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罕見疾病治療市場 - 2018-2028 年全球產業規模、佔有率、趨勢、機會和預測，按治療領域、給藥途徑、藥物類型、配銷通路、地區、競爭細分。

市場調查報告書

商品編碼

1364027

罕見疾病治療市場 - 2018-2028 年全球產業規模、佔有率、趨勢、機會和預測，按治療領域、給藥途徑、藥物類型、配銷通路、地區、競爭細分。

Rare Disease Therapeutics Market, 2028- Global Industry Size, Share, Trends, Opportunity, and Forecast, 2018-2028 Segmented By Therapeutic Area, By Route of Administration, By Drug Type, By Distribution Channel, By Region, By Competition.

出版日期: 2023年10月03日 | 出版商:

TechSci Research | 英文 190 Pages | 商品交期: 2-3個工作天內

價格

簡介目錄

2022年全球罕見疾病治療市場價值為986.1億美元，預計到2028年年複合成長率將達到10.42%。全球罕見疾病治療市場是醫療保健領域一個充滿活力且日益重要的領域。罕見疾病也稱為孤兒病，其特徵是在人群中患病率較低，影響的人數有限。儘管罕見疾病很罕見，但總的來說，罕見疾病影響著全世界數百萬人。這個市場的定義是專注於為這些經常改變生命和危及生命的疾病開發和提供專門的治療方法。

該市場成長的關鍵驅動力之一是基因研究和理解的進步。隨著科學家深入研究罕見疾病的遺傳基礎，他們發現了創新療法的潛在目標，包括基因療法和精準醫學方法。這些突破開闢了治療發展的新途徑，並為患有以前無法治療的罕見疾病的患者帶來了新的希望。監管激勵措施，例如美國的《孤兒藥法案》和其他地區的類似法律，在刺激罕見疾病治療投資方面發揮了關鍵作用。這些激勵措施為專注於開發罕見疾病治療方法的製藥公司和研究人員提供了財務和監管優勢，使其成為更具吸引力的事業。此外，在患者權益團體和醫療保健組織的推動下，人們對罕見疾病的認知不斷提高，人們更加認知到該領域未滿足的醫療需求。這種意識的提高導致更多的資源被用於罕見疾病的研究和治療發展。

專門針對罕見疾病設計的標靶治療是市場成長的另一個驅動力。這些療法是根據疾病獨特的遺傳和分子特徵量身定做的，與傳統療法相比，有望提高療效並減少副作用。隨著更多罕見疾病病例的發現和診斷，患者群體的擴大進一步放大了市場的重要性。此外，以 CRISPR-Cas9 等創新方法為特徵的基因治療進步有可能透過直接解決罕見疾病的遺傳原因來徹底改變罕見疾病的治療方法。

市場概況
預測期	2024-2028
2022 年市場規模	986.1億美元
2028 年市場規模	1768.3億美元
2023-2028 年年複合成長率	10.42%
成長最快的細分市場	血液系統疾病
最大的市場	北美洲

學術界和工業界之間的合作以及全球醫療保健計劃的參與正在創造一個加速罕見疾病治療發展的協作環境。在科學進步、監管支持以及對滿足罕見疾病患者需求的日益承諾的推動下，罕見疾病治療市場不斷發展和擴大，為受這些疾病影響的人們帶來了新的希望並提高了生活品質。

主要市場促進因素

罕見疾病患者數量不斷增加

醫學界有很多罕見疾病。有些是眾所周知的，例如囊性纖維化或貓眼綜合症，而有些則比較模糊。大多數癌症都很罕見，有罕見的大腦和神經系統疾病、代謝疾病、染色體失調、皮膚、骨骼和骨骼疾病，以及影響心臟、血管、肺、腎臟和其他器官和系統的疾病。許多罕見疾病是以首次診斷的醫生命名的，而另一些則以患者甚至首次診斷的醫院命名。

政府和市場參與者採取的舉措

市場的成長預計將得到促進產品開發的政府法規的支持，例如《孤兒藥法》，該法將孤兒藥的分類分配給製藥公司開發的候選藥物。該計劃透過研究和開發的進步促進孤兒疾病的治療。

此外，2021年，藥物評估與研究中心（CDER）批准了約26種治療罕見疾病的新孤兒藥。其中一些新的孤兒藥包括 Lumakras、Scelendrix、Welireg、Amondys、Cytalux、Besremi、Empavelli、Evkeeza、Exkivity、fexinodmaze 和 Zymonta。

國家罕見疾病政策 (NPRD) 於 2020 年 4 月針對受大流行影響的罕見疾病患者啟動了 COVID-19「緊急救濟」計畫。根據該計劃，NPRD 每年向孤兒疾病患者和監護人提供高達 1,000 美元的經濟援助，以滿足他們的非醫療需求。這些計劃預計將在未來幾年增加罕見疾病治療的採用。

印度政府的「2021年罕見疾病」國家政策旨在透過綜合全面的預防策略來減少罕見疾病的盛行率和發病率。這包括提高認知、婚前、婚後、懷孕前和懷孕後篩檢和諮詢計劃，以防止出生患有罕見疾病的兒童，並在資源有限和相互衝突的醫療保健優先事項的範圍內，提供為有資格接受拋棄式治療或相對便宜的治療的罕見疾病患者提供負擔得起的醫療服務。

2020 年，美國 FDA 批准了治療膀胱功能障礙疾病（稱為神經源性逼尿肌過度活動）的藥物，這種疾病在脊髓損傷和多發性硬化症患者中觀察到。

2022年5月，美國食品藥物管理局（FDA）制定了一項名為「加速罕見疾病治癒」（ARC）計畫的新計劃，旨在加速罕見疾病新療法的開發，該計畫由藥物評估和研究中心管理。研究（CDER）。

2022 年5 月，歐洲藥品管理局(EMA) 授予Xenpozyme (olipudasealfa) 上市許可，該藥物用於治療非中樞神經系統(NCS)、酸性鞘磷脂酶缺乏症(ASMD) 的表現，這是一種罕見的退化性遺傳疾病紊亂。

治療罕見疾病的非生物藥物的需求不斷增加

用於罕見疾病的非生物藥物是透過化學合成或透過常規製藥製程生產的治療劑，而不是從生物來源獲得的。這些藥物通常被稱為小分子藥物，是醫學上最廣泛使用和認可的藥物類別。

由於與此類產品相關的免疫原性和最小的副作用，非生物製劑藥物領域預計在預測期內將以令人印象深刻的速度成長。這些藥物被認為可有效治療某些病症，包括 APDS（活化磷酸肌醇 3-激酶 δ 症候群）和 Felty 症候群。這些因素加上臨床研究的成本效益和非複雜性，預計將增加罕見疾病患者對非生物藥物的消費。

