Maria Dimitrova1*†
Ivett Jakab2†
Zornitsa Mitkova1
Maria Kamusheva1
Konstantin Tachkov1
Bertalan Nemeth2
Antal Zemplenyi2,3
Dalia Dawoud4,5
Diana M. J. Delnoij6,7
François Houýez8
Zoltan Kalo2,9- 1Faculty of Pharmacy, Medical University of Sofia, Sofia, Bulgaria
- 2Syreon Research Institute, Budapest, Hungary
- 3Center for Health Technology Assessment and Pharmacoeconomics Research, Faculty of Pharmacy, University of Pécs, Pécs, Hungary
- 4National Institute for Health and Care Excellence, London, United Kingdom
- 5Faculty of Pharmacy, Cairo University, Cairo, Egypt
- 6Erasmus School of Health Policy and Management, Erasmus University, Rotterdam, Netherlands
- 7National Health Care Institute (Zorginstituut Nederland), Diemen, Netherlands
- 8EURORDIS: Eurordis, European Organization for Rare Diseases, Paris, France
- 9Center for Health Technology Assessment, Semmelweis University, Budapest, Hungary
Patients' perspectives are important to identify preferences, estimate values and appreciate unmet medical needs in the process of research and development and subsequent assessment of new health technologies. Patient and public involvement in health technology assessment (HTA) is essential in understanding and assessing wider implications of coverage and reimbursement decisions for patients, their relatives, caregivers, and the general population. There are two approaches to incorporating the patients' voice in HTA, preferably used in a mix. In the first one, patients, caregivers and/or their representatives directly participate at discussions in different stages of the HTA process, often at the same table with other stakeholders. Secondly, patient involvement activities can be supported by evidence on patient value and experience collected directly from patients, caregivers and/or their representatives often by patient groups Patient involvement practices, however, are limited in Central and Eastern European (CEE) countries without clear methodology or regulatory mechanisms to guide patient involvement in the HTA process. This poses the question of transferability of practices used in other countries, and might call for the development of new CEE-specific guidelines and methods. In this study we aim to map potential barriers of patient involvement in HTA in countries of the CEE region.
Introduction
Patients' perspectives are important to identify preferences, estimate values and appreciate unmet medical needs in the process of research and development and subsequent assessment of new health technologies (1). Health technology assessment (HTA) is a multidisciplinary process that uses explicit methods to determine the value of a health technology from different dimensions. Such health technology can be a medical test, device, medicine, vaccine, medical procedure, program, or even a health policy intervention (2). Patient and public involvement in HTA is essential in understanding and assessing wider implications of coverage and reimbursement decisions for patients, their relatives, caregivers, and the general population (1).
Patient involvement is intended to inform all the elements of an HTA from shaping research questions, early dialogues, informing cost-effectiveness models and/or the deliberation process (3, 4). There are two approaches to incorporating the patients' voice in HTA, preferably used in a mix. In the first one, patients, caregivers and/or their representatives directly participate at discussions in different stages of the HTA process, often at the same table with other stakeholders (5). Within this approach, several different methods can be used reflecting different aspects and levels of involvement [e.g., call for written comments, organizing a patient panel, inviting patient(s) to an Advisory Board or focus groups]. Secondly, patient involvement activities can be supported by evidence on patient value and experience collected directly from patients, caregivers and/or their representatives often by patient groups (6). Subsequently, the need for scientific justification of the point of view of patients and society is enhanced by the increasing number of studies to measure not only patients‘ preferences (i.e., time trade-off, standard gamble, etc.) but also patients‘ involvement in the collection of patient‘s reported outcomes (7, 8). Evidence generated through patient preference studies is becoming recognized by HTA organizations as a valuable addition to health technology submissions (9).
The general guide for patient involvement and mix of methods to use should be developed together with patients and fitting the local regulatory environment (2). The method and level of HTA is determined at the national and regional level, along with the method and level of patient involvement. There are European level initiatives such as the European network for Health Technology Assessment (EUnetHTA) jointly assessing selected new technologies and just recently there was a new harmonized regulation on HTA adopted in the European Union (EU), but no EU regulation of HTA applies until January 2025 (10). Thereby, to this date, the approach to patient involvement differs greatly by countries and regions (2, 11, 12). A survey by the European Patients' Forum in 2012 concluded that there are regional differences in the proportion of HTA agencies with and without patient involvement within Europe (13). These findings are in line with results of a survey performed by Health Technology Assessment International's Patient and Citizen Involvement Group (HTAi PCIG) in 2016 (14). The following countries reported patient involvement activities in one or more of their HTA organizations: Australia, Canada, Columbia, England, France, Germany, Italy, Netherlands, Sweden, Taiwan, Poland, Scotland and Wales. Remarkably, there was only one HTA organization from the Central Eastern European (CEE) region reporting on patient involvement activities—the Agency for Health Technology Assessment in Poland. Other CEE countries' HTA organizations did not respond to the survey, thereby there might have been some unreported patient involvement activities in CEE countries. However, the regional difference in response in itself warrants further investigation of patient involvement in HTA in CEE countries.
