REIMBURSEMENT TRENDS AND EVIDENCE REQUIREMENTS FOR ULTRA-ORPHAN THERAPIES ACROSS EUROPE- OPTIMISING MARKET ACCESS IN INCREASINGLY CHALLENGING MARKETS
Author(s)
Morawski J1, Paul A2, Ransom JF3, Spinner DS4, Doyle JJ5, Faulkner EC6
1Quintiles, Cambridge, MA, USA, 2Quintiles Consulting, Durham, NC, USA, 3Quintiles Global Consulting, Hawthorne, NY, USA, 4Quintiles, Durham, NC, USA, 5Quintiles, Hawthorne, NY, USA, 6Institute for Pharmacogenomics and Individualized Therapy, Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC, USA
OBJECTIVES: Ultra-orphan diseases are extremely rare conditions many of which are severe, chronic, and progressive with high mortality rates. There is a growing number of therapies for ultra-rare diseases currently on the market. Reimbursement decisions for these therapies have been characterized by reduced evidence requirements with unmet need weighing heavily into health technology assessment (HTA) and reimbursement decision-making; as well as a generally wide pricing latitude. To gain insight into evolving market access requirements, we conducted a review of pan-European ultra-orphan therapy HTA requirements and reimbursement decisions. METHODS: Applying the National Institute for Health and Care Excellence (NICE) definition for ultra-orphan diseases (prevalence of ≤ 1/50,000), full European HTA reports on ultra-orphan therapies published through May 2014 were identified and reviewed to compare evidence requirements and reimbursement decisions across countries for health economic, clinical, and value based criteria. RESULTS: Over sixty published ultra-orphan HTAs were identified across nine markets. A small portion of these submissions were rejected for reimbursement largely due to lack of evidence on clinical benefit. For therapies recommended with access restrictions, payers often requested additional follow-on studies or ongoing monitoring of patients by manufacturers. With respect to economic evidence evaluation, reimbursement decisions predominately hinged on therapy cost per patient per year, rather than cost-effectiveness. More recent assessments also evaluated quality of life evidence and input from patient groups. CONCLUSIONS: As health care budgets become more strained, ultra-orphan therapies priced at a premium have come under increased scrutiny from HTA agencies and payers to demonstrate value for money. In order to achieve optimal market access, manufacturers must consider continually evolving stakeholder evidence requirements and develop clinical and health economic value plans that demonstrate how their ultra-orphan therapies provide health gain instead of disease stabilization.
Conference/Value in Health Info
2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands
Value in Health, Vol. 17, No. 7 (November 2014)
Code
PHP161
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Reimbursement & Access Policy
Disease
Multiple Diseases