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  • Regulatory NewsRegulatory News

    Tufts Outlines Regulatory Initiatives to Spur Innovation in 2016

    Further harmonization among major regulatory agencies globally will look to focus on pediatric drug studies in 2016, according to a new report from the Tufts Center for the Study of Drug Development. The report released this week outlines not only what the drug development industry should expect for 2016, but also how regulators will play a major role in keeping drugs safe and in some cases extending marketing exclusivity periods for certain classes of drugs deemed crit...
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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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    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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    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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    EMA Hopes Early Dialogue With Drugmakers Will Spur Pediatric Drug Development

    The European Medicines Agency (EMA) is piloting a new initiative to offer early meetings with companies to foster discussions about their pediatric development strategies for new medicines. Background Companies are often reluctant to conduct clinical studies in children due to ethical concerns and fear of adverse events during trials. However, conducting clinical research on children is critical to determining whether drugs are safe and effective in pediatric population...
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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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    Bioethics Council Calls for Reforms for Clinical Trials Involving Children

    The Nuffield Council on Bioethics has released a report on the ethical issues surrounding clinical research involving children in the UK and EU. The report, which looks at the current status of clinical research and regulations, argues better regulation and greater involvement from children is critical to increasing the number of clinical trials involving children. Background Companies are often reluctant to conduct clinical studies on children due to ethical concerns ...
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    FDA Explains How to Extrapolate Data for Pediatric Medical Devices in New Guidance

    A new draft guidance document quietly issued last week by the US Food and Drug Administration (FDA) is intended to explain how medical device manufacturers can show their devices are safe for use in pediatric populations. Background There is often a "paucity of scientific evidence available to substantiate submissions for devices that are indicated for use in the diagnosis or treatment of pediatric patients," FDA explains in the forward of its new draft guidance, Leve...
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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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    EU Committee Wants Additional Restrictions on Codeine Use in Children

    A European committee is recommending a series of restrictions on the use of medicines containing codeine in children. PRAC’s Recommendations Citing concerns that codeine can cause breathing problems and other serious adverse effects in children, the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) is recommending codeine be contraindicated for children less than 12 years of age. Additionally, PRAC says that children between ages 12 ...