Assessment Of Cost-effectiveness Research Articles

Carboplatin desensitization in the era of target therapies: still worthwhile?

Unresectable pediatric low-grade gliomas (LGG) usually need adjuvant therapy, and carboplatin hypersensitivity reaction (HR) commonly leads to premature treatment cessation of a standard chemotherapy regimen. In the molecular era, advances in understanding tumor genetic characteristics allowed the development of targeted therapies for this group of tumors; however, cost-effectiveness assessment of treatments, especially in low-income countries, is crucial. The aim is to describe the results of carboplatin desensitization protocol in a single center in a middle-income country. Prospective analysis of children with LGG submitted to carboplatin desensitization from December 2017 to June 2020 with follow-up until April 2024. Nine patients were included. The mean age was 11years. Five patients were male. Seven had optic pathway and two cervicomedullary location. Six had histologic diagnosis and four molecular analyses. The incidence of carboplatin reactions during the study period was 39.1%. Six patients underwent skin prick test, three with positive results. The first HR occurred, on average, around the 9th cycle of treatment. All patients had cutaneous symptoms, and five out of nine had anaphylaxis as the first reaction. 77.7% of the patients completed the protocol, and the clinical benefit rate (stable disease and partial response) was 88.8%. Six patients further required other lines of therapy. Monthly, the total cost for carboplatin was $409.09, and for target therapies (dabrafenib plus trametinib), $4929.28 to $5548.57. Our study presented an interesting and cost-effective option where desensitization allowed children with HR to be treated with first-line therapy, avoiding the discontinuation of an effective treatment.

The Risk Of Perpetuating Health Disparities Through Cost-Effectiveness Analyses.

Cost-effectiveness analyses are commonly used to inform health care and public health policy decisions. However, standard approaches may systematically disadvantage marginalized groups by incorporating assumptions of persisting health inequities. We examined how competing risks, baseline health care costs, and indirect costs can differentially affect cost-effectiveness analyses for racial and ethnic minority populations. We illustrate that these structural factors can reduce estimated quality-adjusted life-years and cost savings for disadvantaged groups, making interventions focused on disadvantaged populations appear less cost-effective. For example, analyses of a sugar-sweetened beverage tax may estimate higher costs per quality-adjusted life-year gained for Black versus White populations because of differences in competing risks and insurance status that manifest in higher health care cost savings from averted disease among White people. To ensure that cost-effectiveness assessments do not perpetuate inequities, alternative approaches are needed that account for the impact of structural factors on different groups and that consider scenarios in which health inequities are reduced. Sensitivity analyses focusing on health equity could help advance interventions that disproportionately benefit disadvantaged communities.

Quantifying the effectiveness of an active treatment in improving highway-railway grade crossing safety in Canada: an empirical Bayes observational before–after study

Transportation engineers face challenging safety investment decisions, particularly for highway-railway grade crossings (HRGCs), where rare collision occurrences and incomplete historical records complicate the assessment of countermeasure cost-effectiveness. This study introduces an empirical Bayes (EB) observational before–after approach to address these challenges, specifically examining the impact of a widely used safety countermeasure: flashing lights, bells, and gates (FLBG). The research covers a total 171 FLBG crossings with pre-implementation conditions varying between standard reflective crossing signs and flashing lights and bells, utilizing collision data from 2000 to 2012 for analysis. This study is the first to substantiate quantitatively the effectiveness of FLBG treatment in improvising the safety of HRGCs and show that the effect is dependent of crossing characteristics, such as track angle, pavement conditions, and train speed limits. These findings can provide valuable support to decision-makers, enabling them to make more effective decisions for grade crossing safety improvement projects.

Financing and cost-effectiveness of emergency medical services in low- and middle-income countries

