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Background. Diabetes mellitus rates and associated costs continue to rise across Europe enhancing health authority focus on its management. The risk of complications is enhanced by poor glycaemic control, with long-acting insulin analogues developed to reduce hypoglycaemia and improve patient convenience. There are concerns though with their considerably higher costs, but moderated by reductions in complications and associated costs. Biosimilars can help further reduce costs. However, to date, price reductions for biosimilar insulin glargine appear limited. In addition, the originator company has switched promotional efforts to more concentrated patented formulations to reduce the impact of biosimilars. There are also concerns with different devices between the manufacturers. As a result, there is a need to assess current utilisation rates for insulins, especially long-acting insulin analogues and biosimilars, and the rationale for patterns seen, among multiple European countries to provide future direction. Methodology. Health authority databases are examined to assess utilisation and expenditure patterns for insulins, including biosimilar insulin glargine. Explanations for patterns seen were provided by senior-level personnel. Results. Typically increasing use of long-acting insulin analogues across Europe including both Western and Central and Eastern European countries reflects perceived patient benefits despite higher prices. However, activities by the originator company to switch patients to more concentrated insulin glargine coupled with lowering prices towards biosimilars have limited biosimilar uptake, with biosimilars not currently launched in a minority of European countries. A number of activities were identified to address this. Enhancing the attractiveness of the biosimilar insulin market is essential to encourage other biosimilar manufacturers to enter the market as more long-acting insulin analogues lose their patents to benefit all key stakeholder groups. Conclusions. There are concerns with the availability and use of insulin glargine biosimilars among European countries despite lower costs. This can be addressed., peer-reviewed

IntroductionConducting joint health technology assessments (HTA) is one of the main goals of the Beneluxa Initiative. To strengthen this collaboration, this study aimed to assess similarities and differences between past assessments of Beneluxa Initiative member countries (Austria, Belgium, Ireland and the Netherlands).MethodsA retrospective comparative analysis was performed that investigated the similarities and differences in drug assessments in the period 2016 to 2020 in (i) the number and type of assessed indications; (ii) the conclusions within assessments performed by at least two member countries; and (iii) the main arguments leading to the conclusions through a qualitative analysis of selected cases, looking into the patient population, the intervention, comparator, outcome, timing, and included evidence.ResultsThe scope of HTA differs between the countries, with Belgium and Ireland assessing most, the Netherlands focusing on drugs above a budget impact threshold and Austria on outpatient drugs. Furthermore, indications might slightly differ between countries. Therefore, only 44 (10%) of the 444 included drug-indication combinations were assessed through a full HTA by all four countries. Between any pair of countries, the overlap was higher, from 63 (Austria-the Netherlands) to 188 (Belgium-Ireland). Added benefit conclusions matched exactly in 62 to 76 percent of the indications, depending on the compared countries. In the remaining cases, often a difference of one added benefit level was observed (e.g., higher versus equal relative effect). Contradictory outcomes were very rare. Differences were observed with regards to whether a cost-effectiveness analysis was performed. When assessing the underlying arguments within the reports for nine cases with different outcomes, it became clear that organizations agree on almost all aspects, and that differences are mostly attributable to slight differences in weighing of some aspects and uncertainties.ConclusionsOverall, which indications are assessed differs, but for those indications that are assessed by multiple member countries, considerations and assessment outcomes are similar.

Performance-based managed entry agreements (PB-MEAs) might allow patient access to new medicines, but practical hurdles make competent authorities for pricing and reimbursement (CAPR) reluctant to implement PB-MEAs. We explored if the feasibility of PB-MEAs might improve by better aligning regulatory postauthorization requirements with the data generation of PB-MEAs and by active collaboration and data sharing. Reviewers from seven CAPRs provided structured assessments of the information available at the European Medicines Agency (EMA) Web site on regulatory postauthorization requirements for fifteen recently authorized products. The reviewers judged to what extent regulatory postauthorization studies could help implement PB-MEAs by addressing uncertainty gaps. Study domains assessed were: patient population, intervention, comparators, outcomes, time horizon, anticipated data quality, and anticipated robustness of analysis. Reviewers shared general comments about PB-MEAs for each product and on cooperation with other CAPRs. Reviewers rated regulatory postauthorization requirements at least partly helpful for most products and across domains except the comparator domain. One quarter of responses indicated that public information provided by the EMA was insufficient to support the implementation of PB-MEAs. Few PB-MEAs were in place for these products, but the potential for implementation of PB-MEAs or collaboration across CAPRs was seen as more favorable. Responses helped delineate a set of conditions where PB-MEAs may help reduce uncertainty. In conclusion, PB-MEAs are not a preferred option for CAPRs, but we identified conditions where PB-MEAs might be worth considering. The complexities of implementing PB-MEAs remain a hurdle, but collaboration across silos and more transparency on postauthorization studies could help overcome some barriers. ispartof: International Journal Of Technology Assessment In Health Care vol:37 issue:e83 pages:1-11 ispartof: location:England status: Published online

