Multiple Myeloma in Central Europe and the Baltics: Supporting early and equitable access to care to improve patient outcomes is an Economist Impact white paper, commissioned by Johnson & Johnson Innovative Medicine. The report provides an independent analysis of multiple myeloma and its growing burden in the Central Europe and the Baltics (CE&B) region. The report evaluates the current care pathways for myeloma in 10 countries in the region (Bulgaria, Croatia, the Czech Republic, Estonia, Hungary, Latvia, Lithuania, Serbia, Slovakia and Slovenia), explore gaps in management, and identifies opportunities for improving patient outcomes.
Key findings from our research include:
The health burden of myeloma in the CE&B region is rising. The rise in the incidence of myeloma in Central and Eastern Europe is striking. Analysis of the Global Burden of Disease (GBD) data from 1990 and 2019 showed a 42.1% and 37.5% increase in the age-standardised incidence rate (ASIR) of myeloma in Eastern and Central Europe, respectively. Eastern Europe was among the top three regions experiencing the greatest increases in ASIR globally. Furthermore, while the global age-standardised death rate (ASDR) has been stable (among men) and decreasing (among women), the ASDR of myeloma in Central and Eastern Europe has increased by roughly 32% between 1990 and 2019 – indicating disparities in access to myeloma care. Data on the economic burden of myeloma in the CE&B region are sparse, and the impact of better holistic care on direct and indirect costs has not been well-studied.
The CE&B region lags Western Europe in myeloma outcomes. The difference in myeloma outcomes between Western Europe and the CE&B region is stark. A global study comparing different regions and countries for the myeloma Quality of Care Index (QCI) calculated the QCI in Western Europe to be 83.2 in 2019 as compared to 61.5 for the CE&B region. The QCI is a composite measure that takes into account four ratios: mortality to incidence, prevalence to incidence, Disability-Adjusted Life Years (DALYs) to prevalence, and Years of Life Lost (YLL) to Years of Life Lost due to Disability (YLD). The higher the score, the better the quality of care.
Lower investments in healthcare, delayed rollout of advances in diagnosis and treatment, and health systems challenges in CE&B contribute to this disparity. All our countries of interest have universal healthcare funded through wage contributions and/or taxation. However, government spending on healthcare as a percentage of the gross domestic product (GDP) in the CE&B region is lower than that of Western Europe. For example, in 2021, government healthcare spending in the United Kingdom (UK) equalled 10.3% of GDP per capita, almost double that in Hungary and Lithuania, at 5.3% and 5.4%, respectively.
Countries in the CE&B region also lag behind their European peers in terms of speed of access to innovative therapies. The W.A.I.T. Indicator 2022 Survey showed that the time duration between the European Medicines Agency’s (EMA) approval of innovative therapies and their availability as reimbursable treatment options for patients was longer in a majority of CE&B countries as compared to the European Union (EU) average. The shortest delay between market authorisation and patient access was 128 days in Germany. In Croatia, the delay was estimated at 499 days, but most CE&B countries recorded a delay greater than the EU average of 517 days. Of our study countries, the delay is greatest in Serbia (811 days).
Access to comprehensive multidisciplinary care in CE&B is limited. Given its many manifestations affecting different organs, strong multidisciplinary care is essential to improve myeloma outcomes. In the CE&B region, haematologists note that most engagement with other specialists, such as nephrologists and orthopaedic surgeons, is done on an informal basis. Multidisciplinary care is concentrated in a few major hospitals, with peripheral hospitals having less access to physiotherapists, psychologists and social workers. Experts also attest to inadequate palliative care services, again with a higher concentration of such services in major hospitals. Issues among myeloma survivors need more attention, especially among the emerging cohort of younger adults with myeloma who face unique challenges related to fertility, reproduction, finances and re-entering the workforce.
There is a lack of emphasis on value-based health care in the region, and robust disease-specific registries linked to payer data do not exist. Reimbursement for first-line treatment for myeloma is not in line with the European Haematology Association-European Society for Medical Oncology (EHA-ESMO) recommendations in many of the countries studied. In the relapsed setting, while individual innovative therapies are often reimbursed, there is a lack of coverage for optimal combinations of these innovative drugs, which have been shown to improve outcomes. Part of this challenge may be attributed to rigid methodologies applied during Health Technology Assessment (HTA). Broad value-based assessments that consider the impact of therapies on quality of life and economic productivity that go beyond budget line impact are lacking, leading to fewer reimbursements of innovative treatments.
