One-Year Medication Treatment Patterns, Healthcare Resource Utilization, and Expenditures for Medicaid Patients with Schizophrenia Starting Oral Atypical Antipsychotic Medication.

Oral atypical antipsychotic (OAAP) medications are the most commonly prescribed treatment for the management of schizophrenia symptoms. This retrospective study, using Medicaid claims data (2016-2020), followed patients for 12 months after initiating OAAP therapy. Study outcomes included OAAP adherence, switching, augmentation, healthcare resource utilization (HRU), and expenditures. All-cause and schizophrenia-related HRU and expenditures were compared between adherent and nonadherent cohorts. Among 13,007 included patients (39.1 ± 12.8 years of age, 57.0% male, 36.1% Black, 31.8% White, 9.7% Hispanic), 25.7% were adherent to OAAPs (proportion of days covered [PDC] ≥ 0.8). During the 1-year follow-up period, Black individuals were in possession of an OAAP for an average of 166 days compared to 198 and 202 days for White and Hispanic patients, respectively. Approximately 16% of patients switched OAAP medications and 3.2% augmented therapy with an OAAP added to their index medication. Nearly 40% of patients were hospitalized during follow-up and 68.4% had emergency department (ED) visits. A greater proportion of nonadherent patients had all-cause inpatient (41.7% vs. 34.1%, p < 0.001) and ED visits (71.7% vs. 58.8%, p < 0.001) compared to adherent patients. Annual total healthcare expenditures were $21,020 per patient; $3481 higher for adherent versus nonadherent patients. Inpatient expenditures comprised 44.6% and 30.6% of total expenditures for nonadherent and adherent patients, respectively. Hospitalized patients' total expenditures were $23,261 higher compared to those without a hospitalization. Adherence to OAAP medication is suboptimal and associated with increased utilization of costly hospital and ED resources. Efforts to improve therapies and increase medication adherence could improve clinical and economic outcomes among individuals with schizophrenia.

Estimating minimal important differences for several scales assessing function and quality of life in patients with attention-deficit/hyperactivity disorder.

OBJECTIVE: Defining minimal important difference (MID) is critical to interpreting patient-reported outcomes data and treatment efficacy in clinical trials. This study estimates the MID for the Weiss Functional Impairment Rating Scale-Parent Report (WFIRS-P) and the Child Health and Illness Profile-Parent Report (CHIP-CE-PRF76) among parents of young people with attention-deficit/hyperactivity disorder (ADHD) in the UK. METHODS: Parents of children (6-12 years; n=100) and adolescents (13-17 years; n=117) with ADHD completed a socio-demographic form, the CHIP-CE-PRF76, the WFIRS-P, and the Pediatric Quality of Life scale at baseline and 4 weeks later. At follow-up, a subset of parents completed anchor questions measuring change in the child/adolescent from baseline. MIDs were estimated using anchor-based and distribution-based methods, and separately for children and adolescents. RESULTS: The MID estimates for overall change in the WFIRS-P total score ranged from 11.31 (standard error of measurement) to 13.47 (anchor) for the total sample. The range of MID estimates for the CHIP-CE-PRF76 varied by domain: 6.80-7.41 (satisfaction), 6.18-7.34 (comfort), 5.60-6.72 (resilience), 6.06-7.57 (risk avoidance), and 4.00-5.63 (achievement) for the total sample. Overall, MID estimates for WFIRS-P MID and CHIP-CE-PRF76 were slightly higher for adolescents than for children. CONCLUSION: This study estimated MIDs for these instruments using several methods. The observed convergence of the MID estimates increases confidence in their reliability and could assist clinicians and decision makers in deriving meaningful interpretations of observed changes in the WFIRS-P and CHIP-CE in clinical trials and practice.

Increasing Patient Involvement in Drug Development.

