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Background The current organization of the pharmaceutical innovation system poses three major challenges for rare disease patients in terms of availability, accessibility and affordability of treatments. While some changes have emerged in the European Union to address some of these challenges, their impacts are not experienced uniformly across member states nor around the world. We have observed niche initiatives that are actively working to address those challenges within their local contexts. In a position paper in this journal, we characterized such initiatives as “social pharmaceutical innovation” (or SPIN): novel collaborations involving diverse sets of actors that break with conventional pharmaceutical innovation practices to develop interventions that address unmet societal needs of rare disease patients and that are not primarily market driven. Results Here we report on 15 cases of SPIN across Brazil, Canada, France and the Netherlands that we studied through semi-structured qualitative interviews ( n  = 151) with players involved in those cases. Our findings show how SPIN initiatives are reconfiguring pharmaceutical innovation networks to include a wider range of actors in redistributed and differentiated roles within innovation processes. Further, we find that SPINs are associated with changes in the ways data is gathered (often in clinical contexts rather than in conventional trials), and how evidence is assembled to improve access to the treatments. Finally, we demonstrate how SPINs are providing new routes for patients to access treatments for rare diseases, often at more affordable prices. Conclusions While promising, SPINs are not perfect solutions for rare disease patients or the broader challenges to the pharmaceutical innovation system. SPINs are specific solutions adapted to the particulars of local framing, institutions, national policy and care contexts of rare diseases, and should be developed as such. Our findings support these recommendations for SPIN: use local knowledge and expertise in crafting SPINs; develop comprehensive strategies for data governance, access and ownership; and explore new economic models to recoup investments and/or sustain future initiatives. We invite collaboration on these topics and emerging SPIN initiatives so as to support efforts at addressing challenges of availability, accessibility and affordability of treatments for rare diseases patients.

Rare diseases are associated with difficulties in addressing unmet medical needs, lack of access to treatment, high prices, evidentiary mismatch, equity, etc. While challenges facing the development of drugs for rare diseases are experienced differently globally (i.e., higher vs. lower and middle income countries), many are also expressed transnationally, which suggests systemic issues. Pharmaceutical innovation is highly regulated and institutionalized, leading to firmly established innovation pathways. While deviating from these innovation pathways is difficult, we take the position that doing so is of critical importance. The reason is that the current model of pharmaceutical innovation alone will not deliver the quantity of products needed to address the unmet needs faced by rare disease patients, nor at a price point that is sustainable for healthcare systems. In light of the problems in rare diseases, we hold that re-thinking innovation is crucial and more room should be provided for alternative innovation pathways. We already observe a significant number and variety of new types of initiatives in the rare diseases field that propose or use alternative pharmaceutical innovation pathways which have in common that they involve a diverse set of societal stakeholders, explicitly address a higher societal goal, or both. Our position is that principles of social innovation can be drawn on in the framing and articulation of such alternative pathways, which we term here social pharmaceutical innovation (SPIN), and that it should be given more room for development. As an interdisciplinary research team in the social sciences, public health and law, the cases of SPIN we investigate are spread transnationally, and include higher income as well as middle income countries. We do this to develop a better understanding of the social pharmaceutical innovation field’s breadth and to advance changes ranging from the bedside to system levels. We seek collaborations with those working in such projects (e.g., patients and patient organisations, researchers in rare diseases, industry, and policy makers). We aim to add comparative and evaluative value to social pharmaceutical innovation, and we seek to ignite further interest in these initiatives, thereby actively contributing to them as a part of our work.

Whilst public engagement in decisions concerning science and technology is widely extolled, research shows that the application of deliberative democratic theory remains – at least in Europe – highly constrained. Science and technology policy requires closer attention to the wider context of governance and the compatibility of public deliberation with established modes of policy-making.

The final report1 of the Ministerial Review Committee on the Science, Technology and Innovation (STI) Landscape is the latest in a series of documents seeking to review South Africa's National System of Innovation (NSI) and to identify the important actions that are required to enhance innovation within the system and 'deliver a sustained and durable knowledge-based economy'. Although not explicitly stated, it is evident that the Minister of Science and Technology is concerned about the underperformance of the NSI, which necessitated this review. The committee's first report presented its view of the reasons for this underperformance, which included inadequate human capital development, the lack of a common understanding of research and innovation, limited horizontal and vertical coherence, poor support for innovation activities other than formal research and development, and inadequate oversight or analysis. The final report outlines how the DST might act to rectify the underperformance, to invigorate the NSI, and in particular, to direct the system such that it addresses the country's main priorities.

Debat: Actor-netwerk theorie, materiële semiotiek en de Nederlandse sociologie

We believe that the system of post-authorisation assessment for orphan drugs needs to be reformed to address these problems. Fast access to high quality data is essential for the improvement of treatment options and to manage costs more effectively.

The final report of the Ministerial Review Committee on the Science, Technology and Innovation (STI) Landscape is the latest in a series of documents seeking to review South Africa's National System of Innovation (NSI) and to identify the important actions that are required to enhance innovation within the system and 'deliver a sustained and durable knowledge-based economy'.

The social and legal implications of forensic DNA are paramount. For this reason, forensic DNA enjoys ample attention from legal, bioethics, and science and technology studies scholars. This article contributes to the scholarship by focusing on the neglected issue of sameness. We investigate a forensic courtroom case which started in the early '90s and focus on three modes of making similarities: (1) creating equality before the law, (2) making identity, and (3) establishing standards. We argue that equality before the law is not merely a principle but a practice. In the context of DNA research, equality refers to using standardized technology and procedures to identify the criminal suspect. Our case shows the work at stake in introducing a new technology into the courtroom and serves as a lens, magnifying how contingencies and uncertainties are managed and ordered in everyday court practices to arrive at an equal treatment of the suspect.

A call from the EU for the set-up of European Reference Networks (ERNs) is expected to be launched in the first quarter of 2016. ERNs are intended to improve the care for patients with low prevalent or rare diseases throughout the EU by, among other things, facilitating the pooling and exchange of experience and knowledge and the development of protocols and guidelines. In the past, for example where costly orphan drugs have been concerned, industry has played an important role in facilitating consensus meetings and publication of guidelines. The ERNs should provide a unique opportunity for healthcare professionals and patients to lead these activities in an independent way. However, currently costs for networking activities are not to be covered by EU funds and alternative sources of funding are being explored. There is growing concern that any involvement of the industry in the funding of ERNs and their core activities may create a risk of undue influence. To date, the European Commission has not been explicit in how industry will be engaged in ERNs. We believe that public funding and a conflict of interest policy are needed at the level of the ERNs, Centers of Expertise (CEs), healthcare professionals and patient organizations with the aim of maintaining scientific integrity and independence. Specific attention is needed where it concerns the development of clinical practice guidelines. A proposal for a conflict of interest policy is presented, which may support the development of a framework to facilitate collaboration, safeguard professional integrity and to establish and maintain public acceptability and trust among patients, their organizations and the general public.