非生物醫藥產品通常由具有明確化學結構的分子組成。這些分子通常在實驗室中以化學反應合成。大多數非生物藥物都是口服給藥，即以片劑、膠囊或液體形式口服。對患者來說，這是一種方便且可自行給藥的給藥途徑。

非生物藥物具有解決廣泛的分子和細胞途徑的潛力，可用於治療各種醫療狀況，例如罕見疾病。眾所周知，非生物藥物非常穩定，可以在室溫下保存，無需冷藏。此外，這些藥物的生產過程成熟，更適合大規模生產。

一旦非生物藥物的專利保護到期，其他製藥公司就可以生產和銷售該藥物的學名藥，從而提高其可用性和可負擔性。

小分子藥物能夠充當疾病過程中涉及的酵素的抑制劑。例如，伊馬替尼用於治療某些形式的白血病和胃腸道間質瘤。合成激素也用於治療罕見的內分泌疾病，例如在左甲狀腺素的幫助下治療甲狀腺功能減退症，在氫化可的鬆的幫助下治療腎上腺功能不全。

某些小分子藥物具有調節免疫系統反應的潛力，因此可用於治療罕見的自體免疫疾病。

中樞神經系統疾病負擔的增加可以推動罕見疾病精準治療的發展

隨著中樞神經系統 (CNS) 罕見疾病的發生率和盛行率的增加，人們越來越需要更了解這些疾病的遺傳、分子和生化基礎。遺傳學和神經科學使研究人員能夠更好地了解罕見中樞神經系統疾病的複雜性，從而識別誘發疾病的突變和途徑。

鑑於影響脊髓和大腦神經細胞、導致肌肉控制喪失的疾病盛行率不斷上升，預計中樞神經系統罕見疾病治療市場價值將在預測期內加速成長。根據國家醫學圖書館的資料，全球每 10 萬人中約有 5 人患有肌萎縮性側索硬化症。此外，對精準醫學發展的強烈關注將進一步推動對治療罕見疾病的療法的需求。

精準醫學是一種根據每位患者的獨特需求，考慮遺傳、生活方式和環境等因素而量身定做的醫療形式。在中樞神經系統（CNS）罕見疾病中，每種疾病都是由不同的基因突變引起的，因此標靶治療變得越來越可行。了解患者疾病的基因組成使臨床醫生能夠設計針對特定分子缺陷的標靶療法。

精準醫學嚴重依賴生物標記。這些是可以透過測量來顯示疾病的存在或進展的東西。開發針對中樞神經系統罕見疾病的精準治療方法通常涉及尋找和測試與某些疾病相關的生物標記。這些生物標記可以幫助早期診斷、追蹤疾病進展並衡量治療效果。

由於罕見中樞神經系統疾病的治療選擇有限，精準醫學方法涉及檢查針對其他疾病開發的藥物治療罕見疾病的潛力。透過闡明與罕見疾病和其他常見疾病相關的分子途徑，研究人員或許能夠辨識出可能治療罕見中樞神經系統疾病的藥物。

中樞神經系統（CNS）罕見疾病的流行促使研究人員和藥物製造商更加關注這些疾病。精準醫學技術的發展使得對罕見疾病進行針對性研究變得更加簡單，從而加速了藥物的開發和整體市場的成長。

主要市場挑戰

精準診斷

找到診斷是成功治療的關鍵，在某些情況下也是參與臨床試驗的關鍵。然而，對罕見疾病進行準確、及時的診斷對於患者來說可能具有挑戰性，在某些情況下，準確的診斷可能需要長達五年的時間。

在極少數情況下，患者可能會出現醫療專業人員可能無法識別與疾病相關或由疾病引起的徵兆和症狀。在這種情況下，可以進行廣泛的基因測試來識別可能與疾病表現相關的遺傳變異。確保將患者分配給熟悉與解釋遺傳資料相關的困難的臨床遺傳學家或/或醫生總是有益的。

患者可用性和招募

招募患者參與孤兒藥領域的臨床試驗是一項具有挑戰性的任務。不僅確定目標患者群體具有挑戰性，而且找到專門治療這些患者的研究人員也具有挑戰性。這些困難可能會帶來招募風險，並且需要採用客製化的可行性方法來降低每項罕見疾病研究的風險。

由於患者群體可能在地理上分佈廣泛，因此患者可能需要長途跋涉才能到達孤兒病臨床試驗的研究地點。雖然差旅報銷可能會降低這種風險，但組織還應該探索新穎的解決方案和技術，以在家中收集資料並最大程度地減少現場訪問。

端點選擇

在設計針對罕見疾病的治療方法時，重要的是要確保臨床研究終點能夠衡量藥物如何改變患者疾病的進展。為了區分藥物效應和標準疾病進展，臨床研究和終點設計必須充分反映監管要求和付款人考慮因素。

建議申辦者與監管機構合作，在臨床研究之前確定療效終點。美國食品藥物管理局組織針對特定疾病的論壇，以識別和討論從患者或照護者的角度來看相關的終點；這些端點可能包括經過驗證的患者報告的結果，其中包括患者的聲音。罕見疾病通常需要多個地區多個地點的參與

主要市場趨勢

不斷成長的產品創新

對罕見疾病的需求不斷成長，導致了針對該疾病的各種療法的開發。例如，2023 年 2 月，賽諾菲計劃於今年初或明年在印度推出兩種治療罕見疾病的藥物。該公司已獲得主題專家委員會的推薦，在印度進口並投放兩種新產品：用於治療龐貝病的 Nexviazyme（Aveloglucosidase Alfa 粉末）和用於治療 Niemann 病的 Xenpozyme 和 Olipudase Alfa 粉末匹克病（ASMD）。根據公司說明，這兩種產品都具有孤兒藥地位，並在美國、澳洲、歐盟、英國和日本等多個國家獲得批准。此外，該公司還享有後期臨床試驗（III期和IV期）的豁免。

精準醫學 (PPM)、巨量資料和基因組學的進步

沒有跡象表明罕見疾病領域未來的受歡迎程度會下降，因為目前的藥物開發模式非常適合新興的醫學領域。個人化精準醫學（PPM）、巨量資料和基因組學的發展使得具有挑戰性的疾病診斷和治療形式的靶向性得到顯著改善，同時對病理遺傳基礎的更好理解使得罕見疾病的描述和靶向成為可能。疾病

監管遊戲規則改變者

1983年美國《孤兒藥法案》的頒布被廣泛認為是罕見疾病藥物開發史上的轉捩點。該立法旨在透過向製藥公司提供激勵措施來解決市場失靈問題，否則這些製藥公司將不願意投資可能只使少數患者受益的藥物。據藥品貿易組織(PhRMA) 稱，自《孤兒藥法案》實施以來，已有600 多種孤兒藥獲得FDA 批准，但只有5% 的罕見疾病有FDA 批准的治療方法，這意味著未來幾年仍有大量未開發的成長潛力。例如，2022年，FDA宣布啟動新的加速罕見疾病治癒（ARC）計畫。

