Factors influencing patients' willingness-to-pay for disease-modifying therapies for multiple sclerosis.

BACKGROUND: High and ever-increasing costs of Multiple Sclerosis (MS) disease-modifying therapies (DMTs) have impaired patient access to DMTs in the US. Patients' willingness-to-pay (WTPs) for DMTs were recently examined, but their influencers were not determined. Thus, the objective of this study was to examine factors influencing patients' WTPs for DMTs for multiple sclerosis (MS). METHODS: Data were obtained from a previous survey of 1,200 US patients with MS on their preferences and WTPs for DMTs. Patients' characteristics (i.e., age, gender, race, marital status, education, employment status, comorbidity, health status, and health insurance) and their MS experiences (i.e., number of years with MS, MS type, number of relapses, fatigue, mood-change, MS symptom, and DMTs experience) were investigated as influencing factors. Patient's WTP for a DMT was obtained from a direct question in the survey. A two-part model was estimated using logistic regression and generalized linear regression. RESULTS: Responses from 480 patients were analyzed. Their average age was 53 years old. Most of them were female (79%), white (97%), and married (71%). Approximately 61% of them had a four-year college degree or lower, 54% were either unemployed, retired, or students, 59% were enrolled in private insurance, 81% had at least one comorbid condition, and 73% considered themselves having good or better health status. Approximately 44% had at least one relapse in two years, 89% experienced fatigue, 37% experienced mood-change, and on average had MS for more than 13 years. The majority of them had relapsing-remitting MS (66%), considered themselves to have some levels of disability for MS (78%), and had used or were currently using DMTs (97%). The average WTP for a DMT was $579 per month. Patients with professional degrees, or with one or more comorbid conditions were more likely willing to pay for a DMT. Patients who were white, had a professional degree, or were in fair or better health status were willing to pay a significantly higher amount for a DMT. Patients, who were female, were employed, did not have private insurance, had a higher number of MS experience years, or who experienced mood change were willing to pay significantly less amount for a DMT. CONCLUSION: Various patients' characteristics and MS experiences, including gender, race, education, employment, health insurance, comorbidity, health status, DMT experience, and mood change influenced patients' WTPs for a DMT.

Cost effectiveness of collagen crosslinking for progressive keratoconus in the UK NHS.

BACKGROUND: Keratoconus is a progressive degenerative corneal disorder of children and young adults that is traditionally managed by refractive error correction, with corneal transplantation reserved for the most severe cases. UVA collagen crosslinking is a novel procedure that aims to prevent disease progression, currently being considered for use in the UK NHS. We assess whether it might be a cost-effective alternative to standard management for patients with progressive keratoconus. METHODS: We constructed a Markov model in which we estimated disease progression from prospective follow-up studies, derived costs derived from the NHS National Tariff, and calculated utilities from linear regression models of visual acuity in the better-seeing eye. We performed deterministic and probabilistic sensitivity analyses to assess the impact of possible variations in the model parameters. RESULTS: Collagen crosslinking is cost effective compared with standard management at an incremental cost of £ 3174 per QALY in the base case. Deterministic sensitivity analysis shows that this could rise above £ 33,263 per QALY if the duration of treatment efficacy is limited to 5 years. Other model parameters are not decision significant. Collagen crosslinking is cost effective in 85% of simulations at a willingness-to-pay threshold of £ 30,000 per QALY. CONCLUSION: UVA collagen crosslinking is very likely to be cost effective, compared with standard management, for the treatment of progressive keratoconus. However, further research to explore its efficacy beyond 5 years is desirable.

Validity of using vision-related quality of life as a treatment end point in intermediate and posterior uveitis.

AIM: To evaluate the responsiveness of the Vision core module 1 (VCM1) vision-related quality of life (VR-QOL) questionnaire to changes in visual acuity in patients with posterior and intermediate uveitis and to validate its use as a clinical end point in uveitis. METHODS: Logarithm of the minimum angle of resolution visual acuity and VR-QOL using the VCM1 questionnaire were prospectively recorded in 37 patients with active posterior segment intraocular inflammation before starting systemic immunosuppression with ciclosporin, tacrolimus or the anti-tumour necrosis factor (TNF) agent, p55TNFr-Ig, and again 3 months later. Spearman analysis was used to correlate improvements in visual acuity and VR-QOL between baseline and 3 months. RESULTS: The correlation between changes in visual acuity and VR-QOL was moderate to good for the worse eye (r = 0.47, p = 0.003), but poor for the better eye (r = -0.05, p = 0.91). The responsiveness indices effect size and standardised response mean were 0.57 and 0.59, respectively, showing that the VCM1 questionnaire is moderately responsive to immunsosuppressive therapy for active uveitis. CONCLUSION: Changes in VR-QOL measured with the VCM1 questionnaire correlated moderately well with changes in the worse eye visual acuity, suggesting that the VCM1 is a valid instrument for monitoring response to treatment in uveitis.

The assessment of lens opacities in clinical practice: results of a national survey.

AIM: To investigate the examination of lens opacities in routine ophthalmic clinical practice. METHOD: A questionnaire survey was mailed to 703 consultant ophthalmologists in the UK. The surgeons were asked which lens feature(s) they assessed in their clinics when deciding whether to offer cataract surgery. RESULTS: 489 replies were received. A broad range of lens opacities was assessed, with differences between surgeons for some opacities with high prevalences in the population, particularly cortical opacities. Many (74% of 467) surgeons assessed one or more lens opacities (anterior subcapsular cataract, vacuoles, water clefts, coronary flakes, focal dots, retrodots, fibre folds) which may be visually important but which have received relatively little attention by researchers. CONCLUSIONS: Some classes of lens opacity which are traditionally measured by researchers may be ignored in clinical practice and opacities which are traditionally ignored by some researchers are regarded as clinically important by a substantial number of surgeons.