Health technology assessments (HTAs) have traditionally included clinical and cost-effectiveness evaluation of new health technologies (HTs). However, new HTs can generate important organizational impacts (OIs) that influence their overall value. OIs are currently not clearly identified and evaluated in HTA procedures and tools are limited. To address this issue, a comprehensive framework that allows to assess OIs of new HTs in HTAs is proposed.

A working and methodological group identified the Oslo Manual 2018, 4th edition, OECD/Eurostat, on the objectives and outcomes of commercial innovations as the basis for the OIs framework for HTAs. The Oslo Manual was translated to the healthcare sector and adapted to HTA procedures through a three-step process.

The framework is composed of three main parts. Part I tackles the context of the evaluation, Part II the categories of impacts and the specific impacts – in total, 16 OIs were identified – and Part III the stakeholders involved. The central part of the framework is Part II, and consists of three categories of impacts: (i) on the care process, (ii) on the stakeholders’ capabilities and skills, and (iii) on society or the community.

This framework provides a comprehensive and structured basis to document OIs of new HTs. It thus contributes to the extension of HTA evaluation criteria to other dimensions than clinical and economic aspects, that is, organizational aspects. Some of its intrinsic limitations and the questions they raise in the field for policy-makers, practitioners, and researchers are discussed.

The objective was to identify and describe the published guidance and current academic discourse of ethical issues and standards related to the use of Social Media Research for generating patient insights for the use by health technology assessment (HTA) or health policy decisions.

A scoping review of the literature was conducted in PubMed and Embase and identified 935 potential references published between January 2017 and June 2021. After title and abstract screening by three reviewers, 40 publications were included, the relevant information was extracted and data were collected in a mind map, which was then used to structure the output of the review.

Social Media Research may reveal new insights of relevance to HTA or health policies into patient needs, patient experiences, or patient behaviors. However, the research approaches, methods, data use, interpretation, and communication may expose those who post the data in social media channels to risks and potential harms relating to privacy, anonymity/confidentiality, authenticity, context, and rapidly changing technologies.

An actively engaged approach to ensuring ethical innocuousness is recommended that carefully follows best practices throughout planning, conduct, and communication of the research. Throughout the process and as a follow-up, there should be a discourse with the ethical experts to maximally protect the current and future users of social media, to support their trust in the research, and to advance the knowledge in parallel to the advancement of the media themselves, the technologies, and the research tools.

The aim of this initiative was to examine collaboratively, in a multi-stakeholder team (health technology assessment (HTA) practitioners with patient involvement expertise, health technology industry, patient advocates, health policy experts, patient engagement experts), whether evidence generated through social media research (SMR) fills current information gaps relating to insights on specific aspects of patient experiences, preferences, or patient needs and delivers additional value to HTA.

The framing of the project was done in a co-creative, deliberative multi-stakeholder process. Challenge and refinement happened through discussions with 25 independent stakeholders from HTA bodies, industry, academia, and patient advocacy. For critical themes identified during the framing phase, scoping literature reviews were performed including the state of methods and examples for the use of SMR in HTA.

The framing and stakeholder discussions specified a set of expectations and requirements, and the scoping reviews revealed the current state of methods and usage of SMR in health-policy decision making.

The project concluded that SMR can contribute new, relevant evidence to HTA. It is however recommended to evolve the science through defining best practices when planning, conducting, and using SMR and to conduct multi-stakeholder pilot SMR projects to address questions relevant to current HTAs and to validate and improve the proposed practices.

Horizon scanning for health technology appraisal (HTA) in England involves topic notification to the National Institute for Health and Care Excellence (NICE) via technology briefings. This activity is undertaken by the Innovation Observatory with submission timelines designed to ensure that HTA decisions align with regulatory approval time. In this paper, we aimed to track and assess the progression and current status of the topics notified for HTA and provide a descriptive analysis of these topics.

Technology briefings were mapped from submission to NICE technology appraisal/highly specialized technologies recommendations from April 2017 until October 2021. This was done using a combination of searches on Google and NICE website, searching a downloadable spreadsheet containing NICE topic selection decisions, and querying NICE Topic Selection team. Analysis was undertaken regarding type of indications and interventions of submitted topics and published guidance.

