The European Medicines Agency has released a draft guidance to assist drug developers using its Priority Medicines (PRIME) scheme to accelerate authorization of new therapies.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...

In a nearly 350-page report to Congress on the federal government’s COVID-19 response, the Government Accountability Office (GAO) calls on the US Food and Drug Administration (FDA) to review its inspections approach and come up with a plan to address its looming backlog.   Pandemic inspection alternatives   In the early days of the pandemic, FDA halted non-mission-critical foreign and domestic inspections and relied on alternative tools, such as inspection reports ...

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...

In guidance released Monday, the US Food and Drug Administration (FDA) provides the timelines it will stick to when reviewing responses to complete response letters (CRLs) for drugs and biologics when a facility assessment or inspection is required in order for the agency to make a decision on the application.   FDA’s ability to conduct on-site inspections has been dramatically curtailed amid the COVID-19 pandemic due to public health concerns and travel restrictions. ...

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. ...

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...

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...

The US Food and Drug Administration (FDA) is seeking to withdraw the approval of the only drug indicated to prevent recurrent preterm birth, based on a post-approval study showing a lack of efficacy.   Makena (hydroxyprogesterone caproate injection) was originally granted accelerated approval in 2001 and remains on the market. Makena’s manufacturer, AMAG Pharmaceuticals, has requested a public hearing to explore the issue. It is up to the FDA commissioner whether to gr...

The US Food and Drug Administration (FDA) has been using the Risk Evaluation and Mitigation Strategies (REMS) program as a way to mitigate adverse events stemming from opioid misuse and abuse, but that program is a poor tool for fighting the opioid epidemic, the Health and Human Services Office of the Inspector General concluded in a new report .   The REMS program is a long-standing drug safety program that has allowed the FDA to require manufacturers to implement ...

Two new draft guidances from the US Food and Drug Administration give investigators a roadmap for clinical trials of adjuvant drug or biologic therapy for two common cancers. The first of the guidances released on 1 October addresses adjuvant treatment for bladder cancer, and the other addresses adjuvant treatment of renal cell carcinoma (RCC).   “Currently, there is significant variability in the design, conduct, and analysis of clinical trials” that examine adjuvant ...

The US Food and Drug Administration (FDA) on Wednesday issued an immediately effective frequently asked questions guidance  explaining its approach to inspections and manufacturing and supply chain changes during the coronavirus disease (COVID-19) pandemic.   Inspections   For the time being, FDA says it is still following the approach to resuming domestic inspections it laid out last month and will continue to postpone non-mission-critical foreign pre-approval an...

Months after halting most inspections amid the coronavirus disease (COVID-19) pandemic, the US Food and Drug Administration (FDA) on Friday said it plans to resume on-site domestic inspections beginning the week of 20 July 2020.   As a safety measure, FDA will pre-announce all inspections, save for retail tobacco inspections, for the foreseeable future.   The announcement comes nearly two months after the agency said it will implement a phased approach to restartin...