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Rare Diseases market is projected to grow at a CAGR of 8.8% by 2034: Visiongain
Visiongain has published a new report entitled Rare Diseases Market Report 2024-2034: Forecasts by Diseases (Cancer, Metabolic Diseases, Neurological Conditions, Hematologic Disorders, Infectious Diseases, Musculoskeletal Disorders, Cardiovascular Disorders, Others), by Type (Non Biologics, Biologics), by End-users (Specialty Pharmacies, Hospital Pharmacies, Retail Pharmacies), by Age Group (Adult, Paediatric) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies.
The global rare diseases market is estimated at US$204.67 billion in 2024 and is projected to grow at a CAGR of 8.8% during the forecast period 2024-2034.
Increasing Demand from Emerging Economies
The burgeoning demand for rare disease treatments from emerging economies presents a pivotal opportunity for growth within the rare diseases market. As healthcare infrastructure in these regions continues to improve and access to medical care expands, there is a corresponding increase in the diagnosis and treatment of rare diseases. This rising demand is fuelled by several factors, including greater awareness among healthcare professionals and patients, as well as an evolving regulatory landscape that prioritizes rare disease care.
As emerging economies experience demographic shifts and lifestyle changes, there is a notable uptick in the prevalence of chronic and genetic conditions, further driving the need for specialized treatments. Pharmaceutical companies, recognizing the potential of these markets, are increasingly investing in research and development initiatives tailored to address the unique needs of patients in emerging economies.
Moreover, advancements in technology and healthcare delivery models are facilitating greater access to rare disease treatments, further bolstering demand. This growing market presents opportunities for collaboration and partnerships between local and international stakeholders, fostering innovation and expanding the reach of rare disease therapies. Overall, the increasing demand from emerging economies not only signifies a significant growth driver for the rare diseases market but also underscores the importance of equitable access to healthcare on a global scale.
For instance, on 15th March 2024, Novartis announced that it is ramping up its commitment in Singapore by breaking ground on a US$256 million expansion of its biopharmaceutical production facility. The expansion aims to address the surging demand in the Asian market for antibodies. Novartis plans to introduce digitalization and automation at the site to increase manufacturing productivity and to upskill its workforce.
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How will this Report Benefit you?
Visiongain’s 344-page report provides 126 tables, 185 charts. Our new study is suitable for anyone requiring commercial, in-depth analyses for the rare diseases market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for rare diseases. Get financial analysis of the overall market and different segments including type, process, upstream, downstream, and company size and capture higher market share. We believe that there are strong opportunities in this fast-growing rare diseases market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.
What are the Current Market Drivers?
Rising Number of Drug Approvals
The rising number of drug approvals specifically targeting rare diseases serves as a crucial driver for market growth. Each approval represents a breakthrough in addressing previously unmet medical needs, expanding treatment options for patients with rare conditions. Additionally, these approvals signal confidence in the viability of rare disease drug development, attracting investment and fostering innovation within the sector. As the pipeline of approved treatments continues to grow, patients, healthcare providers, and investors alike benefit from increased access to effective therapies, driving market expansion and improving patient outcomes.
For instance, on 2nd January 2024, nature reviews drug discovery stated that in 2023, the FDA's Center for Drug Evaluation and Research (CDER) greenlit 55 new drugs, marking a significant expansion of the small molecule and biologic inventory. This approval cohort is nearly 50% larger than the one in 2022, which deviated from the approval trend line. The ten-year rolling average for new CDER approvals has now reached 46 per year, the highest figure seen in over 20 years.
Robust Pipeline by Major Players
The robust pipeline of rare disease treatments developed by major pharmaceutical players serves as a potent driver for market growth. These pipelines reflect significant investments in research and development, showcasing a commitment to addressing unmet medical needs within the rare disease space. With promising candidates advancing through clinical trials, there's heightened anticipation for the introduction of novel therapies that could revolutionize treatment paradigms for various rare conditions. As these therapies progress towards approval, they not only expand the treatment options available but also contribute to the overall growth and sustainability of the rare diseases market.
