Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Pierpaolo Mincarone, Carlo Giacomo Leo, Saverio Sabina, Antonio Sarriá-Santamera, Domenica Taruscio, Pedro Guillermo Serrano-Aguilar, Panos Kanavos

Research output: Contribution to journalReview article

Abstract

The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential improvements. Current strategies and suggestions are presented along 4 dimensions: (1) comprehensive value assessment, (2) early dialogs among relevant stakeholders, (3) innovative reimbursement approaches, and (4) societal participation in producing ODs. Comprehensive value assessment could be achieved by clarifying the approach of distributive justice to adopt, ensuring a representative participation of stakeholders, and with a broad consideration of value-bearing factors. With respect to early dialogs, cross-border cooperation can be determinant to companies and agencies. The cost-benefit ratio of early dialogs needs to be demonstrated and the "regulatory capture" effect should be monitored. Innovative reimbursement approaches were developed to balance the need for evidence-based decisions with the timely access to innovative drugs. The societal participation in producing ODs needs to be recognised in a collaborating framework where adaptive agreements can be developed with mutual satisfaction. Such agreements could also impact on coverage and reimbursement decisions as additional elements for the determination of a comprehensive societal value of ODs. Further research is needed to investigate the highlighted open challenges so that RDs will not remain, in practical terms, orphan diseases.

Original languageEnglish
Pages (from-to)1-8
Number of pages8
JournalPublic Health Genomics
Volume20
Issue number1
DOIs
Publication statusPublished - 2017

Fingerprint

Orphan Drug Production
Rare Diseases
Costs and Cost Analysis
Drug and Narcotic Control
Social Justice
Reward
Cost-Benefit Analysis
Research
Pharmaceutical Preparations

Keywords

  • Cost-Benefit Analysis
  • Drug Costs
  • Drug Industry/economics
  • Humans
  • Interprofessional Relations
  • Orphan Drug Production/economics
  • Rare Diseases/drug therapy
  • Reimbursement Mechanisms/economics

Cite this

Mincarone, P., Leo, C. G., Sabina, S., Sarriá-Santamera, A., Taruscio, D., Serrano-Aguilar, P. G., & Kanavos, P. (2017). Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements. Public Health Genomics, 20(1), 1-8. https://doi.org/10.1159/000464100

Reimbursed Price of Orphan Drugs : Current Strategies and Potential Improvements. / Mincarone, Pierpaolo; Leo, Carlo Giacomo; Sabina, Saverio; Sarriá-Santamera, Antonio; Taruscio, Domenica; Serrano-Aguilar, Pedro Guillermo; Kanavos, Panos.

In: Public Health Genomics, Vol. 20, No. 1, 2017, p. 1-8.

Research output: Contribution to journalReview article

Mincarone, P, Leo, CG, Sabina, S, Sarriá-Santamera, A, Taruscio, D, Serrano-Aguilar, PG & Kanavos, P 2017, 'Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements', Public Health Genomics, vol. 20, no. 1, pp. 1-8. https://doi.org/10.1159/000464100
Mincarone P, Leo CG, Sabina S, Sarriá-Santamera A, Taruscio D, Serrano-Aguilar PG et al. Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements. Public Health Genomics. 2017;20(1):1-8. https://doi.org/10.1159/000464100
Mincarone, Pierpaolo ; Leo, Carlo Giacomo ; Sabina, Saverio ; Sarriá-Santamera, Antonio ; Taruscio, Domenica ; Serrano-Aguilar, Pedro Guillermo ; Kanavos, Panos. / Reimbursed Price of Orphan Drugs : Current Strategies and Potential Improvements. In: Public Health Genomics. 2017 ; Vol. 20, No. 1. pp. 1-8.
@article{38494863a94241718d5fdc35690155db,
title = "Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements",
abstract = "The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential improvements. Current strategies and suggestions are presented along 4 dimensions: (1) comprehensive value assessment, (2) early dialogs among relevant stakeholders, (3) innovative reimbursement approaches, and (4) societal participation in producing ODs. Comprehensive value assessment could be achieved by clarifying the approach of distributive justice to adopt, ensuring a representative participation of stakeholders, and with a broad consideration of value-bearing factors. With respect to early dialogs, cross-border cooperation can be determinant to companies and agencies. The cost-benefit ratio of early dialogs needs to be demonstrated and the {"}regulatory capture{"} effect should be monitored. Innovative reimbursement approaches were developed to balance the need for evidence-based decisions with the timely access to innovative drugs. The societal participation in producing ODs needs to be recognised in a collaborating framework where adaptive agreements can be developed with mutual satisfaction. Such agreements could also impact on coverage and reimbursement decisions as additional elements for the determination of a comprehensive societal value of ODs. Further research is needed to investigate the highlighted open challenges so that RDs will not remain, in practical terms, orphan diseases.",
keywords = "Cost-Benefit Analysis, Drug Costs, Drug Industry/economics, Humans, Interprofessional Relations, Orphan Drug Production/economics, Rare Diseases/drug therapy, Reimbursement Mechanisms/economics",
author = "Pierpaolo Mincarone and Leo, {Carlo Giacomo} and Saverio Sabina and Antonio Sarri{\'a}-Santamera and Domenica Taruscio and Serrano-Aguilar, {Pedro Guillermo} and Panos Kanavos",
note = "{\circledC} 2017 S. Karger AG, Basel.",
year = "2017",
doi = "10.1159/000464100",
language = "English",
volume = "20",
pages = "1--8",
journal = "Public Health Genomics",
issn = "1662-4246",
publisher = "S. Karger AG",
number = "1",

