• 1 イントロダクション

  • 1.1 調査の前提条件と市場の定義

  • 1.2 本調査の範囲

• 2 調査手法

• 3 エグゼクティブサマリー

• 4 市場力学

  • 4.1 市場概要

  • 4.2 市場の促進要因

    • 4.2.1 増加する神経系希少疾患の有病率

    • 4.2.2 希少神経疾患治療薬の有望なパイプラインについて

    • 4.2.3 診断プロセスのスピードアップに向けた世界各国の好意的な政府政策

  • 4.3 市場の抑制要因

    • 4.3.1 高い治療コスト

  • 4.4 ポーターのファイブフォース分析

    • 4.4.1 新規参入の脅威

    • 4.4.2 買い手・消費者の交渉力

    • 4.4.3 サプライヤーの交渉力

    • 4.4.4 代替品の脅威

    • 4.4.5 競合・競争状況の激しさ

• 5 市場セグメンテーション（市場規模別、金額、百万米ドル）

  • 5.1 薬品タイプ別

    • 5.1.1 生物製剤

    • 5.1.2 低分子化合物

  • 5.2 投与形態別

    • 5.2.1 インジェクタブル

    • 5.2.2 オーラル

    • 5.2.3 その他の管理方法

  • 5.3 地域別

    • 5.3.1 北米

      • 5.3.1.1 米国
      • 5.3.1.2 カナダ
      • 5.3.1.3 メキシコ

    • 5.3.2 ヨーロッパ

      • 5.3.2.1 ドイツ
      • 5.3.2.2 英国
      • 5.3.2.3 フランス
      • 5.3.2.4 イタリア
      • 5.3.2.5 スペイン
      • 5.3.2.6 その他ヨーロッパ

    • 5.3.3 アジア太平洋

      • 5.3.3.1 中国
      • 5.3.3.2 日本
      • 5.3.3.3 インド
      • 5.3.3.4 オーストラリア
      • 5.3.3.5 韓国
      • 5.3.3.6 その他アジア太平洋

    • 5.3.4 中東・アフリカ

      • 5.3.4.1 GCC地域
      • 5.3.4.2 南アフリカ
      • 5.3.4.3 その他中東・アフリカ

    • 5.3.5 南米

      • 5.3.5.1 ブラジル
      • 5.3.5.2 アルゼンチン
      • 5.3.5.3 その他南米

• 6 競合情勢

  • 6.1 企業プロファイル

    • 6.1.1 US WorldMeds LLC (Solstice Neurosciences LLC)

    • 6.1.2 CSL Ltd

    • 6.1.3 Merz Pharma GmbH & Co. KGaA

    • 6.1.4 Aquestive Therapeutics Inc

    • 6.1.5 Kedrion Biopharma Inc

    • 6.1.6 Bayer AG

    • 6.1.7 Pfizer Inc

    • 6.1.8 Novartis AG

    • 6.1.9 Merck & Co. Inc. (EMD Serono Inc.)

• 7 市場機会と今後の動向

The rare neurological disease treatment market is expected to register a CAGR of 8.6% during the forecast period.

The COVID-19 pandemic represents an international health crisis that is challenging to all governments. Health practitioners in different fields must guide people and governments to achieve safe health practices. In June 2020, the World Health Organization issued the results of a survey conducted by 155 countries during the three weeks of May 2020, which showed that the prevention and treatment services of various chronic diseases, including various rare neurological diseases such as amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy, and Huntington's disease, were severely disrupted, becoming a significant concern as people living with chronic diseases are at higher risk of severe COVID-19 related illness and death. As per the study titled "Rare neurological disorder documented following COVID-19 vaccination", published in June 2021, the frequency of Guillain-Barre syndrome in India and England is estimated to be up to 10 times greater than expected after taking the COVID-19 vaccine. Thus, the COVID-19 pandemic has significantly impacted the market’s growth.

