Global Rare Diseases Treatment Market Size, Share & Industry Trends Analysis Report By Distribution Channel (Specialty Pharmacy, Hospital Pharmacy, and Online Pharmacy), By Route of Administration, By Therapeutic Area, By Regional Outlook and Forecast, 20

Global Rare Diseases Treatment Market Size, Share & Industry Trends Analysis Report By Distribution Channel (Specialty Pharmacy, Hospital Pharmacy, and Online Pharmacy), By Route of Administration, By Therapeutic Area, By Regional Outlook and Forecast, 2022 – 2028

The Global Rare Diseases Treatment Market size is expected to reach $255.4 billion by 2028, rising at a market growth of 12.5% CAGR during the forecast period.

A rare disease is one that impacts a small proportion of the population. In some parts of the world, an orphan disease is a rare disease that lacks a large enough market to gain support & resources for uncovering treatments for it, unless the government grants economically advantageous conditions for developing and selling such treatments. Rare disease treatments are those that have been created or sold for this purpose.

A multidisciplinary approach based on innovative initiatives for the development of novel SMA drugs to address unmet needs in rare disease treatment would surge the demand for rare diseases treatment. In addition, basic research on patient access, clinical development challenges, and regulatory approval processes are major characteristics highlighted in policy proposals.

The condition frequently affects a patient's entire family, and continuous care is taxing. Even if only a small number of people are affected by a disease, it can put a massively disproportionate strain on healthcare & social services. The value chain can be extended to achieve forward integration. To achieve a high return on investment, operating efficiency is being increased. This can be accomplished by shifting the production of important medicines to orphan drugs.

Covid-19 Impact Analysis

The ongoing COVID-19 pandemic is a major public health concern that has presented numerous challenges to researchers, healthcare providers, and patients. Because of the risk of coronavirus exposure, R&D organizations and biopharma companies are not able to concentrate on the development of new treatments. To address such challenges, the FDA is proactively collaborating with manufacturers to provide advanced treatments to maintain health care continuity by focusing on ongoing clinical trials. As a result, during the pandemic period, drugs for rare disease treatments are all being developed in a very limited quantity and thus limiting the market growth for rare disease treatments in the upcoming years.

Market Growth Factors

The increasing presence of rare diseases

Because of the high prevalence of individuals suffering from rare diseases, special drugs will be required, which is accelerating the acceptance of rare disease treatments. These treatments are aimed at specific genes that have been transformed in order to cure the disease. Because of their simplicity and major non-invasive treatments, these treatments have grown in popularity. With this rise in popularity, rare disease treatment is expected to expand rapidly. Furthermore, these treatments are becoming more popular as healthcare infrastructure around the world improves.

Rising R&D initiative for rare diseases treatment

The increasing R&D investments by popular vendors for orphan drug development of novel product offerings is one of the crucial driving factors prevailing in the market. Because public awareness and understanding of rare diseases have grown, a large number of key clinical-stage biopharmaceutical companies and established market players now have orphan drug pipeline candidates in various stages of clinical trials. This growing interest in rare disorder therapeutics is due to the fact that major pharmaceutical breakthroughs resulting in blockbuster drug developments are more likely in rare disorders than in traditional pharmaceutical portfolios.

Market Restraining Factors

High costs involved in R&D of the rare diseases treatment

Despite the increased focus on the development and marketing of rare disease therapeutics around the world, certain constraints are limiting the market growth. The high cost of researching and developing pipeline candidates for the treatment of rare diseases leads to an increase in drug prices. This huge cost of treatments has seriously limited the wide adoption of these product lines, particularly in developing countries. Such high treatment costs are also a source of concern in developed countries, where many drugs might not be adequately paid back, and hence, significantly increasing the patient's out-of-pocket costs.

Drug Type outlook

By drug type, the rare disease treatment market is divided into biologics, biosimilar, and small molecule. The small molecule segment generated a substantial revenue share in the rare diseases treatment market in 2021. One of the factors influencing the demand for small molecule drugs is the small and medium-sized pharmaceutical companies' increasing demand for outsourcing. Also, the strict regulations on the development, manufacturing & quality control are further supporting the market growth in this segment.