公司啟動計劃計劃

公司正在採取各種措施來幫助患有罕見疾病的人。例如，2022 年，Horizon 為患有罕見疾病的成年人和青少年推出了新的支持計劃。成為罕見疾病患者可能充滿挑戰且孤獨。為了緩解美國大約 500-600 名囊性纖維化患者的痛苦，Horizon Therapeutic 發起了兩項不同的措施。此外，Horizon Therapeutics 在2021 年還宣布推出每月一次的虛擬講故事體驗#RAREis，以突出患有罕見疾病的兒童社區的聲音和故事，並提高人們對Make-A- Wish Mid-Atlantic和Make-A- Wish 的認知並籌集資金。- 願望加拿大。這個照相亭是地平線全球使命的一部分，即支持患有改變生活的罕見疾病的兒童的願望，並在困難時期給他們帶來微笑。此外，為了支持罕見疾病社區，Horizon 位於伊利諾州迪爾菲爾德的美國總部和位於愛爾蘭都柏林的全球總部點亮了紫色、粉紅色、綠色和藍色，這是罕見病日的官方顏色。

細分市場洞察

治療領域見解

血液疾病涵蓋影響血液和相關組織的多種疾病，由於幾個令人信服的原因，它在全球罕見疾病治療市場中佔據主導地位。首先，這些疾病在全球罕見疾病中佔很大比例，使其成為市場的重要組成部分。例子包括鐮狀細胞性貧血、血友病和各種罕見的血液疾病。

其次，研究和治療開發的進步推動了血液學罕見疾病新療法的出現。特別是基因治療和精準醫學方法在解決以前無法治療或管理不善的疾病方面已顯示出有希望的結果。這項治療創新吸引了製藥公司和研究人員的大量投資和興趣。此外，血液學罕見疾病往往對患者的生活品質和壽命產生重大影響，推動醫學和社會迫切需要有效的治療。這種緊迫感促進了醫療保健提供者、研究人員和倡導團體之間的合作，進一步加速了治療發展。

最後，監管機構認知到血液學罕見疾病未被滿足的醫療需求，已經實施了快速核准途徑，以促進更快地獲得治療。這些因素共同使血液學罕見疾病成為全球罕見疾病治療市場的主導力量，推動研究、創新和改善患者治療結果。

管理途徑見解

由於幾個令人信服的原因，注射劑在全球罕見疾病治療市場中佔據主導地位。首先，許多罕見疾病具有複雜的病理生理學特徵，需要精確和受控的藥物輸送，這使得注射製劑成為最佳選擇。這種給藥途徑可確保藥物快速到達血液，從而產生更直接的治療效果，這對於治療嚴重或危及生命的罕見疾病至關重要。

其次，在口服或其他給藥方法因吸收不良、胃腸道併發症或需要高生物利用度等問題而效果較差的情況下，注射劑通常是首選。在罕見疾病的治療選擇有限的情況下，注射劑提供了一種可靠的方法，可以將治療直接輸送到血液中，從而最佳化其療效。此外，監管機構已經認知到注射劑在治療罕見疾病中的重要性，並加快了創新療法的批准。這鼓勵製藥公司和研究人員專注於開發罕見疾病的注射療法，推動該領域的進一步進步和投資。

最後，注射劑可實現的高特異性和標靶遞送使其成為治療的首選，包括酵素替代療法和基因療法，這對於治療許多罕見疾病至關重要。因此，注射繼續主導全球罕見疾病治療市場，為患有這些具有挑戰性的疾病的患者帶來希望並改善治療結果。

區域洞察

由於幾個令人信服的原因，北美在全球罕見疾病治療市場中佔據主導地位。首先，該地區受益於強大的醫療基礎設施和先進的研究設施，為罕見疾病治療的開發和採用奠定了肥沃的土壤。北美擁有大量致力於解決罕見疾病的著名醫療機構、製藥公司和研究機構。

其次，該地區對創新的承諾體現在對研發的大量投資。北美的政府機構、私人基金會和製藥公司為罕見疾病研究和治療開發分配了大量資金，激勵尖端治療方法的發展。此外，北美擁有龐大的患者群體，這使其成為製藥公司投資罕見疾病療法的一個有吸引力的市場。例如，美國的《孤兒藥法案》激勵藥物開發商專注於罕見疾病，進一步刺激研究和商業化工作。

此外，北美的監管環境有利於罕見疾病治療的開發，加速核准流程和孤兒藥指定有助於更快獲得創新療法。這些因素加上醫療創新和卓越研究的文化，鞏固了北美在全球罕見疾病治療市場的主導地位。

主要市場參與者

諾華公司
阿斯特捷利康公司
輝瑞公司
賽諾菲公司
艾伯維公司
百時美施貴寶公司
拜耳公司
F 霍夫曼-拉羅氏有限公司
安進公司
衛材有限公司
諾和諾德公司

報告範圍：

在本報告中，除了以下詳細介紹的產業趨勢外，全球罕見疾病治療市場也分為以下幾類：

罕見疾病治療市場，依治療領域：

血液系統疾病
癌症
傳染性疾病
心血管疾病
代謝性疾病
內分泌疾病
肌肉骨骼疾病
其他

罕見疾病治療市場，依給藥途徑分類：

可注射
口服
其他

罕見疾病治療市場，依藥物類型：

生物製劑
生物相似藥
小分子

罕見疾病治療市場，依配銷通路：

專業藥房
醫院藥房
網路藥局

罕見疾病治療市場，依地區：

北美洲
- 美國
- 加拿大
- 墨西哥
歐洲
- 法國
- 英國
- 義大利
- 德國
- 西班牙
亞太
- 中國
- 印度
- 日本
- 澳洲
- 韓國
南美洲
- 巴西
- 阿根廷
- 哥倫比亞
中東和非洲
- 南非
- 沙烏地阿拉伯
- 阿拉伯聯合大公國

競爭格局

公司概況：罕見疾病治療市場主要公司的詳細分析。

可用的客製化：

全球罕見疾病治療市場報告根據給定的市場資料，技術科學研究根據公司的具體需求提供客製化服務。該報告可以使用以下自訂選項：

公司資訊

其他市場參與者（最多五個）的詳細分析和概況分析。

市場規模及預測
- 按價值
市佔率及預測
- 依治療領域（血液疾病、癌症、傳染病、心血管疾病、代謝疾病、內分泌疾病、肌肉骨骼疾病、其他）
- 依給藥途徑（注射、口服、其他）
- 依藥物類型（生物製劑、生物相似藥、小分子）
- 依配銷通路（專科藥房、醫院藥房、網路藥房）
- 按地區
- 按公司分類（前 5 名市場參與者的股份）
市場地圖
- 按治療領域
- 依給藥途徑
- 依藥物類型
- 按配銷通路
- 按地區

