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, costs and their driving attributes.
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:
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.