Short-term Therapies Research Articles

The impact of capping health system cost savings on the projected cost-effectiveness of etranacogene dezaparvovec compared with factor IX prophylaxis for the treatment of hemophilia B.

This viewpoint discusses cost-effectiveness estimates for EtranaDez, a gene therapy for hemophilia B, using the Institute for Clinical and Economic Review's (ICER) framework for single and short-term therapies (SSTs). EtranaDez offers long-term benefits from a single administration, in contrast to the high costs and frequent dosing required by current factor IX prophylaxis. However, the projected gains in health from EtranaDez are small relative to the cost implications of the therapy, and consequently, how the cost offsets associated with EtranaDez are counted has a substantial impact on assessing its cost-effectiveness. Strategies for assessing cost offsets used in the ICER SST framework include a 50/50 cost-sharing model between the health care system and the manufacturer and a cap of $150,000 annually on health care cost offsets. Results from the standard full cost-offset analysis as reported by ICER depicted EtranaDez as a dominant therapy with substantial cost savings compared with factor IX prophylaxis. However, while considering the ICER SST framework, particularly the $150,000 annual cap scenario, the cost-effectiveness was significantly reduced. The incremental cost-effectiveness ratio varied notably between these scenarios, challenging the conventional perception of value of gene therapy in health care. These cost-sharing scenarios highlight the potential of the ICER SST framework to help curtail inefficient health care spending. In cases in which the cost of existing treatment is exceedingly high, the application of such frameworks would improve efficiency in resource allocation, fostering a balance between incentives for innovation and economic sustainability in managed care systems.

The importance of antibiotic treatment duration in antimicrobial resistance.

Antimicrobial resistance (AMR) poses a significant threat to global health, leading to increased deaths from drug-resistant infections and escalates healthcare costs. Often termed a "silent pandemic," AMR occurs when pathogens become resistant to antimicrobial drugs, enabling their proliferation and spread. Inappropriate antibiotic usage is a major contributor to this phenomenon, which also extends to fungal infections. In particular, the duration of antibiotic therapy is a crucial aspect, with evidence suggesting that prolonged use can heighten bacterial resistance and harm the human microbiota. In fact, studies comparing short-term versus long-term antibiotic therapies show no significant difference in traditional treatments. In addition, therapeutic drug monitoring allows personalized antibiotic regimens, optimizing dosage and duration to minimize resistance and adverse effects. As a result, clinicians should regularly reassess treatment effectiveness, utilizing techniques like antibiotic timeout and de-escalation therapy to avoid prolonged antibiotic use and mitigate resistance. All these strategies are crucial to prevent and counter the phenomenon of antimicrobial resistance.

Dose-response relationship in cognitive behavioral therapy for depression: A nonlinear metaregression analysis.

Evidence on the optimal "dose" of cognitive behavioral therapy (CBT) for treating major depressive disorder is sparse. This analysis aimed to evaluate the dose-response curve in CBT using a nonlinear approach, whereby "dose" was defined as number of treatment sessions. The dose-response curve of CBT was compared to other psychotherapies and pharmacological treatments for depression. A systematic review and metaregression analysis of randomized controlled trials (RCTs) examining the efficacy of CBT in adults with acute depression was conducted. Treatment arms examining other psychosocial or pharmacological interventions were also analyzed. Cubic spline metaregression techniques were used to model nonlinear dose-response curves. Seventy-two studies and 7,377 participants were included. Modeling the dose-response curve between change of depression symptom severity and the number of CBT sessions resulted in a nonlinear curve characterized by a strong improvement in symptom severity from baseline within the first eight sessions. Symptom reduction continues in the further course of the treatment, but at a slower pace. A similar pattern of symptom development was found for other therapies as well, although the prominence of early improvement and overall effect sizes vary across treatment arms. Results imply a general tendency for the strongest alleviation of depressive symptom severity in early stages of CBT treatment, thus, if aiming at symptom alleviation, speak for short CBT interventions. However, these findings have to be discussed in the light of the limited data regarding the sustainability of treatment effects in short-term therapies and effects beyond symptomatic changes. (PsycInfo Database Record (c) 2024 APA, all rights reserved).

