The Current Use and Future Perspectives of Biosimilars in Pediatric Healthcare: A Narrative Review

ABSTRACT

Background and Aims

The integration of biosimilars into pediatric healthcare represents a significant evolution in treatment accessibility and cost‐effectiveness. Biosimilars—biological products highly similar to already approved biologics, have emerged as promising alternatives for managing chronic and rare pediatric conditions, including inflammatory bowel disease, juvenile idiopathic arthritis, psoriasis, asthma, lupus nephritis, and growth hormone deficiencies. Despite regulatory, clinical, and societal challenges, current data suggest biosimilars demonstrate comparable safety, efficacy, and immunogenicity profiles to their originator products. The review consolidates findings from recent clinical trials, real‐world data, and regulatory landscapes across the US and EU, emphasizing the growing role of biosimilars in pediatric pharmacotherapy. It also explores future directions in biosimilar development, highlighting the need for improved pediatric‐specific research, regulatory harmonization, and stakeholder education to ensure wider adoption and optimized outcomes in children.

Method

A structured literature search of PubMed, Scopus, Web of Science, and Google Scholar (2003–March 2026) identified English‐language studies on pediatric biosimilars. Relevant clinical, regulatory, and review articles were thematically synthesized.

Results

In pediatric patients with chronic conditions such as inflammatory bowel disease and rheumatologic disorders, biosimilars like infliximab and adalimumab offer efficacy and safety comparable to originator biologics, while lowering treatment costs. Economic analyses demonstrate that biosimilars are typically 15%–45% cheaper than reference products in Europe, and in some markets up to 85% lower, improving affordability and access to treatment in pediatric populations.

Conclusion

Biosimilars have a great deal of promise to improve therapeutic access and lower medical expenses associated with pediatric care. However, to maximize results and guarantee therapeutic fairness in pediatric medicine, broader adoption necessitates increased age‐specific clinical trials, harmonized regulations, better training for healthcare providers, and increased stakeholder trust.

1. Introduction

One of the most powerful tools available to physicians for preventing, managing and controlling diseases is pharmaceutical interventions and the advancements in the field of pharmaceuticals constitute a cornerstone of modern medical practice. The sequencing of the human genome has expanded our understanding and identification of the molecular mechanisms of a disease [1]. This has laid the foundation for discovering numerous proteins and biomolecular pathways associated with diseases and for curating therapeutic components that modulate physiological signaling pathways to enhance therapeutic efficacy. A notable year in the history of biopharmaceutical innovation was 1982, when Humulin was approved by the US Food and Drug Administration, the first recombinant human insulin, which marked the beginning of a new era of biologic therapeutics [2]. Since then, there has been a substantial growth in the application of biologics and related agents, particularly in areas like oncology, autoimmune and inflammatory disorders, and cardiometabolic diseases [3].

In contrast to chemically synthesized small‐molecule drugs with defined structure and functions, biological agents are derived from living sources such as animals or microorganisms and are categorized by their structural composition and complexity. They are broadly classified into categories including monoclonal antibodies, vaccines, blood and blood components, allergenics, gene therapies, tissues and recombinant therapeutic proteins, etc., with production relying on living systems such as cultures of mammalian cells, microorganisms, or plant‐based platforms [4]. The manufacturing process often involves sophisticated techniques such as recombinant DNA technology, gene cloning, and protein purification, to name a few. These procedures require high‐level technological proficiency to maintain consistency in potency and the overall quality of the product [5]. Stringent and well‐defined evaluation criteria are applied to ensure biosimilars remain within the permitted variability limits relative to the reference biologic and to confirm biosimilars demonstrate high similarity to originator products [6]. According to the Health Canada's guidance, biosimilars should be highly comparable to the reference biologic enabling the regulatory authorities to make two key judgements, one of which being that minor differences in quality will not negatively influence the therapeutic effectiveness and second, that the data already established from non‐clinical and clinical studies from the originator product remains the same for the biosimilar [7]. The similarity in biosimilar products, hence, refers to the similarity in terms of structure, function, efficacy, and immunogenicity profiles [6].

