Understanding patient and caregiver perspectives using a dyad approach for data collection: A systematic review of the literature

Treatments outside of a clinical setting may be managed independently by the pediatric patient, independently by a caregiver, or by the patient and caregiver together. Best practices for pediatric clinical outcome assessment (COA) recommend patient-reported outcome (PRO) and/or observer-reported outcome (ObsRO) measures to assess the patient experience of a condition or its treatment. However, a dyad approach where patients and caregivers can complete assessments together may be useful for assessing a shared treatment experience that may not be adequately captured by a PRO and/or an ObsRO. A systematic, targeted literature review of empirical literature was conducted to identify and describe published studies detailing dyad patient-caregiver outcome reporting approaches. The search was run in the MEDLINE®, Embase, and PsycINFO® databases using the OvidSP platform and was limited to English-language studies published within 10 years of the conducted search on 28 September 2021, and 13 articles were selected for full-text review based on pre-specified criteria. Advantages and disadvantages for use of a dyad data collection approach are discussed. Though not appropriate for all settings, dyad data collection may be useful for situations where the best practice approach to measurement does not capture all relevant perspectives, or the use of PRO and ObsRO also does not comprehensively capture all relevant concepts. In following, it may offer a pragmatic solution that can minimize the use of proxy assessment and limit missing data, particularly in research involving a shared patient and caregiver treatment experience. Experience Framework This article is associated with the Staff & Provider Engagement lens of The Beryl Institute Experience Framework (https://www.theberylinstitute.org/ExperienceFramework). Access other PXJ articles related to this lens. Access other resources related to this lens

Cost-Effectiveness Model for Neovascular Age-Related Macular Degeneration: Comparing Early and Late Treatment with Pegaptanib Sodium Based on Visual Acuity

AbstractObjectiveTo compare the cost-effectiveness of pegaptanib and usual care within three distinct cohorts of subfoveal neovascular age-related macular degeneration (NV-AMD) patients, that is, those with early, moderate, and late disease, using a comprehensive economic model.MethodsA Markov framework was used to model lifetime movement of a subfoveal NV-AMD cohort through health states based on visual acuity. The model takes a US payer perspective of patients over the age of 65 years. Clinical efficacy was based on published results for the 0.3 mg pegaptanib and usual care groups. Expert interviews were conducted to determine adverse event treatment patterns and vision rehabilitation resource use. Incidence and costs of comorbidities such as depression and fractures associated with the effects of declining visual acuity were based on our previously published analysis of Medicare data. Transition probabilities were derived from published clinical trial data for each 3-month cycle. Utilities were derived from published sources. Three runs of the model were conducted with cohorts of newly diagnosed patients. Patients were classified as having early, moderate, or late NV-AMD defined as visual acuity in the better-seeing eye of 20/40 to more than 20/80, 20/80 to more than 20/200, and 20/200 to more than 20/400, respectively. Costs and outcomes were discounted 3.0% per annum.ResultsIncremental costs per vision-year gained and per quality-adjusted life-year (QALY) gained for early NV-AMD patients were approximately one-third those of patients with late disease ($15,279 vs.$57,230 and $36,282 vs.$132,381, respectively). On average, patients treated early with either pegaptanib or usual care incurred lower lifetime total direct costs than those treated later. Sensitivity analysis showed that base-case incremental costs per QALY gained for pegaptanib versus usual care were relatively robust.ConclusionsFor patients with subfoveal NV-AMD, treatment with pegaptanib should be started as early as possible to maximize the clinical and economic benefits

A Diagnostic Aid For Detecting Multiple Mental Disorders In Primary Care: The Symptom Driven Diagnostic System For Primary Care (sdds-pc*).

