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Peter Palumbo, Jordan Kruger, Christopher L. Robinson
No abstract is available for this record.
Mayeya Paul Mayeya
Results-Based Financing (RBF) has been promoted as an innovative health financing mechanism to improve service delivery and health outcomes in low-resource settings. However, evidence on its impact within devolved health systems remains limited. This study examines how RBF influences key performance indicators (KPIs) in Zambia's devolved district health services. An embedded multiple-case study was conducted across 12 districts in Northern Province, Zambia. Mixed methods were employed, combining qualitative interviews with 44 stakeholders and quantitative analysis of health facility data. The study focused on maternal and child health indicators, service utilization patterns, and health worker motivation following RBF implementation. RBF implementation was associated with improvements in several KPIs: maternal health outcomes (100% of facilities reported improvements), medicine availability (91%), and the quality of primary healthcare services (67%). Health worker motivation increased, with 42% agreeing and 24% strongly agreeing that RBF positively affected service delivery. Community-based volunteers responded positively to incentive structures. However, challenges included delayed fund disbursement (91.7% reported), inadequate funding (83.3%), and monitoring gaps (50%). RBF can enhance key health indicators in devolved systems when properly designed and implemented. Success depends on timely incentive disbursement, robust monitoring systems, and integration with existing community health structures. The study provides evidence for policymakers considering RBF scale-up in decentralized health systems.
Mayeya Paul Mayeya
Zambia has implemented significant health-sector decentralization since 1992, culminating in the devolution of district health services to local authorities under the 2016 Constitutional Amendment. Results-Based Financing (RBF) has been piloted as a performance incentive mechanism, but its institutionalization within devolved structures remains largely unexplored. This study explores the opportunities and challenges of embedding RBF within Zambia's devolved health system, with a focus on Northern Province. A qualitative case study design was used, involving forty-four participants from twelve districts. Purposive sampling selected health workers from provincial and district health offices, local authority representatives, and national stakeholders. Data collection included semi-structured interviews, document reviews, and observations, with thematic analysis conducted using NVivo 9. Most respondents (82%) reported involvement in RBF implementation, and fifty-three% believed that increased Constituency Development Fund (CDF) allocations improved district health services. Key benefits cited were increased accountability (81.8%) and greater community participation (77.3%). Challenges included insufficient funding (83.3%), delays in disbursing funds (91.7%), and limited understanding among local authority implementers. Infrastructure development and procurement of medical equipment were identified as primary areas for CDF improvements (56%). Respondents also agreed (53%) that the Ministry of Health and the Ministry of Local Government would support the institutionalization of RBF. Effective integration of RBF into devolved systems requires harmonizing policies between health and local government ministries, building capacity within local authorities, and aligning RBF with other domestic financing mechanisms, such as CDFs. A phased approach to integration, with clearly defined governance structures, is recommended to ensure sustainable scaling.
Ashar Prima, Dewi Gayatri, Yati Afiyanti, Christantie Effendy
Background: Indonesia faces a growing double burden of non-communicable diseases, particularly cancer. The latest data from the Global Cancer Observatory (Globocan) indicates over 408,661 new cases and 242,099 cancer-related deaths in 2022, with a projected 63% increase in the case burden between 2025 and 2040 without strategic intervention. Although a new legal framework through Health Law No. 17 of 2023 and the Minister of Health Decree (KMK) No. HK.01.07/MENKES/2180/2023 has mandated palliative care as an integral component of health services, its implementation still faces significant systemic barriers. Policy and Implications: This policy brief analyzes the disconnection between the policy mandate and on-the-ground reality, identifying critical gaps in accessibility, healthcare workforce capacity particularly among nursesand financing mechanisms through the National Health Insurance (JKN) program. The failure to effectively integrate palliative care not only causes unnecessary suffering for millions of patients but also burdens the health system with inefficient costs and suboptimal end-of-life care, reflected in the high "financial toxicity" experienced by patients. Recommendations: We recommend a four-pillar strategy: (1) Formalize and standardize palliative services within the JKN benefits package with a clear financing model to address regulatory ambiguity; (2) Develop a national competency-based palliative education and training strategy for all health workers, with a focus on empowering nurses in primary care; (3) Implement a decentralized and tiered palliative care delivery model centered on Community Health Centers (Puskesmas) to ensure equitable access; and (4) Launch a national public education campaign to destigmatize palliative care and increase awareness. Conclusion: The integration of palliative care is not merely an option but a strategic and ethical imperative for achieving Universal Health Coverage (UHC) in Indonesia. It is a cost-effective investment to improve patients' quality of life, support families, and ensure the sustainability of the national health system in facing future non-communicable disease challenges.
