Counterfeit and substandard medicines remain a major global public health threat, with the World Health Organization estimating that up to 10% of medicines in low- and middle-income countries are falsified or substandard, exceeding 20% for some therapeutic classes in sub-Saharan Africa. Blockchain technology, with its decentralised, immutable, and transparent digital ledger architecture, has been proposed as a promising solution for strengthening pharmaceutical supply chain traceability, yet little is known about the readiness of community pharmacies in Nigeria to adopt it. This study assessed the knowledge, current practices, and implementation readiness of blockchain-based traceability for counterfeit medicine prevention among community pharmacists in Bayelsa State, Nigeria, and examined the barriers and enablers influencing adoption. A descriptive cross-sectional survey design was employed, using a structured, validated questionnaire administered electronically through the Association of Community Pharmacists of Nigeria (ACPN), Bayelsa State Chapter. A total of 119 valid responses were obtained, exceeding the minimum sample size derived from Yamane's and Cochran's formulae, representing a 79.3% response rate. Data were analysed using descriptive and inferential statistics, including Chi-square tests. Findings revealed that although 71.4% of respondents had heard of blockchain technology, mean knowledge scores across core blockchain concepts were uniformly low (1.26-1.72 on a 5-point scale), reflecting a substantial awareness-comprehension gap. Authentication practices were overwhelmingly manual, with 77.3% relying on visual inspection of NAFDAC numbers and only 9.2% using digital scanning; 68.9% of pharmacists had encountered suspected counterfeit medicines in the past year. Implementation readiness was below average (Mean = 2.70/5.0), driven by strong training willingness (Mean = 3.74) but deficient infrastructure (Mean = 1.80). Resistance to change and high cost were the leading barriers, while user-friendly applications and mandatory regulation were the strongest enablers. The study concludes that community pharmacies in Bayelsa State are not yet structurally ready for blockchain-based traceability, although a receptive attitudinal environment and strong professional motivation exist to support a phased transition. It is recommended that NAFDAC and the Pharmacists Council of Nigeria develop a phased regulatory framework and integrate blockchain literacy into continuing professional development, that government prioritise infrastructure investment, and that technology developers design mobile-first, user-friendly, offline-capable systems, to enable a coordinated transition toward blockchain-enabled pharmaceutical traceability in Nigeria.
Objectives: The authors explore how large pharmaceutical corporations may integrate emerging decentralized technologies-such as blockchain and decentralized autonomous organizations (DAOs)-within their merger, acquisition and partnership frameworks, and how these strategies intersect with broader innovation and external sourcing models. In this context, blockchain is considered primarily as an enabling infrastructure for decentralized governance and programmable coordination-supporting mechanisms such as tokenized incentives, auditable decision trails, and new forms of intellectual property (IP) and collaboration structures. Methods: This study employed a qualitative case study methodology, combining document analysis and semi-structured interviews with internal stakeholders from a leading large-cap pharmaceutical company (herein after "Company"). Participants included executives and professionals from corporate development, scientific research, external innovation, and digital strategy units.The analysis examined how a large-cap "Company" approaches mergers, acquisitions, and partnerships, and how emerging technologies may influence these frameworks. The study focused on strategy alignment, organisational attitudes towards decentralisation, integration constraints, and perceptions of innovation value along the external sourcing continuum. Results: Acquisition and innovation strategy by the "Company" is driven by long-term alignment between external opportunities and internal priorities. Over time, the "Company" increasingly turned to external sources of innovation, leveraging technologies to improve innovation scouting, target identification, and operational forecasting. While decentralisation technologies such as DAOs are viewed as promising for early-stage innovation and collaboration, their integration is hindered by legal ambiguity, internal governance rigidity, and unfamiliarity with token-based economics. The "Company" views mergers and acquisitions (M&As) and licensing as critical to sustaining its pipeline, and sees potential for emerging technologies to accelerate preclinical decision-making and improve visibility into academic and biotech ecosystems. Conclusions: This study contributes insights into how large-cap pharmaceutical firms might adapt their innovation models in response to technological change and external pressures. While established mechanisms such as M&A and partnerships remain dominant, digital and decentralized technologies offer complementary tools for scouting, collaboration, and portfolio expansion.
