Application of four pricing models for orphan medicines: a case study for lumasiran
Abstract Background The combination of high prices and uncertain effectiveness is a growing challenge in the field of orphan medicines, hampering health technology assessments. Hence, new methods for establishing price benchmarks might be necessary to support reimbursement negotiations. In this stud...
Saved in:
| Main Authors: | , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2024-12-01
|
| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-024-03446-w |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1850086553310724096 |
|---|---|
| author | Noa Rosenberg Evert Manders Sibren van den Berg Lisa J. Deesker Sander F. Garrelfs Saco J. de Visser Jaap W. Groothoff Carla E. M. Hollak |
| author_facet | Noa Rosenberg Evert Manders Sibren van den Berg Lisa J. Deesker Sander F. Garrelfs Saco J. de Visser Jaap W. Groothoff Carla E. M. Hollak |
| author_sort | Noa Rosenberg |
| collection | DOAJ |
| description | Abstract Background The combination of high prices and uncertain effectiveness is a growing challenge in the field of orphan medicines, hampering health technology assessments. Hence, new methods for establishing price benchmarks might be necessary to support reimbursement negotiations. In this study, we applied several pricing models containing cost-based elements to the case of lumasiran for treating primary hyperoxaluria type 1. Methods Price ranges were calculated by estimating minimum and maximum scenarios for four pricing models: Novel Cancer Pricing Model (NCP-model), AIM Model for Innovative Medicines (AIM-model), Discounted Cash Flow model (DCF-model), and the Real-Option Rate Of Return model (ROROR-model). Data was gathered from disease registries, scientific literature, Security and Exchange Committee filings, and expert opinion. A sensitivity analysis was performed to assess the parameters with the largest influence. Results Outcomes resulting from the NCP-model ranged between €87,000 and €224,000 per patient per year, between €33,000 and €340,000 for the AIM-model, between €182,000 and €748,000 for the DCF-model, and between €81,000 and €273,000 for the ROROR-model. Conclusion Outcomes of the four pricing models show wide and heterogeneous price ranges. The DCF-model might be most compatible with the case of lumasiran, due to inclusion of parameters for prevalence, incidence, prescription restrictions and cost of capital. The minimum DCF price could serve as a starting point for pricing and reimbursement negotiations. Uncertainties can be solved by more transparency on input variables. |
| format | Article |
| id | doaj-art-3a4d2baa0e59486991f920b2d6832c4f |
| institution | DOAJ |
| issn | 1750-1172 |
| language | English |
| publishDate | 2024-12-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj-art-3a4d2baa0e59486991f920b2d6832c4f2025-08-20T02:43:27ZengBMCOrphanet Journal of Rare Diseases1750-11722024-12-0119111410.1186/s13023-024-03446-wApplication of four pricing models for orphan medicines: a case study for lumasiranNoa Rosenberg0Evert Manders1Sibren van den Berg2Lisa J. Deesker3Sander F. Garrelfs4Saco J. de Visser5Jaap W. Groothoff6Carla E. M. Hollak7Medicine for Society, Platform at Amsterdam University Medical Center - University of AmsterdamMedicine for Society, Platform at Amsterdam University Medical Center - University of AmsterdamMedicine for Society, Platform at Amsterdam University Medical Center - University of AmsterdamDepartment of Pediatric Nephrology, Emma Children’s Hospital, Amsterdam University Medical Center - University of AmsterdamDepartment of Pediatric Nephrology, Emma Children’s Hospital, Amsterdam University Medical Center - University of AmsterdamMedicine for Society, Platform at Amsterdam University Medical Center - University of AmsterdamDepartment of Pediatric Nephrology, Emma Children’s Hospital, Amsterdam University Medical Center - University of AmsterdamMedicine for Society, Platform at Amsterdam University Medical Center - University of AmsterdamAbstract Background The combination of high prices and uncertain effectiveness is a growing challenge in the field of orphan medicines, hampering health technology assessments. Hence, new methods for establishing price benchmarks might be necessary to support reimbursement negotiations. In this study, we applied several pricing models containing cost-based elements to the case of lumasiran for treating primary hyperoxaluria type 1. Methods Price ranges were calculated by estimating minimum and maximum scenarios for four pricing models: Novel Cancer Pricing Model (NCP-model), AIM Model for Innovative Medicines (AIM-model), Discounted Cash Flow model (DCF-model), and the Real-Option Rate Of Return model (ROROR-model). Data was gathered from disease registries, scientific literature, Security and Exchange Committee filings, and expert opinion. A sensitivity analysis was performed to assess the parameters with the largest influence. Results Outcomes resulting from the NCP-model ranged between €87,000 and €224,000 per patient per year, between €33,000 and €340,000 for the AIM-model, between €182,000 and €748,000 for the DCF-model, and between €81,000 and €273,000 for the ROROR-model. Conclusion Outcomes of the four pricing models show wide and heterogeneous price ranges. The DCF-model might be most compatible with the case of lumasiran, due to inclusion of parameters for prevalence, incidence, prescription restrictions and cost of capital. The minimum DCF price could serve as a starting point for pricing and reimbursement negotiations. Uncertainties can be solved by more transparency on input variables.https://doi.org/10.1186/s13023-024-03446-wPricing modelsLumasiranOrphan medicinal productsPrimary hyperoxaluria type 1 |
| spellingShingle | Noa Rosenberg Evert Manders Sibren van den Berg Lisa J. Deesker Sander F. Garrelfs Saco J. de Visser Jaap W. Groothoff Carla E. M. Hollak Application of four pricing models for orphan medicines: a case study for lumasiran Orphanet Journal of Rare Diseases Pricing models Lumasiran Orphan medicinal products Primary hyperoxaluria type 1 |
| title | Application of four pricing models for orphan medicines: a case study for lumasiran |
| title_full | Application of four pricing models for orphan medicines: a case study for lumasiran |
| title_fullStr | Application of four pricing models for orphan medicines: a case study for lumasiran |
| title_full_unstemmed | Application of four pricing models for orphan medicines: a case study for lumasiran |
| title_short | Application of four pricing models for orphan medicines: a case study for lumasiran |
| title_sort | application of four pricing models for orphan medicines a case study for lumasiran |
| topic | Pricing models Lumasiran Orphan medicinal products Primary hyperoxaluria type 1 |
| url | https://doi.org/10.1186/s13023-024-03446-w |
| work_keys_str_mv | AT noarosenberg applicationoffourpricingmodelsfororphanmedicinesacasestudyforlumasiran AT evertmanders applicationoffourpricingmodelsfororphanmedicinesacasestudyforlumasiran AT sibrenvandenberg applicationoffourpricingmodelsfororphanmedicinesacasestudyforlumasiran AT lisajdeesker applicationoffourpricingmodelsfororphanmedicinesacasestudyforlumasiran AT sanderfgarrelfs applicationoffourpricingmodelsfororphanmedicinesacasestudyforlumasiran AT sacojdevisser applicationoffourpricingmodelsfororphanmedicinesacasestudyforlumasiran AT jaapwgroothoff applicationoffourpricingmodelsfororphanmedicinesacasestudyforlumasiran AT carlaemhollak applicationoffourpricingmodelsfororphanmedicinesacasestudyforlumasiran |