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    EMA Offers New Draft Guidance on Developing Alzheimer’s Treatments

    The European Medicines Agency (EMA) on Monday released new draft guidance on the development of Alzheimer’s disease treatments that would allow treatments to be evaluated in earlier disease stages before dementia sets in. The new guidance comes as EMA recognizes that the field of Alzheimer’s disease (AD) research and development “witnessed a recent paradigm shift in the diagnostic framework of AD which is now considered a continuum with a long-lasting presymptomatic phas...
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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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    First-in-Class, Rare Disease Drug Approvals Dominated in 2015: A Breakdown from FDA

    Last year was a banner year for the US Food and Drug Administration (FDA) in terms of new drug approvals, with many of the new drugs coming to market to help rare disease patients and offer new first-in-class options. The total of 45 approvals was well ahead of FDA’s average of about 28 novel approvals between 2006 and 2014, and 36% of those new approvals were for first-in-class drugs while almost half (47%) were to treat rare diseases, or ailments that impact 200,000 or...
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    FDA Faces Sensitive Challenge With Review of DMD Drug

    The blanketing of snow on the East Coast this past weekend prompted the postponement of a high-profile US Food and Drug Administration (FDA) advisory committee hearing for a Duchene Muscular Dystrophy (DMD) drug that is stirring up public controversy and will eventually lead to an up or down decision testing FDA’s resilience to public pressure. The advisory committee meeting, which the Jett Foundation, a DMD advocacy group, said Tuesday will likely be rescheduled withi...
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    Experts Call for New Regulatory Pathways for Antibiotics

    Faced with the increasing threat of antimicrobial resistance (AMR), experts are calling for more incentives to boost investment in new antibiotics. At the FDA/CMS Summit in Washington, DC today, representatives from several companies developing new antibiotic treatments said that while recent initiatives such as the Generating Antibiotics Now Act (GAIN Act) have helped revitalize investment in antibiotic development, more needs to be done to encourage research and deve...
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    Health Advocacy Groups Call for Changes to Priority Review Voucher Program

    A group of health advocacy organizations are calling on the Senate Committee on Health, Education, Labor and Pensions (HELP) to tighten requirements for obtaining priority review vouchers for drugs for tropical diseases. In a letter addressed to HELP Committee Chairman Lamar Alexander (R-TN) on Tuesday, seven organizations including Médecins Sans Frontières (MSF), Drugs for Neglected Diseases Initiative (DNDi), Center for Global Health Policy, TB Alliance, Treatment Acti...
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    Senators Propose New Exclusivity Voucher, Priority Review Voucher Programs

    Senators on both sides of the aisle are trying to incentivize the development of new drugs for newborn babies and medical countermeasures via a new exclusivity voucher and a priority review voucher system that are similar to the voucher programs already linked to tropical and rare pediatric disease treatments. Neonatal Exclusivity Voucher Last month, Sen. Robert Casey (D-PA) introduced a bill, known as Promoting Life-Saving New Therapies for Neonates Act of 201...
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    Senators Introduce Two Bills to Streamline FDA Reviews of Medical Devices

    Sens. Cory Gardner (R-CO) and Joe Donnelly (D-IN) late Wednesday announced the introduction of two new bills that would further streamline the US Food and Drug Administration (FDA) approval process for certain medical devices. One of the bills -- the Rare Device Innovation Act -- would expand the threshold by which FDA could approve medical devices that treat rare diseases, including ALS, cerebral palsy, Hodgkin’s lymphoma, mesothelioma and tuberculosis. Currently, FDA...
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    Harvard Professor Questions Success of FDA's Priority Review Voucher Program

    As the Senate debates whether to extend or make permanent the US Food and Drug Administration's (FDA) priority review voucher (PRV) program, a Harvard medicine professor says that so far, at least for tropical diseases, there's "little reliable evidence" that the program has spurred novel drug development. Background Since 2007, the FDA has issued seven PRVs (the most recent of which came earlier this month), which allow sponsors to speed the review of any one...
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    FDA Raises Fee Rate for Rare Pediatric Disease Priority Reviews for FY2016

    The US Food and Drug Administration (FDA) on Friday announced that it would increase the rare pediatric disease priority review voucher fee rate for FY 2016 by about $200,000. The new rate -- $2,727,000 – is effective on 1 October and will remain in effect for the next year. FDA previously set the fee rates at $2,562,000 for FY 2015 and $2,325,000 for FY 2014. The new rate is based on FDA's estimate that the cost of a standard review for new molecular entity (NME) new dr...
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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...