• Regulatory NewsRegulatory News

    Bill Would Give Some Reformulated Drugs Extra Protection from Generics

    New legislation introduced in the US House of Representatives would give some pharmaceutical products an extra two years of marketing exclusivity if their sponsors are able to demonstrate that the redesigned or reformulated drugs improve adherence, decrease abuse or otherwise make the drug safer to take. Background The bill, the Promoting Access for Treatments Ideal in Enhancing New Therapies (PATIENT) Act of 2015 , was introduced in the House on 13 March 2015 by Rep...
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    Medical Device Risk Management by Regulatory Authorities

    Regulatory Authorities are challenged to keep their markets open and to ensure that patients have proper access to devices while at the same time keeping non-compliant, possibly dangerous devices away from their populations. To achieve this they need to use risk control strategies. Authorities that are rethinking their approaches to risk management could consider a three-tier system: A 100% check on all devices for a limited number of items per device; A more extensive...
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    UK Regulator Issues First Positive Opinion in Early Access Program

    Merck’s new skin cancer treatment has become the first product approved under a new accelerated review process by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA). The product, Keytruda, has been shown to be effective in slowing the progression of advanced melanoma, a deadly form of skin cancer with few treatment options. Accelerated Approval Keytruda is the first product to be approved under MHRA’s early access to medicines scheme (EAMS), whic...
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    From 100 Hours to 1: FDA Dramatically Simplifies its Compassionate Use Process

    In response to repeated criticism that terminally ill patients find it too difficult to obtain access to experimental and potentially life-saving drugs, the US Food and Drug Administration (FDA) has announced changes to its "compassionate use" process in the hopes of making it easier for physicians to request access to medical products on behalf of their patients. Background FDA's "compassionate use" process—formally known as its Expanded Access Program—is meant to allo...
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    FDA Sees 92% Increase in Requests for Compassionate Access to Medicines in 2014

    Last year, the US Food and Drug Administration (FDA) received—and approved—substantially more requests for "compassionate" access to potentially life-saving treatments than any time since at least 2010, according to new data provided by the regulator. Background The data relate to FDA's "expanded access" program—sometimes called its "compassionate use" program—which was created to allow a pathway for terminally or critically ill patients to obtain access to drugs they...
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    Pharmaceutical Companies Look to Create Better Compassionate Use Process

    • 11 December 2014
    The prominent US pharmaceutical trade group PhRMA has released an updated set of principles it says reflects the US pharmaceutical industry's "commitment" to provide patients with life-threatening diseases access to investigational therapies on a compassionate-use basis. Background The policy is meant to accommodate patients who are otherwise not eligible to participate in a clinical trial for a new drug, either because they are too sick to participate, have a disqualif...
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    Legislation Seeks to Overhaul FDA's Compassionate Use Program

    New legislation introduced in the House of Representatives would makes changes to the US Food and Drug Administration's (FDA) "expanded access"—sometimes called " compassionate use "—policies in the hopes of making it easier for terminally ill patients to access potentially life-saving drugs. Background Under normal circumstances, companies seeking FDA approval for a new drug will first ask regulators for permission to begin a clinical trial using an application known a...
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    Laws Spreading That Allow Terminal Patients Access to Experimental Drugs

    Earlier this month, Arizona voters  approved a referendum  that allows terminally ill patients to receive experimental drugs and devices. It’s the fifth state to approve a “right-to-try” law this year. Supporters say the laws give dying patients faster access to potentially life-saving therapies than the Food and Drug Administration’s existing  “expanded-access” program , often referred to as “compassionate use.” But critics charge they’re feel-good laws that don’t addre...
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    Company's Compassion Leads to Clinical Hold on Experimental Drug

    If you're trying to understand why some drug companies are reluctant to allow desperate patients to enroll in their early-stage clinical trials, look no further than the case of CytRx Corporation, whose clinical trial for the drug compound aldoxorubic is now partially on hold following the death of a patient enrolled in the trial on a compassionate use basis. Background Companies seeking approval to market a drug product in the US must first prove to the US Food and Dru...
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    Think Tank Seeks Ebola Info from FDA, Calling it a Test Case for Compassionate Use System

    A libertarian-leaning think tank is pressuring the US Food and Drug Administration (FDA) to release information regarding the internal approval processes it used to permit two US citizens stricken with Ebola access to Mapp Biopharmaceuticals' ZMapp, an experimental drug. Background In August 2014, the American public became fixated on an outbreak of Ebola in West Africa after two US missionaries, Dr. Kent Brantley and Nancy Writebol, became infected with the disease. In...
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    FDA Leverages Compassionate Use Program to Allow Access to Experimental Ebola Drug

    Canadian biopharmaceutical company Tekmira has announced that the US Food and Drug Administration (FDA) will allow its investigational Ebola drug, TKM-Ebola, to be used by patients under an emergency expanded access program—a stark reversal for the product, which two months ago was subject to a complete clinical hold. Background As Regulatory Focus extensively explained in August , developing new treatments for Ebola is a long and arduous process. Because Ebola, a he...
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    After New Drugs Delayed by DEA, Legislators Seek Overhaul of Drug Scheduling Process

    For most pharmaceutical products in the US, obtaining approval from the US Food and Drug Administration (FDA) to market a product means a company can begin selling its product as soon as it wants. But for a subset of products subject to the Controlled Substances Act (CSA ) , the Drug Enforcement Administration (DEA)—not FDA—is the ultimate arbiter of when market access occurs and the extent to which it occurs. Now new legislation wants to make the process by which DEA ...