scholarly journals Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations

2020 ◽  
Vol 25 (7) ◽  
pp. 1223-1231
Author(s):  
Rick A. Vreman ◽  
Angela S. de Ruijter ◽  
Anna Zawada ◽  
Giovanni Tafuri ◽  
Violeta Stoyanova-Beninska ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Relative Effectiveness ◽  
Health Technology ◽  
Significant Benefit ◽  
Medicinal Products ◽  
Orphan Medicinal Products

scholarly journals Health Technology Assessment Challenges for Orphan Medicinal Products in Low- and Middle-Income Countries in Europe

2021 ◽  
Vol 70 (1) ◽  
Author(s):  
Anna Mateva ◽  
Konstantin Tachkov ◽  
Antoniya Yanakieva ◽  
Petya Milushewa
Keyword(s):  
Health Care ◽  
Health Technology Assessment ◽  
Health Care System ◽  
Technology Assessment ◽  
Health Technology ◽  
Medicinal Products ◽  
Middle Income ◽  
Middle Income Countries ◽  
Orphan Medicinal Products ◽  
Low And Middle Income

The purpose of this research is to provide a short overview of the differences in access to and health technology assessment of OMPs in selected low- and middle-income countries in Europe and discuss these in terms of some challenges in the face of HTA comparing with high-income countries. This is a retrospective, desktop study of already published official inquiries of national or international institutions, scientific publications, and reports from national governing bodies who regulate and monitor HTA in low- and middleincome economies in Europe, with further regards to HTA of orphan medicinal products. Among all selected low- and middle-income countries in Europe - Bulgaria, Serbia, North Macedonia, Moldova, Ukraine, and Georgia, have available and up-to-date information regarding their health care legislation. During 2000-2019 EMA approves 164 orphan medicines and out of them from 23 to 133 were found in the selected countries. We found that Bulgaria has a special HTA body. Since 2015 in Ukraine the health care system is experienced dynamic reform. In particular, a legislative framework has been adopted that regulates pharmaceutical pricing and reimbursement policies, the introduction of health technology assessment (HTA), etc. Despite the fact that Serbia is not part of the European Union there is an HTA commission established by the Ministry of Health where members of the HTA committee have 5- year mandates. According to our research, Georgia and North Macedonia do not use HTA. Meanwhile, United Kingdom is well known to show a strong interest in evidence health care. system. On the other hand, ?he Statutory health insurance system (SHI reform), since 2000, established a federally funded HTA program with a database of relevant HTA results. Even though that the high-income countries have more developed HTA guidelines and stronger and more secure health systems, all the countries mentioned in this work are facing similar challenges. All the countries have limite

VP67 The Value Of The European Network For Health Technology Assessment (EUnetHTA) Outputs For National Health Technology Assessment: The French Experience

2017 ◽  
Vol 33 (S1) ◽  
pp. 180-180
Author(s):  
Irena Guzina ◽  
François Meyer
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Joint Action ◽  
Relative Effectiveness ◽  
Health Technology ◽  
Specific Work ◽  
European Network ◽  
Know How ◽  
French Experience ◽  
Methodological Guidelines

INTRODUCTION:The French National Authority for Health (HAS) has participated in the work of the European Network for Health Technology Assessment (EUnetHTA) since its creation in 2006. HAS has been an active partner in most EUnetHTA activities, and the lead partner of specific work packages.METHODS:This report presents a summary of the main contribution of HAS to the two latest EUnetHTA projects (Joint Action (JA) 1 and Joint Action 2 (JA2)), their impact on national production, and Health Technology Assessment (HTA) doers feedback as to the opportunities and challenges of participating to the network and using its outputs.RESULTS:In JA 1 and JA 2 projects, HAS has: coordinated activities related to Early Dialogues and Additional Evidence Generation; coordinated the development of nine JA1 methodological guidelines for rapid relative effectiveness assessment (REA) of pharmaceuticals; participated in the production of two JA2 methodological guidelines; participated in the production of one JA1 and seven JA2 rapid REA reports, and two JA2 full HTAs.The national uptake of EUnetHTA outputs included entire adoptation of one REA report and adaptation of another. EUnetHTA templates and methodological guidelines have been taken into account when updating or developing national ones. Thanks to the network, HAS HTA doers could exchange on ongoing assessments with European colleagues, have enhanced their methodological know-how and enlarged their professional network.As for the challenges encountered, it turned out that the re-use of EUnetHTA reports for a technology of interest to HAS was not always possible, mainly due to discordances in deadlines or assessment questions between EUnetHTA and national productions.CONCLUSIONS:HAS has actively participated in different EUnetHTA projects since the network's creation. This collaboration has enabled HAS HTA doers, among others, to optimize national assessments and enhance their methodological know-how.

