Mapping Studies to Estimate Health-State Utilities From Nonpreference-Based Outcome Measures: A Systematic Review on How Repeated Measurements are Taken Into Account.

OBJECTIVES: Mapping algorithms are developed using data sets containing patient responses to a preference-based questionnaire and another health-related quality-of-life questionnaire. When data sets include repeated measurements from the same individuals over time, the assumption of observations' independence, required by standard models, is violated, and standard errors are underestimated. This review aimed to identify how studies deal with methodological challenges of repeated measurements, provide an overview of practice to date, and potential implications for future work. METHODS: We conducted a systematic literature search of MEDLINE, Cumulative Index to Nursing and Allied Health Literature, specialized databases, and previous systematic reviews. A data template was used to extract, among others, start and target instruments if the data set(s) used for estimation and validation had repeated measurements per patient, used regression techniques, and which (if any) adjustments were made for repeated measurements. RESULTS: We identified 278 publications developing at least 1 mapping algorithm. Of the 278 publications, 121 used a data set with repeated measurements, among which 92 used multiple time points for estimation, and 39 selected specific time points to have 1 observation per participant. A total of 36 studies did not account for repeated measurements. An adjustment was conducted using cluster-robust standard errors (21), random-effects models (30), generalized estimating equations (7), and other methods (7). CONCLUSIONS: The inconsistent use of methods to account for interdependent observations in the literature indicates that mapping guidelines should include recommendations on how to deal with repeated measurements, and journals should update their guidelines accordingly.

How did the COVID-19 pandemic affect inpatient care for children in Germany? An exploratory analysis based on national hospital discharge data.

BACKGROUND: The delivery of health services around the world faced considerable disruptions during the COVID-19 pandemic. While this has been discussed for a number of conditions in the adult population, related patterns have been studied less for children. In light of the detrimental effects of the pandemic, particularly for children and young people under the age of 18, it is pivotal to explore this issue further. METHODS: Based on complete national hospital discharge data available via the German National Institute for the Reimbursement of Hospitals (InEK) data browser, we compare the top 30 diagnoses for which children were hospitalised in 2019, 2020, 2021 and 2022. We analyse the development of monthly admissions between January 2019 and December 2022 for three tracers of variable time-sensitivity: acute lymphoblastic leukaemia (ALL), appendicitis/appendectomy and tonsillectomy/adenoidectomy. RESULTS: Compared to 2019, total admissions were approximately 20% lower in 2020 and 2021, and 13% lower in 2022. The composition of the most frequent principal diagnoses remained similar across years, although changes in rank were observed. Decreases were observed in 2020 for respiratory and gastrointestinal infections, with cases increasing again in 2021. The number of ALL admissions showed an upward trend and a periodicity prima vista unrelated to pandemic factors. Appendicitis admissions decreased by about 9% in 2020 and a further 8% in 2021 and 4% in 2022, while tonsillectomies/adenoidectomies decreased by more than 40% in 2020 and a further 32% in 2021 before increasing in 2022; for these tracers, monthly changes are in line with pandemic waves. CONCLUSIONS: Hospital care for critical and urgent conditions among patients under the age of 18 was largely upheld in Germany during the COVID-19 pandemic, potentially at the expense of elective treatments. There is an alignment between observed variations in hospitalisations and pandemic mitigation measures, possibly also reflecting changes in demand. This study highlights the need for comprehensive, intersectoral data that would be necessary to better understand changing demand, unmet need/foregone care and shifts from inpatient to outpatient care, as well as their link to patient outcomes and health care efficiency.

Utilization of innovative medical technologies in German inpatient care: does evidence matter?