Compared other regions of Europe, CEE countries are in general at less advanced stages of implementing HTA in spite of the great need for evidence-based resource allocation decisions (15). Some exceptions exist, and a general positive trend can be observed, but the gap between the CEE region and the rest of Europe is still detectable. There are additional—albeit relating—cultural, historical, economic, organizational differences to be taken into consideration when applying good practices of HTA to the CEE context. This is particularly the case for patient involvement in HTA. Patient involvement practices are limited in CEE countries without clear methodology or regulatory mechanisms to guide patient involvement in the HTA process. This poses the question of transferability of practices used in other countries, and might call for the development of new CEE-specific guidelines and methods. However, this requires more insight into patient involvement HTA practices in CEE and the factors that promote or inhibit this. Therefore, in this study we aim to map potential barriers of patient involvement in HTA in countries of the CEE region.
Methodology
This research was conducted as part of the HTx project. HTx is a Horizon 2020 project supported by the European Union lasting for 5 years from January 2019 (www.htx-h2020.eu). The main aim of HTx is to create a framework for the Next Generation Health Technology Assessment to support patient-centered, societally oriented, real-time decision-making on access to and reimbursement for health technologies throughout Europe. Through Work Package 5, HTx aims to assess transferability aspects of novel HTA methodology from Western Europe (WE) countries to CEE countries and form recommendations. Patient-centered and socially-oriented HTA being in the focus of HTx, patient involvement in HTA was selected as a good practice to be included in such an assessment.
The study was conducted in three phases: (1) a scoping literature review to identify potential barriers of patient involvement in HTA, (2) a workshop with relevant stakeholders from CEE countries and experts from the HTx consortium to identify additional barriers, (3) an iterative process ran throughout these phases by CEE researchers from the HTx consortium deduplicating, merging and categorizing identified barriers.
Literature Review
The scoping literature review aimed to identify publications discussing potential barriers of patient involvement in HTA. The literature search was conducted through the PubMed database on the 30th of September in 2020, using the combination of the following keywords: patient; public; health technology assessment; HTA; involve; engage. The search was limited to English-language papers published in the past 10 years. Websites of relevant European Commission funded policy research projects (Innovative Medicines Initiative, Horizon 2020) were screened for project deliverables concerning patient involvement in HTA. Additionally we included experts of the field (patient involvement in HTA) that could propose additional peer-reviewed articles deem important but missed by the literature search.
Identified articles were deduplicated and screened first in the title and abstract screening phase, then those included were reviewed in full-text. The following exclusion criteria were used: (1) No abstract; (2) Not English language, (3) Not published in the past 10 years; (4) Not discussing HTA; (5) Not focusing on patient involvement; (6) Not mentioning any barriers of patient involvement in HTA. Identified barriers were extracted and served as a basis for the workshop and the iterative process.
Webinar
The second step of the study was a live webinar organized for HTx consortium members and CEE stakeholders, including payers, academics, healthcare professionals, industry and patient representatives. The aim of the webinar was to present study results and further identify barriers of patient involvement in HTA from the different perspectives, relevant for the CEE region. Because of the challenges related with the existing COVID-19 pandemic the webinar was held online 0.73 attendees from 12 CEE countries managed to joined the webinar on the 4th of December 2020. All invited attendees got pre-meeting materials for preparations and the interim results of the scoping literature review were presented as a basis for discussion. Based on their expertise and perspective the attendees provided written comments on the most important barriers identified through the literature search process.
Iterative Process
From September 2020 to January 2021, parallel to the other research phases, the main research team conducted the iterative process. The main research team consisted of eight researchers from the two CEE partners within the HTx consortium coming from different areas of health economics and patient-centered research. Final consensus for the identified barriers was reached after series of regular research team meetings for deduplication, merge, categorization and clarification of the identified barriers.
Results
Thirty-two (n = 32) published scientific articles and two gray literature sources meeting the predefined criteria were identified by the scoping literature review (Figure 1).
Figure 1. Flow-diagram of articles screened in the scoping literature review.
The list with the included papers (n = 34) with basic characteristics can be found in Table 1.
Table 1. List of papers included in the scoping literature review.