Knowledge and practice of emergency physicians regarding drug therapy for acute myocardial infarction

Objective: This study aimed to assess the knowledge and practice of emergency physicians regarding drug therapy for acute myocardial infarction (AMI). Methods: A cross-sectional survey of emergency physicians aged between 35 and 60 years was carried out. Emergency physicians were surveyed regarding drug therapy interventions in acute myocardial infarction. Results: Around 267 (89.0%) of the 300 emergency physicians who responded were under 40 years. Among emergency physicians, 176 were men (58.0%). Emergency physicians responded to the study they treated 1 to 4 patients with AMI (n = 183, 61.0%) and more than 5 (n = 117, 39.0%). According to the current results, the demographic data of responses to emergency physicians (age and gender) had a significant effect on the knowledge and practice of emergency physicians regarding drug therapy for acute myocardial infarction. Conclusion: This study indicated that emergency physicians were significantly more aware of and skilled in using most short-term pharmacological therapies while treating AMI. The treatment of patients with AMI, based on evidence-based medicine, is one of the most basic examples of the purpose and responsibilities of the emergency physician.

Comparison of the Effectiveness of Emotion-Focused Therapy and Intensive Short-Term Psychodynamic Therapy on Sexual Dysfunction and Quality of Sexual Life in Women

Objective: The present study aimed to compare the effectiveness of emotion-focused therapy and short-term psychodynamic therapy on sexual dysfunction and the quality of sexual life in women attending counseling and psychotherapy centers in Isfahan. Methods: The research design was a quasi-experimental pre-test, post-test, and follow-up. The population included all women in Isfahan province suffering from sexual dysfunction in 2023. Sixty individuals were selected through purposive sampling and randomly assigned into three groups: twenty in the first experimental group, twenty in the second experimental group, and twenty in the control group. The first experimental group underwent emotion-focused psychological intervention based on the therapeutic protocol by Gilbert (2014) in fifteen 60-minute sessions, and the second experimental group received short-term psychodynamic psychological intervention based on the therapeutic protocol (Davanloo, 2013) in twelve 60-minute sessions held weekly, followed by a one-month follow-up. The control group was placed on a two-month waiting list for the most effective intervention. Data collection for analysis was conducted using the Rosen et al. (2000) Women's Sexual Dysfunction Questionnaire and the Simonds et al. (2005) Women's Sexual Life Quality Questionnaire. Data analysis was performed using mixed ANOVA with SPSS version 25. Findings: The findings indicated that both treatments were effective in reducing sexual dysfunction in women (F = 73.08, p < .001) and improving the quality of sexual life (F = 18.02, p < .001). However, there was a significant difference between the effectiveness of the two therapeutic methods in reducing sexual dysfunction and increasing the quality of sexual life in women (p < .001). Conclusion: It can be concluded that both emotion-focused and short-term psychodynamic therapies are effective in reducing sexual dysfunction and improving the quality of sexual life in women. Both treatments can be used to reduce psychological issues in women attending counseling centers.

The combination of natural compounds Crila and epigallocatechin gallate showed enhanced antiproliferative effects on human uterine fibroid cells compared with single treatments

To investigate the combined effects of Crila and green tea extract, epigallocatechin gallate (EGCG), compared with single treatments, on human uterine fibroid cells. Human uterine leiomyoma (HuLM) cells were treated with different concentrations of Crila, alone or in combination with EGCG, and several experiments were employed. A laboratory study. N/A. Crila, EGCG. Cell proliferation assay, drug synergy using combination index, protein and gene expression analysis of proliferation marker proliferating cell nuclear antigen, and apoptosis marker BAX using western blotting and quantitative polymerase chain reaction, respectively. Results showed that tested Crila concentrations, when combined with 25 and 50 μM EGCG, exerted synergistic growth inhibitory effects on HuLM viability. This inhibitory effect on HuLM cell viability was because of decreased cell proliferation, as shown by a decrease in the proliferation marker proliferating cell nuclear antigen at messenger RNA and protein levels, rather than inducing apoptosis. Our study concludes that the utility of natural compounds may provide a safe and cost-effective alternative to currently used short-term hormonal therapies against uterine fibroids.

