Wouter Boon

Social Impacts of Transport: Literature Review and the State of the Practice of Transport Appraisal in the Netherlands and the United Kingdom

Abstract Transport appraisals in European countries increasingly address three dimensions of sustainability—economic, ecological and social. However, social impacts of transport have been underexposed in (ex‐ante) transport project appraisal, at least in the Netherlands. Firstly, this article presents a theoretical framework describing the relationships between determinants of social impacts of transport; it also provides a definition and categorization of those impacts. Secondly, the article reviews the state of the practice of national transport project appraisal in the Netherlands and the United Kingdom. The article shows that social impacts of transport investments can take on many forms and their levels of importance may vary widely, in project appraisal. The UK transport appraisal guidance includes a spectrum of social impacts through quantitative and qualitative assessments that is broader than the Dutch appraisal guidance. However, it does not cover the full range as identified in the literature. This holds, in particular, for the temporary impacts of transport investments, health impacts, social cohesion, the distribution and accumulation of impacts across population groups and social justice. All in all, it can be concluded that there is a long way to go before social impacts of transport projects are completely included in appraisals, in a way that allows us to compare them to economic and ecological effects.

The role of users in innovation in the pharmaceutical industry.

Traditionally, innovation in the pharmaceutical industry is organised according to the linear model. Over the past two decades this model lost its meaning as a result of rising costs, increased competition, new scientific developments and better-informed, more demanding users. The linear model is not well equipped to involve these new actors and to include their feedback. Starting from a systemic approach, the involvement of actors in pharmaceutical innovation processes, more in particular users, is put central. It is discussed and illustrated with three cases why a systemic model may be more effective to cope with present developments and why users should be involved. To wind up, conclusions are drawn regarding the implications of a systemic approach for policymakers, researchers and firms.

Conditional Approval and Approval Under Exceptional Circumstances as Regulatory Instruments for Stimulating Responsible Drug Innovation in Europe

The need for fast drug innovation and the public demand for risk‐free drugs creates a dilemma for regulatory authorities: less restrictive procedures involve uncertainties about benefit/risk profiles of new drugs. The European Union has introduced two instruments that regulate early market access: conditional approvals (CAs) and approvals under exceptional circumstances (ECs). We have studied whether these instruments compromise the safety of new drugs and whether they lead to earlier access to innovative drugs. Our study shows that neither of these regulatory pathways accelerates the approval process for innovative drugs. However, the CA pathway shortens the clinical development period. Approvals under ECs are associated with longer clinical development periods, but this regulatory pathway may open up opportunities for specific drugs to be admitted into the market because less comprehensive data are required. Despite the fact that these advanced approvals are based on limited safety databases, there are no special safety issues associated with using these pathways.

The role of patient advocacy organisations in neuromuscular disease R&D - The case of the Dutch neuromuscular disease association VSN

This article investigates to what extent patient advocacy organisations play a role in influencing R&D and policymaking for rare neuromuscular diseases. The Dutch neuromuscular disease organisation VSN is studied in depth. A brief history of the VSN is sketched along with the international embedding of the organisation. Then, a more general perspective is provided on the reasons and extent of the involvement of patient organisations (and especially the VSN) in innovation processes. Lastly, internal mechanisms are presented that can best be applied by these organisations. The VSN adheres to a rare, long-term vision on drug innovation that requires long-term planning and policy and vision creation and steering the direction of science and technology. At the same time, other actors like scientific organisations and science policymakers and managers can benefit from these lessons to learn how to deal with patients and patient organisations in the future.

Use of the conditional marketing authorization pathway for oncology medicines in Europe

Conditional marketing authorization (CMA) in the European Union (EU) is an early access pathway for medicines that show promising therapeutic effects, but for which comprehensive data are not available. Using a mixed quantitative‐qualitative research design, we evaluated how CMA has been used in marketing authorization of oncology medicines in the period 2006 to 2013. We show that compared to full marketing authorization, CMA is granted based on less comprehensive data. However, this is accompanied by significantly longer assessment times and less consensus among regulators about marketing authorization. Moreover, development time from first‐in‐human testing to marketing authorization did not differ between full marketing authorization and CMA, but was significantly longer for CMA compared to accelerated approved products in the United States (US). Results indicate that CMA is not used by companies as a prospectively planned pathway to obtain early access, but as a “rescue option” when submitted data are not strong enough to justify full marketing authorization.

Governance of conditional reimbursement practices in the Netherlands

When entering the market, orphan drugs are associated with substantial prices and a high degree of uncertainty regarding safety and effectiveness. This makes decision making about the reimbursement of these drugs a complex exercise. To advance on this, the Dutch government introduced a conditional reimbursement trajectory that requires a re-evaluation after four years. This article focuses on the origins, governance and outcomes of such a conditional reimbursement trajectory for orphan drugs. We find that the conditional reimbursement scheme is the result of years of discussion and returning public pressure about unequal access to expensive drugs. During the implementation of the scheme the actors involved went through a learning process about the regulation. Our analysis shows that previous collaborations or already existing organisational structures led to faster production of the required data on cost-effectiveness. However, cost-effectiveness evidence resulting from additional research seems to weigh less than political, judicial and ethical considerations in decision making on reimbursement of orphan drugs in The Netherlands.

USER-PRODUCER INTERACTIONS IN EMERGING PHARMACEUTICAL AND FOOD INNOVATIONS

AbstractThe following sections are included:IntroductionTheory and Classification of UPIsResearch MethodologyResultsDemand articulation of a patient organisation in the context of Pompe drug developmentInteractive learning of a consortium in the context of nutrigenomics developmentsConcluding Remarks and DiscussionReferences

Prospective positioning of industrial players: the case of theranostics

How are the perceptions of industrial agents affected by newly emerging technological fields? One way to investigate this is to explore the prospective roles that companies involved attribute to themselves and to others by using positioning theory as the point of departure. An appropriate example of an emerging technology to study is theranostics (dedicated diagnostics linked with therapy) given that this affects multiple, relatively unrelated, industries (old and new) simultaneously. By studying annual reports we gain insight into how industries react strategically towards emerging technological fields. We also make a contribution to positioning theory itself.

Influence of 3D printing on transport: a theory and experts judgment based conceptual model

ABSTRACT Consumer 3D printing is on the rise and has the potential to significantly change the transport and logistics sector. Current literature on 3D printing and transport studies does not provide a systematic model of the impact of 3D printing on transport and related (policy relevant) areas, such as traffic safety, location decisions, accessibility and environmental effects. Based on a literature review and two rounds of expert consultation, we propose and refine a conceptual model as a way to approach this gap in the literature. The expert consultation yields that the conceptual model comprises the relevant and important elements for assessing the impact of 3D printing on transport and transport-related challenges. Location, needs and transport resistance are important: (a) city-level hubs are the most likely locations for 3D printers because they can coordinate material flows and gather expertise; (b) mass-individualisation and personification dictates the needs for 3D printers; (c) distribution networks will be organised more efficiently, less empty vehicles, but raw materials still need shipping. However, experts’ opinions diverged on the impact of 3D printing on transport volumes and environmental impacts.

Innovation in Europe — three questions

Three long-standing questions still need to be addressed to stimulate innovation in the European Union ...