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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 Challenges in Orphan Drug Development In August, FDA released a draft guidance intended to address the most common issues faced by drugmakers developing treatments for rare diseases .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'...
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    Bipartisan Group of Senators Introduces Bill to Speed Development of Rare Disease Drugs

    A new bipartisan bill aims to help accelerate the development of targeted drugs to treat rare diseases, including Duchenne muscular dystrophy, cystic fibrosis, some cancers and other genetic diseases. Although it's unclear how exactly the bill will aid in the development of targeted rare disease treatments, one of the cosponsors, Sen. Elizabeth Warren (D-MA), said the bill would clarify the US Food and Drug Administration's (FDA) current authority to consider resear...
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    EMA Wants to Help Drugmakers Show Their Orphan Products' Benefits

    The European Medicines Agency (EMA) is looking help sponsors developing new orphan drugs by clearing up questions on how they can demonstrate their product's improvement over existing therapies. To do so, the agency will be hosting a workshop on 7 December 2015, where it says industry, regulators, health technology assessment (HTA) bodies and other groups will have the opportunity to discuss approaches to demonstrating "significant benefit" for orphan products. Orphan D...
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    What Causes Variations in Review at CDER? It's All About the Designation

    Last year, a study by the Manhattan Institute for Policy Research (MIPR) found variations in review time at the US Food and Drug Administration's (FDA) various drug review divisions were caused by inefficiencies at the agency. Now, FDA representatives are making the case that the variation in review times can be explained by the proportion of applications receiving accelerated review in different therapeutic areas. The MIPR study concluded that the variations in rev...
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    New FDA Guidance Addresses Common Issues in Orphan Drug Development

    The US Food and Drug Administration (FDA) has released a draft guidance intended to help drugmakers tackle common issues encountered in the development of drugs to treat rare diseases. Background In the US, orphan drugs, or drugs used to treat rare diseases which affect fewer than 200,000 people, are given incentives, such as a longer period of marketing exclusivity, tax credits and user fee waivers. These incentives were first instituted with the passage of the 1983 ...
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    Japan Edges out FDA for Fastest Approvals

    In 2014, Japan's regulator, the Pharmaceuticals and Medical Devices Agency (PMDA) edged out the US Food and Drug Administration (FDA) as the world's fastest regulator, with a median approval time of 306 days for new active substances (NASs). New Drug Approvals in ICH Countries A new report by the Centre for Innovation in Regulatory Science (CIRS), New Drug Approvals in ICH Countries 2005-2014 , examines NAS approvals in the US, EU and Japan. The report finds that FD...