BACKGROUND: To ensure the creation of treatments that maximize value at the lowest cost, all aspects of the health care system need to align with patient needs and preferences. Despite growing efforts to engage patients in research and regulatory activities, the pharmaceutical industry has yet to maximize patient involvement in the drug development process. OBJECTIVE: To gain a better understanding of the present state of patient involvement in drug development. METHODS: Through a semistructured interview methodology, we sought to identify opportunities, barriers, and examples of patient involvement in the drug development process. Telephone interviews were conducted with six senior leaders of evidence generation within the pharmaceutical industry and four patients. These interviews were supplemented with interviews with a research funder, a regulator, a patient advocacy group, and a caregiver. RESULTS: Although our interviewees spoke of the potential benefits of aligning research around the needs of patients, there were few examples they could share to suggest this was occurring at scale. A number of barriers were identified including the added burden associated with adverse event reporting, concerns about patient representativeness or their ability to participate in drug development conversations, and the costs in time and resources involved relative to returns on investment. CONCLUSIONS: As health care systems continue to evolve and establish patients as the primary stakeholder in their health care decision making, the pharmaceutical industry will need to be innovative to demonstrate the value of their products relative to the outcomes experienced by patients. Pharmaceutical companies should recognize the value of involving patients across the entire product life cycle and work to transform present perceptions and practices throughout their organizations.

Persistency, medication prescribing patterns, and medical resource use associated with multiple sclerosis patients receiving oral disease-modifying therapies: a retrospective medical record review.

BACKGROUND: In the US, the approved multiple sclerosis (MS) oral disease-modifying therapies (ODMTs) are fingolimod (FTY), teriflunomide (TFN), and dimethyl fumarate (DMF). FTY and TFN are recommended with once-daily doses with no up-titration, whereas DMF treatment is recommended twice-daily (BID) and is initiated with a 7-day starter dose of 120 mg BID before up-titration to the maintenance dose of 240 mg BID. Limited information exists regarding real-world ODMT prescribing patterns to aid physician/patient decision-making. METHODS: Eligible patients for this retrospective medical record review were ≥18 years, had one visit related to ODMT initiation (index visit), and ≥1 visit within 12 months before and after the index visit. Primary objectives were to assess post-index ODMT persistency (i.e., discontinuation), prescribing patterns (medication switching, dose up-titrations, dose reduction, re-starts, and add-ons) and medical resource utilization (office-visits, MRI procedures, and mobility indicators) at distinct time windows of 3, 6, 9, and 12 months. Chi-square or Wilcoxon Rank Sum tests were used for 3-way ODMT group comparisons. RESULTS: Medical records of 293 MS-diagnosed patients using ODMTs were abstracted from 19 US-based neurology clinics between December 31, 2010 and June 30, 2014 (FTY: 101; DMF: 133; TFN: 59). Persistency rates among ODMT groups were similar. MS-related medication switching, dose reduction, re-starts, and add-ons were infrequently observed and were similar across ODMT groups. Of DMF patients with a confirmed starting dose of 120 mg BID with ≥12 months follow-up (n = 26), the percentage who were prescribed dose up-titrations to the recommended maintenance DMF dose was 23.1 % at 1-3 months, 26.9 % at 4-6 months, 42.3 % at 7-9 months, and 0 % at 10-12 months. There were no significant differences at any time window among the ODMT groups in the number of office visits or percent of patients receiving MRIs. Mobility indicator patterns (proportion of patients with abnormal gait, wheelchair use, etc.) were consistent over time. CONCLUSIONS: There was no difference in persistency, prescribing patterns (medication switching, dose reduction, re-starts, and add-ons) or medical resource utilization (office-visits, MRI procedures, and mobility indicators) among the ODMTs. However, in a small sub-group of patients, delays of up to 9 months in DMF dose-up titration to the recommended maintenance dose were observed.

Relationship between brain volume loss and cognitive outcomes among patients with multiple sclerosis: a systematic literature review.

Patients with multiple sclerosis (MS) experience varying rates of brain volume (BV) loss ranging from 0.5 to 1.5 % per year. In addition, 66 % of patients with MS experience cognitive impairment, resulting in impact on daily activities. A systematic literature review (2003-2013) was conducted to identify all studies reporting a relationship between whole BV measures and selected patient outcomes measuring cognition, including the Symbol Digit Modalities Test (SDMT), Paced Auditory Serial Addition Test (PASAT) and MS Functional Composite (MSFC) scores. We identified 18 studies reporting associations between whole BV and cognitive outcomes. Six studies (33 %) examined the association between BV and SDMT; all six studies reported that BV loss (BVL) was significantly associated with a decline in SDMT scores (all p < 0.05). Among 14 studies (78 %) that examined the association between BV and PASAT scores, 12 (86 %) found a significant relationship between BVL and lower PASAT scores (all p < 0.05). Of the seven studies (39 %) that looked at BV and MSFC, six studies (86 %) found BVL significantly associated with lower MSFC scores (all p < 0.05). Our study demonstrated that BVL is associated with declines in cognition in MS patients across several cognition measures. The results of this study suggest that BV is a critical component of disease activity and progression in MS and has implications for treatment decisions to minimize BVL and preserve cognitive functioning.