Substandard medicines in Nepal represent an under-documented yet critical threat to universal health coverage (UHC) and patient safety, disproportionately impacting marginalized communities. This opinion piece seeks to illuminate the prevalence, drivers, and far-reaching consequences of compromised pharmaceuticals, drawing on both local and global evidence. This paper offers pragmatic strategies to strengthen regulatory capacity and accountability by spotlighting ethical imperatives and systemic gaps. Bridging best practices from international frameworks with Nepal's unique context, I aim to catalyze policy discourse, cultivate public awareness, and spur multi-sectoral collaboration. Ultimately, the goal is to foster equitable and high-quality healthcare for all, reinforcing the moral imperative of safeguarding the integrity of Nepal's medicine supply.Substandard medicines are a pressing, under-recognized public health crisis in Nepal. Despite expanded healthcare access, compromised pharmaceuticals undermine patient well-being and public trust (1,2). The World Health Organization (WHO) estimates that at least one in ten medicines in low-and middle-income countries (LMICs) is substandard or falsified (3). In Africa, reports indicate up to 18.7% of sampled pharmaceuticals are compromised (4). This global issue is significantly influenced by two of the world's major suppliers of medicine, both domestically and internationally: India and China (5). According to estimates from India's Health Ministry, 5% of the nation's medicines are counterfeit and 0.3% are spurious, with one study suggesting that 75% of counterfeit medicines supplied globally originate in India (6). While recent, precise prevalence data for counterfeit drugs from China is not readily available, its role as a global manufacturing powerhouse is undisputed; for example, the country produces approximately 120 billion aspirin tablets annually for the North American market alone (7). A 2024 literature review highlights the historical scale of the issue, noting that 31% of the world's falsified and counterfeit medications are produced in China and Hong Kong (8). This geopolitical context is critically important for Nepal. The country's porous borders with these major pharmaceutical producers (9), combined with its own under-regulated supply chains, significantly heighten its susceptibility to the circulation of substandard drugs (10).A Nepal Health Research Council (NHRC) study found that 15.16% of 244 tested medicine batches failed pharmacopeial standards (11). Alarmingly, 62.16% of these were government-supplied. Compromised products included essential antibiotics, analgesics, and iron supplements, with failures in dissolution, assay, fill volume, and sterility. A 2010-2020 review of drug recall notices in Nepal revealed a surge in recalled products, most frequently antimicrobials (2). These findings indicate an ethical breach: public health facilities primarily serve the underprivileged, disproportionately imposing the burden of substandard medicines on them-a direct affront to the principle of justice (12). Patients, often unaware of compromised drug quality due to identical appearance, are denied meaningful informed consent. This erodes trust in the healthcare system and fuels antimicrobial resistance, a global health security threat with severe implications (3).Limited regulatory capacity exacerbates these dangers. In the Nepalese context, chronic under-resourcing of the Department of Drug Administration (DDA) and the National Medicines Laboratory hampers rapid testing and enforcement (13,14). Testing delays can extend for months, allowing compromised medications to circulate (15). While decentralizing drug procurement to local governments was intended to improve responsiveness, this policy has inadvertently weakened quality control due to a lack of local testing facilities and specialized personnel (14).Nepal's crisis of substandard medicines undermines human rights and core ethical principles.Tolerating this compromise of patient safety is incompatible with universal health coverage (UHC) (16). In Nepal, where a significant portion of the population relies on public health facilities for essential medicines, the prevalence of substandard drugs, especially in public health facilities (17), directly undermines the core tenets of UHC. Specifically, it violates the principle of access to quality healthcare: even if services are nominally affordable or free, receiving ineffective or harmful medication means that true, effective healthcare is not being delivered. Furthermore, the consequences of substandard medicines -prolonged illness, treatment failure, or adverse drug reactions -often lead to increased financial hardship for patients who may require further treatment, hospitalization, or experience loss of income due to illness. This directly contradicts the UHC goal of protecting people from financial risks associated with healthcare (18). This creates a two-tiered system, where those with resources can seek higher-quality care in the private sector, while the most vulnerable are left exposed to potentially dangerous treatments. Addressing this inequity and ensuring the integrity of the medicine supply is not just a matter of policy but a fundamental requirement for social justice. Success requires sustained political commitment, collaboration across sectors, and a steadfast focus on the public interest. Nepal must act decisively to eliminate substandard medicines and ensure equitable, high-quality healthcare for every citizen. This is not merely a matter of improving healthcare statistics; it is about upholding the fundamental dignity and rights of every citizen of Nepal.To address this crisis comprehensively, I argue that Nepal must move beyond isolated interventions and adopt a multi-pronged, systemic approach grounded in international best practices and adapted to the local context. The solution to the challenge of substandard medicines is complex, requiring a foundational shift from reactive detection to proactive prevention and quality assurance throughout the entire supply chain.First, the cornerstone of any effective strategy must be the robust implementation of a national quality assurance framework based on established WHO principles (19). This involves