Rare Disease – Thematic Intelligence

Reasons to buy the ‘Rare Disease’ thematic intelligence report:

• The report consolidates GlobalData’s latest thinking and forecasts of how the healthcare, macroeconomic, technology, and regulatory trends will impact the rare disease space.
• It provides insights into the leading players and future disruptors across the value chain. The report also gives insights into key drugs and markets from GlobalData’s Pharma Intelligence Center.
• This report covers strategic planners, competitive intelligence professionals, and key stakeholders in the pharmaceutical industry with a clear view of the opportunities and risks over the foreseeable future for rare diseases.

How is our ‘Rare Disease’ report unique from other reports in the market?

• Identify the key trends accelerating or hindering the rare disease space.
• See market forecasts for different therapies within rare diseases up to 2027.
• Understand recent and influential developments in rare diseases.
• Get access to reviews of leaders and disruptors across the rare disease value chain.

We recommend this valuable source of information to anyone involved in:

• ​Pharmaceutical Industries (Big Pharma, Small Biotech, Start-ups, etc.),
• Pharmaceutical Industry Suppliers (e.g., Consulting Companies, CMOs, CDMOs, CROs, Technology Vendors)
• Consulting and Professional Services (e.g., Investment Companies, Investment Banks, Equity Companies, etc.)
• Pharma Manufacturers (Innovative, Biotech, Generics, Biosimilars, Rare/Orphan Disease) / Distributors
• Financial Institutions (Investors in Pharma/Biotech, Pricing Consultancies)/Parallel Trade Organisations
• Healthcare Organisations (Payers, HTA Bodies, Reimbursement Groups, Government Healthcare Organizations)
• Clinical Research Organisations (CROs)

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Rare Disease Thematic Report Overview

In recent years, rare diseases have become a global health priority, leading to increased research efforts, policy initiatives, and collaborative partnerships aimed at accelerating progress in this field. Key drivers of innovation in the rare disease space include advances in genomics, molecular diagnostics, precision medicine, and therapeutic development, which hold promise for personalized approaches to diagnosis and treatment.

The ‘Rare Disease’ thematic intelligence report provides a focused chapter on the key healthcare, technology, macroeconomic, and regulatory trends impacting rare diseases. The report also offers a review of how these trends will accelerate or hinder the rare disease market.

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Rare Disease – Trends

The main trends shaping the rare disease theme over the next 12 to 24 months are classified into healthcare, technology, macroeconomic, and regulatory.

Healthcare Trends: The main healthcare trends shaping the rare disease theme over the next 12 to 24 months are the role of genomics in drug discovery, the surge in cell & gene therapy, and the use of telehealth among others.

Technology Trends: The main technology trends shaping the rare disease theme over the next 12 to 24 months are the rising use of AI & big data, cloud computing, and 3D printing; the evolution of the wearable technology market; and mobile health among others.

Macroeconomic Trends: The main macroeconomic trends shaping the rare disease theme over the next 12 to 24 months are social inclusion & diversity, COVID-19, and drug pricing and reimbursement.

Regulatory Trends: The main regulatory trends shaping the rare disease theme over the next 12 to 24 months are orphan drug designation, reimbursement landscape, and data privacy concerns among others.

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Rare Disease - Value Chain Analysis

GlobalData’s rare disease value chain is split into drug discovery and development, clinical trials, manufacturing, and patient access.

Drug discovery & development: Drug discovery and development play a pivotal role in the intricate value chain for rare diseases, addressing the unique challenges associated with these often overlooked and medically underserved conditions. The journey begins with rigorous research endeavors aimed at unraveling the complexities of rare diseases, identifying potential therapeutic targets, and conducting preclinical studies to validate their viability.

The Rare Disease Value Chain

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Rare Disease – Competitive Landscape

A few of the leading public companies associated with rare disease themes are:

• Vertex
• Takeda
• Roche

A few of the leading private companies associated with rare disease themes are:

• Curebase
• Raremark
• HiberCell

Leading Companies Making Their Mark Within the Rare Disease Theme

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Sector Score Card Analysis

The scorecard approach is used to predict tomorrow’s leading companies within each sector through a thematic screen, a valuation screen, and a risk screen.  The thematic screen ranks companies based on overall leadership in the 10 themes that matter most to their industry, generating a leading indicator of future performance.

Drug Development Sector Scorecard
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