Six-hundred and ninety-three topics entered the NICE scoping process, of which 94 percent were prioritized. As of November 2021, approximately 39 percent of prioritized topics were in scoping/in progress, 31 percent were proposed/completed, 20 percent were suspended/terminated, and 4 percent were referred back to Innovation Observatory (IO) for further monitoring.

Our work demonstrates that horizon scanning for HTA is a complex and time-intensive process. Timelines and progress through HTA is challenging due to the growing number of innovative medicines, significant uncertainties, and limited transparency in clinical development and regulatory pathways. A better understanding of clinical trials and regulatory requirements may help eliminate some of this uncertainty and improve timely HTA.

Health technology assessment (HTA) is an area that remains less implemented in low- and lower middle-income countries. The aim of the study is to understand the perceptions of stakeholders in Uganda toward HTA and its role in decision making, in order to inform its potential implementation in the country.

The study takes a cross-sectional mixed methods approach, utilizing an adapted version of the International Decision Support Initiative questionnaire with both semi-structured and open-ended questions. We interviewed thirty key informants from different stakeholder institutions in Uganda that support policy and decision making in the health sector.

All participants perceived HTA as an important tool for decision making. Allocative efficiency was regarded as the most important use of HTA receiving the highest average score (8.8 out of 10), followed by quality of healthcare (7.8/10), transparency (7.6/10), budget control (7.5/10), and equity (6.5/10). There was concern that some of the uses of HTA may not be achieved in reality if there was political interference during the HTA process. The study participants identified development partners as the most likely potential users of HTA (66.7 percent of participants), followed by Ministry of Health (43.3 percent).

Interviewed stakeholders in Uganda viewed the role of HTA positively, suggesting that there exists a promising environment for the establishment and operationalization of HTA as a tool for decision making within the health sector. However, sustainable development and application of HTA in Uganda will require adequate capacity both to undertake HTAs and to support their use and uptake.

Patients with diabetes have a higher risk of developing chronic kidney disease (CKD). Early detection of CKD through microalbuminuria screening, followed by treatment, delays the progression of CKD. We evaluated the cost-effectiveness of population-based screening of microalbuminuria among normotensive type 2 diabetes mellitus patients aged >40 years compared with no screening scenario using a decision tree combined with the Markov model.

We considered two scenarios: Scenario I – dipstick microalbuminuria followed by spot-urine albumin–creatinine ratio (ACR) and serum creatinine in sequence; Scenario II – spot urine ACR plus serum creatinine. A mathematical cohort of the target population was simulated over a lifetime horizon with an annual cycle. Data for the model were obtained from secondary resources. The incremental cost-effectiveness ratios (ICERs) were estimated for screening scenarios compared to nonscreening scenario, along with sensitivity analyses.

The discounted ICER per quality-adjusted life years gained for annual microalbuminuria screening in the normotensive diabetic population in India were ₹ 24,114 (US$ 308) and ₹ 13,790 (US$ 176) for scenarios I and II, respectively. Annual screening by scenarios I and II resulted in a reduction of 180 and 193 end-stage renal disease (ESRD) cases per 100,000 population, respectively, resulting in a cost saving of ₹ 12.3 and 13.3 Crore spent on ESRD management over 10 years. Both scenarios were also cost-effective even at the screening frequencies of 5 and 10 yearly.

Microalbuminuria screening was cost-effective at the threshold of one-time GDP per capita in India.

Health technology assessment (HTA) traditionally informs decision making for single health technologies, which could lead to ill-informed decisions, suboptimal care, and system inefficiencies. We explored opportunities for conceptualizing the decision space in HTA as a disease management question versus an intervention management question.

Semistructured interviews were conducted between April 2022 and October 2022 with purposefully selected individuals from national and provincial HTA agencies and related organizations in Canada. We conducted manual line by line coding of data informed by our interview guide and sensitizing concepts from the literature. One author coded the data, and findings were independently verified by a second author who coded a subset of transcripts

Twenty-four invitations were distributed, and eighteen individuals agreed to participate. A disease management approach to HTA was differentiated from traditional approaches as being disease-based, multi-interventional, and dynamic. There was general support for an explicit care pathway approach to HTA by informing discussions around patient choice and suboptimal care, creating a space where decision makers can collaborate on shared objectives, and in setting up a platform for open dialogue about managing high-cost and high-severity diseases. There are opportunities for a care pathway approach to be implemented that build on the strengths of the existing HTA system in Canada.