A Look into Pharmacy Practices among the Purépecha Indigenous Community.

This report describes the adoption and integration of Western medicine in Cherán K'eri after the social changes in the 1940s which led to the transition from healer to pharmacist. There are various health models that rely heavily on community pharmacies. The place used as the basis for this report was a clinic managed by a Purépecha-speaking physician and pharmacist that served primarily monolingual indigenous Purépecha patients, whose population was around 9550 according to the 2010 census. Twelve major differences were observed between community pharmacies in the United States and the community pharmacies of Cherán. It was also observed that the modern approach to the health of the indigenous population used a combination of Western medicine together with traditional methods and only resorted to short-term therapies with Western medicines lasting five days or less. A formulary from the clinic's community pharmacy compiled in 2022 listed the 38 most common medications. Medications used included anti-infectives (n = 3), central nervous system (n = 2), endocrine/hormonal (n = 3), gastrointestinal (n = 3), musculoskeletal (n = 17), respiratory or allergy (n = 6), and genitourinary (n = 2).

Assessing the impact of single or short-term administration on a therapy’s cost-effectiveness: a hypothetical disease-agnostic model

AimsAssessing the value of single or short-term therapies (SSTs) within traditional cost-effectiveness analyses (CEAs) has been a topic of discussion as the number of SSTs increases, particularly regarding the effect of discounting on valuation. To quantify the impact of discounting in economic evaluations, a CEA of a hypothetical SST and equivalent chronic therapy was conducted using standard methods.Materials and methodsA lifetime Markov model was developed for a hypothetical chronic, progressive disease that could be treated with an SST, chronic therapy, or no novel treatment, termed standard of care (SoC). Incremental cost-effectiveness ratios (ICERs) with quality-adjusted life years (QALYs) comparing SST vs. SoC and an equivalent chronic therapy vs. SoC were assessed from a payer perspective. Both treatments had equal benefits and undiscounted lifetime costs; 3% discounting was applied to costs/benefits in the base case, and the impact of discounting was assessed.ResultsIn the base case example, both the SST and equivalent chronic therapy vs. SoC had ICERs of $86,000/QALY without discounting. With 3% discounting, the ICER for the SST increased by 116% ($186,000/QALY) while the ICER for the chronic therapy increased by 10% ($95,000/QALY) despite equal clinical benefit. In scenario analyses, the ICER of the SST was consistently higher than the equivalent chronic therapy across a range of assumptions/inputs. Varying the cost/benefit discount rates had a greater impact on the SST. Differences in the ICERs between the therapies increased with increasing life expectancy/time horizon.LimitationsThe simple model structure may not be reflective of acute or more complex diseases. Also, the scenario of perfect equivalency in efficacy and lifetime costs is hypothetical.ConclusionsThis quantitative assessment showed the extent to which SST CEAs are highly sensitive to discounting, resulting in worse value assessments for SSTs than equivalent chronic therapies.

Effectiveness of Adjunctive Therapies Following Botulinum Toxin Type A Injections in Children with Cerebral Palsy

Objectives: To investigate the effectiveness of intermittent vs. continuous adjunctive therapies following BoNT-A injections for children with cerebral palsy (CP).Methods: A quasi-experimental study was conducted with 80 participants with CP who received adjunctive therapies including physiotherapy and functional electrical stimulation after BoNT-A injections. The participants were randomly divided into two groups. In group A, half of the participants received intermittently adjunctive therapies. In group B, adjunctive therapies were organized continuously for another half. We measured changes in spasticity and dynamic spasticity used by the Modified Ashworth Scale and the Modified Tardieu Scale, and gross motor function used the Gross Motor Function Measure-88. Measurement of spasticity was carried out pre-injections and then 1- and 3-months post-injections. Measurement of gross motor function was organized pre- and post-injections.Results: The effectiveness of BoNT-A injections presented significant improvement in spasticity and gross motor function when it was combined with adjunctive therapies. The continuous adjunctive therapies had a greater reduction of spasticity. Both intermittent and continuous adjunctive therapies had a significant improvement in gross motor function.Conclusions: Our findings add to the evidence of the effectiveness of using different intervals of short-term adjunctive therapies for children with CP after BoNT-A injections.

