全球孤儿药市场的成长机会

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个工作天内

价格

简介目录

排他性的下降和竞争的加剧推动了对更有效率的研发和数位解决方案的需求

在这项研究中，Frost & Sullivan 的转型健康团队提供了对全球孤儿药 (OD) 行业的重要见解，确定了成长机会、收益、监管变化以及影响成长的技术趋势。虽然大约 5% 的罕见疾病 (RD) 获得美国FDA 药物核准，但高达 15% 的 RD 至少有一种药物显示出治疗或预防疾病的潜力。对未满足的罕见疾病日益增长的需求是研究和开发的主要动力。治疗 RD 需要新型治疗药物，目前治疗方法选择有限。精准医学和资讯学的最新进展，例如巨量资料分析、多组体学、奈米医学、基因编辑技术和下一代诊断学，正在为开发特异性和个性化的 RD治疗方法创造机会。癌症与 RD 的融合正变得越来越明显。精准肿瘤学和罕见肿瘤的个体化医疗已成为该领域的关键主题，推动了 OD 产业的扩张。

OD的开发采用了多种方法，包括核酸药物、基因治疗、细胞治疗和人工蛋白，重点是个人化医疗的持续研究和开发。一半以上的 OD 药物由中小企业 (SME) 开发，因为它们受益于科学支援、核准后流程以及上市核准申请的较低成本。创业投资的早期投资正在推动这一势头。

OD 政策改善了 RD 治疗的机会，使当地和全球人民受益。这些行动显示了各国对公共卫生和研究的承诺，并支持潜在的伙伴关係和投资。卫生系统需要控制当前和未来的卫生支出。付款人正在认真审查医疗保健成本和患者的就诊水平，以确保最佳平衡。随着政府考虑《孤儿药法案》和《降低通货膨胀法案》(IRA) 变更的影响，健康计画发起人正在考虑下一步。

调查过程/调查方法
预测考虑因素
成长指标
收益预测
收益预测分析
按产品类型分類的收益预测
预测分析 - 小分子
预测分析 -生物製药
预测期间值得关注的关键资产
按技术类型分類的收入比例
按治疗类型预测收入比例
癌症的主要罕见适应症
核准的抗癌药物的特征 - 孤儿药与非孤儿药
癌症以外的主要适应症 - 免疫/肌肉骨骼系统
癌症以外的主要适应症 - 中枢神经系统
非癌症领域主要适应症－血液疾病/代谢疾病
价格趋势
竞争环境
收益占有率
收益占有率分析

成长机会宇宙

成长机会1：核酸疗法
成长机会2：利用生物资讯学进行药物再利用
成长机会3：在中东建立策略伙伴关係，改善诊断与治疗
成长机会4：利用人工智慧改善诊断与治疗

下一步

简介目录

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