Distribution Channel Outlook

Based on distribution channel, the rare diseases treatment market is classified into hospital pharmacy, specialty pharmacy and online pharmacy. In 2021, the specialty segment witnessed the largest revenue share in the rare diseases treatment market. The growth is attributed to major players' strategic initiatives, like the acquisition of specialty pharmacies for the distribution of their drugs. During the forecast period, such mergers are anticipated to boost segment growth. Specialty drugs are extremely sophisticated drugs, usually based on biology, which structurally resemble substances found in the body.

Route of Administration Outlook

On the basis of route of administration, the rare disease treatment market is fragmented into oral, injectable and others. The oral segment covered a significant revenue share in the rare disease treatment market in 2021. The market share is due to several benefits such as safety, compliance, ease of ingestion, and no risk of pain in the oral route of administration. As a result of growing awareness about the efficacy of such drugs, the oral route of administration has become more popular in the treatment of rare diseases.

Therapeutic Area Outlook

Based on therapeutic area, the rare disease treatment market is segmented into cancer, neurological conditions, cardiovascular conditions, musculoskeletal conditions, hematologic disorders, infectious diseases, metabolic disorders, endocrine disorders and others. In 2021, the cancer segment dominated the rare disease treatment market by generating the highest revenue share. The high prevalence & recurrence rate of rare cancer evidence is the major factor responsible for this dominance. In addition, new treatments are continuously being updated for curing patients suffering from rare cancers. This is expected to rise the demand for rare disease treatment and thus supporting market growth in this segment.

Regional Outlook

Region wise, the rare disease treatment market is analyzed across the North America, Europe, Asia Pacific and LAMEA. In 2021, the North America region dominated the rare diseases treatment market with the maximum revenue share. This is because of the high prevalence of diseases, favorable healthcare infrastructure, and new product approvals for treatment. Access to products may enhance patient compliance, thereby expanding the customer base in the nations such as Canada, and the United States are propelling the growth of rare disease treatment in the North America region.

Recent Strategies deployed in Rare Disease Treatment Market

Partnerships, Collaboration & Agreements

Jul-2022: PTC Therapeutics partnered with Saphetor, a global precision medicine company. The partnership would be centred around supporting awareness of clinical programs for rare diseases. With this partnership, PTC would look forward to leveraging Saphetor’s new approach to bring together the global genomics community to build awareness of the rare disease programs.

Jun-2022: Pfizer partnered with Sirana Pharma, a biotechnology company. The partnership would help Pfizer in investigating the potential identification & validation of a novel treatment concept for rare and debilitating bone diseases. Furthermore, the acquisition would initially involve using Sirana’s proprietary microRNA approach, which targets substantial regenerative recovery of diseased muscle & bone tissue.

Apr-2022: Takeda entered into a partnership with Centogene, the commercial-stage essential biodata life science partner for rare and neurodegenerative diseases. Under this partnership, CENTOGENE would continue providing Takeda access to diagnostic testing for patients across the globe. This partnership would support the expansion of Takeda’s world-class enzyme replacement treatments & most essentially, bring real solutions to under-served patient communities.

Mar-2022: AbbVie joins hands with Gedeon Richter, a Hungarian multinational pharmaceutical and biotechnology company. Under the collaboration, the company focused on research, develop & commercialization of novel dopamine receptor modulators for the potential treatment of neuropsychiatric diseases. Further, through the collaboration, the companies would continue to build on the research that seeks to provide additional insights into the understanding of cariprazine's clinical pharmacology and explore novel chemistry to identify innovative dopamine receptor modulators.

Dec-2021: AstraZeneca signed an agreement with Ionis Pharmaceuticals, a biotechnology company. Under this agreement, the companies would design and commercialize eplontersen also known as IONIS-TTR-LRX. This product is developed to reduce the production of transthyretin (TTR protein) to treat hereditary as well as non-hereditary forms of TTR amyloidosis (ATTR).

Jul-2021: Pfizer teamed up with AbbVie, an American publicly traded Biopharmaceutical Company. Through the acquisition, Pfizer would carefully assess how biomedical data at this unprecedented scale is best utilized and hopefully accelerate the path from genetic discoveries to novel therapeutics.

Acquisitions & Mergers

Aug-2022: Pfizer signed an agreement to acquire GBT, a biopharmaceutical company. The acquisition would complement & improve Pfizer’s more than 30-year heritage in rare hematology and reinforce the company’s commitment to SCD by bringing expertise and a leading portfolio and pipeline with the potential to address the full spectrum of critical requirements in this underserved community.

