International Journal of Technology Assessment in Health Care最新文献

Objectives: The National Institute for Health and Care Excellence (NICE) in England introduced early value assessments (EVAs) as an evidence-based method of accelerating access to promising health technologies that could address unmet needs and contribute to the National Health Service's Long Term Plan. However, there are currently no published works considering differences and commonalities in methods used between Assessment Reports for EVAs.

Methods: This rapid scoping review included all completed EVAs published on the NICE website up to 23 July 2024. One reviewer screened potentially relevant records for eligibility, checked by a second reviewer. Pairs of independent reviewers extracted information on the methods used in included EVAs using a prepiloted form; these were checked for accuracy. Data were described in graphical or tabular format with an accompanying narrative summary.

Results: In total, seventeen EVA Reports of sixteen EVAs were included in this scoping review. Five Reports did not specify how many reviewers undertook screening, whereas five did not report data extraction methods. Five EVAs planned to conduct meta-analyses, nine planned narrative syntheses, and seven planned narrative summaries. Eleven conceptual decision models were presented, with available evidence used to construct cost-utility analyses (N = 5); cost-effectiveness analyses (CEAs; N = 4); a mix of CEAs and cost-consequence analyses (CCA; N = 2); one CCA; and one cost-comparison.

Conclusion: Future EVA Reports should enhance the transparency of the methods used. Furthermore, EVAs could provide opportunities for the adoption of innovative methodological approaches and more flexible communication between EVA authors and key stakeholders, including patients and clinicians, companies, and NICE.

Introduction: Horizon scanning (HS) is a methodology that aims to capture signals and trends that highlight future opportunities and challenges. The National Institute for Health and Care Research (NIHR) Innovation Observatory routinely scans for medical technologies and therapeutics to inform policy and practice for healthcare in the United Kingdom (UK). To date, there is no standardized terminology for horizon scanning in healthcare. Here, we discuss the development of a data glossary and the IOAtlas web app.

Methods: We extracted data points from 4 years' worth of NIHR Innovation Observatory HS projects and collated them by technology type and descriptive family. A source repository was established by extracting a list of all sources used in NIHR Innovation Observatory briefing notes between 2017 and 2021. The repository was validated by external HS organizations and experts, and sources were then mapped to the appropriate time horizons. The glossary and repository were converted to an SQLite database format and connected to a free web app, IOAtlas.

Results: After de-duplication and consolidation, a total of 148 data points were included in the glossary. The source repository consists of 149 sources, with 99 percent being compliant with searching for two or more technology types. The final SQLite database contained 35 tables with 36 relationships.

Conclusions: We present a data glossary to provide globalized standardization for the terminology used in HS projects. The glossary can be accessed through the IOAtlas web app. Furthermore, we provide users with an interface to generate downloadable data extraction templates within IOAtlas.

Objectives: The UK Early Access to Medicines Scheme (EAMS), launched in 2014, enables pre-license access to medicines for areas of high unmet medical need. This study aimed to evaluate the outcomes of the scheme by analyzing subsequent marketing authorization (MA), health technology assessment (HTA), and commissioning decisions.

Methods: We conducted a retrospective analysis of all completed EAMS programs from 2014 to April 2025, reviewing MA, HTA, and commissioning outcomes.

Results: Fifty-one EAMS programs were completed, over half in oncology. Median times from Scientific Opinion (SO) to MA, and reimbursement outcomes in England and Scotland were 4.3 (Q1: 2.6 and Q3: 7.3), 14.5 (Q1: 9.4 and Q3: 20.9), and 15.0 months (Q1: 11.4 and Q3: 18.1), respectively. Of 48 products appraised by the National Institute for Health and Care Excellence (NICE) or National Health Service (NHS) England, 50 percent received positive recommendations, 44 percent were optimized, and 6 percent were rejected. Of 45 products appraised by the Scottish Medicines Consortium or NHS Scotland, 73 percent were positive, 18 percent optimized, and 9 percent rejected. EAMS was qualitatively referenced in 48 percent of NICE appraisals and quantitatively in 18 percent.

Conclusions: Compared to non-EAMS products, those entering the scheme achieve faster MA and HTA timelines and higher regulatory success. However, EAMS is referenced quantitatively in less than a fifth of NICE appraisals, and fewer than half of Promising Innovative Medicine designations progress to a full SO. Administrative burdens, data demands, and liability concerns may limit uptake; addressing these barriers could enhance the scheme's impact.

Objectives: Patient involvement enhances transparency, legitimacy, and responsiveness in pharmaceutical reimbursement decisions. Guided by the mosaic model, this study recognizes effective patient engagement requires diverse context-specific approaches. Despite Taiwan's National Health Insurance Administration (NHIA) implementing policies, gaps remain between intent and practice. This study evaluates NHIA's incorporation of patient inputs into reimbursement decisions and examines factors influencing involvement.

Methods: We analyzed pharmaceutical company-initiated reimbursement submissions for catastrophic illnesses reviewed by the Pharmaceutical Benefit and Reimbursement Scheme Joint Committee (PBRS) from 2016 to 2023. Data sources included PBRS meeting records, the Online Patient Opinion Platform (OPOP), and NHIA notification E-mails. Generalized linear models identified predictors of patient involvement. The association between patient involvement and PBRS decisions was also explored.

Results: Patient involvement occurred in 28.4 percent (80/282) of all submissions, increasing from 17 percent (2016) to 44 percent (2023). Despite aligning with OPOP criteria, patient involvement remained incomplete. Discussion-type submissions, oncology drugs, and new drug applications showed higher involvement, whereas autoimmune diseases and new indication submissions had lower involvement. Budget impact and innovation categories were not significant predictors in adjusted models. The presence of patient involvement was not significantly associated with the PBRS approval rate. Ad hoc analysis revealed increased involvement for new indications following policy expansion.