Mohit Pandey, T. R. Dilip, Amit Chopade, Shailesh V. Shrikhande · 5 authors
No abstract is available for this record.
Javier David Benítez Fuentes, Rodrigo Lastra del Prado, Miguel Borregon-Rivilla, Alicia de Luna Aguilar · 15 authors
Despite significant advances in oncology, cancer care globally continues to face critical challenges, including stark disparities in access, insufficient preventive focus, fragmented primary health care (PHC) integration, unsustainable financing models, workforce shortages, and inadequate community involvement. This paper revisits the Alma Ata Declaration's principles-health equity, universal access, preventive care, and community participation-as a conceptual framework to address these persistent issues in cancer care. We highlight opportunities to strategically integrate oncology services within strengthened PHC systems, balancing centralized specialist resources with decentralized community-based care. Evidence from diverse settings illustrates how reinforcing PHC infrastructures enhances preventive measures, early detection, and survivorship care, thus mitigating geographic and socioeconomic disparities. Sustainable financing mechanisms and targeted workforce strategies, including task-shifting and multidisciplinary training, are proposed as essential components. Effective community engagement models demonstrate improved care relevance, acceptance, and outcomes. Additionally, we emphasize the critical role of health policy alignment with universal health coverage objectives, robust pharmacoeconomic evaluations, and evidence-based national cancer control plans. Integrating Alma Ata's principles into contemporary oncology provides a viable, scalable model to advance equitable, accessible, and sustainable cancer care globally, laying the theoretical groundwork for future research initiatives and informed policy development.
Rolf H. Weber
Decentralized Autonomous Organizations (DAOs), as open, scalable, and self-organized networks, do not easily fit into the available corporate legal forms in Swiss law. The completely new regulations for DAOs developed abroad have some merits; however, they involve lengthy legislative efforts and initially create legal uncertainty. In contrast, Swiss association law appears largely suitable for application to DAOs, containing appropriate rules regarding legal personality and limitation of liability. However, some specific adjustments should be made to the incorporation documents (e.g., ballot voting, multiple voting rights, and/or the design of management competences). Consequently, there is no need for a specific DAO regulation in Switzerland.
Angela Hemesath, William M. Tian, Bryce W. Polascik, Suzanna Joseph · 10 authors
Purpose:. To combine the perspectives of health and commercialization experts on the ethical and regulatory needs for non-fungible token (NFT) implementation in healthcare.Design:. PerspectiveMethods:. For a multidisciplinary perspective by an interdisciplinary group, current event articles and research articles were interpreted and assessed.Results:. Health data has become fragmented and disorganized, resulting in poor accessibility, increased administrative costs, and integrity vulnerability. Healthcare is uniquely suited to adopt blockchain and NFT technology as potential solutions. The incorporation of blockchain technology may offer multiple improvements in data-sharing through consensus, tokenization, and decentralization. However, the current regulatory infrastructure to support blockchain is poorly defined.Conclusions:. Healthcare NFTs would revolutionize patient control over their health data and promote more ethical transparency of data ownership while also reducing administrative security costs. However, blockchain poses unprecedented requirements of healthcare regulation within the unique realms of patient privacy and data ownership. Large-scale implementation of blockchain cannot be achieved without regulatory collaboration.
Jaime Espín, David Epstein, C Martin Saborido
No abstract is available for this record.
K. Jayasankara Reddy
No abstract is available for this record.
Roberto Fantozzi, Stefania Gabriele, Alberto Zanardi
• A reform in 2023 introduced new criteria for allocating healthcare funding in Italy. • Socio-economic variables were included among the criteria for allocating funding across Regions. • We simulate the new scheme and compare it with an alternative in which age and socio-economic indicators are jointly considered in estimating health needs. • It turns out that more resources would be allocated to the Regions with greater deprivation. This paper discusses a reform recently implemented in the Italian National Health Service, aimed at adding some socio-economic indicators to the criteria adopted for allocating healthcare funding to Regions. The reform is based on international experience in healthcare financing in decentralized settings and provides a case study of special interest since Italy is a country with significant territorial disparities and severe budget constraints. The paper first discusses the long-standing debate between Italian Regions which led to the reform. Second, it reviews the main features of the reform which provides for the inclusion of socio-economic indicators via a simplified formula. Moreover, a possible revision of the reform is proposed, fully exploiting the information on the heterogeneity of health needs according to age and socio-economic indicators. By integrating the information on deprivation inside the risk adjustment mechanism, the weight of the different drivers is determined by the distribution of needs and not on a discretionary basis. Simulating the proposed revision suggests that more resources could be allocated to the Regions with higher levels of deprivation compared to a scenario that closely replicates the reform.