the mandatory adoption and enforcement of Good Manufacturing Practices (GMP) for domestic producers, ensuring that quality is built into products from the outset (20).Critically, this must be complemented by the nationwide implementation of Good Storage Practices (GSP) and Good Distribution Practices (GDP), which are presently lacking in Nepal (2). As detailed in WHO guidelines, this requires establishing a comprehensive Quality Management System that governs every stage of the supply chain, from procurement to delivery. This includes temperature-controlled storage and transport, proper documentation to ensure traceability, and rigorous training for all personnel involved (21,22). The goal is to create a secure, transparent, and controlled environment where opportunities for degradation or the introduction of falsified products are minimized.Second, this preventative framework must be supported by strengthening practices at the point of care through the enforcement of Good Pharmacy Practices (GPP). While pharmacistled detection of visibly substandard medicines is challenging and should not be the primary line of defense, pharmacists are integral to maintaining quality assurance (23). The US Agency for International Development (USAID) funded Medicines, Technologies, and Pharmaceutical Services (MTaPS) program has already highlighted the significant impact of GPP implementation on product quality and patient safety in Nepal (24). The urgency of this is underscored by recent local evidence from the Supervision, Performance Assessment, and Recognition Strategy (SPARS) pilot. The baseline assessment from the SPARS pilot, conducted in 2022 across 284 health facilities, revealed alarmingly poor medicines management practices, with an overall median score of only 34% across key performance indicators. Particularly low scores in domains such as storage management and stock management highlight critical, systemic gaps that a robust GPP framework is precisely designed to address (25). Therefore, a renewed focus on GPP-including proper storage, inventory management using the First-Expired-First-Out (FEFO) principle, and patient counselling (26,27)-is not merely a recommendation but an evidence-based necessity to safeguard medicine quality at the final stage of the supply chain.Third, a fundamental overhaul of the national regulatory body is imperative. While establishing a new, fully autonomous commission with civil society oversight represents an ideal long-term goal, a more pragmatic and immediate strategy is to empower the existing Department of Drug Administration (DDA). Given Nepal's resource constraints, strengthening the current regulatory infrastructure is a more feasible first step. Following successful models from other nations, such as the autonomous South African Health Products Regulatory Authority (SAHPRA) (28), granting the DDA greater operational and financial autonomy would be transformative. An empowered DDA could then engage in the kind of international collaboration essential for modern pharmacovigilance, such as the recent Memorandum of Understanding between SAHPRA and Australia's Therapeutic Goods Administration (TGA) to share regulatory information and expertise (29). Recent progress within Nepal indicates a readiness for such advancement; for instance, with support from USAID MTaPS, the DDA has begun modernizing its document management system by installing secure, access-controlled repositories to increase efficiency and security (30).While these foundational improvements are commendable, they must be viewed as the first steps in a much longer journey. Full regulatory maturity requires sustained investment to build upon these gains. This must include a significant increase in the number of trained inspectors and a shift towards a risk-based inspection model that prioritizes systematic laboratory testing of products from importers and wholesalers before they enter the market, thereby ensuring quality at the source rather than attempting to recall products that have already been dispensed.Finally, these systemic and regulatory reforms should be amplified by leveraging modern technology and strengthening post-market surveillance. A vital component underpinning all these reforms is compliance with Good Practice (GxP) principles. GxP is an acronym for a collection of quality guidelines and regulations designed to ensure that products within heavily regulated industries, such as pharmaceuticals, are consistently safe, effective, and fit for their intended use (31). This framework is not a single standard but an ecosystem of practices-including GMP, GSP, and GPP-that collectively safeguard product integrity.While technology is not a panacea, rigorous track-and-trace systems-using technologies like blockchain or simple 2D barcoding-offer a powerful tool for enforcing GxP compliance and achieving end-to-end visibility. This enables regulators to verify authenticity and rapidly identify diversions in the supply chain (32,33). Such systems, proven to be cost-effective in settings like Bangladesh (34), would support the broader GxP framework (31) and provide crucial data for the empowered DDA. Within this enhanced surveillance system, pharmacists, supported by legally protected reporting mechanisms, become vital nodes for reporting suspected adverse events or quality issues, thereby creating a feedback loop that strengthens the entire regulatory ecosystem (35).The integrity of a nation's medicine supply is a direct reflection of its commitment to health equity and social justice. For Nepal, the continued presence of substandard medicines in the supply chain represents a fundamental contradiction to its goal of achieving universal health coverage. The systemic reforms proposed-implementing a robust GxP framework, empowering an autonomous regulator, and leveraging technology for surveillance-are not merely technical exercises; they are essential acts of building trust with the nation's most vulnerable citizens and affirming their right to safe, effective healthcare. Moving forward, the challenge is not one of awareness, but of political will and sustained action. Ensuring the quality of every pill and vial is not just a matter of public health policy-it is a fundamental test of Nepal's promise of equitable healthcare for all.
