Pharmaceutical access in Brazil: challenges and opportunities

Abstract

Background

Brazil faces challenges in ensuring equitable access to prescription drugs for its population. Socioeconomic disparities contribute to health inequalities, impacting access to health care services, including medicines. Brazil’s Unified Health System aims to provide universal health care coverage, but only 30·5% of people obtain all of their prescribed medications through public channels free of charge. The objective is to characterize the Brazilian pharmaceutical market to better understand the underlying factors affecting medicine accessibility.

Methods

We conducted a literature review to assess the current state of the pharmaceutical market and prescription drug access in Brazil.

Findings

The Brazilian pharmaceutical market is extensive but highly concentrated, characterized by low investment in original research and development (R&D) and a heavy reliance on imported active pharmaceutical ingredients. Challenges include vulnerability to economic and external factors. Additionally, high medicine prices, prioritization of production of expensive new medicines over those for prevalent diseases, and shortages contribute to inequalities in access. Regulatory issues, underfunding of health care, and legal discrepancies exacerbate these challenges but also present opportunities for reform. Interpretation: The policymakers should prioritize enhancing R&D investment, reducing dependency on international markets, strengthening regulatory frameworks, and improving health care system efficiency to ensure reliable access to essential medicines.

Introduction

Brazil, with a population of 203·1 million spread across 8·51 million km² and a density of 23·86 inhabitants/km², faces socioeconomic disparities amidst a Gross Domestic Product (GDP) of USD 3·40 trillion dollar in Purchasing Power Parity (PPP) and an inflation rate of 5·79%, coupled with an 8·1% unemployment rate [1]. Over 50% of the population earns less than USD 1,136 in PPP monthly, with extreme poverty affecting 5·9% of the people. Coexisting problems relate to basic infrastructure, with incomplete access to water supply (14·5%), to sewage collection network (36·8%), and lack of garbage collection (14·0%). The five major regions – North, Northeast, South, Southeast, and Midwest – exhibit important geographic and cultural differences, translating into disparities that contribute to health inequalities. The infant mortality rate of 12·6 per thousand live births, access to health care services, mainly medicines, varies significantly, posing a challenge for equitable healthcare provision nationwide and emphasizing the need for effective measures to ensure the well-being of all citizens [2].

According to the 1988 Federal Constitution, Brazil’s Unified Health System (SUS) is designed to guarantee universal access to health care, including essential medicines, as a right of all citizens and a duty of the state. While 71.5% of the population depends on SUS, access to prescribed medicines remains a significant challenge, particularly within the public sector. In 2019, only 30.5% (6.2 million individuals) obtained at least one of the medicines prescribed during their most recent health appointment from public health services. Disparities in access were observed across regions and social groups: lower rates were found in the Center-West (24.9%) and Northeast (28.9%) regions; individuals with higher education (12.5%) accessed medicines less frequently through the public sector compared to those with less education (38.7%); and black individuals (34.4%) had greater success than white individuals (27.9%). In addition, people living in rural areas (36.8%) and those with lower household incomes (42.3% among those earning up to ¼ of the minimum wage) had better access than urban residents (29.6%) and wealthier groups (7.1% among those earning more than five minimum wages) [2]. These inequalities highlight the structural challenges in guaranteeing equitable access to pharmaceutical treatment. In light of these issues, this article aims to comprehensively characterize the Brazilian pharmaceutical market to better understand the underlying factors affecting medicine accessibility. A glossary of key terms and acronyms is provided at the end of the manuscript to support reader comprehension.

Methods

We conducted a comprehensive literature review and collected data from government databases to characterize the current state of the Brazilian pharmaceutical market. Our description covered population health issues, health system characteristics, and financing. The inclusion criteria incorporated keywords such as medications, pharmaceutical market, access to medicines, official laboratories, pharmaceutical market transition, productive development partnership, and patents. Our sources included scientific journals from major national and international databases, technical books from stakeholders, and relevant legislation and government reports from the Ministry of Health, including agencies such as the Brazilian Health Regulatory Agency (Anvisa), Medicines Market Regulation Chamber (Cmed), and National Committee for Health Technology Incorporation at the SUS (Conitec).

Epidemiology

Among the leading causes of death in Brazil are diseases of the circulatory system, accounting for 25·9% of deaths, followed by cancer at 15·8%, diseases of the respiratory system at 11·4%, and infectious diseases at 8·5%. However, external causes of morbidity and mortality, related to such factors as violence, represent about 10% of deaths in the country. These data highlight the need for integrated approaches to prevent and control these various health conditions (Graph 1) [3].

Graph 1.

