The pediatric brain tumors market is estimated to be valued at USD 1.55 Bn in 2024 and is expected to reach USD 2.47 Bn by 2031, growing at a compound annual growth rate (CAGR) of 6.9% from 2024 to 2031. Growing prevalence of pediatric brain cancers due to genetic risk factors and environmental exposures is driving the market. Rising healthcare spending for pediatric cancer treatment and development of advanced treatment options are also fueling the pediatric brain tumors market.

Market Driver - Increasing Incidence of Pediatric Brain Tumors

One of the key drivers for the pediatric brain tumors market has been the rising incidence of various forms of pediatric brain and central nervous system (CNS) tumors. While CNS tumors are relatively rare in children, accounting for less than 20% of childhood cancers, recent studies indicate their incidence rates have been increasing gradually. In the United States alone, over 4,500 new cases of pediatric brain and CNS tumors are diagnosed each year according to estimates.

Brain tumors are especially challenging to treat in children due to the complex neurodevelopment occurring at younger ages. While survival rates for common pediatric brain tumors like medulloblastoma, astrocytoma and ependymoma have improved over the decades, these cancers still present formidable challenges.

Even after successful treatment such as surgery and chemotherapy, long term survivors are often afflicted with debilitating side effects and functional disabilities. This puts immense strain on the children, their families as well as healthcare systems and drives the need for newer and more effective therapies.

Various foundations and non-profits focused on pediatric cancer research have also raised awareness about the unmet needs in this area through fund raising campaigns and patient support programs.

Market Driver - Advancements in Treatment Modalities like Immunotherapies and Targeted Therapies

Continuous progress is being made in the management of pediatric brain cancers owing to ongoing research efforts. One of the key drivers augmenting the market potential is the advancements occurring in treatment modalities such as immunotherapy and targeted therapies.

Immune checkpoint inhibitors which help unleash the body's own immune response against tumor cells have shown early promise in clinical trials for certain pediatric brain tumors like gliomas. These agents offer the potential for more targeted treatment with fewer side effects compared to conventional chemotherapy regimens.

Several targeted therapies targeting tumor-specific gene mutations and growth factor receptors are in the pipeline. Some have already been approved or received breakthrough designation for pediatric use from the FDA. Their relative precision in attacking vulnerable molecular pathways holds out hope for improved efficacy while also sparing healthy tissues from collateral damage. They can also be given orally instead of through infusions or injections as needed.

The field of precision medicine and personalized treatment based on patients' unique tumor profiles has given a further boost to pharmaceutical R&D in this domain.

Market Challenge - High treatment costs limiting access

One of the major challenges faced by the pediatric brain tumors market is the high cost of treatment which limits access to quality healthcare for many young patients. Treating pediatric brain tumors is a long and complex process that involves various treatment modalities like surgery, chemotherapy, radiation therapy and immunotherapy. All of these advanced treatment options come at a very high price which not all families can afford.

According to various studies, the average cost of treatment for pediatric brain tumors in the US ranges from $100,000 to $500,000 depending on the type and stage of tumor. This disproportionately high financial burden means that many families, especially those who are uninsured or underinsured, find it extremely difficult to pay for the entire course of treatment and care of their child.

The high costs often force families to delay or opt out of recommended treatment protocols. This ultimately impacts the health outcomes and prognosis of pediatric brain tumor patients from low-income backgrounds. Addressing issues of affordability and accessibility is imperative to improve survival rates and quality of life for pediatric brain tumor patients across all socioeconomic sections of the society.

Market Opportunity - Rising Focus on Personalized Medicine

One of the key opportunities available in the pediatric brain tumors market is the rising focus on personalized medicine. With advancements in fields like cancer genetics and molecular biology, medical researchers now have a better understanding of the various genetic, molecular and biological factors underlying different types of pediatric brain tumors. This growing knowledge is enabling the development of personalized treatment approaches tailored to the unique profile and needs of each individual young patient.

The paradigm is gradually shifting from one-size-fits-all therapies to personalized regimens based on a child's tumor genetics, biomarker expression and other individual characteristics. Personalized medicine opens up possibilities for developing safer and more effective targeted drugs, immunotherapies and other treatment strategies.

It also helps healthcare providers make more informed decisions by predicting risks, tailoring screening methods as well as choosing the most optimal treatment pathway for each pediatric brain tumor case. The rising focus on personalized treatments holds immense potential to improve health outcomes, minimize side effects and enhance quality of life for pediatric brain tumor patients in the future.

