Impact of Measuring Patient-Reported Outcomes in Dermatology Drug Development.

Although some symptoms of dermatologic diseases, such as pruritus and pain, can be subjectively assessed only by patients, the most commonly used endpoints in dermatology drug research traditionally have been clinician-reported outcomes. Research has found that patient-reported outcomes (PROs) were included in only one-quarter of 125 trials conducted between 1994 and 2001. Our objective was to characterize the impact of PROs in dermatology drug development from the patient, prescriber, regulator, payer, and manufacturer perspectives using a case study approach. We conducted a structured literature review for pivotal clinical trials using PROs for six dermatologic products (MAS063DP, onabotulinumtoxinA, calcipotriene hydrate plus betamethasone dipropionate, pimecrolimus, tacrolimus, and ustekinumab). We also searched regulatory websites to identify product labeling and the UK National Institute for Health and Care Excellence website to identify submissions for the products of interest. A total of 32 articles illustrating the various perspectives were selected for inclusion. Clinical trials that include PROs allow patients to differentiate among treatments based on the experience of other patients participating in trials and enable prescribers to understand the benefit-risk profile of new treatments. The inclusion of PROs enables regulators to evaluate product benefits with a patient-centered perspective; five of the products of interest obtained eight total product labeling statements. PRO data supported manufacturers' dissemination of product benefits in the form of publications and PRO labeling for the product. For payers, PRO data were used in an analysis of cost effectiveness of new treatments. Inclusion of PROs in dermatology drug development programs benefits patients, prescribers, regulators, manufacturers, and payers.

Benefit-risk tradeoff preferences for chronic hand eczema treatments.

Hand eczema affects approximately 16% of the US population. The long-term prognosis is poor, and 5-7% experience severe chronic hand eczema (sCHE) that interferes with daily activities. Treatments for CHE may be ineffective or associated with adverse events (AEs) that may dissuade patients from pursuing or continuing treatment. For quantification of patient experiences and benefit-risk preferences for outcomes associated with CHE treatments, a web-based discrete choice experiment survey was administered to patients in the United States with a self-reported physician diagnosis of CHE and severe symptoms not resolved with topical agents. Respondents answered a series of treatment choice questions, each requiring evaluation of a pair of hypothetical profiles of medications for sCHE defined by efficacy and risk of several AEs. Improvement in CHE clearing of 25-50% was rated from 1.5 to 3.1 times as important as eliminating a 5% risk of permanent bone problems. The mean maximum acceptable risk of permanent vision problems in exchange for an improvement in CHE clearing of 25-50% ranged from 3.4% to 4.8%. This study demonstrated that patients with CHE rated efficacy improvements associated with treatment of sCHE as more important than eliminating the risks of specific AEs.

Assessing United States Patient and Dermatologist Experiences with Severe Chronic Hand Eczema.

OBJECTIVE: Patients with severe chronic hand eczema often have persistent symptoms that interfere with daily activities, social functioning, and employment. Many patients are refractory to topical corticosteroids. This survey-based study was performed to characterize treatment experiences, impact on productivity, and quality of life of patients with severe chronic hand eczema; understand dermatologists' severe chronic hand eczema treatment patterns. DESIGN: A web-based survey in the United States queried pre-identified patients with severe chronic hand eczema regarding symptoms, treatment history, quality of life, work productivity, treatment satisfaction, and healthcare utilization. In a separate survey, dermatologists were asked about treatment patterns and satisfaction with currently available therapies. RESULTS: The most commonly reported symptoms currently experienced by patients (n=163) were dryness/flaking (81%), itchiness (75%), and cracking/tearing of the skin (71%). Over the last three months, 84 percent of patients with severe chronic hand eczema self-reported using topical steroids, and 30 percent used systemic corticosteroids or retinoids. Approximately 30 percent reported impairment while working and productivity loss. Patient quality of life was negatively impacted. Dermatologists (n=125) reported most often treating severe chronic hand eczema with topical corticosteroids (99%), followed by topical immunomodulators (71%) and systemic treatments (70%). Only two percent were very satisfied with currently available products. CONCLUSION: Patients with severe chronic hand eczema experience symptoms that negatively impact work productivity and quality of life. Few dermatologists are very satisfied with currently available severe chronic hand eczema treatment options.

A Cost-Effectiveness Analysis between Amlodipine and Angiotensin II Receptor Blockers in Stroke and Myocardial Infarction Prevention among Hypertension Patients in China.