Causes of death in the Brazilian population by international classification of diseases chapters, 10. Brazil, 2022

Unified health system

Established by the 1988 Federal Constitution, the SUS is grounded in the principles of universality, comprehensiveness, and equity. As the world’s largest universal public health system, SUS ensures free access to health care services and essential medicines for all citizens. Its structure is decentralized, with responsibilities shared among federal, state, and municipal governments. The system is primarily tax-funded and offers most services at no direct cost to the patient [4].

Brazil also maintains a supplementary private health sector, financed through private insurance, direct payments, or corporate plans. This coexistence results in dual access pathways: public provision via SUS and private acquisition. The SUS is the main provider of hospital care in Brazil, accounting for approximately 64% of all hospital admissions — about 13 million per year. In 2023, the leading causes of hospitalization included pregnancy, childbirth, and puerperium (16•8%), external causes of morbidity and mortality (11•1%), diseases of the digestive system (10•8%), respiratory system (9•9%), and circulatory system (9•7%) (Graph 2) [3].

Graph 2.

Hospitalizations in SUS by International Classification of Diseases chapters, 10. Brazil, 2023

While the SUS is primarily tax-funded and offers free access to services, the supplementary system is financed through a combination of private and public sources and may provide access to medicines, depending on the coverage.

Pharmaceutical assistance

Medicines within SUS are dispensed through public health facilities, while private access often involves copayment or full out-of-pocket spending. Pharmaceutical assistance within SUS is structured into three main components, each aligned with the level of care, disease complexity, and funding mechanisms. The Basic Component of Pharmaceutical Assistance (CBAF) covers medicines for common conditions typically managed in primary care, such as hypertension and diabetes. The Strategic Component (CESAF) provides medicines for diseases of public health importance, including tuberculosis, HIV, and leprosy. The Specialized Component (CEAF) supplies high-cost and complex medicines, often related to chronic, rare, or autoimmune diseases, and is generally associated with secondary or tertiary levels of care.

The Brazilian also have a model called Farmácia Popular Program, which provides selected medicines and health products at subsidized prices in private pharmacies through public-private partnerships. This mixed provision model requires context-specific understanding, especially compared to countries where copayment or insurance-based systems dominate [5].

Financing and access disparities

This discrepancy between the demand for medications and the SUS’s capacity to meet it represents a key challenge for Brazil. The financing of medicines in Brazil involves a complex interplay between the public and private sectors, with the government, particularly through the SUS, serving as the larger purchaser. Budget constraints pose challenges to the government’s ability to fully fund pharmaceutical assistance. Brazil’s public health system relies on tax and social contribution resources at federal, state, and municipal levels. There are minimum financing thresholds corresponding to a percentage of tax revenue and funds distributed by the central government to lower levels of government (states) determined by amendment to the country’s constitutional text.

In 2021 the general health expenditure was 9·7% of the GDP, with the general government health expenditure making up 41·6%. The remaining 58·4% would have been covered by private spending, which includes out-of-pocket expenses by individuals, private insurance, and other non-governmental sources. Despite this, Brazil’s health care spending on medications remains relatively low compared to higher-income countries at 1·9% of GDP, with a substantial portion of medication financing coming from the private sector [6]. This peculiarity positions Brazil among the few countries with a nationally organized health system with universal coverage but with a predominant role played by the private sector. This structural imbalance between public and private financing reflects broader global challenges, particularly in countries with universal health systems facing increasing pressure from high-priced technologies.

Considering that a large part of the Brazilian population has the SUS as the main source to access healthcare, equitable access to medicines is still strongly compromised by the low availability of essential medicines as SUS distributes these technologies. In addition, almost 70% of the acquisition of medicines is made through direct disbursement by the population [6] also resulting in high rates of judicialization to obtain them.

From research to access

Getting medications to patients is a complex process involving multiple regulatory stages (Graph 3). Anvisa is the central authority responsible for regulating medicines, ensuring their quality, safety, and efficacy before they are authorized for commercialization. Anvisa also authorizes clinical trials, evaluates dossiers for marketing authorization, and oversees post-marketing surveillance. Like many other regulatory authorities worldwide, Anvisa aligns its regulatory processes with international harmonization standards, such as those issued by the International Council for Harmonization and the World Health Organization (WHO). This ensures regulatory convergence and facilitates the adoption of internationally accepted quality, safety, and efficacy criteria.

Graph 3.

Infographic of the medication life in Brazil. Note: Anvisa – Brazilian Health Regulatory agency. CMED – Drug market regulation chamber

The institution operates within the broader structure of the National Health Surveillance System, which includes federal, state, and municipal authorities. The state and municipal components are responsible not only for inspections but also for implementing surveillance policies and conducting risk-based health monitoring. Their role encompasses surveillance of health services and products, guidance to health professionals, and the enforcement of regulatory compliance across healthcare facilities and pharmaceutical establishments.