VP08 Real-World Data Use In Health Technology Assessments: A Comparison Of Five Health Technology Assessment Agencies

2017 ◽  
Vol 33 (S1) ◽  
pp. 149-150
Author(s):  
Amr Makady ◽  
Ard van Veelen ◽  
Anthonius de Boer ◽  
Hans Hillege ◽  
Olaf Klunger ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Relative Effectiveness ◽  
Internal Validity ◽  
Health Technology ◽  
Real World Data ◽  
World Data ◽  
The Impact

INTRODUCTION:Reimbursement decisions are usually based on evidence from randomized controlled trials (RCT) with high internal validity but lower external validity. Real-World Data (RWD) may provide complimentary evidence for relative effectiveness assessments (REA's) and cost-effectiveness assessments (CEA's) of treatments. This study explores to which extent RWD is incorporated in REA's and CEA's of drugs used to treat metastatic melanoma (MM) by five Health Technology Assessment (HTA) agencies.METHODS:Dossiers for MM drugs published between 1 January 2011 and 31 December 2016 were retrieved for HTA agencies in five countries: the United Kingdom (NICE), Scotland (SMC), France (HAS), Germany (IQWiG) and the Netherlands (ZIN). A standardized data-extraction form was used to extract data on RWD mentioned in the assessment and its impact on appraisal (for example, positive, negative, neutral or unknown) for both REA and CEA.RESULTS:In total, fourty-nine dossiers were retrieved: NICE = 10, SMC = 13, IQWiG = 16, HAS = 8 and ZIN = 2. Nine dossiers (18.4 percent) included RWD in REA's for several parameters: to describe effectiveness (n = 5) and/or the safety (n = 2) of the drug, and/or the prevalence of MM (n = 4). CEA's were included in 25/49 dossiers (IQWiG and HAS did not perform CEA's). Of the twenty-five CEA's, twenty (80 percent) included RWD to extrapolate long-term effectiveness (n = 19), and/or identify costs associated with treatments (n = 7). When RWD was included in REA's (n = 9), its impact on the appraisal was negative (n = 4), neutral (n = 2), unknown (n = 1) or was not discussed in the appraisal (n = 2). When RWD was included in CEA's (n = 11), its impact on the appraisal varied between positive (n = 2), negative (n = 5) and unknown (n = 4).CONCLUSIONS:Generally, RWD is more often included in CEA's than REA's (80 percent versus 18.4 percent, respectively). When included, RWD was mostly used to describe the effectiveness of the drug (REA) or to predict long-term effectiveness (CEA). The impact of RWD on the appraisal varied greatly within both REA's and CEA's.

PRICING, REIMBURSEMENT, AND HEALTH TECHNOLOGY ASSESSMENT OF MEDICINAL PRODUCTS IN BULGARIA

2017 ◽  
Vol 33 (3) ◽  
pp. 365-370 ◽  
Author(s):  
Tatyana Benisheva-Dimitrova ◽  
Dobriana Sidjimova ◽  
Daniela Cherneva ◽  
Nikolay Kralimarkov
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Objective Assessment ◽  
Historical Analysis ◽  
Health Technology ◽  
National Council ◽  
The European Union ◽  
Medicinal Products ◽  
Policy Objective ◽  
Pricing And Reimbursement

