Pediatric Brain Cancer - Market Insight, Epidemiology, and Market Forecast - 2034

Key Highlights

• Brain tumors are the most common type of solid cancer in children and are the second most common childhood cancer overall, after leukemia.
• Tackling brain tumors is tough. Medulloblastoma, juvenile pilocytic astrocytoma, ependymoma, and low-grade glioma are just a few of the more than 100 types and subtypes of pediatric brain cancers for which researchers are trying to develop treatments. A combination of surgery, radiation therapy, and chemotherapy is mainly used for the treatment of pediatric brain tumors.
• For nearly four decades, pediatric patients with low-grade gliomas were treated with the same old chemotherapy until TAFINLAR + MEKINIST were approved.
• Drugs approved for the treatment of pediatric brain cancer include OJEMDA (Day One Biopharmaceuticals), TAFINLAR + MEKINIST (Novartis), AFINITOR DISPERZ (Novartis), and others.
• In April 2024, Day One biopharmaceuticals announced that the US Food and Drug Administration (FDA) had approved OJEMDA (tovorafenib). This indication is approved under accelerated approval based on response rate and duration of response. With the approval, Day One received a rare pediatric disease priority review voucher from the FDA.
• The FDA has granted Rare Pediatric Disease (RPD) Designation to LAM561 (idroxioleic acid, sodium), an investigational drug candidate for patients with Pediatric-Type Diffuse High-Grade Glioma (pdHGG).
• FDA is reviewing Chimerix’s NDA for dordaviprone to treat recurrent H3 K27M-mutant diffuse glioma.

PDUFA action date is set for August 18, 2025.

DelveInsight's “Pediatric brain cancer– Market Insight, Epidemiology and Market Forecast – 2034” report delivers an in-depth analysis of Pediatric brain cancer epidemiology, market, and clinical development in Pediatric brain cancer. In addition to this, the report provides historical and forecasted epidemiology and market data as well as a detailed analysis of the Pediatric brain cancer market trends in the United States, EU4 (Germany, France, Italy, and Spain ), the United Kingdom, and Japan.

The Pediatric brain cancer market report provides real-world prescription pattern analysis, emerging drugs assessment, market share, and uptake/adoption pattern of individual therapies, as well as historical and forecasted Pediatric brain cancer market size from 2020 to 2034 in 7MM. The report also covers current Pediatric brain cancer treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s underlying potential.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Pediatric Brain Cancer Understanding and Treatment Algorithm

Pediatric Brain Cancer Overview and Diagnosis

A brain tumor arises from genetic alterations in brain cells, leading to a mass of abnormal cells. Primary brain tumors start in the brain, unlike secondary tumors that spread from other body parts. They can be low-grade (less aggressive) or high-grade (very aggressive). The exact cause is unknown, with some tumors linked to hereditary germline mutations, but most result from non-hereditary somatic mutations. Central nervous system tumors, including brain and spine tumors, are the most common solid tumors in children, with around 4,500 new cases yearly, and are the leading cause of cancer deaths. About 60% of pediatric brain tumors are in the posterior fossa, with the most common types being medulloblastoma, juvenile pilocytic astrocytoma (JPA), ependymoma, diffuse intrinsic pontine glioma (DIPG), and atypical teratoid rhabdoid tumor (ATRT). The remaining 40% occur in the cerebral hemispheres and include astrocytomas, gangliogliomas, craniopharyngiomas, supratentorial primitive neuroectodermal tumors (PNET), germ cell tumors, dysembryoplastic neuroepithelial tumors (DNET), oligodendrogliomas, and meningiomas.

When a child shows symptoms of a brain tumor, a pediatrician or pediatric neurologist typically performs a thorough evaluation, often starting with an MRI scan. If a tumor is detected, a neurosurgical consultation follows to develop an appropriate treatment plan involving various specialists, such as pediatric oncologists and ophthalmologists. Key factors in planning treatment include determining the tumor's location via imaging, identifying the tumor type through microscopic examination, and assessing the tumor grade to understand its aggressiveness.

The Pediatric brain cancer report provides an overview of Pediatric brain cancer pathophysiology, and diagnostic approaches, along with a real-world scenario of a patient’s journey beginning from the first symptom, the time taken for diagnosis, to the entire treatment process.