Managed Entry Agreements (MEAs), which serve as cost/risk-sharing arrangements between pharmaceutical companies and payers, are primarily finance-based MEAs, involving price concessions and volume caps, especially when utilised to reimburse innovative therapies. Performance-based MEAs, when used, often lack high-quality data collection to study the impact of therapies on outcomes. The Czech Republic has a myeloma-specific disease registry, “Registry of Monoclonal Gammopathies”, which some centres in Slovakia are also contributing to. However, across the region, the development and implementation of myeloma-specific registries face challenges such as sustenance of funding, workforce allocation and confidentiality issues in data transfer. Thus far, myeloma-specific registries have yet to be effectively leveraged to support performance-based MEAs. Furthermore, data on the economic burden of myeloma are not included in these registries.
Based on these findings, we propose the following calls to action to improve the provision of comprehensive care for myeloma in the CE&B region:
Facilitate early diagnosis and specialist referral. Facilitating early diagnosis of myeloma in CE&B needs improved awareness among physicians and the public, combined with universal access to basic laboratory tests that can confirm suspect cases. Establishing guidelines for optimal timeframes for referral and treatment, while concurrently streamlining the referral pathway, would expedite myeloma management and reduce the likelihood of irreversible organ damage.
Invest in strengthening healthcare systems to improve the care pathway. Myeloma is a complex disease that requires specialised care from multidisciplinary teams, including haematologists, oncologists, nurses and other healthcare professionals. Investing in strengthening healthcare systems ensures that patients have access to these specialised services, leading to better management of the disease and improved patient outcomes.
Healthcare systems in CE&B countries should be optimised to provide better ambulatory care for myeloma. Better outpatient management of myeloma would hinge on supporting decentralisation of care with a hub-and-spoke model, developing a specialist nurse service for task-sharing, and a gradual move towards home-based administration of certain injectable drugs that can be given subcutaneously. These efforts, combined with improving the infrastructure for multidisciplinary care, including rehabilitation, palliative care and survivorship, will benefit the care pathway of not just myeloma but a broad spectrum of chronic diseases.
Improve access to innovation. A structured HTA is critical to the reimbursement decision-making framework, especially in countries with publicly funded healthcare systems. However, HTA processes in the majority of countries in CE&B rely heavily on Incremental Cost-Effectiveness Ratios (ICER) to determine reimbursement. Balancing the price of a new medication against its perceived value to patients and broader society is complex, yet restricting reimbursement decisions to ICER assessments is currently limiting patient access to guideline-recommended myeloma treatment options in CE&B. HTAs require a more holistic and multidisciplinary process that incorporates economic, social, organisational and ethical considerations to guide decision-making.
Improving access to innovative treatments will require more holistic HTAs and the development of better funding models. MEAs are often used in CE&B to facilitate access to more expensive therapies, but most of them are finance-based MEAs, which are based on confidential discounts and capping. There is a need for more performance-based MEAs to facilitate value-based healthcare. In addition, countries must strive to increase their attractiveness for clinical trials by improving their administrative capacity, supporting healthcare staff to run trials, and creating awareness among patients to improve enrolment.
Enhance estimates of the burden of myeloma and the impact of comprehensive care. Improving estimates of the burden of myeloma requires the development of more robust disease-specific registries across the region that link to payer data. To achieve this goal, government investment in technology, infrastructure and human resources for data collection is crucial. Mandating the reporting of myeloma to these disease-specific registries will also help improve the completeness of data. The development of automated data collection systems with the capability to transfer information from electronic health records directly into registries will improve the efficiency of data management. Efforts to link myeloma-specific registries to payer data, such as in the ongoing development of the Czech National Information System and the Lithuanian Cancer Registry, will facilitate the estimation of direct costs. More robust databases, which include both direct and indirect costs, can help to accurately estimate the impact of treatment. This will also serve as a foundation for the development of better performance-based MEAs and value-based payment models for innovation. The CE&B region can be a forerunner in this space, given the small populations, existing national cancer registries and unified payment data.
Strengthen stakeholder collaboration towards the delivery of high-quality care. Effective collaboration between all stakeholders, including policymakers, healthcare professionals, patients and pharmaceutical companies, is key to offering the best myeloma care. Experts describe how patient organisations across the region are pioneering the development of rehabilitation programmes with the support of health ministries, improving support for adults with myeloma, and seeking funding for the development of new programmes such as CAR-T cell therapy. Funding offered by pharmaceutical companies has facilitated the growth of the “Registry of Monoclonal Gammopathies” in the Czech Republic. Haematologists have been actively engaging with policymakers to improve access to innovative therapies. In the Czech Republic, they have taken the initiative to raise awareness among policymakers regarding game-changing therapies versus “me-too” regimens. Hungarian haematologists have developed national guidelines that elucidate the gap between EHA-ESMO recommendations and local access to treatments.
Explore our findings in the full report.
