HTA capacity building in Asia: towards one goal.

OBJECTIVES: The aim of the 2022 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) was to discuss experiences and challenges around health technology assessment (HTA) capacity building for both HTA agencies and companies in the Asia region and to identify possible solutions as part of a capacity building roadmap. METHODS: Discussions during the 2022 APF, informed by a pre-meeting survey of HTA agencies and industry attendees from the region, form the basis of this paper. RESULTS: HTA is an essential element of priority-setting in healthcare; however, the scarcity of skilled technical HTA practitioners is a rate-limiting step in the conduct of HTA. The lack of investment in HTA and the political will to mandate the use of HTA in decision-making may be due to a lack of understanding of the value of the HTA process, and how HTA is interpreted and used in the healthcare decision-making process. CONCLUSIONS: Increased demand for HTA is created when the value of HTA is recognized. HTA capacity-building challenges may be mitigated by educating stakeholders, particularly policymakers, on the value of, and the need to invest in, HTA as a transparent process to ensure equitable access to health care for all. Investigating a means of funding and implementing an HTA intern program between agencies, in partnership with industry, to facilitate a supportive environment to foster HTA skills and knowledge, build capacity or strengthen existing capacity should be a priority.

"What's Keeping Me Up at Night?" Reflections on the COVID-19 Pandemic in Asia.

OBJECTIVES: The 2021 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) aimed to explore the challenges and opportunities presented by the global COVID-19 pandemic for health systems and industry in the Asia region, to discuss how HTA changed during the pandemic, and what its role may be moving forward into a postpandemic era. METHODS: Discussions during the virtual 2021 APF, informed by a premeeting survey of HTA agencies and industry attendees from the region describing their experience during the pandemic, form the basis of this paper. RESULTS: During the pandemic, regulatory approval for COVID-related technologies was fast-tracked with fewer evidentiary requirements, and little or no HTA being conducted before these technologies were implemented in health systems in the region. "Living HTA" incorporating real-world evidence (RWE) as it was generated became part of the HTA landscape. In comparison, non-COVID technologies experienced regulatory approval and HTA delays. A major concern of APF members was future pandemic preparedness, and to ensure that lessons are learned from COVID-19. Governments need to continue to invest in innovation and allow early regulatory approvals with the increased use of RWE. Capacity building was identified as a key investment, including capacity in HTA, modeling, as well as local production of necessary supplies and equipment. Finally, collaboration at all levels of the health system was viewed as vital. CONCLUSIONS: Post-COVID, different forms of HTA must be embraced as part of the new HTA landscape in addition to increased intra- and intercountry collaboration across all sectors of the health system, including regulatory bodies.

Ferric Carboxymaltose for Anemic Perioperative Populations: A Systematic Literature Review of Randomized Controlled Trials.

IMPORTANCE: Perioperative anemia is a common comorbid condition associated with increased risk of morbidity and mortality in patients undergoing elective surgical procedures. OBJECTIVE: We conducted a systematic literature review (SLR) to determine the efficacy and safety of the use of intravenous ferric carboxymaltose (FCM) for the treatment of perioperative anemia in preoperative, intraoperative, and postoperative elective surgical care. EVIDENCE REVIEW: Studies meeting inclusion criteria for the SLR reported on treatment efficacy in an adult study population randomly allocated to FCM for the treatment of perioperative anemia during the perioperative period. After screening, 10 of 181 identified studies from searches in MEDLINE and EMBASE databases were identified for inclusion in this review. FINDINGS: Preoperative treatment was reported in six studies, intraoperative treatment in one study, postoperative treatment in two studies, and both pre- and postoperative treatment in one study. Together, 1975 patients were studied, of whom 943 were randomized to FCM, of whom 914 received FCM treatment. The 10 studies reported elective surgical populations for colorectal, gastric, orthopedic, abdominal, urologic, plastic, neck, gynecologic, and otolaryngologic procedures. Given the clinical and methodological heterogeneity of the studies, the analyses were limited to qualitative assessments without meta-analyses. All 10 studies reported statistically greater changes in hemoglobin concentration, serum ferritin, and/or transferrin saturation with FCM treatment compared with comparators (placebo, oral iron, standard care, or a combination of these). Two studies reported statistically significant differences in transfusion rate and 2 studies reported significant differences in length of hospital stay between FCM and its comparator(s). CONCLUSIONS AND RELEVANCE: This SLR adds to existing data that administration of FCM in preoperative and postoperative settings improves hematologic parameters. Several studies in the review supported the beneficial effects of FCM in reducing transfusion rate and length of stay. Larger, well-designed, longer-term studies may be needed to further establish the efficacy and safety of FCM in elective surgery patients with perioperative anemia.

