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    GOP Tax Reform Bill Would Repeal Orphan Drug Research Credits

    Republicans on Thursday rolled out their new tax reform bill, which among other provisions to lower taxes for Americans and businesses, proposes to repeal a provision that might cause the biopharma industry some concern. Under the House bill’s Subtitle E, section 3401 would repeal what amounts to half of the qualified clinical research costs for designated orphan drug products. Background Under the  Orphan Drug Act  of 1983, Congress sought to incentivize the d...
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    FDA Analyst Counters Critiques of Orphan Drug Act

    While recent reports have claimed that drugmakers are manipulating the incentives provided by the Orphan Drug Act , Mike Lanthier, an operations research analyst at the US Food and Drug Administration (FDA), says that in most cases, the act is working as intended. "My sense is that the Orphan Drug Act has been a successful catalyst for spurring rare disease development," Lanthier said at the National Organization for Rare Disorders' (NORD) Summit in Washington, DC on ...
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    Rare Diseases: FDA Awards Grants for 21 Clinical, Natural History Studies

    The US Food and Drug Administration (FDA) on Friday awarded $32 million in research grants to fund 15 clinical studies and six natural history studies for rare diseases. The grants are being funded through FDA's orphan products grants program , with $22 million going to the clinical studies and $9.8 million in combined funding from FDA and the National Center for Advancing Translational Sciences' (NCATS) Therapeutics for Rare and Neglected Diseases program going to fund...
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    Protalex Wins $403K Grant from FDA Office of Orphan Products Development

    Clinical-stage biopharma Protalex, Inc. on Thursday said it has been awarded a $403,000 grant from the US Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) to support future clinical development activity of PRTX-100 as a treatment for immune thrombocytopenia (ITP). The grant is part of a larger program that will award $23 million in research grants for 21 clinical trials over the next four years to boost the development of rare dise...
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    FDA Plots Elimination of the Orphan Drug Designation Request Backlog

    With a flood of new orphan drug designation requests, the US Food and Drug Administration (FDA) on Thursday released its new plan to eliminate the backlog of requests in 90 days and to respond to 100% of all new orphan drug designation requests within 90 days. In 2016, the Office of Orphan Products Development (OOPD) received 568 new requests for designation – more than double the number of requests received in 2012. According to FDA’s database of 4,174 orphan d...
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    FDA Commissioner Vows to Eliminate Backlog of Orphan Drug Designation Requests

    At a Senate hearing on President Donald Trump's FY2018 budget request for the US Food and Drug Administration (FDA), Commissioner Scott Gottlieb vowed to eliminate the backlog of orphan drug designation requests and said he will soon release "modern and risk-based" tools for assessing new treatments, especially for rare diseases and conditions with no effective treatments. "Right now we have a backlog of about 200 orphan drug designation requests where we haven't respond...
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    European Commission and EMA Offer Guidance on Brexit for Pharma Companies

    As marketing authorisation holders (MAHs) based in the UK begin to form plans to transfer their marketing authorizations (MAs) to holders established in the EU, Norway, Iceland and Liechtenstein, the European Medicines Agency (EMA) and European Commission on Wednesday released guidance to prepare industry for the UK’s withdrawal from the EU. The question and answer document is the first in a series of guidances and follows a notice sent earlier this month to ...
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    TGA to Move Forward With Orphan Drug Revamp

    Australia's pharmaceutical industry is "broadly supportive" of proposed changes to the country's orphan drug program, the Therapeutic Goods Administration (TGA) says. On Tuesday, TGA released feedback it received in response to a 2016 consultation on the proposed changes, which include raising the population threshold for rare diseases, taking the seriousness of the condition into account when making designations and requiring sponsors to demonstrate significant benefit ...
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    Sen. Grassley Opens Inquiry Into Orphan Drug Act

    Sen. Charles Grassley (R-IA) on Friday confirmed to Focus that he is gathering more information and discussing with his staff and interested parties a possible inquiry into the Orphan Drug Act ’s abuses leading to high drug prices. “Based on the reporting from Kaiser Health News [KHN] about how the orphan drug provisions appear to be stretched beyond their original intent, and the strong consumer concern about high drug prices, I'm interested in learning whether...
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    European Commission Clears up Questions on Orphan Drug Regulation

    Following a public consultation in November 2015, the European Commission (EC) has released a new communication intended to clarify some lingering questions about the EU's Orphan Regulation . The consultation sought to address five issues that have arisen since the Orphan Regulation came into effect in January 2000: Clarifying the meaning of "significant benefit" How to apply the Orphan Regulation to emerging diseases, such as Ebola, that are not present within...
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    EMA Offers New Guidance on Type II Variations and MA Extensions of Orphan Drugs

    The European Medicines Agency (EMA) has updated its post-authorization guidance on how extensions of marketing authorizations and type II variations may impact orphan-designated medicines. The updates follow the publication of a new  notice by the European Commission on the application of Articles 3, 5 and 7 of Regulation (EC) No. 141/2000 on orphan medicinal products . In this notice, the European Commission provides guidance on the application of the Orphan Regulation,...
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    FDA Helps Fund 21 Early Phase Trials for Rare Disease Treatments

    The US Food and Drug Administration (FDA) on Monday announced it is awarding $23 million in research grants for 21 new clinical trials over the next four years to boost the development of rare disease treatments. The grants, awarded to researchers from academia and industry at domestic and international clinical sites, are part of FDA’s Orphan Products Clinical Trials Grants Program, created in 1983, which has provided more than $370 million (including $19 million ...