Orphan Drugs CDMO Market Size, Share & Trends Analysis Report By Drug Type (Biologics, Non-biologics), By Therapy Type (Oncology, Neuromuscular, Respiratory, Hematology, Others), By End-User (Pharmaceutical companies, Biotechnology companies, CROs, Other End Users), By Region, And By Segment Forecasts, 2025-2034

Report Id: 2150 Pages: 179 Last Updated: 09 May 2025 Format: PDF / PPT / Excel / Power BI
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Global Orphan Drugs CDMO Market Size is valued at USD 13.5 Bn in 2024 and is predicted to reach USD 27.6 Bn by the year 2034 at a 7.5% CAGR during the forecast period for 2025-2034.

Orphan drugs, also known as orphan medicines or orphan therapies, are pharmaceuticals designed to treat rare medical illnesses known as orphan diseases. These disorders often impact a small proportion of the population, making them commercially undesirable for drug companies to research and market in the absence of additional incentives. Several important drivers have influenced the Orphan Drugs CDMO (Contract Development and Manufacturing Organization) market, contributing to its growth and relevance in the pharmaceutical sector.

Orphan Drugs CDMO Market

Orphan medications are in high demand due to increased awareness and diagnosis of rare diseases. As more uncommon diseases are discovered and characterized, the demand for specialized CDMOs to support their development and manufacture grows.However, the COVID-19 pandemic substantially impacted numerous sectors of the pharmaceutical business, particularly the CDMO (Contract Development and Manufacturing Organization) market for orphan drugs. The COVID-19 pandemic had a greater impact on specific pharmaceutical markets due to the deferral of non-life-threatening disease diagnosis and treatment. This was especially true for people suffering from uncommon diseases since various governments concentrated more financial and healthcare resources towards combating the pandemic.

Competitive Landscape

Some Major Key Players In The Orphan Drugs CDMO Market:

  • Novartis AG
  • Hoffmann-La Roche Ltd
  • Celgene,
  • Bristol-Myers Squibb Company
  • Sanofi
  • Bayer Healthcare
  • Doppel
  • Lubrizol Life Science (LLS) Health
  • Others

Market Segmentation:

The Orphan Drugs CDMO Market segmentation consists of drug type, therapy type, and end-user. As per the drug type, the market is segmented as Biologics and Non-biologics. The therapy type segment includes Oncology, Neuromuscular, Respiratory, Hematology, and Others. The end-user segment includes Pharmaceutical companies, Biotechnology companies, CROs, and Other End-users.

Based On Drug Type, The Biologics Segment Is A Major Contributor In The Orphan Drugs CDMO Market. 

The Biologics category is expected to hold a major share of the global Orphan Drugs CDMO Market in 2022 due to the overwhelming prevalence of product offers classed as biologics. According to the U.S. FDA, the organization's Office of Orphan Products Development (OOPD) has developed and marketed approximately 600 medicines and biologic products to treat uncommon illnesses since 1983. 

The Personal Care Segment Witnessed Growth At A Rapid Rate.

The personal care catagory is projected to grow at a rapid rate in the global Orphan Drugs CDMO Market. The Orphan Drugs Contract Development and Manufacture Organisation (CDMO) market assists pharmaceutical companies with orphan medication development and manufacture. The fundamental reason for the segment's dominance is that a considerable number of medications must be administered intravenously in hospitals by qualified healthcare workers.

In The Region, The North America Orphan Drugs CDMO Market Holds A Significant Revenue Share.

The North America Orphan Drugs CDMO Market is projected to record the highest market share in terms of revenue in the near future. The substantial amount spent on orphan pharmaceuticals, the large patient population, and the existence of major market players all contributed to North America's dominance in the creation of sophisticated and cutting-edge technologies. For instance, the Genetic and Rare Diseases (GARD) Information Centre estimates that there are more than 10,000 rare diseases that afflict 30 million Americans, or about 1 in 10 persons, in the U.S. This contributes to North America's dominance in the worldwide market, together with the favourable reimbursement practices in the U.S. Due to a growing patient population in the region and a large uptake of cutting-edge treatments for rare diseases, the European market is expected to grow at a significant CAGR.

