Evidence on bringing specialised care to the primary level-effects on the Quadruple Aim and cost-effectiveness: a systematic review.

BACKGROUND: To achieve the Quadruple Aim of improving population health, enhancing the patient experience of care, reducing costs and improving professional satisfaction requires reorganisation of health care. One way to accomplish this aim is by integrating healthcare services on different levels. This systematic review aims to determine whether it is cost-effective to bring a hospital specialist into primary care from the perspectives of commissioners, patients and professionals. METHODS: The review follows the PRISMA guidelines. We searched PubMed, Scopus and EBSCO (CINAHL and Academic Search Ultimate) for the period of 1992-2022. In total, 4254 articles were found, and 21 original articles that reported on both quality and costs, were included. The JBI and ROBINS-I tools were used for quality appraisal. In data synthesis, vote counting and effect direction plots were used together with a sign test. The strength of evidence was evaluated with the GRADE. RESULTS: Cost-effectiveness was only measured in two studies, and it remains unclear. Costs and cost drivers for commissioners were lower in the intervention in 52% of the studies; this proportion rose to 67% of the studies when cost for patients was also considered, while health outcomes, patient experience and professional satisfaction mostly improved but at least remained the same. Costs for the patient, where measured, were mainly lower in the intervention group. Professional satisfaction was reported in 48% of the studies; in 80% it was higher in the intervention group. In 24% of the studies, higher monetary costs were reported for commissioners, whereas the clinical outcomes, patient experience and costs for the patient mainly improved. CONCLUSIONS: The cost-effectiveness of the hospital specialist in primary care model remains inconclusive. Only a few studies have comprehensively calculated costs, evaluating cost drivers. However, it seems that when the service is well organised and the population is large enough, the concept can be profitable for the commissioner also. From the patient's perspective, the model is superior and could even promote equity through improved access. Professional satisfaction is mostly higher compared to the traditional model. The certainty of evidence is very low for cost and low for quality. TRIAL REGISTRATION: PROSPERO CRD42022325232, 12.4.2022.

The Hospital Frailty Risk Score-outcomes in specialised services.

BACKGROUND: Frailty is increasingly used to risk stratify older people, but across specialised services there is no standardised approach. The aim of this study was to assess if the Hospital Frailty Risk Score (HFRS) could describe outcomes for older people within English specialised services. DESIGN: A retrospective cohort study was performed using the Secondary Uses Service (SUS) electronic database for people aged 75 or older admitted between April 2017 and March 2018. METHODS: Based on HFRS, the populations were risk stratified into mild, moderate and severe frailty risk. The relationships with length of stay, readmission rate, mortality and some selected condition specific treatment complications were quantified using descriptive statistics. RESULTS: Very few individuals (<2%) could not be risk stratified for frailty risk. Frailty was differentially distributed across the specialties; around one-third had mild frailty; another third had moderate frailty and one-quarter severe frailty. Increasing frailty risk was associated with increased length of stay for the index admission, more days in hospital in the year following intervention and increased risk of dying in hospital. Severe frailty was a powerful discriminator of the risk of death; between 25 and 40% of those with severe frailty risk died at 30 months across all specialties. CONCLUSIONS: This study demonstrates the first application of the HFRS to a national dataset to describe service outcomes and mortality for older people undergoing a range of specialised interventions. This information could be used to identify those that might benefit from holistic assessment, aid prognostication, commissioning and service planning.

Managing patients with severe asthma in Australia: Current challenges with the existing models of care.