A high rate of undiagnosed mental disorders in primary care has been well documented for nearly three decades. (Shepherd et al 1966; Regier et al 1978,1993; Goldberg et al 1980; Van Hermert et al 1993; Olfson and Klerman, 1992; Barrett et al 1988). Failure to recognize mental illness has been shown to lead to undertreatment, greater impairment, and a longer duration of illness (Ormel et al 1991). The few efforts to change the diagnostic practice of primary care physicians, either through providing information from a patient screen completed prior to the physician visit or through physician education, have provided equivocal results. (Higgins 1994; Rand et al 1988; Goldberg 1980). The effects of improved physician recognition on decreasing patients’ health care use have also been equivocal, probably because there are many steps between recognition and patient outcome. These steps include accurate diagnosis, proper treatment, patient compliance, and timely follow-up with adjustment in treatment, as needed. Efforts to improve detection of mental disorders have concentrated on developing patient screens, which usually includea list ofpsychiatric symptoms independent of specific diagnoses, (Goldberg et al 1980; Borgquist et al 1993; Von Korff et al 1987) or which screen for one diagnosis; depression or alcoholism (Selzer 1971; Babor et al 1992). The former approach is limited because symptoms are not directly related to a treatable disorder, the latter, because patients may have more than one treatable psychiatric disorder (Kessler et al 1994). Moreover, screens are not widely used in primary care (Nelson and Berwick, 1989). Because most primary care visits are 15 minutes long or less, there is a need to develop rapid assessment methods that can be incorporated into routine care (Barrett 1991; Mitchell et al 1988; Anderson and Mattsson 1989)

Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR Good Research Practices for the Assessment of Patient-Reported Outcomes in Children and Adolescents Task Force

Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR Good Research Practices for the Assessment of Patient-Reported Outcomes in Children and Adolescents Task Force Abstract. Patient-reported outcome (PRO) instruments for children and adolescents are often included in clinical trials with the intention of collecting data to support claims in a medical product label. The purpose of the current task force report is to recommend good practices for pediatric PRO research that is conducted to inform regulatory decision making and support claims made in medical product labeling. The recommendations are based on the consensus of an interdisciplinary group of researchers who were assembled for a task force associated with the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). In those areas in which supporting evidence is limited or in which general principles may not apply to every situation, this task force report identifies factors to consider when making decisions about the design and use of pediatric PRO instruments, while highlighting issues that require further research. Five good research practices are discussed: 1) Consider developmental differences and determine age-based criteria for PRO administration: Four age groups are discussed on the basis of previous research

Development of ACRODAT®, a new software medical device to assess disease activity in patients with acromegaly

Despite availability of multimodal treatment options for acromegaly, achievement of long-term disease control is suboptimal in a significant number of patients. Furthermore, disease control as defined by biochemical normalization may not always show concordance with disease-related symptoms or patient's perceived quality of life. We developed and validated a tool to measure disease activity in acromegaly to support decision-making in clinical practice. An international expert panel (n = 10) convened to define the most critical indicators of disease activity. Patient scenarios were constructed based on these chosen parameters. Subsequently, a panel of 21 renowned endocrinologists at pituitary centers (Europe and Canada) categorized each scenario as stable, mild, or significant disease activity in an online validation study. From expert opinion, five parameters emerged as the best overall indicators to evaluate disease activity: insulin-like growth factor I (IGF-I) level, tumor status, presence of comorbidities (cardiovascular disease, diabetes, sleep apnea), symptoms, and health-related quality of life. In the validation study, IGF-I and tumor status became the predominant parameters selected for classification of patients with moderate or severe disease activity. If IGF-I level was ≤1.2x upper limit of normal and tumor size not significantly increased, the remaining three parameters contributed to the decision in a compensatory manner. The validation study underlined IGF-I and tumor status for routine clinical decision-making, whereas patient-oriented outcome measures received less medical attention. An Acromegaly Disease Activity Tool (ACRODAT) is in development that might assist clinicians towards a more holistic approach to patient management in acromegaly

Results from an international multicenter trial evaluating the ease-of-use of and preference for a newly developed disposable injection pen for the treatment of growth hormone deficiency in treatment-naive children and adults