Objectives: The aim of this study was to investigate the analysis, discussion, and challenges of the price and reimbursement process of medicinal products in Bulgaria in the period 2000–15 and health technology assessment (HTA) role in these processes.Methods: The dynamics of the reform, with respect to the healthcare and pharmaceutical sectors, are tracked by documentary review of regulations, articles, and reports in the European Union (EU), as well as analytical and historical analysis.Results: Pricing and reimbursement processes have passed through a variety of committees between 2003 and 2012. Separate units for pricing and reimbursement of medicinal products were established in Bulgaria for the first time, in 2013, when an independent body, the National Council at Prices and Reimbursement of Medicinal Products, was set up to approve medicinal products with new international nonproprietary names (INN) for reimbursement in Bulgaria. Over the course of 2 years (2013–14), thirty-three new INNs were approved for reimbursement. In December 2015, a new HTA body was introduced, and assigned to the National Centre for Public Health and Analyses.Conclusions: Although Bulgaria has current legislation on pricing and reimbursement which is in accordance with the EU rules, there is no mechanism for reporting and monitoring these processes or the financial resources annually, so as to provide an overall objective assessment and analysis by year. Therefore, this financial assessment should become a national policy objective for the future.

scholarly journals Health Technology Assessment of Advanced Therapy Medicinal Products: Comparison Among 3 European Countries

2021 ◽  
Vol 12 ◽  
Author(s):  
Lucia Gozzo ◽  
Giovanni Luca Romano ◽  
Francesca Romano ◽  
Serena Brancati ◽  
Laura Longo ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
National Level ◽  
Health Technology ◽  
European Countries ◽  
Added Value ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Therapeutic Value ◽  
Advanced Therapy

Even for centrally approved products, each European country is responsible for the effective national market access. This step can result in inequalities in terms of access, due to different opinions about the therapeutic value assessed by health technology assessment (HTA) bodies. Advanced therapy medicinal products (ATMPs) represent a major issue with regard to the HTA in order to make them available at a national level. These products are based on genes, tissues, or cells, commonly developed as one-shot treatment for rare or ultrarare diseases and mandatorily authorized by the EMA with a central procedure. This study aims to provide a comparative analysis of HTA recommendations issued by European countries (France, Germany, and Italy) following EMA approval of ATMPs. We found a low rate of agreement on the therapeutic value (in particular the “added value” compared to the standard of care) of ATMPs. Despite the differences in terms of clinical assessment, the access has been usually guaranteed, even with different timing and limitations. In view of the importance of ATMPs as innovative therapies for unmet needs, it is crucial to understand and act on the causes of disagreement among the HTA. In addition, the adoption of the new EU regulation on HTA would be useful to reduce disparities of medicine’s assessment among European countries.

OP162 Stakeholder Involvement In EUnetHTA Relative Effectiveness Assessments

2019 ◽  
Vol 35 (S1) ◽  
pp. 35-35
Author(s):  
María Sánchez González ◽  
Iñaki Imaz-Iglesia ◽  
Juan Pablo Chalco-Orrego
Keyword(s):  
Health Care ◽  
Focus Groups ◽  
Health Technology Assessment ◽  
Health Care Providers ◽  
Technology Assessment ◽  
Relative Effectiveness ◽  
Stakeholder Involvement ◽  
Health Technology ◽  
Care Providers ◽  
Project Plan

IntroductionAppropriate involvement of stakeholders is one of the founding principles of the European Cooperation on Health Technology Assessment. The European Network for Health Technology Assessment (EUnetHTA) produces Rapid Relative Effectiveness Assessments (REAs) to assess pharmaceutical (PT) or other technologies (OT). Stakeholders essentially participate in the scoping, the draft assessment phase, or both.MethodsAll REAs published since 2013 were reviewed. Stakeholder participation in scoping (project plan) and draft assessment was evaluated. We aggregated categories of stakeholders in four groups (Health Care Providers and Academia, Patients and Consumers, Manufacturers, and Regulators and Payers). Means of collaboration (meetings, comments to project plan and draft assessment, questionnaires, focus groups) are also analyzed. Data is continuously updated with new REAs.ResultsMore than 20 REAs have been published at the moment, with a higher number of OT. Health Care Providers and Academia acted as experts in both phases, participating in all REA of OT, and less of PT. Manufacturers participated in all REA in the scoping phase. Regulators and Payers, less involved, participated mainly in the scoping phase. The main methods are providing comments in a standardized form and meetings. Patients' contribution, similar in OT and PT, has increased over the years. Questionnaires or interviews were the main method of involvement, followed by participation in meetings and focus groups. Visibility and transparency have also improved, with a clearer reporting of the stakeholder contribution in the last assessments.ConclusionsThe stakeholder involvement in EUnetHTA REAs is steadily growing, with the different nature of stakeholders’ categories reflected in their contribution to the assessments. EUnetHTA is standardizing stakeholder involvement procedures taking into account the particularities of each group when generating guidance for stakeholder involvement.

