全球孤兒藥市場的成長機會

Global Orphan Drug Growth Opportunities

出版日期: 2024年03月01日 | 出版商:

Frost & Sullivan | 英文 78 Pages | 商品交期: 最快1-2個工作天內

價格

簡介目錄

排他性的下降和競爭的加劇推動了對更有效率的研發和數位解決方案的需求

在這項研究中，Frost & Sullivan 的轉型健康團隊提供了對全球孤兒藥 (OD) 行業的重要見解，確定了成長機會、收益、監管變化以及影響成長的技術趨勢。雖然大約 5% 的罕見疾病 (RD) 獲得美國FDA 藥物核准，但高達 15% 的 RD 至少有一種藥物顯示出治療或預防疾病的潛力。對未滿足的罕見疾病日益成長的需求是研究和開發的主要動力。治療 RD 需要新型治療藥物，目前治療方法選擇有限。精準醫學和資訊學的最新進展，例如巨量資料分析、多組體學、奈米醫學、基因編輯技術和下一代診斷學，正在為開發特異性和個性化的 RD治療方法創造機會。癌症與 RD 的融合正變得越來越明顯。精準腫瘤學和罕見腫瘤的個體化醫療已成為該領域的關鍵主題，推動了 OD 產業的擴張。

OD的開發採用了多種方法，包括核酸藥物、基因治療、細胞治療和人工蛋白，重點是個人化醫療的持續研究和開發。一半以上的 OD 藥物由中小企業 (SME) 開發，因為它們受益於科學支援、核准後流程以及上市核准申請的較低成本。創業投資的早期投資正在推動這一勢頭。

OD 政策改善了 RD 治療的機會，使當地和全球人民受益。這些行動顯示了各國對公共衛生和研究的承諾，並支持潛在的夥伴關係和投資。衛生系統需要控制當前和未來的衛生支出。付款人正在認真審查醫療保健成本和患者的就診水平，以確保最佳平衡。隨著政府考慮《孤兒藥法案》和《降低通貨膨脹法案》(IRA) 變更的影響，健康計畫發起人正在考慮下一步。

調查過程/調查方法
預測考慮因素
成長指標
收益預測
收益預測分析
按產品類型分類的收益預測
預測分析 - 小分子
預測分析 -生物製藥
預測期間值得關注的關鍵資產
按技術類型分類的收入比例
按治療類型預測收入比例
癌症的主要罕見適應症
核准的抗癌藥物的特徵 - 孤兒藥與非孤兒藥
癌症以外的主要適應症 - 免疫/肌肉骨骼系統
癌症以外的主要適應症 - 中樞神經系統
非癌症領域主要適應症－血液疾病/代謝疾病
價格趨勢
競爭環境
收益佔有率
收益佔有率分析

成長機會宇宙

成長機會1：核酸療法
成長機會2：利用生物資訊學進行藥物再利用
成長機會3：在中東建立策略夥伴關係，改善診斷與治療
成長機會4：利用人工智慧改善診斷與治療

下一步

簡介目錄

Product Code: PFA6-52

Reduced Exclusivity and Increased Competition will Drive Demand for More Efficient R&D and Digital Solutions

In this study, Frost & Sullivan's Transformational Health team provides critical insights into the global orphan drug (OD) industry and highlights growth opportunities, revenue, regulatory changes, and technology trends influencing growth. Approximately 5% of rare diseases (RDs) have received US FDA approval for a drug, while up to 15% of RDs have at least 1 drug that exhibits potential in terms of disease treatment or prevention. The growing number of unaddressed RD needs is a major catalyst for R&D. There is a need for novel medicine to treat RDs that currently have limited therapeutic choices. Recent advancements in precision medicine and informatics, such as big data analytics, multi-omics, nanomedicine, gene-editing techniques, and next-generation diagnostics, have created opportunities to develop specific and individualized therapies for RDs. The convergence of cancer and RDs is becoming evident. Precision oncology and tailored medicine for rare tumors are emerging as prominent themes in the discipline, facilitating the OD industry's expansion.

Various techniques are used to develop ODs, mainly due to the rise of R&D in personalized medicine, including nucleic acid drugs, gene therapies, cell therapies, and engineered proteins. Small and medium-sized enterprises (SMEs) develop more than half of ODD medication because they benefit from lower prices for scientific assistance, pre- and post-authorization processes, and marketing authorization applications. The early investments obtained from venture capitalists buoy the momentum.

RD treatment access has improved with OD policies, benefiting local and global populations. These actions showcase countries' commitment to public health and research, boosting probable partnerships and investments. Healthcare systems face increasing requirements to contain present and future healthcare spending. Payers are diligently scrutinizing medicine costs and patient access levels to guarantee optimal equilibrium. Healthcare plan sponsors are considering their next move as governments contemplate Orphan Drug Act changes and the impact of the Inflation Reduction Act (IRA).

Key Issues Addressed:

How much revenue will the global OD industry generate in 2028?
Which segments will influence revenue growth rate?
What challenges do patients face in their RD journeys?
Which business models, technologies, and trends must stakeholders and participants watch during the forecast period?
What are the major OD growth opportunities?

Strategic Imperatives

Why is it Increasingly Difficult to Grow?
The Strategic Imperative 8™
The Impact of the Top 3 Strategic Imperatives on the Orphan Drug Industry
Growth Opportunities Fuel the Growth Pipeline Engine™

Growth Environment

Scope of Analysis
Segmentation
Rare Disease Patient Journey Challenges
Critical Future Steps in the Development of Novel Drugs for Rare Diseases
Growth Opportunities
Vendor Landscape
Growth Drivers
Growth Restraints

Growth Environment-Pipeline Analysis

Pipeline Analysis and Summary by Therapeutic Segment
Pipeline Summary by Technology Type

Regional Analysis

Incentives to Develop Treatments for Rare Diseases-A Regional Overview
Regional Synopsis
Regional Attractiveness
Recent Regulatory Policies and Initiatives by Region
Impact of the IRA on the US Orphan Drugs Industry
Special US FDA Designations for Drug Development-Orphan, Fast Track, Accelerated Approval, Priority Review, and Breakthrough Therapy

Business Models and Investment Overview

OD Business Models
M&A Assessment
Venture Financing Assessment

Digital Technology Implementation

Market Trends Advancing Sustainability in the Pharma Value Chain
Digital Novel Solution Applications
Industry Use Case-UCB's Drug for Myasthenia Gravis

Growth Opportunity Analysis

Research Process and Methodology
Forecast Considerations
Growth Metrics
Revenue Forecast
Revenue Forecast Analysis
Revenue Forecast by Product Type
Forecast Analysis-Small Molecules
Forecast Analysis-Biologics
Key Assets to Watch During the Forecast Period
Percent Revenue Breakdown by Technology Type
Percent Revenue Forecast by Therapy Type
Key Oncology Rare Indications
Approved Oncology Drugs' Characteristics-Orphans versus Non-orphans
Key Non-oncology Indications-Immunology/Musculoskeletal System
Key Non-oncology Indications-CNS
Key Non-oncology Indications-Blood Disorders and Cardio-metabolic Diseases
Pricing Trends
Competitive Environment
Revenue Share
Revenue Share Analysis

Growth Opportunity Universe

Growth Opportunity 1: Nucleic Acid-based Therapeutics
Growth Opportunity 2: Leveraging Bioinformatics for Drug Repurposing
Growth Opportunity 3: Strategic Partnerships in the Middle East to Improve Diagnosis and Treatment
Growth Opportunity 4: Utilizing AI to Improve Diagnosis and Treatment