М. М. Бабенко, К. Л. Косяченко
The aim: to develop a typology of the current management systems for health technology assessment (HTA) based on the identification of typological features in order to scientifically and practically substantiate a typological model that combines the most stable properties and can be implemented in a variety of modifications, taking into account the dynamic development of the health care system (HCS). Materials and methods: The study used scientific publications, official information from the websites of national or regional bodies/agencies, international organizations on HTA, reports, databases and official documents of the World Health Organization (WHO). The research used the following methods: the system analysis, content analysis, institutional analysis, structural-functional analysis, generalization, comparison, systematization, classification, synthesis, typology, and modeling. To conduct a typological analysis, 34 countries were selected in which the HTA has been implemented in the decision-making process for the use and financing of medical technologies (MTs). Research results. An institutional analysis of national HTA systems was conducted. The status of HTA in the national health care systems of the selected countries and, in particular, the role of HTA in the decision-making process regarding the use of certain MTs were studied. The author analyzes the institutional capacity of the HTA system (availability of a special authorized body, level of centralization/decentralization, financing, regulatory framework and human resources). The functionality and areas of activity of HTA bodies (organizations), the level of accountability, openness and interaction with various stakeholders are analyzed. The systematization and generalization of foreign experience made it possible to conduct a typological analysis by characteristic features). Four types of HTA management systems are identified (starting, centralized, decentralized, and balanced). Conclusions: The study identifies and analyzes the areas of activity of the bodies/organizations in most countries of the world that carry out HTA in terms of their mission, vision and functionality, as well as assesses the level of their openness and interaction with various stakeholders. The scientific generalization and systematization of modern approaches and models of HTA systems made it possible to typologize them on the basis of certain characteristic classification features
Damilare Peter Oyinloye, Jingyue Li
Blockchain is a distributed ledger where partici-pating users with diverse and varying trust levels come to an agreement on the content of a ledger using a mechanism called consensus protocols. As the interest and adaptability of the blockchain technology deepens, the quest for consensus protocols that strike a harmonious balance between throughput, energy efficiency, and security remains a priority. This paper introduces Proof of Success Rate (PoSR), a past behaviour-based consensus protocol, where past successes and failures of a node determines its fate in the network. PoSR leverages the success rating of nodes as a dynamic metric to finely scale the network difficulty of nodes. Nodes with significantly high success rates will find the target hash faster and do less work in mining compared to those with lower rates. We simulated PoSR on SIM-P, a flexible consensus protocol simulator and evaluated its performance using through-put, energy consumption and resistance against 51% attacks as metrics. The performance of PoSR was subsequently compared to three (3) existing consensus protocols (PoW, PoRX, and poC) that were previously simulated on SIM-P. The experimental results shows that PoSR has a higher throughput, records lower energy consumption, and demonstrates better resistance against a 51% attack than PoW. This result highlights the effectiveness of the success rate in PoSR, which scales network difficulty based on the nodes' success rates, allowing nodes with higher rates to find the target hash faster. Additionally, the result shows that it is more expensive for a 51% attacker to succeed in PoSR than in PoW.
Maximilian Unfried
In an era marked by scientific stagnation, Decentralized Science (DeSci) challenges the inefficiencies of traditional funding and publishing systems. DeSci employs blockchain technology to address the misalignment of incentives in academic research, emphasizing transparency, rapid funding, and open-source principles. Centralized institutions have been linked to a deceleration of progress, which is acutely felt in the field of longevity science-a critical discipline as aging is the #1 risk factor for most diseases. DeSci proposes a transformative model where decentralized autonomous organizations (DAOs) facilitate community-driven funding, promoting high-risk, high-reward research. DeSci, particularly within longevity research, could catalyze a paradigm shift towards an equitable, efficient, and progressive scientific future.
