Sofosbuvir-based medicines, approved in late 2013, offer a long-sought after cure for patients with hepatitis C, a virus that disproportionately affects marginalized populations around the world. But the prices set by its manufacturer at approximately $90,000 for a three- month regimen intensified a global debate about the pricing of breakthrough medicines. The dominant economic explanations for pricing have centered on ‘risk’, with prices representing the costly and failure-ridden process of drug development, and ‘value’, with higher prices said to reflect improvements in patient health as well as savings from averted downstream medical expenses. These economic explanations are limited, however, by their focus on prices at the point of exchange between drug manufacturers and public health systems. Instead, I took a historical view, using the case of sofosbuvir to trace the political- economic dynamics and organizational relations of power across the innovation process – from early stage science to deployment. Data from documentary sources, semi-structured interviews, databases, and observations at meetings allowed me to build an account of the sofosbuvir case. Combining this data with sociological and political economy literatures on the roles of an entrepreneurial state, the rise of financial capital, and the pricing and valuation strategies used by businesses, I argue that sofosbuvir’s prices did not represent the tangible costs of innovation or the health value for patients. Rather, the prices were a product of financialization: a pattern of accumulation in which growth was pursued through the capitalization and control of intangible hepatitis C assets in financial markets. As part of this pattern, I map the mobilization of speculative capitals behind Pharmasset, a small biotechnology company that emerged from public investments to develop the compound sofosbuvir, as well as the extractive logics driving the shareholders of Gilead Sciences, a large publicly traded pharmaceutical company that ultimately acquired Pharmasset and then set the prices for the therapy. I demonstrate that though an entrepreneurial state shaped the direction of the innovation process towards a curative therapy, the processes of financialization disconnected the distribution of risks and rewards, undermined the sustainability of future innovation, and diminished patient and public health outcomes. I conclude by responding to dominant economic answers on drug pricing in light of the evidence on financialization.

Prescription drugs play an increasingly significant role in the Canadian healthcare system. Drug spending accounts for a considerable share of total healthcare expenditure and continues to be one of the fastest growing expenditure components in Canada. But, drug manufacturers’ price setting behaviours are not well understood in the literature. I develop a framework of oligopoly theory with two-dimension product differentiation based on a synthesis of the literature on the institutional history and development of the Canadian pharmaceutical system. I find that: (1) The differentiation in perceived quality between brand-name and generic drugs can explain the generic competition paradox. The degree of the product differentiation can be pivotal in shaping the brand-name drug manufacturers’ price setting behaviours in response to the shift in patients’ preference and changes in government policies. (2) Copay and generic drug price-cap policies are commonly adopted by the Canadian public drug plans to contain drug reimbursement cost. Policy-makers should use caution when applying these policies in combination or separately in order to reach the intended outcomes. (3) The generic drug price-cap can elicit competition among brand-name drug manufacturers, but it may need coordinated regulations on patented drug prices. Without full coordination among major stakeholders and across jurisdictions, the benefits of lowered drug prices for some can become additional costs for others. I innovatively adopt the multilevel model to analyze the pharmaceutical market structure and evaluate the net effect of the generic competition paradox. The empirical research on the drug price dynamics is consistent with the predictions of the previously developed theory. I find that: (1) More generic substitutes in a drug molecule are associated with a net effect of increases in drug prices, after other contextual variables are properly controlled for. (2) More therapeutic substitutes do not have a net effect of lowering drug prices. (3) When a generic substitution policy is in place, the studied brand-name drugs maintain net price premiums over their generic substitutes. But, the net price premiums in the case when there is a generic substitution policy are lower than those where there is no such policy.