Introduction/Objectives: Health authorities are facing increasing challenges to the sustainability of their healthcare systems because of the growing expenditures on medicines, including new, high-priced oncology medicines, and changes in disease prevalence in their ageing populations. Medicine prices in European countries are greatly affected by the ability to negotiate reasonable prices. Concerns have been expressed that prices of patented medicines do not fall sufficiently after the introduction of lower-cost generic oncology medicines. The objective of this study was to examine the associations over time in selected European countries between the prices of oral oncology medicines, population size, and gross domestic product (GDP) before and after the introduction of generic versions. Evidence of periodic reassessments of the price, value, and place in treatment of these medicines was also looked for. The goal of this review was to stimulate debate about possible improvements in approaches to reimbursement negotiations. Methodology: Analysis was performed of reimbursed prices of three oral oncology medicines (imatinib, erlotinib and fludarabine) between 2013 and 2017 across Europe. Correlations were explored between GDP, population size, and prices. Findings were compared with previous research regarding prices of generic oral oncology medicines. Results: The prices of imatinib, erlotinib and fludarabine varied among European countries, and there was limited price erosion over time in the absence of generics. There appeared to be no correlation between population size and price, but higher prices of on-patent oral cancer medicines were seen among countries with higher GDP per capita. Conclusion: Limited price erosion for patented medicines contributed to increases in oncology medicine budgets across the region. There was also a concerning lack of evidence re-assessments of the price, value, and place in treatment of patented oncology medicines following the loss of patent protection of standard medicines. The use of such proactive re-assessments in negotiating tactics might positively impact global expenditures for oncology medicines.

The potential benefits of early patient access to new medicines in areas of high unmet medical need are recognised, but uncertainties concerning effectiveness, safety and added value when new medicines are authorised, and subsequently funded based on initial preliminary data only, have important implications. In 2016 olaratumab received accelerated conditional approval from both the European Medicines Agency and the US Food and Drug Administration for the treatment of soft-tissue sarcoma, based on the claims of a substantial reduction in the risk of death with an 11.8-month improvement in median overall survival in a phase II trial in combination with doxorubicin vs. doxorubicin alone. The failure to confirm these benefits in the post-authorisation pivotal trial has highlighted key concerns regarding early access and conditional approvals for new medicines. Concerns include potentially considerable clinical and economic costs, so that patients may have received suboptimal treatment and any money spent has foregone the opportunity to improve access to effective treatments. As a result, it seems reasonable to reconsider current marketing authorisation models and approaches. Potential pathways forward include closer collaboration between regulators, pharmaceutical companies and payers to enhance the generation of rapid and comparative confirmatory trials in a safe and fair manner, with minimal patient exposure as required to achieve robust evidence. Additionally, it may be time to review early access systems, and to explore new avenues regarding who should pay or part pay for new treatments whilst information is being collected as part of any obligations for conditional marketing authorisation. Greater co-operation between countries regarding the collection of data in routine clinical care, and further research on post-marketing data analysis and interpretation, may also contribute to improved appraisal and continued access to new innovative cancer treatments.