From 2013 to 2023, Anvisa registered 1,974 clinical studies, with an increasing annual trend and an average of 141 studies per year [7]. For a drug to receive marketing authorization, its regulatory dossier must demonstrate quality, safety, and efficacy. The comparative evaluation with a standard treatment is not mandatory in all cases; it depends on the nature of the submitted evidence [8, 9].

As seen globally, the lack of comparators, the increasing use of expedited approval pathways and reliance on surrogate endpoints, rather than clinically relevant outcomes, has raised concerns regarding the real-world effectiveness of newly approved therapies [10].

As of December 2023, there were 10,125 medicines with marketing authorization in Brazil. Of these, 80.1% (n = 8,201) contained a single active pharmaceutical ingredient, representing 1,328 unique substances across various pharmaceutical forms and presentations. The most frequent therapeutic classes were antineoplastic and immunomodulating agents (17.8%), alimentary tract and metabolism (13.0%), nervous system (13.0%), anti-infectives for systemic use (11.4%), and cardiovascular system (8.3%) [7].

Research & development

The National Policy on Science, Technology, and Innovation in Health, reformulated in 2008, became an integral part of the national health policy and aims to foster sustainable national development. It does so by promoting the generation of technical and scientific knowledge aligned with Brazil’s economic, social, cultural, and political context. Initiatives led by the Ministry of Health to broaden access to technologies for prevention and care include efforts to stimulate the production and distribution of strategic technologies. One major strategic framework is the Economic-Industrial Health Complex, which seeks to integrate industrial development with public health needs as defined by SUS, focusing on areas such as vaccines, biotechnologies, and essential medicines [11].

Both public and private institutions conduct research and Development (R&D) activities. However, Brazil still faces challenges in fostering pharmaceutical innovation. R&D investment remains comparatively low, particularly in the development of drugs tailored to local health needs. One key limiting factor is Brazil’s heavy dependence on imported active pharmaceutical ingredients (APIs)—with only around 15% produced domestically [6]. API costs may account for up to 80% of a drug’s total production cost [12].

The development of the national pharmaceutical industry has followed divergent paths. While the innovative pharmaceutical sector experienced growth in the 2000s, the generic drug market has increasingly lost ground to multinational companies. Despite being the largest pharmaceutical market in Latin America, Brazil has accumulated a significant trade deficit [12]. In 2023, pharmaceutical imports totaled USD 7.2 billion, while exports reached only USD 519 million, generating a deficit of USD 6.7 billion [13]. This pattern mirrors broader global market asymmetries, where a small number of transnational corporations dominates high-value segments such as biotechnology and innovative pharmaceuticals. Consequently, middle-income countries like Brazil often struggle to establish competitive innovation ecosystems, despite having large domestic markets [14].

Patent protection remains a key incentive for private sector innovation, but also creates barriers to access, especially during exclusivity periods when prices are highest. Although compulsory licensing is permitted under the World Trade Organization’s Doha Declaration, it remains rarely used. Brazil’s 2005 compulsory license for antiretroviral drugs is one of the few successful examples globally, underlining the potential of such legal tools to improve access to essential medicines [15].

Another barrier is the delay in patent examinations by Brazil’s National Institute of Industrial Property, which has been criticized for limiting pharmaceutical investment due to a backlog of applications and a shortage of examiners [16]. Although Anvisa’s prior consent for granting pharmaceutical patents was introduced in 2001, as a public health safeguard to align industrial property with health needs, this requirement was eliminated in 2021 [17, 18]. The removal of this mechanism may affect how public health considerations are integrated into patent decisions.

Brazil’s position in global pharmaceutical patent filings remains modest. In 2022, only 6.5% of the country’s 6,909 patent applications were for pharmaceutical products. Brazil ranked 54th out of 132 countries in the Global Innovation Index 2022, reflecting persistent structural barriers to advancing pharmaceutical innovation [19].

Drug pricing

In Brazil, once a medication obtains marketing authorization from Anvisa, its commercialization is subject to price regulation by the CMED [20]. CMED is responsible for defining price ceilings and monitoring the dynamics of the pharmaceutical market with the aim of promoting pharmaceutical assistance and stimulating both competitiveness and access to medicines. Its regulatory framework includes distinct provisions for public and private sector pricing, tax considerations, and periodic updates to reflect economic conditions [20].

Classification categories are used to establish maximum prices for new pharmaceutical products. New medicines are classified under Categories I or II. Category I applies to products with patented active ingredients in Brazil that demonstrate proven therapeutic benefits—such as increased efficacy, fewer side effects, or lower overall treatment costs. Category II encompasses new products without clear therapeutic advantages. Other categories (III–VI) cover new presentations, fixed-dose combinations, and generic medications. Omitted cases, such as orphan drugs, are decided by CMED’s Technical-Executive Committee [21].