Included articles focused on (1) incentives and expectations of patient involvement in HTA (2) different methods of inclusion (3) methods of impact assessment (4) mapping current practices and actual impact. All included articles mentioned at least one potential barrier of patient involvement in HTA. The scoping review shows that, after 2015, there is a significant rise in the published articles looking into challenges and possible barriers of patient involvement in HTA.
The final set of potential barriers came along as a result of the scoping review, workshop and the parallel iterative process. Twenty-five (n = 25) potential barriers were identified and investigated. First, barriers were classified based on whose perspective they relate to from the two main stakeholder groups included in these patient involvement activities in HTA—payers/HTA organization representatives and patients. Fourteen (n = 14) barriers appeared from the side of payers/HTA bodies and eleven (n = 11) from the side of patients (Table 2). Then to ease understanding these barriers were grouped as follows: (1) payer/HTA side: Limited willingness to involve patients; Conflict of interest and confidentiality; Difficulties to finding the “right” patient; Lack of human resources at relevant public institutes; Not knowing how to involve patients. (2) Patient side: Lack of understanding the decision context; Lack of knowledge and guidance of evidence-based advocacy; Lack of resources to be spent on meaningful patient representation; Lack of ethical guidance for representativeness.
Table 2. Potential barriers of patient involvement in HTA in CEE countries.
Most of the barriers coming from the perspective of the payers/HTA bodies are related to the lack of defined rules how and when to include patients‘ representatives which increases the risk for lack of trust and fear to include patients in the HTA process.
From patients‘ perspective the most of the identified barriers were associated with lack of sufficiently explained methodology for the patient‘s role in the HTA process—lack of HTA and regulatory processes knowledge, medical language knowledge, etc. This could be attributed to the lack of organized programs in the health care systems at all for patient engagement in collecting patient reported outcomes and real world evidence.
Discussion
Patient engagement in HTA is considered as a transformative strategy which still needs to be adopted in most European countries (47). To a certain extent, the difficulties identified here are not specific for patient engagement in HTA nor for CEE countries, but emerge in patient involvement in healthcare decision-making in general and worldwide. For example, De Graaff et al. elaborate on questions such as whom to involve, how to involve patients and the public, and how to value their input in healthcare decision-making. They argue that more attention should be paid to the work needed for patient and public involvement to explain the gap between expected and current practice (48). Specific barriers such as time investment and budgetary constraints have been reported by Wiering et al. in their study of patient involvement in the development of Patient-Reported Outcome Measures (8). Peeters et al. describe patient organizations' involvement in quality improvement projects in The Netherlands as vulnerable, because of insecure funding and lack of negotiating power (49). Wiig et al. mapped patient involvement in regulatory practice in Norway, England, the Netherlands, and Australia. Their study pointed to several difficulties, such as how to incorporate patients' input, lack of willingness of patients to be involved, time and costs required, barriers related to organizational procedures, and dealing with emotions (50). Finally, in their analysis of decision-making in Dutch HTA practice, Moes et al. point to the risk of so-called epistemic injustice that occurs if patients are being “frustrated in their capacity to be heard and make themselves understood” (51).
Some studies also raise the questions of how we can build trust, partnership and collaborative working environment and what tools and knowledge could be effective to bring together patients/public and policy makers (52, 53).
However, despite the fact that several difficulties are of a more general nature, there are specific barriers for implementation in CEE countries, that call for specific guidelines of patient engagement (37). These specific barriers are, for instance, uncertainty in the role of civil societies, including patient organizations in political processes, or more pressing budgetary constraints. The development of CEE specific guidelines can build on general guidelines on patient involvement in HTA incorporating learnings from the identified barriers. Regarding patient engagement and patient preferences in HTA, many approaches were identified and listed as good practice documents, tracing possible mechanisms for inclusion of the patients in the decision-making process. These approaches also aim to establish guidance on different stakeholder involvement, identify emerging strategies and state of art methods do overcome challenges related to the patient engagement in the HTA process (25, 54–68). Notably, the European Patients' Academy on Therapeutic Innovation (EUPATI) published four guidance papers for patient involvement, one of which was about HTA. This guideline includes suggested working practices for both HTA agencies and patient organizations, as well as suggested patient involvement activities for general HTA processes and individual HTAs. Additionally, both EUnetHTA and Heath Technology Assessment International's Patient and Citizen Involvement Interest Group (HTAi PCIG) regularly publishes and updates guides and templates to aid patient involvement practices (69–71).
