scholarly journals PBI16 Betibeglogene Autotemcel GENE Therapy (BETI-CEL) Is Cost-Effective Versus Standard of Care in Patients with Transfusion-Dependent B-Thalassemia (TDT) in France

2020 ◽  
Vol 23 ◽  
pp. S413
Author(s):  
L. Undreiner ◽  
S. Roze ◽  
M. Caillon
2021 ◽  
Vol 11 (10) ◽  
pp. 4640
Author(s):  
Jordan J. Becker ◽  
Tara L. McIsaac ◽  
Shawn L. Copeland ◽  
Rajal G. Cohen

Background: Alexander technique private lessons have been shown to reduce chronic neck pain and are thought to work by different mechanisms than exercise. Group classes may also be effective and would be cost-effective. Design: A two-group pre-test/post-test design. Participants were assigned to either a general Alexander technique class or an exercise class designed to target neck pain. Both groups met over 5 weeks for two 60 min sessions/week. Participants: A total of 16 participants with chronic neck pain (aged 50+/−16 years) completed this study. Interventions: The Alexander class used awareness-building methods to teach participants to reduce habitual tension during everyday activities. The exercise class was based on physical therapy standard of care to strengthen neck and back muscles thought to be important for posture. Measures: We assessed neck pain/disability, pain self-efficacy, activation of the sternocleidomastoid muscles during the cranio-cervical flexion test, and posture while participants played a video game. Results: Both groups reported decreased neck pain/disability after the interventions. Sternocleidomastoid activation decreased only in the Alexander group. Conclusion: In this small preliminary study, Alexander classes were at least as effective as exercise classes in reducing neck pain and seemed to work via a different mechanism. Larger, multi-site studies are justified.


2015 ◽  
Vol 40 (4) ◽  
pp. 298-305 ◽  
Author(s):  
Yoshitsugu Obi ◽  
Rieko Eriguchi ◽  
Shuo-Ming Ou ◽  
Connie M. Rhee ◽  
Kamyar Kalantar-Zadeh

Background: The 2006 Kidney Disease Outcomes Quality Initiative guidelines suggest twice-weekly or incremental hemodialysis for patients with substantial residual kidney function (RKF). However, in most affluent nations de novo and abrupt transition to thrice-weekly hemodialysis is routinely prescribed for all dialysis-naïve patients regardless of their RKF. We review historical developments in hemodialysis therapy initiation and revisit twice-weekly hemodialysis as an individualized, incremental treatment especially upon first transitioning to hemodialysis therapy. Summary: In the 1960's, hemodialysis treatment was first offered as a life-sustaining treatment in the form of long sessions (≥10 hours) administered every 5 to 7 days. Twice- and then thrice-weekly treatment regimens were subsequently developed to prevent uremic symptoms on a long-term basis. The thrice-weekly regimen has since become the ‘standard of care' despite a lack of comparative studies. Some clinical studies have shown benefits of high hemodialysis dose by more frequent or longer treatment times mainly among patients with limited or no RKF. Conversely, in selected patients with higher levels of RKF and particularly higher urine volume, incremental or twice-weekly hemodialysis may preserve RKF and vascular access longer without compromising clinical outcomes. Proposed criteria for twice-weekly hemodialysis include urine output >500 ml/day, limited interdialytic weight gain, smaller body size relative to RKF, and favorable nutritional status, quality of life, and comorbidity profile. Key Messages: Incremental hemodialysis including twice-weekly regimens may be safe and cost-effective treatment regimens that provide better quality of life for incident dialysis patients who have substantial RKF. These proposed criteria may guide incremental hemodialysis frequency and warrant future randomized controlled trials.


2009 ◽  
Vol 91 (4) ◽  
pp. 305-309 ◽  
Author(s):  
Nadine Coull ◽  
Giles Rottenberg ◽  
Sheila Rankin ◽  
Maria Pardos-Martinez ◽  
Bola Coker ◽  
...  