第 6 章：北美罕見疾病治療市場展望

市場規模及預測
- 按價值
市佔率及預測
- 按治療領域
- 依給藥途徑
- 依藥物類型
- 按配銷通路
- 按國家/地區

第 7 章：歐洲罕見疾病治療市場展望

市場規模及預測
- 按價值
市佔率及預測
- 按治療領域
- 依給藥途徑
- 依藥物類型
- 按配銷通路
- 按國家/地區

第 8 章：亞太地區罕見疾病治療市場展望

市場規模及預測
- 按價值
市佔率及預測
- 按治療領域
- 依給藥途徑
- 依藥物類型
- 按配銷通路
- 按國家/地區

第 9 章：南美洲罕見疾病治療市場展望

市場規模及預測
- 按價值
市佔率及預測
- 按治療領域
- 依給藥途徑
- 依藥物類型
- 按配銷通路
- 按國家/地區

第 10 章：中東和非洲罕見疾病治療市場展望

市場規模及預測
- 按價值
市佔率及預測
- 按治療領域
- 依給藥途徑
- 依藥物類型
- 按配銷通路
- 按國家/地區

第 11 章：市場動態

動力
挑戰

第 12 章：市場趨勢與發展

近期發展
併購
技術發布

第13章：競爭格局

諾華公司
- Business Overview
- Treatment Offerings
- Recent Developments
- Key Personnel
- SWOT Analysis
阿斯特捷利康公司
輝瑞公司
賽諾菲公司
艾伯維公司
百時美施貴寶公司
拜耳公司
F霍夫曼-拉羅氏有限公司
安進公司
衛材有限公司
諾和諾德公司

第 14 章：策略建議

第 15 章：關於我們與免責聲明

簡介目錄

Product Code: 7609

Global Rare Disease Therapeutics Market has valued at USD 98.61 billion in 2022 and is anticipated to reach a CAGR of 10.42% through 2028. The global rare disease therapeutic market is a dynamic and increasingly significant sector in the realm of healthcare. Rare diseases, also known as orphan diseases, are characterized by their low prevalence in the population, affecting a limited number of individuals. Despite their rarity, collectively, rare diseases impact millions of people worldwide. This market is defined by its focus on developing and providing specialized treatments for these often life-altering and life-threatening conditions.

One of the key drivers of this market's growth is the advances in genetic research and understanding. As scientists delve deeper into the genetic underpinnings of rare diseases, they uncover potential targets for innovative therapies, including gene therapies and precision medicine approaches. These breakthroughs have opened new avenues for therapeutic development and offer renewed hope for patients with previously untreatable rare diseases. Regulatory incentives, such as the Orphan Drug Act in the United States and equivalent laws in other regions, have played a pivotal role in spurring investment in rare disease therapeutics. These incentives provide financial and regulatory advantages to pharmaceutical companies and researchers focused on developing treatments for rare diseases, making it a more attractive endeavor. Furthermore, increased awareness of rare diseases, driven by patient advocacy groups and healthcare organizations, has led to greater recognition of the unmet medical needs within this space. This heightened awareness has resulted in more resources being directed toward rare disease research and treatment development.

Targeted therapies designed specifically for rare diseases are another driving force behind market growth. These therapies are tailored to the unique genetic and molecular characteristics of the disease, promising improved efficacy and fewer side effects compared to traditional treatments. Expanding patient populations, as more rare disease cases are identified and diagnosed, further amplify the market's significance. Moreover, gene therapy advancements, characterized by innovative approaches like CRISPR-Cas9, have the potential to revolutionize rare disease treatment by directly addressing the genetic causes of these conditions.

Market Overview
Forecast Period	2024-2028
Market Size 2022	USD 98.61 Billion
Market Size 2028	USD 176.83 Billion
CAGR 2023-2028	10.42%
Fastest Growing Segment	Hematologic Diseases
Largest Market	North America

Collaborations between academia and industry, as well as the involvement of global healthcare initiatives, are fostering a collaborative environment that accelerates rare disease therapeutic development. The market for rare disease therapeutics, driven by scientific progress, regulatory support, and a growing commitment to addressing the needs of rare disease patients, continues to evolve and expand, offering renewed hope and improved quality of life for those affected by these conditions.

Key Market Drivers

Rising Number of Patients with Rare Diseases

There are a lot of rare diseases in the medical world. Some are well-known, like cystic fibrosis or cat eye syndrome, while others are more obscure. Most cancers are rare and there are rare diseases of the brain and nervous system, metabolic diseases, chromosome disorders, skin, bone and skeletal, and diseases that affect the heart, blood vessels, lungs, kidneys and other organs and systems. A lot of rare diseases are named after the doctors who first diagnosed them, while others are named after patients or even the hospitals where they were first diagnosed.

An article published in the Journal of Rare Disorders in 2020 estimates that approximately 7,000 rare diseases have been diagnosed, with approximately 70% of them having no treatment available. As a result, research into unmet need of rare disease treatments will ensure that the market continues to expand in the future.

It is estimated that 6% to 8% of the population of the European Union is afflicted with a rare disease, as reported by EURORDIS. Furthermore, EURORDIS states that between 50-60% of rare diseases begin in infancy, and some of these conditions can be fatal. Consequently, it is expected that in the coming years, the demand for certain therapeutic drugs will rise significantly due to the growing pediatric population affected by rare diseases.

Initiatives taken by Government and Market Players

The growth of the market is expected to be supported by government regulations that promote the development of products, such as the orphan drug law, which allocates the classification of an orphan drug to drug candidates developed by pharmaceutical companies. This program facilitates the treatment of orphan diseases through the advancement of research and development.

Additionally, in 2021, Center for Drug Evaluation and Research (CDER) approved about 26 new orphan drugs to treat rare diseases. Some of these new orphan drugs include Lumakras, Scelendrix, Welireg, Amondys, Cytalux, Besremi, Empavelli, Evkeeza, Exkivity, fexinodazole, and Zymonta.

The COVID-19 "Critical Relief" program was launched in April 2020 by the National Policy for rare disorders (NPRD) for patients with rare diseases affected by the pandemic. Under this program, the NPRD provided orphan disease patients and guardians with financial assistance of up to USD 1,000 per year to cover their non-medical needs. These programs are expected to increase the adoption of rare disease treatment in the coming years.

The Indian government's National Policy on 'Rare Diseases 2021' was designed to reduce the prevalence and incidence of rare diseases through an integrated and comprehensive prevention strategy. This includes awareness generation, pre-marriage, post-marriage, pre-pregnancy, and post-pregnancy screening and counselling programs to prevent the birth of children with rare conditions, and, within the limitations of resources and conflicting health care priorities, to provide affordable health care to those with rare diseases who are eligible for one-time treatments or relatively inexpensive therapy.

In 2020, the U.S. FDA approved drugs to treat a disease with bladder dysfunction called neurogenic detrusor overactivity, observed in patients with spinal cord injury and multiple sclerosis.

