市場調查報告書
商品編碼
1383503
到 2030 年孤兒藥市場預測:按疾病類型、藥物類型、銷售、藥物、配銷通路和地區進行的全球分析Orphan Drugs Market Forecasts to 2030 - Global Analysis By Disease Type, Drug Type, Sale, Drug, Distribution Channel and By Geography |
根據 Stratistics MRC 的數據,2023 年全球孤兒藥市場規模為 1,758 億美元,預計到 2030 年將達到 4,455 億美元,預測期內年複合成長率為 14.2%。
用於識別和治療罕見疾病的藥物稱為孤兒藥。孤兒藥物可以有效治療多種疾病,包括癌症、代謝性疾病、血液疾病、慢性疾病、進行性疾病、病毒性疾病和神經系統疾病。孤兒藥通常針對罕見疾病的特定遺傳或分子異常採取有針對性的方法。其結果是提供更準確和有效的治療方案,從而改善患者的治療結果,包括提高存活率、症狀控制和整體生活品質。
根據遺傳和罕見疾病(GARD)資訊中心的數據,美國已知的罕見疾病超過10,000種,影響著十分之一的人,美國有3000萬人。
增加疾病知識是推動孤兒藥市場的一個主要因素,因為它提高了人們對罕見疾病的認知並推動了宣傳、資助和研究。此外,患者、醫療保健專業人員和倡導團體意識的提高增加了對有效治療方法的需求,並激勵製藥公司資助孤兒藥的研發。最終,我們將透過提供更容易獲得的專業治療方法來擴大市場並改善罕見疾病患者的生活。這還可以帶來財政支持、法規激勵和加強夥伴關係。
開發治療罕見疾病的藥物的過程需要廣泛的研究、臨床試驗和法規程序,成本高昂。此外,孤兒藥通常很昂貴,因為這些費用必須由藥品價格來支付。它們的高成本使得難以廣泛提供且價格合理的孤兒藥,並可能給保險公司、醫療保健系統和患者帶來挑戰。因此,患者的治療選擇有限,罕見疾病患者未滿足的醫療需求可能無法解決。
這些激勵措施包括擴大市場獨佔權、稅收優惠和更快的核准程序。這些激勵措施透過降低財務風險和縮短上市時間來激勵製藥公司投資孤兒藥開發。此外,此類激勵措施使這些項目在財務上更加可行,從而促進創新,讓更多人能夠滿足罕見疾病患者未滿足的醫療需求,孤兒藥變得可用。製藥公司和罕見疾病患者都可以從雙贏的局面中受益。因此,政府法規提供有利的獎勵,鼓勵罕見疾病治療方法的開發,是推動孤兒藥產業的主要因素。
有限的市場潛力可能會阻礙公司開拓孤兒藥市場,導致罕見疾病患者的治療選擇減少,並延遲獲得創新治療方法。此外,被稱為孤兒藥的藥物是為了治療僅影響少數人的罕見疾病而創建的。由於受影響的患者數量較少,這些藥物的市場規模比針對更常見疾病的藥物要小。此外,製藥公司可能會發現投資孤兒藥研發在經濟上並不可取,進而為市場擴張帶來障礙。
COVID-19 的爆發擾亂了世界各地醫療領域的工作流程。許多行業,包括一些與醫療保健相關的子行業,因這種疾病而被迫暫時關閉。孤兒藥市場也正在經歷負成長,包括篩檢服務減少、接觸專家的機會減少、治療中斷、大多數行業的業務限制、學術機構和研究缺乏資金以及許多問題在起作用,包括暫時關閉學術機構、供應鏈中斷以及提供所需服務和售後服務的困難。
腫瘤學領域估計佔最大佔有率。孤兒藥在腫瘤疾病的治療中扮演重要角色。專門用於治療僅影響少數患者的罕見疾病的藥物稱為孤兒藥。即使個別癌症類型不是罕見疾病,癌症類型中的某些亞型或突變也可能被歸類為罕見疾病。孤兒藥針對導致癌症發展和擴散的特定遺傳異常或分子途徑。有利的法規激勵措施、向精準醫療的轉變以及罕見癌症亞型盛行率的增加正在推動腫瘤孤兒藥物市場顯著成長。
預計生技藥品領域將在預測期內出現良好的成長。對於患者數量較少的罕見疾病,可以開發生物製藥來針對導致疾病的潛在遺傳異常或分子途徑。這種集中的方法減少了可能的副作用並提高了治療效果。單株抗體是已開發的生物製藥之一。單株抗體特異性結合癌細胞上的某些蛋白質,導致免疫系統攻擊腫瘤。孤兒藥市場正在開發和核准用於治療罕見疾病(包括罕見癌症)的生物製藥越來越多。
由於工業基礎設施的成長、可支配所得的增加以及有利的政府政策,亞太地區在預測期內佔據了最大的市場佔有率。此外,亞太地區的臨床試驗成長預計將快於美國和歐洲,有助於該地區的市場擴張。此外,政府配合措施擴大腫瘤中心和社區健康中心的數量預計將擴大開發中國家孤兒藥的供應。
由於主要市場參與者和大量罕見疾病患者的存在,預計北美在預測期內將實現盈利成長。此外,孤兒藥核准數量的增加、處於市場開拓階段的孤兒藥數量的增加以及該地區孤兒藥支出的增加是推動北美地區孤兒藥市場的其他因素。一個促成因素。
According to Stratistics MRC, the Global Orphan Drugs Market is accounted for $175.8 billion in 2023 and is expected to reach $445.5 billion by 2030 growing at a CAGR of 14.2% during the forecast period. Drugs used to identify and treat uncommon medical conditions are known as orphan drugs. Numerous oncological, metabolic, hematologic, chronic, progressive, viral, and neurological illnesses can be effectively treated with them. Orphan medications often employ a targeted approach, concentrating on the particular genetic or molecular abnormalities that underlie a rare disease. This results in more precise and effective treatment options, leading to improved patient outcomes, including increased survival rates, symptom management, and overall quality of life.