An overview of current trends and gaps in patient-reported outcome measures used in haemophilia.

AIM: This review summarises the importance, recent progress and issues in measuring patient-reported outcomes (PROs) in haemophilia research. METHODS: A critical review of recent advances and trends in measuring haemophilia-related PROs was conducted, using current regulatory guidelines and methodological recommendations to evaluate these instruments. RESULTS: Although regulators, payers and policymakers increasingly consider the patient's perspective to be important in treatment decision-making, to date, few haemophilia intervention studies have meaningfully applied PRO endpoints. Condition-specific PRO instruments have been developed, but most are not fully validated; sensitivity to subgroup differences and changes over time is unclear. Generic PROs and instruments developed for other conditions have been used to measure health-related quality of life (HRQL) in haemophilia patients, but little evidence of their validity for this purpose exists. Haemophilia presents a number of challenges to developing valid, reliable and responsive PRO instruments, including the rarity of the disorder; necessitating research in multiple counties to attain sufficient sample size; the chronic nature of the condition; acute exacerbations of illness; age and geographical region variations with respect to treatment; differences in treatment regimens, range of disease severity and phenotypes; and changes in patients' perceived health status over time. Given that haemophilia begins at birth, the illness has an impact on the lives of caregivers, although the extent of the impact is largely unknown. CONCLUSIONS: Patient perspectives are crucial to understanding the best and most cost-effective haemophilia treatment approaches. More research is needed on the ability of current disease-specific and generic PRO instruments to capture responsiveness to treatments over time and subgroup differences in outcomes. Inclusion of PROs in clinical trials is necessary to answer these questions.

Illustrating the impact of mild/moderate and severe haemophilia on health-related quality of life: hypothesised conceptual models.

INTRODUCTION/AIMS: Haemophilia and its treatment have a significant impact on patients' lives. The study objectives were to understand the impacts of haemophilia and its treatment from the patient perspective and to inform the development of comprehensive health-related quality-of-life (HRQL) conceptual models to illustrate these impacts. METHODS: The study included two phases. Phase I involved a review of literature published from 1995 to 2010, qualitative analysis of six patient (N = 31) and three healthcare provider (N = 15) focus group transcripts, and interviews with two experts to inform draft conceptual models of mild/moderate and severe haemophilia. Phase II involved interviews with 20 haemophilia patients and qualitative analysis of transcripts to confirm the concepts and structure of the conceptual models. RESULTS: The literature search resulted in 66 publications assessing HRQL, four of which were qualitative studies on the impact of haemophilia from the patient perspective. Results from Phase I indicated that acute bleeding events result in pain, swelling, bruising and restricted joint movement; repeated joint bleeds result in chronic symptoms, such as pain and arthropathy. Acute bleeds cause interruptions in daily activities and interfere with work/school. Patients have fears about having bleeds, which can affect their participation in activities, such as sports or crowded events. Patients also expressed feelings of depression, frustration, isolation and embarrassment. Results of Phase II corroborated findings from Phase I. CONCLUSIONS: The conceptual models illustrate the substantial impact of haemophilia and its treatments on patients' lives and can help inform clinical study design and the selection of endpoints to assess treatment benefit.

Patient-reported experience of bleeding events in haemophilia.