To request pricing approval, companies must provide detailed documentation, including international reference prices, cost of the API, projected number of patients, proposed market price, tax burden, estimated marketing expenses, and prices of existing substitutes. External reference pricing includes comparisons with up to ten countries, among them Australia, Canada, Spain, the United States, France, Greece, Italy, New Zealand, Portugal, and the country of origin [20].

CMED’s regulation distinguishes between the Maximum Price to the Government (MPG) and the Maximum Price to the Consumer (MPC) for the private market. The MPG is subject to a mandatory discount coefficient, which is periodically updated and applies to medicines purchases made by the SUS. Additionally, different federal and state taxes apply to medicines—ranging from 12–22%—depending on the product type and state of sale [20].

Affordability, sustainability, and innovation are important policy goals broadly pursued in the national pharmaceutical policy. However, these are not direct, explicit criteria in CMED’s pricing methodology. The system seeks to promote competition and predictability, enabling manufacturers to offer commercial discounts within established price ceilings [14].

While this model has contributed to the containment of pharmaceutical inflation and increased market access, new challenges has appeared. Structural factors—such as high interest rates, exchange rate volatility, and dependency on imported APIs—limit national innovation and industrial autonomy. Recent shifts in the global market, including reduced availability of generics and increased reliance on high-cost therapies, have led to inflation in drug prices surpassing both general and healthcare-specific inflation indices [11].

Furthermore, the global trend toward approving high-cost medicines based on limited clinical evidence poses an additional challenge. These products, sometimes lacking robust comparative data, may receive premium prices without a clear demonstration of cost-effectiveness [33].

Additionally, the reliance on international reference pricing can further reinforce elevated prices set in high-income countries, exacerbating inequities in access. To promote affordability and sustainability, pricing and regulatory frameworks must evolve to better assess therapeutic value, safeguard against excessive costs, and strengthen national negotiation power in a context of increasing global interdependence [33].

National pharmaceutical market

The Brazilian pharmaceutical market is highly concentrated, with a few large companies dominating a substantial share that can impact prices and access, particularly concerning essential medicines. In 2018, Brazil boasted 450 pharmaceutical companies and 59 drug-importing companies. In 2023, the private industries’ revenues were 58·16 billion USD in PPP, with prescription drugs constituting a large portion of revenue. Vaccines for coronavirus, antineoplastic monoclonal antibodies, and non-narcotic analgesics topped revenue percentages. Companies producing reference and biological medicines garnered the highest revenues, followed by those producing generic and similar medications. References and biological medications accounted for a substantial portion of products on the market (Table 1). A reference medicine is an innovator product registered with the Anvisa, with scientifically proven efficacy, safety, and quality at the time of registration. A similar medicine is a drug that contains the same active ingredient, concentration, dosage form, administration route, and therapeutic indications as the reference product. It may differ only in aspects such as shape, size, excipients, shelf life, packaging, and branding. Unlike generics, which are marketed under the nonproprietary name, similar medicines are marketed under a brand name and must demonstrate bioequivalence with the reference medicine [22]. The category of specific medicines includes, for example, products to prevent dehydration and medicines based on vitamins, minerals, and amino acids that are exempt from medical prescription. They, regardless of nature or origin, are not subject to bioequivalence testing against a compared product [13].

Table 1.

Distribution of pharmaceutical products in the Brazilian market, 2023

	Products	Number of manufacturers	Number of drugs	Number of drug forms*	Quantities sold	Revenue (Billions USD)
References	1,150	121	961	2,709	968,408,939	$19,58
Biologicals	350	81	251	661	94,122,398	$15,80
Similar	2,229	136	781	4,545	1,525,829,189	$10,16
Generics	2,557	90	528	4,665	2,479,397,641	$8,52
Specifics	515	98	258	1,272	673,365,103	$3,75
Phytotherapeutics	149	44	63	233	28,666,311	$0,35
Total	6,950	570	2,842	14,085	5,769,789,581	$58,16

Source: Anvisa/Cmed/Anuário Estatístico 2023 [15, 23] *Different form = tablet, capsule, syrup, injection

*Different form = tablet, capsule, syrup, injection

Productive Development Partnerships (PDPs) are public-private agreements aimed at transferring technology from private to public pharmaceutical companies for the production of specific medicines, intending to bolster local industry, reducing import dependence, and enhancing technological capacity in Brazil. The PDPs have faced challenges impacting their effectiveness. Although they were designed to strengthen the Health Economic-Industrial Complex and expand access to strategic medicines for the SUS, the PDPs failed to fully achieve their objectives. Difficulties in implementation, a lack of focus on genuine innovation, coordination problems between public and private partners, and a complex regulatory structure all contributed as well. The dependence on government subsidies without a sustainable model of industrial development and insufficient technology transfer resulted in vulnerability to political and economic changes, compromising national autonomy and productive capacity in the pharmaceutical sector [24].

