For almost ten years a cyclic appropriate care program has been in place in The Netherlands, known as Zinnige Zorg. The program spanned the full International Classification of Diseases (10th Edition). In 2016 a project on mental health was started. A full cycle consisted of four phases: screening (including priority setting), in-depth analysis, implementation and evaluation. During the in-depth analysis phase, the mental health practice as it was provided was compared to the advice in the guidelines. The mental health project is now in the implementation phase. Professionals, mental healthcare institutions, health insurers and patients are now collaborating to reach the goals that have been set at the closure of the analysis phase.

Project documentation was analysed to describe the way stakeholders were involved in priority setting as well as their subsequent involvement in implementation of appropriate care actions.

The present case report describes two factors that are important in engaging stakeholders:

(i) Priority setting started with interviews with different stakeholders. This led to a selection of 9 themes for investigating appropriate care.

(ii) For these themes stakeholders formulated 45 issues, together with their consequences for mental health patients, without formulating solutions. If necessary they were reformulated as: [group of patients x] experiences [bottleneck y in mental healthcare], this leads to the patients [negative consequence z]. Next, 9 issues were prioritized and 4 selected, with input from the stakeholders.

Finally, two diseases were selected for which the issues were investigated in depth. This focus enables development of specific implementation steps and evaluation of their effects.

Currently, stakeholders are collaborating in a constructive manner in the implementation phase of this cyclic appropriate care program to improve mental health care for patients experiencing psychosis or post-traumatic stress disorder. Important characteristic of the process that might have supported the present collaborative effort in implementation were (i) early involvement of the stakeholders and (ii) an orientation on problems experienced by patients in the priority setting phase.

Since 2013, the National Health Care Institute in the Netherlands has systematically analyzed the appropriateness of care provided under public health insurance. Here we present the method used, the results up to now, and what we have learned from it.

The appropriate care program consists of four phases: screening, in-depth analysis, implementation, and evaluation. Stakeholder involvement is a central part of the process. For every ICD-10 area, a screening took place to select care trajectories for in-depth analysis with a potential for wiser choices and more appropriate care. The in-depth analysis indicates which improvements can be made to reach more appropriate care, by assessing guideline adherence. During the implementation phase, which is primarily carried out by clinicians, patients and health insurers, actions are taken to improve care on the identified points. In the evaluation phase, we examine to what extent improvements have been achieved.

Currently, all ICD-10 areas have been screened and 29 selected care trajectories have been subjected to in-depth analyses. The analyses resulted in the identification of more than a hundred areas for potential improvement of the appropriateness of care. For most topics implementation of changes is currently taking place. The four most important impact-enhancing lessons learned by applying the working method are: (i) ICD-10 areas as a starting point for screening are not the most efficient method to reach the biggest impact. (ii) The screening should take a societal perspective. (iii) All public and private parties involved should fulfill their role and take responsibility. (iv) To fulfill our own role better, the working method should be more connected to health technology assessment for reimbursement decisions.

The program has resulted in the identification of many valuable points for improvement which could lead to more appropriate care in the coming years. The impact of the program could be increased through priority setting from a societal perspective and improving the connection to our other health technology assessment processes.

The Revised International Staging System (R-ISS) International Myeloma Working Group (IMWG) recommends a minimal panel to detect high-risk cytogenetics (del17p, t[4;14], t[14;16]) for patients with multiple myeloma (MM). In the Brazilian Public Health System, the use of FISH is currently authorized for rare diseases only, not including MM. In 2021, the Brazilian National Committee for Health Technology Incorporation, with the purpose of broadening the use of FISH to MM patients, requested a review to be undertaken by the Health Technology Assessment Center of University of Campinas’ Teaching Hospital. This study presents the results of a meta-analysis comparing FISH vs CC to the detection of the above-mentioned aberrations in MM patients.

On 25 June 2021, a pre-structured search on four databases (Embase, MEDLINE, Cochrane and LILACS) was performed to identify studies comparing FISH and CC results in MM patients for the detection of high-risk cytogenetics (del17p, t[4;14], and t[14;16]) in MM patients’ bone marrow samples. Study selection, risk of bias assessment, data extraction (frequency of positive tests) and quality of evidence assessment were performed by two independent researchers. Conflicts were solved in agreement meetings with a third researcher. Meta-analysis was performed using frequency of positives to obtain Risk Difference (RD), a surrogate measure of the surplus positive tests between FISH and CC.

