Koichi Mikami

Orphans in the Market: The History of Orphan Drug Policy

Summary This paper examines the history of orphan drug policy, from the emergence of ‘orphans’ in the American pharmaceutical market in the 1960s, through the debates and agitations that resulted in the passage of the US Orphan Drug Act of 1983, to attempts in the 1990s to prevent abuse of that Act and restore its original intentions. Although an increased number of drugs for rare diseases have since been developed and marketed, the extremely high price of some such drugs is considered a major public health issue internationally. The present paper traces the origins of this issue to the market-based approach to resolving the problem of orphan drugs embodied in the 1983 Act. The paper also makes visible an alternative trajectory that existed for a while in the United Kingdom but was eventually abandoned in order to help the biotechnology industry grow in the context of an increasingly integrated European drug market.

The Case of Inferred Doability: An Analysis of the Socio-institutional Background of the STAP Cell Scandal

Abstract On 28 January 2014, Haruko Obokata and her colleagues held a press conference regarding their new method of producing stem cells. The cells, named STAP (stimulus-triggered acquisition of pluripotency) cells, were of considerable interest not only for stem cell scientists but also for the wider society in Japan because both its government and citizens enjoyed the international reputation earned for the country by Shinya Yamanaka’s earlier success in developing a novel technique of cell reprogramming. However, it was soon pointed out that the data in the research article seemed fabricated and was hence suggested that their claims lacked scientific credibility. What was initially considered another triumph of Japanese stem cell research thus resulted in a major national scandal. Instead of seeing this case merely as one of scientific misconduct, this article examines it as a window into the local culture of stem cell research and argues that the socio-institutional background of Obokata’s work incited the researchers involved to infer doability of STAP cell research. The problem with this inference surfaced after those who did not share the culture challenged the robustness of her work, suggesting some cultures of science may be more vulnerable to scientific misconduct than others.

Patient organization involvement and the challenge of securing access to treatments for rare diseases: report of a policy engagement workshop

Plain English summaryPatients with rare diseases often help to develop new treatments for their conditions. But once developed, those treatments are sometimes priced too high for many patients to access them. We became aware that this is a problem in the course of a social science research project that examines the place of rare diseases in health policy. We therefore organized a two-day workshop to try and understand why this problem occurs and what might be done about it.The people who participated in our workshop were: representatives of rare disease patient organizations, experts in matters of drug regulation and assessment of new health technologies, consultants involved with companies producing treatments for rare diseases, and social scientists researching related issues.The main conclusions to emerge from the discussions were as follows:Problems of access to treatments for rare diseases are not just due to high prices; procedures for regulating, assessing and delivering new treatments also need to be better organized. Patients and patient organizations have much to contribute to this process. However, their resources are often very limited. Consequently, more needs to be done to help them use those resources as effectively as possible. In particular, regulators and healthcare providers need to ensure that their procedures are clear and efficiently managed, so as not to waste patient organizations’ time and money. Clearer guidance is needed on what patient organizations can do to provide evidence of the effectiveness of new drugs. Insights gained in tackling rare diseases might also be applicable to common disorders. Finally, the consequences of Brexit for UK policies on rare diseases urgently need to be assessed.AbstractSince the enactment of orphan drug legislation in the USA, Europe and several other countries, an increasing number of treatments for rare diseases have been developed and many of them been approved for marketing. However, such treatments tend to be priced very high, and access to effective treatments remains a major challenge for patients with rare diseases – despite active involvement of patients and their support organizations in various stages of basic and applied research and commercial development. In order to allow patients to benefit from treatments proved effective for their diseases, we need to better understand why this challenge persists, and what steps might be taken to address it. To that end, we organized a policy-engagement workshop, bringing together individuals and organizations with direct experience of trying to secure access to a treatment for a rare disease along with individuals with relevant expertise in regulatory and commissioning processes for new medicines. With additional input from social scientists who offered different perspectives on the value of patient involvement, the workshop aimed to initiate a dialogue among the participants about how to address the challenge in a sustainable manner. Discussions at the workshop stressed that active involvement of patients is as valuable in the regulatory and commissioning processes as in the research and development of new medicines. However, it also highlighted certain risks and costs associated with such involvement. These include the costs of adjusting to abrupt changes in regulatory and commissioning processes, and the risk of being perceived as too close to commercial interests. To optimize use of scarce resources and ensure continuing active involvement, such risks and costs need to be better managed. Participants also noted that, owing to advances in genomic technologies, common diseases are also becoming divided into rare sub-categories, which are equally eligible for orphan drug designation. Consequently, involvement of wider patient communities beyond rare disease communities will be critical for continuing discussions about patients’ involvement in regulatory and commissioning processes, and to consider how patients and their support organizations can best work with other stakeholders – including companies, regulators and policymakers – to ensure access to effective medicines.