Dependency on international markets and limited domestic production capacity for essential medicines heighten Brazil’s vulnerability in its pharmaceutical market. Additionally, market practices such as speculation by pharmacies or distributors can exacerbate drug shortages, creating artificial scarcity situations. For example, in 2022, during the COVID-19 public health emergency, shortages of certain essential medicines were reported in the public sector. In response, CMED issued a temporary resolution that allowed a temporary price adjustment mechanism for a limited number of medicines at risk of shortage [25]. This regulatory flexibility permitted manufacturers to market these products above the previously established ceiling prices, with the goal of restoring availability in the health system [25]. The medicines reappeared—right after the price hike.

The pharmaceutical industry faces a critical juncture in which commercial interests have been viewed to supersede public health concerns, leading to product discontinuations. From 2018 to 2022, 3817 medicines received marketing authorization in Brazil. There were discontinuations in the production of 1,868 medicines (48·9%of total), particularly in therapeutic classes such as alimentary tract and metabolism (15·6%), anti-infectives for systemic use (13·8%), and nervous system medications (12·7%).⁷ This trend reflects a broader issue of dwindling investments in research and development, as multinational pharmaceutical companies prioritize profitable sectors and allocate revenue towards financial activities like share buybacks rather than innovation, exacerbating the imbalance between private profit motives and public health needs [26]. Of the 46 drugs that received marketing authorization as new since 2022, 47·8% were antineoplastic and immunomodulating agents, and 26·1% were anti-infectives for systemic use [7].

Brazil has a network of 33 official pharmaceutical laboratories distributed in the five regions of the country. These laboratories are linked to the federal government (Ministry of Health and Armed Forces), state governments (Departments of Health and Science and Technology), and Universities. They play a crucial role in supplying public health services, yet they face challenges such as limited technological capacity and shortages of qualified personnel. Despite these obstacles, official laboratories contribute to public health by supplying vaccines, serums, synthetic and biological medicines, and other health products [27].

Overall, while the Brazilian pharmaceutical market presents vast opportunities, addressing challenges such as market concentration, dependence on international markets, and regulatory gaps is crucial for ensuring equitable access to medicines and strengthening the resilience of the healthcare system.

Public access to medicines

From the perspective of universal health coverage, the entire Brazilian population has the right to access medicines selected and standardized by Conitec. Since 1964, Brazil has maintained a National List of Essential Medicines (Rename), which has been periodically updated. With Conitec assuming responsibility for these updates, the scope of Rename has expanded to include medicines and pharmaceutical ingredients for the treatment of prevalent diseases in the population—including rare diseases—based on the best available scientific evidence. In addition to the national list, states and municipalities may adopt their own complementary lists. These must include all Rename medicines but may also add others, funded with local resources. The provision of medicines through the SUS is guided by Conitec’s evaluation process, which determines whether a medicine should be included in Rename. As of 2024, 2,011 medicines listed in Rename are guaranteed for free access.

The Brazilian Constitution defined health as a universal right and a state responsibility [4, 28, 29]. But, there are discrepancies in the interpretation of universality - all medications or for all? It has spurred health judicialization, in which courts compel SUS to provide drugs, challenging health care equity [30]. The inadequate availability of essential medicines at health care facilities exacerbates this issue, particularly affecting chronic disease management, underscoring the necessity for ongoing system enhancements to ensure universal access. Studies indicate regional disparities and deficiencies in the availability of only 52·9% of essential medicines, particularly for chronic diseases and conditions of epidemiological importance [31].

The aging demographic and increasing prevalence of chronic diseases emphasize the critical role of SUS in addressing the complex healthcare needs of the population. Additionally, the Farmácia Popular do Brasil Program, initiated in 2004, aimed to improve medication access by accrediting private pharmacies. However, its expansion to a copayment model raises concerns about higher government costs and potential implications for healthcare quality and efficiency [5]. Furthermore, the Farmácia Popular Program mainly reaches municipalities with the largest populations and the highest MHDI and is therefore not enough to address the inequalities in access pointed out [32]. The evolving landscape of healthcare delivery and funding necessitates continuous evaluation of such programs. Therefore, sustaining equitable medication access through SUS remains pivotal for ensuring public health welfare and mitigating healthcare disparities amidst evolving healthcare paradigms.

Challenges and opportunities

Given the persistent and multifaceted challenges surrounding access to pharmacological treatment in Brazil, this study aims to map key issues and suggest possible directions for action. Table 2 outlines each identified challenge alongside corresponding policy and programmatic opportunities that could be pursued within Brazil’s pharmaceutical and healthcare sectors. While not all topics could be explored in depth within this manuscript, we recognize the importance of articulating problems with feasible solutions and intend to examine each area more thoroughly in future studies using robust, systematic methodologies.

Table 2.