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.
Open access
Pharmaceutical Economics and Policy
Health Systems, Economic Evaluations, Quality of Life
Matthew M. Kavanagh, Luis Gil Abinader, Fatima Hassan, Eli A. Friedman
Scientific advances to fight infectious diseases have been remarkable. International law and global governance have sought, and often failed, to keep pace, secure equity, and stop outbreaks. We trace the law and governance model emerging from early failure in the AIDS response and identify four elements: use of law by national governments to compel sharing; decentralized generic manufacturing; mechanisms for voluntary sharing of patents and technology transfer; international funding. In combination, these created a remarkable new ecosystem. We find that when COVID-19 hit and mRNA vaccines were rapidly developed, global North governments opposed mobilizing this synergistic model. Instead, equity efforts focused on financing purchase of vaccines from originator companies with little use of law. Amidst monopolies and scarcity of doses, vaccine nationalism fatally undermined this effort. Whether more synergistic law and governance emerges from rapidly changing global health law will likely dictate the efficacy of future global infectious disease response.
Mark Gaynor, Kathleen N. Gillespie, Allison Roe, Erica F. Crannage · 5 authors
Background: In recent years, blockchain technology has made great strides in diverse industries but has fallen behind within the pharmaceutical industry. The pharmaceutical industry is complex and would benefit greatly from the distributed database and emphasis of information privacy promoted by blockchain technology. This paper identifies the potential best application for blockchain technology in the United States pharmaceutical industry by identifying current trends, companies exploring the possibilities of blockchain technology, and industry concerns with opportunities for improvement. Methods: We utilized a 4D framework using ease of implementation, novelty, necessity, and fit of the overall industry to examine the adoption of blockchain technology in the pharmaceutical industry. Based on the 2D framework of difficulty and novelty as driving factors for the development of foundational technologies in the world of business by Iansiti and Lakhani in The Harvard Business Review, each application was ranked and scored for the best potential implementation. The potential applications proposed in this paper can be grouped into two main categories. The first category, management, includes best use cases such as health records, clinical trials, and inventory systems. The second category, monitoring, highlights cases such as pharmaceutical products, preventing counterfeits, optimizing supply chains, and addressing prescription misuse and abuse.Results: Each application was ranked by the four metrics in the framework, giving the greatest weight to necessity and ease of implementation. Using the highlighted methodology above, the applications for best implementation include Prescription Drug Misuse and Abuse Prevention, Prevention of Counterfeits, Clinical Trial Outcomes, and Smart Contracts. Conclusion: Blockchain technology offers a new and promising solution to pharmaceutical industry needs. Each application of blockchain technology must fit within the framework of necessary, ease of implementation, familiarity amongst stakeholders, and fit of the overall industry to promote the most appropriate use. By using the extended framework proposed by Iansiti and Lakhani we show that blockchain, in all these domains, shows promise to improve pharmaceutical industry performance.
The pharma supply chain system utilized smart contracts aims to enhance transparency and efficiency in the drug distribution of process.Smart contracts, deployed on a blockchain, secure various supply chain stages, such as manufacturing, distribution, etc.This system ensures real-time visibility, reduces counterfeiting risks, and enables swift response to issues like recalls.Additionally, it fosters trust among stakeholders by providing an immutable record of transactions, ultimately promoting integrity and accountability within the pharma supply chain.