BACKGROUND: The reimbursement of new technologies in inpatient care is not always linked to a requirement for evidence-based evaluation of patient benefit. In Germany, every new technology approved for market was until recently eligible for reimbursement in inpatient care unless explicitly excluded. The aim of this work was (1) to investigate the type of evidence that was available at the time of introduction of 25 innovative technologies and how this evidence evolved over time, and (2) to explore the relationship between clinical evidence and utilization for these technologies in German inpatient care. METHODS: This study combined different methods. A systematic search for evidence published between 2003 and 2017 was conducted in four bibliographic databases, clinical trial registries, resources for clinical guidelines, and health technology assessment-databases. Information was also collected on funding mechanisms and safety notices. Utilization was measured by hospital procedures captured in claims data. The body of evidence, funding and safety notices per technology were analyzed descriptively. The relationship between utilization and evidence was explored empirically using a multilevel regression analysis. RESULTS: The number of included publications per technology ranges from two to 498. For all technologies, non-comparative studies form the bulk of the evidence. The number of randomized controlled clinical trials per technology ranges from zero to 19. Some technologies were utilized for several years without an adequate evidence base. A relationship between evidence and utilization could be shown for several but not all technologies. CONCLUSIONS: This study reveals a mixed picture regarding the evidence available for new technologies, and the relationship between the development of evidence and the use of technologies over time. Although the influence of funding and safety notices requires further investigation, these results re-emphasize the need for strengthening market approval standards and HTA pathways as well as approaches such as coverage with evidence development.

Health care patterns and policies in 18 European countries during the first wave of the COVID-19 pandemic: an observational study.

BACKGROUND: The coronavirus disease 2019 (COVID-19) pandemic has developed into an unprecedented global challenge. Differences between countries in testing strategies, hospitalization protocols as well as ensuring and managing ICU capacities can illustrate initial responses to a major health system shock, and steer future preparedness activities. METHODS: Publicly available daily data for 18 European countries were retrieved manually from official sources and documented in an Excel table (March-July 2020). The ratio of tests to cases, the share of hospitalizations out of all cases and the share of ICU admissions out of all hospitalizations were computed using 7-day rolling averages per 100 000 population. Information on country policies was collected from the COVID-19 Health System Response Monitor of the European Observatory on Health Systems and Policies. Information on health care capacities, expenditure and utilization was extracted from the Eurostat health database. RESULTS: There was substantial variation across countries for all studied variables. In all countries, the ratio of tests to cases increased over time, albeit to varying degrees, while the shares of hospitalizations and ICU admissions stabilized, reflecting the evolution of testing strategies and the adaptation of COVID-19 health care delivery pathways, respectively. Health care patterns for COVID-19 at the outset of the pandemic did not necessarily follow the usual health service delivery pattern of each health system. CONCLUSIONS: This study enables a general understanding of how the early evolution of the pandemic influenced and was influenced by country responses and clearly demonstrates the immense potential for cross-country learning.

Reimbursement decisions for medical services in Austria: an analysis of influencing factors for the hospital individual services catalogue between 2008 and 2020.

OBJECTIVES: To (1) describe the (evidence-based) reimbursement process of hospital individual services, (2) evaluate the accordance between evidence-based recommendations and reimbursement decision of individual services and (3) elaborate potential aspects that play a role in the decision-making process in Austria. METHOD: The reimbursement process is described based on selected relevant sources such as official documents. Evidence-based recommendations and subsequent reimbursement decisions for the annual maintenance of the hospital individual service catalogue in Austria between 2008 and 2020 were analysed using a mixed methods approach, encompassing descriptive statistics and a focus group with Austrian decision makers. RESULTS: 118 evidence-based recommendations were analysed. There were 93 (78.8%) negative and 25 (21.2%) positive evidence-based recommendations. In total, 107 out of 118 evidence-based recommendations (90.1%) did not lead to a deviating reimbursement decision. We identified six aspects that may have played a role in the decision-making process for the annual maintenance of the hospital individual service catalogue, with clinical evidence being the most notable. Further aspects included quality assurance/organisational aspects (i.e., structural quality assurance), costs (if comparable to already existing medical services, not: cost-effectiveness), procedural aspects (e.g., if certain criteria for adoption have not been met formally through the proposals), "other countries" (i.e., taking into account how other countries decided) and situational aspects (such as the COVID-19 pandemic). CONCLUSIONS: There is good accordance between evidence-based recommendations and reimbursement decisions regarding hospital individual services in Austria. Beyond clinical evidence, organisational aspects seem to be considered often with regard to quality assurance but costs do not appear to play a major role. The Austrian system has mechanisms in place that can restrict widespread adoption of novel hospital individual services with uncertain clinical benefits. Future studies could investigate how well these mechanisms work and how they compare to other health systems in Europe.

