市场调查报告书
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到 2030 年孤儿药市场预测:按疾病类型、药物类型、销售、药物、配销通路和地区进行的全球分析Orphan Drugs Market Forecasts to 2030 - Global Analysis By Disease Type, Drug Type, Sale, Drug, Distribution Channel and By Geography |
根据 Stratistics MRC 的数据,2023 年全球孤儿药市场规模为 1,758 亿美元,预计到 2030 年将达到 4,455 亿美元,预测期内年复合成长率为 14.2%。
用于识别和治疗罕见疾病的药物称为孤儿药。孤儿药物可以有效治疗多种疾病,包括癌症、代谢性疾病、血液疾病、慢性疾病、进行性疾病、病毒性疾病和神经系统疾病。孤儿药通常针对罕见疾病的特定遗传或分子异常采取有针对性的方法。其结果是提供更准确和有效的治疗方案,从而改善患者的治疗结果,包括提高存活率、症状控制和整体生活品质。
根据遗传和罕见疾病(GARD)资讯中心的数据,美国已知的罕见疾病超过10,000种,影响着十分之一的人,美国有3000万人。
增加疾病知识是推动孤儿药市场的一个主要因素,因为它提高了人们对罕见疾病的认识并推动了宣传、资助和研究。此外,患者、医疗保健专业人员和倡导团体意识的提高增加了对有效治疗方法的需求,并激励製药公司资助孤儿药的研发。最终,我们将透过提供更容易获得的专业治疗方法来扩大市场并改善罕见疾病患者的生活。这还可以带来财政支持、法规激励和加强伙伴关係。
开发治疗罕见疾病的药物的过程需要广泛的研究、临床试验和法规程序,成本高昂。此外,孤儿药通常很昂贵,因为这些费用必须由药品价格来支付。它们的高成本使得难以广泛提供且价格合理的孤儿药,并可能给保险公司、医疗保健系统和患者带来挑战。因此,患者的治疗选择有限,罕见疾病患者未满足的医疗需求可能无法解决。
这些激励措施包括扩大市场独占权、税收优惠和更快的核准程序。这些激励措施透过降低财务风险和缩短上市时间来激励製药公司投资孤儿药开发。此外,此类激励措施使这些项目在财务上更加可行,从而促进创新,让更多人能够满足罕见疾病患者未满足的医疗需求,孤儿药变得可用。製药公司和罕见疾病患者都可以从双赢的局面中受益。因此,政府法规提供有利的奖励,鼓励罕见疾病治疗方法的开发,是推动孤儿药产业的主要因素。
有限的市场潜力可能会阻碍公司开拓孤儿药市场,导致罕见疾病患者的治疗选择减少,并延迟获得创新治疗方法。此外,被称为孤儿药的药物是为了治疗仅影响少数人的罕见疾病而创建的。由于受影响的患者数量较少,这些药物的市场规模比针对更常见疾病的药物要小。此外,製药公司可能会发现投资孤儿药研发在经济上并不可取,进而为市场扩张带来障碍。
COVID-19 的爆发扰乱了世界各地医疗领域的工作流程。许多行业,包括一些与医疗保健相关的子行业,因这种疾病而被迫暂时关闭。孤儿药市场也正在经历负成长,包括筛检服务减少、接触专家的机会减少、治疗中断、大多数行业的业务限制、学术机构和研究缺乏资金以及许多问题在起作用,包括暂时关闭学术机构、供应链中断以及提供所需服务和售后服务的困难。
肿瘤学领域估计占最大份额。孤儿药在肿瘤疾病的治疗中扮演重要角色。专门用于治疗仅影响少数患者的罕见疾病的药物称为孤儿药。即使个别癌症类型不是罕见疾病,癌症类型中的某些亚型或突变也可能被归类为罕见疾病。孤儿药针对导致癌症发展和扩散的特定遗传异常或分子途径。有利的法规激励措施、向精准医疗的转变以及罕见癌症亚型盛行率的增加正在推动肿瘤孤儿药物市场显着增长。
预计生技药品领域将在预测期内出现良好的成长。对于患者数量较少的罕见疾病,可以开发生物製药来针对导致疾病的潜在遗传异常或分子途径。这种集中的方法减少了可能的副作用并提高了治疗效果。单株抗体是已开发的生物製药之一。单株抗体特异性结合癌细胞上的某些蛋白质,导致免疫系统攻击肿瘤。孤儿药市场正在开发和核准用于治疗罕见疾病(包括罕见癌症)的生物製药越来越多。
由于工业基础设施的成长、可支配所得的增加以及有利的政府政策,亚太地区在预测期内占据了最大的市场占有率。此外,亚太地区的临床试验成长预计将快于美国和欧洲,有助于该地区的市场扩张。此外,政府配合措施扩大肿瘤中心和社区健康中心的数量预计将扩大开发中国家孤儿药的供应。
由于主要市场参与者和大量罕见疾病患者的存在,预计北美在预测期内将实现盈利增长。此外,孤儿药核准数量的增加、处于市场开拓阶段的孤儿药数量的增加以及该地区孤儿药支出的增加是推动北美地区孤儿药市场的其他因素。一个促成因素。
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.