Further details related to country-based variations in diagnosis are provided in the report.

Pediatric brain cancer Treatment

The treatment of pediatric brain cancer typically involves a multidisciplinary approach tailored to the individual child's condition. This often includes a combination of surgery, radiation therapy, and chemotherapy. Surgery aims to remove as much of the tumor as possible without causing damage to surrounding healthy tissue. Radiation therapy uses high-energy rays to target and destroy cancer cells, while chemotherapy involves the use of powerful drugs to kill cancer cells or stop them from growing. Additionally, other treatments such as targeted therapy, immunotherapy, and stem cell transplant may be considered depending on the specific type and stage of the cancer. Pediatric patients must receive comprehensive care that addresses not only the cancer itself but also its potential effects on neurological function and overall well-being.

Further details related to treatment are provided in the report.

Pediatric brain cancer Epidemiology

The Pediatric brain cancer epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented as total incident cases of pediatric brain cancer, gender-specific cases of pediatric brain cancer, tumor grade-specific cases of pediatric brain cancer, age-specific cases of pediatric brain cancer, histology-specific cases of pediatric brain cancer, and total treated cases of pediatric brain cancer in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), and the United Kingdom, and Japan from 2020 to 2034.

• The incidence of pediatric brain tumors varies among different countries. It ranges from 1.15 to 5.14 cases per 100,000 children, with the highest rates reported in the United States.
• Ependymoma accounts for about 9% of all childhood brain and spinal cord tumors, affecting about 200 children per year in the United States.
• Males are more likely than females to develop an aggressive form of brain cancer called glioblastoma. There is also evidence that females tend to respond better than males to standard therapy for this disease.
• In Germany, approximately 500 children and young people are newly diagnosed with brain cancer every year, and about 40 children and adolescents under the age of 18 years are newly diagnosed with ependymoma each year.
• Medulloblastoma is the second most common brain tumor in children. It is the most common malignant (high-grade) children’s brain tumor. Around 52 children are diagnosed with medulloblastoma each year in the UK.

Pediatric Brain Cancer Drug Chapters

The drug chapter segment of the Pediatric brain cancer report encloses a detailed analysis of Pediatric brain cancer marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into Pediatric brain cancer's pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.

Marketed Drugs

OJEMDA (tovorafenib): Day One Biopharmaceuticals

OJEMDA (tovorafenib) is a type II RAF inhibitor manufactured by Day One Biopharmaceuticals for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication was approved under accelerated approval based on response rate and duration of response. It was approved by the US FDA in April 2024.

With OJEMDA now available and the first prescriptions being written, patients have begun enrolling in EveryDay Support from Day One, a comprehensive program that offers personalized services for OJEMDA patients and their care teams, including insurance coverage support, financial assistance options, and educational resources throughout the treatment journey.

TAFINLAR (dabrafenib) + MEKINIST (trametinib): Novartis

TAFINLAR (dabrafenib) + MEKINIST (trametinib) was approved by the US FDA in March 2023 for the treatment of pediatric patients 1 year of age and older with LGG with a BRAF V600E mutation who require systemic therapy. The FDA also approved liquid formulations of TAFINLAR and MEKINIST, marking the first time a BRAF/MEK inhibitor was developed in a formulation suitable for patients as young as one year of age.

Emerging Drugs

LAM561: Laminar Pharmaceuticals

LAM561 (2-hydroxyoleic acid) is a synthetic derivative of oleic acid that can be taken orally and that could be able to reach cells in the brain by crossing the blood-brain barrier. This drug alters the composition of the plasma membrane in cancer cells, reducing the activity of membrane-associated signaling proteins that are known to promote tumor growth. Administering LAM561 has shown promising results in the treatment of aggressive brain tumors and glioblastomas. The company is currently conducting a Phase II clinical trial of LAM561 in pediatric glioma patients, and anticipates reaching the market between 2028-2029.

ONC201 (dordaviprone): Chimerix

ONC201 is a first-in-class, highly selective antagonist of dopamine receptor D2 (DRD2) and ClpP agonist that can penetrate the blood-brain barrier effectively. ONC201 engages proven anti-cancer pathways that lead to apoptosis in cancer cells. The US FDA has granted ONC201 a Rare Pediatric Disease Designation for the treatment of H3 K27M mutant glioma. Chimerix is currently conducting a Phase III trial of the drug in pediatric patients with newly diagnosed DIPG and recurrent/refractory H3 K27M gliomas.

