When the late Freddie Mercury penned these words (1), perhaps he had in mind bringing innovative medicines and devices to patients more quickly.

Background: Adaptive approaches to the introduction of drugs and medical devices involve the use of an evolving evidence base rather than conventional single-point-in-time evaluations as a proposed means to promote patient access to innovation, reduce clinical uncertainty, ensure effectiveness, and improve the health technology development process.

Methods: This report summarizes a Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations from invited experts, discussions among attendees about real-world case examples, and background paper.

Results: For adaptive approaches to be understood, accepted, and implemented, the Forum identified several key issues that must be addressed. These include the need to define the goals of and to set priorities for adaptive approaches; to examine evidence collection approaches; to clarify the roles and responsibilities of stakeholders; to understand the implications of adaptive approaches on current legal and ethical standards; to determine costs of such approaches and how they will be met; and to identify differences in applying adaptive approaches to drugs versus medical devices. The Forum also explored the different implications of adaptive approaches for various stakeholders, including patients, regulators, HTA/coverage bodies, health systems, clinicians, and industry.

Conclusions: A key outcome of the meeting was a clearer understanding of the opportunities and challenges adaptive approaches present. Furthermore, the Forum brought to light the critical importance of recognizing and including a full range of stakeholders as contributors to a shared decision-making model implicit in adaptive pathways in future discussions on, and implementation of, adaptive approaches.

Transcatheter aortic valve implantation (TAVI or TAVR) is a newly introduced medical procedure enabled by a new device. Although its introduction does not represent that of all medical devices, it does illustrate a situation in which a device provides a new alternative to standard care in a specific group of patients. Unlike drugs, the medical device category is highly heterogeneous and includes both diagnostic and treatment (accessories, implantable, enablers) and capital equipment. The TAVI- case was discussed as one of the examples in the Policy Forum Meeting and the authors were acting as facilitators that summed up both the case as presented and the discussion between the members that followed. We wish to give a special thanks to the participants of the group work for their input to the discussion.

The most recent discussions of the HTAi Policy Forum are being showcased in this issue of our Journal. The topic of adaptive approaches to technology management has been a policy area of continuing importance and relevance to health technology assessment agencies and policy makers as well as industry. The latest Policy Forum publication (Husereau et al., in this issue) demonstrates that the discussions at the meeting about this complex topic were very wide indeed.

Objectives: The aim of this study was to assess incidence, time frame, and outcome of “Coverage with Evidence Development” (CED) decisions in the Swiss Basic Health Insurance scheme.

Methods: Analysis of all controversial medical technologies submitted to review by the Swiss Federal Office of Public Health (FOPH) from 1996 to 2012 with focus on decisions with constraints. Description of types of technology, type of initial decision, duration of evaluation period, final decision, and search for potential factors associated with changes over time.

Results: Forty-five (37.5 percent) of 120 controversial health technologies were classified as “yes, in evaluation, reimbursed” for a certain period of time and thirty-five (29.2 percent) as “no, in evaluation, not reimbursed” by the Federal Department of Home Affairs from 1996 to 2012. The rate of CED decisions ranged between zero and nine per year and was influenced by type of technology and calendar year. Forty-four of forty-five decisions were subject to further restrictions, to a “center or a specialist” (76 percent), “indications” (49 percent), “registry” (31 percent), or “other” (49 percent). The time to a final decision ranged from 1.5 to 11 years (median, 6 years). No factors associated with initial decision and final outcome could be identified.

Conclusions: CED as a reality in Switzerland might have enabled patients to obtain access to promising technologies early in their life cycle. CED might have acted as a trigger to a successful implementation of a comprehensive national registry. The lack of qualitative data stresses the urgent need for evaluation of the HTA decisions and their impact on patient outcome and costs.

