KYLE Margaret

This doctoral thesis focuses on the challenges faced by rare disease patients. It is structured in three parts, each of which is dedicated to the issues of an actor at the heart of the diagnostic and therapeutic Odyssey of rare disease patients. The first part of the thesis focuses on the patient and his social network. Chapter 1 considers the sources of delay in access to diagnosis, and explores in particular the effect of social capital on the delay in obtaining a diagnosis. Chapter 2 discusses the negative externalities on maternal health of a diagnosis of a chronic disease in children. The second part of the thesis is dedicated to the pharmaceutical industry and focuses on R&D investment decisions targeting rare diseases. Chapter 3 assesses the causal effect of the Orphan Drug Policy on research effort, and Chapter 4 considers the inequalities in the allocation of R&D investments between rare diseases. Part 3 is dedicated to public policy makers and discusses the issues of evaluating the benefits of therapeutic innovation and defining the conditions of access to this innovation. Chapter 5 assesses the causal effect of therapeutic innovation on the longevity of rare disease patients. Chapter 6 is a critical discussion of the use of tools.

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This work focuses on the idea that organizational rationality is bounded: decision-makers seek solutions that offer minimal satisfaction and think in a way that is typical for humans. The thesis explores this interaction between the organizational search process and decision makers' cognition and demonstrates that certain biases (distortions) in characteristic aspects of our thinking can be instruments of behavioral strategy.As a starting point, I complement the first primitive of bounded rationality, i.e. the generation and evaluation of alternatives, with embedded elements of human cognition, such as intuitive thinking, specifically affect heuristics, and imperfect mental representations. Using computational models, I study the effects of biases corresponding to these elements of human cognition (affective preferences and systematic errors in mental representations) over time as organizations adapt to complex environments. This allows me to identify the life cycles of the elements of human cognition and to show that organizations should manage (rather than eliminate) certain biases. Finally, I make proposals and empirically test a subset of my predictions.In conclusion, this work aims to advance the emerging theory of behavioral strategy by jointly considering different primitives of bounded rationality and integrating them with existing knowledge in organizational science. A general question motivating this work is how organizations can manage the many limitations of human rationality.

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This paper focuses on the relationship between therapeutic value and different measures of market rewards (the number of patents, price, market share, and total revenues) of a new treatment. Using an assessment of therapeutic value provided by the French Haute Authorité de Santé (HAS), I find a weak relationship between most measures of rewards and this assessment of therapeutic value, suggesting that the returns to developing a "me-too" product are not very different from developing treatments with greater therapeutic effects. One interpretation is that the HAS score is a poor assessment of therapeutic value, in which case the use of similar health technology assessments by governments and other payers should be re-examined. Alternatively, if the HAS score is informative, the results suggest countries are spending too much on less innovative products, and that a re-balancing of innovation incentives may be worth considering if therapeutic value is highly related to social welfare.

This thesis studies different aspects of firm dynamics, both theoretically and empirically. All chapters make extensive use of different microeconomic databases to test the theoretical predictions. The first chapter focuses on the innovation premium, i.e. the response of the wages of employees of a firm that increases its R&D intensity and thus becomes closer to the technological frontier. The evaluation of this response is done using a database on the wage of 1% of the UK-based workers. The second chapter looks at the response of firms' innovation and productivity to an export demand shock, considering French firms with at least one patent, and using both tax and customs databases. Finally, the third chapter studies the role of adjustment costs of production factors, and in particular of corporate real estate, on the employment dynamics of firms following a productivity shock. This chapter uses a large sample of French single-establishment firms. All three chapters study different dimensions of the response of firms to a demand or productivity shock, whether it is a response in terms of employment, wages, innovation or size.

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The dissertation integrates external and internal analyses into competitive strategy in order to contribute to the analysis of the microfoundations of competition. The rationale for the thesis is motivated by two hiatuses identified in the literature that constitute a unique gap in the literature. First, the field has tended to seek advantage in either resource or positioning with little integration of the two dimensions. Second, the field has neglected how the heterogeneous resource and positioning form an endogenous microenvironment and interact during transactions. The thesis approach adopts a game-theoretic approach that harmonizes (a) firm heterogeneity and thus echoes the internal analysis, (b) strategic interaction between firms that captures the external analysis, and (c) the endogenous microenvironment. Under this general theme, the thesis focuses on three scenarios, in three distinct chapters, which share four common connections beyond the concerted effort to decipher the black box: (i) All three chapters are deeply rooted in transactions, (ii) Cumulative patterns and processes that influence firms' competitive advantage are analyzed, (iii) The set of concepts are located at the firm level, (iv) External and internal determinants are considered simultaneously. Concretely, Chapter 1 introduces the ex ante cost perspective that explains the ex post cost and ex ante incentive analyses. Chapter 2 presents the theoretical framework of evolutionary advantage through the lens of contracting strategy. Chapter 3 shows that the difference in firms' bargaining behavior supports an additional dimension of strategic maneuverability that ultimately affects value appropriation. Overall, the thesis contributes to the understanding of the dynamics of transactions between firms. It offers a valuable precedent in the integration of external and internal analyses, while at the same time providing a complement to the analysis of microfoundations.