Severe asthma leads to debilitating symptoms for patients and excessive socioeconomic burden for the community. Comprehensive models of care are required to address complex issues, risk factors and comorbidities in patients with severe asthma, and to identify patients most appropriate for specialised treatments. Dedicated severe asthma services improve asthma control, reduce asthma exacerbations and hospital admissions, and improve quality of life. Currently, diverse models of care exist for managing severe asthma across Australia. Most referrals to severe asthma services are from respiratory physicians seeking a second opinion or from primary care for poorly controlled asthma. Despite benefits of specialised severe asthma services, many patients are not referred and resources are limited, often resulting in long waiting times. Patient referral is often unstructured and there are considerable variations in the management of severe asthma with limited access to other health care professionals such as speech pathologists and dieticians, and restricted scope to optimise patient work-up before referral. Ongoing communication between the specialist and referring clinician is essential for continuity of care but is often lacking. Referral pathways can be optimised by developing referral criteria and guidelines to triage patients with severe asthma and to improve resource efficiency. Additional education and tools for assessing and managing severe asthma are needed, and mechanisms should be developed for involving primary care in the management of stabilised patients. Strategies to increase patient access to multidisciplinary services are recommended.

Practical application of brief cognitive tests.

INTRODUCTION: Brief cognitive tests (BCT) may help detect cognitive impairment (CI) in the clinical setting. Several BCT have been developed and/or validated in our country, but we lack specific recommendations for use. DEVELOPMENT: Review of studies on the diagnostic accuracy of BCT for CI, using studies conducted in Spain with BCT which take less than 20 min. We provide recommendations of use based on expert consensus and established on the basis of BCT characteristics and study results. CONCLUSION: The Fototest, the Memory Impairment Screen (MIS) and the Mini-Mental State Examination (MMSE) are the preferred options in primary care; other BCT (Clock Drawing Test [CDT], test of verbal fluency [TVF]) may also be administered in cases of negative results with persistent suspected CI or concern (stepwise approach). In the specialised care setting, a systematic assessment of the different cognitive domains should be conducted using the Montreal Cognitive Assessment, the MMSE, the Rowland Universal Dementia Assessment, the Addenbrooke's Cognitive Examination, or by means of a stepwise or combined approach involving more simple tests (CDT, TVF, Fototest, MIS, Memory Alteration Test, Eurotest). Associating an informant questionnaire (IQ) with the BCT is superior to the BCT alone for the detection of CI. The choice of instruments will depend on the patient's characteristics, the clinician's experience, and available time. The BCT and IQ must reinforce - but never substitute - clinical judgment, patient-doctor communication, and inter-professional dialogue.

Safety incidents involving confused and forgetful older patients in a specialised care setting--analysis of the safety incidents reported to the HaiPro reporting system.

AIMS AND OBJECTIVES: To describe the safety incidents involving confused and forgetful older patients in a specialised care setting entered in the HaiPro reporting system. BACKGROUND: About 10% of patients experience a safety incident during hospitalisation, which causes or could cause them harm. The possibility of a safety incident during hospitalisation increases significantly with age. A mild or moderate memory disorder and acute confusion are often present in the safety incidents originating with an older patient. DESIGN: The design of the study was action research with this study using findings from one of the first-phase studies, which included qualitative and quantitative analysed data. METHODS: Data were collected from the reporting system for safety incidents (HaiPro) in a university hospital in Finland. There were 672 reported safety incidents from four acute medical wards during the years 2009-2011, which were scrutinised. Seventy-five of them were linked to a confused patient and were analysed. RESULTS: The majority of the safety incidents analysed involved patient-related accidents. In addition to challenging behaviour, contributing factors included ward routines, shortage of nursing staff, environmental factors and staff knowledge and skills. Nurses tried to secure the patient safety in many different ways, but the modes of actions were insufficient. CONCLUSION AND RELEVANCE TO CLINICAL PRACTICE: Nursing staff need evidence-based information on how to assess the cognitive status of a confused patient and how to encounter such patients. The number of nursing staff and ward routines should be examined critically and put in proportion to the care intensity demands caused by the patient's confused state. The findings can be used as a starting point in the prevention of safety incidents and in improving the care of older patients.

Addiction interprofessional experiences of care: Stress, coping and transformation.

