The CTR Classification Guide is a free European online clinical trial decision tool developed by Paul Janssen Futurelab Leiden and the Dutch Central Committee on Research Involving Human Subjects (CCMO). It helps investigators determine whether a planned study with medicinal products falls within the scope of the EU Clinical Trials Regulation (CTR 536/2014), whether it qualifies as a low‑intervention clinical trial, or whether it lies outside the CTR framework. Through a structured series of questions, the tool clarifies whether a study is considered a clinical trial of medicinal products in the European Union/European Economic Area and indicates when adapted rules for low‑intervention trials may apply. Investigators can use the guide early in study planning to identify the applicable regulatory pathway and to prepare appropriate submissions to ethics committees and competent authorities.

The resource is a 14‑page recommendations document issued in April 2017 by the European Commission expert group on clinical trials to support implementation of Regulation (EU) No 536/2014 on clinical trials on medicinal products for human use. It sets out how to apply risk‑proportionate approaches to the design, conduct and oversight of clinical trials, so that trial requirements are adapted to the actual risks to participants, trial integrity and data reliability, rather than following a “one size fits all” model. The guidance explains risk‑based quality management principles and describes how risk assessment and mitigation can inform monitoring, safety reporting, investigational medicinal product handling and trial documentation, including the Trial Master File.

The European Medicines Agency maintains an online list of national competent authorities responsible for human medicines in each European Union and European Economic Area Member State. The national competent authorities are primarily responsible for the authorisation of medicines available in the EU that do not fall under the scope of the centralised procedure. This directory is part of the European medicines regulatory network information and is regularly updated. Investigators and sponsors planning clinical trials or other regulatory activities in one or more European countries can use it to quickly identify and contact the relevant national competent authority for clinical trial authorisations and other regulatory queries.
 

EudraLex Volume 4 contains the EU Guidelines to Good Manufacturing Practice (GMP) for medicinal products for human and veterinary use, setting out the detailed GMP principles manufacturers must meet in manufacture and control. It includes Annex 13 on investigational medicinal products (IMPs), which covers manufacture, packaging, labelling and quality control of IMPs used in clinical trials, and forms a key regulatory reference for sponsors and investigators. The European Medicines Agency coordinates GMP inspections and related activities at European level, in line with these guidelines. Until 15 July 2026 the guidelines apply to both human and veterinary medicinal products; from 16 July 2026 specific implementing regulations will apply for veterinary medicinal products and their active substances, while remaining aligned with the GMP framework for human use.

Developed by the European Clinical Research Infrastructure Network (ECRIN) within the ERA4Health Partnership, the document shares pratical guidance on how to prepare data sharing plans for clinical studies, and how to design General Data Protection Regulation (GDPR)‑compliant strategies for sharing Individual Patient Data (IPD), including informed consent for secondary use and long‑term storage in repositories that follow FAIR (Findable, Accessible, Interoperable and Reusable) principles. It aligns data sharing planning with expectations from European and international funders and is aimed at investigators, funders, research staff and other stakeholders involved in investigator‑initiated clinical trials.

The ICH E9 (R1) addendum “Estimands and Sensitivity Analysis in Clinical Trials” (EMA/CHMP/ICH/436221/2017) was adopted by the ICH Assembly at Step 4 on 20 November 2019 and came into effect in the European Union on 30 July 2020. It introduces a structured framework to align clinical trial objectives, design, conduct, analysis and interpretation through the explicit definition of estimands: precise descriptions of the treatment effect that reflect how intercurrent events such as treatment discontinuation, rescue medication or death are handled. The addendum describes five main strategies for addressing intercurrent events (treatment policy, hypothetical, composite, while‑on‑treatment and principal stratum) and explains how these choices influence trial design, data collection and analysis. It clarifies the role of sensitivity analysis, which must target the same estimand as the main estimator and is used to assess the robustness of conclusions to deviations from modelling assumptions and data limitations. The principle is that trial protocols should pre‑specify primary and key secondary estimands, aligned estimators and planned sensitivity analyses so that regulators, sponsors and investigators have a shared understanding of what treatment effect is being estimated and how reliable the resulting evidence is for decision making.

This free, self‑paced online course provides a general introduction to adverse events in clinical research and how to manage them when they occur. It explains why adverse events should be collected, how they are defined and categorised, and what information needs to be documented and reported. The course outlines mechanisms for detecting adverse events, evaluating their seriousness and relatedness to the study intervention, and planning appropriate follow‑up. It also summarises what data are typically included in adverse event reports and who should receive them. The training is suitable for all members of a clinical research team, including those new to safety reporting in investigator‑initiated studies.