BACKGROUND: Intellectual property (IP) is a substantial competitive advantage in the health care industry. However, the COVID-19 pandemic highlighted the need for open innovation and collaboration for the greater good. Despite this, the industry faces challenges with innovation owing to organizational and departmental barriers. A secure platform is necessary to facilitate IP sharing without compromising the rights of IP owners. OBJECTIVE: This study proposes a blockchain-based framework to secure IP transactions in health care and bring social impact. METHODS: This study reviews existing researches, publications, practical cases, firm and organization websites, and conferences related to blockchain technology, blockchain in health care, blockchain in IP management, IP pledge research, and practice of IP management blockchain. The platform architecture has 7 components: pledgers, advanced research technology (ART), IP pledge platforms, IP databases, health care research, seeking ART, and transaction condition setting. These components work together seamlessly to support the sharing and pledging of ART and knowledge, while ensuring the platform's transparency, security, and trust. RESULTS: The open IP pledge framework can promote technology dissemination and use, reduce research and development costs, foster collaboration, and serve the public interest. Medical organizations' leadership and support and active participation from stakeholders are necessary for success. By leveraging blockchain technology, the platform ensures tamper-proof and transparent transactions and protects the rights of IP owners. In addition, the platform offers incentive mechanisms through pledge tokens that encourage stakeholders to share their ART and contribute to the platform. CONCLUSIONS: Overall, the proposed framework can facilitate technological innovation, tackle various challenges, and secure IP transactions. It provides a secure platform for stakeholders to share their IP without compromising their rights, promoting collaboration and progress in the health care industry. The implementation of the framework has the potential to revolutionize the industry's approach to innovation, allowing a more open and collaborative environment driven by the greater good.
Abhijeet Ghadge, Michael Bourlakis, Sachin Kamble, Stefan Seuring
Research on Blockchain implementation in the Pharmaceutical Supply Chains (PSC) is lacking despite its strong potential to overcome conventional supply chain challenges. Thus, this study aims to provide critical insight into the nexus between Blockchain and PSC and further build a conceptual framework for implementation within the pharmaceutical industry. Following a systematic literature review and text mining approach, 65 interdisciplinary articles published between 2010 and 2021 were studied to capture the decade long developments. Descriptive and thematic analysis showcases nascent developments of Blockchain in PSC. The drivers and barriers to adoption, implementation stages, and applications identified through the thematic analysis guide in setting the agenda for future research, primarily focussing on the use of Blockchain for drug counterfeiting, recall issues, along with other sector-specific challenges such as patient privacy, regulations and clinical trials. Research on Blockchain for PSC has been slow compared to other sectors, but has accelerated since the Covid-19 pandemic. Identified influential factors, implementation process and apparent applications are expected to influence researchers and practitioners in developing a roadmap for adopting Blockchain in the pharmaceutical industry. The proposed conceptual framework is novel and provides valuable directions to producers, regulators and governments to implement Blockchain in the pharmaceutical industry.
Pharmaceutical supply chains are complex structures that include various participants. Furthermore, blockchains are viewed as a promising solution to increase effectiveness and overcome some of the main challenges in these supply chains-especially lack of trust. The European Union (EU) set strict rules in the domain of pharmaceutical supply chains in order to protect patient safety and public health. In addition, blockchains bring legal requirements. Among these requirements, personal data protection is of utmost importance. This is because, as has been argued for years, blockchains and the EU data protection regime are in conflict by their natures. However, it is also claimed that when rightly designed and combined with other technological solutions, blockchains potentially offer great opportunities to enhance data protection. Nevertheless, potential for blockchains in the pharmaceutical supply chain is not yet been realized as most use cases are in the proof of concept or pilot stage. This article examines the debates surrounding blockchains and data protection. The goal is to draw constructive conclusions on whether blockchain solutions can be designed in data protection-enhancing ways and whether this might help realize the potential for blockchain in pharmaceutical supply chains-particularly by creating trust. For this purpose, the example of an ongoing EU-funded innovative research project called PharmaLedger as a case study to concretize its theoretical examinations is examined. This project is chosen because it gathers a wide variety of stakeholders representing different interests and aims to create a digital trust ecosystem in health care by providing a widely trusted platform that supports the design and adoption of blockchain-enabled healthcare solutions while accelerating the delivery of innovation that benefits the entire ecosystem from manufacturers to patients.
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.
Open access
Health Systems, Economic Evaluations, Quality of Life
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.
