• Regulatory NewsRegulatory News

    ICH survey shows strong level of guideline implementation

    Most of the 55 guidelines from the International Council on Harmonisation that relate to quality, safety and efficacy, as well as multidisciplinary guidelines, have been adopted among its 10 non-founding members and observers and 30 pharmaceutical companies, according to the results of a new survey.   The survey was designed to monitor the adequacy and level of implementation to ICH guidelines by regulators and industry and complements one released in 2019 on the adopt...
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    COVID-19 therapeutics tracker

    More than a year into the pandemic, only a handful of repurposed therapeutics have been approved to treat COVID-19: dexamethasone in the UK and Japan; Avigan (favilavir) in China, Italy and Russia; and Veklury (remdesivir) in the US, UK and Japan.   Regulators around the world have allowed a handful of treatments to be used on an emergency-use basis including Eli Lilly and Company’s monoclonal antibodies bamlanivimab with etesevimab; Regeneron’s casirivimab and imdevim...
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    FDA report: Drug quality tops OPQ's list of product defects

    A new report from the US Food and Drug Administration's (FDA's) Office of Pharmaceutical Quality (OPQ) details the top drug quality issues seen in from 2016-2020, with questions of product quality topping the list.   In all, just over a quarter (27%) of all Product Quality Defect (PQD) reports were related to product quality issues, while device and packaging issues together made up another third of the PDQ reports over the 5-year period.   The PQD reports came fro...
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    EMA recommends bluebird bio's Skysona, seven other medicines

    The European Medicines Agency (EMA) issued a favorable opinion for bluebird bio’s Skysona (elivaldogene autotemcel), a gene therapy that treats early cerebral adrenoleukodystrophy (CALD). Seven other medicines also received positive recommendations from the agency’s Committee for Medicinal Products for Human Use (CHMP) during its May meeting.   Skysona’s status as an advanced therapy medicinal product (ATMP) means it was first assessed by EMA’s Committee for Advanced T...
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    Drug labeling authority, UDI program reviewed by Harvard-based researchers

    Two papers in JAMA Internal Medicine highlighted separate, but important issues concerning the US Food and Drug Administration’s authority to modify package inserts and the effect of a recently shuttered agency initiative.   In a Viewpoint, Bishal Gyawali, MD, PhD, and colleagues at the Program on Regulation, Therapeutics and Law (PORTAL) research group at Harvard Medical School and Brigham & Women’s Hospital suggested improving FDA’s authority to modify drug package...
  • ReconRecon

    Recon: Kerfuffle over WHO's Wuhan mission; BMS inks $1.3 B cancer deal

    Recon:   Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US New, Daily Coronavirus Cases Drop Below 100K For First Time In Months ( NPR ) U.S. Continues To Accelerate Vaccination Strategy ( NPR ) Seven coronavirus variants potentially similar to UK type have been detected in US: study ( The Hill ) ( NYT ) Fauci Awarded $1 Million Israeli Prize For 'Speaking Truth To Power' Amid Pandemic ( NPR ) Pentago...
  • FDA’s OPQ reports on drug quality efforts in 2020

    The office tasked with overseeing pharmaceutical quality within the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) released its annual report for 2020 on Thursday, highlighting its effort in a year marked by the COVID-19 pandemic.   In its previous annual report, the Office of Pharmaceutical Quality (OPQ) touted its participation in nearly one third of the preapproval inspections FDA carried out in 2019. For 2020, OPQ instead foc...
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    New toolbox available for EMA's PRIME designees

    The European Medicines Agency has released a draft guidance to assist drug developers using its Priority Medicines (PRIME) scheme to accelerate authorization of new therapies.   “Experience to date has shown that applicants face challenges to complete quality and manufacturing development and data requirements during development of products in early access approaches,” wrote EMA in the executive summary of the guidance.   Challenges for those developing breakthroug...
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    Perspective: Drug price transparency in the Biden era

    The Trump administration’s efforts to increase drug price transparency are likely to survive the transition to the Biden administration, according to a perspective piece published 13 January in the New England Journal of Medicine . Included in the Transparency in Coverage final rule, issued in late October 2020, the drug price transparency provisions aim to require that health insurers publish list prices and historical net prices for prescription drugs. The inform...
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    EC’s new pharmaceutical strategy highlights support for innovation, R&D

    The European Commission (EC) has adopted its pharmaceutical strategy for Europe. The announcement follows a period of consultation earlier this year and meets the EC’s goal of finalizing the strategy by year’s end.   The strategy is not focused on the COVID-19 pandemic, but strategies and considerations included in the document were informed by the impact of the novel coronavirus in Europe and globally.  The primary objectives of the strategy fall into four categories. ...
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    Experts propose changes to FDA’s Accelerated Approval Program

    A new white paper from the Friends of Cancer Research offers a blueprint for reimagining the US Food and Drug Administration (FDA) Accelerated Approval Program for drugs and biologics that treat serious and life-threatening illnesses. The white paper , released during the group’s annual meeting, recommends explicitly adding a more comprehensive risk-benefit assessment to the surrogate endpoints currently used as the basis for approval, a framework that would move the U...
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    EMA's CHMP gives Gamifant a second thumbs down; approves five new medicines

    In its November meeting, a committee of the European Medicines Agency gave its second thumbs down to the monoclonal antibody Gamifant (emapalumab), confirming a July 2020 negative opinion on the Novimmune SA drug.   The US FDA approved Gamifant for pediatric use in primary hemophagocytic lymphohistiocytosis in November 2018; this was the same indication for which EMA has twice declined authorization.   EMA’s human medicines committee (CHMP) had positive recommendat...