The ECRIN Paediatric Tools – Outcomes provide practical guidance for planning efficacy and safety outcome assessment in paediatric and neonatal clinical trials. The collection supports protocol development by offering recommendations and examples for selecting short- and long-term outcomes, defining data items and planning outcome measurement throughout the study. These tools help researchers develop clinically meaningful, feasible and standardised outcome assessment strategies that are adapted to paediatric populations and particularly relevant for rare disease clinical trials.

This reflection paper from EMA adopted by GCP Inspectors Working Group in February 2012, provides guidance for laboratories that perform analyse or evaluate human samples collected in clinical trials, to help them establish and maintain quality systems that comply with European directives, national regulations and associated guidance. It also sets out what good clinical practice inspectors expect when they inspect laboratories performing work in support of human clinical trials, including investigator‑initiated studies.

Guidance from the European Commission on preparing summaries of clinical trial results for laypersons, in line with Clinical Trials Regulation (EU) No. 536/2014. Good Lay Summary Practice (GLSP) provides recommendations on planning, writing, translating and disseminating plain‑language summaries of clinical trial results, covering all ten mandatory lay summary content elements specified in Annex V of the Regulation. It aims to support transparent, understandable communication of trial outcomes to participants and the public. GLSP is mandatory guidance for interventional clinical trials with medicinal products conducted in the EU/EEA and published in EudraLex Volume 10.

Cochrane PICO search is a powerful discovery tool for finding Cochrane Reviews. Clinical trials start with a clearly defined research question. The PICO framework, which underpins the PICO search tool, is widely used to structure clinical research questions. PICO search allows researchers to use these PICO terms to find the Cochrane Reviews most relevant to their healthcare question. 

Guideline from the European Medicines Agency on the use of computerised systems and electronic data in clinical trials (EMA/INS/GCP/112288/2023). It sets principles and requirements for instruments, software and “as a service” solutions used to create, capture, process, store and archive electronic clinical data across the full data life cycle. Topics include data integrity and ALCOA++ principles, roles and responsibilities, system validation, user management, security, audit trails, electronic signatures, data protection, cloud solutions and database decommissioning. It applies to systems such as electronic medical records, electronic case report forms, electronic clinical outcome assessment and patient‑reported outcome tools, wearables, interactive response technologies, electronic informed consent, electronic trial master files, clinical trial management systems, pharmacovigilance databases and artificial intelligence‑based tools, and replaces the 2010 Reflection Paper on electronic source data.

This EMA guideline provides specific pharmacovigilance considerations for monitoring and evaluating the safety of medicinal products used in paediatric populations. It addresses age-specific aspects of adverse event monitoring, safety reporting, risk management and benefit–risk assessment throughout clinical development and post-authorisation use. Although focused on paediatric medicines, the guidance is highly relevant to rare disease clinical trials, where many conditions have childhood onset and where safety assessment can be challenging due to small populations, limited evidence and long-term treatment needs.

The ICH E9 guideline “Statistical Principles for Clinical Trials” is a harmonised tripartite guideline, adopted at Step 4 on 5 February 1998, for marketing applications in Europe, Japan and the United States. It gives direction to sponsors on the design, conduct, analysis and evaluation of clinical trials of an investigational product within its overall clinical development, and supports experts preparing application summaries or assessing evidence of efficacy and safety, mainly from later‑phase confirmatory trials. It sets out core statistical principles for minimising bias and maximising precision, without prescribing specific methods, and assumes that an appropriately qualified statistician is responsible for implementing these principles.
 

The NCCIH Clinical Research Toolbox is a web‑based information repository for investigators and staff involved in NCCIH‑funded clinical research. Developed by the National Center for Complementary and Integrative Health (US NIH), it brings together required templates, sample forms, FAQs, policies and guidance to support the development and conduct of high‑quality clinical research studies. Content is organised into sections such as clinical research start‑up, documents used during study conduct, on‑site monitoring, milestone documents and additional training and recruitment resources, making it a practical reference point for investigator‑initiated trials. Although some elements are specific to the NCCIH research, many sections on trial planning and development are also relevant to European researchers conducting investigator‑initiated studies.

