US Orphan Drug Market, Drug Sales, Price, Dosage and Clinical

US Orphan Drug Market, Drug Sales, Price, Dosage and Clinical

Highlights of the US Orphan Drug Market, Drug Sales, Price, Dosage and Clinical Trial Insights Report 2028:

* US Orphan Drug Market Opportunities: > USD 150 billion by 2028
* Dosage, price and treatment costs of orphan drugs: > 350 Orphan drug
* Annual and quarterly sales insights (2019 – Q1’2023): > 100 orphan drugs
* Orphan Drug Reimbursement Scenario in the US: Medicare, Medicaid, Private Insurers
* Insight into active clinical trials by company, indication and phase: 1000 orphan drugs
* Clinical insight into marketed orphan drugs by company and indication: > 400 orphan drugs
* Competitive landscape: 75 companies

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https://www.kuickresearch.com/report-us-orphan-drug-market-size

Given the increasing burden of patients suffering from rare and orphan diseases, the US FDA has taken several steps to increase the interest of research and pharmaceutical companies in these neglected diseases. One of the fundamental actions taken by the FDA is the granting of orphan drug designation, which provides benefits to developers in the context of research and development, clinical evaluation, and marketing of the drug. Orphan drug designation has been granted to more than 6,500 drugs and compounds, and approximately 100 of these have received regulatory approvals. Therefore, these drugs, although the number is lower, have been essential in improving the treatment outcomes of patients suffering from rare orphan diseases. Given the continued success of several drugs in the US pharmaceutical market, it is clear that these therapeutic products are doing well despite the small patient base, making it an interesting area to explore.

Earlier, rare diseases were neglected due to low profitability, but the implementation of the FDA’s Orphan Drug Act of 1983, subsequent regulatory reforms, government support, and extensive research by pharmaceutical companies led to exponential growth in the US orphan drug market. These favorable parameters resulted in more than 1,000 orphan drugs in clinical trials and more than 400 orphan drugs commercially available in the market. This number is expected to further surpass 500 orphan drugs in the market by 2028, driven by the increasing focus of pharmaceutical companies on rare diseases. The US orphan drug market was valued at approximately US$ 100 billion in 2022 and is expected to further surpass US$ 180 billion by 2028, making it one of the highest revenue-generating segments of the global orphan drug market.

Many drugs that have received orphan drug designation for rare indications have already received FDA approval for a different, non-orphan indication. Observational studies, anecdotal evidence, or unexpected clinical results from clinical trials in patients with unrelated indications lead researchers to conclude that the same drug can also be used for a rare indication. As a result, pharmaceutical companies often fail to realize the potential of their products as therapeutic treatments for rare diseases. On the positive side, completing clinical trials for rare diseases and then receiving orphan drug designation can help grow the patient base for the company and the drug, which will subsequently increase revenue.

For example, Eylea, which was initially approved in 2011 for the treatment of wet age-related macular degeneration, received its first orphan drug designation for the treatment of retinopathy of prematurity in February 2023 and was immediately approved for the same indication. Since 2011, Eylea has received eight approvals prior to this recent new approval, supporting the fact that many pharmaceutical companies cannot anticipate the potential of their products in orphan indications. This only emphasizes the importance of conducting clinical trials for orphan indications with underlying characteristics similar to more prevalent diseases.

It has been observed that most orphan drugs are developed by small biotech companies and then acquired by leading pharmaceutical companies. When it comes to researching under-researched diseases and conditions, smaller biotech companies have some distinct advantages. Their founders often have more knowledge and experience in conducting research and developing drugs for rare diseases. Furthermore, these companies do not have the complex bureaucracy that you see in larger companies, which simplifies decision-making. Furthermore, they have less risk of losing money if the project does not succeed. Therefore, the majority of these small biotech companies are acquired by larger companies if their therapies are successful, which significantly increases their credibility in the orphan drug market. In the same vein, larger companies have also been known to purchase orphan drug candidates in exchange for royalties and milestone payments.

For example, Amgen and Horizon Therapeutics reached a deal in December 2022 under which Amgen would buy the company and add Horizon’s orphan drugs Actimmune, Ravicti, Tepezza and Uplizna to its pipeline. While the deal has not yet closed, it gives Amgen the opportunity to expand its assembly of orphan drugs for rare diseases.

In recent years, the number of clinical candidates receiving orphan drug designation has increased dramatically, which can be attributed to the benefits offered by the FDA. Temferon, RAG-17, INB-400, Batiraxcept, and ZYIL1 are just a few candidates that will receive orphan drug designation in 2023 for various rare diseases. It is expected that more drug candidates for these indications will enter the pipeline as more companies understand the unmet needs of patients with rare diseases. The U.S. currently leads in the sales and development of orphan drugs due to the existence of numerous companies dedicated to treating rare diseases, favorable government regulations such as Medicare/Medicaid reimbursements, and the extensive network of patient advocacy organizations that work to support people with rare diseases.

KuicK research
Delhi
India

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This press release was published on openPR.