Hypogonadism: cardiometabolism and gonadal function in men

Hypogonadism is a relatively rare condition in men, which increases in frequency as men age, but also as they become less active and gain weight. In the past 20 years, developing knowledge on the relationship between hypogonadism and cardiovascular and cerebrovascular health and on aspects of metabolic health has become clearer. The relationship between hypogonadism and specific endocrine abnormalities of spermatogenesis is much longer established. Long- and short-term testosterone replacement therapies have well-recognised effects on cardiovascular and cerebrovascular health and on aspects of metabolic health. This leads to a sense of safety when it comes to considering these options as ways of managing the recognised symptoms of hypogonadism and the hidden adverse findings. That confidence has yet to be proven by long-term randomised controlled studies. The use of exogenous gonadotrophins to raise endogenous testosterone levels is a cost-efficient method of achieving spermatogenesis but is not suitable for long-term testosterone maintenance therapy.

Examining Psychotherapeutic Processes with Depressed Adolescents: A Comparative Study of Two Psychodynamic Therapies

To understand processes associated with better or poorer psychotherapy outcomes is vital. This study examined and contrasted interaction patterns between one therapist and two depressed 17-year-old girls, Johanna (good outcome) and Sonja (poor outcome), in short-term psychoanalytic therapies selected from an RCT. Outcome data were collected regarding level of inter- and intra-personal functioning and symptoms of depression. Process data were obtained using the Adolescent Psychotherapy Q-Set on all available sessions. Analyses yielded five relational patterns or "interaction structures" in the two therapy processes; Three explained most of the variance in sessions with Johanna (i.e., 'positive working alliance', 'therapist's active use of psychodynamic techniques', and 'a receptive patient') and two explained more of the variance in sessions with Sonja (i.e., 'therapist using a more problem-solving and symptom-oriented approach' and 'patient displaying limited capacity for mentalization'). The processes in the two cases presented differences related to mentalization, psychological mindedness, and attachment style of the patients. The therapist used different therapeutic approaches, favouring more psychodynamic interventions in the good outcome case and a more problem-solving and symptom-oriented approach with the poor outcome case. In the latter case, the relationship seemed to be more of a struggle.

The Medicinal Chemistry of Chalcones as Anti-Mycobacterium tuberculosis Agents.

Tuberculosis (TB), a highly fatal infectious disease, is caused by Mycobacterium tuberculosis (Mtb) that has inflicted mankind for several centuries. In 2019, the staggering number of new cases reached 10 million resulting in 1.2 million deaths. The emergence of multidrug-resistance- Mycobacterium tuberculosis (MDR-TB) and extensively drug-resistant-Mycobacterium tuberculosis (XDR-TB) is a global concern that requires the search for novel, effective, and safer short-term therapies. Nowadays, among the few alternatives available to treat resistant-Mtb strains, the majority have limitations, which include drug-drug interactions, long-term treatment, and chronic induced toxicities. Therefore, it is mandatory to develop new anti-Mtb agents to achieve health policy goals to mitigate the disease by 2035. Among the several bioactive anti-Mtb compounds, chalcones have been described as the privileged scaffold useful for drug design. Overall, this review explores and analyzes 37 chalcones that exhibited anti-Mtb activity described in the literature up to April 2021 with minimum inhibitory concentration (MIC90) values inferior to 20 μM and selective index superior to 10. In addition, the correlation of some properties for most active compounds was evaluated, and the main targets for these compounds were discussed.

Blood pressure-lowering treatment for prevention of major cardiovascular diseases in people with and without type 2 diabetes: an individual participant-level data meta-analysis

