Living with Cerebrotendinous Xanthomatosis: Patient, Caregiver, and Expert Perspectives.

In this article, patients with cerebrotendinous xanthomatosis (CTX) and caregivers detail their experience with lifelong symptoms, diagnosis, treatment and efficacy, and ongoing disease management. One patient and four caregivers describe the challenges associated with pursuing a correct diagnosis for years before testing confirmed a CTX diagnosis. They also detail their ongoing struggles and desire for greater access to physicians with CTX knowledge and to reliable online resources to continue their education about the disease and strategies for symptom management. The expert perspective is a direct response by three CTX researchers, including physicians who are treating patients with CTX in the United States and experts whose laboratories provide genetic and biochemical testing for CTX. They respond to many of the patient and caregiver concerns, including steps that are being taken to identify CTX earlier and provide access to confirmatory diagnostic testing sooner, and suggest the best online resources for CTX-related information and access to webinars and support groups. While the expert perspective is a direct response to the patient and caregiver authors' CTX journeys, it should be beneficial to any patient with CTX or their caregivers.

Concerns Around Budget Impact Thresholds: Not All Drugs Are the Same.

BACKGROUND: The use of budget thresholds is a recent development in the United States (e.g., the Institute for Clinical and Economic Review drug assessments). Budget thresholds establish limits that require some type of budgetary action if exceeded. This research focused on the advisability of using product-level budget thresholds as fixed spending caps by examining whether they are likely to improve or worsen market efficiency over status quo. OBJECTIVE: The aim of this study was to determine whether fixed product-level spending caps are advisable for biopharmaceuticals. METHODS: We systematically examined 5-year, postlaunch revenue for drugs that launched in the United States between 2003 and 2014 using the IMS MIDAS database. For products launched between 2011 and 2014, we used historical revenue as the baseline and trended out 60 months postlaunch based on exponential smoothing. Forecasted fifth-year revenue was compared to analyst reports. Fifth-year revenue was compared against a hypothetical $904 million spending cap to determine the amount of annual spending that might require reallocation. Descriptive statistics of 5-year, postlaunch revenue and annual spending requiring reallocation were calculated. RESULTS: Adhering to a $904 million product-level spending cap requires that approximately one-third of new drug spending be reallocated to other goods and services that have the potential to be less cost-effective due to significant barriers. CONCLUSION: Fixed product-level spending caps have the potential to reduce market efficiency due to their independence from value and the presence of important operational challenges.