Zusammenfassung Ziel Ziel des Artikels ist die Darstellung rechtlicher Rahmenbedingungen sowie Erfahrungen in der grenzüberschreitenden Zusammenarbeit bei der Bewertung von Medizinprodukten als Grundlage für Erstattungsentscheidungen. Der mögliche Nutzen transnationaler Kooperation für verschiedene Stakeholdergruppen soll erläutert werden. Methodik Prozesse und Herausforderungen der abgeschlossenen European Network for Health Technology Assessment (EUnetHTA) Joint Action 2 (JA2) werden zusammengefasst und diskutiert. Die dem Artikel zugrunde liegenden Erfahrungen beruhen auf der Erstellung und Evaluierung von insgesamt 6 Rapid Assessments zu Medizinprodukten, welche während der EUnetHTA JA2 in internationaler Kollaboration verfasst und publiziert wurden. Nutzen, Ziele und Chancen der laufenden EUnetHTA Joint Action 3 (JA3) werden dargestellt. Ergebnisse Herausforderungen in der europäischen Medizinproduktebewertung umfassen die Themenauswahl und den Zeitpunkt der Bewertung, die Intransparenz des Medizinproduktemarkts sowie das Fehlen europäischer Standards zum systematischen Einbeziehen von PatientInnen. Charakteristika wie inkrementelle Veränderungen und Lernkurven machen es notwendig, Medizinprodukte über ihren gesamten Lebenszyklus hinweg zu begleiten. Der Nutzen der europäischen Zusammenarbeit für Stakeholder ist vielfältig: die aufgrund fehlender Evidenz herrschende Unsicherheit zum Zusatznutzen einer Technologie kann durch Early Dialogues reduziert werden; ein europaweit harmonisierter, transparenter Bewertungsprozess steigert die Nachvollziehbarkeit und Qualität der Berichte; die Arbeitsteilung unter den Health Technology Assessment (HTA)-Organisationen ermöglicht eine ressourcenschonende Bewertung einer größeren Anzahl an Technologien; die Patientenbeteiligung gewährleistet die Berücksichtigung patientenrelevanter Endpunkte. Die Wichtigkeit der grenzübergreifenden Zusammenarbeit im Bereich HTA zeigt sich in der Weiterführung des EUnetHTA Projekts, welches die Festigung der internationalen Kollaboration auch nach Auslaufen der EU-Förderungen zum Ziel hat. Schlussfolgerung Eine nachhaltige transnationale Kooperation in der Bewertung von Medizintechnologien kann die grenzüberschreitende Gesundheitsversorgung und die effiziente Zusammenarbeit der nationalen Gesundheitssysteme sichern. Der Fokus sollte dabei auf einer flächendeckenden Implementierung gemeinsam entwickelter Methoden und Qualitätsstandards liegen. Die europäische Zusammenarbeit kann zu einem konkreten Nutzen für PatientInnen, ÄrztInnen, HTA-Organisationen und nationale EntscheidungsträgerInnen führen.

Objectives: The regularly structured adaptation of health technology assessment (HTA) programs is of utmost importance to sustain the relevance of the products for stakeholders and to justify investment of scarce financial resources. This study describes internal adjustments and external measures taken to ensure the Horizon Scanning Programme in Oncology (HSO) is current.Methods: Formal evaluation methods comprising a survey, a download, an environmental analysis, and a Web site questionnaire were used to evaluate user satisfaction.Results: The evaluation showed that users were satisfied with HSO outputs in terms of timeliness, topics selected, and depth of information provided. Discussion of these findings with an expert panel led to changes such as an improved dissemination strategy and the introduction of an additional output, that is, the publication of a league table of emerging oncology drugs. The rather high level of international usage and the environmental analysis highlighted a considerable overlap in topics assessed and, thus, the potential for international collaboration. As a consequence, thirteen reports were jointly published based on eleven “calls for collaboration.” To further facilitate collaboration and the usability of reports for other agencies, HSO reports will be adjusted according to tools developed at a European level.Conclusions: Evaluation of the impact of HTA programs allows the tailoring of outputs to fit the needs of the target population. However, within a fast developing HTA community, estimates of impact will increasingly be determined by international collaborative efforts. Refined methods and a broader definition of impact are needed to ultimately capture the efficiency of national HTA programs.

