Dear readers,
Welcome to the fourth edition of the ASCERTAIN newsletter!
In this issue, we are excited to highlight our efforts to make complex healthcare policies and pricing mechanisms more accessible. Over the past months, our consortium has continued to advance research on pricing models, reimbursement frameworks, and patient engagement —all aimed at improving equitable access to innovative health technologies.
One of our key initiatives has been the “3 Things About” video series, designed to simplify crucial topics such as market access, cost-effectiveness, and reimbursement. In six videos Carin Uyl-de Groot and Frederick Thielen explain “three things you might not know about…”.
In this edition, you will also find updates from our work packages, including progress on the ACCESS2MEDS framework, structured expert elicitation for cost-effectiveness analysis, and our efforts to refine access-based pricing models. Additionally, we showcase the vital role of patient engagement, ensuring that their perspectives remain at the core of our work.
Enjoy reading this newsletter!
Your ASCERTAIN team
MAKING HEALTHCARE COSTS UNDERSTANDABLE: TEACHING, RESEARCH, AND THE ‘3 THINGS ABOUT’ VIDEO SERIES
What is your role at Erasmus University, and how does it relate to health technology assessment?
Frederick: As an assistant professor, my work is divided between teaching and research. Much of my time is dedicated to teaching courses such as HTA, advanced health economic modelling, and pharmaceutical pricing and market access. This covers everything from the fundamentals of how medicine prices are determined to advanced modelling techniques that assess whether new treatments are worth their ever-increasing costs.
In my role as international programme director, I ensure that the curriculum of the European Health Economics and Management (Eu-HEM) Master’s programme remains relevant, up-to-date, and intellectually stimulating for students.
My research at Erasmus University focuses on advanced therapeutic medicinal products (ATMPs) and how to best assess their costs, effectiveness, and environmental impact. Did you know that we often might end up paying for expensive new medication but that the “old” cheaper treatment would have more effective for many patients?
How do you see the value of online videos for learning?
Frederick: From my teaching experience, I know that videos are a great way to facilitate learning and teaching – for both students and teachers. For students, the flexibility of on-demand content allows them to learn at their own pace, re-watch complex concepts, and even speed up playback during boring parts or those they find less challenging. And sometimes people in videos speak soooo slooow. Me included by the way.
For teachers, preparing videos can take more time initially, but it allows for better structuring of content and reduces the need to repeat the same information in every lecture. This frees up time for more interactive discussions, such as Q&A sessions, which enhance the learning experience. Ultimately, it is a win-win situation.
What was it like filming the “3 Things About” series, and what topics can viewers expect to learn about?
Frederick: Filming the series was great fun! The Erasmus Studio at Erasmus University is a fully equipped, professional recording studio, and the team working there is both highly skilled and fun to work with. Have you ever tried warming up your voice by repeatedly saying, “She sells seashells by the seashore”? It is harder than it sounds!
The videos cover key topics related to health economics, including reimbursement, cost-effectiveness, and market access. These are crucial for understanding how healthcare systems make decisions about which treatments to fund and how to allocate limited resources effectively.
Your videos focus on reimbursement, cost-effectiveness, and market access—why are these topics so important for healthcare systems?
Frederick: Healthcare budgets are limited, and new treatments are continuously being developed—often at very high costs. Decisions about reimbursement and market access determine which treatments are available to patients and at what cost to the system. If you look at life expectancy across Europe, you will still find huge differences between countries. Of course, there are many different factors influencing this and many of these factors are harder to influence than others. But how can we sit still when you can buy a happy meal in every European city, but entire European Members States do not have access to life-saving cancer treatments for children?
Cost-effectiveness analysis plays a crucial role in ensuring that healthcare resources are used efficiently. It helps policymakers and insurers assess whether a new treatment provides sufficient benefits compared to existing alternatives, considering both costs and patient outcomes. We can spend money only once. But if we do, it better be for the most effective thing. And maybe we also want to take the health of planet into account.
Without a structured approach to evaluating new treatments, healthcare systems could either overspend on high-cost interventions that provide limited benefit or fail to adopt innovative therapies that could significantly improve patient outcomes. Balancing affordability, effectiveness, and access is key to ensuring sustainable healthcare.
What impact do you hope the series will have on patients and other stakeholders?
Frederick: I hope the series helps to demystify some of the complex economic concepts and make them more accessible to a wider audience. Many people are unaware of the processes that determine whether a new treatment becomes available in their country or why certain medicines are reimbursed while others are not.
For patients, a better understanding of these topics can empower them to advocate for access to necessary treatments but also to understand why access is different between countries. For policymakers and healthcare professionals, the series provides insights into the trade-offs involved in decision-making.
Ultimately, I hope that making these concepts more transparent will contribute to more informed discussions about healthcare funding and the value of medical innovations. And if people want to know, we have a ton of learning material available through our master programmes and at the institute for medical Technology Assessment (iMTA).
CHECK OUT OUR VIDEO SERIES
We are excited to announce the release of the ‘3 Things About’ series. With these six videos we aim to make complex issues more accessible, fostering a deeper understanding of the challenges and opportunities in topics around ASCERTAIN.
