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  • Regulatory NewsRegulatory News

    FDA Helps Fund 21 Early Phase Trials for Rare Disease Treatments

    The US Food and Drug Administration (FDA) on Monday announced it is awarding $23 million in research grants for 21 new clinical trials over the next four years to boost the development of rare disease treatments. The grants, awarded to researchers from academia and industry at domestic and international clinical sites, are part of FDA’s Orphan Products Clinical Trials Grants Program, created in 1983, which has provided more than $370 million (including $19 million ...
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    European Commission to Overhaul Concept of ‘Similar’ Medicines Within Context of Orphan Legislation

    The European Commission on Friday proposed to review the concept of “similar medicinal products” in the context of its orphan legislation as part of a wider effort to adapt the text to technical progress. Fifteen years after the implementation of the orphan legislation, the commission says it is currently launching initiatives to improve the implementation of the regulatory framework with a view to ensuring timely access to medicinal products.  In this context, the Comm...
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    Regulators, Industry Weigh in on Proposed EU Orphan Regulation Changes

    As the number of orphan drug approvals continues to rise in the EU, the European Commission is now considering feedback from European governments, regulators and industry on how changes to five aspects of the Orphan Regulation may further incentivize the development of drugs for rare diseases. To date, the commission has authorized 117 orphan medicines, 82% of which consist of new active substances, according to a new report .  And while the number of products authorize...
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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...
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    Orphan Medicines in the EU: A 15-Year Review

    With the 15th anniversary of the adoption of the Orphan Regulation in the EU, the European Commission notes in a new report that this landmark regulation has incentivized the development, approval and marketing of more than 100 rare disease treatments, or orphan drugs. According to the European Commission’s fourth inventory of orphan medicines, released Friday, somewhere between 5,000 and 8,000 different rare diseases exist in the EU, impacting between 27 million and 36 ...
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    Consumer Advocacy Group Warns on Orphan Provisions in 'Cures' Act

    Orphan drug provisions in the House-passed 21st Century Cures Act could increase US healthcare spending by billions of dollars over the next decade, consumer advocacy group Public Citizen warns, though the bill still remains in limbo in the Senate. The provisions in question would add an additional six months of marketing exclusivity for approved drugs that later go on to be approved to treat rare diseases. The extended period of exclusivity would apply to all indicati...
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    Johns Hopkins Researchers Call for Orphan Drug Reform

    A team of researchers at Johns Hopkins are calling for reforms to the Orphan Drug Act , saying that loopholes have allowed drug companies to skirt the act's intent by taking advantage of its incentives for non-orphan conditions. Background The Orphan Drug Act of 1983 was passed to spur drug development for rare diseases. The act created incentives for drugs that treat diseases affecting fewer than 200,000 individuals in the US per year. The incentives include seven-...
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    Industry Seeks More Specifics on FDA's Flexibility With Orphan Drug Guidance

    Two industry associations and a rare disease patient advocacy group say they want the US Food and Drug Administration (FDA) to clarify its "regulatory flexibility" with orphan drug reviews. Challenges in Orphan Drug Development In August, FDA released a draft guidance intended to address the most common issues faced by drugmakers developing treatments for rare diseases . The guidance notes that FDA's "regulations provide flexibility in applying regulatory standards" a...
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    Report: Competition Quickly Follows First-In-Class Approvals

    By the time "first-in-class" drugs receive marketing approval in the US, nearly all later iterations of those products have begun clinical testing or filed a new drug application with the US Food and Drug Administration (FDA), according to a new study by Tufts Center for the Study of Drug Development. The study, which compared 40 drug classes with a first-in-class approval between 1998 and 2011, found that the race for marketing approval for new classes of drugs and biol...
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    European Regulatory Roundup: CHMP Updates Breast Cancer Guidelines (15 October 2015)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. EMA Updates Data Quality Control, IT Papers as Introduction of New Process Nears The European Medicines Agency (EMA) has updated documents on data quality control and electronic filings ahead of the introduction of a new process in November. EMA will start using the new process on 4 November, at which time the regulator will start sending out alerts every time it vali...
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    Asia Regulatory Roundup: Japanese Regulators Receive Orphan Drug Training from EMA (6 October 2015)

    Welcome to our Asia Regulatory Roundup, our weekly overview of the top regulatory news in Asia. Japanese Regulators Receive Orphan Drug Training from EMA Staffers at Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) have received training on orphan medicines from the European Medicines Agency (EMA). The training session was led by Dr. Segundo Mariz, a member of EMA’s Committee for Orphan Medicinal Products (COMP). Mariz has worked on the committee since ...
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    Studies Raise New Concerns Over FDA's Expedited Approval Designations, Supplemental Indications

    An increasing number of newly-approved drugs by the US Food and Drug Administration (FDA) have been associated with expedited development or review programs, though many of these approved drugs are not first in class and potentially less innovative, according to two new studies published in the British Medical Journal (BMJ). Background Under the standard regulatory review process, FDA has 10 months to review a potential new drug's safety and efficacy based on a company'...