BASEL, Switzerland – DARWIN EU is starting to collect more real-world data (RWD) and gain more adherents as an increasing number of regulators and industry groups recognize the value of RWD, Peter Arlett, head of the European Medicine Agency’s data analytics and methods task force, said at DIA Europe 2023.   RWD saw a significant boost from the COVID-19 pandemic, Arlett said. Pre-pandemic, when EMA recommended the establishment of the Data Analysis and Real World Inter...

The Medicines and Healthcare products Regulatory Agency (MHRA) has committed to embarking on the UK’s “biggest overhaul of trial regulation in 20 years” after a consultation revealed support for many of the draft proposals.   At the start of 2022, MHRA sought feedback on proposals to take advantage of the UK’s split with the EU and “deliver a world-class sovereign regulatory environment for clinical trials.”  The goals of the proposed legislative amendments included st...

Welcome to Regulatory Reconnaissance, your daily regulatory news and intelligence briefing.   In Focus: US Moderna to Price Its Covid-19 Vaccine at $130 a Dose ( Wall Street Journal ) Delays in reporting led FDA to late cancer warning on breast implants, advocates say ( Medtech Dive ) Moderna CEO defends price of COVID shot at Senate hearing ( Healthcare Dive ) 'Banding together': 50 female biotech executives lay out plans for board diversity, new companies and men...

Welcome to the March issue of RF Quarterly, which examines patient-focused regulatory practice and includes articles on patient-reported outcomes in regulatory decision making; real-world evidence and postmarket surveillance data; generating evidence for racial and ethnic minorities in oncologic drug development; structured, patient-focused benefit-risk assessment; and the role of the high-performance integrated virtual environment in research using patient-generated data....

Patient-reported outcome measures (PROMs) have been increasingly mandated to measure the success of clinical outcomes in regulatory decision making. However, these outcome measures require expertise in their application and interpretation to allow realistic assessment of the results. Published literature and guidances from the US Food and Drug Administration (FDA) can help with understanding how best these measures can be used in regulatory practice. Particularly, recent w...

This article compares real-world evidence (RWE) and postmarket surveillance (PMS) data collected outside of a randomized, controlled, clinical trial. Real-world data (RWD) include individual patient data points such as complaints and anecdotal findings. The recent explosion in RWD collection has led to new challenges in RWD evaluation globally. Solutions are rapidly evolving to analyze these relatively random data points for scientifically valid purposes. This article disc...

Discussions regarding diversity, equity, and inclusion in healthcare and clinical research have been ongoing for decades. However, the COVID-19 global pandemic brought about a new focus on this issue. As the pharmaceutical industry began strategizing on how to improve diversity in its clinical trials, the Reagan-Udall Foundation for the FDA in collaboration with the US Food and Drug Administration’s (FDA’s) Oncology Center of Excellence convened a series of interviews and ...

Benefit-risk (BR) assessment, a central part of regulatory decision making, has become increasingly structured in response to the need for consistency and transparency, and in alignment with the US Food and Drug Administration’s (FDA’s) 2021 draft BR guidance. Early planning and established processes are critical to successful execution. To help regulatory affairs professionals guide clinical development teams in planning and conducting patient-focused structured BR assess...

Patient-provided information (PPI) and patient-generated health data (PGHD) are critical sources of real-world data and evidence. Collecting this data empowers patients to become partners in informed decision making by all healthcare stakeholders. However, the increased demand for this type of data has given rise to logistic, technological, and ethical challenges. Technologies and operating systems such as the high-performance integrated virtual environment (HIVE) could si...

After the US Food and Drug Administration (FDA) solicited comments on an educational program for a new Empower Rare Disease Drug Developers (LEADER 3D) initiative in the Accelerating Rare disease Cures (ARC) program, nearly two dozen stakeholders offered feedback on what topics they believe are important for inclusion in educational materials on rare disease drug development.   FDA’s Center for Drug Evaluation and Research (CDER) asked for comments from rare disease st...

There’s no one correct path into regulatory affairs.  If anything, one of the most common ways to get into regulatory affairs is “by accident.” That’s how Eleanor Panico, director of regulatory affairs at Eisai, describes the beginning of her regulatory affairs journey.  Eleanor joined us at RAPS Convergence 2022 in Phoenix for a chat about the regulatory profession, her path into it and much more. This interview has been lightly edited for clarity.  Why do you thin...

The head of the National Cancer Institute’s (NCI) investigational drug research branch said that recent draft guidance from the US Food and Administration (FDA) on finding the optimal dosage for cancer drugs could slow drug development. On the other hand, several stakeholders praised the guidance while asking for additional details such as how to factor in dosing for multi-drug treatments.   Earlier this year FDA published a draft guidance to help sponsors identify the...