The increasing number of studies and clinical experiences highlights the use of biosimilars in children for several medical conditions [8]. Chronic inflammatory conditions such as inflammatory bowel disease (IBD) and juvenile idiopathic arthritis (JIA) among the pediatric population are being reported with growing frequency and prevalence. Pediatric IBD for instance, is more severe when compared to the adult population suffering and often requires early intervention with biologics [9]. For the treatment of pediatric IBD, biosimilars of infliximab have been used and proven to offer equivalent therapeutic safety and benefit compared to the originator drug. Similarly, the use of adalimumab biosimilars, a crucial one for the treatment of pediatric IBD and JIA, is becoming more prevalent in clinical settings. Those suffering from growth‐related disorders, the biosimilars of growth hormone are being used in pediatric cases and have proven to be effective [10]. The objective of this review is to consolidate existing studies on the application of biosimilars in pediatric care, focusing on their therapeutic efficacy, influence on patient outcomes, and implications for healthcare expenditure. See the key stages in biosimilar development in Figure 1.

Figure 1.

Biosimilar development workflow [11]. A schematic illustration of the sequential stages of biosimilar production from cell line generation to final packaging is depicted. Step 1: creation of the cell line—gene synthesis, vector insertion an clone selection. Step 2: cultivation and large‐scale production—cultivation and scale‐up in bioreactors. Step 3: recovery and refinement—protein recovery, purification, and CQA evaluation. Step 4: product formulation and finalization—formulation, sterile filling, packaging, and confirmation of biosimilarity.

1.1. Literature Search Strategy

A structured literature search was conducted to identify relevant evidence on the current use and future perspectives of biosimilars in pediatric healthcare. Databases including PubMed, Scopus, Web of Science, and Google Scholar were systematically searched for studies published between 2003 and March 2026, reflecting the period of increasing biosimilar development and clinical adoption.

The search strategy used combinations of keywords such as “biosimilars,” “paediatric,” or “pediatric,” “children,” “biologics,” “biosimilar drugs in children,” “clinical use of biosimilars,” “regulatory perspectives,” and “future of biosimilars in paediatrics,” Additionally, reference lists of selected articles were manually screened to identify further relevant studies.

Inclusion criteria comprised English language publications focusing on biosimilars and addressing the use, efficacy, safety, or regulatory aspects, and their uses in pediatric indications. Eligible study types included clinical trials, observational studies, review articles, and expert opinions. Non‐English publications, conference abstracts with insufficient data, and duplicate records were excluded. Duplicates were removed during the screening process.

Approximately 200 articles were initially screened based on titles and abstracts, followed by full‐text review, with around 66 studies included in the final synthesis. Findings were thematically analyzed to provide a comprehensive overview of current evidence and emerging trends.

1.2. Regulatory Landscape

Due to the complexities in inherent properties and manufacturing processes of biologicals, including biosimilars, the development and regulation is an excruciating process. When biosimilars are used for pediatric patients, a demographic group with unique physiological and pharmacological profiles, regulatory pathways governing the approval of biosimilars must very intricately navigate the scientific as well as ethical considerations in order to ensure the safety and efficacy. Although the regulatory frameworks set by the United States Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the World Health Organization (WHO) are stringent for approval processes of all drugs, a particular emphasis is given on some specific guidelines and requirements that pertain to the approval process of biosimilars for pediatric patients. Biosimilars are developed in an abbreviated pathway compared to novel biologics by avoiding the repetition of longer clinical trials and taking advantage of the reference product's existing clinical knowledge. In 2004, the EMA (a pioneer in the field of biosimilars) launched a regulatory framework that highlighted a step‐by‐step process centered on strong similarity studies to the reference product to ensure no meaningful clinical differences exist between the two [12]. Likewise, the FDA implemented an abbreviated approval pathway in the year 2010 under the Biologics Price Competition and Innovation Act (BPCI Act), which made it compulsory for the developers of biosimilars to establish a high level of similarity to an already FDA‐approved reference biologic. The pathway also addresses guidelines for interchangeability, allowing substitutions applicable by state laws [13]. Although the approval procedures for biosimilars are well defined by several regulatory bodies, pediatric applications require special considerations due to their unique physiological and developmental factors. In the United States, the Pediatric Research Equity Act (PREA), enforced in the year 2003, requires biosimilar sponsors to conduct pediatric assessments unless a waiver or deferral is justified and granted.