Pediatric Brain Cancer Market Outlook

The market outlook for pediatric brain cancer treatment is becoming increasingly optimistic due to advancements in multidisciplinary approaches and innovative therapies. Traditional treatments, such as surgery, radiation therapy, and chemotherapy, continue to form the cornerstone of pediatric brain cancer management. However, emerging modalities like targeted therapy, immunotherapy, and proton beam therapy are offering new hope. Low-grade tumors often benefit from surgical intervention alone, while high-grade tumors necessitate a combination of treatments to ensure efficacy and minimize recurrence. The past decade has seen significant strides in chemical biology, providing deeper insights into the molecular mechanisms of childhood brain cancers. This progress is paving the way for more effective and personalized therapeutic options, suggesting a promising future in combating pediatric brain tumors.

The recent approvals of OJEMDA (tovorafenib) in 2024 and the combination of TAFINLAR (dabrafenib) with MEKINIST (trametinib) in 2023 have created improved opportunities for pediatric patients with brain cancer. Key players, such as Chimerix, Laminar Pharmaceuticals, and others, are evaluating their lead candidates in different stages of clinical development, respectively. They aim to investigate their products for the treatment of Pediatric brain cancer.

• In 2024, the United States accounts for the largest market size of Pediatric brain cancer, in comparison to EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Among the EU4 and the UK, the United Kingdom had the highest market size, while France had the smallest market size for Pediatric brain cancer in 2024.

Pediatric brain cancer drug uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2025–2034, which depends on the competitive landscape, safety, and efficacy data, along with the order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.

Further detailed analysis of emerging therapies' drug uptake in the report…

Pediatric brain cancer Activities

The report provides insights into different therapeutic candidates in the Phase III and Phase II stages. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Pediatric brain cancer therapies.

KOL Views

To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Medical/scientific writers, Professors, and Others.

DelveInsight’s analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as UT Health San Antonio MD Anderson Cancer Center, Virginia Mason Medical Center, University of Bari, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or Pediatric brain cancer market trends.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial’s primary and secondary outcome measures are evaluated; for instance, in event-free survival, one of the most important primary outcome measures is event-free survival and overall survival.

Further, the therapies’ safety is evaluated, wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

Reimbursement may be referred to as the negotiation of a price between a manufacturer and a payer that allows the manufacturer access to the market. It is provided to reduce the high costs and make the essential drugs affordable. Health technology assessment (HTA) plays an important role in reimbursement decision-making and recommending the use of a drug. These recommendations vary widely throughout the seven major markets, even for the same drug. In the US healthcare system, both Public and Private health insurance coverage are included. Also, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs, including Medicare, Medicaid, Health Insurance Program (CHIP), and the state and federal health insurance marketplaces, are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs), and third-party organizations that provide services and educational programs to aid patients are also present.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report

• The report covers a segment of key events, an executive summary, a descriptive overview of Pediatric brain cancer, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of the diagnosis rate, and disease progression along treatment guidelines.
• Additionally, an all-inclusive account of both the current and emerging therapies, along with the elaborate profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
• A detailed review of the Pediatric brain cancer market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM Pediatric brain cancer market.

Pediatric Brain Cancer Report Insights

• Patient Population
• Therapeutic Approaches
• Pediatric brain cancer Pipeline Analysis
• Pediatric brain cancer Market Size and Trends
• Existing and future Market Opportunity

Pediatric Brain Cancer Report Key Strengths

• 10 Years Forecast
• 7MM Coverage
• Pediatric brain cancer Epidemiology Segmentation
• Key Cross Competition
• Conjoint analysis
• Drugs Uptake and Key Market Forecast Assumptions

Pediatric Brain Cancer Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs

• What is the historical and forecasted Pediatric brain cancer patient pool/patient burden in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• Which combination of treatment approaches will have a significant impact on the Pediatric brain cancer drug treatment market size?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• What are the current and emerging options for the treatment of Pediatric brain cancer?
• How many companies are developing therapies for the treatment of Pediatric brain cancer?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies?

Reasons to buy

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Pediatric brain cancer market.
• Insights on patient burden/disease Incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• Highlights of access and reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

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