Zusammenfassung Einleitung Zusammenarbeit anhand klar definierter Prozesse und Methoden tragt zur Reduktion von Redundanzen und damit zur europaischen Standardisierung von HTA bei. Das europaische HTA-Netzwerk EUnetHTA hat etwa mit dem HTA Core Model® ein Instrument geschaffen, das die transparente Erstellung standardisierter und qualitativ hochwertiger Assessments in internationaler Zusammenarbeit gewahrleisten soll. Methoden Erfahrungen mit bereits publizierten EUnetHTA Assessments werden ebenso beschrieben, wie Moglichkeiten der nationalen/lokalen Adaption dieser Berichte fur den eigenen Kontext. Die Integration von gemeinsam entwickelten Methoden in Routineprozesse einzelner HTA-Institute werden anhand eines ausgewahlten Beispiels beschrieben und weitere europaische Methodenprojekte uberblicksmasig dargestellt. Ergebnisse Im Rahmen von EUnetHTA Joint Action 2 (2012 - 2015) wurden bisher 4 Rapid Assessments in europaischer Zusammenarbeit von 6–9 HTA-Instituten verfasst. Zwei der Assessments befassen sich mit Medikamenten und zwei mit nicht-pharmakologischen Interventionen. Die Gesamtdauer betrug zwischen 7 und 9 Monate. Erste Hinweise bezuglich Haufigkeit und Art der Weiterverwendung fur nationale/lokale HTA-Berichte liegen vor. Insgesamt 28 HTA-Institute gaben an, die Assessments im eigenen Kontext verwenden zu wollen. In Osterreich wurden 2 Berichte basierend auf den EUnetHTA Assessments vom Ludwig Boltzmann Institut fur Health Technology Assessment (LBI-HTA) publiziert. Als weitere Anwendung kann auch die Implementierung dieses Tools im nationalen und regionalen Kontext einen wichtigen Schritt in Richtung landerubergreifendem Informationsaustausch und internationaler Ubertragbarkeit darstellen. Um die Ubertragbarkeit von HTAs zu erhohen und damit einen weiteren Beitrag zur Reduktion von Redundanzen zu leisten, werden am LBI-HTA daher ab 2015 zwei Programmlinien an das Format des HTA Core Models® angepasst. Diskussion Barrieren fur europaische Zusammenarbeit betreffen die Relevanz der Themen fur einzelne HTA-Institute sowie den Zeitpunkt der gemeinsamen Assessments. Die Implementierung gemeinsam entwickelter Methoden in die Prozesse lokaler/nationaler HTA-Institute wird vor allem durch rechtliche Rahmenbedingungen erschwert. Schlussfolgerung Trotz erster positiver Erfahrungen mit internationaler projektbezogener und methodischer Zusammenarbeit, wird sich erst in den kommenden Jahren zeigen, ob bestehende Hurden dauerhaft und nachhaltig uberwunden werden konnen.

Summary Background With the growing use and importance of health technology assessment (HTA) in decision making during recent years, health technology assessors, decision makers and stakeholders are confronted with methodological challenges due to specific characteristics of health technologies (e. g., pharmaceuticals, diagnostic tests, screening programs), their developmental environment, and their regulation process. Being aware of the necessity to use HTA as a policy instrument for sustainable health care systems in a regulatory environment of decentralized Conformite Europeenne (CE) marking, the European Union (EU) is increasingly supporting the development of methods for the assessment of medical devices (MD) on different levels: within the scope of European research projects and within joint assessment activities of the member states of the European network for Health Technology Assessment (EUnetHTA). Objective First, this article describes three projects: MedtecHTA—Methods for Health Technology Assessment of Medical Devices, a European Perspective Work Package 3 (WP3), Comparative Effectiveness of Medical Devices led by the University for Health Sciences, Medical Informatics and Technology (UMIT). Second, we discuss the experiences of the Ludwig Boltzmann Institute Health Technology Assessment (LBI HTA) with the joint production of rapid assessments of medical devices by several European HTA agencies within EUnetHTA. Third, a brief outline is given of the framework of joint methodological guideline elaboration by the EUnetHTA partner organizations because a guideline for therapeutic MD is also being developed here. Methods We will describe aims, methods and some preliminary results of MedtecHTA and EUnetHTA Joint Action 2 Work Package 5 Strand B (WP5B) applying the HTA Core Model for Rapid Assessment for national adaptation and reporting, and give an overview of the development process of methodological guidelines within WP 7 of EUnetHTA Joint Action 2. Results Based on a literature review in MedtecHTA WP3 incremental development, context dependency and the physical mode of action of MD were identified as those characteristics making therapeutic MD different from drugs with regard to evaluation methods. In addition, regulation does not stipulate clinical trials. These characteristics were also identified as challenges for the production of joint assessments of MD within the HTA network EUnetHTA. Furthermore, adequate timing of assessment production, the variety of involved manufacturers, the non-transparent regulation process of MD in Europe and the often poor evidence base pose a challenge to EUnetHTA assessors. As a consequence, processes and methods for the joint production of rapid assessments must be continuously adapted and improved. Discussion Research on HTA methods for the assessment of MD tries to provide tools to deal with rapidly developing devices during evidence generation, dependence of clinical effectiveness of MD on user experience and context factors. There are also tools to integrate evidence from different sources adjusting for different levels of validity, but these methods are not established and need high epidemiological and statistical expertise. A framework for deciding whether additional evidence is needed to reduce uncertainty regarding safety, clinical effectiveness and cost-effectiveness will be adapted to MD. The whole process of evidence generation before and after market access has to be considered to provide an environment for conclusive HTA recommendations informing health care decision making. In Joint Action 2, EUnetHTA develops transparent processes for the early dialogue with stakeholders and fosters dissemination of appropriate HTA methods. In the case of MD, there are special accumulated needs for such efforts.