Controversy exists as to whether the threshold for blood pressure-lowering treatment should differ between people with and without type 2 diabetes. We aimed to investigate the effects of blood pressure-lowering treatment on the risk of major cardiovascular events by type 2 diabetes status, as well as by baseline levels of systolic blood pressure. We conducted a one-stage individual participant-level data meta-analysis of major randomised controlled trials using the Blood Pressure Lowering Treatment Trialists' Collaboration dataset. Trials with information on type 2 diabetes status at baseline were eligible if they compared blood pressure-lowering medications versus placebo or other classes of blood pressure-lowering medications, or an intensive versus a standard blood pressure-lowering strategy, and reported at least 1000 persons-years of follow-up in each group. Trials exclusively on participants with heart failure or with short-term therapies and acute myocardial infarction or other acute settings were excluded. We expressed treatment effect per 5 mm Hg reduction in systolic blood pressure on the risk of developing a major cardiovascular event as the primary outcome, defined as the first occurrence of fatal or non-fatal stroke or cerebrovascular disease, fatal or non-fatal ischaemic heart disease, or heart failure causing death or requiring hospitalisation. Cox proportional hazard models, stratified by trial, were used to estimate hazard ratios (HRs) separately by type 2 diabetes status at baseline, with further stratification by baseline categories of systolic blood pressure (in 10 mm Hg increments from <120 mm Hg to ≥170 mm Hg). To estimate absolute risk reductions, we used a Poisson regression model over the follow-up duration. The effect of each of the five major blood pressure-lowering drug classes, including angiotensin-converting enzyme inhibitors, angiotensin II receptor blockers, β blockers, calcium channel blockers, and thiazide diuretics, was estimated using a network meta-analysis framework. This study is registered with PROSPERO, CRD42018099283. We included data from 51 randomised clinical trials published between 1981 and 2014 involving 358 533 participants (58% men), among whom 103 325 (29%) had known type 2 diabetes at baseline. The baseline mean systolic/diastolic blood pressure of those with and without type 2 diabetes was 149/84 mm Hg (SD 19/11) and 153/88 mm Hg (SD 21/12), respectively. Over 4·2 years median follow-up (IQR 3·0-5·0), a 5 mm Hg reduction in systolic blood pressure decreased the risk of major cardiovascular events in both groups, but with a weaker relative treatment effect in participants with type 2 diabetes (HR 0·94 [95% CI 0·91-0·98]) compared with those without type 2 diabetes (0·89 [0·87-0·92]; pinteraction=0·0013). However, absolute risk reductions did not differ substantially between people with and without type 2 diabetes because of the higher absolute cardiovascular risk among participants with type 2 diabetes. We found no reliable evidence for heterogeneity of treatment effects by baseline systolic blood pressure in either group. In keeping with the primary findings, analysis using stratified network meta-analysis showed no evidence that relative treatment effects differed substantially between participants with type 2 diabetes and those without for any of the drug classes investigated. Although the relative beneficial effects of blood pressure reduction on major cardiovascular events were weaker in participants with type 2 diabetes than in those without, absolute effects were similar. The difference in relative risk reduction was not related to the baseline blood pressure or allocation to different drug classes. Therefore, the adoption of differential blood pressure thresholds, intensities of blood pressure lowering, or drug classes used in people with and without type 2 diabetes is not warranted. British Heart Foundation, UK National Institute for Health Research, and Oxford Martin School.

Pre-Discharge and Post-Discharge Management and Treatment Optimization in Acute Heart Failure.

Acute heart failure is associated with high mortality and rehospitalization rates and required urgent evaluation and early initiation or intensification of therapy. The risk of death and heart failure rehospitalization is greatest in the early post-discharge period, particularly within the first 3-6 months, and declines over time, which is referred as a vulnerable period of acute heart failure hospitalization. Therefore, implementation of guidelines-directed optimal therapy is not only so crucial in the acute phase but also very important in the pre-discharge and early post- discharge period in reducing mortality and rehospitalization rates. The pre-discharge period represents a window of opportunity for treatment optimization which includes to eliminate congestion, to treat comorbidities or precipitating factors, and to initiate or uptitrate oral ther- apy before discharge. Early assessment in the post-discharge period based on clinical evalu- ation and laboratory exams, further optimization of disease-modifying therapy is associated with lower 30-day hospitalization for heart failure. In clinical practice, clinicians usually focus on acute phase intravenous medications and short-term device therapies and, in fact, neglect short- and long-term comprehensive approaches. This paper reviews management strategies that may help reduce mortality and heart failure rehospitalizations in pre-discharge and post- discharge periods and include adopting holistic approaches for heart failure, increasing optimi- zation of evidence-based therapies, treating cardiac and noncardiac comorbidities, improving care transitions, monitoring, and disease management.