INTRODUCTION Conventional publicly funded out-patient services in many specialties are characterised by delays, fragmented diagnostic processes, and overloaded clinics. This is bad for patients as it is clinically dangerous; bad for managers who spend hours managing the failure; bad for doctors who respond by overloading clinics; and bad for purchasers who have to fund the multiple out-patient visits needed. Sound clinical and financial reasons exist for introducing more efficient diagnostic processes. PATIENTS AND METHODS A total of 330 consecutive patients referred to the urology department of Guy' and St Thomas' NHS Foundation Trust were invited to attend one of nine one-stop clinics staffed by consultant urologists with specialist registrars, nurses, and clerical staff. Pre-clinic blood and urine tests were ordered based on the referral letter. Clinics had facilities to perform cystoscopy, ultrasound, and urinary flow studies. Correspondence was generated in real time, and a copy given to the patient. RESULTS Overall, 257 patients attended the clinics. Twenty-three patients cancelled appointments and 50 patients did not attend. Pre-clinic tests were requested in 133 patients and were completed by 86% of the patients who attended. Of patients, 42% were diagnosed and discharged; 28% were listed for surgery, extracorporeal shock wave lithotripsy (ESWL), or referred to another specialty. About 30% of patients needed further out-patient review; in approximately two-thirds to complete a diagnosis and one-third to review the results of therapy initiated. An estimated 350 appointments and 550 patient visits to hospital were saved. CONCLUSIONS A one-stop method of consultation is efficient across a range of urological presenting complaints, and dramatically reduces the need for follow–up consultations. It has potential to: (i) reduce delays to being seen in out-patients; (ii) lead to more cost-effective care; and (iii) increase safety and patient satisfaction. It should become the standard of care in urology, and is probably applicable in many other disciplines.


2021 ◽  
Vol 9 ◽  
Author(s):  
Stephen Okoboi ◽  
Barbara Castelnuovo ◽  
Jean-Pierre Van Geertruyden ◽  
Oucul Lazarus ◽  
Lung Vu ◽  
...  

Introduction: Distribution of HIV self-testing (HIVST) kits through MSM peer networks is a novel and effective strategy to increase HIV testing coverage in this high-risk population. No study has evaluated the cost or cost effectiveness of peer distribution of HIVST strategies among MSM in sub-Saharan Africa.Methods: From June to August 2018, we conducted a pilot study of secondary MSM peer HIVST kit distribution at The AIDS Support Organization at Entebbe and Masaka. We used an ingredients approach to estimate the cost of MSM peer HIVST kit distribution relative to standard-of-care (SOC) hotspot testing using programme expenditure data reported in US dollars. The provider perspective was used to estimate incremental cost-effective ratios per HIV infection averted using the difference in HIV annual transmission rates between MSM with HIV who knew their status and were not virologically suppressed and MSM with HIV who did not know their status.Results: We enrolled 297 participants of whom 150 received MSM peer HIVST kit distribution (intervention group) and 147 received TASO standard of care HIV testing (control group). Provider cost for the intervention was $2,276 compared with $1,827 for SOC during the 3-month study period. Overall, the intervention resulted in higher HIV positivity yield (4.9 vs. 1.4%) and averted more HIV infections per quarter (0.364 vs. 0.104) compared with SOC. The cost per person tested was higher for the intervention compared to SOC ($15.90 vs. $12.40). Importantly, the cost per new HIV diagnosis ($325 vs. $914) and cost per transmission averted ($6,253 vs. $ 17,567) were lower for the intervention approach relative to SOC. The incremental cost per HIV transmission averted by the self-testing program was $1,727. The incremental cost to providers per additional HIV-positive person identified by the intervention was $147.30.Conclusion: The intervention strategy was cost-effective, and identified more undiagnosed HIV infections than SOC hotspot testing at a cost-effectiveness threshold of US $2,129. Secondary distribution of HIVST kits through peers should further be evaluated with longer duration aimed at diagnosing 95% of all persons with HIV by 2030; the first UNAIDS 95-95-95 target.


2018 ◽  
Vol 1 (4) ◽  
Author(s):  
Timothy Porter

No abstract available. Editor’s note: As patients start to demand access to telemedicine,1 it is imperative for physicians to understand how to make these types of appointments available in their practice. Without telemedicine adoption as a standard of care, physicians run the risk of losing patients to on-demand telemedicine organizations. Through telemedicine, not only do patients get a more convenient and cost-effective experience, providers have the opportunity to grow their practice and increase patient satisfaction. In this article, Dr. Timothy Porter, a community pediatrician in Chicago, shares his perspective.