Els Torreele, Heather Sherwin
The Covid-19 vaccine market concentration and hoarding that left many Global South countries unable to access vaccines in a timely manner have led to calls to expand and decentralize manufacturing capacity as a critical element towards more equitable access to vaccines globally Multiple initiatives are underway to build manufacturing infrastructure in low-and middle income countries (LMICs). However, without proper attention to who owns and controls the production and underlying technologies, there is a risk that well-meaning donor investments reinforce market dynamics that favour a handful of major international producers over truly local efforts This is particularly relevant for the African Vaccine Manufacturing Accelerator (AVMA), the new US$ 1Bn financing instrument approved by the Board of Gavi, the Vaccine Alliance, in December 2023
María Teresa Jiménez-Buñuales, Pilar León Sanz, Paulino González Diego, María Leonor González Menorca
In Spain, the National Health System, of a public nature, provides care to Spaniards and other residents in Spain. It is a system adapted to the State of Autonomies, decentralized. Each Auton-omous Community has legislative capacity in its organization and management. We studied the agreement of private hospitals by the public system in La Rioja, an Autonomous Community of Spain located in the North of the Iberian Peninsula. Documentary sources from the General archives of La Rioja, the Institute of Riojan Studies, municipal funds (Logroño, Haro, Calahorra), and in-formation from catalogs and censuses of hospitals from the Ministry of Health, National Institute of Statistics, and Institute of Statistics of La Rioja have been reviewed. The hospital model of La Rioja is characterized by a predominance of public beds over private ones, although there is a growing trend in the number of private beds from 2013 onwards, due to the incorporation of two conva-lescent socio-health hospitals. A characteristic element of La Rioja has been the promotion of pub-lic-private collaboration, conceived as a strategic alliance. Regarding agreements, in La Rioja there has been a specialization in the socio-health space, whose formulas have been the management service contract and the concession of work. The development of the Health System in La Rioja, 1986-2019, has been determined by the presence of a mixed public-private hospital system and a progressively lower dependence on specialized hospitals from other Autonomous Communities.
Jia Lin Hau, Erick Delage, Mohammad Ghavamzadeh, Marek Petrik
Optimizing static risk-averse objectives in Markov decision processes is difficult because they do not admit standard dynamic programming equations common in Reinforcement Learning (RL) algorithms. Dynamic programming decompositions that augment the state space with discrete risk levels have recently gained popularity in the RL community. Prior work has shown that these decompositions are optimal when the risk level is discretized sufficiently. However, we show that these popular decompositions for Conditional-Value-at-Risk (CVaR) and Entropic-Value-at-Risk (EVaR) are inherently suboptimal regardless of the discretization level. In particular, we show that a saddle point property assumed to hold in prior literature may be violated. However, a decomposition does hold for Value-at-Risk and our proof demonstrates how this risk measure differs from CVaR and EVaR. Our findings are significant because risk-averse algorithms are used in high-stake environments, making their correctness much more critical.
Precilia Kong, Chang Lu, C. Reyes-de la Cruz
No abstract is available for this record.
Anna Kollerup, Sarah Wadmann, Toke Bek, Jakob Kjellberg
No abstract is available for this record.
Don Husereau, Terrence Sullivan, Harriet Feilotter, Marcio M. Gomes · 9 authors
AIMS: The Canadian province of Ontario provides full coverage for its residents (pop.14.8 M) for hospital-based diagnostic testing. Historical governance of the healthcare system and a legacy scheme of health technology assessment (HTA) and financing has led to a suboptimal approach of adopting advanced diagnostic technology (i.e. protein expression, cytogenetic, and molecular/genetic) for guiding therapeutic decisions. The aim of this research is to explore systemic barriers and provide guidance to improve patient and care provider experiences by reducing delays and inequity of access to testing, while benefitting laboratory innovators and maximizing system efficiency. MATERIALS AND METHODS: = 2). The forum considered evidence of good practices in adoption, implementation, and financing laboratory services and identified barriers as well as feasible options for improving advanced diagnostic testing in Ontario. RESULTS: Overarching challenges identified included: barriers to define what is needed; need for a clear approach to adoption; and the need for more oversight and coordination. Recommendations to address these included a shift to an anticipatory system of test adoption, creating a fit-for-purpose system of health technology management that consolidates existing evaluation processes, and modernizing the governance and financing of testing so that it is managed at a care-delivery level. CONCLUSIONS: The proposals for change in Ontario highlight the role that HTA, governance, and financing of health technology play along the continuum of a health technology life cycle within a healthcare system where decision-making is highly decentralized. Resource availability and capacity were not a concern - instead, solutions require higher levels of coordination and system integration along with innovative approaches to HTA.