BACKGROUND: Patients with haemophilia experience bleeds because of absent or reduced clotting factor. The study objective was to understand the bleeding experience from the patients' perspective. MATERIALS AND METHODS: Individuals with moderate/severe haemophilia participated in interviews and were asked to describe their most recent bleeding experience, including symptoms, signs of onset, impacts, when bleeding stopped and treatment effectiveness. Interview transcripts were analysed using a thematic analysis involving the coding of transcripts to identify key concepts and themes. RESULTS: Twenty males [10 adults, mean age = 41 (19-52); 10 adolescents, mean age = 13 (12-17)] with moderate (n = 5) or severe (n = 15) haemophilia participated. Symptoms signalling bleed onset included pain, swelling, stiffness, tingling/numbness and/or warmth. Participants reported feeling anger and frustration due to the unpredictable nature, pain and inconvenience of the episode. Adults sometimes reported delaying treatment due to inconvenience or cost; adolescents generally treated right away. Reported bleed severity was influenced by pain level, speed of symptom progression, location, continued use of the affected area, recurrence in same location of recent bleed and treatment delay. Participants reported that it was 'easy' to know when the bleed had stopped. Participants reported that symptoms might linger for days before they returned back to 'normal'. CONCLUSIONS: This qualitative study details the substantial impact of an acute bleed from the patient perspective. Given that treatment was reported to be delayed in part due to inconvenience, more convenient treatment options could help reduce delays in treating bleeds and thereby minimise bleed-related impacts. Clinical studies in haemophilia should include validated patient-reported measurements of acute symptoms and bleed severity to comprehensively assess the bleeding event.

Parent preferences regarding stimulant therapies for ADHD: a comparison across six European countries.

The objective is to identify attributes of ADHD stimulant medications that influence treatment preferences of parents of children and adolescents with ADHD across six European countries, using a discrete choice experiment (DCE). Different attributes (and associated levels) of stimulant therapies were identified through literature review and clinician input. Attributes included duration and degree of symptom control after each dose, frequency of medication dosing, potential for treatment to be abused, the side effects of vomiting, loss of appetite, and sleep disturbance. Attributes and levels were combined using an orthogonal design to produce a number of discrete hypothetical treatments. Parents were recruited via patient panels in different countries and asked to complete a survey. DCE data were analyzed using conditional logit models to explore the impact of each attribute on participants' choices. Six hundred individuals (220 parents of adolescents and 380 parents of children) participated. All attributes were significant predictors of choice (p < 0.01). 'Degree of symptom control' was the most important attribute whereby the odds of choosing 'very much improved symptoms' compared with 'minimally improved' was 4.85 [95 % confidence interval (CI) = 4.28-5.49] for the adolescent group and 6.37 (95 % CI = 5.79-7.01) for the child group. Some inter-country differences emerged, e.g., achieving the best degree of symptom control was more important to parents in some countries than others. In conclusion, the study showed that duration and degree of symptom control were the most important aspects of treatment for parents in all countries. The findings revealed cultural differences in the relative importance of attributes.

Association Between Persistence with Oral Atypical Antipsychotic Medications and Hospital and Emergency Department Utilization in Medicaid Patients with Schizophrenia.

Purpose: To examine 1-year persistence with oral atypical antipsychotics (OAAPs) for Medicaid patients with schizophrenia and assess the association between OAAP persistence and hospital and emergency department (ED) resource utilization. Patients and Methods: Using 2016-2020 multi-state Medicaid claims data, this retrospective study followed patients diagnosed with schizophrenia for 12 months after initiating OAAP therapy. Patients started on an OAAP with no evidence of antipsychotic use in the previous 6 months were included if they had a diagnosis of schizophrenia, were not dually enrolled in Medicaid and Medicare, did not switch to a long-acting injectable antipsychotic, and were continuously eligible 6 months before and 12 months after the initial OAAP prescription (index date). OAAP persistence was measured allowing for a <60-day gap. All-cause and schizophrenia-related inpatient and emergency department (ED) resource utilization during the follow-up period were compared between OAAP persistent and non-persistent groups. Results: The study sample of 13,007 had an average age of 39.1 years and 57.0% were male. Patients were persistent with their index OAAP for 135 days on average and 73.1% had a ≥60-day gap in antipsychotic therapy post-index. While 32.8% and 28.6% of patients who did not persist with their index OAAP restarted the index OAAP or switched to a different OAAP medication later in the year, respectively, a larger proportion (38.6%) had no further OAAP prescriptions. After adjustment for demographic and clinical variables, compared to non-persistent patients, persisting with OAAPs was significantly associated with fewer all-cause and schizophrenia-related hospitalizations (Incidence Rate Ratio [IRR]=0.742, p<0.001; IRR=0.823, p<0.001; respectively) and ED visits (IRR=0.759, p<0.001; IRR=0.773, p<0.001; respectively). Conclusion: Non-persistence with OAAP medication is common among patients with schizophrenia and associated with negative outcomes including increased utilization of hospital and ED resources. Patient-centered interventions that improve antipsychotic persistence should be implemented to facilitate optimal outcomes in this population.

