Visiongain has published a new report entitled Orphan Drugs Market Report 2023-2033: Forecasts by Therapeutic Area (Cancer, Neurological Diseases, Cardiovascular Diseases, Hematologic Disorders, Infectious Diseases, Metabolic Diseases, Others), by Drug Type (Biologics, Biosimilar, Others) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies AND COVID-19 Impact and Recovery Pattern Analysis.

The Orphan Drugs market is valued at US$187.6 billion in 2022 and is projected to grow at a CAGR of 11.1% during the forecast period 2023-2033.

Market growth in the orphan drug sector is being primarily fuelled by the grant of market exclusivity, a key driver during the projected period. Due to the limited patient populations and the specialized nature of rare diseases, competition within the orphan drugs market remains restricted. Market exclusivity offers pharmaceutical companies a unique opportunity to establish a dominant position, virtually devoid of competition for a specific duration. This, in turn, mitigates the risk of generic or biosimilar alternatives challenging their market share and pricing control. Additionally, market exclusivity empowers pharmaceutical firms to set premium prices for orphan drugs. Given the scarcity of treatment options for rare diseases, patients and healthcare systems are often willing to invest more in effective therapies. The combination of elevated pricing and the protection of market exclusivity has the potential to generate substantial revenue for these companies, rendering the development of orphan drugs financially viable. Notably, key drugs such as Trikafta/Kaftrio, Darzalex, Hemlibra, and Ultomiris are anticipated to maintain their market exclusivity throughout the forecasted period.

How has COVID-19 had a Significant Impact on the Orphan Drugs Market?
In recent years, the orphan drugs market has experienced both direct and indirect consequences as a result of the COVID-19 pandemic. The global supply chains have been disrupted due to the pandemic, which has had repercussions on the manufacturing and distribution of pharmaceutical products, including orphan drugs. The implementation of international travel restrictions, lockdown measures, and the heightened demand for healthcare resources have presented significant challenges in ensuring a consistent supply of medications. Unfortunately, this has had an adverse impact on patient access to orphan drugs.

Furthermore, the pandemic has created disruptions in the ongoing clinical trials related to orphan drugs. Many of these trials have encountered delays or interruptions as a consequence of site closures, restricted patient access, and the redirection of resources towards COVID-19 research priorities. These disruptions have the potential to impede the progress of orphan drug development and approval, consequently affecting the anticipated timelines and market entry for these medications.

How will this Report Benefit you?
Visiongain’s 258-page report provides 96 tables and 143 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the orphan drugs market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for orphan drugs. 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 orphan drugs 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?

Increasing R&D Activities in the Development of Novel Therapies
Enhancing research and development (R&D) endeavors aimed at creating orphan drugs plays a pivotal role in addressing the unmet medical requirements of individuals afflicted with rare diseases. The promotion of increased collaboration among academic institutions, market stakeholders, and regulatory authorities holds the potential to encourage the exchange of knowledge and expedite the advancement of orphan drug development. Collaboration serves as a catalyst for improved efficiency, the reduction of redundant efforts, and the seamless translation of research discoveries into tangible therapeutic solutions. As an illustrative example, according to the Global Genes’ NEXT 2022 report, published in January 2022, the pharmaceutical and biotechnology sectors, along with research institutes, managed to raise approximately US$22.9 billion in funding for the creation of innovative therapies targeting rare diseases in 2021, drawing from both public and private sources.

Unmet Medical Needs Anticipated to Boost Industry Growth
Unmet medical needs, characterized by the scarcity of treatment options for rare and often life-threatening diseases, serve as a potent catalyst driving the growth of the orphan drugs market. With a limited patient population facing severe suffering and high mortality rates, there exists a compelling demand for innovative and effective treatments. Pharmaceutical companies are incentivized to invest in research and development to address these pressing medical needs, encouraged by regulatory support, patient advocacy, and the unique market viability that allows for premium pricing. This convergence of urgency, regulatory incentives, and financial viability has fueled significant investment in orphan drug development, offering hope and life-saving interventions to those facing rare diseases with limited therapeutic options.

Where are the Market Opportunities?

High Growth Potential in Emerging Economies
Developing economies, characterized by their sizable and diverse populations, offer substantial growth prospects for orphan drugs. For example, India alone is estimated to have 72 to 96 million people affected by rare diseases, creating a substantial market opportunity. Limited access to effective treatments in these regions has resulted in a higher burden of untreated or under-treated rare diseases, further underscoring the potential for orphan drugs to address unmet medical needs. Additionally, emerging economies are actively enhancing their healthcare infrastructure, including research institutions and regulatory frameworks, which bolsters their capacity to support orphan drug development and distribution. Improved healthcare systems also contribute to heightened awareness, diagnosis rates, and demand for orphan drugs. Government initiatives like India's National Policy for Rare Diseases further stimulate growth opportunities for orphan drug manufacturers. Furthermore, regulatory authorities are streamlining approval processes, offering incentives, and reducing fees to attract manufacturers, thereby enhancing affordability and patient access to these critical treatments in developing economies. Manufacturers offering cost-effective solutions stand to capture a larger market share.

High Growth Potential in Emerging Economies
In the global orphan drugs market, there's a significant trend towards expanding the applications of existing orphan drugs beyond their original rare disease indications. This expansion is driven by ongoing research and development efforts by market players, aiming to broaden the usage of these drugs for different rare diseases. For instance, a study by the National Organization for Rare Disorders (NORD) found that several orphan drugs were approved for multiple rare diseases, increasing their revenue potential and reducing the cost of developing new orphan drugs. Additionally, combination therapies involving orphan drugs are being explored to enhance treatment outcomes, and these drugs may also prove effective for specific genetic mutations or age groups within the rare disease population. This diversification of applications holds promise for both patients and the orphan drug market.

Competitive Landscape
The major players operating in the orphan drugs market are AbbVie Inc., AstraZeneca, Biogen, Bristol-Myers Squibb Company, F. Hoffmann-La Roche Ltd., GSK plc, Johnson & Johnson Services, Inc., Merck & Co., Inc., Novartis AG, Pfizer Inc., Sanofi, and Vertex Pharmaceuticals Incorporated among others. 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 16th January 2023, CARsgen Therapeutics Holdings Limited and Huadong Medicine (Hangzhou) Co., Ltd., a wholly-owned subsidiary of Huadong Medicine Co., Ltd. Collaborated for the commercialization of CARsgen's BCMA CAR-T product CT053 in mainland China.
• On 21st November 2022, Merck and Canada, and Imago BioSciences, Inc. entered into a definitive agreement under which Merck, through a subsidiary, will acquire Imago for $36.00 per share in cash for an approximate total equity value of $1.35 billion.

Notes for Editors
If you are interested in a more detailed overview of this report, please send an e-mail to contactus@visiongain.com or call +44 (0) 207 336 6100.

About Visiongain
Visiongain is one of the fastest-growing and most innovative independent media companies in Europe. Based in London, UK, Visiongain produces a host of business-to-business reports focusing on the automotive, aviation, chemicals, cyber, defence, energy, food & drink, materials, packaging, pharmaceutical and utilities sectors.

Visiongain publishes reports produced by analysts who are qualified experts in their field. Visiongain has firmly established itself as the first port of call for the business professional who needs independent, high-quality, original material to rely and depend on.