What Works for Whom: Patients' Psychological Resources and Vulnerabilities as Common and Specific Predictors of Working Alliance in Different Psychotherapies.

BackgroundAcross different types of psychotherapy, one of the most robust predictors of better therapeutic outcomes is a good working alliance between patient and therapist. Yet there is little comparative research on whether particular patients more likely achieve a better alliance in certain treatments which represent particular therapeutic approaches or durations.Methods326 patients suffering from depressive and/or anxiety disorder were randomized into two short-term (solution-focused or psychodynamic) and one long-term (psychodynamic) therapy models. Treatments lasted ~7 and 36 months, respectively. Before randomization, patients were assessed with the interview-based Suitability for Psychotherapy Scale and filled Childhood Family Atmosphere and Life Orientation Test questionnaires. Patients filled Working Alliance Inventory after 3rd therapy session and at end of treatment; the long-term therapy patients, additionally, at 7 months' time point. Linear regression models were used.ResultsGreater psychological resources (e.g., capacity for self-reflection, affect regulation, flexible interaction) had little effect on alliance during the course of the short-term therapies. However, they did predict better working alliances at end of long-term as opposed to short-term therapy. Childhood adversities impacted alliances already at 7 months.ConclusionsAlthough patients with certain qualities achieve better alliances in long-term as opposed to short-term therapies, apparently the theoretical orientation of therapy makes little difference. For patients with childhood adversities, differences between long-term (psychodynamic) treatment vs. various brief therapy models may be particularly salient.

Ego Impairment Index (EII-2) as a predictor of outcome in short- and long-term psychotherapy during a 5-year follow-up.

ObjectiveThis study examined the predictive ability of the Rorschach‐based Ego Impairment Index (EII‐2) on outcome of psychotherapy in different types and durations of therapy.MethodA total of 326 outpatients suffering from depressive or anxiety disorders were randomized into receiving solution‐focused (n = 97), short‐term psychodynamic (n = 101), or long‐term psychodynamic psychotherapy (n = 128). Psychotherapy outcome assessments during the 5‐year follow‐up period covered psychiatric symptoms, social functioning, and work ability.ResultsLower EII‐2 values, which indicate less problematic ego functioning, were found to predict faster improvement in both short‐term therapies as compared to long‐term psychotherapy.ConclusionThe results provide preliminary support for the utility of EII‐2 as a complementary measure to interview‐based methods for selecting between short‐ and long‐term therapies.

Trajectories of change in general psychopathology levels among depressed adolescents in short-term psychotherapies

Objective to identify and describe trajectories of change in general psychopathology (p) levels among depressed adolescents who received one of three types of short-term therapies (namely Cognitive–Behavioural Therapy, Short-Term Psychoanalytic Psychotherapy, and a Brief Psychosocial Intervention). Method Participants were 465 adolescents with MDD who participated in an RCT comparing three treatments for depression. Narrow-band measures of depression, anxiety, obsessions-compulsions, and conduct problems were assessed at six-time points, and bifactor analysis was performed to extract p factor scores. These scores were submitted to Latent Class Growth Analyses to identify patterns of change over time. Results Three different trajectories of change in p were identified. Two trajectories displayed reductions in p across time-points: one a rapid decrease, and the other slower but steady improvement. The third trajectory indicated a limited decrease in p up until the 12th week after baseline but no further improvement at subsequent time-points. Patients’ baseline p significantly predicted their outcome trajectories. Conclusion Exploring change in p seemed to describe more parsimoniously the patients’ outcomes than the narrow-band assessment of depressive symptoms. Patients with high baseline p were more likely to have poorer outcomes, potentially indicating a need to develop more intensive and tailored treatments for this population.

What patient characteristics predict treatment success or failure in short- and long-term psychotherapies?