A disease management approach may enhance the impact of HTA by supporting dynamic decision making that could better inform a proactive, resilient, and sustainable healthcare system in Canada.

This article presents the mapping of horizons scanning systems (HSS) for medical devices, conducted by the Medical Devices Working Group of the International Horizon Scanning Initiative (IHSI MDWG). It provides an overview of the identified HSS, highlights similarities and differences between the systems, and lessons learned.

Potentially relevant HSS were identified through literature searches, scan of an overview of EuroScan members, and input from the IHSI MDWG members. Structured information was collected from organizations that confirmed having an HSS for medical devices.

Sixteen initiatives could be identified, of which 11 are currently ongoing. The purposes of the HSS range from raising awareness of trends and new developments to managing informed decisions on innovative health services in hospitals. The time-horizon is most often 3 years up to a few months before market entry. Three models of identification of new technologies crystallized: a reactive (stakeholders outside HSS inform), a pro-active (actively searching multifold sources), and a hybrid model. Prioritization is often conducted by separate committees via scoring or debate. The outputs focus either on in-depth information of single technologies or on a class of technologies or on technologies in specific disease areas.

The identified HSS share the common experience that horizon scanning (HS) for medical devices is a resource-intensive exercise that requires a dedicated and skilled team. Insights into the identified HSS and their experiences will be used in the continued work of the IHSI MDWG on its proposal for an IHSI HSS for medical devices.

Limited health budgets and continual advancement of health technologies require mechanisms for prioritization. Israel, with a publicly funded health service basket, has implemented and optimized such a health technology assessment process since 1999.

We describe the process of evaluating technologies according to the Israeli model, analyze its outputs and benefits over two decades of implementation, and compare its key features with international experience.

Retrospective data were collected between 1998 and 2023, including work processes, committee composition, number of applications submitted and approved by a clinical domain, and yearly cost of the basket. Features were evaluated within the evidence-informed deliberative process (EDP) framework.

This national model involves relevant stake holders in a participatory and transparent process, in a timely manner, and is accepted by the public, health professionals, and policy makers, facilitating early adoption of the newest medical technologies. Between 11 and 19 percent of applications are approved for reimbursement annually, mostly pharmaceuticals. On average 26 percent of approved technologies are added to the list without additional budget. Major domains of approved technologies were oncology, cardiology, and neurology.

Israel created a unique model for the expansion of the health service basket. Despite an increasing number of applications and rising costs, the mechanism enables a consensus to be reached on which technologies to fund, while remaining within budget constraints and facilitating immediate implementation. The process, which prioritizes transparency and stake holder involvement, allows just a resource allocation while maximizing the adoption of novel technologies, contributing to an outstanding national level of health despite relatively low health spending.

Germany’s 2019 Digital Healthcare Act (Digitale-Versorgung-Gesetz, or DVG) created a number of opportunities for the digital transformation of the healthcare delivery system. Key among these was the creation of a reimbursement pathway for patient-centered digital health applications (digitale Gesundheitsanwendungen, or DiGA). Worldwide, this is the first structured pathway for “prescribable” health applications at scale. As of October 10, 2023, 49 DiGA were listed in the official directory maintained by Germany’s Federal Institute for Drugs and Medical Devices (BfArM); these are prescribable by physicians and psychotherapists and reimbursed by the German statutory health insurance system for all its 73 million beneficiaries. Looking ahead, a major challenge facing DiGA manufacturers will be the generation of the evidence required for ongoing price negotiations and reimbursement. Current health technology assessment (HTA) methods will need to be adapted for DiGA.

We describe the core issues that distinguish HTA in this setting: (i) explicit allowance for more flexible research designs, (ii) the nature of initial evidence generation, which can be delivered (in its final form) up to one year after becoming reimbursable, and (iii) the dynamic nature of both product development and product evaluation. We present the digital health applications in the German DiGA scheme as a case study and highlight the role of RWE in the successful evaluation of DiGA on an ongoing basis.