According to the Genetic and Rare Diseases (GARD) Information Center, there are more than 10,000 known rare diseases in the U.S. that affect about 1 in 10 people or 30 million people in the U.S.
Growing knowledge of diseases is a major factor driving the market for orphan medications since it makes rare diseases better recognized, which encourages advocacy, funding, and research. Moreover, an increase in patient, healthcare professional and advocacy group awareness drives up the need for efficient therapies, which in turn motivates pharmaceutical corporations to fund research and development of orphan drugs. In the end, it expands the market and enhances the lives of people with rare diseases by making specialized therapies more accessible. Therefore, it also results in increased support for funding, regulatory incentives, and partnerships.
The process of creating medications for uncommon diseases can be costly due to the extensive research, clinical trials, and regulatory procedures involved. Additionally, orphan medications are generally costly since these expenses must be covered by drug prices. It can be difficult to deliver rare disease therapies that are both widely available and reasonably priced due to the high expenses, which can also pose access challenges for insurers, healthcare systems, and patients. Hence, this can leave patients with limited treatment options and hinder progress in addressing the unmet medical needs of individuals with rare diseases.
Extended market exclusivity, tax rebates, and accelerated approval procedures are all included in these incentives. They incentivize pharmaceutical companies to spend money on the development of orphan drugs by lowering financial risks and accelerating time to market. Furthermore, these kinds of incentives make these ventures more financially viable, which in turn encourage innovation and make more orphan medications available to meet the unmet medical requirements of people with rare diseases. Pharmaceutical businesses and those with rare illnesses benefit from this in a win-win scenario. Hence, government regulations that provide favourable incentives to encourage the development of therapies for rare diseases are a major factor driving the orphan drug industry.
The limited market potential can discourage companies from pursuing orphan drug development, resulting in fewer treatment options for patients with rare diseases and potential delays in accessing innovative therapies. Moreover, drugs known as orphans are created to treat uncommon conditions that only a tiny number of people have. The market for these medications is less than that of medications for more prevalent disorders because of the small number of patients. Furthermore, pharmaceutical corporations may find it financially undesirable to invest in the research and development of orphan medications as a result, which is a drawback of the market's expansion.
The outbreak of COVID-19 has disrupted workflows in the health care sector around the world. Numerous industries, including some healthcare-related sub-sectors, have been forced to temporarily close their doors due to the disease. The market for orphan drugs has also experienced negative growth, which can be linked to a number of issues, including a drop in screening services, a decrease in access to specialists, treatment interruptions, restricted operations in the majority of industries, insufficient funding for academic institutions and research, the temporary closure of significant academic institutions, supply chain disruptions, and difficulties delivering necessary or post-sales services.
The oncological diseases segment is estimated to hold the largest share. Orphan drugs play a crucial role in the treatment of oncological diseases. A medication created especially to treat uncommon diseases that only affect a small number of patients is known as an orphan drug. Certain subtypes or mutations within a cancer type can be classified as rare diseases, even though individual cancer types might not be. These orphan medications target particular genetic defects or molecular pathways that contribute to the development and spread of cancer. Due to favourable regulatory incentives, a shift towards precision medicine, and the rising prevalence of rare cancer subtypes, the market for orphan drugs for oncological diseases has experienced significant growth.
The biologics segment is anticipated to have lucrative growth during the forecast period. Biologics can be created to target the underlying genetic abnormalities or molecular pathways that cause the disease in the case of orphan diseases, where the patient population is small. This focused approach lowers possible side effects and improves treatment efficacy. One sort of biologic that has been developed is the monoclonal antibody. It binds specifically to certain proteins on cancer cells, causing the immune system to attack the tumor. The orphan drug market has witnessed a growing number of biologic drugs being developed and approved for rare diseases, including rare forms of cancer.
Asia Pacific commanded the largest market share during the extrapolated period owing to the growing infrastructure of industries, rising disposable incomes, and favourable government policies in the region. Furthermore, clinical trial growth in Asia-Pacific is anticipated to accelerate beyond that of the United States and Europe, contributing to the region's market expansion. Moreover, it is anticipated that an increase in government initiatives to expand the number of oncology and community health centers will broaden the availability of orphan medications in developing nations.
North America is expected to witness profitable growth over the projection period, due to the presence of major market players and a large population of patients with orphan diseases. Additionally, a growing number of orphan product approvals, a sizable number of orphan drugs in the development stage, and rising orphan drug spending in the area are the other factors driving the orphan drug market in the North American region.
Some of the key players in the Orphan Drugs Market include: Bayer AG, Novartis AG, F. Hoffmann-La Roche Ltd, Bristol-Myers Squibb Company, Amgen Inc., Novo Nordisk A/S, Alexion Pharmaceuticals Inc., GlaxoSmithKline plc, AstraZeneca, Celgene Corporation, Mylan, Biogen, Merck KGaA, Takeda Pharmaceutical Company Limited, Eli Lilly and Company and Shire Pharmaceutical.
In March 2023, F. Hoffmann-La Roche Ltd: Roche announces collaboration with Lilly to enhance early diagnosis of Alzheimer's disease.
In January 2022, Amgen and Generate Biomedicines announced a research collaboration agreement to discover and create protein therapeutics for five clinical targets across several therapeutic areas and multiple modalities.
Note: Tables for North America, Europe, APAC, South America, and Middle East & Africa Regions are also represented in the same manner as above.