Medical technology offers enormous potential for scalable medicine—to improve the quality and access in health care while simultaneously reducing cost. However, current medical device innovation within companies often only offers incremental advances on existing products, or originates from engineers with limited knowledge of the clinical complexities. We describe how the Hacking Medicine Initiative, based at Massachusetts Institute of Technology has developed an innovative “healthcare hackathon” approach, bringing diverse teams together to rapidly validate clinical needs and develop solutions. Hackathons are based on three core principles; emphasis on a problem-based approach, cross-pollination of disciplines, and “pivoting” on or rapidly iterating on ideas. Hackathons also offer enormous potential for innovation in global health by focusing on local needs and resources as well as addressing feasibility and cultural contextualization. Although relatively new, the success of this approach is clear, as evidenced by the development of successful startup companies, pioneering product design, and the incorporation of creative people from outside traditional life science backgrounds who are working with clinicians and other scientists to create transformative innovation in health care.

Objectives: The aim of this study was to analyze whether health technology assessment (HTA) reports published by SBU have influenced decisions, guidelines, clinical practice, or research priorities in Sweden.

Methods: All twenty-six SBU reports between 2006 and 2010 were analyzed. For each project, we searched publications and documentation that reflected impact on decisions, guidelines, research or clinical practice. Written documentation, before–after surveys or register-based time series data were used when available. Based on a conceptual model and on the available evidence, we determined whether HTA reports had a high, moderate, or low impact.

Results: HTA reports influenced comprehensive decisions to a high or moderate degree. In the case of fortifying flour with folic acid to a high degree. In ten cases, HTA reports were the primary source of clinical guidelines developed by the National Board of Health and Welfare (NBHW) or professional associations. In the cases of dyspepsia and gastro-esophageal reflux, as well as mild head injury, the HTA reports had a high impact on clinical practice. It was also obvious from this review that research had been initiated as a result of the knowledge gaps identified by HTA reports. In three cases, we had no adequate documentation, suggesting that the impact of the HTA report had been low.

Conclusions: Many interrelated forces change practice, but the cases presented here indicate that HTA reports have had a high impact on clinical guidelines, as well as a moderate or high impact on comprehensive decisions, the initiation of research and changes in clinical practice.

Objectives: The study question was whether dual-energy X-ray absorptiometry (DXA) alone is more cost-effective for identifying postmenopausal women with osteoporosis than a two-step procedure with quantitative ultrasound sonography (QUS) plus DXA. To answer this question, a systematic review was performed.

Methods: Electronic databases (PubMed, INAHTA, Health Evidence Network, NIHR, the Health Technology Assessment program, the NHS Economic Evaluation Database, Research Papers in Economics, Web of Science, Scopus, and EconLit) were searched for cost-effectiveness publications. Two independent reviewers selected eligible publications based on the inclusion/exclusion criteria. Quality assessment of economic evaluations was undertaken using the Drummond checklist.

Results: Seven journal articles and four reports were reviewed. The cost per true positive case diagnosed by DXA was found to be higher than that for diagnosis by QUS+DXA in two articles. In one article it was found to be lower. In three studies, the results were not conclusive. These articles were characterized by the differences in the types of devices, parameters and thresholds on the QUS and DXA tests and the unit costs of the DXA and QUS tests as well as by variability in the sensitivity and specificity of the techniques and the prevalence of osteoporosis.

Conclusions: The publications reviewed did not provide clear-cut evidence for drawing conclusions about which screening test may be more cost-effective for identifying postmenopausal women with osteoporosis.

Objectives: The aim of this study was to evaluate the efficacy and safety of rituximab and tocilizumab compared with adalimumab, etanercept, and infliximab, in patients with rheumatoid arthritis not responding to first-line treatment, and to compare the efficacy and safety of rituximab versus tocilizumab in patients not responding to anti-tumor necrosis factor α (anti-TNF) therapy.

Methods: A literature search of randomized controlled trials, controlled clinical trials, and systematic reviews was performed to evaluate efficacy and safety of rituximab and tocilizumab.

Results: Twenty-four RCTs were included in this systematic review with 6,357 participants; 3,450 treated with biological DMARD and 2,907 with standard care. In patients not responding to first-line treatment, rituximab shows lower response rate in at least 50 percent improvement in the American College of Rheumatology criteria (ACR50) and ACR70 compared with etanercept, at 6 months of follow-up. Tocilizumab shows higher ACR70 response rate compared with infliximab, at the same follow-up time. Other results showed no significant differences. Indirect comparisons between rituximab and tocilizumab in patients not responding for at least one anti-TNF, shows higher ACR20 response rate for tocilizumab at 6 months of follow-up. Regarding safety, adalimumab and etanercept were associated with significant fewer withdrawals due to adverse events compared with infliximab.

