Market Insight

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 experiencing notable growth, with increasing focus on orphan medicines for rare diseases. These specialized drugs address unmet medical needs for patients with rare conditions, driving market expansion. Government incentives and streamlined regulatory pathways are encouraging pharmaceutical companies to invest in research and development for orphan medicines, fostering market growth and accessibility.

Orphan Drugs Boomers & Decliners

Patients suffering from uncommon diseases all over the world are looking for effective treatment options. Expanding R&D initiatives and growing advocacy by patient groups for effective treatment are driving the launch of newer and more enticing product solutions. Such patterns have resulted in the development of multiple blockbuster pharmaceuticals for the treatment of various malignancies, immune illnesses, and other conditions. For example, in June 2020, Chiasma, Inc. reported that the FDA had approved its Mycapssa product offering for the treatment of acromegaly. As a result, the introduction of such sophisticated orphan therapies by established corporations has contributed to increased uptake of these medications. This, in turn, is likely to contribute to the growth of the global orphan medicines market over the forecast period.

Another significant driving factor in the global market is the increasing R&D spending by leading players in orphan medication development and unique product offerings. Since the increased awareness and knowledge of rare diseases, several significant clinical-stage biopharmaceutical startups and established market players have solid pipeline candidates for orphan medications in various phases of clinical trials. Thus, this growing interest in rare disorder treatments is due to the fact that important pharmaceutical breakthroughs leading to blockbuster drug creations are more likely in uncommon disorders than in typical pharmaceutical portfolios.

Furthermore, a key motivator for this is that pharmaceutical companies are required to do substantial research for standard medicines for diseases such as diabetes and coronary artery disease (CAD) for approval, as opposed to rare disorders. All of these are expected to drive market expansion over the forecast period.

Segment Review

The global orphan drugs market is studied for the segments of drug type, sale, drug, and therapy class.

By the segment of drug type, the market has included biologics and non-biologics. The biologics grabbed the market value of USD 75,103.32 million in the year 2017.

By the sale segment, the market has included generics and prescribed.

By the segment of the drug, the market has included Rituxan, Revlimid, keytruda, opdivo, Soliris, imbruvica, pomalyst, jakaf, spinraza, darzalex, and adcetris.

The therapy class segment has included blood, oncology, endocrine, central nervous systems, cardiovascular, and respiratory.

Regional Framework

The Americas grabbed the largest Orphan Drugs Market share of 39.98% in the year 2017. The Americas is estimated to lead the global orphan drugs market in the anticipated period. The market growth in this region is mostly owing to the rising prevalence of rare disorders.

The European market for orphan drugs might become the second-largest during the review period. The rising number of pharmaceutical companies exporting products outside or within Europe is expected to contribute to market growth. Several European companies are engaged in research and development of orphan drugs as per the need of patients. The orphan drugs market in Europe might expand several folds in the coming years owing to numerous favorable factors such as favorable pricing, the introduction of innovative technologies, unmet medical necessities, reimbursement, and a robust clinical pipeline.

Asia-Pacific is also in the line of estimation to be the fastest-growing market owing to the developing healthcare infrastructure. Owing to the rising population, Asia has enormous long-term potential for orphan drug medications and treatments. Also, due to the rising prevalence of other diseases such as cardiovascular, endocrinology, and lymphatic systems, as well as respiratory disorders, the market expansion for orphan drugs is witnessed in this region.

Top Players

The top players in the global orphan drugs market are listed as Mylan, F. Hoffmann-La Roche AG, Novartis AG, Celgene Corporation, Takeda Pharmaceutical Company Limited, Biogen, Eli Lilly and Company, Merck KGaA, Sanofi, Shire Pharmaceutical, Alexion Pharmaceuticals Inc., Pfizer Inc., Bristol Meyer Squibb.

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