Global Orphan Drugs Market Revenue, Opportunity, Segment and Key Trends 2018-2024

Our latest research report entitled Orphan Drugs Market (by disease type (oncology, gastrointestinal, pulmonary, infectious diseases), product types (biologic and non-biologic), distribution channel (hospital pharmacies, specialty pharmacies, and retail pharmacies)) provides complete and deep insights into the market dynamics and growth of Orphan Drugs. Latest information on market risks, industry chain structure Orphan Drugs cost structure and opportunities are offered in this report. The past, present and forecast market information will lead to investment feasibility by studying the essential Orphan Drugs growth factors.

The forecast Orphan Drugs Market information is based on the present market situation, growth opportunities, development factors, and opinion of the industry experts. An in-depth analysis of the company profiles, Orphan Drugs on a global and regional level and applications is conducted. The analysis of downstream buyers, sales channel, raw materials, and industry verticals is offered in this report. According to the report the global orphan drugs market is projected to grow at a CAGR of 6.5% over the forecast period of 2018-2024.

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Growing Demand of Medicines for Rare Disease Treatment to Promote the Growth of Orphan Drug Market

Economic incentives to encourage drug companies to develop and market medicines for rare disease treatment is the key driving factor in the orphan drug market. The rising occurrence of rare cancer cases, availability of orphan drugs at the hospital pharmacy and increasing hospitalization treatment for rare diseases are expected to enhance the growth of orphan drug market over the forecast period. On the other hand, the cost associated with production and R&D is more as compared with other drugs that may be hampering the market growth. Moreover, the rising awareness of the rare disease among the population and increasing investments in R&D is projected to create several opportunities in upcoming years.

North America Dominates the Market of Orphan Drugs

North America dominates the market of orphan drugs owing to its highly developed healthcare infrastructure and favorable government regulations. In North America, the U.S. holds the largest market share in this market due to economic incentives and rising awareness about the orphan drug. Moreover, the increasing investments to treat and diagnose the rare disease also to facilitate the growth in the orphan’s drug market.

New Treatments for Rare Cancers Getting Quick Approval Under ‘Orphan Drug’ Process

In August 2018, The FDA is approving drugs for rare cancers at a record pace. Most people with these diseases are happy with the news, but there are concerns about high prices. When Ann Graham was diagnosed at age 43 with osteosarcoma, a rare bone cancer that typically occurs in children and young adults, all she really heard was “cancer,” not the “rare” part. Historically, patients with rare cancers have had few effective treatment options. Many rare cancers still have no treatment options at all.

Tetra Submits Orphan Drug Applications for Cannabinoid-Based Treatment

In August 2018, Tetra Bio-Pharma has submitted multiple orphan drug applications to the FDA for cannabinoid treatment of rare cancers and ocular diseases, the company announced in a release. An orphan drug designation would allow for fast track approvals, tax credits on clinical research and reduced registration fees, the release said. Managing rare forms of cancer presents a huge challenge for physicians,” Tetra Bio-Pharma interim CEO and chief scientific officer Guy Chamberland, MSc, Ph.D., said. “Our R&D team has submitted numerous orphan drug designation applications to the FDA over the past 6 months, which not only holds promise to improve patient care but lines up with our corporate strategy to develop and commercialize cannabinoid prescription drug products for both rare cancers and ocular diseases in the United States.”

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Orphan Drug Designation Granted to Bietti's Crystalline Dystrophy Gene Therapy

In August 2018, Reflection Biotechnologies Limited’s AAV-based gene therapy, the RBIO-101 program (AAV.CYP4V2), was granted an orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of Bietti's crystalline dystrophy (BCD), a rare retinal degeneration. "Receiving orphan drug designation from the FDA is a milestone,” said Richard R. Yang, founder, and CEO of ReflectionBio, in a recent statement. “This brings hope to BCD patients and their families because BCD is a devastating blinding disease for which, currently, there is no approved treatment. BCD is estimated to affect more than 100,000 patients worldwide. As the next step, we plan to advance BCD gene therapy into a human clinical trial.”

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