Exploring variation of coverage and access to dental care for adults in 11 European countries: a vignette approach.

BACKGROUND: Oral health, coupled with rising awareness on the impact that limited dental care coverage has on oral health and general health and well-being, has received increased attention over the past few years. The purpose of the study was to compare the statutory coverage and access to dental care for adult services in 11 European countries using a vignette approach. METHODS: We used three patient vignettes to highlight the differences of the dimensions of coverage and access to dental care (coverage, cost-sharing and accessibility). The three vignettes describe typical care pathways for patients with the most common oral health conditions (caries, periodontal disease, edentulism). The vignettes were completed by health services researchers knowledgeable on dental care, dentists, or teams consisting of a health systems expert working together with dental specialists. RESULTS: Completed vignettes were received from 11 countries: Bulgaria, Estonia, France, Germany, Republic of Ireland (Ireland), Lithuania, the Netherlands, Poland, Portugal, Slovakia and Sweden. While emergency dental care, tooth extraction and restorative care for acute pain due to carious lesions are covered in most responding countries, root canal treatment, periodontal care and prosthetic restoration often require cost-sharing or are entirely excluded from the benefit basket. Regular dental visits are also limited to one visit per year in many countries. Beyond financial barriers due to out-of-pocket payments, patients may experience very different physical barriers to accessing dental care. The limited availability of contracted dentists (especially in rural areas) and the unequal distribution and lack of specialised dentists are major access barriers to public dental care. CONCLUSIONS: According to the results, statutory coverage of dental care varies across European countries, while access barriers are largely similar. Many dental services require substantial cost-sharing in most countries, leading to high out-of-pocket spending. Socioeconomic status is thus a main determinant for access to dental care, but other factors such as geography, age and comorbidities can also inhibit access and affect outcomes. Moreover, coverage in most oral health systems is targeted at treatment and less at preventative oral health care.

Portable continuous wave Doppler ultrasound for primary healthcare in South Africa: can the EUnetHTA Core Model guide evaluation before technology adoption?

BACKGROUND: This study had a threefold aim: to test the value of stakeholder involvement in HTA to reduce evidence gaps and interpret findings; and to assess a medical device by applying the EUnetHTA Core Model (CM) in South Africa and thus ultimately provide a first overview of evidence for potential widespread adoption of the technology in a primary health care (PHC) setting. Used in primary healthcare setting for obstetric use, the technology under assessment is a low-cost continuous wave Doppler ultrasound (DUS). METHODS: The scoping of the assessment was defined by involving policy makers in selecting the domains and corresponding questions relevant to the ultrasound and its use. Additionally, hospital managers were invited to respond to dichotomous questions on the criteria for procurement. To substantiate evidence obtained from an initial literature review, different stakeholders were identified and consulted. The evidence generated fromall steps was used to populate the high-ranked assessment elements of the CM. RESULTS: The HTA on continuous-wave DUS incorporated the evidence on organizational, ethical, and social value of its use together with effectiveness, safety, and cost-effectiveness of the technology. The domains on "health problem" and "safety" had a higher rank than the rest of the nine domains. Unexplained fetal mortality is the largest single contributor to perinatal deaths in South Africa. Pregnant women in PHC setting were examined using a continuous-wave DUS, after their routine antenatal visit. The healthcare professionals interviewed, indicated the benefit in the use of continuous-wave DUS in the PHC setting and the need for training. CONCLUSIONS: Collection and generation of evidence based on the HTA CM and the chosen decision criteria provided a generalized but structured guidance on the methodology. Several questions were not applicable for the technology and the context of its use and elimination of those that are inappropriate for the African context, resulted in a pragmatic solution. Engaging and consulting local stakeholders was imperative to understand the context, reduce evidence gaps, and address the uncertainties in the evidence, ultimately paving the way for technology adoption. Given the ongoing studies and the evolving evidence base, the potential of this technology should be reassessed.

