生技製藥產業創新技術之價值創造模式-以新興細胞治療公司為例

Abstract

「創新」是科技進步的主要動力來源，更是企業創造價值的關鍵能力，在全球市場邁入知識經濟的時代，高效率且持續不斷的創新，已成為產業技術領先及企業競爭優勢之重要因素。近年來諸多暢銷藥(Blockbuster)藥品專利陸續到期，加上各國政府為了降低整體醫療費用支出，紛紛鼓勵使用學名藥(Generic drugs)，專利藥品銷售額往往在專利到期後隔年急遽下降，形成所謂的專利懸崖(Patent cliff)現象。然而，對生技製藥產業而言，持續性的研究開發與技術創新，並透過創新技術或產品取得專利保護，以確保產品成功上市後，能免於競爭而賺取超額利潤，才是生技製藥公司永續經營的重要關鍵因素。 細胞治療(Cell therapy)即是近年來相當熱門的新興生物技術，為再生醫學(Regenerative medicine)領域中最重要的一環，主要就是利用健康的細胞來進行修復、取代已受損或壞死的細胞以及因疾病、外傷而受損的組織或器官。儘管目前細胞治療技術或產品尚屬於萌芽階段，但對於創新效率不彰或能量飽和的大型製藥公司而言，極具潛力成為延續企業創新價值及突破專利懸崖窘境的利器。本研究主要透過新創公司Juno Therapeutics Inc.進行個案研究，探討生技製藥產業的創新技術如何創造價值，透過文獻比對瞭解細胞治療產業現況及未來的發展趨勢，藉由新創公司的商業營運模式進行架構分析，瞭解新興細胞治療領域之價值創造策略及模式，評估細胞治療產業是否有機會融入全球生技製藥產業價值鏈，成為創造營收的嶄新動能。 研究結果發現，個案透過公司核心能耐的轉化，形成其價值主張包含提供創新CAR及TCR技術、鎖定未被滿足的醫藥需求市場、緊密結合尖端臨床醫療機構以及客製化個人醫療產品及服務，作為細胞治療技術之價值創造策略。此外，藉由產業關鍵成功因素之掌握，個案以臨床試驗療效展現技術創新價值，同時經由對美國FDA審查時效及法規的高度熟稔，以滿足為未滿足的醫藥需求(Unmet medical need)市場，作為公司核心之價值創造模式。 研究結果也發現，細胞治療技術相當新穎，產品特性無法與現有藥物比擬，技術專利涵蓋範圍評價不易，創新技術的臨床試驗成效才是價值創造的重要關鍵。隨著再生醫學領域將朝細胞治療領邁進，整體市場價值將橫跨癌症標靶(Targeted)藥物及幹細胞藥物市場。根據IMS health統計，2013年癌症藥物市場約910美元，癌症標靶藥物約占46%，約418億美元，而由Visiongain研究指出，2016年全球幹細胞市場將達88.8億美元，意謂著全球細胞治療產業至少超過500億美元，細胞治療產業將改變再生醫學傳統模式並提升整體產業價值，成為全球生技製藥產業價值鏈創造營收的嶄新動能。

Innovation would be the major driving force for industry evolution; moreover, it is the core competence for a corporate value creation, as global market towards knowledge-based economy, to have a high efficiency and continuous innovation become the key success factor for corporate to keep their competitive advantage and the capabilities of advanced technology among industry. Recently, many blockbuster patents have expired, and governments have been encouraging the use of generic drugs in order to reduce overall healthcare cost, and this result to the phenomenon of Patent Cliff as patented drugs expired and the sales would be dramatically drops in the next year. However, to acquire patents through continuous R&D and technological innovation can be the most important element for biopharmaceutical industry to ensure they are able to gain from premium profit instead of competition while product launched. Cell therapy is the emerging biotechnology and the most important part of regenerative medicine that utilize healthy cells to repair, replace damaged or necrotic cells and due to illness, injury and damaged tissues or organs. Although cell therapy technologies or products still belong infancy, it has a great potential to become the drive for the continuation of the value innovation and the breakthrough of patent cliff dilemma, especially in big pharma or startup biotech company. In this study, a startup company, Juno Therapeutics Inc. has conducted as a case study to explore how innovative technologies in biopharmaceutical industry create values; this study also includes the status quo of the cell therapy industry and the views on future trends through literature review and various market research reports. An aim to comprehend the strategy and model of the value creation in the emerging cell therapy field via analyzing the business model of this startup case study, this analysis result can be used in cell therapy industry to increase revenue momentum and an assessment whether it’s an opportunity to integrate into the value chain of global biopharmaceutical industry or not. From this case study, we found that the transformation of core competence would formulate its value proposition, which includes providing innovative CAR and TCR technology, focusing on the unmet medical needs market, collaborating closely with advanced clinical institutions, as well as customizing personal medical products and services, the value creation strategy of cell therapy technology would base on these moves. In addition to the key success factors (KSFs) managed effectively, this case demonstrates technological innovation value via the efficiency of clinical trials, and familiar with the US FDA regulations and processes; to fulfill the unmet medical need markets, these are the KSFs for shaping this case company's value creation model. We also found from this study that the cell therapy technology is lacking spec benchmarking reference base due to its too new to the market and hard to compare with the current products. Only the efficacy of clinical trials would dominate the key of industry success, which is the most important element to the value creation. Along with the regenerative medicine field towards cell therapy field, the market value will be crossing the targeted cancer and stem cell therapy market. According to the IMS health and Visiongain statistics, in 2013, the targeted cancer drug market was around $ 41.8 billion, and the stem cell market will reach $ 8.88 billion in 2016. It means the global cell therapy market will reach more than $50 billion and become the new momentum to generate revenue for global biopharmaceutical industry.