To our best knowledge, this is the first published study focusing on barriers of patient involvement in HTA specifically in CEE countries. However, a report with similar scope was identified through the gray literature review. The PARADIGM project was a public-private Innovative Medicines Initiative (IMI) partnership active between 2018 and 2020 (72). The project's mission was to provide a unique framework that enables structured, effective, meaningful, ethical, innovative, and sustainable patient engagement and demonstrates the “return on the engagement” for all players. Within the sustainability assessment of the patient engagement roadmap developed through the project, a workshop was held for stakeholders across the CEE region (37). Four end goals were identified for the European patient engagement landscape: (1) Establish an ethical, trust-based collaboration among all patient engagement stakeholders involved in medicines development; (2) Secure inclusive and diverse patient engagement; (3) Embed patient engagement in the mind-set and at every step and across organizations; (4) Ensure dedicated leadership and operational time, resources and funding. Forty-three (n = 43) barriers were identified challenging the reach to these four end goals. The concept was tested through a CEE workshop, however, the deliverable did not focus on CEE-specific barriers and the scope was research and development, not HTA.
A recent systematic literature review by Gagnon and colleagues (5) in 2021 aimed to summarize current evidence on patient and public involvement in HTA and to propose a framework to assess its impact. Thirty-one (n = 31) studies were included described in 36 publications. One study reporting on the a HTA case study of palliative care involved two CEE countries amongst others, Lithuania and Poland (73). All other studies had settings other than the CEE region; most of the included studies were conducted in Canada, followed by Italy, England, Germany and Finland, Austria, Ireland, Scotland, South Korea and Spain. Barriers published in the paper are in line with our findings and there are certain limitations of our research. Firstly, in terms of methodology, a non-systematic approach was taken when reviewing the literature and we limited our search to articles published in English. There is a chance some papers are missed from our review Secondly, CEE stakeholders included in the webinar had the chance to comment on the list of barriers and propose missing ones, however, the collated barriers were not ranked in terms of priority. We plan to continue our research with this step. Thirdly, our study did not cover potential action plans and recommendations to address the identified barriers. We deem this step to be crucial in advocating for a change of patient involvement practices, thereby our future steps will cover this aspect as well.
Next steps of the research include the ranking of identified barriers and proposing solutions by a broad CEE stakeholder group. The results of the latter studies are to be published in the future. All future studies are planned to be published to serve as a tool for meaningful patient involvement in HTA and pricing and reimbursement decisions in the CEE region.
Conclusion
In conclusion, there is a lack of published evidence on real-world extent of and barriers to patient involvement in HTA in CEE countries. On the other hand, there are available guidelines and best practices that could be adopted in these settings. Twenty-five potential barriers of patient involvement in HTA were identified relevant for the CEE context. These will need further investigation to assess their relative importance and develop potential solutions and recommendations for action to address them.
Data Availability Statement
The raw data supporting the conclusions of this article is available upon request from authors.
Author Contributions
MD performed the scoping review. MD and IJ drafted the manuscript. MK, ZM, KT, BN, AZ, DDa DDe, FH, and ZK reviewed the paper. All authors have provided valuable contributions to the manuscript, read, and approved the final manuscript.
Funding
The HTx project has received funding from the European Unions Horizon 2020 research and innovation program under grant agreement N° 825162. This dissemination reflects only the author's view and the Commission is not responsible for any use that may be made of the information it contains.
Conflict of Interest
Authors IJ, AZ, BN, and ZK are employed by Syreon Research Institute. At the time of the study IJ was the President of the European Patients' Forum Youth Group and a Board of Trustees member at the EUPATI Foundation.
The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
Publisher's Note
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article, or claim that may be made by its manufacturer, is not guaranteed or endorsed by the publisher.
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Keywords: patient engagement, health technology assessment (HTA), barrier, central and eastern EU countries, potential
Citation: Dimitrova M, Jakab I, Mitkova Z, Kamusheva M, Tachkov K, Nemeth B, Zemplenyi A, Dawoud D, Delnoij DMJ, Houýez F and Kalo Z (2022) Potential Barriers of Patient Involvement in Health Technology Assessment in Central and Eastern European Countries. Front. Public Health 10:922708. doi: 10.3389/fpubh.2022.922708
Received: 18 April 2022; Accepted: 13 June 2022;
Published: 28 July 2022.
Edited by:
Chiara de Waure, University of Perugia, ItalyReviewed by:
Janet L. Wale, HTAi Patient and Citizen Involvement in HTA Interest Group, CanadaKalina Andreevska, Sofia University, Bulgaria
Copyright © 2022 Dimitrova, Jakab, Mitkova, Kamusheva, Tachkov, Nemeth, Zemplenyi, Dawoud, Delnoij, Houýez and Kalo. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
*Correspondence: Maria Dimitrova, bWRpbWl0cm92YSYjeDAwMDQwO3BoYXJtZmFjLm11LXNvZmlhLmJn
†These authors have contributed equally to this work and share first authorship