This study aimed to explore healthcare professionals' attribution of meaning to the concept of addiction, treatment and recovery. The study was conducted through semi-structured interviews with 18 healthcare professionals in the field of addiction: nine nurses, six psychologists, a psychiatrist, a social worker and a psychosocial technician. Based on a qualitative methodology, a thematic analysis was performed using the NVivo software. Regarding professional stress, the results revealed several stress factors related to the intervention (e.g. strategic powerlessness and uncertainty of intervention's effectiveness), the addicted individuals (e.g. motivation, difficulties in adhering to treatment, patient behaviour, negative patient emotionality and lack of socio-economic resources) as well as the healthcare services (e.g. team stress). Adaptive coping strategies were reported, both used in the work context (e.g. cognitive coping strategies and acceptance) and in personal life (e.g. work-family boundaries and family support and closeness). Despite the professional stress experienced, the participants' narratives indicated positive transformations of the self, which suggests resilient professional trajectories.

Effects of municipality factors on care transitions.

AIMS: To analyse whether transitions between care settings differ between municipalities in the last 2 years of life among older people in Finland. METHODS: Data were derived from Finnish national registers, and include all those who died in 2002 and 2003 at the age of 70 or older except those living in very small municipalities (n=67,027). Data include admissions and discharges from health and social care facilities (university hospitals, general hospitals, health centres, residential care facilities) and time spent outside care facilities for 730 days prior to death. Three-level negative binomial regression analyses were performed to study the effect of municipal factors on (1) the total number of all care transitions, (2) the number of transitions between home and different care facilities, and (3) transitions between different care facilities. RESULTS: The municipality of residence had only a minor effect on the total number of care transitions, but greater variation between municipalities was found when different types of care transition were examined separately. Largest differences were found in care transitions involving specialised care. Age structure, urbanity, and economic situation of the municipality had an impact on several different care transitions. CONCLUSION: The total number of care transitions in 2 final years of life was approximately similar irrespective of the municipality of residence, but the findings imply differences in transitioning specialised care. Potentially, this may suggest inequality between the municipalities, but more detailed studies are needed to confirm the factors underlying these differences.

Pay-for-Performance incentives for specialised services in England: a mixed methods evaluation.

BACKGROUND: A Pay-for-Performance (P4P) programme, known as Prescribed Specialised Services Commissioning for Quality and Innovation (PSS CQUIN), was introduced for specialised services in the English NHS in 2013/2014. These services treat patients with rare and complex conditions. We evaluate the implementation of PSS CQUIN contracts between 2016/2017 and 2018/2019. METHODS: We used a mixed methods evaluative approach. In the quantitative analysis, we used a difference-in-differences design to evaluate the effectiveness of ten PSS CQUIN schemes across a range of targeted outcomes. Potential selection bias was addressed using propensity score matching. We also estimated impacts on costs by scheme and financial year. In the qualitative analysis, we conducted semi-structured interviews and focus group discussions to gain insights into the complexities of contract design and programme implementation. Qualitative data analysis was based on the constant comparative method, inductively generating themes. RESULTS: The ten PSS CQUIN schemes had limited impact on the targeted outcomes. A statistically significant improvement was found for only one scheme: in the clinical area of trauma, the incentive scheme increased the probability of being discharged from Adult Critical Care within four hours of being clinically ready by 7%. The limited impact may be due to the size of the incentive payments, the complexity of the schemes' design, and issues around ownership, contracting and flexibility. CONCLUSION: The PSS CQUIN schemes had little or no impact on quality improvements in specialised services. Future P4P programmes in healthcare could benefit from lessons learnt from this study on incentive design and programme implementation.

Driving time-based identification of gaps in specialised care coverage: An example of neuroinflammatory diseases in Germany.

