ArticleOrphanet journal of rare diseases2019
An analysis of orphan medicine expenditure in Europe: is it sustainable?
Article in Orphanet journal of rare diseases, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 1 of them a synthesis that pooled it.
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Who cites it
22 citing papers in PubMed, 1 synthesis or guideline pooled it, 36 citations in OpenAlex.
- How Much Does It Cost to Research and Develop a New Drug? A Systematic Review and Assessment.PharmacoEconomics · 2021Pooled it
- Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study.Orphanet journal of rare diseases · 2026Article
- Global access to management of primary hyperoxaluria: a survey on behalf of OxalEurope, G&K Working Group of the ERA and ESPN.Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association · 2025Article
- Advances in Gene Therapy for Rare Diseases: Targeting Functional Haploinsufficiency Through AAV and mRNA Approaches.International journal of molecular sciences · 2025Review
- Orphan medical devices: addressing the regulatory and access gaps in the EU and US.Frontiers in public health · 2025Article
- Budget impact analysis of high-priced orphan medicinal products intended for the treatment of rare diseases in China: evidence from a densely populated metropolis of Chengdu.BMC health services research · 2024Article
- Rare diseases: still on the fringes of universal health coverage in Europe.The Lancet regional health. Europe · 2024Review
- Costs of orphan medicinal products: longitudinal analysis of expenditure in Wales.Orphanet journal of rare diseases · 2023Article
- Clinical applications of gene therapy for rare diseases: A review.International journal of experimental pathology · 2023Review
- How to Predict Drug Expenditure: A Markov Model Approach with Risk Classes.PharmacoEconomics · 2023Article
- Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges.International journal of environmental research and public health · 2023Review
- The challenges of access to innovative medicines with limited evidence in the European Union.Frontiers in pharmacology · 2023Review
- Pharmaceutical policy and innovation for rare diseases: A narrative review.F1000Research · 2023Review
- Exploring alternative financing models and early access schemes for orphan drugs: a Belgian case study.Orphanet journal of rare diseases · 2022Article
- Comparison of US and EU Prices for Orphan Drugs in the Perspective of the Considered US Orphan Drugs Act Modifications and Discussed Price-Regulation Mechanisms Adjustments in US and European Union.International journal of environmental research and public health · 2022Article
- Making Sure That Orphan Incentives Tip the Right Way in Europe.Healthcare (Basel, Switzerland) · 2022Article
- Availability and Accessibility of Orphan Medicinal Products to Patients in Slovakia in the Years 2010-2019.Frontiers in pharmacology · 2022Article
- Trends in orphan drug use and spending among children and adolescents during 2010-2020 in Korea.Frontiers in pharmacology · 2022Article
- Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group).Frontiers in pharmacology · 2021Article
- Methodological Quality Assessment of Budget Impact Analyses for Orphan Drugs: A Systematic Review.Frontiers in pharmacology · 2021Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundOrphan medicinal product (OMP) prices are considered by some to be a challenge to the sustainability of healthcare expenditure. These concerns are compounded by the increasing number of OMPs receiving marketing authorisation (MA) annually. The aim of this study was to explore the sustainability of OMP expenditure within the context of total European pharmaceutical expenditure.
methodsUsing historical IQVIA data, an analysis was conducted on total pharmaceutical and OMP expenditure in eight countries (using values / volumes) in the branded, non-branded and overall pharmaceutical market. Country level and aggregated data was considered for EU5 countries, Austria, Belgium and Ireland. Three key analyses were conducted: 1.The OMP share of total pharmaceutical expenditure was calculated from 2000 to 2017, to assess its evolution over time.2.The results of this analysis were compared with a 2011 forecast of OMP budget impact.3.The evolution of the total pharmaceutical market and its different segments (branded OMPs, non-OMP branded and unbranded) were assessed by estimating the compound annual growth rate (CAGR) and percentage of pharmaceutical expenditure for each market segment from 2010 to 2017.
resultsAcross countries, OMP share of total pharmaceutical expenditure has increased each year since 2000, rising to 7.2% of total pharmaceutical expenditure in 2017. OMP expenditure has increased at a CAGR of 16% since 2010. The number of OMPs receiving MA each year showed a CAGR of 11% since 2001, four percentage points greater than the CAGR for all medicines receiving MA over the same period. OMP share of total pharmaceutical expenditure is higher than forecasted in 2011 due to slower than expected growth in the non-OMP market. OMP growth has been offset by reduced expenditure in the general market and increased use of generics and biosimilars.
conclusionsRelative spending on OMPs has increased over the last 20 years, but this has been largely compensated for within the current allocation of total pharmaceutical spending by flat expenditure for non-OMPs and increased volumes of (lower-priced) generics/biosimilars, reflecting a shift towards expenditure in higher cost, lower volume patient populations and a shift in drug development towards more specialised targeting of diseases.
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Registered trials
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