Strategies to face the challenges of access to medicines in a universal health coverage system

	Actions
Low investment in R&D	Enhance R&D Infrastructure: Augment the Ministry of ST&I and Ministry of Health’s investments in R&D infrastructure and explore new sources of resources, investments, and partnerships by the Official Pharmaceutical Laboratories [27,36]. Sustainable Industrial Transitions: Foster sustainable transitions through industrial and innovation policies, integrating subsystems into the circular economy. R&D Investment for National Revenue: Channel investments into R&D to generate national revenue, noting that companies in the pharmaceutical supply chain, particularly biotechnology firms, tend to yield higher excess returns than the S&P 500 [37]. Economies of Scale: Encourage large R&D organizations to leverage their size for greater efficiency and competitiveness compared to smaller entities [38]. Ministry of Health’s Cultural Shift: Advocate for a cultural shift within the Ministry of Health to serve not only as a monitor and collector of results but also as a guiding and facilitating agent for the activities of the Official Pharmaceutical Laboratories in collaboration with institutions like National Institute of Industrial Property, Anvisa, and control bodies [27]. Strategic Instrument Utilization: Promote the use of strategic instruments outlined by the National Policy for Technological Innovation in Health, beyond technological orders and Health Compensation Measures, to foster agreements for Technology Transfer and, importantly, for local development projects of original technologies or acquisitions that benefit the country [27]. Re-establishment of PDPs: Reintroduce PDPs with a new list of strategic products where a balance between health and economic considerations is paramount [27]. Balancing R&D Rewards: Adjust the relationship between excess rewards and the global R&D composition to prevent overinvestment in certain areas and under-supply in others [39].
High dependency on API	Educational and Technological Incentives: Stimulate incentives in education, technological training, and international partnerships to enhance field expertise [31]. Public Company Infrastructure: Invest in the enhancement of manufacturing infrastructure for public companies to develop new medicines [40]. Economic Regulation of the Sector: Economically regulate the sector by employing a competitive exchange rate that encourages exports, boosts domestic savings, and ensures competitiveness for companies with cutting-edge technologies. Strengthening SUS Coordination: Fortify the coordination between the SUS and the productive system.
High prices of drugs (mainly new drugs)	Drug Pricing Policy Reformulation: Reform drug pricing policies to promote the dynamic efficiency of pharmaceutical R&D, addressing distortions from conventional cost-effectiveness analysis and market competition [41]. Value-Based Pricing: Ensure that new medications are priced fairly, reflecting the added benefit of the new technology, and align with the country’s budget [42]. Transparency in Production Costs: Demand transparency from companies regarding production costs and pricing strategies. Proactive Measures for New Medicines: Implement proactive measures to improve the managed entry of new medicines, including pre-launch horizon scanning and the expansion of managed entry agreements [42]. Compulsory Assessment of New Drugs: Mandate the assessment of the impact of prices and effectiveness of new drugs post-market inclusion [42].
Patents	Patent Standards and Compulsory Licensing Review: Reevaluate aspects of compulsory licensing for medicines critical to public health. Collaboration with the Medicines Patent Pool: Encourage collaborations with the Medicines Patent Pool to promote equitable access to essential medicines globally [43]. Review of Patent Standards for Drug Innovation: Implement a review of the current patent standards to define drug patents, establishing objective criteria for innovation. This action aims to ensure that patents are granted based on genuine advancements in pharmaceuticals that contribute to significant therapeutic improvements. The criteria should be clear, measurable, and designed to encourage true innovation while preventing the extension of patent life for minor modifications that do not translate into clinical benefits.
Regulatory issues	Adherence to Guidelines and Essential Medicines List: Ensure adherence to guidelines and the national list of essential medicines, addressing decision-making levels in both public and private sectors [44,45]. Legal Framework Changes for PDPs: Propose changes in the legal framework of the PDP [27]. Prioritize Strategic Investments: The regulatory system should define clear priorities and allocate investments strategically over time.
Underfunding of the SUS	Ensuring Stability in Medication Funding: Establishing stability in funding over time through the implementation of robust financing mechanisms that can withstand economic fluctuations and ensure consistent investment in medications. Public health expenditures, especially on medications, should reflect a percentage of the GDP compatible with systems of universal health coverage. Federal and Sub-national Funding: Increase federal expenditures in conjunction with contributions from sub-national entities to secure funding stability [46].
Shortage of drugs	Supply Chain and Logistics: Strengthen the supply chain and implement robust logistics systems. Municipal Investment in Pharmaceutical Services: Urge municipalities to boost investment in pharmaceutical services to maximize benefits and ensure the fundamental right to medicine access. Stock Management: Address stock losses and manage inventory effectively. Essential Medication Production: Guarantee that companies continue to produce essential medications for the national market, especially older yet effective drugs with lower prices, and prevent their replacement with more expensive alternatives. Public Pharmaceuticals’ Role: Highlight the central role of public pharmaceuticals in fostering sustainable and endogenous innovation strategies focused on neglected diseases and local epidemiological profiles. Commercial and Public Interest Balance: Establish standards and practices to rebalance commercial and public interests, prioritizing health outcomes over profit motives [47]. Expand Production: In the event of a potential production disruption, collaboration with manufacturers is essential to determine if other companies can and are willing to increase production. This proactive approach is crucial for filling supply gaps [48]. Expedite Inspections and Reviews: Accelerating inspections and reviews of processes to assess manufacturing facilities and identify potential issues in advance is important. This helps prevent medication shortages [48]. Temporary Regulatory Flexibility: In situations where new sources of essential medications emerge, temporary regulatory flexibility should be exercised. This allows for quick adjustments to resolve shortage issues [48]. Develop Risk Management Plans: Manufacturers should be encouraged to create risk management plans for locations where specific drugs are produced, in order to identify and mitigate potential supply interruptions [48]. Continuous Collaboration and Monitoring: Continuous monitoring of the challenges related to medication supply and collaboration with manufacturers to improve availability is crucial. During critical periods, such as pandemics, manufacturers should assess and ensure the continuity of the entire supply chain [48].
Inequality in access to medication	Care Fragmentation Reduction: Reduce care fragmentation caused by the Popular Pharmacy program and ensure financial resources are allocated to a comprehensive model serving the entire population. Administrative Process Modernization: Modernize administrative processes to improve efficiency and reduce barriers to medication access. Information Dissemination: Ensure information is provided to users and healthcare professionals about medication availability and access methods. Health Production Regionalization: Propose the regionalization of health production activities, incorporating digital technologies like artificial intelligence and blockchain for infrastructure and service improvements. Understanding SUS Principles: Ensure uniform understanding of SUS principles, with universality as the guarantee for all Brazilian citizens, and comprehensiveness in healthcare Conitec’s Essential Medications: Ensure that medication provision strictly includes those deemed essential by Conitec. Real-World Data Utilization: Employ real-world data for decision-making, considering treatment effectiveness in actual use.49 Social Determinants and Health Outcomes: Establish connections between social determinants and health outcomes.