In 2023, the 76th World Health Assembly declared coordinated emergency, critical, and operative care services fundamental for comprehensive universal health coverage in low- and middle-income countries. With increasing mortality from noncommunicable diseases, an organized emergency care system has the capacity to treat a variety of conditions with a common set of resources, optimizing per-unit cost efficiency by applying economies of scope and increasing cost-effectiveness. However, the financing and cost-effectiveness of emergency medical services remain poorly understood despite affordability and financial barriers comprising some of the most significant obstacles to development. Cost-effectiveness analyses generate incremental cost-effectiveness ratios for comparison against per-capita gross domestic product thresholds to determine cost-effectiveness, promoted by the World Health Organization’s Choosing Interventions that are Cost-Effective program. Incremental cost-effectiveness ratios may be used as context-specific indicators of value alongside budget impact and feasibility considerations. Currently, there are few high-quality cost-effectiveness studies of emergency care in low- and middle-income countries, demonstrating significant methodologic heterogeneity, little geographic diversity, neglecting descriptions of assumptions used in cost-effectiveness calculations and comparators used, and lacking incremental cost-effectiveness ratios for comparison. The assessment of emergency care cost-effectiveness is challenging, given the significant breadth of conditions encountered and difficulty in projecting subsequent impact. Without improved epidemiologic surveillance and data-collection infrastructure, data inputs for cost-effectiveness calculations will remain limited. Future efforts should practice standard cost-effectiveness methodologies to permit comparison of incremental cost-effectiveness ratios across interventions and settings while incorporating trauma registry data to longitudinally track patient outcomes over sufficient time horizons to determine impact. New indices that expand the scope of analysis to capture broader secondary impacts of emergency care for future cost-effectiveness studies are needed. In this article, we summarize the key steps for economic evaluations for prehospital care systems and recommend considerations for future prehospital emergency care cost-effectiveness analyses, determining the optimal structure for financing mechanisms well-suited to resource-limited settings are critical for future investigation.

Uncertainty in Long-Term Relative Effectiveness of Medicines in Health Technology Assessment

ObjectivesUncertainty regarding the long-term relative effectiveness is an important factor in health technology assessment (HTA) of medicines. This study investigated how different HTA bodies address this uncertainty in their assessments. MethodsA total of 49 HTA reports from 6 national HTA bodies, assessing 9 medicines for spinal muscular atrophy, cystic fibrosis, and hypercholesterolemia, were included. In these reports, 81 relative effectiveness assessments and 45 cost-effectiveness assessments were performed on an indication level. We collected information on included trials, assessment outcomes, uncertainty regarding the long-term effectiveness, proposed managed entry agreements, and reassessments. ResultsUncertainty regarding the long-term effectiveness was an important consideration in almost all cost-effectiveness assessments (91%) and three-quarters of relative effectiveness assessments (74%), despite differences in methodologies among HTA bodies. There were considerable differences in the amount and type of long-term effectiveness data included by HTA bodies due to timing and inclusion criteria. In total 23 managed entry agreements were proposed of which 14 were linked to uncertainty regarding the long-term effectiveness. In addition, 13 reassessments were performed of which 4 led to an increase in patient access because of more available long-term effectiveness data. ConclusionsUncertainty regarding the long-term effectiveness is an important challenge for HTA bodies. There are large differences in the acceptance of evidence among HTA bodies, which leads to heterogeneity in the inclusion of available long-term effectiveness data for decision making. In cases with large uncertainty regarding the long-term effectiveness, outcome-based agreements and reassessments are used by HTA bodies, but differently between HTA bodies and indications.

Antifungal pipeline: Is there light at the end of the tunnel?

The misuse and overuse of classic antifungals have accelerated the development of resistance mechanisms, diminishing the efficacy of established therapeutic pathways and necessitating a shift towards alternative targets. Despite this pressing need for new treatments, the antifungal drug pipeline has been largely stagnant for the past three decades, primarily due to the high risks and costs associated with antifungal drug development, compounded by uncertain market returns. Extensive research durations, special patient populations and rigorous regulatory demands pose significant barriers to bringing novel antifungal agents to market. In response, the “push-pull” incentive model has emerged as a vital strategy to invigorate the pipeline and encourage innovation. This editorial critically examines the current clinical landscape and spotlights emerging antifungal agents, such as Fosmanogepix, Ibrexafungerp, and Olorofim, while also unraveling the multifaceted challenges faced in new antifungal drug development. The generation of novel antifungals offers a beacon of hope in the battle against antimicrobial resistance, but it is premature to declare them as definitive solutions. Their future role hinges on thorough clinical validation, cost-effectiveness assessments, and continuous post-marketing surveillance. Only through strategic implementation and integration with market strategies we can transform the landscape of antifungal development, addressing both the resistance crisis and the treatment challenges.

A comprehensive review of cell transplantation and platelet-rich plasma therapy for the treatment of disc degeneration-related back and neck pain: A systematic evidence-based analysis.