The objectives of pediatric assessments are to demonstrate the safety and effectiveness for its intended pediatric indications and ensure appropriate dosing and delivery methods for each pediatric age group [14]. This procedure is usually initiated by the submission of an initial Pediatric Study Plan (iPSP) during the early stages of development. PREA deferrals were granted when the indication had orphan drug exclusivity or lacked a pediatric drug formulation [15]. In the absence of such waivers, biosimilars were approved for the same pediatric indications based on the available scientific data from the reference product [15]. Hence, to prevent any unnecessary delays in the approval of biosimilars in the USA, early preparation of the iPSP, pediatric dosing device development plan, and human factor study plan should be done [16]. Pediatric‐specific regulations were implemented by the European Union in the year 2007, aimed at promoting development and improving accessibility of medicines for patients [17]. However, biosimilars and generics are excluded from pediatric assessment requirements. As a result, all the approved biosimilars in the EU are generally authorized for the same therapeutic indications except for Rituximab [18]. The originator rituximab (MabThera) is approved for treating non‐Hodgkin's lymphoma, granulomatosis with polyangiitis (GPA), and microscopic polyangiitis (MPA) among pediatric patients; however, its biosimilar counterparts (Rixathon, Truxima/Tuxella/Ritemvia/Blitzima, and Ruxience) lack approval for use in patients under 18 years of age.

1.3. Pediatric Considerations for Generics, Biologics, and Biosimilars

The use of generics, biologics, and biosimilars in pediatric pharmacotherapy must be cautiously assessed due to developmental alterations in drug metabolism, insufficient information from clinical trials, and uncertainties regarding effectiveness and safety.

Generics are chemically equivalent to their patented equivalents and are often accepted in pediatric treatment after bioequivalence is proven. Nevertheless, differences in inactive components might alter medication absorption and tolerance in children, particularly newborns and young children. Furthermore, the absence of pediatric‐friendly formulations, such as liquid suspensions, might complicate dosage and adherence.

Biologics are complex compounds derived from live creatures that are used to treat a wide range of ailments, including autoimmune disorders and cancer. The use of biologics in children is sometimes hampered by a paucity of substantial clinical trials, necessitating dependency on data derived from research on adults. This technique is problematic due to variations in immune system development and illness progression in children. Furthermore, immunogenicity, or the body's immunological reaction to the biologic, can influence both the safety and efficacy among young patients [19].

Biosimilars, on the other hand, are very similar to their parent biologics but not identical. The use of biosimilars in pediatric populations is treated with extreme caution. They are more readily available and less expensive, but there are still issues with their immunogenicity and the habit of projecting data from adults to children [20]. Studies have demonstrated, for example, that although biosimilars such as infliximab are successful in treating juvenile IBD, the strategy of transitioning from originator biologics to biosimilars should be meticulously evaluated, particularly in patients who are in remission. To demonstrate biosimilarity, regulatory authorities like the FDA and EMA seek extensive analytical and experimental proof; nonetheless, data focused on pediatric tests are usually unavailable [21].

1.4. Current Use of Biosimilars in Pediatrics

The use of biosimilars in pediatric healthcare is rapidly growing, offering more affordable alternatives to original biologic treatments for diseases such as rheumatic conditions, inflammatory bowel disease, and growth hormone deficiencies. Studies indicate that biosimilars like infliximab and recombinant human growth hormone demonstrate equivalent safety and effectiveness compared to their reference biologics in children, with promising clinical outcomes. Nevertheless, challenges persist, including a scarcity of pediatric‐specific data, regulatory complexities, and the need for improved education among clinicians and caregivers. Ongoing research and transparent communication are essential to optimize biosimilar adoption in pediatric populations. Refer to Table 1 for the approved biosimilar list for pediatric patients.

Table 1.

Approved biosimilar for pediatric indications.

Biosimilar name	Approval date	Active substance	Reference biologic	Pediatric indications	Reference
CT‐P13 (Inflectra/Remsima)	EMA: 2013, FDA: 2016	Infliximab	Remicade	Pediatric Crohn's disease, ulcerative colitis, others	[8]
Amjevita	FDA: 2016	Adalimumab	Humira	Juvenile idiopathic arthritis, Crohn's disease, ulcerative colitis, others	[22]
Cyltezo	FDA: 2021	Adalimumab	Humira	Juvenile idiopathic arthritis, Crohn's disease, ulcerative colitis, others	[23]
IXIFI	FDA: 2017	Infliximab	Remicade	Pediatric Crohn's disease, ulcerative colitis, others	[24]
Nepexto	EMA: 2020	Etanercept	Enbrel	Juvenile idiopathic arthritis, pediatric psoriasis	[25]
Tocilizumab‐bavi	FDA: 2023	Tocilizumab	Actemra	Polyarticular juvenile idiopathic arthritis ( ≥ 2 years), systemic JIA ( ≥ 2 years)	[26]
Nypozi (filgrastim‐txid)	FDA: 2024	Filgrastim	Neupogen	Febrile neutropenia	[27]
Pyzchiva (ustekinumab‐ttwe)	FDA: 2024	Ustekinumab	Stelara	Plaque psoriasis, psoriatic arthritis (pediatric ≥ 6 years via extrapolation)	[28]