Background Patient factors make a strong contribution to psychotherapy outcome. Pre-treatment patient characteristics – demographic, pathology and personal – may either facilitate achievement of recovery and lasting benefits or constitute a risk for different kinds of treatment failure. Methods A narrative review focused on evaluating the role of pre-treatment patient characteristics on the success or failure of short- and long-term psychotherapy for mood and anxiety disorders. Success was conceptualized as significant improvement or recovery and failure as non-improvement, deterioration, or non-attendance/dropout. Results There is no consistent evidence of demographic variables as predictors of treatment failure, except for lower socioeconomic status, being male and ethnic minority status for therapy non-attendance. Patients’ pathology, i.e. severity of psychiatric symptoms, greater functional impairment, personality disorder and other comorbidities, have been shown to be mostly associated with lesser recovery across different types of disorders, especially in short-term therapies, but it does not consistently predict treatment failure. Some pre-treatment personal characteristics (e.g. secure attachment style) and capacities (e.g. motivation, self-observing capacity and good overall psychological suitability) mostly increase the likelihood of therapy success whereas some vulnerabilities and poor psychological suitability, or overall complexity (i.e. combination of demographic, clinical and personality factors), increase the risk of poor outcome mostly in short-term but not in long-term therapy, or moderate dropout. Likewise, the subsequent match between therapy type and patient’s pre-treatment expectations and preferences may account for different aspects of treatment failure. Conclusions The relative importance of pre-treatment patient characteristics on treatment failure is not known. Meta-analyses on this issue are needed.

Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations

Gene therapy for hemophilia is designed to produce health gains for patients over many years. Rewarding that value creation on the basis of a one-time treatment implies a large upfront cost. This cost can only be justified by long-term health benefits and being cost-effective compared with conventional treatments. Yet, uncertainties about the long-term benefits make it challenging to assess clinical and economic value of gene therapies at launch. We identify and discuss key methodological challenges in assessing the value of gene therapy for hemophilia, including the immaturity of evidence on the durability of benefits, lack of definition and valuation of cure for chronic diseases, absence of randomized controlled trials, limitations of traditional quality of life measures in hemophilia, approach for qualifying cost-savings compared with current treatments, and choice of perspective. The Institute for Clinical and Economic Review has developed a framework for assessing single or short-term therapies (ICER-SST) and has applied it in hemophilia. After reviewing this framework and its application, we recommend the following when assessing the value of hemophilia gene therapies: (1) leveraging expert clinical opinion to justify assumptions on the durability of benefits; (2) using external synthetic controls and lead-in, self-controlled trials to assess comparative effectiveness; (3) addressing limitations of traditional quality of life measures through the use of modified utility collection approaches; (4) adjusting cost offsets from gene therapies with caution; (5) considering outcome-based contracting to address uncertainties about prices and long-term outcomes; and (6) presenting societal and healthcare system perspectives in parallel.

Treatment Patterns in Patients with Diagnostic Imaging for Low Back Pain: A Retrospective Observational Study.

PurposeLow back pain (LBP) is one of the most frequent reasons for medical consultations. Literature suggests a large evidence-performance gap, especially regarding pain management. Therefore, the monitoring of treatment patterns is important to ensure high quality of treatment. This study aimed to describe treatment patterns specific to patients with diagnostic imaging of the spine for LBP.Patients and MethodsThe study was retrospective observational and based on health claims data from 2015 to 2019 provided by a Swiss health insurance company covering around 12% of the population. Patients, ≥18 years of age, with diagnostic imaging of the spine were included and observed 12 months before and after imaging. Patients with back surgery or comorbidities associated with the use of pain medications were excluded.ResultsIn total, 60,822 patients (mean age: 53.5 y, 56.1% female) were included and 85% received at least one pain medication. Of these, non-steroidal anti-inflammatory drugs, paracetamol, or opioids were prescribed in 88.6%, 70.7%, and 40.3% of patients, respectively. Strong opioids were used in 17% of patients given opioids. Patients with combinations of diagnostic imaging methods had the highest odds of receiving pain medication prescriptions (1.81, 95% CI: 1.66, 1.96, P < 0.001). Prescribed defined daily doses corresponded to short-term therapies.ConclusionAlthough the majority of patients received non-opioid short-term therapies, we found a substantial use of opioids, and in particular, a relative high usage of strong opioids. Our results highlighted the importance of both patient and healthcare provider awareness regarding the prudent treatment of LBP.