Global Orphan Drug Growth Opportunities

Revenue Forecast

The revenue estimate for the base year 2023 is $206.82 billion, with a CAGR for the study period (2023–2028) of 10.8%.

The Impact of the Top 3 Strategic Imperatives on the Orphan Drug Industry

Transformative Megatrends

• Why: Healthcare systems are under pressure to contain current and future spending. Payers are diligently scrutinizing medicine costs and patient access levels to ensure 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.
• Frost Perspective: Global collaboration and the formation of consortia are rising. In addition, resource sharing can accelerate research and development (R&D) efforts for rare disorders. Sustained financial support is essential to advance the development of therapies that target rare diseases (RDs) as pharmaceutical firms, payers, and patients face high orphan drug (OD) prices.

Competitive Intensity

• Why: In the last few years, ODs accounted for approximately half of all drug approvals. Biotechnology's progress has created opportunities to address RDs' root causes, enticing both well-established pharmaceutical firms and biotechnology entrepreneurs to invest in the development of groundbreaking medicine.
• Frost Perspective: Rapid technology advancement, including artificial intelligence (AI) and data analytics, has facilitated R&D optimization. Companies that use these technologies hold a competitive advantage in identifying prospective treatment leads; for example, in February 2023, Insilico's AI-discovered small-molecule drug, INS018_055, which treats idiopathic pulmonary fibrosis (IPF), received orphan drug designation (ODD).

Disruptive Technologies

• Why: Digital health technologies, the examination of large-scale datasets through AI, and the use of real-world evidence (RWE) and deep learning techniques are enabling predictive data-driven comprehension of RDs. Genomics and molecular diagnostics' progress is propelling the advancement of precision medicine for certain RDs.
• Frost Perspective: The emergence of cell gene therapy (CGT) and RNA-based therapies will continue to revolutionize the RD treatment landscape. Integrating omics data in drug discovery and development will improve disease understanding and identification of new therapy targets. CRISPR-Cas9 and other technologies have revolutionized gene editing by allowing precise modification or alteration of DNA.

Scope of Analysis

• The study covers the global OD industry, including North America; Europe; the Middle East, North Africa, and South Asia (MENASA); and Latin America.
• It provides insight into the key trends driving the industry and their future implications and assesses industry response to growing demand and the impact of economic factors on growth.
• An integral part of this study represents business models and growth opportunities aligned to industry needs and capabilities.
• RDs are classified as any disease that affects less than 200,000 individuals. Orphan diseases, including RDs, are neglected conditions whose treatment is often not considered profitable due to their high development cost and limited patient population.
• According to the US FDA, an OD is a drug used to treat a rare disease or condition that affects less than 200,000 persons in the United States.
• According to the European Medicines Agency (EMA), an OD is a drug intended to prevent or treat a life-threatening or chronically and seriously debilitating condition affecting not more than 5 in 10,000 people in the EU; moreover, marketing the drug in the EU is not likely to generate sufficient benefit for the affected people or the drug manufacturer to justify the investment.
• The study covers the 2 major OD subsegments, that is, product type and therapeutic category. Examples of prominent pharmaceutical companies with strong RD pipelines include Bristol Myers Squibb, Johnson & Johnson, Novartis, AstraZeneca, Vertex Pharmaceuticals, Roche, Sanofi, Takeda, AbbVie, Pfizer, and Merck & Co.
• Revenue centers around the probable penetration of ODs in the development pipeline based on historical trends. Owing to variations in the patent expiry of drugs across geographies, forecasts are provided by year and not by region. In addition to the approved orphans showing promising sales projections, nearly >500 Phase III, II, or filed drugs are included in the forecast.
• Separate chapters are not provided for subsegments; they are covered in-depth in the Growth Opportunity Analysis section.

Segmentation

Approximately 10,000 RDs impact about 400 million people worldwide, with 30 million in the United States alone. In the EU, the European Medicines Agency estimates that up to 36 million people are diagnosed with an RD; however, <5% of these people have access to approved therapies.

Product Type

• Biologics
  • Therapeutic Category1
    • Oncology
      • Immunomodulators; Musculoskeletal System
    • Non-oncology
      • Central Nervous System
      • Systemic Anti-infectives
      • Blood Disorders, Respiratory System, Cardiovascular System, Endocrine System, and Others2
• Small Molecules

Competitive Environment