Regulating the health workforce in Europe: implications of the COVID-19 pandemic.

In the European free movement zone, various mechanisms aim to harmonize how the competence of physicians and nurses is developed and maintained to facilitate the cross-country movement of professionals. This commentary addresses these mechanisms and discusses their implications during the COVID-19 pandemic, drawing lessons for future policy. It argues that EU-wide regulatory mechanisms should be reviewed to ensure that they provide an adequate foundation for determining competence and enabling health workforce flexibility during health system shocks. Currently, EU regulation focuses on the automatic recognition of the primary education of physicians and nurses. New, flexible mechanisms should be developed for specializations, such as intensive or emergency care. Documenting new skills, such as the ones acquired during rapid training in the pandemic, in a manner that is comparable across countries should be explored, both for usual practice and in light of outbreak preparedness. Initiatives to strengthen continuing education and professional development should be supported further. Funding under the EU4Health programme should be dedicated to this endeavour, along with revisiting the scope of necessary skills following the experience of COVID-19. Mechanisms for cross-country sharing of information on violations of good practice standards should be maintained and strengthened to enable agile reactions when the need for professional mobility becomes urgent.

Reference Pricing in Germany: Implications for U.S. Pharmaceutical Purchasing.

Issue: The German health care system resembles that of the United States in important ways ­ it is financed by multiple private payers and relies principally on negotiation rather than regulation to establish prices. New drugs that offer minimal benefits compared with existing alternatives within a therapeutic class are subject to reference pricing; those with incremental benefits are subject to price negotiations. Together, the reference and negotiated pricing systems have held German prices substantially below U.S. equivalents. Goal: To describe the German reference-pricing system and compare it to tiered formularies and consumer cost-sharing in the United States. Methods: Document review and interviews with leaders in payer, policy, and pharmaceutical industry organizations in Germany. Key Findings and Conclusions: The German pharmaceutical pricing system uses modest levels of consumer cost-sharing to influence consumers' choices for drugs with therapeutically equivalent alternatives. Manufacturers are free to set the prices of their products, but insurers will not pay more for a new drug than for its comparators unless it offers an additional clinical benefit. For drugs covered by reference pricing, the insurers' payment maximum is set at a level that ensures sufficient choices of low-priced options. These models offer an alternative to the U.S. system of tiered formularies.

Scientific Evidence in Health Technology Assessment Reports: An In-Depth Analysis of European Assessments on High-Risk Medical Devices.

BACKGROUND: The aim of this study was to examine the scientific evidence on clinical effectiveness and safety used in health technology assessments (HTAs) of high-risk medical devices (MDs) in Europe. METHODS: We applied a systematic approach to identify European institutions involved in HTA and to select reports assessing MDs considered high-risk according to the definition in the new German health care regulation §137h. Reports published between 2010 and 2015 were considered in our subsequent analysis. We used a structured tool based on widely accepted methodologic principles from Drummond's framework to extract key information on the clinical evidence considered in the reports. RESULTS: Out of 1376 identified reports, 93 were eligible for analysis. All reports based their assessment primarily on direct evidence, in most cases (68%) identified through an independent systematic literature search. In more than half the identified studies considered in the reports, clinical evidence for demonstration of effectiveness and safety was of moderate or low quality. Even when systematic reviews and randomized controlled trials were available for assessment, most studies showed an unclear or high risk of bias. CONCLUSIONS: This study confirms that the quality of scientific evidence used in HTA of high-risk MDs is low and therefore the use of evidence needs improvement. The European Commission recently updated the regulation on MDs but mainly focused on the safety of materials and the CE (Conformité Européene [European Conformity]) mark. Our results show that additional changes are necessary, specifically with regard to the marketing authorization process of MDs, with stricter quality requirements based on methodologically robust trials, possibly in combination with other evidence sources.

HEALTH TECHNOLOGY ASSESSMENT OF MEDICAL DEVICES IN EUROPE: PROCESSES, PRACTICES, AND METHODS.