Chris BONNETT, Tania Stafinski, Evelinda Trindade
Objective: overview of Canadian practices for regulating, financing, and funding prescription drugs. Canada provides universal health coverage for hospital and physician services but excludes universal insurance of prescription medicines. Public plans provide 42% of financing, while private drug insurance covers 35% of expenditures and over 60% of Canadians – mainly through their employer. Canada has relatively high out-of-pocket expenditure (19% of spending) and is currently the tenth largest pharmaceutical market, following Brazil. It is wrestling with inequitable coverage, low use of biosimilars, and affordability and sustainability issues driven by rare disease drugs. Both federal and provincial/territorial governments and their agencies have roles in setting policy and regulating drug prices and costs. These include the federal Patented Medicine Prices Review Board (PMPRB) which ensures prices of new patented drugs are not excessive; the pan-Canadian Pharmaceutical Alliance (pCPA) which negotiates lower patented, generic and biosimilar drug prices on behalf of member jurisdictions; and the Canadian Agency for Drugs and Technologies in Health (CADTH) which provides most public drug plans with robust health technology assessment (HTA), including clinical, economic and budget impact analyses of new drugs. Private drug insurers tend to follow government initiatives, including the use of HTA and confidential Product Listing Agreements. Conclusions: Pharmaceutical coverage in Canada is a “patchwork” of more than 100 public drug plans and 100,000 private insurance plans. As such, it creates gaps in coverage which result in inequitable access and high out-of-pocket drug expenses for some Canadians. Canada’s decentralized health system and the absence of universal drug insurance, among other factors, likely contribute to higher per capita drug expenditure relative to comparable nations that have broader, publicly-funded universal health insurance and more rigourous policy and program strategies.
Nathan A. Yates, Jennifer M. Hinkel
The authors review the literature surrounding the economics of rare disease drug development and access before advancing the case for novel approaches to funding treatments. To fund the next stage of rare disease drugs, which will likely center on gene therapies and molecular medicine, they discuss value frameworks as well as patient-led models of finance, and how these may fit into the existing frameworks in the US to incentivize rare disease drug development and access. “Rare Diseases”, sometimes called “Orphan Diseases”, are those with low prevalence; a systematic review comparing definitions of Rare Disease found the prevalence definition averages around one case per 1700 people, although a common US definition often cites fewer than 200,000 people must be affected by a disease for it to be considered rare.1 Despite low prevalence of each disease fitting this definition, about 1 in 10 Americans, or 30 million, are thought to have been diagnosed with a rare disease, compared to the overall prevalence of much more common diagnoses such as diabetes which affects 10.5% of the US population. Striking in contrast, though, is the consideration that within the “rare disease” population exist 7000 or more distinct diagnoses. From a drug development perspective, the primary challenge to this market remains the balance of funding R&D while market opportunities on the commercialization end remain constrained by small patient populations (i.e., small market sizes). Financial incentives for rare drug development in the US were codified in the 1983 Orphan Drug Act (ODA), which includes tax credits, waives Food and Drug Administration (FDA) user fees, and increases marketing exclusivity for rare indications. A mosaic of programs now exists in the US to de-risk and incentivize rare disease drug development, including voucher programs (e.g., for rare pediatric diseases), grant programs (e.g., enabled under the Rare Disease Act of 2002), Small Business Innovation grants/contracts, targeted research efforts (e.g., Rare Cancer Moonshot) and others mentioned below, and regulatory pathways (e.g., Accelerated Approval). Outside of the US, incentives for development, as well as patient access to resulting treatments, vary widely by country and region. To explore economics and value in rare disease drug development, the authors consider the historical context, current trends, present-day landscape, including insurance coverage and reimbursement trends and “value frameworks” as well as patient-led models of finance, and examine novel methods for rare disease funding and access as well as the “patient–economist” perspective given that both authors are economists and rare disease patients. The current trend of patient-led activism in rare disease financing and discovery is not new, and continues the work led by the National Organization for Rare Disorders (NORD) in the 1980s that resulted in passage of the ODA in 1983. Key ODA provisions include 7-year market exclusivity for orphan drugs, tax credits, development grants, fast-track approval, and waivers of PDUFA fees (a category of FDA user fees for drug developers). Some debate exists regarding whether increased development and discovery in rare disease over the past several decades, particularly with regards to repurposed molecules, is due chiefly to the ODA or to other market and landscape forces. While some researchers have argued that the ODA has not significantly impacted market exclusivity for drugs that would have patent protection