In front of the camera are Carin Uyl-de Groot (project coordinator), Professor of Health Technology Assessment, and Frederick Thielen, Assistant Professor of Health Technology Assessment at the Erasmus University Rotterdam. Each of them explains three topics in the format “three things you might not know about…”.
These videos play a key role in the project’s mission to raise awareness and foster dialogue on the affordability and accessibility of health innovations. The videos are now available here on the ASCERTAIN website and social media channels.
Overview of the Videos
- 3 Things About Market Access
- 3 Things About Tiered Pricing
- 3 Things About Pricing of New Drugs
- 3 Things About Reimbursement
- 3 Things About Cost-Effectiveness
- 3 Things About Value of New Cancer Drugs
SIGNATURES OF LEGAL AGREEMENT AND RELEASE OF THE OPEN ACCESS POLICY
In November 2024, the Consortium achieved an important milestone with the signing of the Joint Controllership Agreement (JCA) between all partners, covering the entire project (D2.4). This followed the previously signed Joint Controllership Agreement among the partners involved in Task 4.1 of WP4 only. The ASCERTAIN Consortium, dedicated to developing open-access tools for healthcare pricing and value assessment, prioritizes data privacy.
The JCA defines that partners actively involved in data processing for specific project tasks act as joint controllers, sharing responsibility for handling personal data. It outlines each partner’s obligations, ensuring compliance with GDPR. The agreement aims to structure data processing responsibilities, considering the joint research purpose, data sharing, open access publication, and adherence to FAIR principles. It specifies which data is collected by whom, how it is shared, and the legal basis for processing (consent or legitimate interests). Data sharing is restricted to partners involved in the same tasks, with aggregated results shared on a common platform and in publications.
All joint controllers have committed to GDPR compliance, implementing necessary measures to protect data subject rights. The JCA also details procedures for managing data subject requests, processor usage, breach notifications, and data protection impact assessments. The final signing of the JCA successfully completed a task initiated at the beginning of 2024.
During the same period, an Open Access Policy was prepared within WP2. This policy was created to outline how the beneficiaries will make their research outputs and resources publicly available. It also establishes principles for sharing research data generated by the ASCERTAIN project, adhering to the FAIR (Findable, Accessible, Interoperable, Reusable) data principles. A dedicated webinar, open to all partners, was held in December.
ADVANCING THE ACCESS2MEDS FRAMEWORK
As part of our ongoing commitment to refining the ACCESS2MEDS Framework, we are continuously updating and improving it. These revisions enhance clarity and readability, strengthen key concepts, and ensure consistency in terminology, aligning with our glossary, which is also regularly updated.
A key focus is making the framework more accessible to all stakeholders by incorporating visual elements—such as graphs, tables, and infographics — alongside streamlined policy insights. Additionally, we are integrating model outputs within the ACCESS2MEDS Toolbox, addressing critical aspects such as pricing models, cost-effectiveness thresholds, and ‘connector’ variables to enhance usability across work packages.
These updates align with core European health values, including universality, equity, and sustainability, while reflecting ongoing developments in EU pharmaceutical legislation and policy frameworks. By reinforcing these connections and improving operationalization, the framework will become an even more robust tool for policy evaluation and decision-making in the pharmaceutical and medical device sectors.
PROGRESS IN DEVELOPING AN ACCESS-BASED PRICING MODEL FOR MEDICINAL PRODUCTS
WP4 has made significant progress in developing a new access-based pricing model for medicinal products. The ASCERTAIN pricing model is structured around the selection of relevant price determinants, i.e., individual components that—taken together—make up a price that aims to ensure access to effective new health technologies. Manufacturers, payers, and other healthcare decision-makers consider different determinants when defining (proposing, negotiating, and setting/regulating) a price for a new health technology. For any given intervention, different determinants for defining a price may become relevant, including some that are linked to the specific health technology.
Over the past six months, we conducted targeted literature reviews on individual price determinants, such as cost and value elements. These reviews have provided a solid foundation for developing a first version of the ASCERTAIN pricing model. Additionally, we continued the analysis of expert interviews that were conducted over the summer of 2024. These interviews have offered valuable insights from a range of stakeholders, including healthcare payers, healthcare professionals, patient organizations, researchers, industry, and investors.
A major milestone was achieved with the successful submission of the first deliverable of this work package. This report describes the research conducted so far and outlines the key principles for the ASCERTAIN access-based pricing model. The deliverable marks a significant step forward in our mission to develop pricing models that balance innovation with patient access.bility, we aim to ensure that health data is managed ethically and securely. This not only builds trust with patients and stakeholders but also paves the way for groundbreaking advancements in health technologies that are both accessible and affordable.
SINGLE-ARM TRIALS AND STRUCTURED EXPERT ELICITATION (SEE)
The evaluation of cost-effectiveness analyses in WP5 relies heavily on documentation based on single-arm trials. Therefore, we started working on how to document the control arm for these settings. This resulted in our first deliverable for WP5 on methods to establish a comparator in non-RCT data and on the use of expert opinion to reduce uncertainty. A report was successfully submitted in November.