OBJECTIVES: To review and compare current Health Technology Assessment (HTA) activities for medical devices (MDs) across European HTA institutions. METHODS: A comprehensive approach was adopted to identify institutions involved in HTA in European countries. We systematically searched institutional Web sites and other online sources by using a structured tool to extract information on the role and link to decision making, structure, scope, process, methodological approach, and available HTA reports for each included institution. RESULTS: Information was obtained from eighty-four institutions, forty-seven of which were analyzed. Fifty-four methodological documents from twenty-three agencies in eighteen countries were identified. Only five agencies had separate documents for the assessment of MDs. A few agencies made separate provisions for the assessment of MDs in their general methods. The amount of publicly available HTA reports on MDs varied by device category and agency remit. CONCLUSIONS: Despite growing consensus on their importance and international initiatives, such as the EUnetHTA Core Model®, specific tools for the assessment of MDs are rarely developed and implemented at the national level. Separate additional signposts incorporated in existing general methods guides may be sufficient for the evaluation of MDs.

Published and unpublished evidence in coverage decision-making for pharmaceuticals in Europe: existing approaches and way forward.

BACKGROUND: Dissemination bias occurs when only some results emerging from clinical research reach their intended audience in the knowledge translation process. Given that coverage decisions increasingly rely on evidence, it is important to explore the types of evidence considered. This paper aimed to examine the evidence base used by regulatory institutions involved in pricing and reimbursement of pharmaceuticals in a broad range of European countries, as well as their awareness of and approach towards dissemination bias. METHODS: A mixed methods approach was adopted. Regulatory documents and published literature were identified in systematic searches and relevant documents were analysed. An online survey was carried out to verify and expand insights. RESULTS: Forty-two relevant regulatory documents and 10 publications were included. The survey had a 35% response rate, yielding valid responses for 13 countries. A fragmented impression was obtained for most countries indicating a general lack of transparency regarding both processes of decision-making and approaches towards unpublished information. Dissemination bias was rarely consistently considered. Practices for the identification and inclusion of all available evidence varied considerably, as did the influence of missing evidence on decision-making. Differences were often attributable to the regulatory context and/or institutional principles. CONCLUSIONS: Best practice is difficult to generalize given the identified variations. Individual exemplary practices support the necessity for institutional exchange at international level. Increased institutional commitment to transparency of methods and processes should be advocated.

Continuity of care in the cross-border context: insights from a survey of German patients treated abroad.

BACKGROUND: Continuity of care is important for outcomes and patient satisfaction and includes additional considerations in the context of cross-border health care. Although this has been discussed in research and was picked up in the recently transposed Directive on patients' rights (2011/24/EU), there is limited evidence about related issues actually encountered by patients crossing borders. METHODS: An anonymous postal survey was carried out by the Techniker Krankenkasse, one of the largest sickness funds in Germany. The questionnaire was sent to 45 189 individuals who had received treatment in EU/EEA countries and included items on relational, management and informational continuity. RESULTS: The survey had a response rate of 41% (n = 17 543). Of those respondents who had travelled for care (n = 3307), ∼19% (n = 570) did so due to a relationship of trust with a given provider. Only 8% of all respondents required emergency follow-up services due to complications, the majority of which was obtained back in Germany. Twelve percentage of those who were prescribed medication abroad (n = 4208) reported problems, spanning unknown products, dispensation and reimbursement. Information exchange between providers across borders was rare and largely carried out by the patients themselves. CONCLUSIONS: Although relational continuity may be important to specific groups of patients travelling for care, it is primarily informational continuity and its interrelation with management continuity that need to be addressed in the cross-border context. Information exchange should be endorsed at European level. Additional focus is required on informing patients about documentation rights and requirements and providing health records that are comprehensive and comprehensible.

CONSIDERING EQUITY IN HEALTH TECHNOLOGY ASSESSMENT: AN EXPLORATORY ANALYSIS OF AGENCY PRACTICES.