regardless of the legislation, others have shown the increase of rare disease approvals as an indicator of the ODA’s relative success.2, 3 Meanwhile, as the rise of “precision medicine” based on molecular diagnostics and next-generation sequencing technologies influences clinical decision-making and patient population definition, potentially more and more diseases, including subindications of more prevalent conditions, can be categorized as “orphan”; as an example, nearly half of requested orphan designations are for rare cancers.4 Rare disease products are comparably more available to US patients than to patients in other countries (primarily due to broad FDA labeling), yet US patients still face a number of barriers, financial and otherwise, as detailed in a 2020 report commissioned by NORD. Given the rising volume of rare disease designations of drug candidates, with 753 in 2020, pressure for market and patient access to rare disease drugs is likely to accelerate in the coming years, along with significant debate as to what constitutes “value” in a rare disease drug. Haendel et al. state that there are approximately 7000 rare diseases according to common classification procedures, but the authors estimate the actual number is closer to 10,000.5 The sheer number of rare diseases, not to mention the paucity of research available on many of these illnesses, creates enormous challenges in drug development. Since much pharmacological research is undertaken by for-profit entities, a large number of rare diseases are never investigated for treatment simply because they afflict so few people. Investing millions of dollars into research to target a disorder affecting 50 people across the globe is unlikely to provide the return on investment sought by the biopharmaceutical industry. Shareholders of public biopharma firms represent another hurdle to pursuing rare disease drug development as such investors are often focused solely on financial returns. Much rare disease drug development resides within smaller biotechnology companies. These firms, often privately held, face fewer demands for immediate earnings and have lower overhead costs than global pharmaceutical companies. After developing a promising drug candidate, such a biotech may be acquired or choose to go public to access the resources necessary to complete clinical trials. Typically, though, we see rare disease treatments marketed by major pharmaceutical companies only after the acquisition of an original developer. This process is certainly unique and frequently suboptimal overall. A recent trend is in patient groups, or in some cases individual patient advocates, seeking to create their own collaborations, funds, and research networks to address rare diseases. In some cases, these patient-led models are blending “traditional” venture-backed biotech approaches with philanthropic funding, cooperatives, and other models to create new and innovative means to accelerate discovery and approval, simultaneously seeking to prioritize the patient perspective. The Rare As One Network, for example, funded by the Chan Zuckerberg Science Initiative, backs 30 grantee patient organizations that are taking on activities usually left to venture-backed biotech, such as pharma partnership development, launching and maintaining clinical registries, building biobanks and tissue repositories, and starting clinical trials. Often, these novel approaches to early-stage development financing are paired with innovations in the development pathway, including “decentralized” or “just in time” clinical trials that allow trials to be opened on a one-off basis across a network of satellite sites so that patients can be accrued without having to travel to a central location, which previously limited trial access and accrual to large research hospitals. Other patient-led innovations include networks for data sharing and analysis, including RARE-X, NORD IAMRARE, and Genetic Alliance PEER, that enable patients to share personal health data with researchers and industry. Few academic publications have thoroughly addressed US insurance coverage and reimbursement trends for rare disease, although the topic is a frequent area of focus for private-sector research and publication. A 2020 study from University of Michigan found that while spending on rare disease therapies increased from 2013 to 2018, patient out-of-pocket costs did as well, nearly doubling from $486 to $866 per year.6 However, coverage across plans is highly variable, with restriction frequency for orphan drugs ranging from 11% to 65% in a 2019 study.7 An earlier study found that 93% of orphan drug approvals are covered by payers, but formulary management and utilization management may lead to restrictions, high cost shares, and similar mechanisms that impact access to such products.8 Assessing the “value” of rare disease treatments presents numerous challenges and is a topic of debate not only in the US but in countries with more formalized Health Technology Assessment (HTA) programs that determine insurance coverage or approval for new therapies. Small population sizes in clinical trials, limited experience with the best outcomes or endpoints to measure in such trials, the lack of existing treatments for many rare diseases, limited validated quality-of-life measurement instruments for rare disease populations, and challenges to project forward how new treatments will impact health utilization and other costs make HTA particularly difficult. The rare disease community has been vocal in criticizing use of measures such as cost-per-QALY (quality-adjusted