Conclusions: Considering efficacy, safety, and the availability of 3 anti-TNFs in the National Medicines Formulary (adalimumab, etanercept, and infliximab), it seems appropriate to remove infliximab from the coverage, and introduce tocilizumab for patients not responding for at least one anti-TNF.

Objectives: To date, IT strategic planning has been mostly theory-based with limited information on “best practices” in this area. This study presents the process and outcomes of IT strategic planning undertaken at a pediatric hospital (PH) in Canada.

Methods: A five-stage sequential and incremental process was adopted. Various tools / approaches were used including review of existing documentation, internal survey (n = 111), fifteen interviews, and twelve workshops.

Results: IT strategic planning was informed by 230 individuals (12 percent of hospital community) and revealed consistency in the themes and concerns raised by participants (e.g., slow IT projects delivery rate, lack of understanding of IT priorities, strained communication with IT staff). Mobile and remote access to patients’ information, and an integrated EMR were identified as top priorities. The methodology and used approach revealed effective, improved internal relationships, and ensured commitment to the final IT strategic plan. Several lessons were learned including: maintaining a dynamic approach capable of adapting to the fast technology evolution; involving stakeholders and ensuring continuous communication; using effective research tools to support strategic planning; and grounding the process and final product in existing models.

Conclusions: This study contributes to the development of “best practices” in IT strategic planning, and illustrates “how” to apply the theoretical principles in this area. This is especially important as IT leaders are encouraged to integrate evidence-based management into their decision making and practices. The methodology and lessons learned may inform practitioners in other hospitals planning to engage in IT strategic planning in the future.

Objectives: The current digital revolution is particularly relevant for interactions of healthcare providers with patients and the community as a whole. The growing public acceptance and distribution of new communication tools such as smart mobile phones provide the prerequisite for information and communication technology (ICT) -assisted healthcare applications. The present study aimed at identifying specifications and perceptions of different interest groups regarding future demands of ICT-supported doctor–patient communication in Austria.

Methods: German-speaking Austrian healthcare experts (n = 73; 74 percent males; mean age, 43.9 years; SD 9.4) representing medical professionals, patient advocates, and administrative personnel participated in a 2-round online Delphi process. Participants evaluated scenario-based benefits and obstacles for possible prospect introduction as well as degree of innovation, desirability, and estimated implementation dates of two medical care-related future set ups.

Results: Panelists expected the future ICT-supported doctor–patient dialogue to especially improve the three factors doctors–patient relationship, patients’ knowledge, and quality of social health care. However, lack of acceptance by doctors, data security, and monetary aspects were considered as the three most relevant barriers for ICT implementation. Furthermore, inter-group comparison regarding desirability of future scenarios showed that medical professionals tended to be more skeptical about health-related technological innovations (p < .001).

Conclusions: The findings of this survey revealed different expectations among interest groups. Thus, we suggest building taskforces and using workshops for establishing a dialogue between stakeholders to positively shape the future of ICT-supported collaboration and communication between doctors and patients.

Objectives: The need to consider the patient perspective in health technology assessments (HTA) has been widely recognized. In July 2012, the Finnish Medicines Agency (Fimea) published a national recommendation for integrating the patient perspective into the HTAs of pharmaceuticals. The aim of this study is to describe the development of the recommendation for integrating the patient perspective into the HTA process of pharmaceuticals in Finland.

Methods: The development of the recommendation was based on a review of international recommendations and experiences of patient and public involvement in HTA. The draft recommendation was tested in two focus group discussions (n = 7 patients) and three individual interviews among diabetes patients (type 1 or 2) using long-acting insulin treatment. The recommendation was open for public consultation in April 2012 and revised according to the comments received.

Results: Patients will be involved in multiple stages of Fimea's HTA process. The recommendation includes step-by-step instructions on how to assess the patient perspective. The main focus is on qualitative interviews, which will be conducted at the beginning of the assessments to gain information, particularly on patient preferences and values, including positive and negative outcomes important to patients and ethical and social aspects of the medicine's use.