Objective: Due to the growing complexity in monitoring and treatment of many disorders, disease-specific care and research networks offer patients certified healthcare. However, the networks' ability to provide health services close to patients' homes usually remains vague. Digital Health Technologies (DHTs) help to provide better care, especially if implemented in a targeted manner in regions undersupplied by specialised networks. Therefore, we used a car travel time-based isochrone approach to identify care gaps using the example of the neuroinflammation-focused German healthcare and research networks for multiple sclerosis (MS), myasthenia gravis (MG), myositis and immune-mediated neuropathy. Methods: Excellence centres were mapped, and isochrones for 30, 60, 90 and 120 minutes were calculated. The resulting geometric figures were aggregated and used to mask the global human settlement population grid 2019 to estimate German inhabitants that can reach centres within the given periods. Results: While 96.48% of Germans can drive to an MS-focused centre within one hour, coverage is lower for the rare disease networks for MG (48.3%), myositis (43.1%) and immune-mediated neuropathy (56.7%). Within 120 minutes, more than 80% of Germans can reach a centre of any network. Besides the generally worse covered rural regions such as North-Eastern Germany, the rare disease networks also show network-specific regional underrepresentation. Conclusion: An isochrone-based approach helps identify regions where specialised care is hard to reach, which might be especially troublesome in the case of an often disabled patient collective. Patient care could be improved by focusing deployments of disease-specific DHTs on these areas.

Reasons for contacting the consultation service of a Dutch assault centre.

A telephone and e-mail helpline known as the Consultation Service, open to all the public, was launched at a Dutch Centre for Sexual Violence to deal with non-acute sexual violence. The aim of this study was to gain insight into case characteristics, reasons for contacting the Consultation Service and whether these reasons differed for victims, their relatives and professionals. Using a mixed methods design, the study included all consultations handled at the Consultation Service in 2018 and 2019. Descriptive statistics described quantitatively the case characteristics, the themes and differences between victims, relatives and professionals. The themes of the reasons for contact were established from the qualitative analysis, using the method of content analysis. Cases were characterised by complexity. Three themes emerged: case complexity, decision-making on care options and reporting to the police, which differed for victims, relatives and professionals. The differences in reasons for contacting the helpline imply that approaches should be adapted and fitted to different clients. Specialised care is needed to guide clients through cases that are challenging and often complex. There is a widespread lack of knowledge of options in addition to the complex multifaceted aspects to decision making about actions post-assault. Assault centres should implement a Consultation Service in which integrated care is offered not only to the victims, but also to their relatives and professionals.

Improvement Strategies for the Challenging Collaboration of General Practitioners and Specialists for Patients with Complex Chronic Conditions: A Scoping Review.

Introduction: Coordination of healthcare professionals seems to be particularly important for patients with complex chronic disease, as they present a challenging interplay of conditions and symptoms. As one solution, to counteract or prevent this, improving collaboration between general practitioners (GPs) and specialists has been the aim of studies by linking or coordinating their services along the continuum of care. This scoping review summarises role distributions and components of this collaboration that have potential for improvement for the care of patients with complex chronic conditions. Methods: Scoping review as a knowledge synthesis for components of collaboration and role distributions between medical specialists and GPs in intervention studies. The PubMed database was searched for literature from 2010-2020. Results: Literature search and reference screening generated 2,174 articles. 30 articles originating from 22 unique projects were included in our synthesis. In the interventions to improve collaboration, the GP is most commonly in charge of patient management and extends the scope of practice. The specialist provides support when needed. Clear definition of roles, resources for knowledge transfer and education from specialists are commonly utilised interventions. Typically, combinations of process and system changes addressing communication and coordination issues are applied. Most interventions improve provider and patient satisfaction, health outcomes, and reduce care fragmentation. Conclusion: This review showed that interventions to improve collaboration between GPs and medical specialists seem promising. Further efforts should be made to test and apply the findings systematically in broad clinical practice.

Development of the professional role of diabetes nurses in specialised diabetes and primary care in Catalonia.