Randomized Placebo-Controlled Trial of Ferric Carboxymaltose in Heart Failure With Iron Deficiency: Rationale and Design.

BACKGROUND: Iron deficiency (ID) has a prevalence of ≈40% to 50% among patients in heart failure (HF) with reduced ejection fraction and is associated with worse prognosis. Several trials demonstrated that intravenous ferric carboxymaltose leads to early and sustained improvement in patient-reported outcomes and functional capacity in patients with HF with reduced ejection fraction with ID, yet morbidity and mortality data are limited. METHODS: The objective of the HEART-FID trial (Ferric Carboxymaltose in Heart Failure With Iron Deficiency) is to assess efficacy and safety of ferric carboxymaltose compared with placebo as treatment for symptomatic HF with reduced ejection fraction with ID. HEART-FID is a multicenter, randomized, double-blind, placebo-controlled trial enrolling ≈3014 patients at ≈300 international centers. Eligible patients are aged ≥18 years in stable chronic HF with New York Heart Association functional class II to IV symptoms, ejection fraction ≤40%, ID (ferritin <100 ng/mL or ferritin 100-300 ng/mL with a transferrin saturation <20%), and documented HF hospitalization or elevated N-terminal pro-brain natriuretic peptide. Consented patients are assigned to ferric carboxymaltose or placebo at baseline, with repeated visits/assessments every 6 months for additional study drug based on hemoglobin and iron indices for the trial duration. The primary end point is a hierarchical composite of death and HF hospitalization at 12 months and change from baseline to 6 months in the 6-minute walk test distance. CONCLUSIONS: The HEART-FID trial will inform clinical practice by clarifying the role of long-term treatment with intravenous ferric carboxymaltose, added to usual care, in ambulatory patients with symptomatic HF with reduced ejection fraction with ID. Registration: URL: https://www.clinicaltrials.gov; Unique identifier: NCT03037931.

Sustainability of healthcare systems in Asia: exploring the roles of horizon scanning and reassessment in the health technology assessment landscape.

OBJECTIVES: The 2019 Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) discussed the role of horizon scanning (HS) and health technology reassessment (HTR) in supporting sustainable healthcare in Asia. METHODS: Discussions and presentations at the 2019 HAPF, informed by a literature review, results of a premeeting survey, and case studies of HTA agencies from the region form the basis of this paper. RESULTS: Five of the fourteen HTA agencies surveyed have established or are developing HS systems (HSSs), and six reported some experience with HTR. Although there were many differences in the respective HSSs, all were proactive and identified technologies within a 1-3-year time horizon. Commonalities included the criteria used to prioritize the technologies including burden and rarity of the disease, and cost to the health system and patients. Experience in HTR was not as extensive with most HTR activity being conducted on an ad hoc basis. Challenges for both HS and HTR include a lack of HTA capacity and good quality data, as well as a need for transparency and collaboration with industry. CONCLUSIONS: With developing universal health care and HTA capacity in many countries in Asia, HS and HTR are in their infancy. Whilst several countries in the region are experienced in all facets of HTA, other countries are developing their HTA skill base. As such, there was a clear appetite for setting up a collaborative network in the Asia region to facilitate information sharing around HTA activities, and more specifically, HS and HTR methodologies, experiences, and assessments.

Outcomes of extended-spectrum beta-lactamases producing Enterobacteriaceae colonization among patients abdominal surgery patients.

