gaucher disease. Background Gaucher disease is a rare, inherited lysosomal storage disorder which affects patients by causing the buildup of lipids in cells and organs, such as the liver, spleen, kidneys and brain. It is thought that about 20,000 patients have Type I Gaucher disease in the US, a...
  • New FDA Guidance Establishes Pediatric Data Submission Standards for Devices

    US Food and Drug Administration (FDA) regulators have finalized a new guidance document that aims to make medical devices a little bit safer for children. Background In 2007, Congress passed the  Food and Drug Administration Amendments Act (FDAAA) , a massive piece of legislation that channeled a significant amount of energy into commitments meant to make products safer for consumers, including pediatric patients, to use. Notably, section 515A of  FDAAA requ...
  • New Guidance Clarifies Development Process for Pediatric Medical Devices

    A new guidance document published by the US Food and Drug Administration's (FDA) Center for Devices and Radiological Health (CDRH) updates the agency's thinking on how to ensure that medical devices are safe and effective for pediatric populations. Background Historically, many medical devices and other medical products have been developed for adults, which constitute the largest market segment for many products. Many of those devices were approved based on data obtaine...
  • Focusing on Pediatric Study Commitments, FDA Prepares to Name and Shame Noncompliant Companies

    Pharmaceutical and biopharmaceutical companies, consider yourselves on notice-very, very public notice. In an announcement made on 26 August 2013 through its FDA Voice blog, the US Food and Drug Administration (FDA) said it will this week begin to publish letters of non-compliance for companies it has found to have missed deadlines to conduct pediatric studies required by federal law. Background Historically, many companies seeking product approval in the US avoided c...
  • European Commission Releases First Report on the Pediatric Regulation

    The European Commission today published a progress report on medicines for children covering the five years since the Pediatric Regulation came into force. Although it will take several more years to fully assess the impact of the legislation due to long drug development cycles, this preliminary review points to significant improvements in the pediatric medicines landscape: better and safer research, more products for children on the EU market and more information for p...