Conclusions: The recommendation will act as a tool to integrate patients’ experiences, needs and preferences into Fimea's HTAs of pharmaceuticals.

Objectives: Licensing of, and coverage decisions on, new therapies should rely on evidence from patient-relevant endpoints such as overall survival (OS). Nevertheless, evidence from surrogate endpoints may also be useful, as it may not only expedite the regulatory approval of new therapies but also inform coverage decisions. It is, therefore, essential that candidate surrogate endpoints be properly validated. However, there is no consensus on statistical methods for such validation and on how the evidence thus derived should be applied by policy makers.

Methods: We review current statistical approaches to surrogate-endpoint validation based on meta-analysis in various advanced-tumor settings. We assessed the suitability of two surrogates (progression-free survival [PFS] and time-to-progression [TTP]) using three current validation frameworks: Elston and Taylor's framework, the German Institute of Quality and Efficiency in Health Care's (IQWiG) framework and the Biomarker-Surrogacy Evaluation Schema (BSES3).

Results: A wide variety of statistical methods have been used to assess surrogacy. The strength of the association between the two surrogates and OS was generally low. The level of evidence (observation-level versus treatment-level) available varied considerably by cancer type, by evaluation tools and was not always consistent even within one specific cancer type.

Conclusions: Not in all solid tumors the treatment-level association between PFS or TTP and OS has been investigated. According to IQWiG's framework, only PFS achieved acceptable evidence of surrogacy in metastatic colorectal and ovarian cancer treated with cytotoxic agents. Our study emphasizes the challenges of surrogate-endpoint validation and the importance of building consensus on the development of evaluation frameworks.

Objectives: In economic evaluations of healthcare technologies, situations arise where data are not randomized and numbers are small. For this reason, obtaining reliable cost estimates of such interventions may be difficult. This study explores two approaches in obtaining cost estimates for pregnant women screened for a fetal cardiac anomaly.

Methods: Two methods to reduce selection bias in health care: regression analyses and propensity scoring methods were applied to the total mean costs of pregnancy for women who received specialist cardiac advice by means of two referral modes: telemedicine and direct referral.

Results: The observed total mean costs of pregnancy were higher for the telemedicine group than the direct referral group (4,918 versus 4,311 GBP). The regression model found that referral mode was not a significant predictor of costs and the cost difference between the two groups was reduced from 607 to 94 GBP. After applying the various propensity score methods, the groups were balanced in terms of sizes and compositions; and again the cost differences between the two groups were smaller ranging from -62 (matching “by hand”) to 333 GBP (kernel matching).

Conclusions: Regression analyses and propensity scoring methods applied to the dataset may have increased the homogeneity and reduced the variance in the adjusted costs; that is, these methods have allowed the observed selection bias to be reduced. I believe that propensity scoring methods worked better for this dataset, because after matching the two groups were similar in terms of background characteristics and the adjusted cost differences were smaller.

Objectives: Health economic models are developed as part of the health technology assessment process to determine whether health interventions represent good value for money. These models are often used to directly inform healthcare decision making and policy. The information needs for the model require the use of other types of information beyond clinical effectiveness evidence to populate the model's parameters. The purpose of this research study was to explore issues concerned with the identification and use of information for the development of such models.

Methods: Three focus groups were held in February 2011 at the University of Sheffield with thirteen UK HTA experts. Attendees included health economic modelers, information specialists and systematic reviewers. Qualitative framework analysis was used to analyze the focus group data.

Results: Six key themes, with related sub-themes, were identified dealing with decisions and judgments; searching methods; selection and rapid review of evidence; team communication; modeler experience and clinical input and reporting methods. There was considerable overlap between themes.

Conclusions: Key issues raised by the respondents included the need for effective communication and teamwork throughout the model development process, the importance of using clinical experts as well as the need for transparent reporting of methods and decisions.

The affiliation listed for author Sergio Iannazzo for the article “Economic Evaluation of Intravenous Iodinated Contrast Media in Italy” was incorrect. The study and writeup were completed while Sergio Iannazzo was an IMS Health employee, and the author's correct affiliation for this paper is “IMS Health Italy.” The publisher regrets the error.