Global Orphan Drugs Market by Disease Type (Oncologic Diseases, Metabolic Diseases, Hematologic and Immunologic Diseases, Infectious Diseases, Neurologic Diseases and Other Rare Diseases), Indication (Non-Hodgkin Lymphoma, Acute Myeloid Leukemia, Cystic Fibrosis, Glioma, Pancreatic Cancer, Ovarian Cancer, Multiple Myeloma, Duchenne Muscular Dystrophy, Graft vs Host Disease, Renal Cell Carcinoma and Others) - Global Opportunity Analysis and Industry Forecast, 2014-2022The pharmaceutical agents that are administered to treat, diagnose, and prevent life-threatening diseases (rare diseases) are known as orphan drugs. The global orphan drugs market accounted for $106 billion in 2015, and is anticipated to reach $169 billion by 2022, registering a CAGR of 6.8% from 2016 to 2022.
Orphan drugs are used to treat rare (orphaned) diseases prevalent in a very small patient pool. Hence, the pharmaceutical industry takes little interest in their development. Moreover, their development requires high cost and the chances of returns on investment are comparatively less when compared to non-orphan drugs. These drugs treat different rare diseases, such as non-Hodgkin lymphoma, acute myeloid leukemia, cystic fibrosis, and others.
Conducive government regulations and market exclusivity for orphan drugs drives the global orphan drugs market. In addition, surge in prevalence of rare diseases and rise in investment in research, drug development, and innovation supplement the market growth. However, limited patient pool for clinical trials & product marketing and high treatment costs per patient impede the market growth. Furthermore, growth in novel indications for known orphan drugs and untapped emerging markets offer lucrative opportunities for the market players.
The orphan drugs market is segmented based on disease type, indication, and geography. Based on disease type, the market is divided into oncologic diseases, metabolic diseases, hematologic and immunologic diseases, infectious diseases, neurologic diseases, and other rare diseases. In the orphan drugs disease type segment, oncologic diseases occupied dominant share in 2015, and the segment is expected to maintain this trend during the forecast period. This is attributed to the array of different forms of rare cancers, such as leukemia, myeloma, angiosarcoma, and others prevalent in the patient population.
Based on indication, this market is classified into non-Hodgkin lymphoma, acute myeloid leukemia, cystic fibrosis, glioma, pancreatic cancer, ovarian cancer, multiple myeloma, Duchenne muscular dystrophy, Graft vs host disease, renal cell carcinoma, and others. The non-Hodgkin lymphoma was the highest revenue contributor in 2015, while renal cell carcinoma is anticipated to grow at the highest rate. The growth for this segment is due to increase in investment in R&D throughout the world along with surge in awareness about kidney cancer.
Geographically, this market is analyzed across North America, Europe, Asia-Pacific, and LAMEA. North America accounted for the largest market share in the orphan drugs market in 2015, and is expected to retain its dominance throughout the forecast period. This is primarily attributed to the higher buying power, availability, accessibility, and treatment options that favor the utilization of orphan drugs in this region. There is also an increase in product approvals for different pharmaceutical products designated as orphan drugs by the U.S. Food and Drug Administration (FDA), which fosters the market growth. However, Asia-Pacific is expected to emerge as a lucrative area with maximum growth potential, owing to the improvement in R&D facilities, available disposable income, and rapidly developing economic conditions.
Product development is the key strategy adopted by market players. The report provides a comprehensive analysis of the key players that operate in the global orphan drugs market.
KEY PLAYERS PROFILED
Aegerion Pharmaceuticals, Inc.
Bristol-Myers Squibb Company
F. Hoffmann-La Roche Ltd.
Johnson & Johnson.
KEY MARKET BENEFITS
The study provides an in-depth analysis of the global orphan drugs market along with the current trends and future estimations to elucidate the imminent investment pockets.
The report presents quantitative analysis of the market from 2014 to 2022 to enable stakeholders to capitalize on the prevailing market opportunities.
