Neurofibromatosis Market
Number of pages: 100 | Report Format: PDF | Published date: June 20, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
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Report Attribute |
Details |
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CAGR |
11.30% |
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Base Year for Estimation |
2022 |
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Forecast Period |
2023 to 2031 |
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Historical Year |
2021 |
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Segments Covered |
Type, Drug Class, Distribution Channel, and Region |
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Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global neurofibromatosis market is expected to register a revenue CAGR of 11.30% during the predicted timeframe from 2023 to 2031.
Neurofibromatosis Market Fundamentals
Neurofibromatosis (NF) is a set of hereditary illnesses that cause tumors in the nerve system to form. Neurofibromas are tumors that can grow on nerves anywhere in the body, including the skin, brain, spinal cord, and peripheral nerves. Mutations in certain genes that affect cell development and division cause neurofibromatosis. Neurofibromatosis is classified into three categories: type 1 (NF1), type 2 (NF2), and schwannomatosis. Some people may have modest symptoms, while others may have more serious consequences and limitations. Treating neurofibromatosis includes pharmacological therapy such as anti-inflammatory, antineoplastic agents, and antihistamines.
Neurofibromatosis Market Dynamics
Growing prevalence of neurofibromatosis is one of the major drivers in global market growth. According to the report by ‘Neurofibromatosis Clinics Association’, it was noted that NF1 occurs one in every 3,000 children born. Similarly, NF2 is a rarer type, occurring in 1:25,000 people worldwide and schwannomatosis occurs in 1:40,000 people. Advances in genetic research and molecular diagnostics may lead to the development of targeted therapies and personalized medicine approaches. Additionally, pharmaceutical companies and research institutions are actively exploring potential treatments for neurofibromatosis, including drug candidates targeting specific pathways involved in tumor growth. There is a growing interest in identifying novel therapies that can effectively treat the symptoms and complications associated with neurofibromatosis. Moreover, ongoing clinical trials play a crucial role in evaluating the safety and efficacy of potential treatments for neurofibromatosis. For instance, in January 2023, the Children’s Tumor Foundation (CTF) entered a new phase in its strategic NF research model by announcing a significant impact investment in a Phase 2b clinical trial at NFlection Therapeutics. The trial involves NFX-179, a MEK inhibitor which shows a promising result in shrinking cutaneous neurofibromas in NF1 patients, is a topical treatment which has successfully passed a Phase 1/2a trial. These trials contribute to the development of new therapies and provide opportunities for patients to access experimental treatments. Increased awareness about neurofibromatosis has led to improved diagnosis rates and better access to healthcare resources. Furthermore, advocacy groups, such as the Children's Tumor Foundation, have been instrumental in raising awareness, promoting research, and supporting affected individuals and their families. Collaboration between academic institutions, biotechnology companies, and pharmaceutical companies is vital for advancing research and development efforts in the field of neurofibromatosis. Partnerships can help accelerate the translation of scientific discoveries into therapeutic interventions. For instance, a breakthrough medication developed by AstraZeneca and Merck & Co. became the first medicine licensed in the European Union for NF1. The kinase inhibitor Koselugo (selumetinib) was licensed in the United States in April 2020.
The integration of patient perspectives and experiences in research and drug development processes is gaining importance. Including patients in clinical trial design, identifying patient-relevant outcomes, and incorporating patient-reported outcomes can help ensure that treatments address the most significant needs and challenges faced by individuals with neurofibromatosis.
Currently, there is no known cure for neurofibromatosis. Treatment options primarily focus on managing symptoms and complications. The absence of a curative treatment presents a significant challenge for patients and healthcare providers. Neurofibromatosis is a highly variable condition, with symptoms and manifestations differing widely among individuals. Such heterogeneity makes it challenging to develop standardized treatment approaches that effectively address the diverse needs of patients. Neurofibromatosis tumors can grow and cause complications over time. The unpredictable nature of tumor growth and the potential for malignant transformation pose challenges in managing the condition and achieving long-term tumor control. The availability of FDA-approved treatments specifically targeting neurofibromatosis is currently absent. While some medications may help manage specific symptoms or complications, there is a need for more targeted therapies that can provide better outcomes and improve quality of life for patients. Neurofibromatosis is a rare condition, and awareness among healthcare professionals, patients, and other care givers may be limited. This can lead to delayed or missed diagnoses, impacting the timely initiation of appropriate management strategies.