Preoperative enteric screening for extended-spectrum ß-lactamase (ESBL)-producing Enterobacteriaceae was conducted in 360 patients prospectively observed for surgical site infection (SSI). ESBL colonization (adjusted odds ratio [aOR], 2.4) and dirty wound classification (aOR, 3.6) were associated with SSI; no association between carbapenem prophylaxis and reduction in SSI was detected.

Thinking outside the system: the integrated care experience in Queensland, Australia.

Health policy-makers are faced with a demand for health care that exceeds supply, driven in part by an ageing population and an increased prevalence of chronic disease. An integrated 'people-centred' model of care across primary, secondary and tertiary health care can strengthen the health system by streamlining services to improve the patient journey and outcomes. Integrated care offers an opportunity to reduce admissions and re-admissions to hospitals, reduce presentations to emergency departments and improve the capacity of outpatient clinics while delivering better outcomes to patients. Queensland's $35 million Integrated Care Innovation Fund (ICIF) has provided seed funding to 24 projects covering a wide spectrum of chronic disease management and complex disease. Programs such as the ICIF offer an opportunity to provide a new approach to caring for vulnerable populations such as the frail and elderly; children with behavioural and developmental issues; children in out-of-home care; rural and remote populations; and people with mental health issues, whose care runs the risk of 'falling through the cracks' with conventional healthcare approaches.

Improving Access to High-Cost Technologies in the Asia Region.

OBJECTIVES: Discussions at the Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) aimed to understand the meaning of "high-cost technologies," and to explore mechanisms to increase access to these technologies in publicly funded health systems in the Asia region. METHODS: Discussions and presentations at the 2018 HAPF, informed by a literature review and a premeeting survey of HTA agencies and industry, form the basis of this paper. RESULTS: Challenges payers in the public health system face when investing in high-cost technologies include a lack of data, especially real-world data, affordability, and the budgetary impact of high-cost technologies. Managed entry schemes (MES) are one means to enable earlier access to high-cost technologies, or at reduced cost to the system. Most countries surveyed had used an MES to introduce a new health technology and most industry representatives had experience with financial-based MES, such as discounts or rebates, with most put in place to increase access to pharmaceuticals. Little experience of outcome-based or evidence-generation MES was reported. CONCLUSIONS: Although it is early days in the implementation of MES in Asia, they have the potential to play an important role enabling access to new, mainly pharmaceutical, health technologies. The development of a "road map" of MES in the region should outline the intent and need for a MES, articulating the "rules of engagement" for all stakeholders-patients, providers, payers, and industry-which will assist countries to clearly identify the problem trying to be solved, and how an MES can be part of the solution.

Fostering healthcare innovation in public hospitals: the Queensland experience.

Faced with scarce resources and a demand for health care that exceeds supply, health policy makers at all levels of government need to adopt some form of rationing when deciding which health services should be funded in the public health system. With a relatively small investment, programs such as Queensland Health's New Technology Funding Evaluation Program (NTFEP) fosters innovation by providing funding and pilot studies for new and innovative healthcare technologies. The NTFEP assists policy makers to make informed decisions regarding investments in new safe and effective technologies based on available evidence gathered from real-world settings relevant to Queensland patients and clinicians. In addition, the NTFEP allows appropriate patient access, especially in rural and remote locations, to potentially beneficial technologies and acts a gatekeeper, protecting them from technologies that may be detrimental or harmful.

Phase 3 adaptive trial design options in treatment of complicated urinary tract infection.

New antimicrobial drugs for treatment of complicated urinary tract infection (cUTI) are generally assessed in randomized, double-blind, noninferiority clinical trials. Robust historical data for the active comparator inform on treatment effect estimation, yet typically do not substitute for the active comparator data in the proposed trial. We report design options for a phase 3 trial of cUTI using a Bayesian hierarchical model and historical data from 2 well-executed phase 3 registrational trials of doripenem. The methodology is directly applicable to other phase 3 noninferiority settings. In addition to the research design application, we provide a novel methodology for assessing the robustness of type I error control. The model borrows heavily from the prior data when the current active comparator parameter estimate approximated the historical estimate. In contrast, the model had restricted borrowing when the 2 estimates were very different. The alternative trial design, with or without the inclusion of futility stopping criteria, provides a framework for future cUTI phase 3 trials.

