이 보고서는 소아 뇌종양에 대한 상세한 분석을 제공하며, 총 발병 건수, 성별, 종양 등급, 연령, 조직형 등의 각종 구분별 발병 건수에 관한 과거 및 미래의 역학 데이터를 제시하고 있습니다. 또한 진단 과정, 처방 패턴, 의사의 견해, 시장 접근, 치료 옵션, 시장 전망 동향을 미국, EU 4개국(독일, 프랑스, 이탈리아, 스페인), 영국, 일본의 주요 7개국 시장에 걸쳐 2020-2034년 기간으로 분석했습니다. 또한 기존의 치료실태와 미충족 수요(Unmet Needs)를 종합적으로 평가하여 시장의 잠재력과 새로운 사업 기회를 명확히 하고 있습니다.
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.
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.
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.
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 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.
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.
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.
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.
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