Low back pain (LBP) and neck pain predominate as the primary causes of disability. Cell- and platelet-rich plasma (PRP) products are potential therapies with clinical trials and reviews promoting their efficacy. Nonetheless, they frequently disregard the clinical significance of reported improvements. In this systematic review, the effectuated improvements in pain, disability, quality of life (QoL), and radiographic images are comprehensively described and scored on their clinical significance. An electronic database literature search was conducted on July 2023 for in-human assessment of cell or PRP products to alleviate discogenic pain. Papers were screened on quantitative pain, disability, QoL, radiographic improvements, and safety outcomes. Risk of bias was assessed through MINORS and Cochrane Source of Bias tools. Reported outcomes were obtained, calculated, and assessed to meet minimal clinically important difference (MCID) standards. From 7623 screened papers, a total of 80 articles met the eligibility criteria, presenting 68 specific studies. These presented at least 1974 treated patients. Overall, cell/PRP injections could alleviate pain and disability, resulting in MCID for pain and disability in up to a 2-year follow-up, similar to those observed in patients undergoing spinal fusion. Included trials predominantly presented high levels of bias, involved heterogeneous study designs, and only a minimal number of randomized controlled trials. Nonetheless, a clear clinically significant impact was observed for cell- and PRP-treated cohorts with overall good safety profiles. These results highlight a strong therapeutic potential but also underline the need for future cost-effectiveness assessments to determine the benefits of cell/PRP treatments.

Is a larger patient benefit always better in healthcare priority setting?

When considering the introduction of a new intervention in a budget constrained healthcare system, priority setting based on fair principles is fundamental. In many jurisdictions, a multi-criteria approach with several different considerations is employed, including severity and cost-effectiveness. Such multi-criteria approaches raise questions about how to balance different considerations against each other, and how to understand the logical or normative relations between them. For example, some jurisdictions make explicit reference to a large patient benefit as such a consideration. However, since patient benefit is part of a cost-effectiveness assessment it is not clear how to balance considerations of greater patient benefit against considerations of severity and cost-effectiveness. The aim of this paper is to explore the role of a large patient benefit as an independent criterion for priority setting in a healthcare system also considering severity and cost-effectiveness. By taking the opportunity cost of new interventions (i.e., the health forgone in patients already receiving treatment) into account, we argue that patient benefit has a complex relationship to priority setting. More specifically, it cannot be reasonably concluded that large patient benefits should be given priority if severity, cost-effectiveness, and opportunity costs are held constant. Since we cannot find general support for taking patient benefit into account as an independent criterion from any of the most discussed theories about distributive justice: utilitarianism, prioritarianism, telic egalitarianism and sufficientarianism, it is reasonable to avoid doing so. Hence, given the complexity of the role of patient benefit, we conclude that in priority practice, a large patient benefit should not be considered as an independent criterion, on top of considerations of severity and cost-effectiveness.

Effectiveness of aerobic training on adverse symptoms related to chemotherapy during treatment: protocol of a randomized controlled trial with cost-effectiveness assessment. (Preprint)

Effectiveness of aerobic training on adverse symptoms related to chemotherapy during treatment: protocol of a randomized controlled trial with cost-effectiveness assessment. (Preprint)

Reducing non-communicable diseases among Palestinian populations in Gaza: A participatory comparative and cost-effectiveness modeling assessment.

We sought to assess the effectiveness and cost-effectiveness of potential new public health and healthcare NCD risk reduction efforts among Palestinians in Gaza. We created a microsimulation model using: (i) a cross-sectional household survey of NCD risk factors among 4,576 Palestinian adults aged ≥40 years old in Gaza; (ii) a modified Delphi process among local public health experts to identify potentially feasible new interventions; and (iii) reviews of intervention cost and effectiveness, modified to the Gazan and refugee contexts. The survey revealed 28.6% tobacco smoking, a 40.4% prevalence of hypertension diagnosis (with a 95.6% medication treatment rate), a 25.6% prevalence of diabetes diagnosis (with 95.3% on treatment), a 21.9% prevalence of dyslipidemia (with 79.6% on a statin), and a 9.8% prevalence of asthma or chronic obstructive pulmonary disease (without known treatment). A calibrated model estimated a loss of 9,516 DALYs per 10,000 population over the 10-year policy horizon. The interventions having an incremental cost-effectiveness ratio (ICER) less than three times the GDP per capita of Palestine per DALY averted (<$10,992 per DALY averted)(<$10,992 per DALY averted) included bans on tobacco smoking in indoor and public places [$34 per incremental DALY averted (95% CI: $17, $50)], treatment of asthma using low dose inhaled beclometasone and short-acting beta-agonists [$140 per DALY averted (95% CI: $77, $207)], treatment of breast cancer stages I and II [$730 per DALY averted (95% CI: $372, $1,100)], implementing a mass media campaign for healthier nutrition [$737 per DALY averted (95% CI: $403, $1,100)], treatment of colorectal cancer stages I and II [$7,657 per DALY averted (95% CI: $3,721, $11,639)], and (screening with mammography [$17,054 per DALY averted (95% CI: $8,693, $25,359)]). Despite high levels of NCD risk factors among Palestinians in Gaza, we estimated that several interventions would be expected to reduce the loss of DALYs within common cost-effectiveness thresholds.

