Rare Disease Drugs Market 2023: Business Growth, Development Factors, Revenue Strategies, Future Trends 2030 | Novartis AG, Pfizer, Inc, Shire plc, Sanofi S.A.

Conditions that only affect a tiny fraction of the population are considered rare or orphan diseases. Treatments for extremely rare diseases often require extremely niche medications, so those drugs are given “orphan drug” status. The FDA classifies a disease as “orphan” if fewer than 200,000 Americans suffer from it. As a result of the Orphan Drugs Act passed in the United States, pharmaceutical companies can apply to the Food and Drug Administration for orphan drug status for medicines designed to treat extremely rare diseases and conditions. Orphan drug designation applications have been on the rise over the past few years, as reported by the FDA in 2017.

Due to a number of unknowns, the global market for drugs targeting rare diseases was not considered lucrative until recently. The study population was small because of the low incidence of the disease. In addition, manufacturers were hesitant to address unmet needs because of a lack of knowledge about the diversity and uniqueness of diseases. Manufacturers’ attention has been drawn to the rare disease drugs market due to the introduction of some expensive orphan drugs like Spinraza for spinal muscular atrophy, which costs US$ 125,000 per vial.

In 2016, the top 10 rare disease drugs brought in a combined $62.5 billion worldwide.

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🠺 Brief Overview to the research study.
🠺 Table of Contents the scope of the study’s coverage
🠺 Leading market participants
🠺 Structure of the report’s research framework
🠺 Coherent Market Insights’ research approach

Competitive Analysis of top 10 organizations in the global rare disease drugs market

In 2016, the Top 10 organizations in the market of global rare disease drugs Market on the basis of the 2016 revenue are:

• Novartis AG
• Celgene Corporation
• Hoffmann-La Roche Ltd.
• Bristol-Myers Squibb Company
• Pfizer, Inc
• Shire plc
• Sanofi S.A.
• Alexion Pharmaceuticals, Inc.
• Bayer
• Biogen, Inc.

1. As a multinational pharmaceutical company, Novartis AG offers a wide variety of pharmaceutical and medical solutions, from cancer drugs to eye care items. The company’s operations are managed by the Innovative Medicines Division, which is split into two subdivisions: the oncology business unit and the Novartis pharmaceutical business units. Progressive pharmaceuticals Ophthalmology, immunology, neuroscience, and dermatology, cardio-metabolic, respiratory and established medicines make up the pharmaceutical business in addition to products for oncology diseases and rare diseases. In 2015, Novartis’s orphan drug Gleevec for the therapy of long-term myeloid leukemia (CML) lost patent protection, and in 2016, generic Gleevec was released by Sun Pharma, cutting into Novartis’s sales.
2. Drugs like Revlimid, Pomalyst, and Otezla are just a few examples of Celgene Corporation’s expertise in oncology and inflammation. The company has a strong pipeline for oncology conditions, and it is currently concentrating on broadening the indication range of the existing therapies. Revlimid (lenalidomide) is being tested for the treatment of , indolent lymphoma, diffuse large B-cell lymphoma and follicular lymphoma in phase III clinical trials. In addition, the firm’s in-house technology platforms for target identification and drug discovery speed up the process of moving from target validation to lead identification and optimization.
3. In 2016, Shire Plc earned nearly US$ 11 billion and took the top spot as the world leader in the orphan drug products category (excluding oncology products). In 2016, the firm purchased Baxalta, along with its hematology/oncology/immunology therapeutic divisions. With an R&D budget of US$ 1.4 billion, Shire Plc is similarly committed to developing treatments for rare diseases. After acquiring Dyax and Baxalta in 2016, the company was able to expand its pipeline to include 37 therapies.
4. Both the supply and demand sides of the market have a significant impact on the global rare disease drugs market. The increasing interest of manufacturers in providing therapies in this sector of the healthcare industry is primarily motivated by the large gap between market needs and potential market offerings. The biopharma industry submitted 582 requests for orphan drug designation to the U.S. Food & Drug Administration (FDA) in 2016. The readily accessible highly effective drug therapies have also contributed to a rise in the sales of orphan drug diseases. Niraparib by Tesaro, Inc., in phase I for the treatment of ovarian cancer, Emicizumab by Roche, in phase III for the treatment of hemophilia, and LentiGlobin by Bluebird Bio, in varying phases for the treatment of sickle cell anemia and beta-thalassemia major, are just a few examples of the potential drug therapies that are present in the pipeline for various cancers and rare diseases. The outlook for the global market for drugs to treat rare diseases is expected to shift if these clinical trials are successful.
5. The study also includes an in-depth analysis of the major players in this market and a discussion of the competitive landscape they occupy. This will help you comprehend the methods utilized by dominant firms in this industry, as well as their ultimate objectives.

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Scope of the Report

• The report presents the analysis of Rare Disease Drugs market for the historical period of 2017-2021 and the forecast period of 2023-2030.
• The report analyses the Rare Disease Drugs Market by value (USD Billion)
• The report analyses the Rare Disease Drugs Market by Drug Type (Biologics, Non-Biologics).
• The report analyses the Rare Disease Drugs Market by Therapeutic Area (Infectious Disease, Oncology, Neurology, Metabolic, Others)
• The report analyses the Rare Disease Drugs Market by Patients (Pediatric, Adults).
• The report analyses the Rare Disease Drugs Market by Route of Administration (Oral, Injectable).
• The report analyses the Rare Disease Drugs Market by Distribution Channel (Hospital & Retail Pharmacies, Online Pharmacies)..
• The report analyses the Rare Disease Drugs Market by Patients (Pediatric, Adults).

Table of Contents with Major Points:

Executive Summary

• Introduction
• Key Findings
• Recommendations
• Definitions and Assumptions

Executive Summary

Market Overview

• Definition of Rare Disease Drugs Market
• Market Dynamics
• Drivers
• Restraints
• Opportunities
• Trends and Developments

Key Insights

• Key Emerging Trends
• Key Developments Mergers and Acquisition
• New Product Launches and Collaboration
• Partnership and Joint Venture
• Latest Technological Advancements
• Insights on Regulatory Scenario
• Porters Five Forces Analysis

Qualitative Insights Impact of COVID-19 on Global Rare Disease Drugs Market

• Supply Chain Challenges
• Steps taken by Government/Companies to overcome this impact
• Potential opportunities due to COVID-19 outbreak

Conclusion 

Appendix

• Data Sources
• Abbreviations
• Disclaimer

TOC Continued…!

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