Open access
Pharmaceutical Economics and Policy
Health Systems, Economic Evaluations, Quality of Life
The award was announced during the ConV2X 2021 conference, themed “Blueprint for a New Digital Health Era,” broadcast November 9-11, 2021. The winning article is titled: "The Last Mile: DSCSA Solution Through Blockchain Technology: Drug Tracking, Tracing, and Verification at the Last Mile of the Pharmaceutical Supply Chain with BRUINchain," by lead author William Chien (PharmD, MBA) and fellow authors from UCLA Health and LedgerDomain. The article is located at https://doi.org/10.30953/bhty.v3.134. The groundbreaking article was part of the FDA’s Pilot Project Program for the Drug Supply Chain Security Act (DSCSA), and centered on a healthcare center pharmacy operating solely on commercial off-the-shelf (COTS) technology. It presented the highest number of BHTY 2020 reader engagements, with the most downloads and views. The study demonstrated a 100% success rate across scanning, expiration detection, and counterfeit detection; and paperwork reduction from approximately 1 hour to less than a minute. Projecting out to 4.2 billion prescriptions being dispensed each year in the United States, the study found that distributed ledger technologies (such as blockchain) would not only save $183 million in annual labor costs, but also avert bad or fraudulent transactions, reduce the need for safety stock, and enhance the detection and removal of potentially dangerous drugs from the drug supply chain to protect U.S. consumers.
Nazik Zakari, Muna Al‐Razgan, Amani Alsaadi, Haya Alshareef · 9 authors
Blockchain technology is accelerating digital transformation across multiple industries, including the pharmaceutical industry. The pharmaceutical industry suffers from a lack of transparency, difficulty tracking products, lack of trust, and the shipment of expired products. Blockchain technology has been applied to solve several of these problems. In this paper, we present a systematic review of the literature focusing on the adoption of blockchain technology in the pharmaceutical industry. We collected, analyzed, qualified, and discussed studies retrieved from seven databases. The initial search yielded 2,185 papers, which were screened, discussed, voted on, critically appraised, and collected by a snowball workflow that finally yielded 38 papers. The blockchain application areas covered in the papers were classified as counterfeit drug prevention, drug distribution, tracking and tracing, and safety and security. The most frequent category was counterfeit drug prevention, which is consistent with the primary objective of the pharmaceutical industry. The newer topics discussed in this study were data governance, data quality, pharmaceutical turnover, and prescription drug monitoring. We discuss issues surrounding each of these topics and research studies, along with their limitations and solutions. We also examine the challenges and future research directions of applying blockchain technology in the pharmaceutical industry.
The integration of blockchain technology into pharmacovigilance infrastructure for national cancer registries presents a transformative approach to managing drug safety data. Traditional pharmacovigilance systems often face challenges such as fragmented data sources, delayed reporting, lack of transparency, and data security risks. Blockchain offers a decentralized, immutable, and transparent framework that can enhance the accuracy, timeliness, and reliability of adverse drug reaction (ADR) reporting. This review explores how blockchain can be applied to cancer registries to create a more efficient and trustworthy pharmacovigilance ecosystem. It highlights the key components of a blockchain-enabled infrastructure, including smart contracts for automating data validation, permissioned ledgers for maintaining patient confidentiality, and interoperability standards to facilitate seamless data exchange among stakeholders. Furthermore, it discusses the potential benefits such as improved patient safety, strengthened regulatory oversight, enhanced collaboration among healthcare providers, and empowerment of patients in the pharmacovigilance process. Despite the promising prospects, challenges including technical complexity, regulatory compliance, scalability, and stakeholder acceptance must be addressed. Overall, blockchain holds significant potential to revolutionize pharmacovigilance practices, particularly in oncology, where timely detection and response to drug-related adverse events are critical for patient outcomes and public health.