R&D = Research and Development; Anvisa = National Health Surveillance Agency, PDPs = Productive Development Partnerships, SUS = Unified Health System; API = Active Pharmaceutical Ingredient

One prominent challenge is the limited investment in R&D for pharmaceutical products, particularly within the Ministry of Health’s infrastructure. They are proposed actions such as increasing R&D investment, fostering public-private and international partnerships, and promoting a cultural shift that enables the Ministry to assume a more proactive role in guiding innovation. In line with recent updates to Brazil’s national innovation policies, the table also emphasizes the need to support sustainable industrial transitions by aligning innovation policies with circular economy principles and technological upgrading strategies.

Another pressing issue is Brazil’s high dependency on imported APIs. With COVID-19 pandemic, this vulnerability was more evident, after the global supply chain disruptions. Table 2 proposes a range of responses, including reinforcing national production capacity through investment in public pharmaceutical companies, creating educational and technological incentives, and introducing economic regulations that promote competitiveness and exports. These proposals align with ongoing efforts to revitalize Brazil’s industrial base, and local production initiatives by Farmanguinhos and other public labs [34].

To tackle the problem of high drug prices, the manuscript highlights the need for reform in the pricing system. Table 2 includes solutions grounded in international best practices and current regulatory discussions in Brazil. Although Brazil’s current pricing model already incorporates value-based elements by applying internal reference pricing when no added therapeutic benefit is demonstrated, and external reference pricing when such benefit is proven, recent developments signal the need for improvement and modernization of these mechanisms. For instance, CMED recently conducted a public consultation (closed on March 28, 2025) to review pricing criteria for advanced therapies [35]. Accordingly, our proposed actions include enhancing value-based pricing, increasing transparency—especially for medicines targeting rare diseases—and strengthening the integration of health technology assessment (HTA) mechanisms. Horizon monitoring and managed entry agreements—already part of regulatory discussions—are also highlighted as relevant strategies to improve cost-effectiveness and ensure the sustainability of public spending.

Regarding patent and regulatory challenges, proposed actions include revising national patent standards in light of flexibilities, supporting fairer access to essential medicines through strengthened collaborations with initiatives like the Medicines Patent Pool, and updating legal frameworks related to PDPs.

Other key challenges include underfunding of the SUS, drug shortages, and persistent inequalities in access to medicines. Table 2 highlights the importance of stable and diversified financing mechanisms, improved coordination between federal and subnational levels, enhanced supply chain management, and the modernization of administrative processes.