Cost-Effectiveness of Recombinant FactorIX Fc Prophylaxis and Recombinant FactorIX On-Demand Treatment in Patients with HaemophiliaB Without Inhibitors.

Recombinant factor IX (rFIX) and recombinant FIX Fc fusion protein (rFIXFc) are standard half-life and extended half-life FIX replacement therapies, respectively, and represent established treatment options indicated for adults and children with haemophiliaB. These FIX replacement therapies can be administered as prophylaxis (to prevent bleeding) or 'on-demand' (to stop bleeding). This analysis aimed to estimate the cost-effectiveness of once-weekly prophylaxis with rFIXFc versus on-demand treatment with rFIX in patients with haemophiliaB without inhibitors in the Italian healthcare setting. A Markov model was developed to assess a hypothetical cohort of adolescent or adult male patients (≥ 12years) with haemophiliaB (FIX level of ≤ 2IU/dL) without inhibitors. Model inputs were derived from the pivotal phase3 clinical studies for rFIXFc and rFIX, published literature and assumptions when published data were unavailable. The model employed a lifelong time horizon with 6-monthly transitions between health states, and it estimated total costs, total quality-adjusted life years (QALYs), number of bleeds, number of surgeries and incremental cost-effectiveness ratio. rFIXFc prophylaxis was associated with lower total costs per patient (€5,308,625 versus €6,564,510) and greater total QALYs per patient (15.936 versus 11.943) compared with rFIX on-demand; rFIXFc prophylaxis was therefore the dominant treatment strategy. The model also demonstrated that rFIXFc prophylaxis was associated with fewer incremental bleeds (- 682.29) and surgeries (- 0.39) compared with rFIX on-demand. rFIXFc prophylaxis provides improved health outcomes and lower costs, and represents a cost-effective treatment option compared with rFIX on-demand for adolescent and adult male patients with haemophiliaB. This comparative assessment of cost-effectiveness should help to inform both clinicians and healthcare policy makers when making treatment decisions for patients with haemophiliaB.

Energy Performance and Cost-Effectiveness Assessment towards the Nearly Zero-Energy School Buildings in Mild Climates

Energy Performance and Cost-Effectiveness Assessment towards the Nearly Zero-Energy School Buildings in Mild Climates

Health economic evaluation of blended collaborative care for older multimorbid heart failure patients: study protocol

BackgroundIntegrated care, in particular the ‘Blended Collaborative Care (BCC)’ strategy, may have the potential to improve health-related quality of life (HRQoL) in multimorbid patients with heart failure (HF) and psychosocial burden at no or low additional cost. The ESCAPE trial is a randomised controlled trial for the evaluation of a BCC approach in five European countries. For the economic evaluation of alongside this trial, the four main objectives were: (i) to document the costs of delivering the intervention, (ii) to assess the running costs across study sites, (iii) to evaluate short-term cost-effectiveness and cost-utility compared to providers’ usual care, and (iv) to examine the budgetary implications.MethodsThe trial-based economic analyses will include cross-country cost-effectiveness and cost-utility assessments from a payer perspective. The cost-utility analysis will calculate quality-adjusted life years (QALYs) using the EQ-5D-5L and national value sets. Cost-effectiveness will include the cost per hospital admission avoided and the cost per depression-free days (DFD). Resource use will be measured from different sources, including electronic medical health records, standardised questionnaires, patient receipts and a care manager survey. Uncertainty will be addressed using bootstrapping.DiscussionThe various methods and approaches used for data acquisition should provide insights into the potential benefits and cost-effectiveness of a BCC intervention. Providing the economic evaluation of ESCAPE will contribute to a country-based structural and organisational planning of BCC (e.g., the number of patients that may benefit, how many care managers are needed). Improved care is expected to enhance health-related quality of life at little or no extra cost.Trial registrationThe study follows CHEERS2022 and is registered at the German Clinical Trials Register (DRKS00025120).