Adult ADHD patient experiences of impairment, service provision and clinical management in England: a qualitative study.

BACKGROUND: There is limited evidence of the unmet needs and experiences of adults with Attention Deficit Hyperactivity Disorder (ADHD) in the published scientific literature. This study aimed to explore the experiences of adults in England with ADHD regarding access to diagnostic and treatment services, ADHD-related impairment and to compare experiences between patients diagnosed during adulthood and childhood. METHODS: In this qualitative study, 30 adults with ADHD were recruited through an ADHD charity (n = 17) and two hospital outpatient clinics for adults with ADHD in England (n = 13). Half of the participants were diagnosed with ADHD during childhood or adolescence and the remainder during adulthood. Semi-structured interviews were conducted and data was analysed using a thematic approach based on Grounded Theory principles. RESULTS: Analysis revealed five core themes: 'An uphill struggle': the challenge of accessing services, 'Accumulated Psychosocial Burden and the Impact of ADHD', 'Weighing up Costs vs. Benefits of ADHD Pharmacological Treatment', 'Value of Non-pharmacological Treatment' and 'Barriers to Treatment Adherence'. Accessing services and the challenges associated with securing a definitive diagnosis of ADHD in adulthood was an 'uphill struggle', often due to sceptical and negative attitudes towards ADHD by healthcare professionals. ADHD-related impairment had an overwhelmingly chaotic impact on every aspect of patients' lives and many felt ill equipped to cope. A persistent sense of failure and missed potential from living with the impact of ADHD impairment had led to an accumulated psychosocial burden, especially among those diagnosed from late adolescence onwards. In contrast, positive adjustment was facilitated by a younger age at diagnosis. Although medication was perceived as necessary in alleviating impairment, many felt strongly that by itself, it was inadequate. Additional support in the form of psychological therapies or psycho-education was strongly desired. However, few patients had access to non-pharmacological treatment. In some, medication use was often inadequately monitored with little or no follow-up by healthcare professionals, leading to poor adherence and a sense of abandonment from the healthcare system. CONCLUSION: The findings suggest that the unmet needs of adults with ADHD are substantial and that there is a wide gap between policy and current practice in England.

Dopamine agonist monotherapy utilization in patients with Parkinson's disease.

Objectives: To characterize patients with Parkinson's disease (PD) who initiated dopamine agonist (DA) monotherapy, describe medication utilization and provider types, and estimate medication adherence and discontinuation rates. Methods: Retrospective study identified patients with PD in the Optum Research Database and included those with ≥1 claim for DA or levodopa between 09/01/2012 and 12/31/2018, ≥2 PD diagnoses, commercial or Medicare Advantage Part D (MAPD) insurance, ≥40 years old, and continuous medical and pharmacy coverage ≥12 months before and after index date. A subset of patients receiving DA monotherapy was selected for this analysis. Variables were analyzed descriptively. Adherence was measured with medication possession ratio (MPR) and proportion of days covered (PDC); defined as ≥0.80. Results: Patients (N = 642) had mean (SD) age of 70.2 (9.9) years, 70.6 % had MAPD coverage, and 61.7 % were male. Neurologists prescribed 64.6 % of DA monotherapy, and 56.9 % of patients had ≥2 PD diagnoses before or on the index date. Index therapy was discontinued by 44.1 % of patients, and 55.9 % persisted for 12 months without change. Mean (SD) time to discontinuation was 102 (79) days. Mean (SD) MPR for patients (n = 562) with ≥2 fills was 0.84 (0.2); 70.3 % were MPR adherent. Mean (SD) PDC for all 642 patients was 0.66 (0.3); 50.5 % were PDC adherent. Conclusion: Adherence and continuation of therapy were suboptimal, which could translate into poor patient outcomes. Future studies could provide insights on the impact of low adherence and persistence with DA monotherapy.

