ReviewEuropean journal of clinical pharmacology2025
Era of surrogate endpoints and accelerated approvals: a comprehensive review on applicability, uncertainties, and challenges from regulatory, payer, and patient perspectives.
Review in European journal of clinical pharmacology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
3 citing papers in PubMed.
- Efficacy and Safety Evidence Supporting Cancer Drug Approvals in Switzerland (2001-2020): A Meta-Analysis of Pivotal Randomised Controlled Trials.The Lancet regional health. Europe · 2026Article
- FDA first to global follow-on: alignment in expedited oncology approvals across EMA, TGA, and PMDA.Frontiers in pharmacology · 2026Article
- Innovations in Alzheimer's disease diagnostic technologies: clinical prospects of novel biomarkers, multimodal integration, and non-invasive detection.Frontiers in neurology · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeThe regulatory landscape in rare diseases and oncology has evolved to address unmet medical needs by implementing expedited approval pathways. The US FDA's Accelerated Approval and the EMA's Conditional Marketing Authorization facilitate earlier patient access to therapies through reliance on surrogate endpoints derived from early-phase clinical trials. The review aims to provide a comprehensive review of the role and utilization of surrogate endpoints in accelerated drug approvals, highlighting their strengths, limitations, and the varying perspectives of stakeholders on their validity and utility.
methodsThis article reviews existing literature and regulatory guidelines to assess the effectiveness and challenges associated with surrogate endpoints in expedited approval pathways. It also examines the post-approval commitment adherence required by regulatory bodies, exploring discrepancies among stakeholder perspectives.
resultsFindings indicate that while surrogate endpoints enable faster market access, uncertainties remain regarding post-approval commitments and their consistency. Differences in stakeholder opinions also persist, reflecting varying levels of confidence in the validity and applicability of surrogate endpoints.
conclusionSurrogate endpoints play a crucial role in accelerating drug approvals in areas with high unmet needs, yet challenges around post-approval commitments and stakeholder acceptance suggest the need for enhanced regulatory clarity and ongoing assessment of surrogate endpoint validity.
Indexed as
Identifiers
40080138What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.