O. Piniazhko, PhD, Director of HTA Department, SEC of Ministry of Health, Kyiv, Ukraine
B. Németh, PhD, Chair of ISPOR CEE Consortium and Principal Health Economist of Syreon Research Institute, Budapest, Hungary
M. Niewada, MD, PhD, Department of Clinical & Experimental Pharmacology Medical University of Warsaw and CEO HealthQuest, Warsaw, Poland
Managed Entry Agreements (MEAs), Coverage with Evidence Development (CED) schemes and Multi-Criteria Decision Analysis (MCDA) are tools for assessing mainly the highly innovative health technologies from more holistic and objective perspectives, including social impact, ethical and organizational dimensions and other issues, critical for proper evaluation of the innovation and its value for patients, caregivers, healthcare professionals, payers and society. Limited examples of utilizing those tools and methods are already available in the Central and Eastern Europe (CEE) region and further research is needed in order to apply them correctly within the context of CEE region healthcare settings, taking into consideration usually limited capacities and capabilities of stakeholders engaged in a process of assessment and appraisal.
During Virtual ISPOR Europe 2021, this topic was discussed in the CEE forum hosted by ISPOR CEE Consortium. The forum was moderated by Oresta Piniazhko and with performed fruitful discussion with speakers Bertalan Németh, Maciej Niewada and Oresta Piniazhko.
Dr. Bertalan Németh, Principal Researcher at Syreon Research Institute, Hungary, explained that many definitions exist in the literature regarding MEAs. He highlighted the importance of mentioning the concepts of risk-sharing to reduce uncertainty of payers, and confidentiality to facilitate differential pricing based on the value of the health technology as well. Naturally, it is conducted to increase patient access. Typically, MEAs can be grouped as financial-based or outcome-based agreements.
Regarding times of a pandemic, while it is not justifiable to delay policy decisions in priority areas, it is also not reasonable to ignore the implications of policy decisions. Methods have been introduced to reduce this uncertainty and to allow temporary, conditional reimbursement for example, coverage with evidence development. These CED schemes can be seen as a reasonable and scientifically sound compromise, offering an opportunity for reducing uncertainty by allowing temporary reimbursement while additional data are collected and assessed. However, the cost of real-world data collection and its burden on healthcare professionals should be better mitigated. CEDs can (and should) be built on existing data with special focus on linking patient records in different databases.
Dr. Németh highlighted that the implementation of MEAs, for example CED schemes can bring benefits for all stakeholders in CEE countries. Therefore, even in CEE countries MEAS, CED (and MCDA) are not just nice 'Buzzwords’. However, the reality must be considered, as there may be barriers in place that can seemingly prevent the widespread use of MEAs. Efforts should be dedicated to overcoming these barriers, and not giving up on MEA implementation.
Suggested reading: Kaló Z, Németh B, Zemplényi A. Can cost-effectiveness principles be ignored in urgent times?. Journal of Comparative Effectiveness Research. 2021 Oct; 11(1):7-9.
Dr. Maciej Niewada, Professor, Department of Clinical and Experimental Pharmacology, Medical University of Warsaw, Poland, presented the legal context and practices for MEAs, CED, and MCDA in Poland. Virtually all kinds of MEAs were introduced with reimbursement regulation in 2011. So far, the majority of these arrangements are mainly financial (i.e. discounts, clawbacks, caps and others) with listed prices unchanged, but a few performance-based were also introduced mainly in oncology. MEAs were subject to three extensive reports by INFARMA published in 2015, 2016 and 2018 (see: https://www.infarma.pl/raporty/ ). Main barriers for implementation of complex, outcome-based MEAs in Polish settings were identified and pragmatically elaborated. Polish HTA Agency reviewed MEAs in reimbursement applications for drugs used in oncological diseases within the specific reimbursement framework of Drug Programs in 2012-2018. The analysis showed discounts being most often proposed.
Certainly, details on MEAs are confidential and unavailable in the public domain, but it is well recognized that decision-makers are likely to adopt financial MEAs, mainly discounts or pay-backs (including upfront price reduction to adjust for non-responders). Outcome-based MEAs are still in the piloting phase. Coverage for responders only is feasible, especially for drugs available in Drugs Programs, which are associated with mandatory electronic data collection in SMPT (dedicated e-system for monitoring and reporting on patients enrolled).
CED was addressed in the recent regulation of the Medical Fund from 2020, which introduced so-called highly innovative medicines in rare diseases and cancer. These recently authorized drugs will be subject to effectiveness and safety analyses by HTA Agency based on the payer run registries. Consequently, local real-world data will be used to inform further reimbursement decisions.
Dr. Oresta Piniazhko, Director of HTA Department, State Expert Center of the Ministry of Health of Ukraine, provided a review for the first time of MEA implementation based on HTA development and roadmap in Ukraine. The article №791 of the Law of Ukraine № 2801-XII states that the aim of MEA concluding is ensuring the availability of innovative medicines for patients at the expense of the state budget. The perspective of providing HTA recommendations for MEA procedures is enshrined in all three legislative acts: the Law of Ukraine № 2801-XII, CMU Decree №1300, CMU Decree №61. The simplified scheme of the process is the following: HTA conclusion prepared by an authorized body (the HTA Department of the State Expert Centre of the Ministry of Health (MoH) of Ukraine) – negotiation – agreement – monitoring – prolongation. On the date of forum 3 December 2021 the authorized body has published 19 HTA conclusions with recommendations, 4 medicines of which contain a recommendation to consider the possibility of negotiating a MEA (https://www.dec.gov.ua/materials/derzhavna-oczinka-medychnyh-tehnologij-zayavy-ta-dosye/)
Ministry of Health of Ukraine already addressed applicants with a proposal to negotiate a MEA. The recently approved legislative acts for HTA and MEA in Ukraine are aimed at improving patients' access to innovative medicines at the expense of the state budget that meets best international practice and showed prospective implementation of use of these tools in practice.
In conclusion, the forum speakers discussed the main obstacles and barriers on the way to introduction of MEAs and how to overcome them and continued the forum with sharing experience and perspective to use MCDA as a complementary approach in the deliberation process. Maciej summed up that many qualitative MCDAs are operating already (e.g. Iowisz - Evaluation Instrument of Investment Motions in Health Care). MCDA was also supported by National Drug Policy 2018-2022, especially to inform coverage decisions on drugs in rare diseases in Poland. Additionally, quantitative MCDA, as a complementary approach to cost-effectiveness analysis, was proposed in Plan for Rare Diseases in 2021. MCDA methods are extensively studied and many local cases study publications are available, which can further facilitate implementation in reimbursement decision making.