From a total of 1346 rendered entries, 11 studies were selected. Only observational studies were available. These studies presented an overall high risk of bias (QUADAS-2). A total of 781 patients were assessed (653 evaluated by FISH and 719 by CC). Meta-analysis results showed that, for t(4;14) FISH detected 12 percent more samples (RD:0.12 [95% confidence interval (CI):0.06-0.19]). For t(14;16), FISH detected 0.42 percent more samples (RD:0.00 [95%CI:-0.01-0.02]). And for del17p, FISH detected 1.6 percent more samples (RD:0.12 [95%CI:0.04-0.20]).

FISH appears to be more effective than CC on the detection of t(4;414) and del17p aberrations, and can be a useful tool in hematology practice. The results of t(14;16) presented non-superiority, probably due to the low frequency of this aberration.

Magnetic resonance imaging guided high-intensity focused ultrasound (MR-HIFU) is a non-invasive technique with a low risk of complications for the patient and few side effects. Integration with MRI allows monitoring of the temperature regime of thermal doses, which protects important structures from overheating, and at the same time directing a high thermal dose to the target tissue.

MR-HIFU in the treatment of uterine fibroids, prostate cancer and the treatment of pain in bone metastases is compared with both traditional methods of treatment (uterine artery embolization, hysterectomy, prostatectomy, etc.).

To assess the clinical effectiveness and safety of non-invasive MR-HIFU, a literature search was performed in the MEDLINE database using the following keywords:“MRgFUS” “MR-HIFU”. The following filters were used: (i) article type: meta-analysis, systematic review, guidance; (ii) date of publication: no later than 5 years (from 2016).

According to the search terms, 104 publications were submitted to MEDLINE for keywords. After using filters, 57 publications were identified to familiarize themselves with research abstracts. The analysis included six publications according to PICO criteria.

The use of non-invasive MR-HIFU therapy for the treatment of uterine fibroids, prostate cancer and various forms of metastatic bone lesions does not have convincing evidence of advantages over standard treatment methods (surgical resection, embolization, etc.) and may be used only as an alternative technique or in addition to standard therapy.

Despite some advantages of the MR-HIFU technology, it is experimental and should only be used as an alternative to surgical treatment. Convincing evidence of the efficacy of MR-HIFU treatment in meta-analyses, systematic reviews, and randomized controlled trials has not yet been published.

Constantly rising costs of pharmaceuticals and biologics spurred a debate in recent years leading to increasingly persistent public calls to overhaul the existing system of pricing and distributing health technologies. The COVID-19 pandemic exposed the controversies of the current model of access to health commodities in all evidence when the existing discrepancy between the global supply and the global demand in health technology can be viewed as an illustration of a conflict between the neoliberal free market ideal of health innovation genesis and ownership and the original democratic principles of what would now be called sustainable human development.

Presented here is an integrative literature review of over 265 publications in peer-reviewed journals on Pubmed and Web of Science, academic and “grey” literature, mainly publicism, published in English. We reviewed and analyzed included literature for the purposes of identifying leading ideas with regards to ethical frameworks for evaluating or referencing value for pricing health commodities.

Seven drug pricing models were analyzed in terms of them satisfying the three most common criteria of price adequacy – its fairness (viewed from the point of view of main schools of ethical thought), accountability-for-reasonableness (including transparency of decisions, relevance, existing mechanisms of revisions and governance to ensure compliance), and price functionality. One of the central ideas under controversy is value, its relative character for different contexts due to the high weight of the willingness-to-pay in the value-based health technology assessment (HTA) decisions and the relative value of money, and the attempts to quantify value in a universal way for institutions, patients, and originators.

While the review scored the pricing models on their “public fairness” with volume-based rather than value-based pricing models leading the rating, the main conclusion of the review is that the main meaningful divide is between value creation and value extraction when pricing health innovation.

