Navigating joint HTA, procurement, and fair pricing: evidence-based insights and practical recommendations - A meeting report from ISPOR regional conference in Warsaw, 2019

ABSTRACT

Introduction: The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) organized its first Central Eastern European regional meeting in 2019 in Warsaw, Poland.

Area covered: The scientific program of the two-day conference covered a broad range of topics presented from the perspective of the region. Specifically, the focus was on cross-country collaboration within different steps of health technology assessment (HTA) and the need for local HTA adaptations in decision-making.

Expert commentary: Attended by approximately 200 delegates from many countries and by several high level ISPOR leaders, the conference provided a valuable opportunity to exchange knowledge and strengthen the scientific network among experts from different stakeholder groups on issues specific to the region.

KEYWORDS:

• Central Eastern Europe
• collaboration
• conference report
• health technology assessment
• International Society for Pharmacoeconomics and Outcomes Research

1. Introduction

The Central Eastern European (CEE) region, one of the most dynamically developing regions of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) community, is characterized by unique features. Even though overall healthcare spending is lower compared to Western European (WE) countries, innovative pharmaceuticals are introduced at the same international public prices as those in high income countries [1]. However, a lower local willingness to pay these prices may result in the fact that without a non-transparent discount or a managed entry agreement (MEA) innovative medicines at international list prices are often not cost-effective in the CEE countries. Although more limited public resources should necessitate the implementation of an even more rigorous health technology assessment (HTA) system, HTA capacity building is less advanced compared to WE countries. Meanwhile, the overall health status is worse [2]. Consequently, CEE countries pay even more penalties for suboptimal coverage decisions.

With the title of ‘Navigating Joint HTA, Procurement, and Fair Pricing: Evidence-Based Insights and Practical Recommendations’, ISPOR Warsaw 2019 was the first regional conference of ISPOR in CEE to discuss issues relevant for this particular region. The conference which was attended by around 200 delegates and many high-level ISPOR leaders, was organized on the 27-28^th of March, 2019 in Warsaw, Poland. The program was comprised of two chapter meetings for both Poland and Ukraine and three sponsored educational symposia on the first day, which were followed by four plenaries held on the second day [3].

2. First day

The first educational symposium on MEAs sponsored by a multinational pharmaceutical company highlighted that providing access to innovative medicines for patients remains a challenge in CEE. MEAs not only share the risk of uncertainty between payers and manufacturers, but also offer opportunities to improve patient access while not conflicting with local cost-effectiveness and budget impact criteria. The second educational symposium sponsored by an umbrella organization for generic and biosimilar manufacturers on sustainable biosimilar policies argued that innovative biologic treatments are associated with significant hidden access restrictions that are implemented to ensure the financial sustainability of their reimbursement. Biosimilar medicines in such an environment should not be used for disinvestment, but to increase the patient access to these active compounds by increasing the number of treated patients [4]. The panel discussion aimed to identify demand-side policy tools that could ensure the long-term sustainability of the biosimilar market. The third education symposia sponsored by a Hungarian pharmaceutical company, which focused on schizophrenia, highlighted the relevance of local HTA adaptations in the healthcare decision-making process. Overall, the three educational symposia on the first day discussed practical challenges in the CEE region and the potential solutions.

3. Second day

At the beginning of the second day ISPOR Executive Director Nancy Berg greeted all delegates. In her speech, she confirmed the significance of the CEE region in the global strategy of ISPOR, reinforced ISPOR’s commitments to methodological programs and introduced ISPOR’s strategic plan aiming to re-invest revenues of the Society into regional conferences like this. The conference was opened by Zbigniew Krol, Deputy Minister of Health for Poland. He emphasized that patient needs should be at the center of healthcare programs and highlighted the new Polish initiative to create a pool of registries for the purpose of analyzing Big Data through Artificial Intelligence.

The aim of the first session was to reflect on the benefits of the early dialogues (EDs) between stakeholders and to align evidence requirements for drug authorization and coverage that focus on CEE countries. In his introduction, Finn Børlum Kristensen (former head of EUnetHTA) highlighted that dialogs between national stakeholders started at the regulatory level and continued with HTA early scientific advice [5,6]. Maggie Galbraith (Haute Autorité de Santé, France) explained the four key benefits of ED: quality, consistency, timeliness, and efficiency and highlighted that an ED takes 4–4.5 months [7]. Considering HTA capacities, Francis Arickx (National Institute for Health and Disability Insurance, Belgium) highlighted that although Belgium is a high-income country, even they face a shortage of experts in specialized fields like orphan drugs. This necessitates international collaboration, for example among the BeNeLux states, in conducting joint HTA that includes a joint writing of the reports. Tomáš Tesař (Union Health Insurance Fund, Slovakia) mentioned that from a payer’s perspective, EDs are expected to facilitate patient access. He claimed that the main challenge is to find policy-relevant comparators in different countries, considering that comparators selected at development may not have the same relevance at registration. Zbigniew Krol acknowledged the importance of a strong HTA Office like the Agency for Health Technology Assessment and Tariff System that has been in operation for more than a decade [8]. Overall, the speakers of the first session strongly underlined the usefulness of EDs in CEE.