Work Loss and Direct and Indirect Costs Associated with Parkinson's Disease.

Purpose: To examine work loss and indirect costs during the three-year periods prior to and following initial diagnosis of Parkinson's disease (PD) in patients and in spouses of PD patients, as well as direct costs of healthcare. Patients and Methods: This is a retrospective, observational cohort study using the MarketScan Commercial and Health and Productivity Management databases. Results: A total of 286 employed PD patients and 153 employed spouses met all diagnostic and enrollment criteria for short-term disability (STD) analysis (PD Patient cohort and Caregiving Spouse cohort). The proportion of PD patients having a STD claim increased from roughly 5% and plateaued at around 12-14% starting in the year prior to first diagnosis of PD. The mean number of days lost from work due to STD per year increased from 1.4 days in the 3rd year prior to diagnosis to 8.6 days in the 3rd year after diagnosis (corresponding to an increase in indirect costs from $174 to $1104). STD use for spouses of patients with PD was lowest in the year after their spouses were diagnosed and then rose dramatically in the 2nd and 3rd years after the spouse's diagnosis. Total all-cause direct health-care costs increased during the years leading up to PD diagnosis and were highest in the years following diagnosis, with PD-related costs contributing ~20-30% of the total. Conclusion: PD has both a significant direct and indirect financial burden on patients and their spouses when analyzed for 3 years before and after diagnosis.

Treatment Patterns and Healthcare Resource Use in Medicare Beneficiaries with Parkinson's Disease.

Background: Studies on real-world treatment patterns and long-term economic burden of Parkinson's disease (PD) have been limited. Objective: To assess treatment patterns, healthcare resource utilization (HRU), and costs associated with PD symptoms and treatment-related adverse events (AEs) among Medicare beneficiaries in the United States. Methods: A 100% Medicare Fee-For-Service data (2006-2020) of patients with PD were analyzed. PD treatment patterns were described for the subset of patients who had no previously observed PD treatments or diagnoses (ie, the incident cohort). HRU and healthcare costs associated with PD symptoms were assessed for all patients with PD (ie, the overall cohort) and that associated with treatment-related AEs were assessed for the subset of patients who received PD treatments after PD diagnosis (ie, the active treatment cohort), using longitudinal models with repeated measures. Results: Overall, 318,582 patients were included (mean age at PD diagnosis: 77.4 years; 53.3% female). Among patients in the incident cohort (N=214,829), 51.1% initiated levodopa monotherapy and 5.9% initiated dopamine agonists (DAs) monotherapy as first-line treatment. The proportion of incident patients treated with DAs and other PD therapies generally increased from post-diagnosis years 1 to 10. The median time from diagnosis to PD treatment initiation was 2.0 months; the median time to treatment discontinuation was the longest with levodopa (18.7 months), followed by DAs (9.5 months). In the overall cohort, PD symptoms, especially motor symptoms and severe motor symptoms, were associated with significantly higher rates of HRU and costs. In the active treatment cohort (N=234,298), treatment-related AEs were associated with significantly higher rates of HRU and medical costs. Conclusion: While levodopa is still the mainstay of PD management, considerable heterogeneity exists in real-world treatment patterns. Overall, PD symptoms and AEs were associated with significantly higher HRU and healthcare costs, suggesting unmet medical needs for PD treatments with better tolerability profiles.

Levodopa treatment patterns in Parkinson's disease: A retrospective chart review.

