Short description:
This thesis focuses on the central role of healthcare outcomes in optimizing the quality of cancer care. The overall aim is to advance both the understanding and the practical application of outcome-based quality assessment in oncology. Part I explores how outcome information can be used for valid and reliable hospital comparisons, thereby stimulating continuous quality improvement, while Part II examines how outcome predictions can support personalised care and improve SDM. Together, these approaches are essential for achieving outcome-driven, value-based cancer care.
In Chapter 2, we review the current landscape of cancer outcome benchmarking in Europe, focusing on quality indicators and the methodology of case-mix adjustment models. In Chapter 3, we develop a case-mix adjustment model for an important outcome indicator in the NBCA: complications after surgery. Since case-mix adjustment alone does not ensure the validity and reliability of an indicator, we introduce a structured framework to evaluate quality indicators on feasibility, discriminative ability, validity, and reliability (Chapter 4), and apply it to both breast cancer (a high-incidence cancer) and oral cavity cancer (a low-incidence cancer; Chapter 5).
In Chapter 6, we externally validate the updated PREDICT tool (version 3.1) for supporting clinical decision in Dutch and Swedish breast cancer patients, with a focus on lobular breast cancer and younger patients, two groups for whom outcomes are harder to pre ict and more uncertain. In Chapter 7, we compare version 3.1 of the PREDICT tool with the previous version 2.2, using data from the Dutch population and 36 clinically relevant subgroups. Finally, in Chapter 8, we develop models to predict trends of health-related quality of life (HRQoL) after breast cancer surgery and reconstruction, using 15 different HRQoL outcomes, aiming to better support patients and clinicians in managing expectations during SDM
English below. The defense will be in Dutch.
Door maatschappelijke veranderingen en druk op het zorgstelsel verschuift de gezondheidszorg van een paternalistisch naar een participatief model, waarin patiënten en zorgprofessionals samen verantwoordelijkheid dragen voor gezondheid en zorg. Een kernbegrip binnen deze verschuiving is zelfmanagement: de manieren waarop mensen omgaan met de fysieke, psychosociale en existentiële gevolgen van ziekte en behandeling.
Aan de hand van een combinatie van kwantitatieve en kwalitatieve onderzoeksmethoden onderzoekt dit proefschrift, vanuit de perspectieven van patiënten en zorgprofessionals, wat zelfmanagement en zorg in de laatste levensfase betekenen voor (1) mensen met vergevorderde ziekte (kanker), en (2) mensen in een situatie van dak of thuisloosheid.
De resultaten tonen dat zelfmanagement in de laatste levensfase sterk persoonsgebonden is en als uitdagend wordt ervaren. Zorgprofessionals vervullen hierbij uiteenlopende ondersteuningsrollen, variërend van instructief tot adviserend en samenwerkend. Voor mensen die dak of thuisloos zijn worden zelfmanagement en palliatieve zorg bemoeilijkt door sociale en structurele kwetsbaarheden, zoals multimorbiditeit, complexe leefomstandigheden, onverzekerdheid en frequente overplaatsingen tussen zorgvoorzieningen.
Het proefschrift concludeert ook dat zelfmanagement in de laatste levensfase een heterogeen begrip is, dat ruimte laat voor uiteenlopende discoursen (sociale rechtvaardigheid, klinische dominantie en individuele verantwoordelijkheid). Deze conceptuele variatie gaat samen met oversimplificatie, miscommunicatie en inconsistente toepassingen. Vier leidende principes kunnen bijdragen aan meer eenduidigheid: zelfmanagement (1) reikt verder dan de medische zorg alleen, (2) omvat ook andere betrokkenen dan de patiënt, (3) is zowel persoonsgebonden als contextafhankelijk, en (4) is onvermijdelijk. Daarnaast laat het proefschrift zien dat er sprake is van gebrekkige afstemming tussen wat mensen zelf doen en wat zorgprofessionals aanbieden. Kijkend naar de onderliggende oorzaken suggereren we oplossingen op meerdere niveaus (individueel en relationeel, zorgsysteem, maatschappelijk).
