Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study
Background: Despite calls for the use of real-world data, the rare diseases (RD) treatment landscape suffers from a scarcity of data referred to orphan medicinal products (OMP) use at the population level.Objectives: We aim to describe the characteristics and patterns of utilization of OMP in a siza...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2022-09-01
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| Series: | Frontiers in Pharmacology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fphar.2022.940010/full |
| _version_ | 1811253721735102464 |
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| author | Monica Mazzucato Cinzia Minichiello Andrea Vianello Laura Visonà dalla Pozza Ema Toto Paola Facchin Paola Facchin |
| author_facet | Monica Mazzucato Cinzia Minichiello Andrea Vianello Laura Visonà dalla Pozza Ema Toto Paola Facchin Paola Facchin |
| author_sort | Monica Mazzucato |
| collection | DOAJ |
| description | Background: Despite calls for the use of real-world data, the rare diseases (RD) treatment landscape suffers from a scarcity of data referred to orphan medicinal products (OMP) use at the population level.Objectives: We aim to describe the characteristics and patterns of utilization of OMP in a sizable group of RD patients globally monitored by an area-based rare diseases registry located in the Veneto region, Italy, during a 3-year period (1 January 2019 to 31 December 2021).Methods: A list of OMP (n = 60) was assembled for study purposes, according to extensive criteria with regard to the status of orphan designation and of national reimbursement decisions.Results: OMP prescriptions involved 1,010 patients, corresponding to the 2.3% of all the patients monitored by the RD registry. Nearly one out of five (22.8%) was a pediatric patient at the time of the first prescription. OMP use interested a limited proportion (17.5%) of diseases approaching the rarity threshold, having a prevalence of less than five per 10,000, while individuals affected by these more common rare conditions represented 49% of all the patients receiving an OMP prescription. A clustering effect of OMP use was observed in selected groups of diseases, mainly, neurological, hematological, and hereditary metabolic ones. Medication plans including an OMP show in the 45.9% of the cases a high level of complexity, both in terms of nature and number of co-prescribed treatments. Off-label use interested 15.3% (n = 155) of all the RD patients with at least an OMP prescription during the study period.Conclusion: Data collected in a real-world setting through population-based registries globally monitoring RD patients, including related medication plans, have the potential to identify which diseases, and thus patients, have less benefit from the advent of OMP so far. Furthermore, in the rapidly evolving RD therapeutic landscape, they can help understand which therapeutic areas are most in need of investment to address existing unmet care needs. |
| first_indexed | 2024-04-12T16:55:22Z |
| format | Article |
| id | doaj.art-1fd28792b8eb4cf2a2841f8f328b9091 |
| institution | Directory Open Access Journal |
| issn | 1663-9812 |
| language | English |
| last_indexed | 2024-04-12T16:55:22Z |
| publishDate | 2022-09-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj.art-1fd28792b8eb4cf2a2841f8f328b90912022-12-22T03:24:14ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122022-09-011310.3389/fphar.2022.940010940010Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry studyMonica Mazzucato0Cinzia Minichiello1Andrea Vianello2Laura Visonà dalla Pozza3Ema Toto4Paola Facchin5Paola Facchin6Veneto region Rare Diseases Coordinating Centre-Registry, Padua University Hospital, Padua, ItalyVeneto region Rare Diseases Coordinating Centre-Registry, Padua University Hospital, Padua, ItalyEpidemiology and Community Medicine Unit, Padua University Hospital, Padua, ItalyVeneto region Rare Diseases Coordinating Centre-Registry, Padua University Hospital, Padua, ItalyVeneto region Rare Diseases Coordinating Centre-Registry, Padua University Hospital, Padua, ItalyVeneto region Rare Diseases Coordinating Centre-Registry, Padua University Hospital, Padua, ItalyEpidemiology and Community Medicine Unit, Padua University Hospital, Padua, ItalyBackground: Despite calls for the use of real-world data, the rare diseases (RD) treatment landscape suffers from a scarcity of data referred to orphan medicinal products (OMP) use at the population level.Objectives: We aim to describe the characteristics and patterns of utilization of OMP in a sizable group of RD patients globally monitored by an area-based rare diseases registry located in the Veneto region, Italy, during a 3-year period (1 January 2019 to 31 December 2021).Methods: A list of OMP (n = 60) was assembled for study purposes, according to extensive criteria with regard to the status of orphan designation and of national reimbursement decisions.Results: OMP prescriptions involved 1,010 patients, corresponding to the 2.3% of all the patients monitored by the RD registry. Nearly one out of five (22.8%) was a pediatric patient at the time of the first prescription. OMP use interested a limited proportion (17.5%) of diseases approaching the rarity threshold, having a prevalence of less than five per 10,000, while individuals affected by these more common rare conditions represented 49% of all the patients receiving an OMP prescription. A clustering effect of OMP use was observed in selected groups of diseases, mainly, neurological, hematological, and hereditary metabolic ones. Medication plans including an OMP show in the 45.9% of the cases a high level of complexity, both in terms of nature and number of co-prescribed treatments. Off-label use interested 15.3% (n = 155) of all the RD patients with at least an OMP prescription during the study period.Conclusion: Data collected in a real-world setting through population-based registries globally monitoring RD patients, including related medication plans, have the potential to identify which diseases, and thus patients, have less benefit from the advent of OMP so far. Furthermore, in the rapidly evolving RD therapeutic landscape, they can help understand which therapeutic areas are most in need of investment to address existing unmet care needs.https://www.frontiersin.org/articles/10.3389/fphar.2022.940010/fullorphan drugsrare diseasespopulation-based registryreal-world dataprescriptions |
| spellingShingle | Monica Mazzucato Cinzia Minichiello Andrea Vianello Laura Visonà dalla Pozza Ema Toto Paola Facchin Paola Facchin Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study Frontiers in Pharmacology orphan drugs rare diseases population-based registry real-world data prescriptions |
| title | Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study |
| title_full | Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study |
| title_fullStr | Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study |
| title_full_unstemmed | Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study |
| title_short | Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study |
| title_sort | real world use of orphan medicinal products omps in rare disease rd patients a population based registry study |
| topic | orphan drugs rare diseases population-based registry real-world data prescriptions |
| url | https://www.frontiersin.org/articles/10.3389/fphar.2022.940010/full |
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