Conclusions: Despite NHIA's efforts, patient involvement implementation remains suboptimal. Structured mechanisms and expanded patient involvement beyond high-profile submissions and PBRS are crucial to broaden patient involvement. This study provides practical insights for East Asian healthcare systems advancing patient involvement amid limited empirical research.

Collaboration is both a process and an outcome. Collaboration is based on the idea that interactions between participants with a common goal, working together as partnerships and sharing resources, can solve complex or "wicked" problems that are not possible to solve in isolation. Collaboration may be simple, occurring between individuals, or more complex interorganizational arrangements across sectors, with the life cycle and size of the collaboration determined by the issue at hand. HTA collaborations may involve a wide range of stakeholders, including HTA agencies at the national, regional, or global level, academia, government (including regulatory authorities), industry, clinicians, providers, and patient organizations. Regardless of the number or type of participants, collaborations need a shared understanding of the common goal, an agreement on aims, and a commitment to shared solutions.Industry and agency members of the Health Technology Assessment International (HTAi) Asia Policy Forum (APF) met in Seoul, South Korea, in November 2024 for open discussions on how to facilitate and improve the collaborative process between all stakeholders in the health system, including government, HTA agencies, industry, academia, clinicians, as well as patients. Over the three days, these discussions identified some of the risks and obstacles to collaboration in the region, how to develop and use collaboration better, as well as articulating the value and benefits of collaboration both in the region and globally.

Objectives: Evaluate the extent to which delivery constraints were considered during the health technology assessment (HTA) of cell and gene therapies.

Methods: Constraints on delivering cell and gene therapies were identified from guidance documents by the National Institute for Health and Care Excellence Technology Appraisal and Highly Specialised Technologies streams until October 2024. Inductive coding was performed to identify delivery constraints reported within the guidance documents. A quantitative analysis established the proportion of guidance documents that reported delivery constraints, and the distribution of these constraints across the guidance documents (frequency, mean range).

Results: Sixteen guidance documents for cell and gene therapies were identified. Thirteen guidance documents (81.3 percent of the sample) reported constraints on delivering cell and gene therapies. Thirty-one examples of delivery constraints were reported. The mean number of constraints per guidance document was 1.9 (range: 0-6 constraints). The reported constraints were grouped by six different themes: provider experience (n = 8); testing constraints (n = 7); geographical constraints (n = 5); payment constraints (n = 5); maturity of developments in care (n = 4); and infrastructure constraints (n = 2).

Conclusion: Formal HTA processes are one effective way to identify constraints on delivering cell and gene therapies. Proactive identification of potential delivery constraints will help decision-makers, providers, and manufacturers generate strategies that improve the implementation of cell and gene therapies. Overcoming delivery constraints will strengthen the likelihood of realizing the expected incremental net health benefit of cost-effective cell and gene therapies for patients across a healthcare system.

Objective: This investigation evaluates the relationships between claims of patient and health system benefit, evidence in support of those claims, and the recommendation outcomes of medical technologies assessed by the National Institute for Health and Care Excellence (NICE).

Methodology: Data on evidence, claims, and recommendation outcomes were gathered from published Medical Technologies Guidances (MTGs) on the NICE Web site between 1 December 2010 and 11 April 2023. Binary logistic regressions and descriptive data analyses were performed to investigate the correlation between claims, evidence, and recommendation outcomes.

Results: The technology was fully or partially recommended in forty-six (67.7 percent) of sixty-eight MTGs. No correlation was found between types and number of claims and type and quantity of clinical evidence. However, claims supported directly by evidence were significantly correlated (p < 0.016) with recommendation.

Conclusion: Evidence supporting claims is crucial for receiving a full or partial guidance recommendation. There is no clear pattern in what kind of or quantity of evidence leads to a recommendation, and to increase the probability of receiving a favorable recommendation, the manufacturer needs to plan early in the development phases on how to articulate and refine the claims and to substantiate claims through clinical evidence. It is therefore advisable to take advantage of the opportunity for scientific advice, which NICE offers.

Patient involvement is an increasingly recognized cornerstone of effective Health Technology Assessment (HTA). Clear, accessible information empowers patient organizations to contribute meaningfully to HTA. Therefore, an international Summary Information for Patient Groups template was developed to provide plain language summaries of new medicines being assessed. Pilots using the template were conducted in Australia in 2021 and England in 2022, providing a trial within the HTA process. In Australia, the Consumer Evidence and Engagement Unit (CEEU) used a workshop and survey, together with key stakeholder interviews, to gather feedback. In England, the National Institute for Health and Care Excellence used public consultation, surveys, and a Short-Life Working Group (SLWG). An advisory board with patient organizations provided additional insights. The feedback enabled members of the HTA International Patient and Citizen Involvement in HTA Interest Group to evaluate the potential to enhance patient organization submissions to HTA bodies and to provide recommendations on the template's implementation in HTA processes. The pilots highlighted that plain language summaries increased confidence and reduced preparation time for patient organization input to HTA. Other nonexpert stakeholders also found them valuable for fostering understanding. However, challenges remain, including mitigating bias in completed templates, allocating sufficient resources, and integrating into existing processes. The evaluation concludes that the approach holds significant potential to enhance patient organization involvement in HTA. Recommendations include setting up multi-stakeholder SLWGs, ensuring early access to summaries, and aligning implementation with local regulations. These insights provide guidance for HTA bodies to develop an approach to support patient involvement.