Objectives: Many of the currently used health technologies have never been systematically assessed or are misused, overused or superseded. Therefore, they may be ineffective. Active identification of ineffectiveness in health care is gaining importance to facilitate best care for patients and optimal use of limited resources. The present research analyzed processes and experiences of programs for identifying ineffective health technologies. The goal of this study was to elucidate factors that facilitate implementation.Methods: Based on an overview article, a systematic literature search and unsystematic hand-search were conducted. Further information was gained from international experts.Results: Seven programs were identified that include identification, prioritization and assessment of ineffective health technologies and dissemination of recommendations. The programs are quite similar regarding their goals, target groups and criteria for identification and prioritization. Outputs, mainly HTA reports or lists, are mostly disseminated by means of the internet. Top–down and bottom–up programs both have benefits in terms of implementation of recommendations, either as binding guidelines and decisions or as nonbinding information for physicians and other stakeholders. Crucial facilitators of implementation are political will, transparent processes and broad stakeholder involvement focusing on physicians.Conclusions: All programs can improve the quality of health care and enable cost reduction in supportive surrounding conditions. Physicians and patients must be continuously involved in the process of evaluating health technologies. Additionally, decision makers must support programs and translate recommendations into concrete actions.

The aim of this paper was to present the legal framework as well as previous experience in transnational collaboration regarding the assessment of medical devices for reimbursement decisions. Furthermore, the possible benefit of transnational collaboration for various stakeholders will be discussed.Experiences gathered with the compilation of overall 6 joint rapid assessments of medical devices within the completed European Network for Health Technology Assessment (EUnetHTA) Joint Action 2 (JA2) are summarised and discussed. Benefits, aims and opportunities of the ongoing EUnetHTA Joint Action 3 (JA3) are described.Challenges in joint European assessment of medical devices encompass the choice of topics and the time point of assessments, the non-transparency of the medical devices market as well as the lack of European standards for systematic patient involvement. Characteristics of medical devices such as incremental changes and learning curves call for monitoring of medical devices through their whole lifecycle. The concrete benefit of European collaboration for stakeholders is manifold: uncertainties with regard to the actual added value of a technology, caused by a lack of evidence, may be reduced by Early Dialogues; harmonized and transparent assessment processes throughout Europe increase the reproducibility and quality of reports; the division of work among the health technology assessment (HTA) organisations allows a resource efficient assessment of a larger amount of technologies; patient involvement facilitates consideration of patient-relevant endpoints. The importance of cross-border collaboration in the field of HTA is shown in the continuation of the EUnetHTA project, which aims to further strengthen international collaboration even after expiration of EU funding.A sustainable transnational collaboration in the assessment of medical devices can ensure cross-border health care as well as efficient collaboration of national health systems. The focus should be on a wide implementation of jointly established methods and quality standards. The European collaboration can lead to a concrete benefit for patients, physicians, HTA organisations and national decision makers.ZIEL: Ziel des Artikels ist die Darstellung rechtlicher Rahmenbedingungen sowie Erfahrungen in der grenzüberschreitenden Zusammenarbeit bei der Bewertung von Medizinprodukten als Grundlage für Erstattungsentscheidungen. Der mögliche Nutzen transnationaler Kooperation für verschiedene Stakeholdergruppen soll erläutert werden.Prozesse und Herausforderungen der