BACKGROUND: Medication regimens for Parkinson's disease (PD) may change as the disease progresses, symptoms fluctuate, or medication-related adverse events occur. This study evaluated treatment trends by observation year for patients initially receiving monotherapy with levodopa and a peripheral dopa decarboxylase inhibitor (PDDI). METHODS: In this retrospective chart review, therapy changes were evaluated for patients across the US diagnosed with PD on or before 6/30/2014 who initially received levodopa-PDDI monotherapy. Index date was the first clinic visit. Post-index was any time between the first 31 days after index and study end (6/30/2019). Index Hoehn-Yahr (H-Y) score and medication changes were also analyzed by index low (<400 mg/day) or high (≥400 mg/day) levodopa doses in the levodopa-PDDI combinations. RESULTS: In the levodopa-PDDI cohort (n = 95), there were 0.39 dose escalations, 0.16 dose reductions, 0.12 discontinuations, 0.19 therapy switches, and 0.24 add-ons per patient per year during the study. Most dose escalations or add-ons occurred within the first 6 months post-index. Of those who ever stopped levodopa-PDDI (n = 34), 31 (91%) restarted within the study period. Most (83%) patients who restarted levodopa-PDDI did so in the same year as stopping treatment. Index low dose users were associated with lower H-Y scores, were more inclined to escalate their dose, and were less inclined to reduce their dose in the first 2 years of treatment than index high dose users. CONCLUSIONS: Prescribers and patients tend to experiment with levodopa-PDDI treatment. Although many patients appeared to stop levodopa-PDDI after an initial course of treatment, most subsequently restarted treatment.

Exploring access to care from the perspective of patients with breast cancer: A qualitative study.

OBJECTIVES: Patients face a myriad of personal and system-based challenges in accessing breast cancer care, but less is known about access as expressed and experienced by patients themselves. The objective of this qualitative study was to further explore the breadth of issues related to access from the perspective of patients with breast cancer across their care journey. METHODS: Twelve women participated in 1-h semi-structured interviews and 48 women participated in 2-h focus groups at six oncology practices in 2018. Grounded theory was used to analyze the data. RESULTS: Six primary themes emerged concerning access to care: information, psychosocial support, health insurance, financial resources, timeliness, and emotions. CONCLUSIONS: This study identified six core dimensions of access to care. Access encompassed not only gaining entrée to care services-in the traditional sense of access-but also the continuing support needed to effectively use those services throughout the cancer care journey. Future strategies aimed at improving access to breast cancer care should attend to these ongoing patient-centric and system-based issues which are mostly amenable to change.

Natural history and patterns of treatment change in Parkinson's disease: A retrospective chart review.

BACKGROUND: Parkinson's disease (PD) management seeks to balance the benefits and harms of current medications and evolves as the disease progresses. The natural history of PD and associated patterns of treatment change were analyzed to identify unmet needs in treatment of PD symptoms. METHODS: Medical charts of patients from clinics across the US diagnosed on or before June 30th, 2014 were retrospectively reviewed. Index date was the first clinic visit, and the post-index period was through study end (June 30th, 2019). Outcomes included the frequency of therapy changes in the post-index period, reasons for therapy change, and adverse events (AE). RESULTS: Patients (n = 203) at index were receiving levodopa-peripheral dopa decarboxylase inhibitor (PDDI) monotherapy (47%), dopaminergic agonist (DA) monotherapy (15%), monoamine oxidase B inhibitor (MAOBI) monotherapy (14%), or combination therapies. The percentage of patients in Hoehn-Yahr disease Stage 1-2 was 52% at index and 20% by the end of the study. Frequencies of motor, non-motor, and neuropsychiatric symptoms increased during the enrollment. Levodopa-PDDI monotherapy and levodopa-PDDI + MAOBI had the lowest rates of therapy changes. Symptom relapse was the most common reason for dose escalation, add-on, and dose reduction, whereas AEs were the most common reason for discontinuation and switching. Dose escalation, add-on, and forward switch were most likely to occur in the first 6 months of treatment. CONCLUSIONS: Therapy changes during the study period reflected the challenging and evolving management of PD as the disease progresses. New or add-on symptomatic treatments are needed that are well-tolerated and able to control PD symptoms.

Methylphenidate delivery mechanisms for the treatment of children with attention deficit hyperactivity disorder: heterogeneity in parent preferences.