Health Technology Wales (HTW) review guidance 3 years after publication to establish if reassessment is warranted because, for example, new evidence has become available. Since the publication of guidance on flash glucose monitoring (FGM) in 2018, HTW introduced a patient and public involvement (PPI) process with novel approaches to flexible engagement. This enabled HTW to include three streams of patient evidence into the review of FGM devices.

HTW’s Patient and Public Involvement Standing Group (PPISG) considered appropriate methods of engagement using the HTW Patient and Public Involvement Mechanism Tool. This tool considers the nature of the health technology, the presence of appropriate patient organizations and questions that can be put to patients, as well as other approaches for obtaining patient evidence.

HTW contacted Diabetes Cymru and met with them to discuss contributing to the appraisal of FGM devices. Diabetes Cymru produced a patient submission summarizing the experiences of their patient network, with particular focus on the expansion of the technology to closed-loop insulin systems. Diabetes Cymru later attended HTW’s Appraisal Panel committee and gave a presentation. Additionally, HTW conducted a patient evidence literature review. This review summarized published qualitative studies on a range of perspectives, including carer perspectives, family perspectives, children and adolescences perspectives as well as considerations from specific environments, such as schools, workplaces, homes, care homes and communities. In addition to new clinical and cost effectiveness evidence, this PPI input was used to formulate new guidance recommending more widespread adoption of FGM.

The introduction of flexible approaches to PPI enabled HTW to gain patient evidence from multiple sources. This ensured greater patient representation and a more detailed understanding of the role of FGM devices across different patient communities. This added considerable richness to the patient evidence, which is vital to understand the everyday impacts of FGM and its use amongst patients. Combining flexible PPI with the new clinical and cost effectiveness evidence resulted in a change in the original guidance recommendation.

It has been suggested that health economists need to adapt their methods in order to meet the challenges of evaluating molecular diagnostics. The aim of this review is to categorize and critically examine the challenges and methodological developments identified from the literature and to suggest how such challenges may be addressed.

We identified challenges and suggested methodological improvements using a systematic rapid review of the literature. We categorized challenges into those common to all economic evaluations, those common to all diagnostic technologies and those relevant to molecular diagnostics. We assessed whether development in the methods of economic evaluation or alternative action was required.

We found forty-one papers which identified twelve challenges. Choice of perspective and time-horizon were challenges common to all economic evaluations. Five challenges were relevant for all diagnostic technologies: complexity of analysis; range of costs; under-developed evidence base; behavioral aspects; and choice of outcome metrics. The final five challenges were specific to molecular diagnostics: heterogeneity of tests and platforms; increasing stratification; capturing personal utility; incidental findings and spill-over effects. The final five challenges may require methodological development. For example, although methods exist to capture the value of a diagnostic test over and above any health gain captured in a quality adjusted life year (‘personal utility’), there is currently no agreed method of incorporating this into a cost-utility analysis. For the other challenges development of evaluation processes is key. In particular, the weak evidence base for diagnostic technologies may require processes to evolve.

Current methods of economic evaluation are generally able to cope with molecular diagnostics although a renewed focus on specific decision-makers’ needs and a willingness to move away from cost-utility analysis may be required. A key issue is the under-developed evidence-base and it may be necessary to rethink translation processes to ensure sufficient, relevant evidence is available to support economic evaluation and adoption of molecular diagnostics.

Since 2019, Canada’s Drug and Health Technology Agency (CADTH) has worked directly with patients through its Patient and Community Advisory Committee (PCAC). In Brazil, Conitec has worked directly with patients since 2021 through testimonials in its committee meetings for all assessed technologies. In this study we explored patients’ perspectives about their participation in these processes.

Two patients directly involved with both CADTH and Conitec were invited to share their perspectives about the strengths and weaknesses of the patient engagement processes.

For CADTH, the strengths were as follows: PCAC focuses on the whole organization, including the patient engagement strategy and strategic plan, and is on the same level as other professional committees; PCAC members are compensated; the ‘Learning Sessions’ show CADTH staff the ramifications of their work in people’s lives; and there is increased patient engagement on other committees throughout CADTH. The weaknesses identified with the CADTH process were that PCAC is an advisory body with no decision-making authority and that the diversity of people on the PCAC could be increased. In addition, while CADTH informs and consults patients, the PCAC is not involved in individual HTAs.

