European payers push back on orphan drug prices

The cost of treating rare diseases in an age when austerity measures are hitting total health care funding across Europe is a highly controversial subject, according to Justpharmareports. It says there is a clear and pressing clinical need, a strong patient voice and the treatments themselves sometimes offer the only hope.

Orphan Drugs in Europe: Pricing, Reimbursement, Funding & Market Access, 2013 Edition, a new publication from Justpharmareports,asks if this is enough to assure premium prices and special status in an era of budget limitations, and growing demands for evidence of clinical and economic value?  It finds that if there is discrimination by payers towards orphan drugs as a group, it is still in a positive way. The products are as diverse as the diseases they treat and some are coming under greater scrutiny.

Four of the first seven orphan drugs to enter Germany’s AMNOG (The Pharmaceuticals Market Reorganisation Act) procedure were considered to bring only minor additional benefit and for two others the benefit was deemed non-quantifiable. Discounts were required by GKV-Spitzenverband for all. Re-evaluation by the Transparency Commission in France has seen both SMR and ASMR scores downgraded with resultant price cuts.