life year), a perspective that has been supported by research demonstrating the insufficiency of such metrics in rare disease and the risk that applying them will lead to unjust policies for rare disease patients.9 Health economists have encouraged the use of broader elements beyond those typically included in cost-per-QALY assessments when evaluating the value of rare disease therapeutics. Alongside innovation coming from patient-led research and development groups, a number of academic and nongovernmental organizations have proposed or pioneered innovative funding models for rare disease drug development as well as business models that reduce risk and channel financing more efficiently. As a real-world case study, academics and venture capitalists alike have pointed to BridgeBio, a rare disease drug company with a portfolio model that reduces risk of developing only one molecule as traditional biotech companies often do. Other novel methods include crowdfunding, “venture philanthropy” that blends venture capital’s search for returns with a philanthropic and social-impact mindset, incentive prizes, disease-specific venture funds, and social impact bonds (SIBs). From the pricing perspective, researchers have proposed a number of mechanisms to allow for risk sharing, including value-based or outcomes-based contracts or cost-based yardstick pricing.10 While the high price of rare disease therapies can create “sticker shock” among the public and politicians, the authors believe it is important to consider the relevant context, emphasizing previous health economic research that has cautioned against applying an overly utilitarian view to rare disease drug development and patient access. The long-term economics and value of rare disease treatments are particularly critical to understand as they evolve over time and not be measurable (although they are possible to model) at the time of drug approval. For example, the cost of a single dose of Zolgensma (onasemnogene abeparvovec-xioi) is over $2.1 million. The uproar following the approval of this drug was immediate, ferocious, and focused singularly on the price. A more comprehensive analysis, however, reveals important details about the economics of the treatment. While onasemnogene abeparvovec-xioi is a one-time treatment, the alternatives require continued doses for life. Evrysdi (risdiplam) costs $3.4 million for one decade of treatment, and Spinraza (nusinersen) costs over $4.1 million for 10 years of therapy, plus the cost of spinal injections. The full scope and cost of all available drugs to treat a disease, as well as the secondary costs and benefits such as avoiding additional hospital stays or reducing other therapies, should be fully assessed before declaring a treatment “unaffordable” in the court of public opinion. Ultimately, the authors would agree to prioritize the development of rare disease drugs that cure or significantly alter the trajectory for the most serious and debilitating conditions affecting humanity, regardless of the size of population affected. We should always value patients by putting them at the center of development, approval, and treatment decisions. On a macro level, drugs that dramatically reduce the lifetime cost of treating rare diseases are also worthwhile to pursue, as doing so could free up capital for investment in other areas of drug discovery and improve sustainability of treating rare diseases in global markets. A two-tiered system whereby some people have access to rare disease drugs and others suffer without treatment is not ethical, but the solution is not to shortsightedly restrict development/approval of expensive medications. Instead, we should focus on economic solutions and innovative outcome-based frameworks that enhance access for all while maintaining strong incentives for research, development, and commercialization of products that can have positive life-altering and life-saving impact. While investment in rare disease therapies has increased over the past four decades, both the number of new drug candidates for and the total number of investment dollars in rare disease—whether coming from “traditional” venture capital and private equity sources, or from new philanthropic, patient-led, and social-impact based backers—are likely to continue an upward trajectory. Alongside funding and development emphasis, rare disease patients and their families, with the present authors as an example, are increasingly taking roles in drug research, policy advocacy, biopharmaceutical business, market access, and financing innovation in ways that meaningfully advance the market for rare disease research, drug development, and drug commercialization. While numerous rare diseases remain without current treatment, the past decade has seen advancement for a number of conditions that were previously thought to be “untreatable”; these “moonshots”—ambitious efforts to treat rare diseases—have paved the way for more economically viable models. There is also a growing consensus that rare disease treatments bring significant value to society, despite the applicability of any one molecule to a relatively small population. With advances in financial innovation and patient-led research, these authors are optimistic that the market for rare disease drugs will continue to attract outside investment, although they acknowledge that market access innovations will increasingly be needed to meet patient demand for global access to the drugs that result from such investment. No funding was received for this work. The authors declared no competing interests for this work.
