Global Orphan Drug Sector Poised for Growth Amidst Unmet Rare Disease Needs

Dublin, April 05, 2024 (GLOBE NEWSWIRE) -- The "Global Orphan Drug Growth Opportunities" report has been added to's offering.

This study 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?

Key Growth Opportunities:

  • 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

Company Coverage: FDA, UCB

Key Topics Covered:

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 Analysis

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