Evaluation of the Cost-Effectiveness of Evidence-Based Interventions to Increase Female Breast and Cervical Cancer Screens: A Systematic Review.

To systematically review published cost-effectiveness analyses of Evidence-Based Interventions (EBIs) recommended by the United States Community Preventive Services Task Force (CPSTF) to increase breast and cervical cancer screening. We searched PubMed and Embase for prospective cost-effectiveness evaluations of EBIs for breast and cervical cancer screening since 1999. We reviewed studies according to the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) and compared the incremental cost-effectiveness ratio (ICERs), defined as cost per additional woman screened, adjusted to 2021 USD, within and across EBIs by cancer type. We identified eleven studies meeting our review criteria: nine were breast cancer-focused, one breast and cervical cancer combined, and one cervical only, which together reported twenty-four cost-effectiveness assessments of outreach programs spanning eight EBIs. One-on-one education programs were the most common EBI evaluated. The average ICER across breast cancer studies was USD 545 (standard deviation [SD] = USD 729.3), while that for cervical cancer studies was USD 197 (SD = 186.6. Provider reminder/recall systems for women already linked to formal care were the most cost-effective, with an average ICERs of USD 41.3 and USD 10.6 for breast and cervical cancer, respectively. Variability in ICERs across and within EBIs reflect the population studied, the specific EBI, and study settings, and was relatively high. ICER estimate uncertainty and the potential for program replicability in other settings and with other populations were not addressed. Given these limitations, using existing cost-effectiveness estimates to inform program funding allocations is not warranted at this time. Additional research is needed on outreach programs for cervical cancer and those which serve minority populations for either of the female cancer screens.

Comparing code-free and bespoke deep learning approaches in ophthalmology.

Code-free deep learning (CFDL) allows clinicians without coding expertise to build high-quality artificial intelligence (AI) models without writing code. In this review, we comprehensively review the advantages that CFDL offers over bespoke expert-designed deep learning (DL). As exemplars, we use the following tasks: (1) diabetic retinopathy screening, (2) retinal multi-disease classification, (3) surgical video classification, (4) oculomics and (5) resource management. We performed a search for studies reporting CFDL applications in ophthalmology in MEDLINE (through PubMed) from inception to June 25, 2023, using the keywords 'autoML' AND 'ophthalmology'. After identifying 5 CFDL studies looking at our target tasks, we performed a subsequent search to find corresponding bespoke DL studies focused on the same tasks. Only English-written articles with full text available were included. Reviews, editorials, protocols and case reports or case series were excluded. We identified ten relevant studies for this review. Overall, studies were optimistic towards CFDL's advantages over bespoke DL in the five ophthalmological tasks. However, much of such discussions were identified to be mono-dimensional and had wide applicability gaps. High-quality assessment of better CFDL applicability over bespoke DL warrants a context-specific, weighted assessment of clinician intent, patient acceptance and cost-effectiveness. We conclude that CFDL and bespoke DL are unique in their own assets and are irreplaceable with each other. Their benefits are differentially valued on a case-to-case basis. Future studies are warranted to perform a multidimensional analysis of both techniques and to improve limitations of suboptimal dataset quality, poor applicability implications and non-regulated study designs. For clinicians without DL expertise and easy access to AI experts, CFDL allows the prototyping of novel clinical AI systems. CFDL models concert with bespoke models, depending on the task at hand. A multidimensional, weighted evaluation of the factors involved in the implementation of those models for a designated task is warranted.

Economic evaluations of assisted reproductive technologies in high-income countries: a systematic review.

