OFF episode quality of life impact scale (OFFELIA): A new measure of quality of life for off episodes in Parkinson's disease.

INTRODUCTION: OFF Episodes occur in people with Parkinson's disease when their medication wears off, and motor and/or non-motor symptoms emerge. Existing measures used to assess OFF Episodes focus on the time spent in OFF Episodes through diaries or by identifying symptoms, but they are limited in their ability to capture the severity and functional impact of OFF episodes. The aim of this study was to develop and validate a new instrument, called "OFFELIA," that measures the impact of OFF episodes on the quality of life of individuals with Parkinson's disease. METHODS: Participants completed a cross-sectional questionnaire, "Impact and Communication on OFF Periods," while enrolled in the online clinical study Fox Insights. The data collected was used to develop OFFELIA. Psychometric testing was performed on 18 candidate items using classical, exploratory factor analysis, and item response theory methods. RESULTS: 569 individuals with Parkinson's disease completed the questionnaire. All items were retained for the final measure, with 17 items aggregated into two multi-item scales (functioning and psychological well-being) and one item reported separately as it did not function well with the other items (employment). Known group comparisons based on average duration, frequency and unpredictability of OFF episodes indicated that OFFELIA subscales were more sensitive than existing generic and condition-specific measures. CONCLUSION: Initial evidence supports the validity of OFFELIA, a new instrument that assesses the impact of OFF periods on daily life. This instrument can be used in assessing clinical therapeutic strategies targeting OFF episodes in Parkinson's disease.

Real-world utilization of top-down and step-up therapy and initial costs in Crohn disease.

BACKGROUND: Medication treatment strategies for Crohn disease (CD) include step-up (SU) therapy, beginning with oral anti-inflammatory agents, and top-down (TD) therapy, beginning with biologics or immunomodulators. The real-world utilization and short-term medical costs associated with these treatment strategies are not well described. OBJECTIVE: To examine the prevalence of TD therapy use over time and compare the first-year direct medical expenditures among patients initiating CD medication treatment with SU and TD therapy in a real-world setting. METHODS: We conducted a retrospective cohort study of Optum Clinformatics Data Mart examining adult patients with CD newly initiated on medication therapy from 2010 to 2018. Included patients had a CD-indicated medication dispensed within 60 days after their initial CD diagnosis, were continuously enrolled in the health plan throughout the study period, and did not have comorbidities treated with a biologic also indicated for CD. A generalized linear model was used to quantify the differences in adjusted mean first-year CD-specific, direct nonpharmacy medical costs between users of TD and SU therapy. RESULTS: We identified 3,157 patients newly initiating medication therapy for CD (2,392 [75.8%] patients treated with SU therapy and 765 [24.2%] treated with TD therapy). The use of TD therapy over the study period increased from 17% in 2011 to 31% in 2017. TD therapy was also associated with a 149.8% ($1,230) higher adjusted average per-patient first-year CD-direct nonpharmacy medical cost compared with SU therapy (adjusted ratio of cost for TD compared with SU [2.498, 95% CI = 2.12-2.95]). CONCLUSIONS: In patients newly initiating medication therapy for CD, TD therapy use increased between 2010 and 2017 and was associated with higher first-year nonpharmacy medical expenditure. These findings align with the strategy of initiating TD therapy in patients with a higher disease burden. Further research is needed to determine long-term overall health care costs and clinical outcomes associated with SU and TD strategies in a real-world setting. DISCLOSURES: Dr Caffrey received research funding from Gilead, Merck, Pfizer, and Shionogi and is a speaker for Merck. The views expressed are those of the author and do not necessarily reflect the position or policy of the US Department of Veterans Affairs. Material is based on work supported, in part, by the Office of Research and Development.

Key learnings from Institute for Clinical and Economic Review's real-world evidence reassessment pilot.

Health technology assessment (HTA) agencies are considering adopting a lifecycle approach to assessments to address uncertainties in the evidence base at launch and to revisit the clinical and economic value of therapies in a dynamic clinical landscape. For reassessments of therapies post launch, HTA agencies are looking to real-world evidence (RWE) to enhance the clinical and economic evidence base, though challenges and concerns in using RWE in decision-making exists. Stakeholders are embarking on demonstration projects to address the challenges and concerns and to further define when and how RWE can be used in HTA decision making. The Institute for Clinical and Economic Review piloted a 24-month observational RWE reassessment. Key learnings from this pilot include identifying the benefits and challenges with using RWE in reassessments and considerations on prioritizing and selecting topics relevant for RWE updates.

Behavior-based diabetes management: impact on care, hospitalizations, and costs.

OBJECTIVES: To (1) examine the impact of the Diabetes Care Rewards (DCR) program on adherence to care standards and (2) evaluate the economic impact of adherence to care standards. STUDY DESIGN: A retrospective observational cohort study design with propensity matching. Additional covariates adjustment was used to minimize residual imbalance. METHODS: Utilization and cost data were compared between individuals enrolled vs individuals eligible for but not enrolled in the DCR program using a standard mean difference. Individuals were employees or their dependents from self-insured companies throughout the United States. Outcomes included adherence to the care standards, service utilization, and costs. RESULTS: A total of 3318 propensity-matched participants were included. Primary analysis revealed that enrolled members increased adherence to semiannual glycated hemoglobin, annual lipid, and annual urine albumin-creatinine ratio testing. Additionally, enrolled members experienced less utilization of high-acuity services and increased rates of physician visits. In a secondary analysis, the enrolled group was associated with greater pharmaceutical costs but lower medical costs. CONCLUSIONS: A behavioral science- and incentive-based diabetes management program was associated with greater rates of adherence to recommended diabetes monitoring care standards, increased routine clinic visits, decreased hospital admissions, and decreased inpatient days. Anticipated increases in pharmaceutical expenditures were offset by overall lower medical expenditures. Results indicate the economic benefits of adherence to evidence-based standards for diabetes care.

Challenges in Evaluating Safety and Efficacy in Drug Development for Rare Diseases: A Review for Pharmacists.

A rare disease, or orphan disease, in the United States is a condition with a national prevalence of fewer than 200,000 diagnoses. As therapies for rare diseases are developed and brought to market, pharmacists should understand the challenges of drug development for rare diseases and aid in educating patients about the approval process for rare disease therapies. Developing drugs for treating rare diseases presents unique challenges in proving the drug's safety and efficacy with adequate study design, power, and validity. Results of the clinical trials for rare diseases may be weakened by small patient populations, limited disease information, and difficulty defining end points and biomarkers. In addition to investigational barriers, pharmaceutical companies face financial barriers in justifying the investment of bringing a rare disease therapy to market. Federal programs, such as the Orphan Drug Act of 1983, expedited review, the Rare Pediatric Disease Priority Review Vouchers (RPD PRV) program, and the 21st Century Cures Act, give pharmaceutical companies motivation to develop therapies for rare diseases. The objective of this article is to provide pharmacists with an understanding of the challenges in designing clinical trials for drugs for rare diseases and discuss federal programs that address efforts to develop safe and efficacious drugs for rare diseases.