全民健康保險制度在台灣開辦以來已經快二十年了，在全民都有保險、給付的範圍從基礎到複雜的醫療照護完整、合約醫療機構相當普遍、就醫的方便性等，得到大多數台灣民眾的肯定。 但全民健保這一路實施下來卻為台灣帶來的龐大國家赤字。健保局針對於每年五千餘億的健保費用其中的藥品費用(為第二大支出，占了其中的25%)多次長期進行多種的藥費及藥價管控措施，低廉的新藥核價加上每兩年的藥價調查後的藥價調降，國外藥廠除了要配合健保的藥價政策外，還需要面對醫療機構對於現有藥品再降價的要求。長久下來，藥廠利潤逐年下降影響甚劇。 其中以今年七月所要實施二代健保中「藥品三同」對於原開發藥廠藥衝擊最大。衛生署所實施的三同政策，即是針對健保給付超過15年的藥品，實施「同成分」、「同品質」、「同價格」，調整為同價格。此一政策倘若實施，原開發藥廠利潤嚴重下降，可能會導致會放棄台灣市場，民眾只剩下國產學名藥可以選擇。再者，三同政策最大的瑕疵，在於只單方面考慮健保給付的藥價，卻忽略了藥廠製藥成本，此舉明顯不顧成本不同卻硬性規定價格，明顯違反市場機制，也違反健保精神。 專利藥到期的原開發藥藥廠要如何因應以縮小品質及價格的爭端，只著眼於一昧砍藥價來填補健保財務虧損，不仔細思考同成分就一定同品質的問題而將藥品砍至同一價格的健保署藥物品質認定及給付標準。 本研究探討之研究問題包括: 1)在台灣健保逐年調降藥價的政策下藥品三同政策前，原開發藥廠在台的經營模式為何,2)在政府政策培育下的學名藥如何日益茁壯來影響專利藥到期的原開發藥廠市場活動，3)藥品三同政策實施後對專利藥到期的原開發藥廠的衝擊4)主要獲利的專利藥品皆到期的台灣武田藥品公司未來的經營模式從主要價值活動面該如何調整因應。 本研究以Afuah A.(2004)經營模式架構，先從過去健保政策下原開發藥廠的營運模式做說明，再以目前健保政策環境因素的變化，如何影響個案公司的市場行銷活動，而活動又如何影響公司的資源、定位及成本。 本研究發現「藥品三同」對台廠學名藥因具有較佳的成本競爭優勢，對原開發藥廠產生威脅，但因原料的不穩定性讓病患、處方醫師無法信任。所以原開發藥廠除了監督政府長期對學名藥廠品質加以把關、用訴訟延緩學名藥的上市外、另外一方面併購國內學名藥廠，以配合醫療院所對於低價高品質藥品的需求。最後，專利藥到期的原開發藥廠從健保政策中與學名藥廠價格競爭獲得利潤，就必須改變營運模式，首要的就是降低成本。如何從採購及製造方面有效降低成本著手，進一步調整有形、無形資產投入、人員配置培養與組織等策略資源，以及定價、市場區隔等定位修正，是原開發藥廠需要深入思考的問題。 / National Health Insurance (NHI) program has been implemented in Taiwan for almost 20 years. It has earned recognition for the compulsory enrollment for all residents in Taiwan, the benefits covered from basic to complicated medical care, the prevalence of contract medical facility and the convenience of going to doctors. NHI, however, has caused fiscal deficit for the past few years. Drug cost, among the over 500,000,000,000 NTD health insurance fee, is the second largest expenditure, which takes up 25 percent of health insurance fee. Therefore, Bureau of National Health Insurance has taken measures to manage and control drug cost and drug price multiple times for a long period. Foreign pharmaceuticals have cooperated on the low-priced pricing for new drugs and have lowered drug price after drug price evaluation every two year. However, there is still demand from medical institution for lowering drug price. The profit is therefore significantly affected in the long run. Among the impacts, the significant one for research-based pharmaceutical companies is “three sameness of drug” in the 2nd generation NHI, which will be implemented in this coming July. This three sameness policy is to adjust those drugs which are covered by NHI for over 15 years and are of the same ingredients and quality to the same price. If this policy is put into practice, the profit of research-based pharmaceutical companies would plunge, which may result in their leave of Taiwan market. The domestic generic drugs, as a result, would be the only choice for the general public. Moreover, the major flaw of this three sameness policy is that it merely considers the drug price NHI covers, not the costs of pharmaceutical companies. That the drug price is set inflexibly without taking costs difference into consideration is against not only market mechanism but also the essence of NHI. The questions this study aimed to investigate were: 1. What have been the operating models of research-based pharmaceutical companies in Taiwan in the circumstances of drug price drop year by year before “three sameness policy”? 2. How have the government-sponsored generic drugs affected the market activities of drug patent expiration of research-based pharmaceutical companies? 3. What have been the impacts of “three sameness policy” on drug patent expiration of research-based pharmaceutical companies? 4. How should the operating model of Takeda Pharmaceuticals Taiwan, Ltd. adapt to, from the aspect of primary value activities, the condition of drug patent expiration of its profitable drugs? Based on the framework of operating model by Afuah A.