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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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    Sanofi Bets Nearly a Quarter Billion on Voucher for Faster Drug Approval

    An unusual regulatory incentive used to accelerate the review of a small number of drug products has just sold to the French pharmaceutical giant Sanofi for the record-setting sum of $245 million. Background The incentive, known as a Rare Pediatric Disease Priority Review Voucher (Pediatric PRV), is an integral part of a program meant to spur the development of new therapies of rare pediatric diseases. The Pediatric PRV program was established under the Food and Dru...
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    Public Health Groups Propose Global Drug Innovation Fund

    Two health advocacy groups, Médecins Sans Frontières (MSF) and the Drugs for Neglected Diseases initiative (DNDi) are urging public health authorities to create a global fund to tackle what they call "deadly gaps in innovation." Citing the rising threat of antimicrobial resistance (AMR), emerging infectious diseases and neglected tropical diseases (NTDs), the two organizations say the pharmaceutical industry is failing to address certain public health challenges. Back...
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    FDA Exercising 'Extraordinary' Flexibility on Drugs for Rare Diseases, New Study Finds

    The US Food and Drug Administration (FDA) is a reasonable regulator. That's the conclusion of a new analysis looking at the measures taken by FDA to review and approve new drugs intended for use in rare diseases affecting small populations of patients. Background The root of this "reasonable" argument starts with a law intended to facilitate the development of new therapies for patients with rare diseases. Under the 1983 Orphan Drug Act , a rare disease is one which a...
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    Australia Wants Input on Orphan Drugs Program as it Conducts Review

    Regulators in Australia today released a discussion paper intended to solicit comments on its orphan drugs program, in light of changes in the landscape for rare diseases since the program's inception in 1997. Orphan Drugs Regulation in Australia Orphan drugs are products intended to treat, diagnose or prevent rare diseases. Because the potential treatment populations for these drugs are small, regulators often offer incentives to encourage the development of treatments...
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    EMA Says Companies Still Need to Report Side Effects of Donated Drugs

    Drug makers are frequently involved in relief and humanitarian aid efforts, often through donating their products. However, in light of recent outbreaks of neglected tropical diseases such as Ebola, the World Health Organization (WHO) has asked the European Medicines Agency (EMA) to clarify its drug safety monitoring requirements for companies that donate medicines outside the EU to treat neglected tropical diseases. According to WHO, neglected tropical diseases are a...
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    Bill Would Make Permanent FDA's Rare Pediatric Voucher Program

    New legislation introduced this week would reauthorize the US Food and Drug Administration's (FDA) rare pediatric disease priority review voucher program, which is currently set to end after triggering a sunset clause in its authorizing statute. Background Priority review vouchers are incentives meant to catalyze the development of new therapies for historically under-served disease areas, such as rare pediatric diseases affecting fewer than 200,000 children in the US...
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    Pediatric Priority Review Voucher Program Set to End After FDA Approves New Drug

    The US Food and Drug Administration's (FDA) rare pediatric disease priority review voucher program is slated to end in one year, after the agency awarded its third-ever rare pediatric voucher, thereby triggering a little-known provision in the voucher program. Background Regular readers of Regulatory Focus will be familiar with FDA's priority review voucher programs. We've written about them extensively over the years, and the vouchers are the focus of our most rec...
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    With New Data in Hand, Could Sanofi and Regeneron Use a Regulatory Shortcut?

    A new drug being co-developed by drugmakers Sanofi and Regeneron could, according to new data , dramatically decrease low-density lipoprotein (LDL) cholesterol in patients and lessen cardiac events. But it's a reduction in something else that could be most important for Sanofi and Regeneron: the time it might take the US Food and Drug Administration (FDA) to review the new drug, Praluent. Background In July 2014, Sanofi and Regeneron announced they had purchased what...
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    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" ...
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    Rare Disease Patient Group Commends EMA, Wants EU-Wide Access to Treatment

    A rare disease advocacy network has applauded the European Medicines Agency’s (EMA) efforts to incentivize the development of treatments for rare diseases , but says  the variance in treatment access across the EU is “unacceptable.” In an interview with EurActiv , Terkel Andersen, president of the advocacy group EURODIS, said that EMA and the European Commission (EC) are “doing their utmost to try to make rare diseases ‘attractive’ for the pharmaceutical industry.” ...