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  • Regulatory NewsRegulatory News

    FDA: 30 Percent of Drugs Used in Expanded Access Programs are Later Approved

    A new paper by a team of US Food and Drug Administration (FDA) officials finds that only about 30%of drugs requested for expanded access go on to be approved by the agency. "Expanded access provides just that: access. There is no guarantee that the product sought will be effective and/or safe, much less that it will be effective and/or safe for the particular patient," the authors write. Background In recent years, the debate over patient access to unapproved drugs has...
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    FDA to Close Loophole Allowing Companies to Skirt Pediatric Study Requirements

    FDA commissioner Scott Gottlieb announced Tuesday that the agency will release guidance to close a loophole that allows companies to avoid their obligation to study pharmaceuticals in pediatric populations. The situation, according to Gottlieb, arises if sponsors received an orphan designation for a pediatric subtype of an otherwise common and non-orphaned adult disease. For example, with a condition like inflammatory bowel disease (IBD), a drug may be approved to...
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    FDA Reports on FY2016 Pediatric Device Approvals

    In a report to Congress released this week, the US Food and Drug Administration (FDA) says it approved 13 high-risk medical devices for pediatric patients in FY2016, the highest number since 2011 when 15 were approved. Under the Food and Drug Administration Amendments Act of 2007 (FDAAA), device makers are required to include information in premarket approval (PMA) and humanitarian device exemption (HDE) applications about pediatric subpopulations that are affected by ...
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    GAO: FDA Improved Expanded Access but More Clarity Necessary

    With the rise of Right-to-Try legislation across states in the US, it might seem like patients with serious or life-threatening ailments and no other comparable medical options might have difficulties in obtaining access to investigational drugs outside of a clinical trial because of the US Food and Drug Administration (FDA). But a new Government Accountability Office report released Tuesday lends credence to many who have voiced concerns with the Right-to-Try laws,...
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    10 Years in, EU Pediatric-Use Marketing Authorization Called a 'Disappointment'

    Biopharmaceutical companies and regulators across Europe have said that in the 10 years since the Pediatric Regulation took effect, some pediatric therapeutic areas have seen progress, though the pediatric-use marketing authorization (PUMA) has not lived up to expectations. Overall, according to a summary of the comments received from 75 stakeholders on the consultation from last November , the European Commission noted that the reward system built into the regul...
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    EMA and FDA Look to Facilitate Development of Gaucher Disease Treatments

    The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) on Monday released a new strategy document to aid in the development of new treatments for Gaucher disease, a rare lysosomal storage disorder. Given the limited number of patients worldwide with Gaucher disease, EMA and FDA put forth two possible approaches to better facilitate investigations of such products: Extrapolation across age groups within a single drug development program...
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    FDA Amends Humanitarian Device Exemption Regulations

    The US Food and Drug Administration (FDA) on Tuesday announced that it is amending its regulations governing humanitarian device exemptions (HDE) to reflect changes brought on by the 21st Century Cures Act . These exemptions provide a pathway for device makers looking to develop medical devices for small patient populations by exempting them from demonstrating a device's effectiveness. Instead, a company must demonstrate that the humanitarian use device (HUD) "will no...
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    FDA Approved Nearly All Expanded Access Requests in FY2016

    The US Food and Drug Administration (FDA) granted more than 99% of requests for patients to access unapproved drugs and biologics in fiscal year 2016, according to data released last week. Through FDA's expanded access program, the agency allows patients with serious or life-threatening diseases to access unapproved drugs. Under the program, physicians can request access to a drug for individual patients or a group of patients under an expanded access investigational ne...
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    FDA Delays Final Rule on Intended Uses

    The US Food and Drug Administration (FDA) on Friday announced it will delay implementation of its final rule clarifying when tobacco products are regulated as medical products until March 2018 in response to a petition from biopharmaceutical industry groups that raised questions about how the rule would alter the agency's definition of intended uses for drugs and medical devices. The petition, filed by on behalf the Medical Information Working Group (MIWG), the Pharmaceu...
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    Pediatric Cancers: New Bill Seeks to Close Clinical Trial Requirement Loopholes

    A new bipartisan bill reintroduced in the House and Senate in late February, which could ultimately be attached to the reauthorized Prescription Drug User Fee Act (PDUFA), seeks to close the loopholes biopharmaceutical companies use to skirt around clinical trial requirements for cancer treatments in pediatric populations. Background Pediatric study requirements are included in both the Pediatric Research Equity Act (PREA), which requires biopharma companies to ...
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    CTTI Offers Recommendations for Pediatric Clinical Trials for Antibiotics

    The Clinical Trials Transformation Initiative (CTTI) has released a set of recommendations it says will improve the use of antibiotics in children. According to CTTI, a public-private partnership aimed at improving the quality and efficacy of clinical trials, challenges in enrolling and completing pediatric clinical trials for antibiotics leads to a situation where such drugs are used in children despite lacking adequate labeling for pediatric populations. Furthermore, ...
  • Regulatory NewsRegulatory News

    Industry Groups Petition FDA Over Revisions to ‘Intended Use’ Rule

    The Medical Information Working Group (MIWG), the Pharmaceutical Research and Manufacturers of America (PhRMA) and the Biotechnology Innovation Organization (BIO) have petitioned the US Food and Drug Administration (FDA) following its “unexpected decision” last month to revise the definitions of “intended use” for drugs and medical devices. The industry groups call for this final rule to be stayed indefinitely and reconsidered because FDA failed to give fair no...