Industry Background:
The rare diseases drug highly conditions as an orphan drug. It is highly utilized in the United States regions. There are near about 7000 rare diseases across the globe. The highest number of patients related to the rare disease drugs in the United States, the European Union, and Japan. The market is growing at a higher rate because of the increased number of current willingness and the increasing number of industry players. With the growing concern towards rare disease drugs, many emerging economies are focusing on development initiatives. For instance, India is looking to set rare diseases drug policy and also developing domestic treatments. In India increasing the occurrence of Duchenne muscular dystrophy among children forced the Ministry of Health and Family Welfare forming the national Policy for the treatment of rare diseases. Globally, also various legislations are developing in concern of the rare disease drugs such as the United States Orphan Drug Act is initiating various rare disease drug development.This growth is primarily driven by Rising Awareness among Consumers
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Attributes | Details |
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Study Period | 2017-2027 |
Base Year | 2021 |
Forecast Period | 2022-2027 |
Volume Unit | K Units |
Value Unit | USD (Million) |
Dominating Type | Pharmacies |
Customization Scope | Avail customization with purchase of this report. Add or modify country, region & or narrow down segments in the final scope subject to feasibility |
The Health Care Supplies sector in the
region has been increasing at a sustainable rate and further growth is expected to be witnessed over the forecast period, owing to the robust investments and expansion in production facilities in the region. Major Players, such as Novartis (Switzerland), Bristol-Myers Squibb Company (United States), Celgene Corporation (United States), F. Hoffmann-La Roche Ltd. (Switzerland), Pfizer, Inc. (United States), Sanofi S.A. (France), Alexion Pharmaceuticals, Inc. (United States), Eli Lilly and Company (United States), Novo Nordisk A/S (Denmark), AstraZeneca (United Kingdom), Eisai Co., Ltd. (Japan) and Daiichi Sankyo Company Limited (Japan), etc have either set up their manufacturing facilities or are planning to start new provision in the dominated region in the upcoming years.
Key Developments in the Market:
In Feb 2019, Ipsen announced the acquisition of the Clementia Pharmaceuticals for the treatment of the key late-stage clinical asset palovarotene, multiple osteochondromas, and others. Through this agreement, the company is aiming to enhance its product portfolio in rare diseases across the globe.
In Aug 2019, Critical path Institute and advocacy organization national organization revealed its new data and analytics platform for rare disorders. This platform is having multiple sources including clinical trials, observational studies, real-time data, and others.The global rare disease drug market is highly competitive and consists of some key players. In terms of market share, few of the key players presently dominate the global market. These market players are leveraging on strategic collaborative creativities to intensification their market share and escalation their profitability.
Regulatory Insights:
The rare disease drugs are regulated by the FDA. Recently, in the market there are three new drugs approved by the FDA for the treatment of two new types of rare diseases including non-Hodgkin lymphomas, and Fabry disease. The new drugs are Mogamulizumab-kpkc injection, Lanadelumab-flyo, and migalastat. Recently, the United States regulators registered COVID-19 as rare disease treatment. For this FDA grant an experimental medicine known as remdeivir for the treatment of coronavirus disease. With the continuously rising COVID-19 cases in the United States region, the health agency's made decisions to provide remdesivir at a lower price (if it will be approved).
Influencing Trend:
Increase in investment from the manufacturers of the leading drug across the globe to developing therapies, invest more in research and focused on offering innovative solutions. Which can bring high potential to offer a solution which impacted on rare diseases. The company is providing drugs to provide patients which includes emerging markets, with advance healthcare.
Market Growth Drivers:
Rising Awareness among Consumers, Technology Advancement in Manufacturing Process and Increasing Number of Rare Diseases in North America Regions, it almost occurs in one in ten American
Challenges:
The high challenge for patients, they are facing huge diversity in their clinical histories. These variables include age, disease progression, and various outcomes, whether they are clinician-reported, observer-reported, or patient-reported outcomes.
Restraints:
Fluctuation in Cost of Raw Materials Associated With Rare Disease Drugs and There are around 95 percent of rare disease which do not have any treatment across the globe
Opportunities:
Development in Drug Manufacturing Countries and Rising Investment of Research and Development on the Rare Disease Drugs, in Terms of Inventing New Treatments
AMA Research follows a focused and realistic research framework that provides the ability to study the crucial market dynamics in several regions across the world. Moreover, an in-depth assessment is mainly conducted by our analysts on geographical regions to provide clients and businesses the opportunity to dominate in niche markets and expand in emerging markets across the globe. This market research study also showcases the spontaneously changing Players landscape impacting the market's growth. Furthermore, our market researchers extensively analyze the products and services offered by multiple players competing to increase their market share and presence.
Data Sources of Rare Disease Drug Market Study
Primary Collection: InMail, LinkedIn Groups, Survey Monkey, Google, and Other professional Forums are some of the mediums utilized to gather primary data through key industry participants and appointees, subject-matter experts, C-level executives of Rare Disease Drug Industry, among others including independent industry consultants, experts, to obtain and verify critical qualitative commentary and opinion and quantitative statistics, to assess future market prospects.
The primary interviews and data collected as per the below protocols: By Designation: C-Level, D-Level, Others
By Company Type: Tier 1, Tier 2, Tier 3
Secondary Data Sources such as Annual reports, Press releases, Analyst meetings, Conference calls, Investor presentations, Management statements, and SEC filings of Rare Disease Drug players along with Regulatory Sites, Association, World bank, etc were used as sources secondary set of data.
Customization in the Report
AMA Research features not only specific market forecasts but also includes significant value-added commentary on:
- Market Trends
- Technological Trends and Innovations
- Market Maturity Indicators
- Growth Drivers and Constraints
- New Entrants into the Market & Entry/Exit Barriers
- To Seize Powerful Market Opportunities
- Identify Key Business Segments, Market Proposition & Gap Analysis
Against this Challenging Backdrop, Rare Disease Drug Study Sheds Light on
The Rare Disease Drug Market status quo and key characteristics. To end this, Analysts at AMA organize and took surveys of the Rare Disease Drug industry Players. The resultant snapshot serves as a basis for understanding why and how the industry can be expected to change.
Where Rare Disease Drug industry is heading and what are the top priorities. Insights are drawn from financial analysis, surveys, and interviews with key executives and industry experts.
How every company in this diverse set of Players can best navigate the emerging competition landscape and follow a strategy that helps them position to hold the value they currently claim or capture the new addressable opportunity.