2018 ◽  
Author(s):  
Ching Lam ◽  
Edward Meinert ◽  
Abrar Alturkistani ◽  
Alison R. Carter ◽  
Jeffrey Karp ◽  
...  

BACKGROUND Decisional tools have demonstrated their importance in informing manufacturing and commercial decisions in the monoclonal antibody domain. Recent approved therapies in regenerative medicine have shown great clinical benefits to patients. OBJECTIVE The objective of this review was to investigate what decisional tools are available and what issues and gaps have been raised for their use in regenerative medicine. METHODS We systematically searched MEDLINE to identify articles on decision support tools relevant to tissue engineering, and cell and gene therapy, with the aim of identifying gaps for future decisional tool development. We included published studies in English including a description of decisional tools in regenerative medicines. We extracted data using a predesigned Excel table and assessed the data both quantitatively and qualitatively. RESULTS We identified 9 articles addressing key decisions in manufacturing and product development challenges in cell therapies. The decision objectives, parameters, assumptions, and solution methods were analyzed in detail. We found that all decisional tools focused on cell therapies, and 6 of the 9 reviews focused on allogeneic cell therapy products. We identified no available tools on tissue-engineering and gene therapy products. These studies addressed key decisions in manufacturing and product development challenges in cell therapies, such as choice of technology, through modeling. CONCLUSIONS Our review identified a limited number of decisional tools. While the monoclonal antibodies and biologics decisional tool domain has been well developed and has shown great importance in driving more cost-effective manufacturing processes and better investment decisions, there is a lot to be learned in the regenerative medicine domain. There is ample space for expansion, especially with regard to autologous cell therapies, tissue engineering, and gene therapies. To consider the problem more comprehensively, the full needle-to-needle process should be modeled and evaluated.


2020 ◽  
Author(s):  
Jacqui Miot ◽  
Trudy Leong ◽  
Simbarashe Takuva ◽  
Andrew Parrish ◽  
Halima Dawood

Abstract Background Cryptococcal meningitis in HIV-infected patients in sub-Saharan Africa accounts for three-quarters of the global cases and 135 000 deaths per annum. Current treatment includes the use of fluconazole and amphotericin B. Recent evidence has shown that the synergistic use of flucytosine improves efficacy and reduces toxicity, however affordability and availability has hampered access to flucytosine in many countries. This study investigated the evidence and cost implications of introducing flucytosine as induction therapy for cryptococcal meningitis in HIV-infected adults in South Africa. Methods A decision analytic cost-effectiveness and budget impact model was developed based on survival estimates from the ACTA trial and local costs for flucytosine as induction therapy in HIV-infected adults with cryptococcal meningitis in a public sector setting in South Africa. The model considered four treatment arms: (a) standard of care; 2-week course of amphotericin B/fluconazole (2wk AmBd/Flu), (b) 2-week course of amphotericin B/flucytosine (2wk AmBd/5FC), (c) short course; 1-week course amphotericin B/flucytosine (1wk AmBd/5FC) and (d) oral course; 2-week oral fluconazole/flucytosine (oral). A sensitivity analysis was conducted on key variables. Results The highest total treatment costs were in the 2-week AmBd/5FC arm followed by the 2-week oral regimen, then the 1-week AmBd/5FC with the lowest cost in the standard of care arm. Compared to standard of care the 1-week flucytosine course is most cost-effective at USD31/QALY, followed by the oral 2-week course at USD155/QALY and the 2-week flucytosine course at USD568/QALY. The budget impact analysis shows that the 1-week course has the lowest incremental cost, followed by the oral course and then the 2-week flucytosine course compared to what is currently spent on standard of care. Sensitivity analyses suggest that the model is most sensitive to the price of flucytosine and hospital costs, particularly length of stay. Conclusions The addition of flucytosine as induction therapy for the treatment of cryptococcal meningitis in patients infected with HIV is cost-effective regardless of whether it is used as a 1-week, 2-week or oral regimen. Savings could be achieved with early discharge of patients as well as a reduction in the price of flucytosine.