(2004), this study began with the operating model of research-based pharmaceutical companies in former NHI policy. It then elaborated on how changes of the present NHI policy have influenced the marketing activities of the case company and how these activities have influenced the company’s resources, position and costs. This study reveals that “three sameness policies” is favorable for Taiwanese generic manufacturers for their costs competition. However, the instability of raw materials could not gain the trust of patients and doctors. Therefore, in order to cooperate on the demand for low-price and high-quality drug, researched-based pharmaceutical companies supervise the government’s check on the quality of generic manufacturers, employ litigation to suspend the launch of generic drugs or merge domestic generic manufacturers. If research-based pharmaceutical companies with drug patent expiration would like to compete with generic manufacturers and make a profit in NHI policy, they would have to change their operating model. How to reduce costs of purchase and manufacture and go further to adjust the input of physical and intangible assets, strengthen professional training of personnel and organization and modify strategies for pricing and market segmentation are the main issues worth considering.

La recherche dans le domaine des maladies sans traitement existant obéit à plusieurs impératifs définis par le législateur européen dans le règlement (CE) n° 141/2000 du 16 décembre 1999 concernant les médicaments orphelins. Les patients atteints de maladies rares et négligées ont le droit à « la même qualité de traitement que les autres » ce qui suppose de prendre les mesures d'incitation nécessaires pour « promouvoir la recherche, le développement et la commercialisation de traitements adéquats ». C'est un « domaine prioritaire ». Après avoir désigné les médicaments concernés, l'Union européenne veut « éviter la dispersion de ressources limitées » notamment par la promotion d'une « coopération transnationale ». Dans une démarche téléologique, cette recherche décrit les effets de la législation européenne sans ignorer la réception de ce droit par les États membres. Des fondements théoriques à même d'assurer une meilleure performativité du droit et des propositions concrètes en vue de conformer le droit positif à l'intention du législateur et aux attentes des parties prenantes sont proposés. En se focalisant sur le seul levier de la propriété intellectuelle, ce droit spécial et incitatif ne semble pas en mesure d'apporter aux patients la plupart des traitements attendus. La régulation de la concurrence et la compétence nationale sur le prix des médicaments perpétuent un seuil de rentabilité éloignant de nombreuses recherches de la phase du développement. L'accès aux médicaments orphelins demeure très restreint. Un changement de paradigme dans la construction de ce droit apparaît nécessaire afin qu'un modèle économique favorable se mette en place. Il convient que le profit des pharmaciens de l'industrie ne dépende plus de la conquête de parts de marché, mais de l'intérêt de leurs inventions pour les besoins de santé non satisfaits. / Research in the field of diseases without an existing treatment is governed by several requirements defined by the European legislator in Regulation (CE) No 141/2000 of 16 December 1999 on orphan medicinal products. Patients with rare and neglected diseases have the right to "the same quality of treatment as other patients" which means taking the necessary incentives to stimulate research, development and bringing to the market of appropriate medications". It is a "priority area". After designating the drugs concerned, the European Union wants to "avoid the dispersion of limited resources", in particular by promoting "cross national co-operation". In a teleological approach, this research analyses the positive law endeavouring to implement the objectives of Orphan Drug Law. It describes the effects of European legislation without ignoring the receipt of this law by the Member States. Theoretical foundations that can ensure a better performativity of the law, as well as concrete proposals to conform the positive law to the intention of the legislator and to the stakeholders' wills are proposed. By focusing solely on the leverage of intellectual property rights, this special and incentive law does not seem to be able to provide patients with many of the expected treatments. Competition regulation and setting of the price which is a national competency perpetuate a high profitability threshold, often withholding research projects from reaching the development phase. Access to orphan drugs remains very limited. A paradigm shift in the construction of this law appears necessary in order for a favorable economic model to emerge. The profit of the industry's pharmacists should no longer depend on the conquest of market shares, but on the value of their inventions for unmet health needs.