The strengths of Conitec’s process were that the volunteer is selected by patients; there are opportunities to consolidate direct patient involvement and promote it in other instances; and the technical support is excellent. On the downside, there was a lack of information about this opportunity; the testimonials last only ten minutes and patients are frequently not questioned; only one patient can participate; there is a lack of transparency about testimonial analysis and its role; and being the only representative during a discussion that culminates in a decision can cause anxiety about performance.

Patients felt heard in the engagement processes and stated that the PCAC embeds patient perspectives throughout CADTH, not just in particular HTAs. One patient felt that her participation was essential for the committees to understand patients’ lived experiences.

Since October 2020, Conitec has made public calls (PCs) to invite patients and family members to express perspectives during committee meetings. This study seeks to reflect about the impact of the newest patient and public involvement (PPI) Conitec strategy and how it can contribute to increase patient direct involvement and public confidence regarding Brazilian health decisions in medical devices.

We conducted a document analysis after searching for PCs addressed to medical devices in the section “Patient Perspectives” on Conitec´s website until Aug 31, 2021.

From 64 PCs, five were related to medical devices. Rotational thromboelastometry for transfusion controls: Ended upon applicant request. Transcatheter aortic valve implantation (TAVI): One patient gave his testimonial, explaining how it impacted his quality of life and daily activities, besides answering questions from the committee. The testimonial was not included in the recommendation report. After the public consultation, the initial recommendation changed from non-reimbursement to reimbursement. Botton™ probe for gastrotomy: Eleven people volunteered and a family member was selected. She expressed the importance of the device in her son’s quality of life, making ordinary activities more accessible, helping in the physiotherapy sessions, reducing hospitalizations, and supporting her son’s socialization. The initial recommendation was to reimburse it. iStent Inject Trabecular Micro-Bypass System™ (glaucoma treatment): No patients volunteered. The final recommendation report informed that Conitec received 55 comments through public consultation, including experiences and opinions about the technology. After the public consultation, there was a change from the initial to the final recommendation, from non-incorporation to incorporation. Non-invasive ventilation (cystic fibrosis treatment): There were five volunteers and a mother of twins, both with cystic fibrosis, gave her testimonial. She expressed the importance of this device in her kids’ quality of life, allowing them to sleep better, eat and study, control the symptoms, and reduce hospitalizations and antibiotic usage.

Aspects such as documenting this participation, communicating and offering support to the public and patients, analysis and transparency are essential for evaluating and improving this strategy.

Patients have knowledge, perspectives and experiences that are unique and can make an essential contribution to Health Technology Assessment (HTA). However, in order for their participation to be effective, they need to be able to understand how HTA reports are generated and the decision-making processes that they inform. The aim is to describe the development and virtualization of training materials for patients, as well as to their implementation in a pilot study.

A working group from Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS) agencies was created to develop educational materials in collaboration with patients. The content was based on international initiatives and feedback from the working group. The project was initiated in November 2020. The team consisted of HTA researchers, technicians with experience in training and virtualization and patients. The final version was obtained after an iterative process and refinement of the content and design.

The materials were published in complete and summary versions, and they were translated into Catalan, Basque and Galician. The online course was designed in an e-learning platform (Moodle) with the aim of being implemented by each of the agencies. The materials include relevant and summarized information on HTA processes, current framework at national and European level, and the role of patients in HTA. Health research and the importance of qualitative and quantitative methods are also addressed. The course also includes a module of practical aspects of patient and citizen participation for achieving an effective contribution to HTA. The course is being piloted with patients in different regions in Spain. The objective of the pilot is to evaluate the usefulness and satisfaction with the course, and it has been designed with the purpose of incorporating the pertinent modifications in the course.

The online training course is intended to facilitate the acquisition of knowledge related to the processes and tools of HTA for patients, as well as to inform them in what phases and in what way they can participate. The pilot will provide relevant information on its use in practice. It is expected that the course will favor capacity building and patient involvement.