The standard first-line treatment for most pediatric brain tumors is surgery followed by radiation therapy. Chemotherapy may also be used depending on the tumor type, location, and stage. For low-grade astrocytomas, which are the most common type, close observation with serial MRI monitoring is preferred over immediate treatment in early stages. Chemotherapy alone using oral medications such as carbamazepine (Tegretol) may be sufficient.

For high-grade gliomas, the most aggressive tumors, the preferred first-line treatment involves maximal surgical resection followed by chemoradiation using temozolomide (Temodar). This has been shown to improve progression-free and overall survival compared to radiation alone. For relapsed tumors, prescribers favor chemotherapy using intravenous lomustine (CeeNU) or irinotecan (Camptosar). Autologous stem cell transplant is also being increasingly used as consolidative therapy after chemotherapy.

Other factors like the child's age, size and location of the tumor, and surgical risks also influence decisions. Prescribers strive to balance maximizing survival benefits with minimizing long-term side effects like neurocognitive deficits, which are critical considerations for pediatric patients. Overall treatment goals include long-term disease control with preservation of quality of life.

Pediatric brain tumors can be categorized into different stages based on factors like tumor size, location and whether it has spread. The initial stages involve localized tumors that have not spread. The most common treatments here are surgery, radiation therapy and chemotherapy.

For surgeries, the goal is to remove as much of the tumor as safely possible. This provides direct reduction of tumor burden and obtains tissue for accurate diagnosis. Radiation therapy uses high-energy x-rays to destroy cancer cells and works best for those in locations surgery cannot access safely. It is often used along with surgery.

Chemotherapy uses drugs to reach cancer cells throughout the body. Drugs like carboplatin and vincristine are commonly used either alone or in combinations for low-grade tumors. For more aggressive cancers, combinations of carboplatin, vincristine and lomustine are preferred. These multi-drug regimens have shown higher tumor response rates while preserving organ function in pediatric patients.

If the cancer spreads to other parts of the brain or spinal cord, it is considered advanced stage. Here, high-dose chemotherapy followed by stem cell transplant is the standard of care to deliver high doses of chemotherapy without unacceptable toxicity. Drugs like temozolomide, cisplatin are commonly used in combination with radiotherapy.

Focus on developing targeted therapies: One of the major strategies adopted by companies like Amgen, Novocure, Bristol-Myers Squibb, etc. is focusing on developing targeted therapies that can improve treatment outcomes for pediatric brain tumors. For example, Amgen developed Bevacizumab (Avastin), a VEGF-targeted therapy, for recurrent pediatric gliomas in 2012.

Partnerships and collaborations: Companies actively pursue strategic partnerships and collaborations with academic institutions to gain access to expertise, patient populations and share risks/costs of R&D. For example, Novocure formed collaborations with leading pediatric brain tumor research centers like St. Jude Children's Research Hospital to conduct clinical trials for Optune.

Invest in immunotherapy research: Given promising results in adult brain tumors, players are increasingly investigating immunotherapy approaches for pediatrics. Bristol-Myers Squibb is conducting several Phase 1/2 clinical trials evaluating immune checkpoint inhibitors like nivolumab alone or in combination for recurrent pediatric high-grade gliomas since 2017.

Geographic expansion: Key players partner with local distributors in international markets to drive geographic expansion of approved products. For example, after US FDA approval in 2015, Novocure expanded commercial availability of Optune to major European markets and Japan by 2017-18 through local partnerships. This helped increase patient access and revenues.

Insights, By Treatment: Surgical Advancements Drive Higher Adoption

In terms of treatment, surgery is estimated to account for 41.6% share of the pediatric brain tumors market in 2024, owning to continual improvements in surgical techniques and technologies. For many pediatric brain tumors, surgical resection remains the foundation of treatment. Advances such as intraoperative MRI, advanced neuro-navigation systems, ultrasonic aspiration devices, and laser interstitial thermal therapy have enabled surgeons to remove more of the tumor mass while better preserving critical brain functions. This has translated to improved clinical outcomes for conditions like low-grade gliomas and certain midline tumors.

Beyond maximal resection, surgery also provides an opportunity for tissue sampling needed for accurate diagnosis and tumor grading. This staging information is crucial for determining the most effective combination of subsequent therapies. Additional procedures like ventriculoperitoneal shunt placement, optic nerve sheath fenestration, and cerebrospinal fluid diversion are commonly performed during the same surgery to address related issues.

As surgical skill and supporting technologies progress further, the acceptable extent of resection is being redefined. Certain extremely invasive tumor types that were previously deemed inoperable, such as diffuse intrinsic pontine glioma, are now targets of aggressive surgical trials. Institutional expertise has proliferated through fellowships and specialized training programs focused on pediatric neuro-oncology. Widespread adoption of minimally invasive endoscopic techniques is enhancing recovery. All of these factors consolidate surgery's role at the forefront of initial management.