abgeschlossenen European Network for Health Technology Assessment (EUnetHTA) Joint Action 2 (JA2) werden zusammengefasst und diskutiert. Die dem Artikel zugrunde liegenden Erfahrungen beruhen auf der Erstellung und Evaluierung von insgesamt 6 Rapid Assessments zu Medizinprodukten, welche während der EUnetHTA JA2 in internationaler Kollaboration verfasst und publiziert wurden. Nutzen, Ziele und Chancen der laufenden EUnetHTA Joint Action 3 (JA3) werden dargestellt.Herausforderungen in der europäischen Medizinproduktebewertung umfassen die Themenauswahl und den Zeitpunkt der Bewertung, die Intransparenz des Medizinproduktemarkts sowie das Fehlen europäischer Standards zum systematischen Einbeziehen von PatientInnen. Charakteristika wie inkrementelle Veränderungen und Lernkurven machen es notwendig, Medizinprodukte über ihren gesamten Lebenszyklus hinweg zu begleiten. Der Nutzen der europäischen Zusammenarbeit für Stakeholder ist vielfältig: die aufgrund fehlender Evidenz herrschende Unsicherheit zum Zusatznutzen einer Technologie kann durch Early Dialogues reduziert werden; ein europaweit harmonisierter, transparenter Bewertungsprozess steigert die Nachvollziehbarkeit und Qualität der Berichte; die Arbeitsteilung unter den Health Technology Assessment (HTA)-Organisationen ermöglicht eine ressourcenschonende Bewertung einer größeren Anzahl an Technologien; die Patientenbeteiligung gewährleistet die Berücksichtigung patientenrelevanter Endpunkte. Die Wichtigkeit der grenzübergreifenden Zusammenarbeit im Bereich HTA zeigt sich in der Weiterführung des EUnetHTA Projekts, welches die Festigung der internationalen Kollaboration auch nach Auslaufen der EU-Förderungen zum Ziel hat.Eine nachhaltige transnationale Kooperation in der Bewertung von Medizintechnologien kann die grenzüberschreitende Gesundheitsversorgung und die effiziente Zusammenarbeit der nationalen Gesundheitssysteme sichern. Der Fokus sollte dabei auf einer flächendeckenden Implementierung gemeinsam entwickelter Methoden und Qualitätsstandards liegen. Die europäische Zusammenarbeit kann zu einem konkreten Nutzen für PatientInnen, ÄrztInnen, HTA-Organisationen und nationale EntscheidungsträgerInnen führen.

Oncologic drug therapies have gained wide attention in the context of health policy priority setting for serious and socially significant diseases with high human and monetary costs. Due to uncertainties and scepticism about the actual therapeutic importance of newly approved oncology products, an early assessment programme was already established in Austria in 2007. The assessment of new oncology products is thereby faced with special challenges, since study populations are frequently not representative or the study design is laid out in such a manner that a definitive assessment of patient-relevant endpoints is not possible (cross-overs after interim assessments, surrogate parameters as primary endpoints, uncontrolled studies or those with unrealistic comparators, invalidated post-hoc identified biomarkers). On account of these major uncertainties, even the European Medicines Agency (EMA) is already contemplating multi-stage, "adaptive" approvals, and national reimbursement institutions are increasingly working with outcome-oriented, conditional reimbursement. (As supplied by publisher).

Esophageal stents are used for the treatment of refractory and recurrent dyphagias. In 2007, esophageal biodegradable stents (EBS) were authorised as an alternative to existing metal and plastic stents in Europe. The advantages claimed for EBS are fewer complications concerning tissue ingrowth, stent migration and stent removal.We performed a systematic review to evaluate the efficacy and safety of EBS compared to fully-covered self-expanding metal stents, self-expanding plastic stents, and esophageal dilation for the treatment of refractory or recurrent benign esophageal stenosis. Three comparative studies (one randomized controlled trial and two cohort studies) were assessed. The studies used different inclusion criteria, had a very small (sample) size and the quality of the evidence was very low. Expert commentary: The current evidence is insufficient to determine the relative efficacy or safety of esophageal biodegradable stents. The results of this systematic review should be updated once new evidence is available.