The table summarizes the approved biosimilars for pediatric use, highlighting dates of approvals by regulatory authorities, active substances, reference biologics, and indications. Almost all of the above‐mentioned biosimilars have received approval from the FDA for pediatric indications. An exception is Nepexto, which was approved for pediatric use by the EMA only in May 2020. The only two IBD biologic therapies, Infliximab and Adalimumab, are biosimilars with FDA approval. Biosimilars play a crucial role in pediatric rheumatology and for children with rheumatic diseases, with the most common ones being Adalimumab, Etanercept, Tocilizumab, and Ustekinumab. Two recently approved biosimilars, Nybozi (Filgrastim) and Pyzchiva (Ustekinumab), expand treatment options for febrile neutropenia and pediatric psoriasis‐related conditions.

1.5. Biosimilars and Indication Extrapolation

In the development of biosimilars, extrapolation refers to the approval of a product for indications of the reference biologic without direct comparison using the clinical trials. This is justified through a scientific framework that integrates the totality of evidence, allowing data from one indication to support others [29]. Such an extrapolation avoids unnecessary duplication of clinical studies and accelerates development. The extrapolated data from one indication to another is permissible when biosimilarity has been demonstrated using a robust framework, incorporating safety, efficacy, and immunogenicity assessments. This principle, grounded with adequate scientific rationale, is widely accepted and has been applied in regulatory decision‐making for many years [30].

1.6. Biosimilar for Pediatric Asthma

Around 6 million children are diagnosed with asthma, a condition that imposes a considerable burden through disability and, in some cases, premature mortality. On an annual basis, asthma leads to over 10 million absences from school [31]. The financial impact is also significant, with emergency department expenses for pediatric asthma reaching roughly $272 million in 2010 alone [32]. Additionally, the disease can compromise the quality of life; findings from a national survey reported that 5.5% of children between the ages 5–17 with symptomatic asthma experienced limitations in their day‐to‐day activities. Advancements in biological therapies have significantly changed the approach to managing asthma by focusing on interventions against specific inflammatory pathways [33]. Although their use in pediatric patients was constrained by age‐related eligibility, with the recent regulatory changes, new approvals have facilitated their use in younger populations [34]. One of the first targeted biologics to be approved by the US Food and Drug Administration in 2003 for the treatment of moderate‐to‐severe allergic asthma (AA) is an anti‐IgE mAb, Omalizumab [35]. It is a subcutaneously administered monoclonal antibody which was previously licensed for use in adults and adolescents aged 12 and above with uncontrolled severe allergic asthma despite a daily dose of treatment regimen on inhaled corticosteroid, along with inhaled LABA [36]. The European Medicines Agency and the Food and Drug Administration authorized the pediatric indication for Omalizumab for children aged 6 and above suffering from asthma in 2009 and 2016, respectively [37]. In a phase III clinical trial, P043 (Zerafil, CinnaGen, Iran) demonstrated equivalent efficacy to Omalizumab in reducing asthma exacerbations with comparable safety profiles. These findings support the clinical use of P043 as a biosimilar to Omalizmab for patients with moderate to severe AA [38]. Omlyclo (omalizumab‐igec) is the first FDA‐approved biosimilar that can be used in place of Xolair. It is used to treat children aged 6 years and older with moderate‐to‐severe allergic asthma that is not well controlled by inhaled corticosteroids. It works as an add‐on treatment and has similar safety and effectiveness as the original medicine [39].

