OLD DRUGS TURNED NEW FOR ULTRA ORPHAN DISEASE- A CHALLENGE FOR PAYERS OR FOR THE INDUSTRY?
Author(s)
Van Tendeloo M, Clerinx C
AX'S Consulting, Chaumont-Gistoux, Belgium
Presentation Documents
OBJECTIVES: During the last 15 years, EMA approved ultra orphan drugs (UOD) that can be defined as “repurposed”: products containing active ingredients that are either approved in a common indication, available as a suboptimal formulation, or requiring pharmacy compounding. This study identifies those products and describes the current market access status in 4 major European countries. The aim is to understand their HTA / access position and identify common patterns and derive success factors. METHODS: : The study screened all EMA approved drugs between 2002 and 2017 meeting the criteria of “repurposed” UODs. For each selected compound a systematic listing was made including approvals, OD designations, scientific evidence and publicly available product information. Market access information was obtained through HTA agency websites, local/hospital formularies and publications/press releases. Interviews with market access stakeholders were conducted to fill the gaps. RESULTS: Six products were retained. France and Germany did better in providing patient access compared to UK and Italy. HAS reviewed five compounds with ASMR rates between 2 and 4. In Germany products were either “pre-AMNOG” or without benefit rating (yet). None of them were NICE reviewed and most had restricted formulary access. SMC assessed 4 out of 6 but only one was recommended. Only one product is reimbursed in Italy, two were rejected. Prices of these UODs were 8- to 70-fold the price of the original. Budget impact considerations played a role whether or not to grant patient access in certain countries for certain products, but not for others. CONCLUSIONS: This study illustrates the variable success of market access of “repurposed” UODs across countries, France and Germany being the most “generous”. Even though the success rates are product- and indication-specific, drivers of success are the level of clinical evidence, availability of compounded or older drugs and budget impact.
Conference/Value in Health Info
2018-11, ISPOR Europe 2018, Barcelona, Spain
Value in Health, Vol. 21, S3 (October 2018)
Code
PHP220
Topic
Health Policy & Regulatory
Topic Subcategory
Reimbursement & Access Policy
Disease
Multiple Diseases