This guidance provides practical recommendations for incorporating pharmacovigilance and risk management into neonatal clinical trial protocols. It supports protocol development by outlining key considerations for planning safety monitoring, adverse event collection and reporting, risk identification, and safety oversight in neonatal studies. The tool helps researchers address the specific safety challenges of neonatal clinical trials and develop protocols that support effective pharmacovigilance while meeting regulatory and ethical expectations.

The Regulatory and Ethical Database (RED) is a regularly updated, online search tool that provides country‑specific information on regulatory and ethical requirements for clinical research across Europe. Developed by the European Clinical Research Infrastructure Network (ECRIN), it offers structured, up‑to‑date content on national legislation, competent authorities, ethics committees and study‑specific provisions for medicinal products, medical devices and other clinical studies. RED is designed to support investigators and sponsors in planning, initiating and conducting mono‑ and multinational clinical studies by providing simple, centralised access to key submission and compliance information by country and study type. It also provides regulatory and ethical information for specific population groups, such as minors, incapacitated persons, emergency situations and pregnant or breastfeeding women. For researchers looking to expand a study to another country, a “Compare” feature enables side‑by‑side review of legislation and other requirements for clinical research in two selected countries.

TranspariMED’s Clinical trial transparency tools page collates practical tools, manuals, workshop materials and case studies that universities and other institutions can use to strengthen clinical trial registration and results reporting, improve registry data quality, and embed transparency within institutional policies and audit practices.

Downloadable guidance document published by the World Health Organization (WHO) that sets out best practices for the design, conduct, registration, oversight and reporting of clinical trials globally. It responds to the World Health Assembly resolution WHA75.8 on strengthening clinical trials, updating earlier WHO work on research capacity for the context of well‑designed and well‑implemented trials. The guidance aims to enhance clinical research efficiency, minimise research waste and support sustainable clinical trial capacity that can function in routine settings and pivot during health emergencies. 

COBWEB (CONSORT‑based WEB tool) is a user friendly online manuscript‑writing aid that guides authors through drafting randomised controlled trial reports in line with the Consolidated Standards of Reporting Trials (CONSORT) and its extensions. Developed by clinical epidemiologists, it helps users write, edit, share and export randomised trial manuscripts structured around CONSORT checklist items. The tool provides tailored templates for different trial designs and intervention types, with bullet‑point prompts and examples of good reporting for each item, consolidating relevant CONSORT guidance into a single, editable document.

ICH E6 (R3) is the current International Council for Harmonisation guideline on Good Clinical Practice, adopted by the European Medicines Agency as a scientific guideline with effect from 23 July 2025. It sets international standards for the design, conduct, recording and reporting of clinical trials involving human subjects, to support the mutual acceptance of clinical trial results by regulatory authorities across ICH member regions It outlines the responsibilities of sponsors, investigators, and other stakeholders to protect the rights, safety, and well-being of trial participants, while ensuring the integrity and credibility of clinical trial data. The guideline is structured as:

• Overarching principles and objectives document;
• Annex 1 focused on interventional clinical trials;
• Annex 2 to address additional Good Clinical Practice considerations for certain non‑traditional interventional trials (pragmatic clinical trials, decentralised clinical trials, trials that incorporate real-world data sources). 

Foundational ICH harmonised guideline on clinical safety data management, adopted at Step 4 on 27 October 1994 and intended for use alongside other Good Clinical Practice guidelines. It defines key terms such as adverse event, adverse drug reaction, serious adverse event and unexpected adverse reaction, and clarifies the distinction between serious and severe events. The guideline sets internationally agreed standards for expedited reporting of serious and unexpected adverse drug reactions in the pre‑approval phase, including reporting time frames and minimum criteria for case reporting. It is an essential reference for clinical trial sponsors and investigators developing safety reporting procedures and regulatory submissions in line with ICH E2A requirements.

This document is a mapping and forward-looking overview of all scientific/regulatory advice mechanisms in the EU medicines system, with a strong focus on coordination, early engagement, and support for better clinical development. The document is essentially a framework overview. It explains what advice exists, how to access it and how the system is evolving.

This MedEthicsEU overview, written and endorsed by a group of national representatives of Medical Research Ethics Committees in EU/EEA member states, summarises Part II clinical trial application requirements per country under the Clinical Trials Regulation (EU) No 536/2014. Version 3.0 (14 March 2025) covers 21 member states and supersedes version 2.0. It is a practical European reference for sponsors compiling country‑specific Part II documentation for multi‑country clinical trial applications in CTIS. For each country it lists, for every Clinical Trials Information System Part II placeholder, which documents are required, whether European Commission or national templates are mandatory or recommended, the legal basis (Regulation, national law, guidelines or other) and links to ethics committee or authority websites. The document is described as a living, non‑binding tool to facilitate submission of clinical trial applications and should be read together with the Clinical Trials Regulation and EudraLex Volume 10 templates.