Due to societal changes and increasing pressure on the healthcare system, healthcare is shifting from a paternalistic to a participatory model, in which patients and healthcare professionals share responsibility for health and healthcare. A key concept within this shift is self‑management: the ways in which people deal with the physical, psychosocial and existential consequences of illness and its treatment.
Using a combination of quantitative and qualitative research methods, this thesis examines— from the perspectives of patients and healthcare professionals—what self‑management and care in the final phase of life mean for (1) people with advanced illness (cancer), and (2) people experiencing homelessness.
The findings show that self‑management in the final phase of life is highly personal and is experienced as challenging. Healthcare professionals fulfil a range of support roles, varying from instructive to advisory and collaborative. For people experiencing homelessness, self‑management and palliative care are further complicated by social and structural vulnerabilities, such as multimorbidity, complex living conditions, lack of health insurance, exclusion from regular care, and frequent transfers between care settings.
The thesis further concludes that self‑management in the final phase of life is a heterogeneous concept, allowing for different discourses (social justice, clinical dominance and individual responsibility). This conceptual variation is associated with oversimplification, miscommunication and inconsistent application. Four guiding principles are proposed to promote clarity: self‑management (1) extends beyond medical care, (2) involves others besides the patient, (3) is both person‑specific and context‑dependent, and (4) is inevitable. In addition, the thesis identifies inadequate alignment between what people do themselves and what healthcare professionals offer. By examining the underlying causes, solutions are suggested at multiple levels (individual and relational, healthcare system, and societal).
Short description
Erasmus MC set its strategic goals for a newly built hospital in 2000. It wanted to create an innovative care environment to cater for its tertiary care patients, often requiring multidisciplinary care, that would combine ‘high tech’ with ‘high touch’. For the built environment itself the ambition was to create a safe, pleasant, and sustainable spaces. Between ambitions and realisation, many stakeholders influence design and decision-making processes. This study reconstructs the engagement of stakeholders in the transformative change process for Erasmus MC’s newly built hospital, and it tries to understand the trade-offs based on stakeholder voices during this process as experienced by end-users, e.g. after the building has been taken into use in 2013 and 2018. Design quality as experiences by end-users is influenced by the balance between bricks, bytes, and behaviour, or by the fit of the human-building-technology interactions. And, collecting lessons learned through evaluation research helps shape better (hospital) buildings.
Gastric cancer is highly fatal, but can be prevented. The challenge is to find the strategies that work best, for whom and at what cost. This thesis investigates which prevention strategies save most lives, while balancing inherent harms related to screening.
The research focuses on two main approaches: screen-and-treat of Helicobacter pylori, the bacterium causing most gastric cancers, and endoscopic screening to catch cancer early. By combining data analysis, ethical reflections and microsimulation modelling, the work evaluates the benefits, harms and trade-offs of these strategies across populations in Europe, Asia and the United States.
A highlight of this thesis is the development of MISCAN-gastric, a new disease model in the MISCAN family, that is readily available for health policy analysis. The research also explores broader challenges, including antibiotic resistance, surveillance of precancerous lesions and the ethics of screening people based on race or ethnicity.
Most lung cancer cases are detected only after the disease has spread to other organs. At this advanced stage, survival remains low—28% after one year—even with new treatments. Earlier detection through CT screening has been shown in large trials to find cancers sooner and reduce lung‑cancer mortality by 24%. However, it was still unclear how these trial results would translate into population‑level costs and health benefits.
The thesis uses microsimulation modelling to project real‑world outcomes at the national level. It estimates the costs of a large‑scale screening program and the number of life‑years that could be gained. The study addresses key questions raised by the Health Council in 2025, such as which risk metrics should determine eligibility,, the optimal screening ages, and whether annual or biennial screening is most effective.