OVERCOMING THE BARRIERS TO ACHIEVING UNIVERSAL HEALTH CARE IN THE ASIAN REGION.

OBJECTIVES: The Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) met to discuss the challenges of achieving universal health care (UHC) in Asia. METHODS: Group discussions and presentations at the 2017 HAPF, informed by a background paper, including a literature review and the results of pre-meeting surveys of health technology assessment (HTA) agencies and industry, formed the basis of this article. RESULTS: Affordability was identified as the greatest barrier to establishing UHC; however, other impediments include the lack of political will to implement UHC, and the cultural issue of deference to expert opinion instead of evidence-based assessments. Although HTA was identified as an important prioritization tool when adding new technologies to benefit packages, it is used inconsistently in the region, resulting in a less transparent decision-making process for stakeholders. Although regional challenges exist around real-world data (RWD), including a lack of capacity to enable information and data sharing, most policy or funding decision makers in the region have access to data. However, there appears to be a disconnect with the experience of industry, whose representatives identify the lack of RWD as their primary issue. To overcome these issues, both HTA agencies and industry agree that collaboration and transparency should be fostered to support the development of robust evidence generation in the region. CONCLUSIONS: There is a willingness for HTA agencies and industry to collaborate to develop HTA methodology for the prioritization of technologies in the Asia region that support healthcare systems to achieve the ultimate outcome of UHC.

Community-Acquired Pneumonia Case Validation in an Anonymized Electronic Medical Record-Linked Expert System.

An electronic anonymized patient portal analysis using radiographic reports and admission and discharge diagnoses had sensitivity, specificity, positive predictive value, and negative predictive value of 84.7%, 78.2%, 75%, and 87%, respectively, for community-acquired pneumonia validated against a blinded expert medical review. This approach can help to track antimicrobial use and resistance.

Platelet-rich plasma: a case study for the identification of disinvestment opportunities using horizon scanning.

Objective This paper discusses the potential for horizon scanning to identify low-value, inappropriate clinical practices that deliver minimal benefit to patients and represent a considerable financial burden on the health system. Methods Platelet-rich plasma (PRP) was identified by routine horizon scanning as a potentially innovative treatment alternative for osteoarthritis of the knee. A rapid, non-systematic assessment of the evidence pertaining to the safety and effectiveness of PRP compared with nonsteroidal anti-inflammatory drugs (NSAIDs) for the treatment of osteoarthritis of the knee was conducted. Results The evidence base supporting the use of PRP for the treatment of osteoarthritis was poor. No comparative studies were identified that compared the use of PRP to NSAIDs, the current treatment option for osteoarthritis of the knee in Australia. Despite the lack of effectiveness evidence, the use of PRP injections was rapidly increasing in the private sector using an inappropriate Medicare Benefits Schedule item number. Conclusions This assessment highlights the potential of using established horizon scanning methodologies to identify targets for full or partial disinvestment of ineffective, inefficient or harmful clinical practices. What is known about the topic? PRP is rapidly diffusing in the private health system in Australia, however the use of a Medicare Benefits Schedule item number meant that this practice was being subsidised by the public reimbursement of treatment fees. What does this paper add? Traditional horizon scanning tends to identify technologies for health systems to invest in. The evidence on the effectiveness of PRP was examined with the purpose of exploring investment in an innovative treatment that may have reduced the number of invasive procedures being performed in the public hospital system. The current evidence base does not support the use of PRP injections for the treatment of osteoarthritis. It does, however, support the use of horizon scanning as an inexpensive methodology to identify possible disinvestment targets associated with potential patient harm and high health service expenditure. What are the implications for practitioners? Practitioners should be aware that public funding for the injection of PRP should not be used for the treatment of osteoarthritis.