• Regulatory NewsRegulatory News

    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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    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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    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.” ...
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    Orphan Drugs in the EU: A Record-Breaking Year

    The European Medicines Agency (EMA) recommended 17 drugs with orphan product designation for approval in 2014, the most ever in a single year. This is much higher than the average of 6.75 per year between 2010 and 2013 and is a significant increase from 2013, when EMA recommended 11 products with orphan designation for approval. Background: Orphan Designation Orphan product designation was first introduced in the EU in 2000 under Regulation (EC) No 141/2000 . The reg...
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    FDA Explains How its New Incentive Program for Rare Pediatric Disease Treatments Works

    The US Food and Drug Administration (FDA) has released a long-anticipated guidance document intended to explain how a new incentive program known as the Rare Pediatric Disease Priority Review Voucher system works. Background Created in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA) , the Rare Pediatric Disease Priority Review Voucher is modeled closely off a similar program known as the Tropical Disease Priority Review Voucher system. T...
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    First Pediatric Priority Review Voucher Goes up for Sale, Fetching $67M

    As far as regulatory incentives go, some are pursued more than others. The US Food and Drug Administration's (FDA) orphan drug exclusivity provisions, for example, have brought about a renaissance of therapies meant to treat patients suffering from rare diseases and conditions. But on the other side of the coin are incentives not often sought out by companies. And perhaps no incentive maintained by FDA is used less than its priority review vouchers. Background: Vouchers...
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    Head of FDA's Rare Disease Division Departs, Leaving Agency Looking for New Leader

    The US Food and Drug Administration (FDA) is looking for a new leader for its Rare Diseases Program. In an email to staff on 16 July 2014, John Jenkins, director of the Office of New Drugs (OND), which oversees the rare disease program, said the program's current leader would be transitioning to a new role at FDA. Anne Pariser, OND associate director for rare diseases, will take on a new position in the Office of Translational Sciences, Jenkins said. Pariser will still ...