Additional Benefit Scoring During Health Technology Assessment in Germany: Its Drivers and Impact on Pricing and Discount for Orphan Drugs

    September 2018 in “ Value in Health
    Y. Oku, Ayako Fukushima, J Dorey, Mondher Toumi
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    TLDR Orphan drug benefit scores in Germany are influenced by phase III data and lack of alternatives, but not linked to price or discounts.
    The document analyzed the factors affecting the additional benefit scoring of orphan drugs (ODs) during health technology assessment (HTA) in Germany and its impact on pricing and discount rates. The study included 33 ODs with 45 indications and found that phase III study data and the absence of comparable treatments were crucial for a high HTA score. However, no significant correlation was observed between the HTA score and the drug's price or discount rate. Additionally, the document covered other healthcare cost topics, including the variation in OD prices across Europe, the economic impact of MRSA infections in China based on a study of 367 inpatients, international drug price comparisons involving 44 top-selling drugs in Korea, and the economic burden of needle-stick injuries in China, estimated at ¥5.93 billion/year. The document calls for more research on the determinants of OD pricing in Europe and the economic effects of digital health interventions.
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