The rising prevalence of rare neurological diseases, increasing R&D procedures for treating neurological diseases, and the growing government policies worldwide to create awareness for these diseases may drive the rare neurological diseases treatment market.

There are about 5,000–8,000 rare diseases. Some neurological disorders include reflex sympathetic dystrophy syndrome, Battaglia-Neri syndrome, Creutzfeldt Jakob Disease, agnosia, Aicardi syndrome, and Aicardi-Goutieres syndrome. As per the report titled "Prevalence and incidence of rare diseases: Bibliographic data", published in January 2022, the prevalence of chronic inflammatory demyelinating polyneuropathy is 3.7 in Europe. However, the diagnosis of rare neurological diseases involves several barricades, such as a lack of awareness about rare neurological diseases within the population. Rare diseases are creating huge healthcare and economic burden worldwide.

The National Organization for Rare Disorders (NORD) Report 2019 estimates that around 7,000 known rare diseases lead to remarkable morbidity and mortality in 25 million affected Americans. In addition, about one-third of these are thought to include neurological components and symptoms. However, the high cost of treatment is expected to hinder the growth of the rare neurological disease treatments market.

Key Market Trends

The Small Molecules Segment is Expected to Hold a Major Share in the Rare Neurological Disease Treatment Market

The small molecules segment holds a significant share in the rare neurological disease treatment market. It is anticipated to show a similar trend over the forecast period due to the higher cost of biological drugs.

As per the report titled "Peer support for the Huntington's community...by the Huntington's community, Huntington's Community Connect ", published in December 2020, over 1,800 people have Huntington's disease in Australia, and approximately 9,000 individuals are at risk of developing the disease. Similarly, a very high concentration of Huntington's disease has been found in Venezuela, where the prevalence is about 700 per 100,000 population. According to the study titled ‘Biologics versus small molecules: Drug costs and patient access in 2021’ published in March 2021 in ‘Science Direct’, the market for small molecules is rapidly expanding. Small molecules are increasingly being used to treat chronic disorders and continue to dominate the pharmaceutical business while being more impacted by policy implementation than biologics, which are still relatively new in healthcare. Therefore, the rising adoption of small molecules in treating chronic diseases like rare neurological disorders is expected to drive the small molecule drug discovery market.

Moreover, the segment’s growth is contributed largely by collaboration among different market players for the research and discovery of small molecules for neurological diseases. For instance, in July 2021, Servier and Nymirum entered a strategic collaboration to identify and develop RNA-modulatory drugs to treat neurological diseases. Under the collaboration agreement, Nymirum will leverage its proprietary DART Platform (Dynamic Atomic-Resolution RNA Targeting Platform) to discover novel small molecule therapeutics for multiple neurological targets.

Increasing incidences of rare neurological diseases worldwide, increasing focus on fast-track approvals, technological advancements, and growing awareness regarding early diagnosis of rare neurological diseases are the key factors driving the small molecules segment.

North America is Expected to Hold a Significant Market Share During the Forecast Period

North America is expected to hold a major share in the rare neurological disease treatment market due to the availability of reimbursement, growing incidence of rare neurological diseases, and increasing R&D in the region. As per the data published by the Center for Rare Neurological Disease Research in January 2021, rare neurological diseases affect approximately 200,000 individuals in the United States.

Additionally, the launch of new treatments for treating these diseases, such as Huntington's disease, may drive the market. For instance, in December 2021, Novartis received approval from the US Food and Drug Administration (FDA) for Fast Track designation for branaplam (LMI070) for the treatment of Huntington’s disease (HD). Moreover, the last day of February is celebrated as Rare Disease Day every year to raise awareness among policymakers and the public about rare diseases and their impact on patients’ lives. In February 2022, this day was celebrated to raise awareness about rare diseases, the people affected, and the collaborations by the National Institute of Health (NIH) that address scientific challenges and advance research for new treatments. Thus, these programs may help raise awareness regarding rare neurological diseases in the country, thus driving the demand for their treatment in the country.