Recent Developments:

  • In Aug 2021, Novartis disclosed that Sabatolimab (MBG453) has been awarded as orphan drug by the EC (European Commission) for the management of myelodysplastic syndromes (MDS). The decision was based on clinical evidence demonstrating a significant response rate in patients with high-risk MDS who received concurrent treatment with sabatolimab and hypomethylating agents (HMAs).

Orphan Drugs CDMO Market Report Scope:

Report Attribute Specifications
Market Size Value In 2024 USD 13.5 Bn
Revenue Forecast In 2034 USD 27.6 Bn
Growth Rate CAGR CAGR of 7.5% from 2025 to 2034
Quantitative Units Representation of revenue in US$ Bn,and CAGR from 2025 to 2034
Historic Year 2021 to 2024
Forecast Year 2025-2034
Report Coverage The forecast of revenue, the position of the company, the competitive market structure, growth prospects, and trends
Segments Covered By Drug Type, Therapy Type, End-User
Regional Scope North America; Europe; Asia Pacific; Latin America; Middle East & Africa
Country Scope U.S.; Canada; U.K.; Germany; China; India; Japan; Brazil; Mexico; The UK; France; Italy; Spain; China; Japan; India; South Korea; South East Asia; South Korea; South East Asia
Competitive Landscape Novartis AG, F. Hoffmann-La Roche Ltd, Celgene, Bristol-Myers Squibb Company, Sanofi, Bayer Healthcare, Doppel, Lubrizol Life Science(LLS) Health, and Others
Customization Scope Free customization report with the procurement of the report and modifications to the regional and segment scope. Particular Geographic competitive landscape.
Pricing And Available Payment Methods Explore pricing alternatives that are customized to your particular study requirements.

Segmentation of Orphan Drugs CDMO Market-

Orphan Drugs CDMO Market By Drug Type-

  • Biologics
  • Non-biologics

Orphan Drugs CDMO Market Seg

Orphan Drugs CDMO Market By Therapy Type-

  • Oncology
  • Neuromuscular
  • Respiratory
  • Hematology
  • Others

Orphan Drugs CDMO Market By End-User-

  • Pharmaceutical companies
  • Biotechnology companies
  • CROs
  • Other End Users

Orphan Drugs CDMO Market By Region-

North America-

  • The US
  • Canada

Europe-

  • Germany
  • The UK
  • France
  • Italy
  • Spain
  • Rest of Europe

Asia-Pacific-

  • China
  • Japan
  • India
  • South Korea
  • Southeast Asia
  • Rest of Asia Pacific

Latin America-

  • Brazil
  • Mexico
  • Argentina
  • Rest of Latin America

 Middle East & Africa-

  • GCC Countries
  • South Africa
  • Rest of Middle East and Africa

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Research Design and Approach

This study employed a multi-step, mixed-method research approach that integrates:

  • Secondary research
  • Primary research
  • Data triangulation
  • Hybrid top-down and bottom-up modelling
  • Forecasting and scenario analysis

This approach ensures a balanced and validated understanding of both macro- and micro-level market factors influencing the market.

Secondary Research

Secondary research for this study involved the collection, review, and analysis of publicly available and paid data sources to build the initial fact base, understand historical market behaviour, identify data gaps, and refine the hypotheses for primary research.

Sources Consulted

Secondary data for the market study was gathered from multiple credible sources, including:

  • Government databases, regulatory bodies, and public institutions
  • International organizations (WHO, OECD, IMF, World Bank, etc.)
  • Commercial and paid databases
  • Industry associations, trade publications, and technical journals
  • Company annual reports, investor presentations, press releases, and SEC filings
  • Academic research papers, patents, and scientific literature
  • Previous market research publications and syndicated reports

These sources were used to compile historical data, market volumes/prices, industry trends, technological developments, and competitive insights.

