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Capricord
Agencia de Evaluación de Tecnologías Sanitarias
Work Package 5 – Uncertainty‑Adapted Pricing System
Overview
Work Package 5 focuses on developing a coherent and practical system to support national authorities when assessing and pricing medicines characterised by high levels of clinical uncertainty. These products, often approved based on preliminary evidence, conditional authorisations or limited datasets, pose significant challenges for pricing, reimbursement and long term budget sustainability.
WP5 provides methodologies, tools and structured processes that help decision makers navigate uncertainty, promote evidence generation, and align pricing with the evolving value of medicines over time.
Objectives
WP5 pursues a set of specific, interlinked objectives aimed at improving decision making under uncertainty:
1. Identify and classify key domains of uncertainty
Development of a structured checklist that allows authorities to systematically identify areas where evidence is insufficient or evolving, ensuring consistent assessment across Member States.
2. Support evidence generation planning
Creation of a template for Evidence Generation Plans (EGPs) to guide what additional data are needed, how they should be collected, and how they will inform pricing and reimbursement decisions over time.
3. Provide a decision tree for uncertainty management
Design of a decision tree that helps authorities select the most appropriate pricing or reimbursement approach depending on the type and magnitude of uncertainty.
4. Develop methods for calculating a Maximum Acceptable Price (MAP)
Definition of methodological approaches to estimate the price level that reflects the medicine’s value under uncertain evidence conditions, adaptable to different national decision frameworks.
5. Design weighting methods to reflect uncertainty in pricing decisions
Development of transparent methods to account for the relative importance of different uncertainty domains, allowing pricing decisions to incorporate the quality and robustness of the evidence.
6. Integrate all tools into a full uncertainty adapted pricing system
Synthesis of all components into a coherent, practical model that can be implemented by competent authorities across the NCAPR network.
Key Activities
WP5 delivers a sequence of methodological and operational activities:
- Development and validation of an uncertainty checklist
- Drafting of the Evidence Generation Plan template
- Mapping of decision pathways and creation of the decision tree
- Comparative assessment of MAP calculation methods
- Design and testing of uncertainty weighting methodologies
- Integration of all outputs into a final uncertainty-adapted pricing system
- Consultation with expert panels, national authorities and stakeholders for refinement
Expected Outcomes
WP5 will produce a set of validated, practical tools that empower national authorities to make more transparent, consistent and evidence aligned pricing and reimbursement decisions for high uncertainty medicines.
Expected outcomes include:
- A harmonised approach to identifying and characterising uncertainty
- Clear guidance on what data are needed and how to generate them responsibly
- Structured pathways to support pricing decisions under uncertainty
- Improved predictability and consistency for national authorities and stakeholders
- A comprehensive pricing system that links price levels to evidence quality and public health value
Ultimately, WP5 supports more sustainable, accountable and patient centred approaches to financing innovative treatments across Europe.
Work Package 6 – Public Engagement and Transparency
Overview
Work Package 6 focuses on improving the capacity of national competent authorities to effectively involve patients and healthcare professionals in pricing and reimbursement (P&R) processes across EU Member States. By strengthening engagement mechanisms and promoting transparent, harmonised approaches, WP6 contributes to more inclusive, evidence based and patient centred decision making in medicine funding.
This Work Package also supports the transition towards a more needs-driven and collaborative model of pricing and reimbursement within the NCAPR network.
Objetives
WP6 is organised around three core objectives:
1. Mapping and analysing current engagement practices
The Work Package reviews existing approaches used by Member States to involve patients and clinicians in P&R procedures, compiling this information into comparable formats to support mutual learning and cross country alignment.
2. Evaluating and testing engagement methods
Through structured assessment, WP6 examines the impact of current practices on P&R processes, generating evidence on what works, for whom and under what conditions. This contributes to developing more effective and transparent models of public involvement.
3. Harmonising approaches and developing practical tools
WP6 produces common guidance documents, templates and educational materials that support countries in implementing high quality engagement processes, including:
- Guidelines for patient and clinician engagement
- Templates to support the preparation of Member State PICO for Joint Clinical Assessment (JCA)
- Educational materials and workshops for patient organisations and healthcare professionals
These deliverables aim to promote consistency, clarity and transparency across Member States.
