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  • Regulatory NewsRegulatory News

    EMA Reflects on Advanced Therapy Classification

    The European Medicines Agency (EMA) has released a new reflection paper discussing how products are classified as advanced therapy medicinal products (ATMPs). Background In the EU, medicinal products derived from genes, cells or tissues are classified as ATMPs. Because these areas of biomedicine are still in early stages of development and are fundamentally different from other types of medicine, the European Commission (EC) regulates them differently. ATMPs are define...
  • Regulatory NewsRegulatory News

    FDA Considers Compounding Restrictions on Four Drugs, Including Acetaminophen

    The US Food and Drug Administration (FDA) is preparing to ask one of its newest advisory committees to decide if some compounding pharmacies should be allowed to make four drugs, including the widely used painkiller acetaminophen. Background The advisory committee, known as the Pharmacy Compounding Advisory Committee (PCAC) , was re-created under a piece of 2013 legislation known as the  Drug Quality and Security Act (DQSA) . The law, passed in the wake of a major drug...
  • Feature ArticlesFeature Articles

    What Influences CDER Advisory Committee Members?

    Over the past two decades, the US Food and Drug Administration’s (FDA’s) Center for Drug and Evaluation and Research (CDER) has been developing new, expedited pathways to help bring innovative medical products to market more efficiently. These pathways include fast track designation, accelerated approval, priority review and, more recently, breakthrough therapy. Through these expedited pathways, some products have been approved with fewer clinical data than traditio...
  • Regulatory NewsRegulatory News

    Marketing Exclusivity Provisions for Rare Disease Drugs Back in 21st Century Cures Bill

    An updated draft of the House Energy and Commerce (E&C) Committee's 21st Century Cures Act has reintroduced a provision which would extend marketing exclusivity for some drugs repurposed to treat rare diseases or conditions by six months. Background The provision, popularly known as the Orphan Product Extensions Now Act , was originally introduced in the US House of Representatives in November 2014. Under the bill, existing pharmaceutical products would be grant...
  • Regulatory NewsRegulatory News

    Following Negative Comments by Reviewer, EU Committee OK's Gene Therapy

    Last month, German regulators postponed their review of UniQure's gene therapy Glybera after the European Medicines Agency (EMA) rapporteur assigned to the product claimed new data showed Glybera lacked efficacy and called for it to be reassessed . Now, EMA's Committee for Advanced Therapies (CAT) has completed its assessment, and is maintaining its positive opinion of Glybera, Reuters reports. Background Glybera is the first gene therapy approved for use in the E...
  • Regulatory NewsRegulatory News

    Senators Question FDA's Authority to Issue 'Placeholder' Name for First Biosimilar

    When the US Food and Drug Administration (FDA) approved the US' first biosimilar product, Zarxio, in March 2015, it gave the drug a unique and temporary nonproprietary name. Now a group of US senators is questioning whether FDA had the legal authority to do so. Background Zarxio is the first drug to be approved under the 351(k) pathway created in 2010 under the Patient Protection and Affordable Care Act . The drug, which is manufactured by Sandoz, is intended to be bio...
  • Regulatory NewsRegulatory News

    German Regulators Decry Lack of Trial Transparency in Look at Stem Cell Transplantation

    The German Institute for Quality and Efficiency in Health Care (IQWiG) has found recent clinical data on the use of stem cell transplantation to treat multiple myeloma—a rare and deadly form of cancer—to be inconclusive . Background and 2012 Conclusion Stem cell transplantation is a therapy used to treat certain cancers, immunodeficiencies and hematologic diseases. It has also been widely used to treat multiple myeloma. Patients undergoing stem cell transplantation a...
  • Regulatory NewsRegulatory News

    UPDATED: New to FDA's Advisory Committee Briefing Documents: Patient Testimony

    Patients and their advocates appear to be getting more prominent attention from the US Food and Drug Administration (FDA) if a recent release of documents in advance of an upcoming advisory committee meeting is any indication. This story has been updated to include additional information from FDA and to correct one inaccuracy: Patient testimonials have always been made available to advisory committee members in advance of meetings, even if they are not publicly po...
  • RAPS' LatestRAPS' Latest

    Regulatory Profession a Promising Field in China, Says RAPS China Advisory Committee Member Lin

    The following article was adapted from a translation of the original interview conducted in Chinese.  Two decades ago, unlike many of her contemporaries, Linda Lin decided not to pursue a job that would have been a more traditional choice for a woman in China, a job like teacher for example, to work in a joint-venture pharmaceutical factory in Guangzhou. Today, she is vice president of international business for Shanghai MicroPort Medical (Group) Co. Ltd. She also serv...
  • Regulatory NewsRegulatory News

    First EU-Authorized Gene Therapy Hits Snag, Benefit Assessment Delayed in Germany as EMA Reviews New Report

    Regulators at the German Federal Joint Commission (G-BA) have postponed their review of the gene therapy Glybera, Reuters reports . The decision was made after the European Medicines Agency (EMA) rapporteur assigned to Glybera found the product lacked efficacy and called for it to be reassessed. Glybera, which treats a rare condition called lipoprotein lipase deficiency (LPLD), made headlines both for being the first gene therapy approved in Europe and for its record-...
  • Regulatory NewsRegulatory News

    FDA Seeks Industry Representation on 17 of its Drug Advisory Committees

    • 15 April 2015
    The US Food and Drug Administration (FDA) is reaching out to the pharmaceutical and biopharmaceutical industries for input on which industry experts should serve on 18 of its influential advisory committees. Background FDA's advisory committees are comprised of experts—approximately 8-17 of them per committee—which make recommendations to FDA on matters of policy and specific products. For example, a committee might recommend a specific product be approved because of it...
  • Regulatory NewsRegulatory News

    EU Committee Confirms Increased Risk with Ibuprofen at High Doses

    A European committee has confirmed that high doses of ibuprofen, a popular over-the-counter (OTC) anti-inflammatory drug, can increase a patient's risk for cardiovascular issues. Background Ibuprofen belongs to a class of drugs, called non-steroidal anti-inflammatory drugs (NSAIDs), which includes drugs such as diclofenac, naproxen and aspirin. NSAIDs already have a warning about increased risk of blood clots included in the information distributed with such products ...