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Introduction 5 Cell and Gene Therapy 9 Modification of splicing: Antisense oligonucleotides and small molecules 21 Tissue Engineering 24 Small trials, difficult statistics, difficult regulation and data reproducibility. 28 Health economics of regenerative medicine 31 Regulation of stem cell therapies and regenerative medicine 41 Ethics 43 Public engagement and trust 54 Conclusions 56

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This is a report of a patient with minimal change disease (MCD) onset after bevacizumab administration. A 72-year-old man with inoperable Grade 3 astrocytoma was treated with a combination of temozolomide and the vascular endothelial growth factor monoclonal antibody bevacizumab. After two biweekly treatments, he developed nephrotic syndrome. Despite cessation of bevacizumab, his renal function deteriorated and a renal biopsy disclosed MCD. Thereafter, he was started on high-dose oral prednisone and renal function immediately improved. Within weeks, the nephrotic syndrome resolved. Although rare, biologic agents can cause various glomerulopathies that can have important therapeutic implications. MCD should be considered in patients who develop nephrotic syndrome while exposed to antiangiogenic agents.

Abstract:As health policy-makers around the world seek to make progress towards universal health coverage they must navigate between two important ethical imperatives: to set national spending priorities fairly and efficiently; and to safeguard the right to health. These imperatives can conflict, leading some to conclude that rights-based approaches present a disruptive influence on health policy, hindering states' efforts to set priorities fairly and efficiently. Here, we challenge this perception. We argue first that these points of tension stem largely from inadequate interpretations of the aims of priority setting as well as the right to health. We then discuss various ways in which the right to health complements traditional concerns of priority setting and vice versa. Finally, we set out a three-step process by which policy-makers may navigate the ethical and legal considerations at play.

Research involving human subjects is much more stringently regulated than many other nonresearch activities that appear to be at least as risky. A number of prominent figures now argue that research is overregulated. We argue that the reasons typically offered to justify the present system of research regulation fail to show that research should be subject to more stringent regulation than other equally risky activities. However, there are three often overlooked reasons for thinking that research should be treated as a special case. First, research typically involves the imposition of risk on people who do not benefit from this risk imposition. Second, research depends on public trust. Third, the complexity of the moral decision making required favors ethics committees as a regulative solution for research.

Health systems that aim to secure universal patient access through a scheme of prepayments-whether through taxes, social insurance, or a combination of the two-need to make decisions on the scope of coverage that they guarantee: such tasks often falling to a priority-setting agency. This article analyzes the decision-making processes at one such agency in particular-the UK's National Institute for Health and Care Excellence (NICE)-and appraises their ethical justifiability. In particular, we consider the extent to which NICE's model can be justified on the basis of Rawls's conception of "reasonableness." This test shares certain features with the well-known Accountability for Reasonableness (AfR) model but also offers an alternative to it, being concerned with how far the values used by priority-setting agencies such as NICE meet substantive conditions of reasonableness irrespective of their procedural virtues. We find that while there are areas in which NICE's processes may be improved, NICE's overall approach to evaluating health technologies and setting priorities for health-care coverage is a reasonable one, making it an exemplar for other health-care systems facing similar coverage dilemmas. In so doing we offer both a framework for analysing the ethical justifiability of NICE's processes and one that might be used to evaluate others.