GesPeDia is a web-based application that provides aggregate clinical information, using outcome and process indicators, and disaggregated patient information. Information is obtained from the electronic medical records. GesPeDia aims to promote people-centered care, improve monitoring of patients’ health outcomes and quality of professional performance. This study aims to evaluate usability, acceptability and satisfaction of GesPeDia.

Nineteen evaluators were included (2 management technicians, 9 health center directors and 8 endocrine consultants). They had access to GesPeDia for two months. Perception of their usefulness for decision-making, acceptability and design satisfaction were measured with an online questionnaire. In addition, suggestions for improvements in the app’s functionalities were collected. Finally, a sample of the evaluators were included in a semi-structured interview to deepen the analysis of dimensions. A descriptive analysis of the data was performed.

The questionnaire was completed by 10 professionals, with mean age of 51.1 years and professional experience 16.5 years. Among the evaluators, 60 percent considered the app quite useful and only 10 percent found it inappropriate for their daily activities. Each of the indicator blocks was rated quite useful. Eight percent considered GesPeDia moderately fast, although for 20 percent navigation within the app was not very intuitive. Appearance was positively valued by 80 percent, despite the fact that 30 percent considered that design does not favor the understanding of contents. Seventy percent considered degree of reliability, relevance and clarity of the contents to be high. Most indicated that information provided by GesPedia is complete for decision-making.

GesPeDia is valued positively by evaluators as a decision-making tool.

Pompe disease (PD) is a rare, progressive neuromuscular disease that severely affects motor and respiratory functions. Late onset PD (LOPD) is the most common phenotype. Current treatment involves enzyme replacement therapy (ERT) with alglucosidase alfa, which was first approved in 2006. In Europe, the treatment landscape is changing as two new ERTs have been filed for regulatory approval: avalglucosidase alfa and cipaglucosidase alfa plus miglustat. We analyzed how health technology assessment (HTA) and reimbursement criteria may be applied to ERTs in key countries for patients with PD.

Eighteen different factors were identified from the pivotal trials (LOTS, COMET, and PROPEL) for the three recombinant enzymes. These covered the categories of trial design, endpoints, quality of life, and other product characteristics. Twenty-six HTA experts and health economists from Denmark, England, France, Germany, Italy, the Netherlands, Spain, and Sweden with rare disease experience were interviewed during the period from July to September 2021. In structured discussions, each participant was asked to rate (from one to seven) the factors in terms of their importance and impact on the HTA evaluation and reimbursement of treatments for adults with PD.

The following factors were highly rated: a well-defined PD trial population; use of an active trial comparator; efficacy in both treatment naïve and experienced subpopulations; a superiority study design; and payer-relevant endpoints and quality of life improvements. The five lowest rated factors were open-label data, biomarkers, innovation, ease of administration, and mode of action. While the results were mostly consistent across countries, the HTA expert viewpoints varied depending on the country. For example, HTA experts in Italy, the Netherlands, and Sweden rated innovation and biomarkers more highly than German experts.

As new ERTs become licensed, achieving reimbursement and successful HTA of them will require a clear exposition of payer-relevant evidence for the LOPD population in the target country, including comparative randomized controlled trial data, benefits for experienced and treatment naïve subgroups, and payer-relevant endpoints and quality of life gains.

Disinvestment of ineffective, low value technologies is growing as a priority for international health policy in order to improve quality and maximize value in health care. Different strategies have been implemented at the international and national level using various methods of evidence gathering and technical assessment. However, the success of these initiatives is mixed, with fewer than half of the empirical studies reporting reductions in the use of low value services.

This review explored the role of stakeholders in the disinvestment process by describing the initiatives and analyzing the methods used for reassessment. We also identified the facilitators and barriers related to disinvestment implementation.

This scoping review was guided by the JBI Manual for Evidence Synthesis and the PRISMA statement for scoping reviews. Strategic literature searches were performed to identify published reviews on disinvestment in health care using the MEDLINE, Web of Science, and Scopus databases. Data were extracted using a predesigned form and then synthesized narratively to identify similarities and differences across the approaches according to prespecified domains.