Moyosore Taiwo, Adebanjo Olowu, Yusuf Olanlokun, Ojo Timothy
As healthcare systems globally transition toward patient-centered and decentralized care models, the role of pharmacists has evolved from traditional dispensing functions to more autonomous, clinically integrated responsibilities. This paradigm shift, driven by the need for efficiency, accessibility, and personalized care, positions pharmacists as critical stakeholders in therapeutic decision-making, especially within primary and community-based health systems. In decentralized healthcare structures, such as those found in integrated care networks and rural outreach programs, pharmacists are increasingly responsible for clinical judgment, medication optimization, patient education, and adverse drug reaction monitoring—functions traditionally reserved for physicians. This study explores the extent and determinants of clinical decision-making autonomy among pharmacists within decentralized healthcare models. It examines how organizational structure, regulatory frameworks, risk management policies, and interprofessional collaboration impact pharmacists’ ability to make independent clinical decisions. Particular attention is paid to the balance between autonomy and accountability, highlighting potential risks such as therapeutic errors and liability concerns, alongside opportunities for improving medication adherence and reducing hospital readmissions. Using a mixed-methods approach involving policy analysis, structured interviews, and clinical case reviews, the study uncovers significant variation in autonomy across regions and care settings. It proposes a framework for risk-informed autonomy, whereby pharmacists operate with expanded clinical responsibility under well-defined governance and support systems. Ultimately, this research underscores the importance of redefining pharmacists’ roles in modern health systems and offers strategic recommendations for empowering them within decentralized models without compromising patient safety or care quality.
Procurement is the most important part of the pharmaceutical logistic cycle. It is the process of acquiring supplies after a properly selected list of products. The procurement system or model depends on the type of organization weather it is governmental or private, centralized or decentralized, autonomous or semiautonomous. The objectives of procurement system is to make available the right drug in an appropriate quantities of adequate quality from a reliable supplier at the right time with the lowest possible prices through an ethical and legal procedures. Prequalification of suppliers is the successful quality assurance activity. Needs and funds can be reconciled and a rational cut can be done by using ABC- VAN matrix technique. Purchasing should be by transparent competition through open tender, restrictive tender, restricted competition or in certain cases by direct negotiation by transparent committee leading to transparent contract. One of the most important procurement practice for the system to succeed is the reliable payment and efficient financial management and monitoring the supplier performance. The system should have an efficient quality assurance program with annual auditing and regular reports.
INTRODUCTION: The amount of mandatory data that needs to be analyzed as part of a medical device postmarket surveillance (PMS) system has grown exponentially in recent times. This is a consequence of increasingly demanding and complex regulatory requirements from Health Authorities, aimed at a better understanding of the medical device safety evaluation. Proactive approaches to PMS processes are becoming more necessary as regulators increase the scrutiny of device safety. New technologies have been explored to address some of the challenges associated with this changing regulatory environment. AREAS COVERED: This paper focuses on the different technical aspects of blockchain and how this new technology has the potential to support the ongoing efforts to improve the PMS system for medical devices. EXPERT OPINION: To address these challenges, we suggest to generate a private PMS data permissioned blockchain with a proof-of-authority consensus mechanism, to which only a restricted number of designated and audited participants have authorization to validate transactions and add them to the PMS data blockchain ledger. Blockchain has the potential to support a more efficient approach, which could offer many advantages to the different stakeholders involved in the PMS process, such as supporting with new regulatory initiatives.
Ching Lam, Michelle Helena van Velthoven, Edward Meinert
BACKGROUND: Advanced therapies, including cell and gene therapies, have shown therapeutic promise in curing life-threatening diseases, such as leukemia and lymphoma. However, these therapies can be complicated and expensive to deliver due to their sensitivity to environment; troublesome tissue, cell, or genetic material sourcing; and complicated regulatory requirements. OBJECTIVE: This study aims to create a novel connected supply chain logistics and manufacturing management platform based on blockchain, with cell and gene therapy as a use case. Objectives are to define the requirements and perform feasibility evaluations on the use of blockchain for standardized manufacturing and establishment of a chain of custody for the needle-to-needle delivery of autologous cell and gene therapies. A way of lowering overall regulatory compliance costs for running a network of facilities operating similar or parallel processes will be evaluated by lowering the monitoring costs through publishing zero-knowledge proofs and product release by exception. METHODS: The study will use blockchain technologies to digitally connect and integrate supply chain with manufacturing to address the security, scheduling, and communication issues between advanced therapy treatment centers and manufacturing facilities in order to realize a transparent, secure, automated, and cost-effective solution to the delivery of these life-saving therapies. An agile software development methodology will be used to develop, implement, and evaluate the system. The system will adhere to the EU and US good manufacturing practices and regulatory requirements. RESULTS: This is a proposed study protocol, and upon acceptance, grant funding will be pursued for its execution in 2021. CONCLUSIONS: The successful implementation of the integrated blockchain solution to supply chain and manufacturing of advanced therapies can push the industry standards toward a safer and more secure therapy delivery process. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/17005.