Wu Zeng
Improving health system efficiency for better health outcomesThe World Health Organization (WHO) placed "value for money" as an important element to move countries towards Universal Health Coverage (UHC) in its 2010 World Health Report, stressing that health systems should pay attention to improving efficiency of using existing funding for better health outcomes (1).A significant amount of health resources are wasted, which would saving millions of lives if they were used wisely and efficiently.The efficiency of using available funding, along with the innovations to generate new resources for health, is critical for achieving UHC.This special series compiles studies that aims to improve the health system efficiency in Afghanistan, Nigeria, Ukraine, Haiti, Botswana and Zimbabwe.Zeng and colleagues evaluate the cost-effectiveness of two financing programs in Nigeria: one is performance-based financing that tied payments to performance indicators, and the other is decentralized facility financing program where payments to health facilities were unconditional.Both programs show promising results in providing essential maternal and child health services.The result suggests that financial incentives can improve the use of underutilized essential services in low-and middle-income countries.Shepard et al. investigate the cost-effectiveness of a similar program in Zimbabwe.The evaluation combined the impact of the performance-based financing on both quality and quantity of target health services.The study shows that the program was associated with the improvement of use of key maternal and child health services.However, the impact of the program on quality improvement was minimal.Overall, the program demonstrates a cost-effective health system investment.In the same country, Mutasa et al. focus on the effect of the performance-based financing program on quality of care only.The result is consistent to what reported in Shepard's article, showing that the performance-based financing program only has an impact on quality for care for institutional deliveries.However, there are a wide variety of contextual variables that may affect the program's effectiveness, including population wealth and availability of human resources.The article highlights that incentive payment programs should be accustomed to the local context to maximize their potential.At the health facility level, Avila and colleagues examine the efficiency of providing antiretroviral treatment (ART) services in Botswana and find a wide range of performance in delivering ART services in the country.One important finding is that the performance of ART services is lower in hospitals than health posts, suggesting the potential to scale up the ART to the lower level of health systems to improve the coverage of the care.The authors suggest decentralizing ART services to primary health care delivery points for better efficiency.In Haiti, Mekonen and colleagues evaluate health facility efficiency of delivering primary health services.The study identifies the critical association between health workforce and the efficiency of health facilities in the county.As many countries are facing a shortage of health human resources and challenges of disruptive medical health services, this study sheds light on strengthening health human resources as a means to improve the efficiency of primary health care systems.In Ukraine, Avila reviews the evolvement of health financing policies in the country.The article highlights the weaknesses of health financing system in the past, and describes the process of health financing reforms that have been undertaken.The review highlights the critical role of strategic purchase as a lever to overhaul the health system in Ukraine to promote the efficiency improvement.In Afghanistan, Farewar and colleagues take the angle of equity to assess the disparity of use of health care services.After health care reforms for more than a decade, there exist inequity in using health care services.The inequity is more salient in using inpatient care and private health services than outpatient care.As Afghanistan still heavily relies on the private sector to provide essential health services, addressing the concern of unequal access to care becomes a prominent issue in the country.In summary, efficiency is an important topic in strengthening health systems, and approaches to evaluating the efficiency have been evolving.The articles that are compiled in this specifical series provide empirical evidence of efficiency evaluation at the health facility and health system levels, and methodology to conduct the evaluation.
Marina Raijche Mattozo Rover, Emília Baierle Faraco, Claudia Marcela Vargas-Peláez, Cláudia Flemming Colussi · 7 authors
This case study aimed to characterize the Specialized Component of Pharmaceutical Services (CEAF) organization in four Brazilian states from diverse regions of the country. Data were collected with representatives of CEAF management from states in different regions, who answered a 21-question questionnaire on scope, organization, financing, hurdles, and facilitators. This information was complemented with data from national health surveys, DataSUS, the applied resources, and socioeconomic indicators. Differences were observed between states on issues such as the proportion of users and the decentralization of services. These characteristics seem to be related to the level of development concerning the socioeconomic indicators used. Advances in access to medicines were highlighted, despite the difficulties complying with the CEAF's objectives, such as insufficient resources, the qualification of human resources, and the provision of necessary visits and exams. The results point to advances, different forms of organization and highlight the need for more in-depth studies on the clinical and economic outcomes achieved as a strategy to outline solutions to achieve the comprehensive and equal care for users.