OBJECTIVES: The aim of this study was to describe therapeutic education programmes in diabetes in Catalonia and the differences according to the healthcare setting in which the patients are attended (primary care [PC] and specialised diabetes care [SDC]). METHOD: We performed a descriptive, cross-sectional, comparative study of diabetes nurses (DN) in SDC and PC in Catalonia. The sample was obtained from all the DN in SDC and a randomised cluster sample of DN in PC. The questionnaire of the Study of European Nurses in Diabetes (SEND) validated in Spanish was used. RESULTS: A total of 287 questionnaires were analysed (24.3% SDC and 75.6% PC). More training in diabetes through masters, postgraduate courses and continuing education was observed in SDC (p<0.001) as well as structured, written, education programmes and the combination of individual and group education strategies (p<0.05). The roles of educator, consultant, researcher, manager, liaison or collaborator and innovator and the telematic follow-up of patients was also more developed in SDC (p<0.05). The grade of work satisfaction was elevated in both groups. CONCLUSIONS: (1) Nursing professionals in SDC assume more roles of specialised clinical nursing and also have more training in diabetes and therapeutic education than PC professionals. (2) Professionals in SDC work with a greater proportion of structured diabetes therapeutic education programmes although this should improve in both settings. According to the results obtained and the scientific evidence currently available, the training of DN working in the care of people with diabetes should be accredited in order to increase the use of structured programmes and investigation by DNs in both healthcare settings.

Validity and reliability of the 9-item Shared Decision Making Questionnaire (SDM-Q-9) in a national survey in Hungary.

BACKGROUND: The nine-item Shared Decision Making Questionnaire (SDM-Q-9) is one of the most frequently applied instruments for assessing patients' involvement in medical decision-making. Our objectives were to develop a Hungarian version of SDM-Q-9, to evaluate its psychometric properties and to compare its performance between primary and specialised care settings. METHODS: In 2019, a sample of adults (n = 537) representative of the Hungarian general population in terms of age, gender and geographic region completed an online survey with respect to a recent health-related decision. Outcome measures included SDM-Q-9 and Control Preferences Scale-post (CPSpost). Item characteristics, internal consistency reliability and the factor structure of SDM-Q-9 were determined. RESULTS: The overall ceiling and floor effects for SDM-Q-9 total scores were 12.3% and 2.2%, respectively. An excellent internal consistency reliability (Cronbach's alpha 0.925) was demonstrated. Exploratory factor analysis resulted in a one-factor model explaining 63.5% of the variance of SDM-Q-9. A confirmatory factor analysis supported the acceptability of this model. Known-groups validity was confirmed with CPSpost categories; mean SDM-Q-9 total scores were higher in the 'Shared decision' category (72.6) compared to both 'Physician decided' (55.1, p = 0.0002) and 'Patient decided' (57.2, p = 0.0086) categories. In most aspects of validity and reliability, there was no statistically significant difference between primary and specialised care. CONCLUSIONS: The overall good measurement properties of the Hungarian SDM-Q-9 make the questionnaire suitable for use in both primary and specialised care settings. SDM-Q-9 may be useful for health policies targeting the implementation of shared decision-making and aiming to improve efficiency and quality of care in Hungary.

Pay for performance for specialised care in England: Strengths and weaknesses.

Pay-for-Performance (P4P) schemes have become increasingly common internationally, yet evidence of their effectiveness remains ambiguous. P4P has been widely used in England for over a decade both in primary and secondary care. A prominent P4P programme in secondary care is the Commissioning for Quality and Innovation (CQUIN) framework. The most recent addition to this framework is Prescribed Specialised Services (PSS) CQUIN, introduced into the NHS in England in 2013. This study offers a review and critique of the PSS CQUIN scheme for specialised care. A key feature of PSS CQUIN is that whilst it is centrally developed, performance targets are agreed locally. This means that there is variation across providers in the schemes selected from the national menu, the achievement level needed to earn payment, and the proportion of the overall payment attached to each scheme. Specific schemes vary in terms of what is incentivised - structure, process and/or outcome - and how they are incentivised. Centralised versus decentralised decision making, the nature of the performance measures, the tiered payment structure and the dynamic nature of the schemes have created a sophisticated but complex P4P programme which requires evaluation to understand the effect of such incentives on specialised care.