INTRODUCTION:Consistently high-quality health care is expected throughout Europe while concurrently, financial resources of member states are decreasing. National Health Technology Assessment (HTA) institutes are informing evidence-based reimbursement decisions in the national context, leading to redundancies in HTA production and tying up limited resources. Since 2006, the European Union project, the European Network for HTA (EUnetHTA) is aiming at enhancing the efficient use of HTA resources and facilitating transnational collaboration. Our aim is to present previous experience in joint assessment of medical devices. Furthermore, possible benefits of European collaboration for stakeholders will be discussed.METHODS:Processes and challenges of the completed EUnetHTA Joint Action (JA) 2 are summarized and discussed. Benefits, aims and opportunities of the ongoing EUnetHTA JA 3 are described.RESULTS:Six rapid assessments of medical devices, focusing on the assessment of effectiveness and safety, were published during EUnetHTA JA 2. Challenges in European medical device assessment encompass the choice of topics, the time point of assessments and the lack of European standards for systematic patient involvement. Characteristics of medical devices, like learning curves, call for monitoring them throughout their lifecycle.The benefit of European collaboration for stakeholders is manifold: uncertainty with regard to actual added value of a technology is minimized through Early Dialogues; harmonized and transparent assessment processes increase the quality of reports; work division among HTA organizations allows a resource-efficient assessment of a bigger amount of technologies; patient involvement ensures consideration of patient relevant endpoints.The importance of cross-border collaboration in HTA is shown in the continuation of the EUnetHTA project, which aims to sustainably strengthen international collaboration even after expiration of EU-funding.CONCLUSIONS:European collaboration in medical device assessment can ensure cross-border health care and efficient cooperation of national health systems. The focus should be set on a wide implementation of jointly established methods and quality standards. The European collaboration can lead to a concrete benefit for various stakeholders.

SincetheEMA/EuropeanMedicinesAgencyhasimplementedaregulationintend-ing to stimulate the developmentof drugs for rare diseases in 2000, 858 drugs havereceived Orphan Drug Designations, but only 62 drugs have yet received MarketingAuthorisation as an “orphan drug”. To benefit from the incentives (reduced fees formarketing-authorisationapplicationsandprotectionfrommarketcompetition),com-panies intending to develop an orphan medicine must submit an application to theEuropeanMedicinesAgencyrequesting’orphandesignation’fortheirmedicine. Theapplication is evaluated by the Agency’sCommittee for Orphan Medicinal Products(COMP), which provides its opinion on whether or not the medicine qualifies as anorphanmedicine for the treatment, preventionor diagnosis of a rare disease, definedas a condition affecting no more than 5 in 10,000 people in the EU. If the COMPissuesa positiveopinion,theEMA maythengrantthemedicineorphanstatus: allofthem registered in the Register of designated Orphan Medicinal Products [1].Even though a wide variety of diseases fulfil this criterion, the largest group(45.2%) for which the COMP has adopted a positive opinion as well as of the 62drugs finally having received marketing authorisation (35.5)% were for oncologytreatments [2]. By April 2011, 21 oncologic drugs (between 1 and 5 per year) havebeenapprovedasorphandrugs(seeTable1),someofthemformorethanoneindica-tion. According to industry-forecasts, the number of designations will “continue toincrease exponentially” [3] within the next months and years and consequently willthe numberof successful marketing authorisations.While the regulationfor orphandrugs, originallyintendedto providea favourableeconomic environment for the development of drugs for which either no marketor a too fragmented markets for the generation of adequate return on investmentexist, is euphorically discussed in pharma-fora and at biotech-conferences, health

Several countries have set up early awareness and alert systems (EAAS) to inform their customers about technologies that may have a significant impact on the health care system [1].