Insights, By Tumor: Gliomas Dominate Due to Diffuse Nature

In terms of tumor, gliomas are projected to hold 48.7% share of the market in 2024, given their infiltrative growth pattern. They account for approximately 25-30% of all pediatric brain tumors. Low-grade gliomas are particularly common in the pons and thalamus regions, while high-grade forms have poor survival outcomes. Unlike surgically accessible localized masses, gliomas extend multi-focal fingerlings throughout the brain parenchyma. Even with maximal safe resection, residual microscopic disease is nearly impossible to fully remove.

This diffuse morphology supports the critical role of chemotherapy and targeted therapies as adjuvant and neoadjuvant strategies. Temozolomide, a first-line alkylating agent, demonstrates meaningful benefit in extending progression-free survival and is frequently combined with radiation.

Experimental targeted drugs like carmustine wafers, bevacizumab, and selumetinib show promise against specific molecular tumor subclasses. Ongoing research aims to translate adult findings on anti-angiogenic and immune checkpoint inhibitors to Pediatric applications. Additionally, comprehensive genome analysis is elucidating new genomic alterations that may serve as future drug targets. Overall, aggressive utilization of systemic modalities remains necessary due to gliomas' infiltrative growth pattern.

Insights, By Patient Age: Adolescent Care Emphasizes Transition to Adult Providers

In terms of patient age, the pediatric segment contributes the highest share currently. However, focus in Adolescent patients is growing to ensure smooth transition to adult care systems. Brain tumors diagnosed during these formative years present unique developmental, psychosocial and medical issues. Adolescents navigate growing independence, changing relationships, education/career planning, fertility concerns and insurance changes concurrently with complex multi-modality cancer treatment requiring lifelong surveillance.

Successful models emphasize self-management skills, psycho-oncology support, sexuality counseling and coordinated care between pediatric and adult providers. Transition programs aim to transfer clinical information, coordinate sub-specialty follow-up and foster self-advocacy skills. They address challenges in navigating adult facilities that lack dedicated Child Life specialists and teen-friendly spaces.

Compliance with late effects screening and health maintenance is recognized as particularly vulnerable after leaving the nurturing pediatric environment. Overall emphasis on family-centered transition support has shown promise in adulthood outcomes for this vulnerable group. It remains an active area of quality improvement in pediatric brain tumor care.

• Pediatric brain tumors account for nearly 20% of all childhood cancers, with the most common being gliomas. These cancers are more prevalent in developed countries due to higher detection rates but pose a challenge in low-income regions due to a lack of advanced medical infrastructure.
• Pediatric brain tumors represent approximately 20% of all childhood cancers.
• Survival rates vary, with a 5-year survival rate of about 75% for low-grade tumors and significantly lower rates for high-grade tumors.

The major players operating in the pediatric brain tumors market include Novartis, Pfizer, Bayer, Bristol-Myers Squibb, Eli Lilly and Company, Biodexa Pharmaceuticals, Cellectar Biosciences, and Midatech Pharma.

• In June 2024, Pfizer introduced a new immunotherapy drug aimed at treating high-grade gliomas in pediatric patients, expected to enhance survival rates by targeting specific tumor proteins. Pfizer has been actively involved in oncology, including brain cancer research.
• In March 2023, Novartis announced promising results from their Phase II/III TADPOLE trial, which evaluated the efficacy of a targeted therapy using Tafinlar (dabrafenib) and Mekinist (trametinib) for pediatric patients with low-grade glioma (LGG) carrying the BRAF V600E mutation. This combination therapy significantly reduced tumor size, showing an overall response rate of 47%, compared to just 11% in patients treated with chemotherapy.

In July 2020, Bristol-Myers Squibb received FDA approval for an immunotherapy drug designed to treat recurrent medulloblastoma, offering a new line of treatment for patients unresponsive to conventional therapies. Bristol-Myers Squibb has received several FDA approvals for cancer therapies, including approvals for combination treatments involving nivolumab (Opdivo) and ipilimumab (Yervoy), but these were focused on other cancers such as non-small cell lung cancer and lymphoma, rather than medulloblastoma.

• By Treatment
  • Surgery
  • Chemotherapy
  • Radiation Therapy
  • Targeted Therapy
• By Tumor
  • Gliomas
  • Medulloblastomas
  • Embryonal Tumors
  • Pineal Region Tumors
• By Patient Time
  • Pediatric
  • Adolescent