An online research data management guide maintained by University College London (UCL) Library Services, covering how to manage the outputs of research projects across the full data lifecycle. It supports researchers from planning to project closure, including writing a data management plan, organising and storing data securely during a study, and choosing where and how to share or preserve data at the end. The guide addresses key issues such as data protection, copyright, long‑term preservation, and compliance with institutional and funder expectations. Relevant for clinical investigators who need to handle research data systematically, protect participants’ information, and meet open research and data sharing requirements.

The CONSORT-C Extension provides recommendations to improve the reporting and dissemination of randomised clinical trials involving children and adolescents. It complements the general CONSORT Statement by addressing paediatric-specific considerations and supporting transparent reporting of trial design, interventions, outcomes and results. The tool is particularly relevant for paediatric rare disease trials, where small populations and limited evidence require high-quality reporting to maximise the value and usability of clinical research findings.

The Easy Guide to Clinical Studies (Easy GCS) is an online tool developed by the Swiss Clinical Trial Organisation (SCTO) Project Management Platform, with input from all SCTO platforms and the Swiss Biobanking Platform. It provides easily accessible, phase‑specific guidance for planning and conducting clinical studies, organised in a grid that combines six study phases with key subjects such as protocol, ethics and laws, study management, safety, data management, statistics, quality and risk, monitoring, and biobanking. Each cell links to topic pages that answer three practical questions: what the topic is and why it matters, what needs to be done, and where to get help. The tool is aimed at sponsor‑investigators, site investigators, research coordinators and other clinical research professionals, and supports investigator‑initiated studies in Switzerland and beyond by clarifying regulatory requirements and operational good practice from study concept through to completion

This EudraLex Volume 10 page, published by the European Commission, provides the official clinical trial application templates for preparation of Part I and Part II documentation for the Clinical Trials Information System (CTIS) under Regulation (EU) No 536/2014 in European Union and European Economic Area member states. It includes document templates for the CTIS form section (statement on compliance with Regulation (EU) 2016/679 on data protection) and Part II application documents, such as compensation for trial participants, investigator curriculum vitae, declaration of interest, site suitability form, informed consent and patient recruitment procedure, and compliance with rules for biological samples. Templates are available in portable 
document format (PDF) and Word format for direct use or adaptation by sponsors.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) can issue qualification opinions or qualification advice on novel methodologies used in medicine development, such as biomarkers, imaging methods and digital tools, within a defined context of use in non‑clinical or clinical studies. 

A web‑based research data management toolkit for life sciences, developed and maintained by the ELIXIR network. It guides researchers and data stewards in managing research data across the full data lifecycle in line with FAIR (Findable, Accessible, Interoperable, Reusable) principles. Content is community‑driven, with contributors from many European countries, and focuses on practical, domain‑specific and role‑based guidance. Particularly relevant for biological and health research, including investigator‑initiated clinical studies that need to ensure that data are well documented, reusable and compliant with funder expectations.

Clinical Trials Regulation (EU) No 536/2014 in practice” is a quick guide produced under EudraLex Volume 10 to help sponsors to apply the Clinical Trials Regulation (EU) No 536/2014 in the European Union and European Economic Area. Prepared by the Clinical Trials Coordination and Advisory Group (CTAG), whose members are the National Contact Points named in the Regulation, it provides a practical overview of the main rules and procedures, to be read alongside the legislative text. The guide outlines key principles of the Clinical Trials Regulation, the transition period and step‑by‑step processes before, during and after a clinical trial, including use of the Clinical Trials Information System (CTIS), safety reporting, transparency, and specific requirements for advanced therapy and genetically modified investigational medicinal products. It is intended to facilitate day‑to‑day implementation of the Regulation for national and multinational clinical trials.

The Protocol Development Toolkit, part of the EDCTP Knowledge Hub, is an online resource developed by the European & Developing Countries Clinical Trials Partnership (EDCTP) and The Global Health Network to support high‑quality health research protocols, particularly for clinical studies in low‑resource settings. It combines step‑by‑step guidance on protocol development with examples, templates and interactive tools, including the Concept Protocol Crowd Review Tool, the SEPTRE electronic protocol builder and the Study Walk‑through toolkit. Linked training materials cover scientific, ethical, regulatory, operational and data‑management aspects of study design, helping investigator‑initiated studies move from a research question to a complete, robust protocol.