The standardisation of European HTA and thus the reduction of redundancies require clearly defined processes and methods. The HTA Core Model®, a tool developed by the European Network EUnetHTA, is intended to ensure the transparent production of standardised and high-quality assessments in international collaboration.The present paper describes the experience with already published EUnetHTA assessments as well as possibilities for national/local adaptations of these assessments. The integration of jointly developed methods in routine processes of individual HTA agencies will be explained on the basis of a selected example. Further methodological initiatives in Europe will be presented.So far, EUnetHTA has published four rapid assessments conducted through European cooperation between 6-9 HTA institutes during Joint Action 2 (2012-2015). Two assessments dealt with pharmaceuticals and two with non-pharmaceutical interventions. The overall duration of these assessments ranged from 7 to 9 months. There is initial information about the frequency and manner in which these assessments have been used for national/local HTA reports. According to a survey, a total of 28 HTA institutes have indicated that they want to make use of these assessments in their own context. In Austria, the Ludwig Boltzmann Institute for Health Technology Assessment (LBI-HTA) has produced two reports based on EUnetHTA assessments. A further step towards cross-border collaboration and harmonisation is the implementation of these tools in a national and regional context. Beginning in 2015 the LBI-HTA will adjust two programme lines to the format of the HTA Core Model® in order to increase the transferability of HTAs and to reduce redundancies.Barriers to European collaboration include the relevance of topics for individual HTA institutes and the timing of joint assessments. Implementing commonly developed methods as standard practice in local/national HTA institutes is mainly impeded by legislative requirements.Despite the initial positive experiences with international collaboration on specific topics and methods, the coming years will have to prove whether existing barriers can be overcome effectively.

Objectives: The aim of this study was to present the first four collaborative health technology assessment (HTA) processes on health technologies of different types and life cycles targeted toward diverse HTA users and facilitators, as well as the barriers of these collaborations.Methods: Retrospective analysis, through four case studies, was performed on the first four collaboration experiences of agencies participating in the EUnetHTA Joint Action project (2010–12), comprising different types and life cycles of health technologies for a diverse target audience, and different types of collaboration. The methods used to initiate collaboration, partner contributions, the assessment methodology, report structure, time frame, and factors acting as possible barriers to and facilitators of this collaboration were described.Results: Two ways were used to initiate collaboration in the first four collaborative HTA processes: active brokering of information, so-called “calls for collaboration,” and individual contact between agencies after identifying a topic common to two agencies in the Planned and Ongoing Projects database. Several success factors are recognized: predefined project management, high degree of commitment to the project; adherence to timelines; high relevance of technology; a common understanding of the methods applied and advanced experience in HTA; finally, acceptance of English-written reports by decision makers in non–English-speaking countries. Barriers like late identification of collaborative partners, nonacceptance of English language and different methodology of assessment should be overcome.Conclusions: Timely and efficient, different collaborative HTA processes on relative efficacy/effectiveness and safety on different types and life cycles of health technologies, targeted toward diverse HTA users in Europe are possible. There are still barriers to overcome.

Oncologic drug therapies have gained wide attention in the context of health policy priority setting for serious and socially significant diseases with high human and monetary costs. Due to uncertainties and scepticism about the actual therapeutic importance of newly approved oncology products, an early assessment programme was already established in Austria in 2007. The assessment of new oncology products is thereby faced with special challenges, since study populations are frequently not representative or the study design is laid out in such a manner that a definitive assessment of patient-relevant endpoints is not possible (cross-overs after interim assessments, surrogate parameters as primary endpoints, uncontrolled studies or those with unrealistic comparators, invalidated post-hoc identified biomarkers). On account of these major uncertainties, even the European Medicines Agency (EMA) is already contemplating multi-stage, "adaptive" approvals, and national reimbursement institutions are increasingly working with outcome-oriented, conditional reimbursement. (As supplied by publisher).

Background Due to increasing expenditures for cancer therapies, an instrument was needed in Austria to facilitate the evidence-based use of new anticancer drugs and to pre-estimate their financial implications. Objectives To describe and analyse the development and implementation of a Horizon Scanning System (HSS) in Austria that allows for the evaluation of new anticancer drugs before their routine introduction into clinical practice in order to inform decision-makers. Methods Common stages involved in HSSs were identified by a literature review and in cooperation with experts. A first concept for an HSS in Oncology was developed and piloted, and further adjustments were made after several feedback rounds with experts in oncology. Results To specifically tailor the five common stages of HSSs to the needs of our HSS, a continually evolving process was required. Now, 21 information sources are regularly scanned, the information is retrieved and extracted in a standardised format, and only anticancer drugs in phase III are included and prioritised by a team of eight experts. Since the HSS in Oncology was implemented as a standard practice, 19 assessments on novel cancer therapies with likely therapeutic and/or financial impacts have been published. Conclusions The successful implementation of an HSS necessitates a repetitive cycle of adjustments in order to meet the objectives set by the individual HSS.