1.7. Biosimilar for Pediatric Lupus Nephritis

Lupus nephritis represents one of the most serious clinical manifestations seen in patients with systemic lupus erythematosus (SLE), which is a chronic autoimmune disease that leads to inflammation and injury across various organ systems, particularly affecting the kidneys [40]. Lupus nephritis occurs more often and is more severe in children, and it has been linked to increased rates of morbidity and mortality [41]. This disease affects approximately 50%–82% of children, while it impacts 20%–40% of adults [42]. In pediatric lupus nephritis, the only biosimilar drug named Rituximab biosimilar BCD020, showed superior efficacy over conventional non‐biologics treatment. Despite years of experience treating SLE, rituximab has not yet been officially approved for use in adult or pediatric therapy since there aren't enough randomized clinical trials to support its effectiveness in treating SLE [43]. Important age‐related safety issues, especially with regard to long‐term hypogammaglobulinemia, are reflected in the distinct regulatory approach to rituximab approval in pediatric versus adult populations. Granulomatosis with polyangiitis and microscopic polyangiitis were only approved by the FDA for pediatric children 2 years of age and older in 2019. Research shows that between 44% and 55.2% of children treated with rituximab develop hypogammaglobulinemia, and 50.5% of these instances persist for more than 6 months [44, 45]. Children's immature bone marrow microenvironment, reduced baseline IgG levels, and fewer memory B cells all contribute to this increased sensitivity [44, 46].

1.8. Biosimilar for Pediatric Inflammatory Bowel Disease

IBDs are chronic, lifelong conditions that affect the gastrointestinal system and exhibit relapsing‐remitting behavior. They include Crohn's disease (CD), ulcerative colitis (UC), and inflammatory bowel disease unclassified (IBD‐U). In order to lessen the financial burden of these chronic conditions, biosimilars are a major topic. The use of biosimilars offers a way to significantly reduce healthcare costs and improve patient access to biologic therapies by reducing overall treatment costs and providing a high standard of patient care. Biosimilars of anti‐TNF‐α have become more and more popular in recent years, even in pediatrics [8]. The first infliximab biosimilar entered the market in September 2013. In 2015, the ESPGHAN IBD Porto group released the first position paper on the use of biosimilars in pediatric IBD. Since then, more data have emerged showing that biosimilars are a safe and efficient substitute to the originator for both adults and children [47]. An infliximab biosimilar (CT‐P13) is presently widely used in the majority of European nations. Studies conducted both in vitro and in vivo have demonstrated that CT‐P13 and the reference drug infliximab are extremely similar. Using evidence from preclinical research and clinical trials in rheumatic indications, CT‐P13 was licensed for treatment in patients with CD [48].

1.9. Biosimilar for Pediatric Arthritis

In 2013, the infliximab biosimilar was launched, representing the beginning of the use of biosimilars in pediatric rheumatology. Since then, new biosimilars have been developed, such as adalimumab, rituximab, and etanercept, giving children with rheumatic diseases more alternatives for therapy [49]. One recent study compared the immunogenicity profile, safety, and effectiveness of biosimilars of etanercept and adalimumab to their original products in children with juvenile idiopathic arthritis. As a result, children with juvenile idiopathic arthritis show a higher overall profile of biosimilars than originators [50].

1.10. Biosimilar for Psoriasis in Pediatric Patients

Children suffering from psoriasis, a chronic inflammatory skin disease, may experience discomfort and irritation resulting from red, scaly areas. The condition can start at any age, including infancy, and affects about 1% of children [51]. With equal performance and safety to biologics from the original manufacturer and an additional advantage of lower prices, biosimilars are showing potential as a preferred therapy option for pediatric psoriasis. Children with moderate to severe plaque psoriasis who do not react to topical treatments or phototherapy might benefit most from these medications. Biosimilars for pediatric usage are now more widely available owing to the latest approvals from regulatory bodies. Clinicians are now presented with additional choices for treating these disorders in children, thanks to the approval [52]. The efficacy of biosimilars in pediatric populations has been demonstrated by clinical data. In a noteworthy case study, a kid treated with an adalimumab biosimilar for 24 weeks saw a total cure of scalp and nail psoriasis, resulting in a Psoriasis Area and Severity Index (PASI) score of 100. This result highlights how biosimilars have the potential to provide pediatric patients with substantial therapeutic advantages [53].