Furthermore, increasing healthcare spending and the presence of well-established healthcare infrastructure are fueling the growth of the overall regional market to a large extent.

Competitive Landscape

The rare neurological disease treatment market is consolidated. The market consists of a few significant players that dominate the market, including CSL Ltd, Kedrion Biopharma Inc., US WorldMeds LLC (Solstice Neurosciences LLC), Merz Pharma GmbH & Co. KGaA, Aquestive Therapeutics Inc., Bayer AG, Pfizer Inc., Novartis AG, Merck & Co. Inc. (EMD Serono Inc.), and Jazz Pharmaceuticals PLC.

Additional Benefits:

• The market estimate (ME) sheet in Excel format
• 3 months of analyst support

• 1 INTRODUCTION

  • 1.1 Study Assumptions and Market Definition

  • 1.2 Scope of the Study

• 2 RESEARCH METHODOLOGY

• 3 EXECUTIVE SUMMARY

• 4 MARKET DYNAMICS

  • 4.1 Market Overview

  • 4.2 Market Drivers

    • 4.2.1 Increasing Prevalence of Rare Neurological Diseases

    • 4.2.2 Promising Pipeline Drugs for Treatment of Rare Neurological Diseases

    • 4.2.3 Favorable Government Policies Worldwide for Speeding up Diagnostic Processes

  • 4.3 Market Restraints

    • 4.3.1 High Cost of Treatment

  • 4.4 Porter's Five Forces Analysis

    • 4.4.1 Threat of New Entrants

    • 4.4.2 Bargaining Power of Buyers/Consumers

    • 4.4.3 Bargaining Power of Suppliers

    • 4.4.4 Threat of Substitute Products

    • 4.4.5 Intensity of Competitive Rivalry

• 5 MARKET SEGMENTATION (Market Size by Value - USD million)

  • 5.1 By Drug Type

    • 5.1.1 Biologics

    • 5.1.2 Small Molecules

  • 5.2 By Mode of Administration

    • 5.2.1 Injectables

    • 5.2.2 Oral

    • 5.2.3 Other Modes of Administration

  • 5.3 By Geography

    • 5.3.1 North America

      • 5.3.1.1 United States
      • 5.3.1.2 Canada
      • 5.3.1.3 Mexico

    • 5.3.2 Europe

      • 5.3.2.1 Germany
      • 5.3.2.2 United Kingdom
      • 5.3.2.3 France
      • 5.3.2.4 Italy
      • 5.3.2.5 Spain
      • 5.3.2.6 Rest of Europe

    • 5.3.3 Asia-Pacific

      • 5.3.3.1 China
      • 5.3.3.2 Japan
      • 5.3.3.3 India
      • 5.3.3.4 Australia
      • 5.3.3.5 South Korea
      • 5.3.3.6 Rest of Asia-Pacific

    • 5.3.4 Middle-East and Africa

      • 5.3.4.1 GCC
      • 5.3.4.2 South Africa
      • 5.3.4.3 Rest of Middle-East and Africa

    • 5.3.5 South America

      • 5.3.5.1 Brazil
      • 5.3.5.2 Argentina
      • 5.3.5.3 Rest of South America

• 6 COMPETITIVE LANDSCAPE

  • 6.1 Company Profiles

    • 6.1.1 US WorldMeds LLC (Solstice Neurosciences LLC)

    • 6.1.2 CSL Ltd

    • 6.1.3 Merz Pharma GmbH & Co. KGaA

    • 6.1.4 Aquestive Therapeutics Inc

    • 6.1.5 Kedrion Biopharma Inc

    • 6.1.6 Bayer AG

    • 6.1.7 Pfizer Inc

    • 6.1.8 Novartis AG

    • 6.1.9 Merck & Co. Inc. (EMD Serono Inc.)

• 7 MARKET OPPORTUNITIES AND FUTURE TRENDS