Key Activities
WP6 includes a series of coordinated activities carried out by national authorities and expert teams:
• Systematic literature review on existing public engagement methodologies
• Cross country surveys targeting patient organisations, clinicians and competent authorities
• Development of Country Fiches summarising national approaches
• Design and delivery of training workshops in participating countries
• External validation of outputs by independent experts
• Preparation of harmonised templates and recommendations for the NCAPR network
These activities ensure that WP6 outputs are robust, relevant and adaptable to the diverse contexts of EU health systems.
Expected Outcomes
WP6 will deliver a set of practical, tested, and consensus based tools to strengthen the role of patients and healthcare professionals in pricing and reimbursement processes across Europe. Expected outcomes include:
• Improved understanding of national engagement practices
• Greater methodological consistency across Member States
• Enhanced transparency of decision making
• Stronger collaboration between competent authorities, patients and clinicians
• Recommendations and templates that can be adopted or adapted by national P&R systems
Ultimately, WP6 contributes to a more responsive, participatory and transparent European landscape for medicine pricing and reimbursement.
Work Package 7 – Strengthening National Capacities for Orphan Drug Pricing and Reimbursement
Overview
Work Package 7 focuses on supporting national competent authorities to better address the complex challenges associated with the pricing and reimbursement (P&R) of orphan medicinal products. Orphan drugs often combine high prices, limited clinical evidence, significant uncertainty, and small patient populations, placing increasing pressure on national healthcare budgets and creating barriers to equitable access.
WP7 responds to these challenges by developing practical tools, shared methodologies and collaborative approaches that reinforce sustainability, transparency, and predictability in orphan drug P&R decision-making.
Objectives
WP7 works toward three main objectives:
- Map current practices and develop an orphan drug P&R toolkit. The Work Package identifies and analyses existing national approaches, including reimbursement pathways, categorisation frameworks, effectiveness assessment models and risk sharing agreements. These insights feed into a continuously updated online toolkit designed to support competent authorities in selecting appropriate P&R strategies, comparing national approaches, and applying practical solutions adapted to orphan drugs.
- Assess the feasibility of simplified cross country real world data collection. Given the scarcity of evidence for rare diseases, WP7 investigates how Member States can share minimal, essential datasets to improve real world evidence availability while avoiding the creation of extensive or burdensome registries. The feasibility study explores data needs, interoperability considerations, bias mitigation and mechanisms for integrating multi country datasets.
- Explore opportunities for joint negotiations and procurement. WP7 analyses existing barriers and identifies realistic, low risk models for joint negotiation among Member States, particularly for orphan drugs with low volumes and limited case numbers.
The study aims to define minimal conditions for collaboration and lay the groundwork for pilot joint negotiations in the future.
Key Activities
To achieve its objectives, WP7 carries out a coordinated set of tasks:
- Systematic mapping of orphan drug P&R practices and risk sharing models
- Creation of a living P&R toolkit integrating new solutions, country examples and recommendations
- Literature reviews and surveys on data collection and joint negotiation models
- Feasibility study on simplified real world data sharing
- Feasibility study on cross country joint negotiations and procurement
- Collaborative workshops and exchanges within the NCAPR network
These activities prioritise actionable, pragmatic outputs tailored to the needs of national P&R authorities.
Expected Outcomes
WP7 will deliver tools and evidence that support more sustainable and equitable decision making for orphan drugs across Europe:
- A practical, evolving orphan drug P&R toolkit accessible to NCAPR members
- Clear guidance on minimal datasets for real world evidence sharing
- Recommendations for future joint negotiation pilots
- Strengthened negotiation power for Member States
- Greater transparency, consistency and predictability in orphan drug P&R processes
Ultimately, WP7 contributes to ensuring that patients with rare diseases gain access to effective treatments through coordinated, evidence based and financially sustainable approaches.
Deliverables
- D7.1 – Orphan P&R Toolkit (online platform)
- D7.2 – Feasibility Study on Simplified Real World Data Collection
- D7.3 – Feasibility Study on Joint Negotiations