Despite the immature state of stem cell medicine, patients are seeking and accessing putative stem cell therapies in an "early market" in which direct-to-consumer advertising via the internet likely plays an important role. We analyzed stem cell clinic websites and appraised the relevant published clinical evidence of stem cell therapies to address three questions about the direct-to-consumer portrayal of stem cell medicine in this early market: What sorts of therapies are being offered? How are they portrayed? Is there clinical evidence to support the use of these therapies? We found that the portrayal of stem cell medicine on provider websites is optimistic and unsubstantiated by peer-reviewed literature.

BackgroundThe rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.ObjectivesThe aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources.MethodsA scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making.ResultsThe scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or ‘opportunity cost’-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework.ConclusionOur framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-014-0235-x) contains supplementary material, which is available to authorized users.

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Objectives To assess the feasibility of using a citizensÕ jury to elicit public values on health technologies and to develop criteria for setting priorities for health technology assessment (HTA).Methods Sixteen individuals were selected from 1600 randomly sampled residents of the Capital Health Region in Alberta, Canada. They participated in a 2½ day jury which comprised presentations by Ôexpert witnessesÕ, who represented innovators, patients, healthcare policy-makers and clinicians, as well as a series of small and large group priority-setting exercises based on actual examples of technologies that had recently been considered for assessment by local and national HTA bodies. The session was audio-taped, and transcripts were independently reviewed by two researchers using content analytical techniques in order to ensure that no important concepts expressed by individual jurors were missed during group development of the final list of priority-setting criteria. Jurors evaluated the process by completing self-administered, semi-structured questionnaires at the end of the session. Responses were analysed using qualitative methods.Results The jury identified 13 criteria, which they subsequently ranked in order of importance. The top two criteria included Ôpotential to benefit a number of peopleÕ and Ôextends life with qualityÕ. Based on feedback from questionnaires, jurors valued the opportunity to become engaged in such a process, and expressed interest in participating in future juries.Conclusions CitizensÕ juries offer a feasible approach to involving the public in priority-setting for HTA. Furthermore, technologies that may benefit a number of people and improve quality of life appear to be of greatest importance to the public.

Health technology assessment (HTA) has become an integral part of decision-making on the coverage of new health technologies in most health systems in the developed world. In recent years, pressure to involve patients and members of the public in HTA has grown. In this article, we summarize findings from peer-reviewed and 'gray' literature, and discussions with key informants to determine potential roles for patients and the public in HTA and coverage decision-making. We also summarize existing roles for both groups in jurisdictions. Although there appears to be a general view that involvement of patients and the public is highly desirable, research offering insights into the effectiveness of different approaches to accomplish this is scarce. Nonetheless, many of the HTA agencies in developed countries have established some mechanism for seeking input from patients or the public in their processes.