2020 ◽  
Vol 3 (Supplement_1) ◽  
pp. 151-152
Author(s):  
S Alrajhi ◽  
A N Barkun ◽  
V Adam ◽  
K Callichurn ◽  
M Martel ◽  
...  

Abstract Background Single-operator cholangioscopy assisted electrohydraulic lithotripsy is the standard of care for difficult common bile duct (CBD) stones with failed clearance using standard ERCP. This technology is expensive and optimal timing of its use in terms of cost-effectiveness in the management algorithm of patients with difficult CBD stones remains unclear Aims To determine the cost-effective timing of SOC-EHL utilization in the management of difficult CBD stones Methods A decision model was developed assessing 4 strategies and progressively delayed introduction of SOC-EHL in relation to ERCP over 6-months. Difficult stones were defined as having failed CBD clearance via standard ERCP. Probability estimates for each health state were obtained from a literature systematic review. For each strategy, outpatients undergoing ERCP underwent different timings of SOC-EHL introduction from the first to the fourth ERCP and were followed for subsequent need for re-intervention, adverse events, need for surgery, and/or successful endoscopic CBD clearance. The unit of effectiveness was complete CBD clearance without need for surgery. Deterministic sensitivity analyses were performed varying all 50 model variables across ranges spanning 30% of their respective values. Costs are in 2018US$ based on US data. Results Performing SOC-EHL immediately during the first ERCP is the least expensive approach when compared to delaying SOC-EHL. This strategy costs $15,528 on average per patient with CBD clearance avoiding surgery and can save between $260 to $720 compared to the 3 other strategies, which introduce SOC-EHL during the second to the fourth ERCP. Effectiveness is clinically comparable between the four strategies ranging from 97–99%. Deterministic sensitivity analysis shows changes in the results when the ERCP complication rate (baseline probability of 6%) decreases to 4.5%, when the SOC-EHL (baseline costs of $2,450) costs more than $2,670, or when the ERCP facility fees (baseline costs of $4,292) are less than $3,425. In all 3 scenarios, delaying the first SOC-EHL use to the fourth procedural attempt becomes the dominant strategy. Variations of the other 47 variables did not alter results. Conclusions Although SOC-EHL is expensive, this analysis demonstrates that among patients who have failed a prior attempt at stone extraction, utilization of SOC-EHL at the next (first subsequent) ERCP is less costly when compared to its delayed introduction. However, postponing the use of SOC-EHL to the fourth ERCP could be identified as the most cost-effective strategy when facility fees or ERCP complications rates are below certain thresholds, or when the costs of SOC-EHL extend beyond a defined threshold. Funding Agencies None


Blood ◽  
1996 ◽  
Vol 87 (12) ◽  
pp. 5095-5103 ◽  
Author(s):  
G Hortelano ◽  
A Al-Hendy ◽  
FA Ofosu ◽  
PL Chang

A potentially cost-effective strategy for gene therapy of hemophilia B is to create universal factor IX-secreting cell lines suitable for implantation into different patients. To avoid graft rejection, the implanted cells are enclosed in alginate-polylysine-alginate microcapsules that are permeable to factor IX diffusion, but impermeable to the hosts' immune mediators. This nonautologous approach was assessed by implanting encapsulated mouse myoblasts secreting human factor IX into allogeneic mice. Human factor IX was detected in the mouse plasma for up to 14 days maximally at approximately 4 ng/mL. Antibodies to human factor IX were detected after 3 weeks at escalating levels, which were sustained throughout the entire experiment (213 days). The antibodies accelerated the clearance of human factor IX from the circulation of the implanted mice and inhibited the detection of human factor IX in the mice plasma in vitro. The encapsulated myoblasts retrieved periodically from the implanted mice up to 213 days postimplantation were viable and continued to secrete human factor IX ex vivo at undiminished rates, hence suggesting continued factor IX gene expression in vivo. Thus, this allogeneic gene therapy strategy represents a potentially feasible alternative to autologous approaches for the treatment of hemophilia B.


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