The CONSORT-Outcomes 2022 extension provides harmonised, evidence‑ and consensus‑based guidance for reporting outcomes in randomised clinical trial reports, adding 17 outcome‑specific items to the CONSORT 2010 (and updated CONSORT 2025) checklist to ensure that outcomes are fully defined, justified, assessed, analysed and reported in ways that enhance trial utility, replicability and transparency, and limit selective non‑reporting of trial results.

Hosted by Trial Forge, this online library provides real‑world Participant Information Leaflets (PILs) and Informed Consent Forms (ICFs) used in randomised trials, assembled through the EXCELSIOR project. The collection allows researchers to see how key trial concepts such as randomisation, placebo use and withdrawal rights are actually explained to potential participants in practice. Investigators can review and compare wording across trials and use these examples to inform the design of their own participant information leaflets and consent forms for future studies.

This resource provides an overview of data-sharing repositories available for paediatric clinical research and highlights opportunities and challenges related to individual patient data (IPD) sharing and reuse. It supports investigators in planning data management strategies, identifying suitable repositories and considering data accessibility and interoperability. The resource is particularly relevant for paediatric rare disease trials, where limited patient numbers and multinational collaboration make responsible data sharing and reuse essential to maximise the value of clinical research data.

A harmonised European template for agreements between trial sponsors and clinical sites that facilitates the setup of multi-country clinical trials. It standardizes key contractual provisions, reduces negotiation time and administrative burden, supports compliance with ICH Good Clinical Practice, and promotes efficient collaboration across participating sites. It is particularly useful during the trial initiation and site activation stages of clinical trial management. Developed by ECRIN within the ERA4Health project, its primary objective is to streamline and standardize contractual negotiations between trial sponsors and participating clinical sites while accommodating national legal requirements.

CONSORT 2025 is the updated Consolidated Standards of Reporting Trials statement, providing a 30‑item checklist and participant flow diagram that set out the minimum information required when reporting randomised trials. It supersedes earlier CONSORT versions (1996, 2001, 2010) and reflects new methodological evidence, open science practices and user feedback. Developed through a scoping review, international Delphi survey (317 participants in round 1) and expert consensus meeting, it aims to improve clarity, completeness and transparency of trial reports. The guideline is endorsed by major journals and editorial organisations and is associated with more complete reporting in journals that require its use. Authors, editors, reviewers and other users are encouraged to use CONSORT 2025 alongside the detailed explanation and elaboration document and expanded checklist when preparing or appraising reports of randomised trials.

An online Data Sharing Toolkit, developed within the EDCTP Knowledge Hub and hosted by The Global Health Network, that collates practical information and resources on sharing clinical and health research data. It brings together guidance on data management basics, step‑by‑step data sharing workflows and a repository finder tool to support researchers in preparing, documenting and depositing datasets in appropriate repositories. 

The EURORDIS Community Advisory Board (CAB) Programme provides a structured framework for involving patient representatives and expert patients in clinical research and development. It supports protocol development by facilitating dialogue between patient communities and research sponsors on topics such as unmet needs, trial design, feasibility, outcome selection, recruitment strategies and participant burden. The programme helps researchers integrate patient perspectives early in the clinical trial planning process, supporting the development of more relevant, acceptable and patient-centred rare disease clinical trials.

Regulation (EU) No 536/2014 is the core European legislative act governing clinical trials on medicinal products for human use. It repeals Directive 2001/20/EC and sets directly applicable, uniform rules for the authorisation, conduct and supervision of clinical trials in all Member States, with specific provisions on low‑intervention clinical trials. It is the primary legal reference for sponsors and investigators planning or conducting clinical trials with medicinal products in Europe.The Regulation aims to ensure that the rights, safety, dignity and well‑being of trial subjects are protected and that data generated in clinical trials are reliable and robust, while enabling efficient authorisation procedures, including for multinational trials.

The Patient Engagement Toolbox, developed under the IMI‑PARADIGM (Innovative Medicines Initiative – Patients Active in Research and Dialogues for an Improved Generation of Medicines) project, centralises co‑created recommendations, practical tools and background materials to make patient engagement in medicines development easier, more consistent and more impactful. The toolbox is organised around three key phases of patient engagement: planning, conduct, reporting and evaluation. 