Extensive analysis of the market based on indications assists in understanding the trends in the industry.
Key market players along with their strategies are thoroughly analyzed to understand the competitive outlook of the industry.
KEY MARKET SEGMENTS
By Disease Type
Hematologic and Immunologic Diseases
Other Rare Diseases
Acute Myeloid Leukemia
Duchenne Muscular Dystrophy
Graft vs Host Disease
Renal Cell Carcinoma
Rest of Europe
Rest of Asia-Pacific
Rest of LAMEA
1.1. REPORT DESCRIPTION
1.2. KEY BENEFITS
1.3. KEY MARKET SEGMENTS
1.4. RESEARCH METHODOLOGY
1.4.1. Primary research
1.4.2. Secondary research
1.4.3. Analyst tools and models
CHAPTER 2. EXECUTIVE SUMMARY
2.1. CXO PERSPECTIVE
CHAPTER 3. MARKET OVERVIEW
3.1. MARKET DEFINITION AND SCOPE
3.2. KEY FINDINGS
3.2.1. Top investment pockets
3.2.2. Top winning strategies
3.3. PORTERS FIVE FORCES ANALYSIS
3.4. PATENT ANALYSIS
3.4.1. Patent analysis by year
3.4.2. Patent analysis by region
3.4.3. Patent analysis by indication
3.6. MARKET DYNAMICS
220.127.116.11. Conducive government legislations
18.104.22.168. Market exclusivity for orphan drugs
22.214.171.124. Surge in prevalence of rare diseases
126.96.36.199. Limited patient pool for clinical trial and product marketing
188.8.131.52. High treatment costs per patient
184.108.40.206. Growing novel indications designated for known orphan drugs
220.127.116.11. Untapped emerging markets
CHAPTER 4. ORPHAN DRUGS MARKET, BY DISEASE TYPE
4.1.1. Market size and forecast
4.2. ONCOLOGIC DISEASES
4.2.1. Key market trends
4.2.2. Growth factors and opportunities
4.2.3. Market size and forecast
4.3. METABOLIC DISEASES
4.3.1. Key market trends
4.3.2. Growth factors and opportunities
4.3.3. Market size and forecast
4.4. HEMATOLOGIC AND IMMUNOLOGIC DISEASES
4.4.1. Key market trends
4.4.2. Growth factors and opportunities
4.4.3. Market size and forecast
4.5. INFECTIOUS DISEASES
4.5.1. Key market trends
4.5.2. Growth factors and opportunities
4.5.3. Market size and forecast
4.6. NEUROLOGIC DISEASES
4.6.1. Key market trends
4.6.2. Growth factors and opportunities
4.6.3. Market size and forecast
4.7. OTHER RARE DISEASES
4.7.1. Key market trends
4.7.2. Growth factors and opportunities
4.7.3. Market size and forecast
CHAPTER 5. ORPHAN DRUGS MARKET, BY INDICATION
5.1.1. Market Size and Forecast
5.2. NON-HODGKINS LYMPHOMA
5.2.1. Market size and forecast
5.3. ACUTE MYELOID LEUKEMIA
5.3.1. Market size and forecast
5.4. CYSTIC FIBROSIS
5.4.1. Market size and forecast
5.5.1. Market size and forecast
5.6. PANCREATIC CANCER
5.6.1. Market size and forecast
5.7. OVARIAN CANCER
5.7.1. Market size and forecast
5.8. MULTIPLE MYELOMA
5.8.1. Market size and forecast
5.9. DUCHENNE MUSCULAR DYSTROPHY
5.9.1. Market size and forecast
5.10. GRAFT VS. HOST DISEASE
5.10.1. Market size and forecast
5.11. RENAL CELL CARCINOMA
5.11.1. Market size and forecast
5.12.1. Market size and forecast
CHAPTER 6. ORPHAN DRUGS MARKET, BY GEOGRAPHY
6.1.1. Market size and forecast
6.2. NORTH AMERICA
6.2.1. Key market trends
6.2.2. Key growth factors and opportunities
6.2.3. Market size and forecast
18.104.22.168. U.S. market size and forecast
22.214.171.124. Canada market size and forecast
126.96.36.199. Mexico market size and forecast
6.3.1. Key market trends
6.3.2. Key growth factors and opportunities
6.3.3. Market size and forecast
188.8.131.52. Germany market size and forecast
184.108.40.206. UK market size and forecast
220.127.116.11. France market size and forecast
18.104.22.168. Italy market size and forecast
22.214.171.124. Spain market size and forecast
126.96.36.199. Rest of Europe market size and forecast
6.4.1. Key market trends
6.4.2. Key growth factors and opportunities
6.4.3. Market size and forecast