Secondary Research

Primary Research

Primary research was conducted to validate secondary data, understand real-time market dynamics, capture price points and adoption trends, and verify the assumptions used in the market modelling.

Stakeholders Interviewed

Primary interviews for this study involved:

  • Manufacturers and suppliers in the market value chain
  • Distributors, channel partners, and integrators
  • End-users / customers (e.g., hospitals, labs, enterprises, consumers, etc., depending on the market)
  • Industry experts, technology specialists, consultants, and regulatory professionals
  • Senior executives (CEOs, CTOs, VPs, Directors) and product managers

Interview Process

Interviews were conducted via:

  • Structured and semi-structured questionnaires
  • Telephonic and video interactions
  • Email correspondences
  • Expert consultation sessions

Primary insights were incorporated into demand modelling, pricing analysis, technology evaluation, and market share estimation.

Data Processing, Normalization, and Validation

All collected data were processed and normalized to ensure consistency and comparability across regions and time frames.

The data validation process included:

  • Standardization of units (currency conversions, volume units, inflation adjustments)
  • Cross-verification of data points across multiple secondary sources
  • Normalization of inconsistent datasets
  • Identification and resolution of data gaps
  • Outlier detection and removal through algorithmic and manual checks
  • Plausibility and coherence checks across segments and geographies

This ensured that the dataset used for modelling was clean, robust, and reliable.

Market Size Estimation and Data Triangulation

Bottom-Up Approach

The bottom-up approach involved aggregating segment-level data, such as:

  • Company revenues
  • Product-level sales
  • Installed base/usage volumes
  • Adoption and penetration rates
  • Pricing analysis

This method was primarily used when detailed micro-level market data were available.

Bottom Up Approach

Top-Down Approach

The top-down approach used macro-level indicators:

  • Parent market benchmarks
  • Global/regional industry trends
  • Economic indicators (GDP, demographics, spending patterns)
  • Penetration and usage ratios

This approach was used for segments where granular data were limited or inconsistent.

Hybrid Triangulation Approach

To ensure accuracy, a triangulated hybrid model was used. This included:

  • Reconciling top-down and bottom-up estimates
  • Cross-checking revenues, volumes, and pricing assumptions
  • Incorporating expert insights to validate segment splits and adoption rates

This multi-angle validation yielded the final market size.

Forecasting Framework and Scenario Modelling

Market forecasts were developed using a combination of time-series modelling, adoption curve analysis, and driver-based forecasting tools.

Forecasting Methods

  • Time-series modelling
  • S-curve and diffusion models (for emerging technologies)
  • Driver-based forecasting (GDP, disposable income, adoption rates, regulatory changes)
  • Price elasticity models
  • Market maturity and lifecycle-based projections

Scenario Analysis

Given inherent uncertainties, three scenarios were constructed:

  • Base-Case Scenario: Expected trajectory under current conditions
  • Optimistic Scenario: High adoption, favourable regulation, strong economic tailwinds
  • Conservative Scenario: Slow adoption, regulatory delays, economic constraints

Sensitivity testing was conducted on key variables, including pricing, demand elasticity, and regional adoption.

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Frequently Asked Questions

Orphan Drugs CDMO Market Size is valued at USD 13.5 Bn in 2024 and is predicted to reach USD 27.6 Bn by the year 2034

Novartis AG, F. Hoffmann-La Roche Ltd, Celgene, Bristol-Myers Squibb Company, Sanofi, Bayer Healthcare, Doppel, Lubrizol Life Science(LLS) Health, and

Drug Type, Therapy Type and End-User are the key segments of the Orphan Drugs CDMO Market

North American region is leading the market.

Global Orphan Drugs CDMO Market is expected to grow at a 7.5% CAGR during the forecast period for 2025-2034.
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