For the Netherlands, screening high‑risk individuals aged 55–75 could prevent around 1,000 deaths per year—about 10% of current lung‑cancer mortality—at an estimated €12,000 per life‑year gained, within acceptable national cost‑effectiveness thresholds. Personalized risk scores could further improve efficiency by adjusting the age of first screening based on individual risk levels. Moreover, the thesis shows that the onset of expensive treatments for late-stage lung cancer have made early detection increasingly cost-efficient.
Short discription of the thesis
The studies underlying this dissertation explores the ethical and psychosocial issues involved in the development and implementation of the bio-artificial pancreas (BAP) for people with type 1 diabetes. The BAP aims to stabilize blood glucose levels and eliminate the need for insulin administration trough transplantation without toxic immunosuppressive medication. However, initiating early-phase clinical trials raises ethical concerns due to the invasive and risky nature of the procedure, uncertain benefits, and existing alternative treatment options. This dissertation addresses four central questions: (1) which ethical considerations are essential for conducting responsible research, (2) which are relevant for responsible implementation, (3) what is the social value of the BAP compared with current therapies for people with type 1 diabetes, and (4) how do diabetes stakeholders evaluate this therapy? The studies combine systematic reviews, ethical analyses, and qualitative research. Interviews with patients and professionals reveal hopes for greater flexibility, and privacy in daily life, alongside concerns about safety, functionality, and equity. The findings provide a foundation for responsible clinical translation and implementation of the BAP.
Short description thesis:
Since the emergence of gene therapy research in the 1990s, people with hemophilia and their healthcare providers have hoped that this innovation might one day provide a cure. However, gene therapy also raises ethical questions. Therefore, this thesis scrutinizes the meaning of ‘cure’ and investigates how gene therapy for hemophilia can be further developed and implemented in an ethically responsible manner, responsive to the needs of stakeholders.
The analysis demonstrates that “cure” can refer to normalization of the body, obtaining a normal life, or change in narrative identity. Given these variations, the term should be used cautiously and context-specifically to prevent misunderstanding and unrealistic expectations.
I end with three concrete recommendations for the further development of gene therapy and hemophilia care in general: to reflect on the goals of gene therapy, to have attention for the ethics of communicating about gene therapy and to design care for hemophilia in a way that can enhance the independence of people living with hemophilia.
Short description of thesis:
Post-acute infection syndromes, such as post COVID-19 condition (PCC) and Q-fever fatigue syndrome (QFS), present a major public health challenge due to the large impact on both affected individuals and on society. Patients experience a wide range of symptoms and varying degrees of functional limitations, which hinder their daily life and societal participation. The heterogenous presentation in terms of symptoms, functioning and longitudinal trajectory presents notable challenges for both patients suffering from these conditions and for professionals tasked with the care for these patients. To gain more insight into these conditions, this thesis aimed to evaluate outcomes in patients with post-acute infection syndromes, to explore determinants associated with these outcomes, to examine heterogeneity in outcomes in this patient population, and to identify opportunities for improving healthcare for these patients.
Short description of thesis:
This thesis investigates the potential effectiveness of pancreatic cancer surveillance. In the first part, we used our microsimulation model to evaluate the impact of several uncertainties (e.g. pancreatic cancer risk, natural disease course) on surveillance effectiveness. We adjusted our microsimulation model to gain insight into the natural disease course of pancreatic cancer and found a 4 year window of opportunity for HGD detection. In the second part, we evaluated the maximum effect of pancreatic cyst surveillance and found a number needed to surveil so high that cyst surveillance seems inefficient with currently used expensive and burdensome tests. In the third part, dedicated to individuals with a hereditary increased pancreatic cancer risk, we performed a clinical surveillance study of high risk individuals. Finally, we used our microsimulation model to evaluate long-term effectiveness of pancreatic cancer surveillance in CDKN2A mutation carriers.