GSS

Cystadrops® is currently indicated in adults and children from 2 years of age diagnosed with cystinosis with corneal crystal accumulation observed.
However administration of Cystadrops® in patients below 2 years old could be of value for these patients and prevent the crystal deposit. It is the reason why as part of the Cystadrops® pediatric investigational plan (PIP), RECORDATI Rare Diseases committed to conduct a clinical study to assess Cystadrops® safety and efficacy in the pediatric population from 6 months to less than 2 years old.

One of the most commonly reported reasons for people not seeking hearing aids (HAs) intervention is the high cost of HAs and the associated audiological fitting services. Because HAs fitted using the audiologist-based service-delivery model (AUD model) are unaffordable, more and more Americans purchase amplification devices via over-the-counter service-delivery models (OTC models) to compensate for their impaired hearing.
Although OTC amplification devices are gaining popularity and are regarded as an important option for promoting accessible and affordable hearing healthcare, it is unclear if they are viable solutions for age-related hearing loss as OTC models exclude professional services. Further, if there are outcome differences between AUD and OTC models, it is unknown if the clinical improvement in outcomes will be offset by the improved value (outcome relative to cost), or if it is possible to identify appropriate candidacy for each model to ensure optimal patient care for all. Finally, no prior research has investigated if "hybrid" service-delivery models, in which professionals provide streamlined services to fit OTC amplification devices, offer affordable and quality amplification interventions as has been recently advocated.
The overall goal of this project is to characterize the differential effect of service-delivery models on provision of amplification so that accessible and affordable hearing healthcare can be facilitated. This project proposes to conduct research that would provide new knowledge about the outcome, value, and candidacy of OTC, hybrid, and AUD models and the effect of professional evaluation/selection services, patient-centered services, and device-centered services on outcome and value. The proposed study will acquire this knowledge through a two-site, double-blinded, randomized controlled field trial. The results obtained will inform patients and hearing healthcare providers about what can be achieved with different service-delivery models, and will help us develop guidelines to facilitate the selection of the most appropriate and cost-effective intervention for a particular patient. The significance of the proposed project from the public health point of view is that it will facilitate not only accessible and affordable, but also quality, hearing healthcare.

OBJECTIVES: I. Determine the proportion of patients with cystinosis who experience a serious adverse effect when treated with a new formulation of cysteamine hydrochloride for corneal cystine accumulation.
II. Determine the proportion of patients with a reduction in corneal crystal density of 1.00 unit when treated with this regimen.