ArticleOrphanet journal of rare diseases2020
Impact of the therapeutic positioning report in the P&R process in Spain: analysis of orphan drugs approved by the European Commission and reimbursed in Spain from 2003 to 2019.
Article in Orphanet journal of rare diseases, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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Who cites it
3 citing papers in PubMed.
- Pricing of innovative medicines and advanced therapies worldwide: an scoping review.The European journal of health economics : HEPAC : health economics in prevention and care · 2026Article
- Challenges and reforms in Spain's health technology assessment system: analysis of criteria influencing medicines' reimbursement decisions between 2019 and 2022 in Spain.The European journal of health economics : HEPAC : health economics in prevention and care · 2025Article
- A comparative analysis of patient participation in health technology assessment systems worldwide: trends and practices.Frontiers in public health · 2025Article
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Authors and funding
6 authors.
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No grant is acknowledged in the PubMed record.
Abstract
backgroundPricing and reimbursement decisions for orphan drugs are faced with differences access between European countries depending on each reimbursement policies, evaluation processes and timings. In 2013, the therapeutic positioning report was introduced in the pricing and reimbursement process in Spain. The present study aims to identify orphan drugs authorised in Spain and approved by the European Commission between January 2003 and December 2019, analyse the impact of the therapeutic positioning report in the pricing and reimbursement process of orphan drugs in Spain and to assess additional potential criteria that could influence pricing and reimbursement decisions for orphan drugs.
resultsNinety-four orphan drugs have been approved by the European Commission between January 2003 and December 2019 and have marketing authorisation in Spain. Out of the 94 orphan drugs, 46 (48.9%) had received pricing and reimbursement approval. Before the inclusion of the therapeutic positioning report in year 2013, the mean time from European Commission approval to pricing and reimbursement approval for orphan drugs in Spain was 25.1 ± 16.5. After 2013, timelines have been reduced by an average of 9 months. The mean regulatory time from European Commission approval to Spanish marketing authorisation has decreased nearly 4 months (from 7.5 ± 10.2 months in years 2003-2013 to 3.8 ± 7.6 months in years 2014-2019). The instauration of the therapeutic positioning report could be associated with a reduction of the mean time from the Spanish marketing authorisation to pricing and reimbursement approval by an average of 5 months (from 17.3 ± 13.1 months in years 2003-2013 to 12.3 ± 5 months in years 2014-2019). In addition, orphan drugs with a positive conclusion in the therapeutic positioning report would be more likely to be reimbursed in Spain (p < 0,0001).
conclusionsThis study shows that the therapeutic positioning report plays a key role in the pricing and reimbursement process in Spain. A positive conclusion of the therapeutic positioning report seems to favourably affect pricing and reimbursement decisions in Spain and, since its introduction, has also contributed to reduce pricing and reimbursement approval timelines in Spain.
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