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  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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...
  • Regulatory NewsRegulatory News

    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 ...
  • Regulatory NewsRegulatory News

    Bill Would Close Loopholes Now Preventing Some Generic Drugs From Being Approved

    A new bill introduced last week would seek to limit the use of safety programs required by the US Food and Drug Administration (FDA) to delay and even prevent the introduction of generic drugs into the market. Background REMS were first introduced under the 2007 Food and Drug Administration Amendments Act (FDAAA) , and were meant to act as a regulatory hedge against potentially risky products. REMS typically include the following elements: A communication plan, Medicat...
  • Regulatory NewsRegulatory News

    Pfizer Launches Expanded Access Program for Breast Cancer Drug

    The US Food and Drug Administration's (FDA) expanded access program, sometimes referred to as its "Compassionate Use" program, has frequently been in the news as of late, as dozens of patients have taken to social media in an attempt to pressure companies into allowing them access to clinical trials they believe might save their lives. But while many companies have seemed reluctant to grant access to those trials, pharmaceutical giant Pfizer is taking a markedly differen...
  • Regulatory NewsRegulatory News

    Senators Want Detailed Data on FDA's Compassionate Use Program, Including Potential Improvements

    Three Republican legislators are pressing the US Food and Drug Administration (FDA) to provide them with information about its application of its expanded access regulations, saying they are in the midst of performing "oversight" over the program. Background FDA's expanded access regulations, sometimes referred to as its "compassionate use" program, allow patients to access clinical trials they otherwise might not be eligible for. The program is meant to offer ho...
  • FDA Launches Long-Awaited Expedited Approval Program Aimed at High-Need Devices

    The US Food and Drug Administration (FDA) has released a long-awaited draft guidance document outlining an expedited approval pathway that can be used by sponsors of medical devices intended to treat life-threatening or "irreversibly debilitating" diseases or conditions. Background: EAP Basics The guidance, which for months has been rumored to be near completion , introduces the "Expedited Access Premarket Approval" program, or EAP for short. Regulatory officials said ...