Which assited reproductive technology (ART) interventions in high-income countries are cost-effective and which are not? Among all ART interventions assessed in economic evaluations, most high-cost interventions, including preimplantation genetic testing for aneuploidy (PGT-A) for a general population and ICSI for unexplained infertility, are unlikely to be cost-effective owing to minimal or no increase in effectiveness. Approaches to reduce costs in order to increase access have been identified as a research priority for future infertility research. There has been an increasing number of ART interventions implemented in routine clinical practice globally, before robust assessments of evidence on economic evaluations. The extent of clinical effectiveness of some studied comparisons has been evaluated in high-quality research, allowing more informative decision making around cost-effectiveness. We performed a systematic review and searched seven databases (MEDLINE, PUBMED, EMBASE, COCHRANE, ECONLIT, SCOPUS, and CINAHL) for studies examining ART interventions for infertility together with an economic evaluation component (cost-effectiveness, cost-benefit, cost-utility, or cost-minimization assessment), in high-income countries, published since January 2011. The last search was 22 June 2022. Two independent reviewers assessed publications and included those fulfilling the eligibility criteria. Studies were examined to assess the cost-effectiveness of the studied intervention, as well as the reporting quality of the study. The chosen outcome measure and payer perspective were also noted. Completeness of reporting was assessed against the Consolidated Health Economic Evaluation Reporting Standard. Results are presented and summarized based on the intervention studied. The review included 40 studies which were conducted in 11 high-income countries. Most studies (n = 34) included a cost-effectiveness analysis. ART interventions included medication or strategies for controlled ovarian stimulation (n = 15), IVF (n = 9), PGT-A (n = 7), single embryo transfer (n = 5), ICSI (n = 3), and freeze-all embryo transfer (n = 1). Live birth was the mostly commonly reported primary outcome (n = 27), and quality-adjusted life years was reported in three studies. The health funder perspective was used in 85% (n = 34) of studies. None of the included studies measured patient preference for treatment. It remains uncertain whether PGT-A improves pregnancy rates compared to IVF cycles managed without PGT-A, and therefore cost-effectiveness could not be demonstrated for this intervention. Similarly, ICSI in non-male factor infertility appears not to be clinically effective compared to standard fertilization in an IVF cycle and is therefore not cost-effective. Interventions such as use of biosimilars or HMG for ovarian stimulation are cheaper but compromise clinical effectiveness. Lack of both preference-based and standardized outcomes limits the comparability of results across studies. The selection of efficacy evidence offered for some interventions for economic evaluations is not always based on high-quality randomized trials and systematic reviews. In addition, there is insufficient knowledge of the willingness to pay thresholds of individuals and state funders for treatment of infertility. There is variable quality of reporting scores, which might increase uncertainty around the cost-effectiveness results. Investment in strategies to help infertile people who utilize ART is justifiable at both personal and population levels. This systematic review may assist ART funders decide how to best invest to maximize the likelihood of delivery of a healthy child. There was no funding for this study. E.C. and R.W. receive salary support from the National Health and Medical Research Council (NHMRC) through their fellowship scheme (EC GNT1159536, RW 2021/GNT2009767). M.D.-T. reports consulting fees from King Fahad Medical School. All other authors have no competing interests to declare. Prospero CRD42021261537.

AI and machine learning in medical imaging: key points from development to translation.

Innovation in medical imaging artificial intelligence (AI)/machine learning (ML) demands extensive data collection, algorithmic advancements, and rigorous performance assessments encompassing aspects such as generalizability, uncertainty, bias, fairness, trustworthiness, and interpretability. Achieving widespread integration of AI/ML algorithms into diverse clinical tasks will demand a steadfast commitment to overcoming issues in model design, development, and performance assessment. The complexities of AI/ML clinical translation present substantial challenges, requiring engagement with relevant stakeholders, assessment of cost-effectiveness for user and patient benefit, timely dissemination of information relevant to robust functioning throughout the AI/ML lifecycle, consideration of regulatory compliance, and feedback loops for real-world performance evidence. This commentary addresses several hurdles for the development and adoption of AI/ML technologies in medical imaging. Comprehensive attention to these underlying and often subtle factors is critical not only for tackling the challenges but also for exploring novel opportunities for the advancement of AI in radiology.

Navigating the Therapeutic Landscape: A Comprehensive Review of Platelet-Rich Plasma and Bone Marrow Aspirate Concentrate in Knee Osteoarthritis.

This comprehensive review provides an in-depth analysis of platelet-rich plasma (PRP) and bone marrow aspirate concentrate (BMAC) as potential treatments for knee osteoarthritis. It explores their mechanisms of action, clinical efficacy, safety considerations, and the importance of personalised treatment approaches. The review highlights promising findings regarding the ability of PRP and BMAC to alleviate symptoms, improve joint function, and potentially slow disease progression. It emphasises the need for further research into long-term outcomes, direct comparative studies, protocol standardisation, biomarker identification, and cost-effectiveness assessments to enhance clinical practice. While the review does not directly compare PRP and BMAC, it provides valuable insights into their respective roles in knee osteoarthritis management. The review aims to contribute to evidence-based advancements in regenerative therapies for knee osteoarthritis by addressing critical research priorities and refining treatment strategies.

