ADL-ability is the most important predictor of societal costs of care in community dwellings irrespective of country and should therefore be central in the economic evaluation of Alzheimer's disease therapies.

In Europe, there were almost twice as many patents granted for medical technology (13,795) compared to pharmaceuticals (7441) in 2018. It is important to ask how to integrate such an amount of innovations into routine clinical practice and how to measure the value it brings to the healthcare system. Given the novelty of digital health interventions (DHI), one can even question whether the quality-adjusted life years (QALY) approach developed for pharmaceuticals can be used or whether we need to develop a new DHI’s value assessment framework. We conducted a systematic literature review of published DHIs’ assessment guidelines. Each publication was analyzed with a 12-items checklist based on a EUnetHTA core model enriched with additional criteria such as usability, interoperability, and data security. In total, 11 value assessment guidelines were identified. The review revealed that safety, clinical effectiveness, usability, economic aspects, and interoperability were most often discussed (seven out of 11). More than half of the guidelines addressed organizational impact, data security, choice of comparator, and technical considerations (six out of 11). The unmet medical needs (three out of 11), along with the ethical (two out of 11) and legal aspects (one out of 11), were given the least attention. No author provided an analytical framework for the calculation of clinical and economic outcomes. We elicited five recommendations for the choice of DHI’s value criteria and a methodological suggestion for the pricing and reimbursement framework. Our conclusions lead to the need for a new DHI’s value assessment framework instead of a QALY approach.

BackgroundThe objective of this study was to assess the potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement (P&R) process with regard to orphan drugs.MethodsA four step approach was designed. Firstly, a systematic literature review was conducted to select the MCDA criteria. Secondly, a database of orphan drugs was established. Thirdly, health technology appraisals (HTA recommendations) were categorized and an MCDA appraisal was conducted. Finally, a comparison of HTA and MCDA outcomes was carried out. An MCDA outcome was considered positive if more than 50 % of the maximum number of points was reached (base case). In the sensitivity analysis, 25 % and 75 % thresholds were tested as well.ResultsOut of 2242 publications, 23 full-text articles were included. The final MCDA tool consisted of ten criteria. In total, 27 distinctive drug-indication pairs regarding 21 drugs were used for the study. Six negative and 21 positive HTA recommendations were issued. In the base case, there were 19 positive MCDA outcomes. Of the 27 cases, there were 12 disagreements between the HTA and MCDA outcomes, the majority of which related to positive HTA guidance for negative MCDA outcomes. All drug-indication pairs with negative HTA recommendations were appraised positively in the MCDA framework. Economic details were available for 12 cases, of which there were 9 positive MCDA outcomes. Amongst the 12 drug-indication pairs, two were negatively appraised in the HTA process, with positive MCDA guidance, and two were appraised in the opposite direction.ConclusionsAn MCDA approach may lead to different P&R outcomes compared to a standard HTA process. On the one hand, enrichment of the list of decision making criteria means further scrutiny of a given health technology and as such increases the odds of a negative P&R outcome. On the other hand, it may uncover additional values and as such increase the odds of positive P&R outcomes.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-016-0388-0) contains supplementary material, which is available to authorized users.

This cost-utility analysis showed vortioxetine to be a good alternative for MDD patients switching therapy in Finland.

BackgroundThere are positive and negative consequences of the implementation of out of pocket (OOP) payments as a source of the healthcare financing. On the one hand, OOP burden increases awareness of treatment costs and limits unnecessary use of healthcare services. On the other hand, it may prevent the sick from accessing needed care. Consequently there are several aspects that ought to be taken into consideration while defining the optimal structure of OOP payments. The objective of this study was twofold. Firstly, it was to understand what actions are taken to decrease the OOP burden. Secondly, it was to address the question whether the implementation of any form of formal OOP payments may impact negatively upon fairness in financial contribution.MethodsThe literature search was conducted using the Pubmed, Embase, Cochrane Library and Center of Review and Dissemination databases. Only studies which measured the Kakwani index of progressivity in at least two time points were included. Articles written in English published between January 2004 and September 2015 were searched. No geographical restriction was imposed. An increment of more than 0.10 in the Kakwani index was considered as a significant health policy impact.ResultsIn total 16 publications were included, of which nine studied attempts to decrease the OOP burden, four described the consequences of the introduction of formal fees, and three covered both topics. Overall, a significant health policy impact was noted in four cases. All of them related to a reduction in the OOP burden, with three and one noting a change towards the progressivity and regressivity of direct healthcare payments respectively. Among jurisdictions which introduced formal fees, none study noted a significant impact on the regressivity of OOP spendings.ConclusionsIn the majority of cases, a health policy impact on the distribution of OOP health payments was insignificant. The reduction of OOP burden cannot be achieved successfully without adequate extension of healthcare coverage or engagement of other sources of healthcare financing. When formal fees are being introduced, protection against catastrophic healthcare payments is needed for the most vulnerable groups.

As budget constraints become more and more visible, there is growing recognition for greater transparency and greater stakeholders' engagement in the pharmaceuticals' pric-ing&reimbursement (P&R) decision making. New frameworks of drugs' value assessments are searched for. Among them, the multi-criteria decision analysis (MCDA) receives more and more attention. In 2014, ISPOR established Task Force to provide methodological recommendations for MCDA utilization in the health care decision making. Still, there is not so much knowledge about the real life experience with MCDA's adaptation to the P&R processes. Areas covered: A systematic literature review was performed to understand the rationale for MCDA adaptation, methodology used as well as its impact on P&R outcomes. Expert commentary: In total 102 hits were found through the search of databases, out of which 18 publications were selected. Although limited in scope, the review highlighted how MCDA can im-prove the decision making processes not only regarding pricing & reimbursement but also contribute to the the risk benefit assessment as well as optimization of treatment outcomes. Still none of re-viewed studies did report how MCDA results actually impacted the real life settings.

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A total of 7 years after the addition of Poland, the Czech Republic and Hungary to the EU, the pricing and reimbursement regulations introduced in these countries are still considered to be not fully compliant with Directive 89/105/EEC, commonly referred to as the 'Transparency Directive' (TD). The TD aims to ensure the transparency of the pricing and reimbursement processes for medicinal products established by the member states. Among the most difficult barriers on the way to successful implementation of the TD discussed are meeting the timelines indicated by the TD, the implementation of objective and verifiable criteria for decisions, and the availability of remedies for negative decisions. Health technology assessment (HTA) has been introduced in to the reimbursement systems in Poland, the Czech Republic and Hungary almost simultaneously to their accession to the EU. Even though Central Eastern European (CEE) countries faced similar challenges during the transformation to a democratic system, certain differences in the extent to which HTA principles have been implemented in these countries could be distinguished. Hence, it is thought-provoking to consider the different views of HTA experts on the role of HTA in the adaptation of the TD in the CEE region. The key objective of this article will be to discuss whether the adaptation of HTA principles has supported or only triggered additional challenges in the process of successful implementation of the TD in the CEE region. In particular, the article will discuss whether the introduction of mandatory HTA recommendations or explicit willingness-to-pay threshold would encourage or discourage implementation of the TD. The importance of the independent HTA agency as a condition for successful introduction of the TD will also be debated.