Jun-2022: Pfizer took over ReViral, a privately held, clinical-stage Biopharmaceutical Company. The acquisition would demonstrate the company's commitment to advancing pioneering science both through in-house expertise as well as work with leading, innovative companies with the goal of delivering innovative breakthroughs to patients suffering from serious infectious diseases.

May-2022: Pfizer signed an agreement to acquire Biohaven Pharmaceutical Holding Company, a clinical-stage biopharmaceutical company. Through this acquisition, Pfizer would be exceptionally positioned to help the portfolio reach its full potential given the company's leading scale & capabilities that includes comprehensive field force engagement with Primary Care Physicians, specialists, and health systems delivering the right information at the right time.

Jun-2022: PTC Therapeutics completed the acquisition of Censa Pharmaceuticals, a biopharmaceutical company developing CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases. The acquisition would add a well-established late-stage PKU clinical program to the increased rare disease portfolio.

Dec-2021: Merck signed an agreement to acquire Chord Therapeutics, a Swiss-based biotech company. By this acquisition, Merck aimed at leveraging its existing capabilities to further develop an oral cladribine product tailored particularly for MS, oncologic disorders, gMG, and NMOSD.

Sep-2021: Merck took over Acceleron Pharma, an American clinical-stage biopharmaceutical company. The company aimed at broadening its portfolio beyond the aging cancer drug Keytruda with potential treatments that could bring in fresh revenue. The acquisition would enable Merck to access Acceleron’s rare disease drug candidate, sotatercept, which the company would expect to be a multi-billion-dollar peak sales opportunity, and would come as Keytruda moves toward the loss of market exclusivity in 2028.

Jul-2021: AstraZeneca took over Alexion Pharmaceuticals, a global biopharmaceutical company. Through this acquisition, the company would be able to enter into medicines for rare diseases and the beginning of a new chapter. Further, the acquisition improved AstraZeneca's scientific presence in immunology and, through Alexion’s new complement-biology platform and robust pipeline, would continue to pioneer the discovery & development of medicines for patients with rare diseases.

Approvals and Trials:

Apr-2022: Novartis got FDA approval for Vijoice, a kinase inhibitor that treats rare overgrowth conditions. Vijoice would be the first FDA-approved treatment for PROS, a spectrum of rare conditions characterized by overgrowths & blood vessel anomalies impacting an estimated 14 people per million. The approval would support the treatment of adult & paediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who need systemic therapy.

The market research report covers the analysis of key stake holders of the market. Key companies profiled in the report include AbbVie, Inc., F. Hoffmann-La Roche Ltd., Bayer AG, Novartis AG, Merck Group, Bristol Myers Squibb Company, Pfizer, Inc., AstraZeneca PLC, Takeda Pharmaceutical Company Limited and PTC Therapeutics, Inc.

Scope of the Study

Market Segments covered in the Report:

By Drug Type

• Biologics
• Biosimilar
• Small Molecule

By Distribution Channel

• Specialty Pharmacy
• Hospital Pharmacy
• Online Pharmacy

By Route of Administration

• Injectable
• Oral
• Others

By Therapeutic Area

• Cancer
• Cardiovascular Conditions
• Infectious Diseases
• Musculoskeletal Conditions
• Endocrine Disorders
• Metabolic Disorders
• Hematologic Disorders
• Neurological Conditions
• Others

By Geography

• North America
• US
• Canada
• Mexico
• Rest of North America
• Europe
• Germany
• UK
• France
• Russia
• Spain
• Italy
• Rest of Europe
• Asia Pacific
• China
• Japan
• India
• South Korea
• Singapore
• Malaysia
• Rest of Asia Pacific
• LAMEA
• Brazil
• Argentina
• UAE
• Saudi Arabia
• South Africa
• Nigeria
• Rest of LAMEA

Companies Profiled

• AbbVie, Inc.
• F. Hoffmann-La Roche Ltd.
• Bayer AG
• Novartis AG
• Merck Group
• Bristol Myers Squibb Company
• Pfizer, Inc.
• AstraZeneca PLC
• Takeda Pharmaceutical Company Limited
• PTC Therapeutics, Inc.

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