OBJECTIVES: Equity is one of the founding principles in most healthcare systems. Financial constraints entail an increased risk of exacerbating inequities and a greater need for evidence-based decisions. It is, therefore, both important and timely to enquire how equity can be addressed in health technology assessment (HTA) practice. We aimed to explore related practices from a broad range of HTA agencies, identify exemplary approaches and common concerns, and offer insights for future considerations. METHODS: HTA agencies for which both methodological guides and HTA reports were publicly available were selected from an initial comprehensive pool. Information was extracted on issues ranging from a general commitment to fairness to specific measures targeting both methodological and process-related elements. RESULTS: Methodological documents and ninety-eight reports from nineteen agencies were analyzed. Our findings indicate that equity was not a standard consideration in HTA report production. The nature of specific approaches and the amount of resources invested into including an equity perspective varied considerably. Specific measures (e.g., appropriate information sources, analytical tools, and schemes) were mentioned by almost half of the agencies analyzed. Albeit sporadic, both horizontal and vertical equity considerations were identified in included HTA reports. CONCLUSIONS: While varying legal contexts and institutional principles can lead to different interpretations of equity at the decision point, a combination of methodological and process-related practices could contribute to more equity-sensitive evaluations, especially in conjunction with enhanced dissemination of existing methodological tools. Networking initiatives on behalf of existing collaborating platforms could play an important role in this direction.

TAXONOMY OF MEDICAL DEVICES IN THE LOGIC OF HEALTH TECHNOLOGY ASSESSMENT.

OBJECTIVES: The suitability of general HTA methodology for medical devices is gaining interest as a topic of scientific discourse. Given the broad range of medical devices, there might be differences between groups of devices that impact both the necessity and the methods of their assessment. Our aim is to develop a taxonomy that provides researchers and policy makers with an orientation tool on how to approach the assessment of different types of medical devices. METHODS: Several classifications for medical devices based on varying rationales for different regulatory and reporting purposes were analyzed in detail to develop a comprehensive taxonomic model. RESULTS: The taxonomy is based on relevant aspects of existing classification schemes incorporating elements of risk and functionality. Its 9 × 6 matrix distinguishes between the diagnostic or therapeutic nature of devices and considers whether the medical device is directly used by patients, constitutes part of a specific procedure, or can be used for a variety of procedures. We considered the relevance of different device categories in regard to HTA to be considerably variable, ranging from high to low. CONCLUSIONS: Existing medical device classifications cannot be used for HTA as they are based on different underlying logics. The developed taxonomy combines different device classification schemes used for different purposes. It aims at providing decision makers with a tool enabling them to consider device characteristics in detail across more than one dimension. The placement of device groups in the matrix can provide decision support on the necessity of conducting a full HTA.

From market access to patient access: overview of evidence-based approaches for the reimbursement and pricing of pharmaceuticals in 36 European countries.

BACKGROUND: Coverage decisions determining the benefit baskets of health systems have been increasingly relying on evidence regarding patient benefit and costs. Relevant structures, methodologies, and processes have especially been established for pharmaceuticals but approaches differ. The objective of this work was thus to identify institutions in a broad range of European countries (n = 36) in charge of determining the value of pharmaceuticals for pricing and reimbursement purposes and to map their decision-making process; to examine the different approaches and consider national and supranational possibilities for best practice. METHODS: Institutions were identified through websites of international networks, ministries, and published literature. Details on institutional practices were supplemented with information from institution websites and linked online sources. RESULTS: The type and extent of information available varied considerably across countries. Different types of public regulatory bodies are involved in pharmaceutical coverage decisions, assuming a range of responsibilities. As a rule, the assessment of scientific evidence is kept structurally separate from its appraisal. Recommendations on value are uniformly issued by specific committees within or commissioned by responsible institutions; these institutions often also act as decision-makers on reimbursement status and level or market price. While effectiveness and costs are important criteria in all countries, the latter are often considered on a case-by-case basis. In all countries, manufacturer applications, including relevant evidence, are used as one of the main sources of information for the assessment. CONCLUSION: Transparency of evidence-based coverage decisions should be enhanced. International collaboration can facilitate knowledge exchange, improve efficiency of information production, and strengthen new or developing systems.