When a DiGA is likely to be updated and assessed regularly, full-scale RCTs are infeasible; we therefore make the case for using real-world data and real-world evidence (RWE) for dynamic HTAs.

Continous evaluation using RWD is a regulatory innovation that can help improve the quality of DiGAs on the market.

The Health Technology Assessment (HTA) process aims to optimize health system funding of technologies. In recent years there has been an increase in what is known as Real-World Evidence (RWE) as a complement to clinical trials. The objective of Health Technology Assessment International’s Latin American Policy Forum 2022 was to explore the utility of incorporating RWE into HTA and decision-making processes in the region.

This article is based on a background document, survey, and the deliberative work of the country representatives who participated in the Forum.

There is a growing interest in the use of Real-World Data / Real-World Evidence in HTA processes in Latin America, although currently there are no specific local guidelines for RWE use by HTA agencies. At present, its use is limited to certain areas such as adding context to HTA reports, the evaluation of adverse events, or cost estimation.

Potential future uses of RWE were identified, including the creation of risk-sharing agreements, the assessment of technology performance in routine practice, providing information on outcomes that are not so easily evaluated in clinical trials (e.g., the identification of specific subpopulations or quality of life), and the estimation of input parameters for economic evaluations.

The participants agreed that there are several areas presenting significant potential to expand the application of RWD/RWE and that the development of normative frameworks for its use could be helpful.

Published evidence on health service interventions should inform decision-making in local health services, but primary effectiveness studies and cost-effectiveness analyses are unlikely to reflect contexts other than those in which the evaluations were undertaken. A ten-step framework was developed and applied to use published evidence as the basis for local-level economic evaluations that estimate the expected costs and effects of new service intervention options in specific local contexts.

Working with a multidisciplinary group of local clinicians, the framework was applied to evaluate intervention options for preventing hospital-acquired hypoglycemia. The framework included: clinical audit and analyses of local health systems data to understand the local context and estimate baseline event rates; pragmatic literature review to identify evidence on relevant intervention options; expert elicitation to adjust published intervention effect estimates to reflect the local context; and modeling to synthesize and calibrate data derived from the disparate data sources.

From forty-seven studies identified in the literature review, the working group selected three interventions for evaluation. The local-level economic evaluation generated estimates of intervention costs and a range of cost, capacity and patient outcome-related consequences, which informed working group recommendations to implement two of the interventions.

The applied framework for modeled local-level economic evaluation was valued by local stakeholders, in particular the structured, formal approach to identifying and interpreting published evidence alongside local data. Key methodological issues included the handling of alternative reported outcomes and the elicitation of the expected intervention effects in the local context.

The adoption of genomic technologies in the context of hospital-based health technology assessment presents multiple practical and organizational challenges.

This study aimed to assist the Instituto Português de Oncologia de Lisboa Francisco Gentil (IPO Lisboa) decision makers in analyzing which acute myeloid leukemia (AML) genomic panel contracting strategies had the highest value-for-money.

A tailored, three-step approach was developed, which included: mapping clinical pathways of AML patients, building a multicriteria value model using the MACBETH approach to evaluate each genomic testing contracting strategy, and estimating the cost of each strategy through Monte Carlo simulation modeling. The value-for-money of three contracting strategies – “Standard of care (S1),” “FoundationOne Heme test (S2),” and “New diagnostic test infrastructure (S3)” – was then analyzed through strategy landscape and value-for-money graphs.

Implementing a larger gene panel (S2) and investing in a new diagnostic test infrastructure (S3) were shown to generate extra value, but also to entail extra costs in comparison with the standard of care, with the extra value being explained by making available additional genetic information that enables more personalized treatment and patient monitoring (S2 and S3), access to a broader range of clinical trials (S2), and more complete databases to potentiate research (S3).

The proposed multimethodology provided IPO Lisboa decision makers with comprehensive and insightful information regarding each strategy’s value-for-money, enabling an informed discussion on whether to move from the current Strategy S1 to other competing strategies.