Estimating Societal Cost of Illness and Patients' Quality of Life of Duchenne Muscular Dystrophy in Egypt.

OBJECTIVES: Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease that causes substantial economic burden. This study aims to measure the DMD cost from societal perspective and the quality of life (QOL) of the Egyptian patients. METHODS: We conducted interviews with caregivers of patients with DMD. The questionnaire included demographics, healthcare resource use, and nonmedical and indirect costs. Total disease burden was estimated with a bottom-up approach. QOL was measured with a disease-specific tool. Costs and utilities were stratified by the disease stage. RESULTS: Caregivers of 97 patients with DMD were interviewed. The mean annual per-patient cost of $17 485 (SD ± 9240) was estimated resulting in a total burden of $138 217 043 in Egypt. Nonmedical costs made up the largest category representing 54% followed by medical then indirect costs. Informal care made the greatest contribution of nonmedical costs whereas physiotherapy was the largest medical subcategory. Nonmedical costs were highest in stage 3 and lowest at early stages whereas medical costs were almost steady among all stages with differences in individual subcategories. Of all medical costs, 95% were out of pocket. The mean utility score was 0.43 (± 0.31), which decreases with disease progression. CONCLUSION: Our study quantified the huge economic burden of DMD on the society and how it differs in different stages. Almost the whole burden is paid by households resulting in catastrophic expenditures, which leads to reduced compliance and quality of care. QOL is also severely compromised. Our findings can inform future healthcare policies and economic evaluation of new DMD therapies.

Early Cost-Utility Analysis of Ataluren and Eteplirsen in the Treatment of Duchenne Muscular Dystrophy in Egypt.

OBJECTIVES: Ataluren and eteplirsen are orphan drugs that delay progression of Duchenne muscular dystrophy in mutation-specific subgroups. They have yet to be approved in Egypt but are expected to reach the market soon. This study describes 2 cost-utility models comparing the drugs with the standard of care. METHODS: We used a partition-survival model with 5 states based on the ambulatory status to model a cohort of ambulatory patients at the age of 5 years. Baseline curves were obtained from a published model; then the ambulation loss curve was updated using the Kaplan-Meier curve of the standard of care from a study by McDonald et al. Other curves were updated by calibration to this curve. Costs and utilities were from a local study. Deterministic and probabilistic sensitivity analyses were conducted. Prices were estimated based on other orphan drugs' prices. RESULTS: In the base case, ataluren 1000 mg and eteplirsen 50 mg/mL resulted in an incremental cost-effectiveness ratio of EGP 51 745 605 and EGP 69 652 533/quality-adjusted life-year, respectively, at their hypothetical prices of EGP 308 600 for ataluren 30-sachet pack and EGP 62 800 for eteplirsen 10 mL vial. The incremental cost-effectiveness ratio was sensitive to health state utilities but not to state costs. At EGP 911 719/quality-adjusted life-year threshold, the value-based prices were EGP 4680 for ataluren 1000 mg and EGP 733 for eteplirsen 10 mL vial. CONCLUSIONS: Based on these models, there is a huge gap between the prices of orphan drugs and their value-based prices, which highlights the need for major policy reforms in the assessment and pricing of orphan drugs.

Descriptive analysis of medication errors reported to the Egyptian national online reporting system during six months.

OBJECTIVES: This study analyzes reports to the Egyptian medication error (ME) reporting system from June to December 2014. METHODS: Fifty hospital pharmacists received training on ME reporting using the national reporting system. All received reports were reviewed and analyzed. The pieces of data analyzed were patient age, gender, clinical setting, stage, type, medication(s), outcome, cause(s), and recommendation(s). RESULTS: Over the course of 6 months, 12,000 valid reports were gathered and included in this analysis. The majority (66%) came from inpatient settings, while 23% came from intensive care units, and 11% came from outpatient departments. Prescribing errors were the most common type of MEs (54%), followed by monitoring (25%) and administration errors (16%). The most frequent error was incorrect dose (20%) followed by drug interactions, incorrect drug, and incorrect frequency. Most reports were potential (25%), prevented (11%), or harmless (51%) errors; only 13% of reported errors lead to patient harm. The top three medication classes involved in reported MEs were antibiotics, drugs acting on the central nervous system, and drugs acting on the cardiovascular system. Causes of MEs were mostly lack of knowledge, environmental factors, lack of drug information sources, and incomplete prescribing. Recommendations for addressing MEs were mainly staff training, local ME reporting, and improving work environment. DISCUSSION: There are common problems among different healthcare systems, so that sharing experiences on the national level is essential to enable learning from MEs. Internationally, there is a great need for standardizing ME terminology, to facilitate knowledge transfer. Underreporting, inaccurate reporting, and a lack of reporter diversity are some limitations of this study. Egypt now has a national database of MEs that allows researchers and decision makers to assess the problem, identify its root causes, and develop preventive strategies.