In May 2022, the U.S. Food and Drug Administration (FDA) created a new program to accelerate the development of new treatments for rare diseases called the "Accelerating Rare Disease Cures" (ARC) Program, which is managed by the Center for Drug Evaluation and Research (CDER).

In May 2022, the European Medicines Agency (EMA) granted a marketing authorization for Xenpozyme (olipudasealfa), a therapy for the treatment of Non-Central Nervous System (NCS), manifestations of Acid Sphingomyelinase Deficiency (ASMD), a rare and degenerative genetic disorder.

Increasing Demand of Non-Biological Drugs to Treat Rare Diseases

Non-biologic drugs for rare diseases are therapeutic agents that have been chemically synthesized or produced through conventional pharmaceutical processes, rather than being obtained from biological sources. These drugs are commonly referred to as small molecule drugs and are the most widely used and recognized drug class in medicine.

Non-biologics drug segment is expected to grow at an impressive rate during the forecast period, on account of the immunogenicity and minimal side effects associated with such products. These drugs are deemed effective for treating certain conditions including APDS (activated phosphoinositide 3-kinase delta syndrome) and Felty Syndrome. These factors, alongside cost-effectiveness and non-complexity in clinical studies, are projected to augment the consumption of non-biologic medications among rare disease patients.

Non-biological medicinal products are generally composed of molecules with well-characterized chemical structure. These molecules are usually synthesized in the laboratory through chemical reactions. Most non-biological medications are orally administered, i.e., taken by mouth in tablet, capsule, or liquid form. This is a convenient and self-administered route of administration for patients.

Non-biologic drugs have the potential to address a broad range of molecular and cellular pathways and are employed in the treatment of a variety of medical conditions, such as rare diseases. Non-biological drugs are known to be highly stable and can be kept at room temperature without the need for refrigeration. Furthermore, the manufacturing process for these drugs is well-established, making them more suitable for large-scale production.

Once the patent protection of a non-biologic drug has expired, other pharmaceutical companies can produce and sell generic versions of the drug, thus increasing its availability and affordability.

Small molecule drugs are capable of functioning as inhibitors of enzymes that are involved in the process of disease. For example, imatinib is used to treat certain forms of leukemia and gastroenterological stromal tumors. Synthetic hormones are also used to treat rare endocrine disorders, such as hypothyroidism, with the aid of levothyroxine, and adrenal insufficiencies with the aid of hydrocortisone.

Certain small molecule drugs have the potential to modulate the immune system response, thus making them useful for the treatment of rare autoimmune conditions.

Growing Burden of CNS disorders can Drive the Development of Precision Treatments for Rare Diseases

As the incidence and prevalence of rare diseases in the central nervous system (CNS) increase, there is an amplified need to gain a better understanding of the genetic, molecular, and biochemical substrates of these diseases. Genetics and neuroscience have enabled researchers to gain a greater understanding of the complexities of rare CNS disorders, allowing for the identification of disease-inducing mutations and pathways.

Rare disease treatment market value from the central nervous system is expected to accelerate in the forecast period, given the rising prevalence of disorders that affect nerve cells in the spinal cord and brain, causing loss of muscle control. Based on data from the National Library of Medicine, amyotrophic lateral sclerosis affects around 5 in 100,000 people worldwide. Moreover, the strong focus on the development of precision medicine will further drive the demand for therapies to treat rare diseases.

Precision medicine is a form of medical treatment that is tailored to the unique needs of each individual patient, considering factors such as genetics, lifestyle, and environment. In rare diseases of the central nervous system (CNS), where each condition is caused by a distinct genetic mutation, targeted therapies are becoming increasingly feasible. Understanding the genetic makeup of a patient's disorder allows clinicians to design targeted therapies that address specific molecular defects.

Precision medicine is heavily reliant on biomarkers. These are things that can be measured to show the presence or progress of a disease. Developing precision treatments for rare diseases of the central nervous system often involves finding and testing biomarkers related to certain disorders. These biomarkers can help early diagnosis, track disease progress, and measure how well treatment is working.

As treatment options for rare CNS diseases are limited, precision medicine approaches involve examining the potential of drugs that have been developed for other conditions to treat a rare disease. By elucidating the molecular pathways associated with both the rare condition and other common conditions, researchers may be able to identify drugs that could potentially treat the rare CNS disorder.

The prevalence of rare diseases in the central nervous system (CNS) has prompted researchers and pharmaceutical manufacturers to pay greater attention to these conditions. The development of precision medicine techniques has made it simpler to conduct specific research on rare diseases, thus accelerating the development of drugs and overall market growth.

Key Market Challenges

Accurate Diagnosis

Finding a diagnosis is the key to successful treatment and, in some cases, clinical trial involvement. However, accurate and timely diagnosis of a rare condition can be challenging for patients and in some cases an accurate diagnosis may take up to five years.

In rare cases, patients may present signs and symptoms that a medical professional may not be able to identify as being related to or caused by the disease. In such cases, extensive genetic testing may be conducted to identify genetic variations that may be associated with the manifestation of the disease. It is always beneficial to ensure that the patient is either assigned to a clinical geneticist or/or a physician who is familiar with the difficulties associated with interpreting genetic data.

Patient Availability and Recruitment

Recruiting patients to participate in clinical trials in the field of orphan medicinal products is a challenging task. Not only is it challenging to determine the target patient population, but also finding investigators who specialize in treating these patients is challenging. These difficulties may pose a recruitment risk and necessitate a customized feasibility approach to reduce risk for each rare disease study.

Because the patient population is likely to be geographically distributed, patients may be required to travel considerable distances to access the study site for an orphan disease clinical trial. While travel reimbursement could potentially reduce this risk, organizations should also explore novel solutions and technologies to collect data from home and minimize site visits.

Endpoint Selection

When designing therapeutics for rare conditions, it is important to ensure that clinical study endpoints measure how the drug changes the progression of patient's disease. To distinguish a drug effect from standard disease progression, clinical studies and endpoint design must adequately reflect regulatory requirements and payer considerations.

It is recommended that sponsors collaborate with regulatory agencies to determine efficacy endpoints prior to the clinical study. The Food and Drug Administration organizes disease-specific forums to identify and discuss endpoints that are relevant from the patient or caregiver's point of view; these endpoints may include verified patient-reported outcomes that include the patient's voice. Rare disease often requires participation from numerous sites across many regions

Key Market Trends

Growing Product Innovation

Rising demand for rare diseases has led to the development of various therapies for the disease. For instance, in February 2023, Sanofi is planning to launch two of its medicines for rare diseases in India early this year or next year. The company has been recommended by the Subject Expert Committee for the import and placing on the market of the two novel products in India: Nexviazyme (Aveloglucosidase Alfa powder) for the treatment of Pompe disease and Xenpozyme and Olipudase Alfa powder, for the treatment of Niemann Pick disease (ASMD). According to a company note, both products have orphan drug status and are approved in several countries including the United States, Australia, the European Union, the United Kingdom, and Japan. In addition, the company has waivers for late-stage clinical trials (Phase III and IV).