An ordinary life: People with dementia living in a residential setting.

The aim of this research was to explore the experiences of care support workers and family members of the impact of a new care approach in a specialised unit as it shifted from a clinical to an inclusive model, focused on creating an ordinary life for people with dementia and their families. The research was a partnership between the unit staff and university researchers. Using a qualitative approach, data were collected in focus groups with 11 family members and nine staff members. Thematic analysis identified the themes personalised care for people with dementia, family involvement - continuing to care, and staff competence and confidence to care. A personalised approach to supporting people with dementia was considered paramount, communicative family-staff relationships enhanced the social environment, and competence enhanced confidence and quality care. Participants identified positive ways of working that benefited staff and families and they reported increased well-being for the people with dementia on the unit. Developing well-articulated and systematically implemented local models of care provides opportunities for family and staff creativity and engagement, enhancing care for people with dementia. Strong and effective leadership is required to enable these approaches to become a reality.

Improving People's Self-Reported Experience with the Health Services: The Role of Non-Clinical Factors.

The main aim of this study was to analyse the association between non-clinical factors and the self-reported experience of people with the main health services of the Spanish public healthcare system. Specifically, we analysed whether factors such as the treatment received from health staff, the confidence transmitted to the patient by the doctor, or waiting time for a diagnostic test had an influence on people reporting a more satisfactory experience with primary, specialised, and hospital care services. We used cross-sectional microdata from the Spanish Healthcare Barometer survey of 2015 comprising a sample of 7800 individuals. We applied a probit-adapted ordinary least squares estimation, which is one of the most widely used methods in recent studies on subjective well-being. Our findings suggest that individuals' interaction with non-clinical factors was positively correlated with the overall health services experience. Treatment received from health staff was one of the most relevant factors to ensure that individuals report a more satisfactory experience with primary care. Time devoted by physicians to each patient and waiting time for a non-emergency admission were the most correlated factors in specialised and hospital care services, respectively. This study could have implications for public policies. First, it shows policy-makers the influence of non-clinical factors when individuals rate their overall experience with the main health services in Spain. Second, it identifies the key factors where the health system could reallocate more public resources to improve people's experience and thus the health system responsiveness.

[Map of resources and healthcare needs for patients with dyslipidaemia in Spain: The MADI study]. / Mapa de recursos y necesidades asistenciales del paciente dislipidémico en España: estudio MADI.

INTRODUCTION: Dyslipidaemia is a major modifiable determining factor of vascular risk and, despite this, a significant number of patients do not achieve lipid goals. The aim of this study is to describe the resources and current needs in clinical practice in Spain, through an analysis of management, organisation and the patient care circuit of dyslipidaemia patient. METHODS: A descriptive, cross-sectional, multicentre study, using a questionnaire, was conducted on physicians, 266 in primary care (PC) and 258 in specialised care (SC), who attended patients with dyslipidaemia in hospitals and centres within the National Health System. Probabilistic analyses were performed, stratifying by care-level, existence of a lipid unit (LU), and geographic area. RESULTS: Observed differences were mostly due to geographic location, rather than the existence of LU in the referral hospitals. Most system deficiencies were found in the southern provinces of the country. Nearly all primary care physicians declared that they diagnose, manage and control dyslipidaemia patients, but a general agreement was lacking for diagnostic and referral criteria. The scarce use of a shared protocol between PC and SC showed evidence of poor coordination between health care providers. Furthermore, there was a remarkably low proportion of patients receiving health care education for their disease. CONCLUSIONS: This study emphasises the need to identify weaknesses in the dyslipidaemia patient care circuit, and to perform the appropriate remedial actions, in particular, to promote coordination between levels of care and to foster patient education about their disease.

Internet-based CBT for social phobia and panic disorder in a specialised anxiety clinic in routine care: Results of a pilot randomised controlled trial.