Khizar Abbas, Muhammad Afaq, Talha Ahmed Khan, Wang‐Cheol Song
From the last decade, pharmaceutical companies are facing difficulties in tracking their products during the supply chain process, allowing the counterfeiters to add their fake medicines into the market. Counterfeit drugs are analyzed as a very big challenge for the pharmaceutical industry worldwide. As indicated by the statistics, yearly business loss of around $200 billion is reported by US pharmaceutical companies due to these counterfeit drugs. These drugs may not help the patients to recover the disease but have many other dangerous side effects. According to the World Health Organization (WHO) survey report, in under-developed countries every 10th drug use by the consumers is counterfeit and has low quality. Hence, a system that can trace and track drug delivery at every phase is needed to solve the counterfeiting problem. The blockchain has the full potential to handle and track the supply chain process very efficiently. In this paper, we have proposed and implemented a novel blockchain and machine learning-based drug supply chain management and recommendation system (DSCMR). Our proposed system consists of two main modules: blockchain-based drug supply chain management and machine learning-based drug recommendation system for consumers. In the first module, the drug supply chain management system is deployed using Hyperledger fabrics which is capable of continuously monitor and track the drug delivery process in the smart pharmaceutical industry. On the other hand, the N-gram, LightGBM models are used in the machine learning module to recommend the top-rated or best medicines to the customers of the pharmaceutical industry. These models have trained on well known publicly available drug reviews dataset provided by the UCI: an open-source machine learning repository. Moreover, the machine learning module is integrated with this blockchain system with the help of the REST API. Finally, we also perform several tests to check the efficiency and usability of our proposed system.
Background: Pharmaceutical corruption is a serious challenge in global health. Digital technologies that can detect and prevent fraud and corruption are particularly important to address barriers to access to medicines, such as medicines availability and affordability, stockouts, shortages, diversion, and infiltration of substandard and falsified medicines.Objectives: To better understand how digital technologies are used to combat corruption, increase transparency, and detect fraud in pharmaceutical procurement systems to improve population health outcomes.Methods: We conducted a multidisciplinary review of the health/medicine, engineering, and computer science literature. Our search queries included keywords associated with medicines procurement and digital technology in combination with terms associated with transparency and anti-corruption initiatives. Our definition of ‘digital technology’ focused on Internet-based communications, including online portals and management systems, supply chain tools, and electronic databases.Results: We extracted 37 articles for in-depth review based on our inclusion criteria focused on the utilization of digital technology to improve medicines procurement. The vast majority of articles focused on electronic data transfer and/or e-procurement systems with fewer articles discussing emerging technologies such as machine learning and blockchain distributed ledger solutions. In the context of e-procurement, slow adoption, justifying cost-savings, and need for technical standards setting were identified as key challenges for current and future utilization.Conclusions: Though there is a significant promise for digital technologies, particularly e-procurement, overall adoption of solutions that can enhance transparency, accountability and concomitantly combat corruption, is still underdeveloped. Future efforts should focus on tying cost-saving measurements with anti-corruption indicators, prioritizing centralization of e-procurement systems, establishing regulatory harmonization with standards setting, and incorporating additional anti-corruption technologies into procurement processes for improving access to medicines and to reach the overall goal of Universal Health Coverage.
Sharing medical data with numerous stakeholders for varied purposes is a perplexing problem facing healthcare systems throughout the world. All this needs to be done, whilst ensuring patient and health data privacy and data integrity. Through this paper, an attempt has been made to present a novel distributed digital health record management system. The blockchain framework has been built using the tools provided by hyper ledger, an open source project. This system empowers patients with a comprehensive, secure, immutable, and easily accessible digital record of their health. Also, this information is available across multiple practitioners and healthcare providers throughout the world. This system can therefore empower the emergence of health data economics, empowering researchers with the health data and giving patients an opportunity to share their health data without losing anonymity. The purpose of this paper is to introduce a prototype of a health data management system utilising blockchain.
Reda Moulouki, Dominique Bernard Kanga, Taif Fatima, Mohamed Azouazi
Rising global costs and the fragmented nature of the healthcare supply chain creates several challenges. The healthcare industry needs efficient solutions that can streamline supply chain operations and processes in a cost- effective manner. Leading healthcare players are exploring blockchain technologies to achieve efficiencies and gain better control over their supply chains. In this paper, we present the current state of the subject and summarize the benefits and the challenges of the distributed organization and management of supply chains management. Focusing on healthcare, and we discuss the applicability of blockchains in the supply chain domain in the healthcare.