Each month we scan the regulatory landscape to bring you the latest developments shaping pharmaceutical and biotechnology innovation. In this roundup you’ll find key publications that caught our attention, plus a curated summary of the most important regulatory news. Our goal is to highlight what matters, cut through the noise, and keep you up to date on changes that could impact drug development and market access in Europe and the US.
News
The FDA announced a Request for Information to identify and eliminate unnecessary regulations
The U.S. Department of Health and Human Services and the FDA have launched a public Request for Information to identify and eliminate outdated or unnecessary regulations, aligning with Executive Order 14192. Led by Secretary Robert F. Kennedy, Jr., the initiative enforces a “10-to-1” deregulatory policy: for each new regulation, at least ten existing ones must be rescinded. Additional measures include a regulatory cost cap (total regulatory costs for FY2025 must be below zero), expanded scope to include informal policies and annual transparency reports. The effort aims to reduce costs, empower providers, and refocus healthcare on patient care. A 60-day public comment period is now open.
The FDA granted breakthrough device designation for the first in vitro diagnostic device that tests blood to aid in diagnosing Alzheimer’s disease
The FDA has cleared the Lumipulse G pTau217/ß-Amyloid 1-42 Plasma Ratio, the first blood test to help diagnose Alzheimer’s disease. Intended for adults aged 55 and older showing cognitive symptoms, the test measures two proteins in blood—pTau217 and β-amyloid 1-42—to detect amyloid plaques, a key indicator of Alzheimer’s disease. Unlike previous tests requiring invasive spinal taps, this test uses a simple blood draw, offering a more accessible option. In a clinical study of 499 cognitively impaired patients, the test showed high accuracy: 91.7% of positive results matched positron-emission tomography- or cerebrospinal fluid-confirmed plaque presence, and 97.3% of negative results aligned with negative findings.
The MHRA released guidance for the use of real-world data in clinical studies to support regulatory decisions
The MHRA has released guidelines to support the use of real-world data (RWD), such as electronic health records, registries and patient-reported outcomes, in clinical research for regulatory decision-making. These guidelines aim to help sponsors design studies that use RWD effectively and ensure the data meet high-quality standards. Emphasis is placed on the quality, provenance, accuracy and relevance of data sources, including how data are captured, linked and validated. The guidelines also stress that principles of robust study design, such as randomisation and bias reduction, remain critical. Digital health technologies, such as wearables, may be used to collect RWD if appropriately validated. Detailed data quality processes must be outlined in the study protocol, and sponsors are expected to ensure data integrity throughout the study lifecycle. The MHRA may inspect systems and data handling practices as part of Good Clinical Practice inspections, particularly in high-interest or high-risk studies. Sponsors are encouraged to seek scientific advice on RWD use early in the planning process.
The MHRA released a draft guideline on the use of external control arms based on real-world data to support regulatory decisions
The MHRA has released a draft guideline on the use of real-world data (RWD) external control arms (ECAs) in clinical trials, developed with input from the Commission on Human Medicines RWD ad hoc group. RWD ECAs involve using patient-level data collected outside of a clinical study as a control group to assess the safety and efficacy of an intervention. The guideline outlines key considerations and principles for sponsors planning to use RWD ECAs in trials requiring regulatory approval. While focused on RWD, the principles also apply to external controls from other sources, such as past clinical trials. A 6-week public consultation invites stakeholder feedback on the guideline’s clarity, consistency and completeness.
The MHRA approved aumolertinib to treat non-small cell lung cancer
On 3 June 2025, the MHRA approved aumolertinib (Aumseqa) for treating adults with advanced or metastatic non-small cell lung cancer who have specific epidermal growth factor receptor (EGFR) mutations. It is approved as a first-line treatment and, in some cases, for previously treated metastatic cancer. Taken orally as a tablet, aumolertinib blocks EGFR activity to help slow or shrink tumours. In a phase 3 trial, it reduced the risk of disease progression or death by 54% compared with gefitinib, an existing treatment.
The European Society for Medical Oncology released version 2 of the magnitude of clinical benefit scale
The European Society for Medical Oncology (ESMO) released ESMO- ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) v2.0, an enhanced version of its scale for evaluating the clinical benefit of cancer therapies. This update introduces 13 critical amendments, including new criteria for single-arm de-escalation studies and toxicity annotations in curative settings. The revision incorporates extensive stakeholder feedback and improves alignment with evolving treatment landscapes. It emphasises transparency, patient-centred considerations and methodological rigor, particularly in evaluating toxicity, data maturity and statistical robustness. ESMO-MCBS v2.0 ensures more accurate, fair and clinically meaningful assessments, supporting informed treatment decisions and better patient outcomes.
The EMA published its 2024 Annual Report
The EMA’s 2024 Annual Report highlights its strategic contributions to public and animal health in the European Union (EU). In 2024, the EMA recommended 114 human medicines for marketing authorisation, including 46 with new active substances. Several of these address pressing public health needs or showcase notable innovation. The digital report features an interactive timeline of milestones and offers deeper insights via supporting materials. Key initiatives in 2024 included ACT EU (Accelerating Clinical Trials in the EU) and DARWIN EU (Data Analysis and Real World Interrogation Network), aimed at improving clinical trial processes and real-world data use. The EMA also advanced the adoption of Artificial Intelligence and innovative manufacturing methods, while reinforcing collaboration across EU and global regulatory networks. The second chapter presents statistics and trends on regulatory activities, with further details available in the EMA’s annual activity report.
Publications that caught our eye...
The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK. Nature Medicine
Insight from the ESMO targeted anticancer therapies congress 2025. Nature Medicine
Optimizing Patient Registries for Regulatory Decision Making - Key Learnings From an HMA/EMA Multistakeholder Workshop. Clinical Pharmacology & Therapeutics.
JAMA Oncology – The Year in Review, 2024. JAMA Oncology
Sotorasib’s Accelerated Approval. Wrong Dose and Indication. JAMA Oncology
New drugs approved by the European Medicines Agency during 2024. European Journal of Medicinal Chemistry
Expedited Approval in Oncology: A Study of European Regulators’ Perspectives and Trade-offs. Clinical Pharmacology & Therapeutics