2. Safety and Efficacy in Pediatric Patients

Since biosimilars provide affordable substitutes for original biologics in a range of conditions, they have grown in importance in pediatric treatment. Over 7000 pediatric patients participated in the multinational post‐marketing surveillance research PATRO Children, which evaluated the extended safety and efficacy of biosimilar recombinant human growth hormone (rhGH). According to the study's findings, biosimilar rhGH is safe and effective, with the majority of side effects being mild to moderate. After 5 years of therapy, all signs showed persistent recuperation [54]. The German BIKER database conducted a prospective observational trial to assess etanercept biosimilars in children with JIA (Juvenile Idiopathic Arthritis). The research observed that biosimilars were as effective and safe as their original counterparts. Adverse events were similar, with only a little increase in injection site responses under biosimilar treatment, which did not affect treatment adherence. In both groups, disease activity either declined or stayed in remission [55]. A Pegaspargase biosimilar was used in the treatment of 72 pediatric ALL patients in a prospective research carried out in India. According to the research, the biosimilar was safe and acceptable, with mild to moderate suspected side effects. Interestingly, no allergic responses were noted, and the majority of side effects were during the treatment's induction phase [56]. The combined findings of this research support the safety and efficacy of biosimilars for pediatric patients in a range of medical specialties. With results similar to those of original biologics, their usage can improve children's access to necessary medicines.

3. Challenges and Barriers

Several key challenges hinder the adoption of biosimilars in pediatric care. Primarily, there is a lack of pediatric‐specific clinical trials, as children are often excluded from studies due to ethical and logistical difficulties. This limited data creates uncertainty about the long‐term safety and efficacy of biosimilars in children, leading to cautious prescribing practices. Hesitancy among healthcare providers and patients further restricts biosimilar use. Many clinicians remain skeptical about switching from originator biologics due to concerns over interchangeability and immunogenicity. Likewise, parents and caregivers may have misconceptions or fears regarding biosimilar safety, which can reduce acceptance and adherence. Economic factors also pose barriers. Although biosimilars are intended to lower treatment costs, high development and regulatory expenses can limit price reductions. Regulatory challenges contribute to delays and limited labeling for pediatric use. Extrapolating adult data to children is approached cautiously, and the lack of harmonized pediatric guidelines complicates approval processes. Differences in regulatory frameworks across regions further affect biosimilar availability [57]. Finally, manufacturing and supply chain complexities impact the consistent availability of biosimilars. The intricate production process requires strict quality control, and any disruptions can cause shortages, adversely affecting children who depend on continuous treatment. In conclusion, addressing these barriers through enhanced pediatric research, education, regulatory clarity, economic strategies, and supply chain stability is essential to improve biosimilar adoption and optimize outcomes in pediatric patients. Refer to Figure 2 for the key challenges and barriers.

Figure 2.

Challenges and barriers of biosimilar in pediatric care [58]. The figure highlights the key challenges, including limited availability of clinical data concerning pediatrics, regulatory restrictions, misconceptions, logistic issues, and economic factors. These barriers collectively hinder the progress of biosimilars' use in pediatric care.

4. Economics of Pediatric Biosimilars

Biosimilars play an important role in reducing the high cost of biologic therapies in pediatric healthcare. Many chronic childhood conditions such as juvenile idiopathic arthritis (JIA), inflammatory bowel disease (IBD), and certain cancers require long‐term or even lifelong treatment with monoclonal antibodies. Because original biologic medicines are very expensive, access can be limited, especially in resource‐constrained settings. In Europe, biosimilars are typically sold at prices 15%–45% lower than their originator (reference) products, and in some markets, they are 25%–85% lower, often costing about 60%–85% of the original price [59]. However, despite these reductions, their absolute cost can still be substantial, which may continue to restrict access in low and middle income countries.

Their complex manufacturing process largely shapes the economics of biosimilars. Large scale cell culture, purification, and stability testing are similar to those required for originator biologics, keeping production costs high. Although abbreviated clinical development pathways lower overall investment, the savings are moderate. In pediatric practice, switching to biosimilars in IBD and hematology has been associated with meaningful cost savings without compromising clinical outcomes. Infliximab biosimilars, in particular, have been introduced at prices approximately 10%–50% lower than their originator products in several markets [60, 61]. Encouraging interchangeability, supporting pediatric‐specific studies, and using competitive tendering systems could further increase savings and allow those funds to be reinvested to expand patient access. Although high development costs limit how inexpensive biosimilars can be, they still reduce the price gap with originator biologics and improve the financial sustainability of monoclonal antibody therapy in children [49]. Overall, pediatric biosimilars provide measurable economic value, but policy frameworks must focus on sustaining trust, continuity of care, and long‐term outcomes rather than price alone. However, the extent of cost‐effectiveness is influenced by the specific country context and the structure of its healthcare system.