188.8.131.52. Japan market size and forecast
184.108.40.206. China market size and forecast
220.127.116.11. India market size and forecast
18.104.22.168. Australia market size and forecast
22.214.171.124. South Korea market size and forecast
126.96.36.199. Taiwan market size and forecast
188.8.131.52. Rest of Asia-Pacific market size and forecast
6.5.1. Key market trends
6.5.2. Key growth factors and opportunities
6.5.3. Market size and forecast
184.108.40.206. Brazil market size and forecast
220.127.116.11. Turkey market size and forecast
18.104.22.168. Saudi Arabia market size and forecast
22.214.171.124. South Africa market size and forecast
126.96.36.199. Rest of LAMEA market size and forecast
CHAPTER 7. COMPANY PROFILES
7.1. ABBVIE INC.
7.1.1. Company overview
7.1.2. Company snapshot
7.1.3. Operating business segments
7.1.4. Product portfolio
7.1.5. Business performance
7.1.6. Key strategic moves and developments
7.2. AEGERION PHARMACEUTICALS, INC. (NOVELION THERAPEUTICS INC.)
7.2.1. Company overview
7.2.2. Company snapshot
7.2.3. Operating business segments
7.2.4. Product portfolio
7.2.5. Business performance
7.2.6. Key strategic moves and developments
7.3. BRISTOL-MYERS SQUIBB COMPANY
7.3.1. Company overview
7.3.2. Company snapshot
7.3.3. Operating business segment
7.3.4. Product portfolio
7.3.5. Business performance
7.3.6. Key strategic moves and developments
7.4. CELGENE CORPORATION
7.4.1. Company overview
7.4.2. Company snapshot
7.4.3. Operating business segments
7.4.4. Product portfolio
7.4.5. Business performance
7.4.6. Key strategic moves and developments
7.5. F. HOFFMANN-LA ROCHE AG
7.5.1. Company overview
7.5.2. Company snapshot
7.5.3. Operating business segments
7.5.4. Product portfolio
7.5.5. Business performance
7.5.6. Key strategic moves and developments
7.6. GLAXOSMITHKLINE PLC
7.6.1. Company overview
7.6.2. Company snapshot
7.6.3. Operating business segments
7.6.4. Product portfolio
7.6.5. Business performance
7.6.6. Key strategic moves and developments
7.7. JOHNSON & JOHNSON
7.7.1. Company overview
7.7.2. Company snapshot
7.7.3. Operating business segments
7.7.4. Product portfolio
7.7.5. Business performance
7.7.6. Key strategic moves and developments
7.8. NOVARTIS AG
7.8.1. Company overview
7.8.2. Company snapshot
7.8.3. Operating business segments
7.8.4. Product portfolio
7.8.5. Business performance
7.8.6. Key strategic moves and developments
7.9. PFIZER INC.
7.9.1. Company overview
7.9.2. Company snapshot
7.9.3. Operating business segments
7.9.4. Product portfolio
7.9.5. Business performance
7.9.6. Key strategic moves and developments
7.10.1. Company overview
7.10.2. Company snapshot
7.10.3. Operating business segments
7.10.4. Product portfolio
7.10.5. Business performance
7.10.6. Key strategic moves and developments
Orphan drugs are specialized pharmaceutical agents that are administered for treatment of rare (orphan) diseases. These diseases have a very low prevalence rate, hence, pharmaceutical companies do not readily invest in these drugs as the returns on investment in orphan drugs is risky when compared with non-orphan drugs. Moreover, multiple clinical trials for drug testing cannot be voluntarily performed due to the small patient population. However, orphan drugs have shown tremendous potential in diagnosis and treatment of cancer, this trend is expected to continue throughout the forecast period. In addition, the increase in indications of orphan drugs to treat an array of different diseases such as lymphoma, leukemia, myeloma, and others boost the market growth.