The cost of treating neurofibromatosis and its associated complications can be substantial. The need for regular medical surveillance, imaging studies, and potential surgical interventions can place a financial burden on patients and their families, limiting access to optimal care. Neurofibromatosis can have significant psychological and social impacts on individuals, including stigma, body image concerns, anxiety, and depression. Addressing the psychosocial aspects of the condition is essential but often overlooked in treatment strategies. Despite advancements in understanding neurofibromatosis, research funding for the condition may still be limited compared to more prevalent diseases. Insufficient funding can slow down the pace of research, hampering the development of new therapies and treatment approaches.
Neurofibromatosis Market Ecosystem
The global neurofibromatosis market has been analyzed from the following perspectives: by type, drug class, distribution channel, and region.
Neurofibromatosis Market by Type
Based on the type, the global neurofibromatosis market is segmented into NF1, NF2, and schwannomatosis.
NF1 accounts for more than half revenue share of the global market, due to the high prevalence compared to other types. NF1 is also known as Recklinghausen disease, von Recklinghausen disease, von Recklinghausen’s phakomatosis, von Recklinghausen’s neurofibromatosis, neurofibroma (multiple), neurofibromatosis-pheochromocytoma-duodenal carcinoid syndrome, or peripheral neurofibromatosis. NF1 typically presents with a wide range of symptoms and manifestations, including café-au-lait spots (pigmented skin lesions), neurofibromas (benign tumors on or under the skin), and other potential complications affecting various organ systems. The diverse clinical presentation of NF1 may contribute to increased awareness and diagnosis rates. Due to its higher prevalence and well-characterized genetic basis, NF1 has been the focus of extensive research efforts.
According to an article by ‘Cancer.Net’, it was estimated that 1 in 3,000 people suffers from NF1 mutation. And approximately 50% of people affected by NF1 do not have any family history of the condition. Scientists and clinicians have dedicated significant resources to studying the molecular mechanisms underlying NF1, identifying potential therapeutic targets, and developing treatment strategies. The extensive research conducted in NF1 has resulted in a broader range of clinical trials and treatment options for individuals with NF1 compared to other forms of neurofibromatosis. This includes investigational therapies targeting specific molecular pathways associated with NF1, such as MEK inhibitors. The availability of clinical trials and treatment options further highlights the dominance of NF1 in the neurofibromatosis market.
Neurofibromatosis Market by Drug Class
Based on the drug class, the global neurofibromatosis market is segmented into antihistamines, antineoplastic agents, and anti-inflammatory drugs.
Antineoplastic agents (anticancer drugs) play an important role in the treatment of neurofibromatosis. Antineoplastic agents are commonly used in the management of tumors, including those associated with neurofibromatosis by slowing down the tumor growth, reducing tumor burden, or control symptoms caused by tumor compression. Some antineoplastic agents or targeted therapies used in the treatment of other types of cancers are being explored in clinical trials for their potential effectiveness against neurofibromatosis-associated tumors. These investigational therapies aim to inhibit specific signaling pathways involved in tumor growth and may hold promise for the future treatment of neurofibromatosis. Furthermore, these antineoplastic agents can also be used in combination with other treatment modalities for neurofibromatosis management. For example, chemotherapy drugs may be used alongside surgical interventions or radiation therapy to enhance treatment outcomes or control tumor growth in individuals with neurofibromatosis. At present selumetinib and carboplatin are the most commonly prescribed antineoplastic drugs used for treatment of neurofibromatosis. Among the different agents, selumetinib is FDA approved for treating NF1.