Acceptability of 'as needed' biologic therapy in psoriasis: insights from a multistakeholder mixed-methods study.

Biologic therapies have led to increasing numbers of patients with psoriasis who have clear or nearly clear skin. It is current practice to continue biologic therapy indefinitely in these patients, which contributes to a substantial long-term drug and healthcare burden. 'As needed' biologic therapy in psoriasis may address this; however, our understanding of patient and clinician perceptions of this strategy is limited. The aim of this mixed-methods study was to gain insight into the perspectives of both patients and clinicians regarding the acceptability of an 'as needed' approach to biologic therapy in psoriasis, including potential barriers and enablers to implementation in routine care. We first conducted UK-wide online scoping surveys of patients with psoriasis and dermatology clinicians to explore their views on 'as needed' biologic therapy. Using topic guides informed by these survey findings, we then carried out qualitative focus groups with patients and clinicians. Themes were identified using reflexive thematic analysis. Of 67 patients and 27 clinicians completing the scoping surveys, 67% (43 of 64 patients) and 78% (21 of 27 clinicians) supported the use of 'as needed' biologic therapy, respectively. Respondents highlighted advantages such as a reduction in healthcare burden and greater ownership of care. Challenges included logistics of 'as needed' drug provision and potential risks of disease flare and drug immunogenicity. Focus groups comprised 15 patients with psoriasis [9 female patients (60%), average disease duration 32 years (range 9-64)] and 9 dermatology clinicians [8 female clinicians (89%), average dermatology experience 20 years (range 8-33)]. Both patients and clinicians felt that an 'as needed' treatment approach will deliver a reduction in treatment burden and present an opportunity for patient-led ownership of care. Both groups highlighted the importance of ensuring ongoing access to medication and discussing the potential impact of psoriasis recurrence. Patient preferences were influenced by their lived experiences, particularly previous difficulties with medication delivery logistics and establishing disease control. Clinician perspectives were informed by personal experience of their patients adapting their own dosing schedules. Clinicians highlighted the importance of targeted patient selection for an 'as needed' approach, ongoing disease monitoring, and prompt reaccess to medications upon psoriasis recurrence. These data indicate that 'as needed' biologic therapy in psoriasis is acceptable for both patients and clinicians. Formal assessment of clinical effectiveness and cost-effectiveness is warranted to enable the real-world potential of this approach to be realized.

Integrating China in the International Consortium for Personalised Medicine: a position paper on innovation and digitalization in Personalized Medicine

BackgroundThe advent of Personalized Medicine (PM) holds significant promise in revolutionizing healthcare by tailoring treatments to individual patients based on their data. However, its successful implementation requires the seamless integration of innovative technologies and presents formidable challenges in terms of sustainability. To tackle these challenges head-on, the International Consortium for Personalized Medicine (ICPerMed) was established, and the IC2PerMed project, as part of this consortium, seeks to foster collaboration between the European Union (EU) and China in the field of Personalized Medicine. Based on the results collected by the project, the objective of this study is to discern the key priorities for the implementation of Personalised Medicine concerning Information and Communication Technologies (ICT) and Big Data and digital solutions, with a particular emphasis on data management and protection.MethodsA Delphi survey was conducted to gather expert’s consensus on the main priorities for actions on Information and Communication Technologies (ICT) and Big Data and digital solutions in the field of Personalized Medicine.ResultsThe survey identified seven priorities in the area of Big Data and digital solutions, including data interoperability, standards, security measures, and international partnerships. Additionally, twelve priorities were identified for the innovation-to-market process, emphasizing cost-effectiveness, need assessment, and value definition in resource allocation.ConclusionsThe effective implementation of new technologies in Personalized Medicine research and practice is essential for the advancement of healthcare systems in both the European and Chinese contexts. The identified priorities play a pivotal role in promoting the sustainability of health systems and driving innovation in the implementation of Personalized Medicine. Addressing challenges related to data interoperability, standards, security, international collaboration, cost-effectiveness, and value assessment is of utmost importance in order to propel the progress of Personalized Medicine in healthcare systems.