Advancements In Precision Medicine (PPM), Big Data, and Genomics

There are no indications that the rare disease space will experience a decline in popularity in the future, as the current drug development landscape is well-suited to the emerging field of medicine. The development of Personalized Precision Medicine (PPM), Big Data, and Genomics has enabled a significant improvement in the targeting of challenging diagnostic and therapeutic forms of disease, while a better comprehension of the genetic substrates of pathologies has enabled the delineation and targeting of rare disorders

Regulatory Game-Changers

The 1983 enactment of the United States Orphan Drug Act is widely regarded as the turning point in the history of rare disease drug development. The legislation sought to address market failure by providing incentives to pharmaceutical companies that would otherwise be reluctant to invest in a drug that could potentially benefit only a small number of patients. According to pharmaceutical trade organization (PhRMA), more than 600 orphan drugs have received FDA approval since Orphan Drug Act's inception, yet only 5 percent of rare conditions have an FDA-approved treatment, meaning there's still plenty of untapped growth potential for years to come. For instance, in 2022, FDA announced the launch of the new Accelerating Rare Disease Cure (ARC) Program.

Companies Launching Initiative Programs

There are various initiatives being taken by companies to help people living with rare diseases. For instance, in 2022, Horizon launched new support initiatives for adults and teens living with rare disease. Being a rare disease sufferer can be challenging and lonely. To provide relief to the estimated 500-600 individuals living with cystic fibrosis in the United States, Horizon Therapeutic launched two distinct initiatives. Additionally in 2021, Horizon Therapeutics announced the launch of monthly virtual storytelling experience, #RAREis, to highlight the voices and stories of the community of children living with rare diseases and to raise awareness and funds for Make-A- Wish Mid-Atlantic and Make-A-Wish Canada. This photobooth is part of Horizon's global mission to support the wishes of children living with life-altering rare diseases and to bring smiles to their faces during a difficult time. Further, in support for the rare disease community, Horizon's U.S headquarters in Deerfield, Illinois and global headquarters in Dublin, Ireland was lit in purple, pink, green, and blue, the official colors of rare disease day.

Segmental Insights

Therapeutic Area Insights

Hematologic diseases, which encompass a wide range of conditions affecting the blood and related tissues, hold a dominant position in the global rare disease therapeutic market for several compelling reasons. Firstly, these diseases collectively represent a significant proportion of rare diseases globally, making them a substantial part of the market. Examples include sickle cell anemia, hemophilia, and various rare blood disorders.

Secondly, advancements in research and therapeutic development have propelled the emergence of novel treatments and therapies for hematologic rare diseases. Gene therapy and precision medicine approaches, in particular, have shown promising results in addressing previously untreatable or poorly managed conditions. This therapeutic innovation has attracted substantial investment and interest from pharmaceutical companies and researchers. Furthermore, hematologic rare diseases often have a significant impact on patients' quality of life and longevity, driving both medical and societal urgency for effective treatments. This sense of urgency fosters collaborations among healthcare providers, researchers, and advocacy groups, further accelerating therapeutic development efforts.

Lastly, regulatory agencies, recognizing the unmet medical need in hematologic rare diseases, have implemented expedited approval pathways, facilitating quicker access to therapies. These factors collectively make hematologic rare diseases a dominant force in the global rare disease therapeutic market, driving research, innovation, and improved patient outcomes.

Route of Administration Insights

Injectables hold a dominant position in the global rare disease therapeutic market for several compelling reasons. Firstly, many rare diseases are characterized by complex pathophysiology and require precise and controlled drug delivery, making injectable formulations an optimal choice. This route of administration ensures that the medication reaches the bloodstream rapidly, allowing for more immediate therapeutic effects, crucial in managing severe or life-threatening rare diseases.

Secondly, injectables are often preferred in cases where oral or other delivery methods may be less effective due to issues like poor absorption, gastrointestinal complications, or the need for high bioavailability. In the context of rare diseases, where treatment options are limited, injectables provide a reliable means of delivering therapies directly to the bloodstream, optimizing their efficacy. Furthermore, regulatory agencies have recognized the importance of injectables in the treatment of rare diseases by expediting approvals for innovative therapies. This has encouraged pharmaceutical companies and researchers to focus on developing injectable treatments for rare diseases, driving further advancements and investments in this area.

Lastly, the high specificity and targeted delivery achievable with injectables make them a favored choice for therapies, including enzyme replacement therapies and gene therapies, which are essential in managing many rare diseases. As a result, injectables continue to dominate the global rare disease therapeutic market, offering hope and improved outcomes for patients with these challenging conditions.

Regional Insights

North America occupies a dominant position in the global rare disease therapeutic market for several compelling reasons. Firstly, the region benefits from a robust healthcare infrastructure and advanced research facilities, which foster a fertile ground for the development and adoption of rare disease treatments. North America boasts a wealth of renowned medical institutions, pharmaceutical companies, and research organizations dedicated to addressing rare diseases.

Secondly, the region's commitment to innovation is exemplified by its significant investments in research and development. Government agencies, private foundations, and pharmaceutical companies in North America allocate substantial funding towards rare disease research and therapeutic development, incentivizing the creation of cutting-edge treatments. Additionally, North America possesses a sizeable patient population, making it an attractive market for pharmaceutical companies to invest in rare disease therapies. The Orphan Drug Act in the United States, for instance, provides incentives for drug developers to focus on rare diseases, further stimulating research and commercialization efforts.

Furthermore, North America's regulatory environment is conducive to rare disease therapeutic development, with expedited approval processes and orphan drug designations facilitating quicker access to innovative treatments. These factors, combined with a culture of medical innovation and research excellence, solidify North America's dominant role in the global rare disease therapeutic market.

Key Market Players

Novartis AG

AstraZeneca PLC

Pfizer Inc.

Sanofi SA

AbbVie Inc

Bristol-Myers Squibb Co

Bayer AG

F Hoffmann-La Roche Ltd.

Amgen Inc

Eisai Co Ltd

Novo Nordisk A/S

Report Scope:

In this report, the Global Rare Disease Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Rare Disease Therapeutics Market, By Therapeutic Area:

Hematologic Diseases
Cancer
Infectious Diseases
Cardiovascular Diseases
Metabolic Diseases
Endocrine Diseases
Musculoskeletal Diseases
Others

Rare Disease Therapeutics Market, By Route of Administration:

Injectable
Oral
Others

Rare Disease Therapeutics Market, By Drug Type:

Biologics
Biosimilar
Small Molecules

Rare Disease Therapeutics Market, By Distribution Channel:

Specialty Pharmacies
Hospital Pharmacies
Online Pharmacies

Rare Disease Therapeutics Market, By Region:

North America
United States
Canada
Mexico
Europe
France
United Kingdom
Italy
Germany
Spain
Asia-Pacific
China
India
Japan
Australia
South Korea
South America
Brazil
Argentina
Colombia
Middle East & Africa
South Africa
Saudi Arabia
UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Rare Disease Therapeutics Market.