The second session provided a multi-perspective discussion on the better use of existing- or currently developed health databases with respect to their availability, access and quality. Jose Valverde (DG CNECT, European Commission) overviewed the Big Data evolution at the EU level. He highlighted two EU level pillars for digital data transformation: 1) making it citizen-centered and 2) regulating the use of citizens’ healthcare data for secondary purposes. Hubert Zycinski (Ministry of Health, Poland) presented a national perspective by highlighting that e-prescriptions, e-dispensations, e-referral systems and patient internet health accounts have already been launched in Poland. Wim Goettsch (Associate Professor, Utrecht University) introduced the HTx project, a Horizon 2020 funded, 5-year collaborative research project on the next generation of HTA methods. He highlighted that the involvement of CEE countries will ensure the transferability of results. Guenka Petrova (Professor, Medical University of Sofia) provided information on a diabetes registry in Bulgaria which can be used for multiple purposes including survival analysis, quality control and evaluation on utilization data. Alexander Berler (Gnomon Informatics) suggested that no universal solutions should be expected when providing interoperability between platforms/databases. The panel discussion, moderated by Katarzyna Kolasa (Associate Professor, Kozminski University) discussed the potential use of Artificial Intelligence on health databases. Although this would be a longer-term concept, in HTx there is also an Artificial Intelligence/machine-learning work package. Nevertheless, it was highlighted that among the main conclusions of this session, Artificial Intelligence is a decision support tool and machines will not take over decision-making, though the use of novel methods of data analysis in healthcare research and decision making is inevitable.

The third session on medical device assessment and procurement focused on the need for the standardization of medical device value assessment in resource-restricted healthcare systems. Ernesto Nogueira (ValueConnected) mentioned that the medical device industry launches the highest number of new health technologies. Claudia Wild (Director, Ludwig Boltzmann Institute) highlighted that even high-income countries like Austria find EDs to be useful tools, given that innovations often enter such countries earlier. She stressed the importance of reducing the overlaps in Europe on conducting HTA, highlighting the usefulness of the EUnetHTA Planned and Ongoing Projects database [9]. Rok Hren (Assistant Professor, University of Ljubljana) introduced two main challenges, 1) methodology and 2) the lack of bridges for communication between the three key stakeholders: HTA offices, payers, and market authorization holders. Bence Takács (Health Economist, National Institute of Pharmacy and Nutrition) highlighted that due to recent changes, there is now communication between the Hungarian HTA office and the industry. He also spoke about the benefits of using joint assessments in order to reduce the workload, since the HTA Office can check these assessments quickly and work more on the economic sections. It was also highlighted that a better categorization of devices is necessary, and high-cost and high-volume devices should have as good evidence as drugs. Overall, the speakers of this session expressed the strong benefits of international collaboration in the field of HTA.

The fourth session discussed joint approaches in clinical evaluation, data generation, joint procurement and pricing negotiations of health technologies. Andrzej Rys, (Director, DG Santé, European Commission) provided an overview on 20 years of project-based EU level cooperation. He highlighted the similarities between collaboration of EU countries at the level of the European Union and the joint efforts of HTA offices in each country. He referred to the first joint procurement of a vaccine by 14 countries as an example to follow. Marcin Czech (Former Vice Minister, Ministry of Health, Poland) summarized the cooperative initiative of Fair and Affordable Pricing initiative with Visegrad V4 and Baltic countries, which aims to provide access to effective and affordable medicines. He argued, however, that such projects cannot replace country-level decision-making. Iga Lipska (National Health Insurance Fund, Warsaw) highlighted that during a joint procurement the focus should be on the value of products delivered to patients, not only on their prices, and that the process should be pre-defined, well-described and transparent. Stanimir Hasardzhiev (National Patients’ Organisation, Sofia) underscored the importance of EU-level cooperation for stability. He also articulated that patients need health now, not in the future. The panel discussion, moderated by Joanna Lis (Market Access Head, Sanofi), overviewed steps of collaboration. By citing the EMA central approval as an example, the concern was emphasized that joint assessment may also trigger joint coverage. Panel members, however, acknowledged that these decisions should be kept at a country level. It was emphasized that in order to respect solidarity, each country’s degree of purchasing and negotiation power should be taken into consideration so that countries do not have to pay for something they cannot afford. However, to achieve a better deal, being able to judge the value of the product is important, therefore countries with a light HTA will not be strong negotiation partners for the industry.

4. Summary

In conclusion, the conference provided an opportunity to engage in discussions with key stakeholders and leaders from the CEE region. Authors are looking forward to ISPOR’s continued support of such events where there is an opportunity to submit also abstracts on posters, podium presentations and panel discussions. Hopefully, such regional conferences will not supersede the increasing acceptance of program proposals involving CEE experts at the Annual European ISPOR conference on topics that are important for the CEE region.

Declaration of interest

A Inotai and B Németh are employees of Syreon Research Institute. Syreon Research Institute received speaker’s honoraria from Medicines for Europe and Gedeon Richter PLC. However, the authors have summarized their independent professional opinions and take full responsibility for any potential errors in the manuscript. The content of this paper, as well as the views and opinions expressed therein are those of the authors and not necessarily their affiliated organizations. No writing assistance was utilized in the production of this manuscript. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.