Despite all the challenges discussed, it is also important to acknowledge the significant progress Brazil has made over the past decades in the regulation and organization of pharmaceutical services. Prior to the 1988 Constitution and the creation of the SUS, access to medicines was marked by severe shortages, uncontrolled pricing, and widespread circulation of counterfeit and substandard products. The establishment of the National Medicines Policy, the Anvisa, and the CMED represent key milestones that have contributed to improving the availability, quality, and safety of medicines.

Yet, these achievements now coexist with a rapidly evolving global pharmaceutical landscape that poses new and complex challenges. To preserve and build upon past gains, Brazil must strengthen its response to both internal vulnerabilities and external pressures.

In addressing these national challenges, it is essential to consider the growing interdependence between domestic pharmaceutical policies and global dynamics. The internationalization of supply chains, cross-border regulatory influences, and the consolidation of multinational pharmaceutical corporations shape Brazil’s capacity to ensure access to safe, effective, and affordable medicines. Harmonization efforts—such as alignment with international standards on HTA, patent flexibilities, and pricing transparency—can support more effective and equitable regulatory frameworks.

Furthermore, global trends, including the increasing approval of high-cost medicines with limited therapeutic value, place pressure on national health budgets and challenge the principles of universality and cost-effectiveness. Brazil must navigate these external pressures by reinforcing regional cooperation, leveraging international mechanisms like the WHO’s Fair Pricing Forum and the Medicines Patent Pool, and advocating for global policies that prioritize public health over market exclusivities.

Taken together, these dynamics highlight the need for coordinated, multi-level strategies to build resilient and responsive pharmaceutical systems—strategies that transcend national borders while respecting local health needs and economic realities.

In summary, this manuscript provides a structured overview of the main barriers to equitable access to pharmacological treatment in Brazil and outlines potential policy solutions. While the proposed strategies do not capture the full complexity of each issue, they are grounded in current regulatory frameworks, recent public policy developments, and emerging debates. Each topic warrants further research using robust methodologies and in-depth evaluations. This article lays the groundwork for such investigations and aims to inform policymakers and researchers committed to building a more sustainable, efficient, and equitable pharmaceutical and healthcare system. It also serves as a foundation for future studies on access to pharmaceutical services in countries with universal health coverage.

Conclusion

In Brazil, access to prescription medications remains a critical challenge, exacerbated by socio-economic disparities, regional variations, and deficiencies within the health care system. These challenges underscore the urgent need for comprehensive reforms. To address this, collaborative efforts are essential: strengthening the investment in the healthcare system and enhancing regulatory frameworks are primordial, as well as strategic investments and definitions of fair pricing. Such efforts will allow prioritization of the distribution of medications with efficacy and safety guaranteed selected by Conitec while fostering the budget balance. Collaboration among government agencies, health care providers, pharmaceutical companies, and civil society organizations is also vital. Achieving equitable access to prescription drugs is fundamental for the well-being and health outcomes.

Although the focus is on the Brazilian health system, the issues raised are relevant to all nations that have universal health coverage systems. They are even more critical for those countries without universal coverage, but who seek to ensure access to medications. The global trend of the pharmaceutical industry to prioritize investments in highly profitable drugs at the expense of those intended to treat more prevalent and low-cost diseases is a cause for global concern. Ensuring that everyone has access to prescribed medications is a social obligation that should be seen as a priority by all governments in the world.

Abbreviations

Anvisa

Brazilian Health Regulatory Agency

API

Active Pharmaceutical Ingredient

CBAF

Basic Component of Pharmaceutical Assistance

CEAF

Specialized Component of Pharmaceutical Assistance

CEsAF

Strategic Component of Pharmaceutical Assistance

CMED

Medicines Market Regulation Chamber

Conitec

National Committee for Health Technology Incorporation at the SUS

GDP

Gross Domestic Product

HTA

Health Technology Assessment

MPC

Maximum Price to the Consumer

MPG

Maximum Price to the Government

PDPs

Productive Development Partnerships

PPP

Purchasing Power Parity

R&D

Research and Development

Rename

National List of Essential Medicines

SNVS

National Health Surveillance System

SUS

Unified Health System

WHO

World Health Organization

Author contributions

CMR has participated in the conception, and design of the work; collecting, analysis, and interpreting of data; and drafting and revising it. RP has participated in the collecting, analysis, and interpreting of data; and drafting and revising it. FAA, JAT, and AAGJ, has participated of the analysis, and interpretation of data; and revising it. ASK has participated in the conception, and design of the work; analysis, and interpretation of data; and and revising it.

Funding

The authors are supported individually or have researchers financed by Arnold Ventures and the Commonwealth Fund. This study was financed in part by the Coordenação de Aperfeiçoamento de Pessoal de Nível Superior - Brasil (CAPES) - Finance Code 001.

Data availability

No datasets were generated or analysed during the current study.

Declarations

Ethical approval

Not applicable because all data are available publicly.

Competing interests

The authors declare no competing interests.