Risuku ka sareru shintai—Gendai igaku to touchi no tekunorojī リスク化される身体 —現代医学と統治のテクノロジ [Body as Risk—Modern Medicine and the Technology of Governance]

I sometimes wonder what the value(s) of critical sociological engagement with science for scientists themselves might be. As discussed in the critique of the so-called deficit model, no system of knowledge production, scientific or not, is inherently superior to others (e.g., Wynne 1992). Hence, we—social scientists of science—celebrate plurality of knowledge. Scientists may well develop an interest in communicating to and engaging with the nonscientist public by recognizing that science has its limitations, as many other systems of knowledge production do, and also by realizing the significance of lay expertise. But can and should scientists learn to engage with their own system as critically as we would? Isn’t the unpacking or deconstructing of scientific knowledge too disturbing for them to remain active in the system? This book—Body as Risk—suggests that it may not be as counterproductive as one might think. Tatsuya Mima, in writing the book, wears two hats, one as a neuroscientist and the other as a medical sociologist, and discusses how risk is calculated in modern society. As its title suggests, the author’s main argument is that now health risk can be identified in one’s own body, which then gets turned into a subject of his or her disciplinary practice. After briefly reviewing several social science approaches to the concept of “risk” in the introduction,Mima explores two cases of medical risk in contemporary Japan to develop his main argument. The first is the idea of metabolic syndrome (chap. 1). He explains that while metabolic syndrome—measured by one’s waist, blood pressure, cholesterol level, and so on—began to be widely recognized as one of the health problems in the country starting around 2005. It has no experienced symptoms and hence is not a disease per se. Rather, it is a state of having high risk of developing cardiovascular disease. For him, it is an example of an attempt to control people’s

The “Life Costs” of Living with Rare Genetic Diseases

Abstract Economic cost analysis of the impact that a treatment or therapy has is arguably successful insofar as judgments can be made about who will benefit the most from a particular therapy. By definition this utilitarian analysis excludes those whose disease might not be “common.” This lack of social accountancy prejudices healthcare systems not only in terms of treatments but also in terms of support, capacity and knowledge made available to ameliorate the “ life costs ” of those living with rare diseases such as Huntington’s disease (HD) or phenylketonuria (PKU). Although life costs also apply to those living with the crippling effects of common disease, we argue that the social, emotional, psychological, and financial costs for those living with life-threatening or -affecting rare genetics conditions are particularly attenuated because of the assumptions about who would benefit (the case of PKU) and the nature of that benefit (the case of HD).

Adoptable packages and the cost of their adoption: the craftwork of making the right cells for regenerative medicine in Japan

The goal of regenerative medicine is to utilize biological properties of cells for therapeutic purposes. Although substantial international investment has been made in this biomedical technology, the issue of which type of cells best serves for these purposes still remains unsettled. Adopting a conceptual framework from Clarke and Fujimura that the rightness of “tools” needs to be socially constructed, this paper examines the interactions of various actors in Japan and demonstrates two kinds of craftwork as examples of attempts to construct the rightness of the cells for the technology. In such attempts, the actors not only produced adoptable packages but also assumed responsibility for their dissemination. However, because the packages are tied to their original practices as well as to their working environment, others would have to bear considerable cost of articulation to adopt them for their job and hence the rightness of the cells has not been firmly upheld.

East Asian Science, Technology and Society

The English title of this book is slightly misleading. Despite its emphasis on social risks, the main discussion of the book is neither what kind of risks technoscience would pose to society nor in what ways such risks ought to be understood as social ones. Themore precise idea of the bookmay be conveyed by its original Japanese title: Technoscience Risks and Sociology: NewDirection in Sociology of Science and Technology (Tekunosaiensu Risuku to Shakaigaku: kagaku-shakaigaku no aratana tenkai テクノサイエンス ・リスクと社会学:科学社会学の新たな展開). In this book, Miwao Matsumoto attempts to demonstrate how sociology, that of science and technology in particular, ought to approach what he refers to as technoscience risks and introduces an approach called the sector model. As he reveals in the afterword, “this book resulted from [his] impression that we need an alternative to the existing approaches to comprehend the interface between technoscience and society” (365). To achieve this end, he needs to justify his impression, and this is exactly what the first half of the book is devoted to. Thus, the book takes on two main tasks: to present a constructive critique of the current status of sociology of science and technology and to introduce and explicate an alternative approach called the sector model. Before examining how well this book carries out these tasks, I need to clarify the premise of his argument, which he mainly deals with in the first chapter. He defines technoscience risks as “the risks that emerge at the interface of technoscience and society” and continues to explain that “in this context, risks ought to be understood as undesirable consequences for human society, in the future-oriented and collective sense, involving some degrees of uncertainty” (11). Here, the existence of such risks is assumed to be taken for granted or, at least, to be known from our past and present experiences. Then, he classifies such risks into two basic categories: the “abrupt type” (Dokan-Gata どかん型), where an event is rather closed, like an ex-