Deciding when physicians are unfit to practise: an analysis of responsibilities, policy and practice in 11 European Union member states.

In 1974, the European Economic Community established mutual recognition of medical qualifications obtained in any of its member states. Subsequently, a series of directives has elaborated on the initial provisions, with the most recent enacted in 2013. However, greater movement of physicians across borders and some high-profile scandals have raised questions about how to prevent a physician sanctioned in one country from simply moving to another, without undermining the principle of free movement. A survey of key informants in 11 European Union (EU) member states was supplemented by a review of peer-reviewed and grey literature, with the results validated by independent reviewers. It examined processes, adjudicative and disciplinary measures that are in place to evaluate physicians about whom concerns arise, and related sanctions, along with other aspects of professional standards and regulation. Overall, responses varied greatly between participating countries, with respect to the institutions responsible for the regulation of medical professions, the investigation processes in place, and the terminology used in each member state. While the types of sanction (removal from the register of medical professionals and/or licence revocation, suspension, dismissal, reprimand, warnings, fines, as well as additional education and training) applied are similar, both the roles of the individuals involved and the level of public disclosure of information vary considerably. However, some key features, such as the involvement of professional peers in disciplinary panels and the involvement of courts in criminal cases, are similar in most member states studied. Given the variation in the regulatory context, individuals and processes involved that is illustrated by our findings, a common understanding of definitions of what constitutes competence to practise, its impairment and its potential impact on patient safety becomes particularly important. Public disclosure of disciplinary outcomes is already applied by some member states, but additional measures should be considered to protect medical professionals from undue consequences.

How health technology assessment agencies address the issue of unpublished data.

OBJECTIVES: Reporting bias potentially threatens the validity of results in health technology assessment (HTA) reports. Our study aimed to explore policies and practices of HTA agencies regarding strategies to include previously unpublished data in their assessments, focusing on requests to industry for unpublished data. METHODS: We included international HTA agencies with publicly available methods papers as well as HTA reports. From the methods papers and recent reports we extracted information on requests to industry and on searches in trial registries, regulatory authority Web sites and for conference abstracts. RESULTS: Eighteen HTA agencies and seventy-three reports were included. Agencies' methods papers showed variability regarding requests to industry (requests are routinely carried out in seven cases, not mentioned in six, at the discretion of HTA authors in three, and based on manufacturer applications in two), which were reflected in the reports investigated. As reporting of requests was limited, it often remained unclear whether unpublished data had been received. Searches in trial registries, at regulatory authorities or for conference abstracts are described as a routine or optional part of the search strategy in the methods papers of 9, 11, and 8 included agencies, respectively. A total of 52 percent, 39 percent, and 16 percent of reports described searches in trial registries, at regulatory agencies, and hand searching of conference proceedings. CONCLUSION: International HTA agencies currently differ considerably in their efforts to address the issue of unpublished data. Requests to industry may constitute one strategy to access and include unpublished data, while agencies can learn from each other concerning successful practice.

Innovations in pharmaceutical policies and learnings for sustainable access to affordable medicines.

Sustainable access to affordable medicines remains a public health issue globally, including for high-income countries. To foster the debate on avenues for the future, the fifth PPRI Conference held in Vienna on 25 and 26 April 2024 will offer a forum for the debate on innovating pharmaceutical policymaking to develop and implement futureproof policy options, which are able to address current and future challenges. The Conference invites a broad audience of stakeholders, including researchers, policymakers, payers, patients, industry and health professionals. The conference topics are organised in three strands: Strand 1 on 'Local challenges, global learnings' aims to contribute to lively discussions on the implementation of pharmaceutical policies across the globe. Best-practice examples will be presented, supplemented by case studies of less effective policies which can offer rich learnings. Strand 2 on 'Strengthening the evidence base' is the place for presentations and discussions on topics such as health technology assessments, managed entry agreements and real-world data. Strand 3 'Futureproofing pharmaceutical policies' is particularly dedicated to explore innovation in policymaking to achieve sustainable access to affordable medicines.