Neurofibromatosis Market by Distribution Channel
Based on the distribution channel, the global neurofibromatosis market is segmented into hospital pharmacies, retail pharmacies, and online pharmacies.
Neurofibromatosis treatment often requires specialized medications, including antineoplastic agents, pain management drugs, and medications to manage associated symptoms and complications. Hospital pharmacies are well-equipped to handle the storage, preparation, and dispensing of these specialized medications, ensuring their availability for patients, thus, dominating the global market. Hospitals occupy over 40% of the global market share in 2022.Hospital pharmacies play a crucial role in supporting this multidisciplinary care by supplying medications to different departments and healthcare providers involved in the treatment of neurofibromatosis patients. Hospital pharmacies cater to both inpatient and outpatient settings. In the case of neurofibromatosis, patients may require hospitalization for surgical interventions, pain management, or other specialized care. Additionally, hospital pharmacies may also provide outpatient services to neurofibromatosis patients, ensuring the continuity of medication supply beyond the hospital stay. Hospital pharmacies are well-equipped to provide compounding services, preparing medications in specific dosages, formulations, or combinations as prescribed by healthcare professionals. Hospital pharmacies often collaborate with research institutions and participate in clinical trials evaluating potential treatments for neurofibromatosis.
Neurofibromatosis Market by Region
Based on the region, the global neurofibromatosis market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa.
Neurofibromatosis has a significant prevalence in North America, thus, leading the global market with a sizeable revenue share. The larger patient population creates a substantial market for neurofibromatosis-related products, services, and research in the region. North America has well-developed healthcare infrastructure, including hospitals, specialized clinics, research institutions, and pharmaceutical companies. This infrastructure facilitates diagnosis, treatment, and research activities related to neurofibromatosis. The availability of advanced medical facilities and expertise supports the growth of the neurofibromatosis market in the region. North America is at the forefront of neurofibromatosis research and development. Numerous academic institutions, research centers, and biotechnology companies in the region are actively engaged in studying the underlying mechanisms of neurofibromatosis, identifying potential therapeutic targets, and developing innovative treatment approaches.
The presence of cutting-edge research contributes to the dominance of North America in the global neurofibromatosis market. The regulatory environment in North America, particularly in the United States, provides a framework for the approval of drugs, medical devices, and treatments related to neurofibromatosis. The rigorous evaluation processes conducted by regulatory authorities, such as the U.S. Food and Drug Administration (FDA), ensure the safety and efficacy of neurofibromatosis-related products, fostering confidence in the market. North America has strong patient advocacy organizations, such as the Children's Tumor Foundation, that work tirelessly to raise awareness, provide support to individuals with neurofibromatosis, and drive research initiatives. These organizations play a crucial role in increasing public awareness, advocating for improved treatments and healthcare access, and mobilizing resources for neurofibromatosis research. The insurance coverage and reimbursement systems in North America, particularly in the United States, are relatively robust. This can facilitate patient access to neurofibromatosis-related healthcare services, including diagnosis, treatments, and medications. Adequate insurance coverage and reimbursement policies contribute to the growth of the neurofibromatosis market by reducing financial barriers for patients.
Competitive Landscape
The prominent players operating in the global neurofibromatosis market are:
Neurofibromatosis (NF), a rare genetic illness which results in benign nerve tumors and growths in other parts of the body, including the skin, is a form of phakomatosis or syndrome with neurological and cutaneous signs.
Merck & Co. Inc., Array BioPharma, and Healx are some of the major players in the global neurofibromatosis market.
Lack of awareness is a critical factor obstructing the growth of the global neurofibromatosis market.
The hospital pharmacies segment dominates the global neurofibromatosis market.
Advancing genetic testing and increasing awareness and diagnosis are some of the major factors driving the global neurofibromatosis market.
*Insights on financial performance are subject to the availability of information in the public domain