Research Methodology – Orphan Drug Market
At Straits Research, we adopt a rigorous 360° research approach that integrates both primary and secondary research methodologies. This ensures accuracy, reliability, and actionable insights for stakeholders. Our methodology for the Orphan Drug Market comprises the following key stages:
Market Indicator & Macro-Factor Analysis
Our baseline thesis for the Orphan Drug Market is developed by integrating key market indicators and macroeconomic variables. These include:
Factors considered while calculating market size and share
- Number of diagnosed patients
- Degree of prevalence/incidence of orphan diseases globally
- Market sales of marketed orphan drugs
- Regulatory policies and incentives for orphan drugs
- Product pricing
- Patent status and exclusivity period
- R&D investments in the sector
- Investments by pharmaceutical companies in orphan drugs
- Entry of generic drugs and biosimilars
- Market competition
Key Market Indicators
- Projected market growth rate
- Global orphan drug sales
- Number of orphan drug designations
- Number of approved orphan drugs
- Orphan drug application rate
- R&D expenditure in orphan drugs
- Market penetration
- Market volatility
Growth Trends
- Increase in the incidence of rare diseases
- Increase in drug approvals and designations
- Escalating investments in R&D by pharmaceutical companies
- Expansion of the target patient pool
- Increasing global healthcare expenditure
- Advancements in biotechnology and pharmaceutical research
- Government incentives and policies encouraging orphan drug development
- Growing collaboration between pharmaceutical companies and research institutes
Secondary Research
Our secondary research forms the foundation of market understanding and scope definition. We collect and analyze information from multiple reliable sources to map the overall ecosystem of the Orphan Drug Market. Key inputs include:
- Annual reports, investor presentations, SEC filings
- Company press releases and product launch announcements
- Public executive interviews and earnings calls
- Strategy briefings and M&A updates
- Country-level industry associations and trade bodies
- Government dossiers, policy frameworks, and official releases
- Whitepapers, working papers, and public R&D initiatives
- Relevant Associations for the Orphan Drug Market
- Broker reports and financial analyst coverage
- Paid databases (Hoovers, Factiva, Refinitiv, Reuters, Statista, etc.)
- Import/export trade data and tariff databases
- Sector-specific journals, magazines, and news portals
- Global macroeconomic indicators and their cascading effect on the industry
- Demand–supply outlook and value chain analysis
- Consumer behaviour, adoption rates, and commercialization trends
Primary Research
To validate and enrich our secondary findings, we conduct extensive primary research with industry stakeholders across the value chain. This ensures we capture both qualitative insights and quantitative validation. Our primary research includes:
- Key Opinion Leader (KOL) Engagements
- Structured interviews with executives, product managers, and domain experts
- Paid and barter-based interviews across manufacturers, distributors, and end-users
- Discussions with stakeholders to validate demand-supply gaps
- Group discussions on emerging technologies, regulatory shifts, and adoption barriers
- Cross-verification of market sizing and forecasts with industry insiders
- Capturing business perspectives on growth opportunities and restraints
Data Triangulation & Forecasting
The final step of our research involves data triangulation ensuring accuracy through cross-verification of:
- Demand-side analysis (consumption patterns, adoption trends, customer spending)
- Supply-side analysis (production, capacity, distribution, and market availability)
- Macroeconomic & microeconomic impact factors
- Time-series analysis
- Regression and correlation studies
- Baseline modeling
- Expert validation at each stage
Outcome
The outcome is a comprehensive and validated market model that captures:
- Market sizing (historical, current, forecast)
- Growth drivers and restraints
- Opportunity mapping and investment hotspots
- Competitive positioning and strategic insights
Available for purchase with detailed segment data, forecasts, and regional insights.
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