OP119 Advanced Therapy Medicinal Products: Are Current Health Technology Assessment Methods Suitable?

2017 ◽  
Vol 33 (S1) ◽  
pp. 54-55
Author(s):  
Grace Hampson ◽  
Adrian Towse
Keyword(s):  
Cell Therapy ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Medicinal Products ◽  
T Cell Therapy ◽  
Car T Cell Therapy ◽  
Advanced Therapy ◽  
Car T ◽  
Regenerative Medicines

INTRODUCTION:There is considerable excitement around the development of regenerative medicines (or advanced therapy medicinal products, ATMPs), with the expectation that they may bring substantial clinical gains and offer cures for previous debilitating and fatal diseases. However, high costs mean that Health Technology Assessment (HTA) and reimbursement decisions are challenging for payers and manufacturers, even when the therapies are expected to offer good value for money.In Europe, seven ATMPs have market authorization, yet only one has achieved national level reimbursement. Statistics such as these put HTA bodies under pressure to review their methods and consider how these can apply to regenerative medicines.METHODS:We present a review of one example, from the United Kingdom's National Institute for Health and Care Excellence (NICE), who commissioned an external organization to undertake a mock appraisal of a hypothetical ATMP using standard methods. The therapeutic area chosen for the mock appraisal was chimeric antigen receptor (CAR) T-cell therapy for treating relapsed or refractory B-cell acute lymphoblastic leukaemia.RESULTS:The role of uncertainty was a key consideration within the report, yet we found that the presentation of uncertainty within the mock appraisal was misleading for decision makers.We found that the exercise represents a thorough mock HTA of CAR T-cell therapy. However, it focused on testing whether ATMPs could fit into the existing HTA pathway for conventional medicines, rather than seeking to identify the most suitable approach for assessing regenerative medicines. We suggest the latter would have been a more relevant question for the mock appraisal.CONCLUSIONS:Any significant departures from the usual HTA process must be based on solid economic rationale if we are to ensure efficient allocation of resources. Thus, in order for regenerative medicines to be given ‘special treatment,’ it must be demonstrated that societal preferences, or the full extent of health (or non-health) benefits, are not being realised for this group of treatments through existing HTA methods.

CURRENT ENVIRONMENT FOR INTRODUCING HEALTH TECHNOLOGY ASSESSMENT IN GREECE

2017 ◽  
Vol 33 (3) ◽  
pp. 396-401 ◽  
Author(s):  
Chara Kani ◽  
Vasilios Kourafalos ◽  
Panagiota Litsa
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Pharmaceutical Expenditure ◽  
Health Technology ◽  
Medicinal Products ◽  
Health Technologies ◽  
External Reference ◽  
Legislative Framework ◽  
Human Use ◽  
Pricing And Reimbursement

Objectives: The aim of this study was to describe the current regulatory environment in Greece to evaluate the potential introduction of health technology assessment (HTA) for medicinal products for human use.Methods: Data sources consist of national legislation on pricing and reimbursement of health technologies to identify the potential need of establishing HTA and its relevant structure.Results: The pricing procedure regarding medicinal products for human use is based on an external reference pricing mechanism which considers the average of the three lowest Euorpean Union prices. Currently, a formal HTA procedure has not been applied in Greece, and the only prerequisite used for the reimbursement of medicinal products for human use is their inclusion in the Positive Reimbursement List. To restrict pharmaceutical expenditure, a variety of measures—such as clawback mechanisms, rebates, monthly budget caps per physician, generics penetration targeting—have been imposed, aiming mainly to regulate the price level rather than control the introduction of medicinal products for human use in the Greek pharmaceutical market.Conclusions: Greece has the opportunity to rapidly build capacity, implement, and take advantage of the application of HTA mechanisms by clearly defining the goals, scope, systems, context, stakeholders, and methods that will be involved in the local HTA processes, taking into account the country's established e-prescription system and the recently adapted legislative framework.

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