OBJECTIVE: Uncontrolled hypertension (HTN) results in strokes, myocardial infarction (MI), and other complications, which are the leading cause of disability, death, and severe economic consequence. We conducted an economic evaluation to determine the costs and quality-adjusted life-years (QALYs) associated with amlodipine (Norvasc) and the angiotensin II receptor blockers (ARBs) in preventing stroke and MI among Chinese HTN patients. METHODS: A cost-utility analysis was conducted from the third-party payer perspective. A Markov model was constructed to estimate 5-year costs and health consequences of amlodipine and valsartan. Effectiveness data were based on a published meta-analysis. Utility data were retrieved from the published literature. Costs of MI were retrieved from China Health Statistics Yearbook. Costs of stroke were obtained from retrospective chart review and follow-up interviews in Chinese tertiary hospitals. Costs included costs of drugs, direct medical costs of HTN management, stroke/MI treatment, and follow-up management. Discounting rate used for costs and QALYs was 3%. RESULTS: Total direct medical and drug costs of amlodipine and valsartan (ARB) users were ¥111,731,716 and ¥132,058,611, respectively; total QALYs of amlodipine and valsartan users were 30,648.5 and 30,520.8, respectively. Amlodipine is dominant with lower costs and higher QALYs. This demonstrated that compared with valsartan, amlodipine is a cost-saving therapy with better QALY outcome. When irbesartan data were used in the comparison, the magnitude of cost saving changed but the overall conclusion remained the same. CONCLUSION: Amlodipine is a cost-saving therapy compared with ARBs in preventing stroke and MI for Chinese HTN patients.

Capecitabine treatment patterns in patients with gastroesophageal cancer in the United States.

AIM: To assess the use of capecitabine-based therapy and associated complication rates in patients with gastroesophageal cancer (GEC) in a real-world treatment setting. METHODS: Patients with claims between 2004 and 2005 were identified from the Thomson Reuters MarketScan databases. Capecitabine regimens were compared with 5-fluorouracil (5-FU) and other chemotherapy regimens, and were stratified by treatment setting. RESULTS: We identified 1013 patients with GEC: approximately half had treatment initiated with a 5-FU regimen, whereas 11% had therapy initiated with a capecitabine regimen. The mean capecitabine dose overall was 2382 +/- 1118 mg/d, and capecitabine was used as monotherapy more often than in combination. Overall, 5-FU regimens were the most common treatment option in neoadjuvant and adjuvant settings, while other non-capecitabine regimens were used more widely in first- and second-line settings. The overall unadjusted complication rate for capecitabine regimens was about half of that seen with 5-FU regimens. In multivariate analyses, capecitabine recipients had a 51% (95% CI: 26%-81%) lower risk of developing any complication than 5-FU recipients did. The risk of developing bone marrow, constitutional, gastrointestinal tract, infectious, or skin complications was lower with capecitabine therapy than with 5-FU. CONCLUSION: Capecitabine appeared to have a favorable side effect profile compared with 5-FU, which indicates that it may be a treatment option for GEC.

Costs associated with complications are lower with capecitabine than with 5-fluorouracil in patients with colorectal cancer.

BACKGROUND: Capecitabine, an oral alternative to 5-fluorouracil (5-FU) in patients with colorectal cancer (CRC), has equal clinical efficacy and a favorable safety profile; however, its use may be limited because of unit cost concerns. In this study, the authors measured the cost of chemotherapy-related complications during treatment with capecitabine- and 5-FU-based regimens. METHODS: Patients with CRC who received at least 1 administration of capecitabine or 5-FU during 2004 and 2005 were identified from the Thomson MarketScan research databases. Monthly frequency and cost for 23 complications were recorded. Logistic regression was used to predict complication probability. General linear models were used to predict monthly complication cost and total monthly expenditure. RESULTS: In total, 4973 patients with CRC met the inclusion criteria for this analysis. Although the most frequently observed complications were the same between capecitabine and 5-FU (nausea and vomiting, infection, anemia, neutropenia, diarrhea), each was observed with greater frequency in 5-FU-based regimens. The mean predicted monthly complication cost was significantly higher (by 136%) with 5-FU monotherapy than with capecitabine monotherapy (difference, $601; 95% confidence interval [95% CI], $469-$737). In addition, the mean predicted monthly complication cost for 5-FU+oxaliplatin was higher than the cost with capecitabine plus oxaliplatin (difference, $1165; 95% CI, $892-$1595). When acquisition, administration, and complication costs were taken into consideration, there were no significant differences in the total cost between capecitabine regimens and 5-FU regimens. CONCLUSIONS: Capecitabine compared well with 5-FU-based therapy in patients with CRC and was associated with lower complication rates and associated costs.

The National Public Health Leadership Institute: evaluation of a team-based approach to developing collaborative public health leaders.

Recent public health literature contains calls for collaborative public health interventions and for leaders capable of guiding them. The National Public Health Leadership Institute aims to develop collaborative leaders and to strengthen networks of leaders who share knowledge and jointly address public health problems. Evaluation results show that completing the institute training increases collaborative leadership and builds knowledge-sharing and problem-solving networks. These practices and networks strengthen interorganizational relationships, coalitions, services, programs, and policies. Intensive team-and project-based learning are key to the program's impact.