The content of the report focused on the use of structured expert elicitation (SEE) as documentation in HTAs, more specifically in cost-effectiveness analysis. SEE is a valuable method to address data gaps and uncertainties, particularly in economic evaluations where traditional evidence may be limited or unavailable. In the report, we describe the SEE frameworks and tools in combination with a systematic review on applications of SEE in cost-effectiveness analyses. SEE is used as a method in use cases in WP5; therefore, a description of the method and an overview of available applications are of great value in the following work. Based on the report, we will shortly submit a scientific paper on the systematic review.
We have also started a new SEE data collection for a different indication, exploring methodological, practical, and technical features of the SEE framework. SEE interviews are being collected, and we are currently planning for a consensus meeting.
ADVANCING FLEXIBLE AND DYNAMIC REIMBURSEMENT MODELS
Work Package 6 continues to expand the scope of reimbursement frameworks, developing dynamic and cyclic reimbursement procedures that incorporate innovative cost-effectiveness threshold (CET) models to better reflect societal values and patient needs. Building on previous scoping reviews, this phase introduces flexible thresholds that adapt to disease- and patient-specific characteristics, broader societal considerations, and country-specific preferences.
A key component of this work is gathering insights from diverse stakeholders—including patients, citizens, policymakers, healthcare professionals, and insurers—through multi-stakeholder surveys and preference elicitation studies conducted across multiple European countries. These studies explore how value-for-money assessments can be refined to align with evolving health system priorities and societal expectations.
A major step in the next phase for the ASCERTAIN preference study was a series of focus groups held two weeks ago, where participants from the general population shared their views on reimbursing costly, innovative medical treatments. The discussions tackled a fundamental question: When is society willing to pay for expensive new treatments—and when not? Key themes included:
- Ethical, economic, and societal factors that should influence reimbursement decisions;
- Balancing healthcare budgets with patient access to high-cost treatments;
- Broader perspectives on whether healthcare costs should have defined limits.
Participants highlighted the complexity of these decisions, emphasizing that funding choices should not be based solely on economic calculations but must also reflect ethical values and societal priorities. Ensuring sustainability and fairness in healthcare requires continuous engagement with the public, ensuring that decision-making processes align with societal expectations and democratic principles.
Initial findings, drawn from 100 European participants in a multi-stakeholder survey, indicate strong support for expanding cost-effectiveness assessments beyond traditional economic factors. By incorporating patient experiences, societal willingness-to-pay, and ethical considerations into cost-effectiveness threshold frameworks, WP6 aims to establish context-sensitive and adaptable reimbursement strategies. The next phase will refine attribute levels using data from focus groups, literature reviews, and real-world case studies, ensuring a more representative and inclusive approach to pricing and reimbursement decisions. These efforts will contribute to enhancing the ACCESS2MEDS Toolbox, strengthening its ability to support policymakers in designing sustainable, equitable, and transparent health technology reimbursement frameworks.
HOW ASCERTAIN IS INCLUDING THE PATIENT PERSPECTIVE
Patient engagement in research projects with a health policy focus helps ensure that research outcomes align with the views and needs of patients, the ultimate beneficiaries of the tools such projects seek to develop. ASCERTAIN is committed to this principle by giving patients an active and meaningful role in the project from the very beginning.
This is achieved through several means, including the involvement of a European patient organisation, Myeloma Patients Europe (MPE), as a partner in the consortium, actively seeking patient feedback on various research tasks, and including representatives of multiple patient organizations in the project’s Advisory Board.
MPE, an organisation representing the interests of patients with a rare type of blood cancer, multiple myeloma, is involved in nearly all ASCERTAIN work packages. Their role is to promote patient understanding and gather feedback from patients and patient advocates on project results. Additionally, patients and patient advocates at national and European levels can provide their perspectives on pricing, cost-effectiveness, and reimbursement through surveys, interviews, and focus groups. They will also be invited to review ASCERTAIN’s key deliverables, including policy tools and the overarching framework.
Finally, the Advisory Board, which oversees the project’s progress throughout its duration, includes representatives from various patient organizations, such as PNH Global Alliance, Inspire2Live, and the European Sickle Cell Federation, alongside stakeholders from other relevant groups.
If you’re interested in learning more about patient engagement in ASCERTAIN or getting involved, contact us at ue.sdem2ssecca@tcatnoc.
Note: PNH refers to Paroxysmal Nocturnal Hemoglobinuria.
“3 Things About Market Access” – Transcript
“3 Things About Tiered Pricing” – Transcript
“3 Things About Pricing Of New Drugs” – Transcript
“3 Things About Reimbursement” – Transcript
“3 Things About Cost-Effectiveness” – Transcript
“3 Things About Value of New Cancer Drugs” – Transcript
EHA2025 Congress
Join partners for the 30th EHA Congress, taking place in Milan!
12.-15. June 2025
ISPOR EUROPE 2025
Join partners from the ASCERTAIN project at ISPOR in Glasgow!
09.-12. November 2025
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