The major factors that drive the global orphan drugs market are conducive government regulations, market exclusivity for orphan drugs, and surge in prevalence of rare diseases. In addition, growth in novel indications designated for known orphan drugs, and untapped emerging markets thereby provide lucrative opportunities for market growth. However, limited patient pool for clinical trial & product marketing and high treatment costs per patient restrain the market growth.
The oncologic disease type segment occupies the greatest share in the orphan drug market owing to the array of diverse forms of rare cancers, such as leukemia, myeloma, angiosarcoma, and others prevalent in the patient population. Whereas, the renal cell carcinoma indication is anticipated to grow at the highest rate. The growth for this segment is due to increase in investment in R&D throughout the world along with surge in awareness about kidney cancer. Multiple myeloma currently dominates the indication segment and is expected to grow at a CAGR of 7.8%.
Key Findings of Orphan Drugs Market
Metabolic diseases type segment is projected to grow at the highest CAGR from 2016 to 2022.
Neurologic disease type segment is poised to witness the fastest growth during the forecast period.
Non-Hodgkin Lymphoma indication generated the highest revenue in 2015 and is expected to continue its dominance ahead as well.
North America dominated the market and is projected to grow at a CAGR of 5.5%.
Asia-Pacific is expected to witness the fastest growth during the forecast period.
In 2015, Asia-Pacific and LAMEA collectively accounted for around two-fifths of the global orphan drugs market and are expected to continue this trend due to rise in awareness regarding orphan drugs in China, India, and other developing economies. Various governmental and non-governmental organizations implement favorable legislations that supplement the growth of orphan drugs market. In addition, increase in investment in R&D for drug development by public and private sectors is also expected to boost the market growth.
The major companies profiled in the report include AbbVie Inc., Aegerion Pharmaceuticals, Inc., Bristol-Myers Squibb Company, Celgene Corporation, F. Hoffmann-La Roche Ltd., GlaxoSmithKline plc, Johnson & Johnson, Novartis AG, Pfizer Inc., and Sanofi.
The analyst offers exhaustive research and analysis based on a wide variety of factual inputs, which largely include interviews with industry participants, reliable statistics, and regional intelligence. The in-house industry experts play an instrumental role in designing analytic tools and models, tailored to the requirements of a particular industry segment. The primary research efforts include reaching out participants through mail, tele-conversations, referrals, professional networks, and face-to-face interactions.
They are also in professional corporate relations with various companies that allow them greater flexibility for reaching out to industry participants and commentators for interviews and discussions.
They also refer to a broad array of industry sources for their secondary research, which typically include; however, not limited to:
- Company SEC filings, annual reports, company websites, broker & financial reports, and investor presentations for competitive scenario and shape of the industry
- Scientific and technical writings for product information and related preemptions
- Regional government and statistical databases for macro analysis
- Authentic news articles and other related releases for market evaluation
- Internal and external proprietary databases, key market indicators, and relevant press releases for market estimates and forecast
Furthermore, the accuracy of the data will be analyzed and validated by conducting additional primaries with various industry experts and KOLs. They also provide robust post-sales support to clients.