DRIVERS OF ORPHAN MEDICINAL PRODUCT VALUE, PRICE AND COSTS – A PRACTICAL EXPLORATORY ANALYSIS

Author(s)

Vermeersch S1, Patris J2, Bruneo H2
1hict, Brugge, VWV, Belgium, 2Alnylam Pharmaceuticals, Zug, Switzerland

OBJECTIVES: There is increasing debate over price and value of innovative medicines, in particular for orphan medicinal products (OMP). We conducted a descriptive exploratory analysis of OMP prices and costs and their driving attributes. METHODS: We selected 15 high-cost (>100k€ annual treatment cost per patient), ultrarare (<15/100,000) OMPs, excluding oncology products, reimbursed in France, Italy, UK, Germany or Spain in the last 10 years. We investigated (i) ex-factory price (price) and annual treatment cost (cost) elasticity; (ii) the relationship between price, cost and estimated total budget impact (budget); and (iii) impact of disease (prevalence) and product (first in class, existence of a comparator, pharmaceutical form, incremental QALY) attributes on price, cost and budget. RESULTS: Our analyses indicate (i) a threshold effect in terms of price for price elasticity: higher priced products vary less in price across countries; (ii) a threshold effect in terms of cost and prevalence: willingness to pay decreases as patient numbers increase – the rate of decrease differs for different levels of budget; (iii) rewards for innovation with some product attributes (first in class, existence of a comparator, pharmaceutical form) leading to higher price and/or cost and a suggested relationship between price, cost (but not budget) and QALY; (iv) a limit to the acceptability of price and cost premiums, with higher prices and costs acceptable if cost versus budget does not exceed that of other OMPs. CONCLUSIONS: Our preliminary findings suggest OMP value drivers that are implicitly and consistently taken into consideration by authorities and companies, with financial considerations acting as limiting factor to price and cost premiums. Further research should focus on extending the dataset for comparable OMPs as per our methodology, as the heterogeneity of OMPs as well as time-related factors explain the lack of results in previous studies.

Conference/Value in Health Info

2018-11, ISPOR Europe 2018, Barcelona, Spain

Value in Health, Vol. 21, S3 (October 2018)

Code

PSY164

Topic

Health Policy & Regulatory

Topic Subcategory

Reimbursement & Access Policy

Disease

Rare and Orphan Diseases

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