Sixteen reviews were included. We identified various disinvestment initiatives across 16 countries, with a minimum of 34 initiatives at different levels of implementation and with various agencies responsible for the activities. Two of the most used methods for facilitating disinvestment decisions were program budgeting and marginal analysis (PBMA) and health technology assessment (HTA). Stakeholder involvement was the most important aspect to be addressed since it acts as both a facilitator and a barrier in implementing disinvestment initiatives. Meaningful engagement may be strengthened with continuous stakeholder participation, transparency in methods and processes, and ongoing knowledge transfer.

This scoping review highlights the role of stakeholder involvement in disinvestment, which is a double-edged sword because it both facilitates and hinders disinvestment implementation. The most common methods for assessing candidates for disinvestment are PBMA and HTA, but there is a lack of clarity on which HTA dimension is suitable for a disinvestment process.

Within Dutch health care, Zorginstituut Nederland regularly selects topics for reassessment of diagnostic and therapeutic interventions. Until now, about 15 topics have been investigated, such as hip/knee arthrosis, stable angina, low back pain, urinary incontinence, palliative care, retinopathy and asthma.

For each topic, stakeholders were asked to report what could be improved. Also a working group was created to analyze: what do guidelines recommend, what does actual care look like seen from administrative data, is there a gap between guidelines and delivered care, what can be improved? A report with recommendations was written with a calculation of the impact. Agreements were made on implementation. This report focused on findings relating to asthma.

We identified the multiple issues in the management of asthma: (i) Only 11 percent of patients had their diagnosis confirmed (with spirometry and reversibility or visit to pulmonologist). (ii) 60,000 patients had intensive short acting broncho-adrenegic agents (SABA) without inhaled corticosteroids (ICS) representing overuse of SABA. (iii) 200,000 patients use inhalers that can be empty, without the patient knowing this, as there is no indicator showing the number of actuation/puffs left, which leads to under treatment. (iv) 60.000 patients have aerosol and powder inhalers together, each requiring a different technique, leading to mistakes. (v) 67 percent of asthma adults get a chest x-ray on referral to hospital, which is a high proportion. (vi) 49 percent of patients use inhalers with propellant, which is needlessly high, given their undesirable impact on climate change. This causes 36 million kg co2 equivalent, the same as 36000 extra gasoline cars. (vii) Only 37 percent of patients receive yearly monitoring. We calculated that EUR 14 million annually can be saved as a result of better diagnosis leading to less overdiagnosis and overtreatment and less spacers.

Despite that ‘HTA (Health Technology Assessment) at the doorstep’ has been applied for asthma interventions, we noticed considerable room for improvement. We consider this method important for real HTA life cycle approach.

The Spanish Ministry of Health asked us about the efficiency of extending the current rotavirus vaccination strategy to all newborns. The current strategy is to vaccinate only to high-risk newborns (premature and those qualified as high-risk by a pediatrician). The objective of this research was to compare three strategies: no-vaccination, vaccination of high-risk newborns and universal vaccination, considering the two vaccines available in Spain: RotaTeq® and Rotarix®.

A cost-utility analysis, based on a de novo Markov model, was carried out both from a societal and a healthcare system perspective. The model follows a cohort of newborns during their life-course. The cycle length is annual and a half-cycle correction was applied. A discount rate of 3 percent was applied in the base case both to costs and utilities. Most of the incidence, probabilities and costs data were Spanish. The Quality Adjusted Life Year (QALY) data were taken from international literature. We assumed a willingness to pay threshold of EUR 25,000 per QALY gained. We performed deterministic one-way sensitivity analysis.

Compared to no-vaccination, the high-risk vaccination strategy is cost-effective assuming the above- mentioned threshold only with Rotarix® from a societal perspective (RotaTeq® EUR 32,008 per QALY; Rotarix® EUR 23,368 per QALY). Universal vaccination is not cost-effective either compared to no-vaccination or compared to the high-risk vaccination strategy and with both perspectives. Vaccine prices and efficacy data are highly sensitive variables. We find that universal vaccination would be cost-effective with a discount of 44.6 and 36.9 percent of the current price of RotaTeq® and Rotarix®, respectively.

