Is it the Right Topic? An Overlooked Stage in the Institutionalization of Health Technology Assessment.

Producing a Health Technology Assessment (HTA) is resource intensive, therefore, an explicit process for Topic Identification, Selection, and Prioritization (TISP) can optimize the use of limited resources to those HTA topics of national importance. TISP does not have to be complicated, however, a formalized process facilitates HTA recommendations that better align with local priorities. The comprehensiveness of TISP processes varies according to countries' needs and to the types of decisions HTA supports. There may be many relevant considerations for TISP, such as the resources available for allocation within the health system, the number of dedicated personnel to complete HTA, and the number of stakeholders and institutions involved in the decision-making process. In countries where HTA-supported decision-making is well-established, the process for TISP is usually formalized. In settings where HTA is emerging, relatively new, or where there may not be the necessary supporting institutional mechanisms, there is limited normative guidance on how to implement TISP. We argue that developing a clear process for TISP is key when institutionalizing HTA. Moreover, insights and experiences from more formalized HTA systems can provide valuable lessons. In this commentary we discuss three institutional aspects that we believe are vital to TISP: 1) Begin topic selection with a clear link to health system feasibility, 2) Ensure legitimacy and impact through transparent TISP processes, and 3) Include the public from the start to embed patient and public engagement throughout HTA.

What principles should govern the use of managed entry agreements?

BACKGROUND: To ensure rapid access to new potentially beneficial health technologies, obtain best value for money, and ensure affordability, healthcare payers are adopting a range of innovative reimbursement approaches that may be called Managed Entry Agreements (MEAs). METHODS: The Health Technology Assessment International (HTAi) Policy Forum sought to identify why MEAs might be used, issues associated with implementation and develop principles for their use. A 2-day deliberative workshop discussed key papers, members' experiences, and collectively addressed four policy questions that resulted in this study. RESULTS: MEAs are used to give access to new technologies where traditional reimbursement is deemed inappropriate. Three different forms of MEAs have been identified: management of budget impact, management of uncertainty relating to clinical and/or cost-effectiveness, and management of utilization to optimize performance. The rationale for using these approaches and their advantages and disadvantages differ. However, all forms of MEA should take the form of a formal written agreement among stakeholders, clearly identifying the rationale for the agreement, aspects to be assessed, methods of data collection and review, and the criteria for ending the agreement. CONCLUSIONS: MEAs should only be used when HTA identifies issues or concerns about key outcomes and/or costs and/or organizational/budget impacts that are material to a reimbursement decision. They provide patient access and can be useful to manage technology diffusion and optimize use. However, they are administratively complex and may be difficult to negotiate and their effectiveness has yet to be evaluated.

Interactions between health technology assessment, coverage, and regulatory processes: emerging issues, goals, and opportunities.

BACKGROUND: The relationship between regulatory approval on the one hand and health technology assessment (HTA) and coverage on the other is receiving growing attention. Those responsible for regulatory approval, HTA, and coverage have different missions and their information requirements reflect these. There is nonetheless an increasingly popular view that improved communication and coordination between these functions could allow them all to be undertaken effectively with a lower overall burden of evidence requirements, thus speeding patient access to new products and reducing unnecessary barriers to innovation. This study summarizes the main points emerging from a recent discussion of this topic at the HTAi Policy Forum. RESULTS AND CONCLUSIONS: After considering the roles of the various bodies, stakeholder perspectives and some current practical initiatives, those present at the Forum meeting discussed possible goals and challenges for improved interactions-in general and at specific stages of the product development life cycle. Opportunities for progress were seen in: continuing the dialogue to promote understanding and interaction between the different bodies and stakeholders; working to align scientific advice for manufacturers on the design and data requirements of pre- and post-marketing evaluation of products (specifically phase 2/3 and phase 4 trials for drugs); and extending the current dialogue to include discussion of product development to address unmet health needs.

Health technology assessment to optimize health technology utilization: using implementation initiatives and monitoring processes.

BACKGROUND: The way in which a health technology is used in any particular health system depends on the decisions and actions of a variety of stakeholders, the local culture, and context. In 2009, the HTAi Policy Forum considered how health technology assessment (HTA) could be improved to optimize the use of technologies (in terms of uptake, change in use, or disinvestment) in such complex systems. METHODS: In scoping, it was agreed to focus on initiatives to implement evidence-based guidance and monitoring activities. A review identified systematic reviews of implementation initiatives and monitoring activities. A two-day deliberative workshop was held to discuss key papers, members' experiences, and collectively address key questions. This consensus paper was developed by email and finalized at a postworkshop meeting. RESULTS: Evidence suggests that the impact and use of HTA could be increased by ensuring timely delivery of relevant reports to clearly determined policy receptor (decision-making) points. To achieve this, the breadth of assessment, implementation initiatives such as incentives and targeted, intelligent dissemination of HTA result, needs to be considered. HTA stakeholders undertake a variety of monitoring activities, which could inform optimal use of a technology. However, the quality of these data varies and is often not submitted to an HTA. CONCLUSIONS: Monitoring data should be sufficiently robust so that they can be used in HTA to inform optimal use of technology. Evidence-based implementation initiatives should be developed for HTA, to better inform decision makers at all levels in a health system about the optimal use of technology.

Phenotype and gene expression of human mesenchymal stem cells in alginate scaffolds.

Human mesenchymal stem cells (MSC) are popular candidates for tissue engineering. MSC are defined by their properties in two-dimensional (2D) culture systems. Cells in 2D are known to differ from their in vivo counterparts in cell shape, proliferation, and gene expression. Little is so far known about the phenotype and gene expression of cells in three-dimensional (3D) culture systems. To begin to unravel the impact of 3D versus 2D culture conditions on MSC, we have established MSC from adipose tissue and bone marrow in 3D cultures in alginate beads covalently modified with the tripeptide arginine-glycine-aspartic acid (RGD), the integrin-binding motif found in several molecules within the extracellular matrix. The MSC changed from their fibroblastoid shape (2D) to a small, compact shape when embedded in RGD alginate (3D). High viability was maintained throughout the experiment. The MSC retained expression of integrins known to bind RGD, and practically ceased to proliferate. Microarray analysis revealed that the gene expression in cells in RGD alginate was different both from the cells cultured in 2D and from prospectively isolated, uncultured MSC, but more similar to 2D cells. As alginate may be entirely dissolved, leaving the cells as single cell suspensions for various analyses, this represents a useful model for the study of cells in 3D cultures.