Orphan Drugs Market Report, Global Forecast to 2032

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The reimbursement recommendations and rebates to encourage the production of ODs escalate the market growth to furthered height.

The worldwide orphan medicine market is expanding at a rapid rate. The market is expanding because to the increasing frequency of rare but significant, life-threatening illnesses such as human papillomavirus (HPV) and HPV16-positive cervical carcinoma. HPV is a viral infection that produces warts on the skin and mucous membranes, as well as several types of cancer. Furthermore, the growing geriatric population has a substantial impact on market growth.   

Moreover, the reimbursement recommendations and rebates to encourage the production of ODs escalate the market growth to furthered height. The orphan pharmaceuticals market was valued at USD 160.78 billion in 2021 and is expected to rise from USD 175.57 billion in 2022 to USD 355.00 billion by 2030, representing a compound annual growth rate (CAGR) of 9.20% during the forecast period (2022- 2030).

The Orphan Drugs market is burgeoning, addressing the unmet medical needs of rare diseases. Orphan medicines, developed for conditions affecting a small patient population, are gaining traction due to regulatory incentives and growing investment in rare disease research. With heightened awareness and advocacy efforts, the market continues to expand, offering hope to patients worldwide.

HTA reforms, as well as the demand for breakthrough diagnostics and treatment processes, are important drivers of market expansion. In addition, government measures aimed at raising awareness and developing orphan medications have a favorable impact on market growth. Furthermore, the launch of new orphan medications and therapies to treat rare pediatric disorders drives market growth.

Conversely, the high production cost of ODs is projected to stifle market expansion. Furthermore, governmental restrictions on profitability have a significant impact on market expansion. Nonetheless, the availability of cutting-edge technology and increased access to treatment will help the market develop throughout the projection period.

Orphan Drugs Market   - Segments

The report is segmented into five dynamics;

By Drug Type: Biologics and Non-Biologics.

By Sale: Generics and Prescribed.

By Drug: Revlimid, Rituxan, Opdivo, Keytruda, Imbruvica, Soliris, Jakaf, Pomalyst, Darzalex, Spinraza, Adcetris, and others.

By Therapy Class: Oncology, Blood, Central Nervous Systems, Endocrine, Cardiovascular, Respiratory, and others.

By Regions: Americas, Europe, Asia-Pacific, Middle East & Africa, and Rest-of-the-World.

Orphan Drugs Market   - Regional Analysis

North America dominates the global orphan drugs market. In 2017, the region accounted for 39.98% of the overall market share. The largest market share attributes to the increasing prevalence of rare diseases and disorders, such as HPV. According to the US Centers for Disease Control & Prevention, HPV causes around 34,000 cases of cancer in the country each year.

Among these, cervical cancer is the most common HPV-associated cancer in American women. HPV16-positive cervical cancer is the next major cause responsible for approximately 50% of cancer cases. Additionally, the vast development of technology drives market growth, improving screening, and manufacturing techniques. Also, the increasing production of prophylactic vaccination against HPV infections fosters the growth of the regional market.

Europe stands second in the global orphan drugs market. The market growth is majorly driven by the presence of many well-established industry players and well-developed healthcare systems. Besides, the massive funding for drug discoveries and developments push the regional market growth. Moreover, the reimbursement recommendations for ODs (reimbursement - T2R) issued by the four well-established European HTA bodies (France, Germany, UK, and Scotland) boost the orphan drugs market in Europe.

The orphan drugs market outlook is brisk in the Asia Pacific region. Factors such as the vast technological advances and rising numbers of healthcare facilities foster regional market growth. The region demonstrates high growth potential for orphan drugs due to the increasing prevalence of diseases such as endocrinology, cardiovascular, lymphatic systems, and respiratory disorders. Moreover, the growing number of pharmaceutical companies with their strong export, favorable pricing, and clinical pipeline contribute to the regional market growth hugely.

Global Orphan Drugs Market - Competitive Landscape

Highly competitive, the orphan drugs market appears to be well-established with the presence of several notable players. To gain a larger competitive share, industry players adopt strategic initiatives such as mergers & acquisitions, collaborations, expansion, and new technologies/product launches. 

Major Players 

Players active in the global orphan drugs market include Bayer, Amryt Pharma PLC, Alexion Pharmaceuticals Inc., AbbVie, Inc., GlaxoSmithKline PLC, and Vertex Pharmaceuticals, Inc., among others. 

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