For instance, on 18th April 2024, two companies, Zura Bio and Benitec Biopharma, secured $152.5 million through private placements in public equity to advance their therapeutics for rare diseases through clinical development. Zura Bio, a clinical stage immunology company focusing on autoimmune and inflammatory diseases, finalized subscription agreements for a private investment in public equity (PIPE) expected to yield approximately $112.5 million in gross proceeds. The financing was led by Access Biotechnology and involved contributions from RA Capital Management, Deep Track Capital, Great Point Partners, Suvretta Capital, funds managed by Allostery Investments, Armistice Capital, and other prominent investment management firms. This funding move underscores a trend among investors in the biotech industry to capitalize on opportunities presented by purchasing private placements in public companies, leveraging the relatively lower valuations in the public markets.
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Where are the Market Opportunities?
Advancement in Research and Development
Advancements in research and development present compelling opportunities within the rare diseases market. As scientific understanding of rare conditions deepens and technologies evolve, there's a growing potential to identify novel therapeutic targets and develop more precise treatments. Additionally, collaborations between academia, industry, and patient advocacy groups are fostering interdisciplinary approaches, accelerating the pace of innovation. With emerging fields such as gene therapy and precision medicine gaining traction, there's renewed optimism for breakthroughs that could transform the lives of individuals affected by rare diseases. These advancements not only improve patient outcomes but also attract investment and drive market expansion.
Orphan Drug Designation Incentives
The designation of orphan drugs by regulatory agencies unlocks a myriad of incentives, propelling them into a prime position within the pharmaceutical landscape. Market exclusivity stands as one of the most impactful benefits, affording the sponsor a considerable advantage by preventing competitors from marketing similar products for the same indication for a specified period, typically 7 to 10 years. This exclusivity not only fosters a conducive environment for recouping investments but also incentivizes companies to undertake the often costly and risky endeavor of developing treatments for rare diseases. Additionally, tax credits for qualified clinical testing expenses provide financial relief, further encouraging investment in orphan drug research and development.
Moreover, regulatory fee waivers or reductions streamline the approval process, mitigating financial burdens associated with regulatory compliance. The allure of expedited approval pathways, such as the U.S. FDA's Accelerated Approval and Priority Review programs, adds another layer of appeal, allowing for faster market access and potentially life-saving treatments for patients with unmet medical needs. Furthermore, the availability of protocol assistance and scientific advice from regulatory agencies facilitates smoother development processes, enhancing the overall efficiency and success rate of orphan drug programs. As a result, orphan drug designation incentives not only serve as a catalyst for innovation but also present lucrative opportunities for pharmaceutical companies to address unmet medical needs and make significant contributions to the rare disease market.
Competitive Landscape
The major players operating in the rare diseases market are AbbVie Inc., Alexion Pharmaceuticals, Inc. (AstraZeneca), Amgen Inc., Amneal Pharmaceuticals LLC, Biogen Inc., Bristol-Myers Squibb Company, Chiesi Farmaceutici S.p.A., Dr. Reddy’s Laboratories Ltd., F. Hoffmann-La Roche Ltd., Johnson & Johnson Services, Inc., Merck & Co Inc., Novartis AG, Pfizer Inc., Sanofi, Takeda Pharmaceutical Company Limited, Teva Pharmaceutical Industries Ltd., and Vertex Pharmaceuticals Incorporated. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launch.
Recent Developments
On 3rd April, 2023, Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics finalized the rolling submission of Biologics License Applications (BLAs) to the U.S. Food and Drug Administration (FDA) for exagamglogene autotemcel (exa-cel), an investigational treatment for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). These BLAs include requests for Priority Review, aiming to accelerate the FDA's assessment process to eight months from submission, compared to the standard 12-month review timeline.
On 31st January 2023, Amgen announced AMJEVITA (adalimumab-atto), a biosimilar to Humira (adalimumab), is now available in the United States.
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