World Health Organization policy and implementation guidance on the sharing and reuse of health‑related data for research purposes. It clarifies how health data collected under the auspices of WHO technical programmes may be reused and onward shared for research, in both emergency and non‑emergency situations. The policy covers research data generated directly by WHO, research funded by WHO and the reuse of other health‑related data for research when WHO is involved. It sets objectives and principles for data sharing that are equitable, ethical, efficient and consistent with FAIR (Findable, Accessible, Interoperable, Reusable) data practices, and is accompanied by implementation guidance to support the development of data management and data sharing plans for each dataset for which WHO is responsible.

ECRIN’s Data Centre Certification Programme identifies non‑commercial clinical trials units that can demonstrate safe, secure, compliant and efficient management of clinical research data. The programme is built on a published set of data centre standards that cover information technology infrastructure, data management processes, treatment allocation and statistical programming. These standards are used both for independent on‑site audits and as a practical reference for good clinical data management practice.

This guide supports the meaningful involvement of patients, caregivers and patient organisations during protocol development. It provides practical recommendations for establishing effective patient partnerships, including identifying appropriate partners, defining roles and expectations, planning communication, and recognising contributions. The guide also highlights how patient input can improve the relevance, feasibility and participant-centredness of a clinical trial by informing the research question, study procedures, outcome selection, recruitment strategies and participant-facing materials. It is a useful resource for researchers seeking to embed patient perspectives from the earliest stages of trial design.
 

This publication provides practical recommendations for incorporating health-related quality of life (HRQoL) and patient-reported outcomes (PROs) into rare disease research. It supports protocol development by discussing the selection of appropriate outcome measures, instrument validity, patient relevance, data collection strategies, and interpretation of HRQoL data in small and heterogeneous patient populations. The resource helps researchers identify meaningful patient-centred endpoints and select fit-for-purpose PRO instruments that strengthen the clinical relevance and quality of rare disease clinical trials.

Endorsed by the EU Clinical Trials Coordination and Advisory Group (CTAG), this European Commission template supports sponsors and investigators in describing recruitment arrangements and informed consent procedures for clinical trials under Regulation (EU) No 536/2014. It is not mandatory, but if it is not used, all items it covers must still be addressed in the protocol as a minimum. The template guides users through how potential participants are identified and approached, how consent is obtained and documented, and how special situations such as incapacitated adults, minors, emergency research and cluster trials are handled. It is most useful for EU/EEA trials needing a structured, regulator‑aligned description of recruitment and informed consent arrangements, including investigator‑initiated CTIMPs.

This review provides guidance on the use of patient-reported outcome measures (PROMs) in rare disease research and clinical trials. It supports protocol development by addressing the selection, development and evaluation of PROMs, including considerations of validity, reliability, responsiveness, feasibility and relevance to patients. The resource helps researchers identify meaningful patient-centred endpoints and incorporate appropriate outcome measures into rare disease clinical trial protocols, particularly when disease heterogeneity and small populations create challenges for outcome assessment.

The Adaptive Platform Trial Toolbox is an online resource developed by ECRIN, European Clinical Research Infrastructure Network, and partners within EU‑funded projects such as RECOVER and EU‑RESPONSE. It collates accumulated knowledge, experience and resources from multiple adaptive platform trials into a practical, guided toolbox to support the planning and conduct of future adaptive platform trials in any therapeutic area. More than 130 tools are organised into categories and subcategories, each with a short description of format and level of validation, helping researchers and sponsors quickly find relevant guidance, templates and examples for complex platform trial designs.

This guideline provides a harmonised framework for using extrapolation to support paediatric drug development when existing evidence from adults or other paediatric populations can be scientifically justified. During protocol development, it helps researchers determine the extent of new clinical data required, identify evidence gaps, and plan efficient study designs using approaches such as modelling and simulation, pharmacokinetic/pharmacodynamic data, and targeted evidence generation. The guideline is particularly relevant for rare diseases, where limited patient populations require innovative and scientifically robust strategies to minimise unnecessary studies while ensuring reliable evidence of safety and efficacy.

The ERICA PROMs Repository provides a centralised resource to identify patient-reported outcome measures (PROMs), observer-reported outcome measures (ObsROs) and other patient-centred clinical outcome assessment tools relevant to rare diseases. It supports protocol development by helping researchers select appropriate outcome measures based on disease area, patient population, age group and assessment type. The repository facilitates the integration of meaningful patient-centred endpoints into rare disease clinical trials and promotes the use of standardised, fit-for-purpose outcome measurement instruments.