Available Customizations:

Global Rare Disease Therapeutics market report with the given market data, Tech Sci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

1. Product Overview

1.1. Market Definition
1.2. Scope of the Market
- 1.2.1. Markets Covered
- 1.2.2. Years Considered for Study
- 1.2.3. Key Market Segmentations

2. Research Methodology

2.1. Objective of the Study
2.2. Baseline Methodology
2.3. Key Industry Partners
2.4. Major Association and Secondary Sources
2.5. Forecasting Methodology
2.6. Data Triangulation & Validations
2.7. Assumptions and Limitations

3. Executive Summary

3.1. Overview of the Market
3.2. Overview of Key Market Segmentations
3.3. Overview of Key Market Players
3.4. Overview of Key Regions/Countries
3.5. Overview of Market Drivers, Challenges, Trends

4. Voice of Customer

5. Global Rare Disease Therapeutics Market Outlook

5.1. Market Size & Forecast
- 5.1.1. By Value
5.2. Market Share & Forecast
- 5.2.1. By Therapeutic Area (Hematologic Diseases, Cancer, Infectious Diseases, Cardiovascular Diseases, Metabolic Diseases, Endocrine Diseases, Musculoskeletal Diseases, Others)
- 5.2.2. By Route of Administration (Injectable, Oral, Others)
- 5.2.3. By Drug Type (Biologics, Biosimilar, Small Molecules)
- 5.2.4. By Distribution Channel (Specialty Pharmacies, Hospital Pharmacies, Online Pharmacies)
- 5.2.5. By Region
- 5.2.6. By Company (Shares of Top 5 Market Players)
5.3. Market Map
- 5.3.1. By Therapeutic Area
- 5.3.2. By Route of Administration
- 5.3.3. By Drug Type
- 5.3.4. By Distribution Channel
- 5.3.5. By Region

6. North America Rare Disease Therapeutics Market Outlook

6.1. Market Size & Forecast
- 6.1.1. By Value
6.2. Market Share & Forecast
- 6.2.1. By Therapeutic Area
- 6.2.2. By Route of Administration
- 6.2.3. By Drug Type
- 6.2.4. By Distribution Channel
- 6.2.5. By Country
  - 6.2.5.1. United States Rare Disease Therapeutics Market Outlook
    - 6.2.5.1.1. Market Size & Forecast
    - 6.2.5.1.1.1. By Value
    - 6.2.5.1.2. Market Share & Forecast
    - 6.2.5.1.2.1. By Therapeutic Area
    - 6.2.5.1.2.2. By Route of Administration
    - 6.2.5.1.2.3. By Drug Type
    - 6.2.5.1.2.4. By Distribution Channel
  - 6.2.5.2. Canada Rare Disease Therapeutics Market Outlook
    - 6.2.5.2.1. Market Size & Forecast
    - 6.2.5.2.1.1. By Value
    - 6.2.5.2.2. Market Share & Forecast
    - 6.2.5.2.2.1. By Therapeutic Area
    - 6.2.5.2.2.2. By Route of Administration
    - 6.2.5.2.2.3. By Drug Type
    - 6.2.5.2.2.4. By Distribution Channel
  - 6.2.5.3. Mexico Rare Disease Therapeutics Market Outlook
    - 6.2.5.3.1. Market Size & Forecast
    - 6.2.5.3.1.1. By Value
    - 6.2.5.3.2. Market Share & Forecast
    - 6.2.5.3.2.1. By Therapeutic Area
    - 6.2.5.3.2.2. By Route of Administration
    - 6.2.5.3.2.3. By Drug Type
    - 6.2.5.3.2.4. By Distribution Channel

7. Europe Rare Disease Therapeutics Market Outlook

7.1. Market Size & Forecast
- 7.1.1. By Value
7.2. Market Share & Forecast
- 7.2.1. By Therapeutic Area
- 7.2.2. By Route of Administration
- 7.2.3. By Drug Type
- 7.2.4. By Distribution Channel
- 7.2.5. By Country
  - 7.2.5.1. France Rare Disease Therapeutics Market Outlook
    - 7.2.5.1.1. Market Size & Forecast
    - 7.2.5.1.1.1. By Value
    - 7.2.5.1.2. Market Share & Forecast
    - 7.2.5.1.2.1. By Therapeutic Area
    - 7.2.5.1.2.2. By Route of Administration
    - 7.2.5.1.2.3. By Drug Type
    - 7.2.5.1.2.4. By Distribution Channel
  - 7.2.5.2. Germany Rare Disease Therapeutics Market Outlook
    - 7.2.5.2.1. Market Size & Forecast
    - 7.2.5.2.1.1. By Value
    - 7.2.5.2.2. Market Share & Forecast
    - 7.2.5.2.2.1. By Therapeutic Area
    - 7.2.5.2.2.2. By Route of Administration
    - 7.2.5.2.2.3. By Drug Type
    - 7.2.5.2.2.4. By Distribution Channel
  - 7.2.5.3. United Kingdom Rare Disease Therapeutics Market Outlook
    - 7.2.5.3.1. Market Size & Forecast
    - 7.2.5.3.1.1. By Value
    - 7.2.5.3.2. Market Share & Forecast
    - 7.2.5.3.2.1. By Therapeutic Area
    - 7.2.5.3.2.2. By Route of Administration
    - 7.2.5.3.2.3. By Drug Type
    - 7.2.5.3.2.4. By Distribution Channel
  - 7.2.5.4. Italy Rare Disease Therapeutics Market Outlook
    - 7.2.5.4.1. Market Size & Forecast
    - 7.2.5.4.1.1. By Value
    - 7.2.5.4.2. Market Share & Forecast
    - 7.2.5.4.2.1. By Therapeutic Area
    - 7.2.5.4.2.2. By Route of Administration
    - 7.2.5.4.2.3. By Drug Type
    - 7.2.5.4.2.4. By Distribution Channel
  - 7.2.5.5. Spain Rare Disease Therapeutics Market Outlook
    - 7.2.5.5.1. Market Size & Forecast
    - 7.2.5.5.1.1. By Value
    - 7.2.5.5.2. Market Share & Forecast
    - 7.2.5.5.2.1. By Therapeutic Area
    - 7.2.5.5.2.2. By Route of Administration
    - 7.2.5.5.2.3. By Drug Type
    - 7.2.5.5.2.4. By Distribution Channel