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We examine the effect of pharmaceutical patent protection on the speed of drug launch, price, and quantity in 60 countries from 2000-2013. The World Trade Organization required its member countries to implement a minimum level of patent protection within a specified time period as part of the TRIPS Agreement. However, members retained the right to impose price controls and to issue compulsory licenses under certain conditions. These countervailing policies were intended to reduce the potential static losses that result from reduced competition during the patent term. We take advantage of the fact that at the product level, selection into TRIPS "treatment" is exogenously determined by compliance deadlines that vary across countries. We find that patents have important consequences for access to new drugs: in the absence of a patent, launch is unlikely. That is, even when no patent barrier exists, generic entry may not occur. Conditional on launch, patented drugs have higher prices but higher sales as well. The price premium associated with patents is smaller in poorer countries. Price discrimination across countries has increased for drugs patented post-TRIPS and prices are negatively related to the burden of disease, suggesting that countervailing policies to offset expected price increases may have had the intended effects.

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Innovation has received much attention from economists. As a result, intellectual property rights (IPRs), which allow the innovator, in order to recoup his R&D cost, to charge a monopoly price, have gained wide acceptance. Balancing the dynamic and static efficiency of IP is a major concern for policy makers. Many studies have addressed this issue, but have not reached a consensus. In the first chapter, I attempt to answer the following question: Should additional market exclusivity be granted for new innovators in the pharmaceutical industry? My results show that the value gained by a new innovation outweighs the loss of market exclusivity, but with a minor benefit. In chapter two (with Roberta Dessi), we turn our attention to the financial aspects of innovative activities. We review the empirical evidence on the impact of venture capital on innovation. We propose a simplified theoretical model to illuminate potential endogeneity signals to identify how venture capital influences innovation. In Chapter Three (with Margaret K. Kyle), we explore the potential effects of intellectual property rights and research exemptions on cumulative advances in drug development. Our results show that patent protection tends to promote innovative activities and facilitate post-launch drug trials. Research exemptions are found to be associated with a decrease in follow-up innovation activities. Each of these three chapters stands alone. Chapters one and three investigate the rationale for granting intellectual property rights to the innovator. Chapter two examines the financial arrangements for innovation activities.

This thesis is presented as a collection of three empirical papers. The overall objective of this thesis is to examine the export behavior of small and medium-sized enterprises (SMEs) in an emerging economy, Malaysia. This study focuses, specifically, on two research areas: export determinants and export strategy. The first and second papers analyze, respectively, the internal and external determinants of export decisions. The third paper examines export strategy in the context of market selection. The results and contributions are discussed in each article.The first article examines the impact of financial factors on the export decision. In particular, we incorporate the two major key financial dimensions, cost and capital, to study how the perception of cost, internal capital capacity, and external capital constraint determines the firm's export status. Our results show that exporters perceive a lower cost of exporting and are less constrained by external capital than non-exporters. However, we find that exporters show a lower internal capital capacity than non-exporters. This paper contributes to the literature while incorporating push and pull factors to understand the combined effect of financial determinants on export decisions. The second paper assesses the effectiveness of export promotion programs. In particular, we examine the level of awareness, frequency of use, and perceived usefulness of these programs between non-exporters and exporters. Our results suggest that exporters have more awareness, are more frequent users, and consider these programs more useful than non-exporters. However, both groups show higher levels of awareness, more frequent use, and higher levels of perceived usefulness of programs related to export information and sponsored international trade shows than those related to financial assistance such as credit counseling. In addition, the analysis also revealed that frequency of use and perceived usefulness for most programs are positively related to export experience, but not to export sales. This study helps us better understand the impact of export programs on export initiation and expansion across the different stages of exporting in an emerging economy. The third paper examines the relationship between competitive strategies and export market selection. Drawing on the literature of comparative advantage for emerging country exporters, and the characteristics of demand in export markets, we test hypotheses on how cost dominance, differentiation, and targeting strategies influence exporting to developed and developing countries. Differentiation strategies show opposite effects to cost, while targeting strategies are negatively associated with exports to both types of markets. This study contributes to the literature by showing that competitive strategies act as a firm-level determinant of export market selection.

This paper investigates how the implementation of intellectual property rights in developing countries under the 1995 TRIPS agreement affected trade in knowledge‐intensive goods. We compare trade in knowledge‐intensive goods with trade in other types of goods for 158 members and observers of the World Trade Organization from 1993–2009. Trade in knowledge‐intensive goods increased relative to a control group after TRIPS implementation. The increase in imports by developing countries was driven by exchange with high‐income countries and was concentrated in the information and communications technology sector. Our findings suggest that the effect of TRIPS on promoting knowledge diffusion from high‐income to developing countries varies by sector.