OBJECTIVES: Extended-release therapies avoid the need for children with attention-deficit/hyperactivity disorder (ADHD) to take medication at school. Recently a transdermal delivery system has been developed which can allow symptom control all day long but with greater dosing flexibility. This study explored the parents' preferences regarding oral and transdermal therapy. METHODS: A nonsystematic and qualitative literature review and in-depth interviews with parents and physicians helped identify salient treatment attributes for a discrete choice experiment. Treatment attributes included mode of administration (tablet or transdermal), speed of onset (30-90 min); duration (lasts until 3-9 pm) and ability to tailor the drug to different needs (no flexibility, limited flexibility, easy to adjust to different days). A convenience sample of parents of children treated for ADHD (n = 200) were recruited using a recruitment agency. Data were analyzed using generalized estimating equations (GEE). RESULTS: Parents' preferred once-a-day oral therapy (odds ratio [OR] = 1.76 [95 percent confidence interval {CI}, 1.43 - 2.18]); rapid speed of onset (OR = 1.22 [95 percent CI, 1.07 - 1.39]), and symptom control until 9 pm (OR = 3.79 [95 percent CI, 2.98 - 4.82]). Analyses identified that 30 percent of parents preferred transdermal treatment and this subgroup preferred treatments with a fast onset of action. CONCLUSIONS: This survey demonstrates that parents of ADHD children have different preferences for the ADHD treatments prescribed for their children. A distinct subgroup of parents prefer the transdermal therapy. These parents were less likely to be working and so monitoring compliance and doing after school activities may have been easier.

An assessment of Parkinson's disease medication treatment patterns in the Medicaid population.

INTRODUCTION: Most Parkinson's disease (PD) medication adherence studies have focused on patients with commercial or Medicare health insurance coverage. However, less is known regarding medication treatment patterns within the Medicaid population. METHODS: This retrospective cohort study utilized 2011-2019 administrative healthcare claims from 7 state Medicaid programs. We compared newly diagnosed patients with PD started on either levodopa or a dopamine agonist (DA). Baseline comorbidities were compared. Outcomes were assessed during a 12-month post-index observation period, and included total medication days, proportion of days covered (PDC), adherence status, persistence to initiating PD medication, and time to non-persistence of initiating PD medication. RESULTS: Our study sample of 805 Medicaid patients had an average age of 54.1 years, with 52.0% being female. Levodopa was the predominant PD medication at initiation (75.4%). Roughly half of patients had a baseline depressive disorder and nearly 40% had an anxiety disorder. Levodopa patients had a significantly higher PDC compared to DA patients (0.621 vs. 0.546, p = 0.007). An adjusted logistic regression model showed no significant difference in the number of adherent patients between the two groups (p = 0.058). An adjusted Cox proportional hazards model controlling for demographic and baseline variables showed a 26% lower risk of non-persistence for levodopa patients versus DA patients (HR 0.740, CI 0.597-0.917, p = 0.006). CONCLUSIONS: Adherence and persistence rates were suboptimal following initiation of either levodopa or DA medication for patients with PD in Medicaid programs, though rates were better for those initiated on levodopa.

Evaluation of access to care issues in patients with breast cancer.

AIMS: System-level efforts have been deployed to improve oncology care and access while reducing utilization and costs. Understanding the nature of access to care from the perspective of patients themselves is an unmet need. This study examined access to care in a population of women with breast cancer and its relationship to overall patient satisfaction. MATERIALS AND METHODS: Patients with breast cancer from six oncology clinics in five states completed a survey during routine office visits. Access to care (higher scores indicated increasing access barriers), overall patient satisfaction, and patient demographic/clinical characteristics were measured. The relationships between access (composite and factor scores) and satisfaction were assessed using multivariable analyses controlling for age (the only significant characteristic from bivariate analyses). RESULTS: A total of 180 patients completed the survey. Factor analysis of access to care items revealed an 8-factor measure - Insurance, Health System, Emotional, Holistic Treatment, Family Support, Knowledge/Understanding, Information Quality, and Financial Support - with high reliability (Composite: Cronbach alpha = 0.93; Factors: Cronbach alpha range = 0.85-0.91). Access composite score was moderately low (mean = 1.90), indicating an overall low level of access barriers, and overall patient satisfaction was high (mean = 4.59). The composite score (p < .001) and the Health System and Knowledge/Understanding factors (p < .01) were significant and negative predictors of overall satisfaction. LIMITATIONS: Study sites were high functioning clinics and all, but one, are Oncology Care Model practices. Thus, the scope of access to care issues for patients of under-resourced clinics might not be well addressed. CONCLUSIONS: Access to care overall and by factor was significantly predictive of patient satisfaction with care. In addition, access to care factors varied across several demographic and clinical characteristics. Future strategies that address access to care challenges should consider these modifiable, patient-centric, and system-based issues.