8. Asia Pacific Rare Disease Therapeutics Market Outlook

8.1. Market Size & Forecast
- 8.1.1. By Value
8.2. Market Share & Forecast
- 8.2.1. By Therapeutic Area
- 8.2.2. By Route of Administration
- 8.2.3. By Drug Type
- 8.2.4. By Distribution Channel
- 8.2.5. By Country
  - 8.2.5.1. China Rare Disease Therapeutics Market Outlook
    - 8.2.5.1.1. Market Size & Forecast
    - 8.2.5.1.1.1. By Value
    - 8.2.5.1.2. Market Share & Forecast
    - 8.2.5.1.2.1. By Therapeutic Area
    - 8.2.5.1.2.2. By Route of Administration
    - 8.2.5.1.2.3. By Drug Type
    - 8.2.5.1.2.4. By Distribution Channel
  - 8.2.5.2. India Rare Disease Therapeutics Market Outlook
    - 8.2.5.2.1. Market Size & Forecast
    - 8.2.5.2.1.1. By Value
    - 8.2.5.2.2. Market Share & Forecast
    - 8.2.5.2.2.1. By Therapeutic Area
    - 8.2.5.2.2.2. By Route of Administration
    - 8.2.5.2.2.3. By Drug Type
    - 8.2.5.2.2.4. By Distribution Channel
  - 8.2.5.3. South Korea Rare Disease Therapeutics Market Outlook
    - 8.2.5.3.1. Market Size & Forecast
    - 8.2.5.3.1.1. By Value
    - 8.2.5.3.2. Market Share & Forecast
    - 8.2.5.3.2.1. By Therapeutic Area
    - 8.2.5.3.2.2. By Route of Administration
    - 8.2.5.3.2.3. By Drug Type
    - 8.2.5.3.2.4. By Distribution Channel
  - 8.2.5.4. Japan Rare Disease Therapeutics Market Outlook
    - 8.2.5.4.1. Market Size & Forecast
    - 8.2.5.4.1.1. By Value
    - 8.2.5.4.2. Market Share & Forecast
    - 8.2.5.4.2.1. By Therapeutic Area
    - 8.2.5.4.2.2. By Route of Administration
    - 8.2.5.4.2.3. By Drug Type
    - 8.2.5.4.2.4. By Distribution Channel
  - 8.2.5.5. Australia Rare Disease Therapeutics Market Outlook
    - 8.2.5.5.1. Market Size & Forecast
    - 8.2.5.5.1.1. By Value
    - 8.2.5.5.2. Market Share & Forecast
    - 8.2.5.5.2.1. By Therapeutic Area
    - 8.2.5.5.2.2. By Route of Administration
    - 8.2.5.5.2.3. By Drug Type
    - 8.2.5.5.2.4. By Distribution Channel

9. South America Rare Disease Therapeutics Market Outlook

9.1. Market Size & Forecast
- 9.1.1. By Value
9.2. Market Share & Forecast
- 9.2.1. By Therapeutic Area
- 9.2.2. By Route of Administration
- 9.2.3. By Drug Type
- 9.2.4. By Distribution Channel
- 9.2.5. By Country
  - 9.2.5.1. Brazil Rare Disease Therapeutics Market Outlook
    - 9.2.5.1.1. Market Size & Forecast
    - 9.2.5.1.1.1. By Value
    - 9.2.5.1.2. Market Share & Forecast
    - 9.2.5.1.2.1. By Therapeutic Area
    - 9.2.5.1.2.2. By Route of Administration
    - 9.2.5.1.2.3. By Drug Type
    - 9.2.5.1.2.4. By Distribution Channel
  - 9.2.5.2. Argentina Rare Disease Therapeutics Market Outlook
    - 9.2.5.2.1. Market Size & Forecast
    - 9.2.5.2.1.1. By Value
    - 9.2.5.2.2. Market Share & Forecast
    - 9.2.5.2.2.1. By Therapeutic Area
    - 9.2.5.2.2.2. By Route of Administration
    - 9.2.5.2.2.3. By Drug Type
    - 9.2.5.2.2.4. By Distribution Channel
  - 9.2.5.3. Colombia Rare Disease Therapeutics Market Outlook
    - 9.2.5.3.1. Market Size & Forecast
    - 9.2.5.3.1.1. By Value
    - 9.2.5.3.2. Market Share & Forecast
    - 9.2.5.3.2.1. By Therapeutic Area
    - 9.2.5.3.2.2. By Route of Administration
    - 9.2.5.3.2.3. By Drug Type
    - 9.2.5.3.2.4. By Distribution Channel

10. Middle East & Africa Rare Disease Therapeutics Market Outlook

10.1. Market Size & Forecast
- 10.1.1. By Value
10.2. Market Share & Forecast
- 10.2.1. By Therapeutic Area
- 10.2.2. By Route of Administration
- 10.2.3. By Drug Type
- 10.2.4. By Distribution Channel
- 10.2.5. By Country
  - 10.2.5.1. South Africa Rare Disease Therapeutics Market Outlook
    - 10.2.5.1.1. Market Size & Forecast
    - 10.2.5.1.1.1. By Value
    - 10.2.5.1.2. Market Share & Forecast
    - 10.2.5.1.2.1. By Therapeutic Area
    - 10.2.5.1.2.2. By Route of Administration
    - 10.2.5.1.2.3. By Drug Type
    - 10.2.5.1.2.4. By Distribution Channel
  - 10.2.5.2. Saudi Arabia Rare Disease Therapeutics Market Outlook
    - 10.2.5.2.1. Market Size & Forecast
    - 10.2.5.2.1.1. By Value
    - 10.2.5.2.2. Market Share & Forecast
    - 10.2.5.2.2.1. By Therapeutic Area
    - 10.2.5.2.2.2. By Route of Administration
    - 10.2.5.2.2.3. By Drug Type
    - 10.2.5.2.2.4. By Distribution Channel
  - 10.2.5.3. UAE Rare Disease Therapeutics Market Outlook
    - 10.2.5.3.1. Market Size & Forecast
    - 10.2.5.3.1.1. By Value
    - 10.2.5.3.2. Market Share & Forecast
    - 10.2.5.3.2.1. By Therapeutic Area
    - 10.2.5.3.2.2. By Route of Administration
    - 10.2.5.3.2.3. By Drug Type
    - 10.2.5.3.2.4. By Distribution Channel

11. Market Dynamics

11.1. Drivers
11.2. Challenges

12. Market Trends & Developments

12.1. Recent Development
12.2. Mergers & Acquisitions
12.3. Technology Launches

13. Competitive Landscape

13.1. Novartis AG
- 13.1.1. Business Overview
- 13.1.2. Treatment Offerings
- 13.1.3. Recent Developments
- 13.1.4. Key Personnel
- 13.1.5. SWOT Analysis
13.2. AstraZeneca PLC
13.3. Pfizer Inc
13.4. Sanofi SA
13.5. AbbVie Inc
13.6. Bristol-Myers Squibb Co
13.7. Bayer AG
13.8. F Hoffmann-La Roche Ltd
13.9. Amgen Inc
13.10. Eisai Co Ltd
13.11. Novo Nordisk A/S