}

TY - JOUR

T1 - Reimbursed Price of Orphan Drugs

T2 - Current Strategies and Potential Improvements

AU - Mincarone, Pierpaolo

AU - Leo, Carlo Giacomo

AU - Sabina, Saverio

AU - Sarriá-Santamera, Antonio

AU - Taruscio, Domenica

AU - Serrano-Aguilar, Pedro Guillermo

AU - Kanavos, Panos

N1 - © 2017 S. Karger AG, Basel.

PY - 2017

Y1 - 2017

N2 - The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential improvements. Current strategies and suggestions are presented along 4 dimensions: (1) comprehensive value assessment, (2) early dialogs among relevant stakeholders, (3) innovative reimbursement approaches, and (4) societal participation in producing ODs. Comprehensive value assessment could be achieved by clarifying the approach of distributive justice to adopt, ensuring a representative participation of stakeholders, and with a broad consideration of value-bearing factors. With respect to early dialogs, cross-border cooperation can be determinant to companies and agencies. The cost-benefit ratio of early dialogs needs to be demonstrated and the "regulatory capture" effect should be monitored. Innovative reimbursement approaches were developed to balance the need for evidence-based decisions with the timely access to innovative drugs. The societal participation in producing ODs needs to be recognised in a collaborating framework where adaptive agreements can be developed with mutual satisfaction. Such agreements could also impact on coverage and reimbursement decisions as additional elements for the determination of a comprehensive societal value of ODs. Further research is needed to investigate the highlighted open challenges so that RDs will not remain, in practical terms, orphan diseases.

AB - The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential improvements. Current strategies and suggestions are presented along 4 dimensions: (1) comprehensive value assessment, (2) early dialogs among relevant stakeholders, (3) innovative reimbursement approaches, and (4) societal participation in producing ODs. Comprehensive value assessment could be achieved by clarifying the approach of distributive justice to adopt, ensuring a representative participation of stakeholders, and with a broad consideration of value-bearing factors. With respect to early dialogs, cross-border cooperation can be determinant to companies and agencies. The cost-benefit ratio of early dialogs needs to be demonstrated and the "regulatory capture" effect should be monitored. Innovative reimbursement approaches were developed to balance the need for evidence-based decisions with the timely access to innovative drugs. The societal participation in producing ODs needs to be recognised in a collaborating framework where adaptive agreements can be developed with mutual satisfaction. Such agreements could also impact on coverage and reimbursement decisions as additional elements for the determination of a comprehensive societal value of ODs. Further research is needed to investigate the highlighted open challenges so that RDs will not remain, in practical terms, orphan diseases.

KW - Cost-Benefit Analysis

KW - Drug Costs

KW - Drug Industry/economics

KW - Humans

KW - Interprofessional Relations

KW - Orphan Drug Production/economics

KW - Rare Diseases/drug therapy

KW - Reimbursement Mechanisms/economics

U2 - 10.1159/000464100

DO - 10.1159/000464100

M3 - Review article

C2 - 28359063

VL - 20

SP - 1

EP - 8

JO - Public Health Genomics

JF - Public Health Genomics

SN - 1662-4246

IS - 1

ER -