Universal vaccination would not be a cost-effective strategy for Spain with either of the two vaccines at current prices. Vaccination of high-risk newborns would be cost-effective at current prices and from a societal perspective only with Rotarix®. Substantial vaccines price reductions could make the universal vaccination a cost-effective option in Spain.

Improving the value of healthcare delivered requires accurate cost information, which can be challenging for rare diseases. We report direct costs of patients with OI using the TDABC methodology.

This research is part of a nationwide observational study to assess the “Value of Healthcare Journey for Patients With Rare Diseases” in SUS. Patient journey and economic data was collected for the actual clinical practice in each medical center enrolled in the project. We set the starting point of disease and map a patient’s path in the healthcare journey, including treatment, exams, procedures and appointments needed. Unit cost, time and amount of resources to perform each activity, such as human resources, materials and medicines, was assessed, disregarding indirect costs. Here we present preliminary results for OI. We considered medical centers from different regions of Brazil. The results are presented in terms of percentage and/or mean and its standard deviation (SD).

Three medical centers completed the data collection. The average [SD] cost of a one year journey of a patient diagnosed with OI is BRL 16,308.07 [11,005.21] (USD 2,886.91 [1,948.36]) per center. Activities with greater cost are medicines, with an average cost of BRL 11,919.47 [12,629.45] (USD 2,109.76 [2,235.52]), followed by materials and human resources, with an average cost of BRL 2,881.91 [3,311.57] (USD 509.92 [585.84]) and BRL 1,506.70 [1,300.46] (USD 266.54 [230.24]), respectively. When assessing the moment of a patient’s journey, the percentage of appointments, diagnosis, treatments and follow-up were 11.2, 25.8, 32.5 and 30.5, respectively. Only 3.3 percent of consumed resources were external to the center (out-of-pocket or private insurance).

The TDABC can efficiently draw the processes and costs associated with it. Medicines are the main driver of annual costs for OI patients in the SUS. This study was funded by the National Council for Scientific and Technological Development – CNPq and the Ministry of Health of Brazil – MoH.

Measuring the performance of the health systems is an important challenge at international level. The main objective of this work is to analyze the outcomes of the Kazakhstan Health Care System in order to establish the main causes of avoidable mortality in the country. Also, to identify benchmarking possibilities that may support public policy decisions to improve the results.

To calculate the avoidable mortality indicators due to preventable and treatable causes, the methodology agreed by the OECD and Eurostat based on the International Classification of Diseases, ICD-10 was applied. Starting from the mortality database of the World Health Organization, the standardized indicators of avoidable mortality was calculated for those countries that had available data based on this classification. Based on the outcomes obtained, a “Two-Step” Cluster Analysis was used to identify and characterize the different clusters of countries that present similar results to identify possible affinities and detect benchmarking possibilities.

The main causes of mortality from treatable diseases in Kazakhstan are those related to the circulatory system, followed by different types of cancer and respiratory diseases.

Applying the cluster analysis in the international context, we find important differences between the different clusters, both in the standardized ratios of avoidable mortality and in its causes. Notable differences have also been identified between Kazakhstan and the countries that make up its cluster. Overall, Kazakhstan presents better avoidable mortality results, both from preventable and treatable causes, than the average of the cluster to which it belongs. However, in some causes of death, it presents worse results and high mortality rates, as in the case of those related to the circulatory and respiratory systems or different types of injuries.

The cluster analysis based on the avoidable mortality indicators reveals different conglomerates of countries that show important similarities between them and also some significant differences. Groups of avoidable diseases that characterize each cluster and subcluster, provide key information for the benchmarking and the design of future actions.

Involving patients in the health technology assessment (HTA) lifecycle is a core principle at the National Institute for Health and Care Excellence (NICE). We include both patient organizations and patient experts, which helps build public confidence in healthcare decision-making. We continually work with patient experts to improve their experience and ability to participate by seeking patient expert feedback after every committee meeting.

We sent patient experts an anonymous experience survey containing a five-point Likert scale and open text boxes to capture qualitative data. The survey covered their overall experience, interaction with the committee Chair, and the support they received from both NICE and the Public Involvement Programme (PIP).