Previous research has reported that ease of use of and preference for a delivery device are associated with greater patient compliance - an important factor in achieving optimal therapeutic results. The objective of this study was to assess the ease-of-use of a new disposable pen (GoQuick((R)), Pfizer, Inc.) versus the current reusable pen (GENOTROPIN Pen((R)), Pfizer, Inc.) to inject a daily dose of recombinant DNA origin human growth hormone, Genotropin((R)) (somatropin) in standard practice. In this randomized, crossover, multicenter, multinational, openlabel study, ease-of-use of and preference for the two pens were assessed in three treatment-naive populations: 1) parents of very young children; 2) parent-child dyads; and 3) adults via use of a validated self-report Injection Pen Assessment Questionnaire (IPAQ) after 2 months of at-homeuse experience. The primary endpoint was the proportion of participants who reported the new disposable pen to be no different from or easier to use than the current reusable pen. Safety was also assessed and reported according to local legal requirements. Of the 120 screened patients, 119 were included in the ease-of-use analysis and all were included in the safety analyses. In all, 67.2% found the new somatropin disposable pen to be no different from or easier to use than the reusable pen (95% confidence interval: 58.8-75.7). Most adverse events were mild or moderate. No deaths or device-or treatment-related serious adverse events were reported. These results suggest that improvements made to the reusable somatropin pen are tangible and recognizable to treatment-naive patients and their caregivers, child-caregiver dyads, and adults, and may positively impact continued compliance with therapy

Economic Evaluation of Systemic Treatments for Cytomegalovirus Retinitis in Patients with AIDS

Objective: To determine the cost of using systemic therapy to treat newly diagnosed cytomegalovirus (CMV) retinitis in persons with AIDS. Design: Incidence-based simulation model of CMV treatment from a government payer perspective. Setting: Swiss healthcare system. Patients and participants: Patients with AIDS and newly diagnosed CMV retinitis. Interventions: Patients were assigned to 1 of 4 treatment regimens for induction and maintenance therapy: 1. intravenous (IV) cidofovir induction and maintenance (cidofovir IV/IV); 2. IV foscarnet induction and maintenance (foscarnet IV/IV); 3. IV ganciclovir induction and maintenance (ganciclovir IV/IV); and 4. IV ganciclovir induction and oral (PO) ganciclovir maintenance (ganciclovir IV/PO). Following a second relapse, patients were assigned to one of the other regimens. Main outcome measures: Time to first and subsequent progression, duration of maintenance treatment and direct medical expenditures [1998 Swiss francs (SwF)]. Results: The median time to first progression was longest for cidofovir IV/IV, followed by foscarnet IV/IV, ganciclovir IV/IV and ganciclovir IV/PO. Mean survival was 13 months and mean costs for this period in the base case were lowest in those initially treated with cidofovir (SwF146 742), followed by initial treatment with foscarnet IV/IV (SwF194 809), ganciclovir IV/PO (SwF195 190) and ganciclovir IV/IV (SwF243 964). Costs were most sensitive to changes in efficacy estimates. Conclusions: Of the regimens studied, initiation of treatment with systemic cidofovir appears least costly over a 13-month period.Antivirals, Cidofovir, Cost analysis, Cytomegalovirus infections, Foscarnet, Ganciclovir, Pharmacoeconomics, Retinitis

Development of ACRODAT®, a new software medical device to assess disease activity in patients with acromegaly

textabstractPurpose: Despite availability of multimodal treatment options for acromegaly, achievement of long-term disease control is suboptimal in a significant number of patients. Furthermore, disease control as defined by biochemical normalization may not always show concordance with disease-related symptoms or patient’s perceived quality of life. We developed and validated a tool to measure disease activity in acromegaly to support decision-making in clinical practice. Methods: An international expert panel (n = 10) convened to define the most critical indicators of disease activity. Patient scenarios were constructed based on these chosen parameters. Subsequently, a panel of 21 renowned endocrinologists at pituitary centers (Europe and Canada) categorized each scenario as stable, mild, or significant disease activity in an online validation study. Results: From expert opinion, five parameters emerged as the best overall indicators to evaluate disease activity: insulin-like growth factor I (IGF-I) level, tumor status, presence of comorbidities (cardiovascular disease, diabetes, sleep apnea), symptoms, and health-related quality of life. In the validation study, IGF-I and tumor status became the predominant parameters selected for classification of patients with moderate or severe disease activity. If IGF-I level was ≤1.2x upper limit of normal and tumor size not significantly increased, the remaining three parameters contributed to the decision in a compensatory manner. Conclusion: The validation study underlined IGF-I and tumor status for routine clinical decision-making, whereas patient-oriented outcome measures received less medical attention. An Acromegaly Disease Activity Tool (ACRODAT) is in development that might assist clinicians towards a more holistic approach to patient management in acromegaly