PMU103 GLOBAL TRENDS IN ALTERNATIVE ACCESS ARRANGEMENTS FOR HIGH-COST ONCOLOGY AND RARE DISEASE DRUGS.

Abstract

Last five years have seen the plethora of Alternative Access Arrangements (AAA) implemented globally by various payers, especially for high-cost Oncology and Rare Disease drugs. We evaluated the trends in such practices based on three different categories of US list prices. We used a non-systematic review of public data sources to identify various AAAs for Oncology and Rare Disease drugs categorized by the US list prices of ‘less-than-$100,000’, ‘between-$100,000-and -$250,000’ and ‘above-$250,000’. Two broad categories – Financial-Based Agreements (FBA) and Outcomes-Based Agreements (OBA), constituted over 140 AAAs. Two-thirds of the total agreements are FBAs, as discounts represent the quickest access solution to entry in most markets, but also skewed heavily by UK’s focus on cost-effectiveness. However, concepts like ‘free drug’ and ‘tiered pricing’ are being tried sporadically in markets with limited experience with such approaches. The total number of OBAs is driven by the UK’s reliance on post-market evidence development, particularly through the Cancer-Drug-Fund. Performance-based agreements are emerging in the US and Germany as opportunities to share risk while facilitating patient access. Italy continues to lead the way in OBAs due to a robust registry system, but details are not public. Truly innovative AAAs are more prevalent for drugs ’above-$250,000’, but payers still appear to be relying heavily on traditional approaches in such high-cost category. Other countries outside of US and EU are slowly experimenting with such novel AAAs as health system start maturing in many such countries (China, Brazil). Although biopharmaceutical manufacturers tend to price drugs based on the intrinsic value of the product, it appears that payer uncertainties around value of such products constitute a gap that necessitated the number and types of AAAs implemented. Robust discussion with payers to manage such uncertainties via optimized clinical trial program can alleviate such gaps to facilitate patient access.