Ample studies have demonstrated that internet-based cognitive behavioural therapy (iCBT) for anxiety disorders is effective and acceptable in controlled settings. Studies assessing the clinical effectiveness of iCBT for anxiety disorders among routine care populations are, however, not as numerous. The purpose of this study was to assess the effectiveness of iCBT among anxiety patients, who were on a waiting list for intensive outpatient treatment, in a specialised routine care clinic. A randomised controlled pilot trial was conducted. Recruited patients were on a waiting list and had a primary diagnosis of either social phobia or panic disorder. Participants were randomised into either receiving iCBT with minimal therapist contact (received access to the programme FearFighter® (FF) and received support from a clinician via telephone) or no treatment (stayed on the waiting list). The primary outcome was self-reported symptomatic change of anxiety on Beck Anxiety Inventory (BAI). The secondary outcomes were comorbid depression measured on Beck Depression Inventory (BDI-II) and quality of life measured with the EuroQol one-item visual-analogue scale (EQ-vas). All results were analysed by intention-to-treat analyses using a mixed-effects approach. N = 158 patients were assessed for eligibility of which N = 67 met all eligibility inclusion criteria, signed informed consent forms, and were randomised. Post-treatment assessment was completed by N = 47 (70%). In the intervention group, N = 11 (31%) completed all modules of FF. No significant differences of change of symptomatic levels were found between the intervention and control group for anxiety (BAI: mean diff. = 2.42; 95% CI - 1.03 to 5.86; p = 0.17; d = 0.06) or for depression (BDI-II: mean diff. 1.87; 95% CI - 2.25 to 6.00; p = 0.37; d = 0.02). A large and significant effect was found in self-reported quality of life in favour of the experimental group (EQ-vas: mean diff. - 20.88; 95% CI - 30.64 to - 11.11; p < 0.001; d = 0.81). This study was not able to document statistically significant clinical effect of iCBT with minimal therapist contact compared to a waiting list control group in a specialised anxiety clinic in routine care. However, a large and significant effect was seen on self-reported quality of life. Although these results offer an interesting perspective on iCBT in specialised care, they should be interpreted with caution, due to the limitations of the study. A large scale fully powered RCT is recommended.

Reimbursement of licensed cell and gene therapies across the major European healthcare markets.

OBJECTIVE: The aim of this research is to identify the pricing, reimbursement, and market access (P&R&MA) considerations most relevant to advanced therapy medicinal products (ATMPs) in the Big5EU, and to inform their manufacturers about the key drivers for securing adoption at a commercially viable reimbursed price. METHODOLOGY: The research was structured following three main steps: 1) Identifying the market access pathways relevant to ATMPs through secondary research; 2) Validating the secondary research findings and addressing any data gaps in primary research, by qualitative interviews with national, regional, and local-level payers and their clinical and economic advisors; 3) Collating of primary and secondary findings to compare results across countries. RESULTS: The incremental clinical benefit forms the basis for all P&R&MA processes. Budget impact is a key consideration, regardless of geography. Cost-effectiveness analyses are increasingly applied; however, only the United Kingdom has a defined threshold that links the cost per quality-adjusted life year (QALY) specifically and methodologically to the reimbursed price. Funding mechanisms to enable adoption of new and more expensive therapies exist in all countries, albeit to varying extents. Willingness to pay is typically higher in smaller patient populations, especially in populations with high disease burden. Outcomes modelling and risk-sharing agreements (RSAs) provide strategies to address the data gap and uncertainties often associated with trials in niche populations. CONCLUSIONS: The high cost of ATMPs, coupled with the uncertainty at launch around their long-term claims, present challenges for their adoption at a commercially viable reimbursed price. Targeting populations of high disease burden and unmet needs may be advantageous, as the potential for improvement in clinical benefit is greater, as well as the potential for capitalising on healthcare cost offsets. Also, targeting small populations can also help reduce both payers' budget impact concerns and the risk of reimbursement restrictions being imposed.