5. Future Perspectives of Biosimilars in Pediatric Care

Current pediatric clinical trials and observational studies of biosimilars primarily evaluate the safety and efficacy of anti‐TNF‐α therapies, including adalimumab and infliximab, in chronic conditions such as inflammatory bowel disease, psoriasis, and non‐infectious uveitis. Adalimumab biosimilars demonstrated a comparable effectiveness and safety profile in pediatric psoriasis. Transitioning from the reference adalimumab to biosimilars did not compromise treatment outcomes or safety. However, a higher likelihood of treatment discontinuation was observed in patients who switched from Humira to biosimilar products [62].

Real‐world evidence registries provide long‐term safety, effectiveness, and immunogenicity data, supporting informed use of biosimilars in pediatric patients. In pediatric populations, switching from originator biologics to biosimilars has been shown to be generally safe and effective. Switching from originator infliximab to its biosimilar appears to be a safe strategy for children with Crohn's disease, with the biosimilar maintaining effectiveness comparable to the original product following the transition [63].

The future of biosimilars in pediatric care is promising, driven by the development of novel biosimilars targeting pediatric‐specific indications. The continuous development of emerging biosimilars for pediatric‐specific indications, such as juvenile idiopathic arthritis and inflammatory bowel disease, promises to broaden treatment access and mitigate healthcare costs. Concurrently, advancements in pharmacovigilance and personalized medicine are crucial; enhanced monitoring systems will facilitate precise safety assessments and allow for individualized treatment strategies, optimizing patient outcomes based on their unique characteristics. Furthermore, fostering collaboration in pediatric research among academia, industry, regulatory bodies, and patient organizations is essential. Such partnerships can accelerate the generation of robust clinical data and real‐world evidence, thereby addressing existing knowledge gaps and instilling greater confidence in biosimilar prescribing [64]. Finally, proactive policy recommendations and advocacy initiatives are vital. This includes developing clear regulatory guidelines, incentivizing pediatric‐focused research, and implementing comprehensive educational campaigns for both healthcare providers and families. Collectively, these efforts will cultivate an environment conducive to the successful integration of biosimilars, ultimately improving treatment accessibility, affordability, and therapeutic results for pediatric patients.

6. Conclusion

Biosimilars are poised to dominate the pharmaceutical market, with projections indicating they could contribute to 50% of top drug sales. Although their use in pediatrics is currently limited, clinical evidence increasingly supports their safety, efficacy, and cost‐effectiveness in treating complex pediatric conditions such as juvenile idiopathic arthritis, inflammatory bowel disease, and certain cancers. This review highlights the growing potential of biosimilars to enhance access and reduce healthcare costs in pediatric care. However, to fully integrate biosimilars into pediatric practice, critical steps are needed: expanding age‐specific clinical trials, streamlining regulatory pathways, improving healthcare provider education, and building trust among caregivers. With these measures, biosimilars can significantly improve therapeutic equity and innovation in pediatric medicine.

Author Contributions

Zubaier Ahmed: conceptualization, data curation, and writing‐original draft. Tasfiah Tasnim Maha: conceptualization, data curation, and writing‐original draft. Sanjana Nowsher: data curation and writing –original draft. Wasima Taslim: data curation and writing – original draft. Fatema‐Tuz‐Zohora: conceptualization, supervision, writing – review, and editing. Sabrina Sharmin: conceptualization, supervision, writing – review and editing. Hasina Yasmin: Addressing revision, writing – review, and editing.

Funding

The authors have nothing to report.

Ethics Statement

This narrative review is based on previously published studies and does not involve human or animal subjects. Therefore, ethical approval and informed consent were not required.

Conflicts of Interest

The authors declare no conflicts of interest.

Transparency Statement

The corresponding authors, Zubaier Ahmed and Fatema‐Tuz‐Zohora, affirm that this manuscript is an honest, accurate, and transparent account of the study being reported; that no important aspects of the study have been omitted; and that any discrepancies from the study as planned (and, if relevant, registered) have been explained.

Data Availability Statement

Data sharing not applicable as no new data was generated, or the article describes entirely theoretical research.