In the 2019 to 2020 period we sent out 59 questionnaires and received 29 responses (47%), all of which were from medicines HTA committee participants. In the 2020 to 2021 period we sent out 120 questionnaires and received 65 responses (54%), of which 64 were from patient experts who attended medicines HTAs and one was from a medical devices HTA committee participant.

Good or excellent experiences were reported by 90 percent of patient experts. The four main success factors noted were: good support before meetings; being welcomed and respected; well organized meetings; and patient expert input being valued. Areas for further improvement included: providing better briefing before meetings; allowing more time to review documents; providing more technical support; and giving more consideration to the opinions of patient experts.

As a result of the feedback received, the PIP now holds monthly group briefing meetings for patient experts. We also publish the anonymized feedback from the patient experts quarterly in a newsletter for committee members and share the data with internal NICE teams. Additionally, NICE aims to: send committee papers out earlier; have the option of holding a technical engagement call before committee meetings; and develop a feedback mechanism to ascertain the impact of patient input.

Reimbursement decision making is based on a relative effectiveness assessment (REA), which may be combined with a cost-effectiveness assessment, by national Health Technology Assessment (HTA) agencies. These assessments are based on clinical data where new interventions are compared to the current standard of care, which may differ between countries. Since most pivotal trials only include a limited number of interventions, indirect treatment comparisons (ITCs) can be used to compare multiple interventions. The aim of this study was to evaluate the use of ITCs in HTA decision making in the Netherlands and England.

All pharmaceutical assessments published between 2015 and 2019 by the National Health Care Institute (ZIN) and the National Institute for Health and Care Excellence (NICE) were reviewed to determine whether an ITC had been used. For detailed analysis we included all assessments of ZIN using an ITC, and a random sample of assessments of NICE using an ITC (10 assessments per publication year).

Between 2015 and 2019 a total of 106 and 265 assessments were conducted by ZIN and NICE, respectively. Of these assessments 48 from ZIN and 150 from NICE included an ITC. The detailed analysis showed that pharmaceutical assessments including indirect comparative evidence led to the REA conclusion of similar therapeutic evidence in 57 percent of 48 assessments by ZIN and in 52 percent of 50 assessments by NICE. Reimbursement recommendations including indirect comparative evidence most often resulted in positive recommendations by ZIN (57% assessments), and in restricted recommendations by NICE (50% assessments). Different methods were employed to incorporate indirect comparative evidence, such as naïve ITCs and network meta-analysis.

Our results showed a significant variability in the use of ITCs between NICE and ZIN, which may contribute to differences in their recommendations. Further analysis will provide deeper insight in these differences and may provide suggestions for a clearer international guidance on the use of ITCs for HTA.

In 2018 the Dutch Ministry of Health (MoH) introduced a new policy regarding the conditional reimbursement (CR) of drugs in the basic health insurance package. This new policy offers patients with a serious, often rare disease for which no effective treatment is yet available, the possibility of obtaining controlled access to new promising drugs. In the meantime, additional data on (cost-) effectiveness is being collected. The aim was to assess whether this new policy allowed improved inclusion of drugs in the basic health insurance package.

Marketing authorization holders (MAH) were able to apply for the CR. The drug had to be registered by the European Medicines Agency (EMA) as an orphan drug, conditional or exceptional and address an unmet medical need. The MAH had to submit a dossier which includes a study protocol together with the professionals’ associations, patients’ associations and a research institute. It was possible to engage an ongoing (international) study in the CR application. Based on the proposed study, the National Health Care Institute (ZIN) assessed whether it is possible to determine if the drug should be reimbursed at the end of the CR period. A reduced price was a condition for CR.

Four drugs are currently reimbursed as part of the CR, being: parathyroid hormone, ataluren, larotrectinib and entrectinib. The proposed studies are ongoing and will generate data to support the final reimbursement decision. Progress will be monitored by the researchers and discussed with ZIN.

Four drugs were successfully conditionally reimbursed, concluding the new CR procedure is feasible. Additional data is being collected to aid in the decision on the definitive reimbursement of these drugs. The upcoming period, the focus will be on the quality of the collected data and whether the inclusion of patients is proceeding as planned. The MoH will be informed by ZIN on the study progress annually. The final reimbursement decision is taken at the end of the CR period.