Abstract

OBJECTIVES: The present work reviews current practices for the definition of the reimbursed price of orphan drugs, and proposes a conceptual framework for their value-based pricing along with a roadmap for its possible implementation. METHODS: Based on a literature analysis, we systematize the current discussions on the topic within a conceptual framework intended to support evaluations and decision-making for determining a value-based reimbursed price for orphan drugs. RESULTS: Our analysis points out the "black box" pricing mechanism of orphan drugs and the limited consistency of some of the constituent domains of Health Technology Assessment when applied to orphan medical products (safety, clinical effectiveness, costs and economic evaluation, ethical analysis, and organizational and social aspects). The proposed framework comprises: a) elements of societal value and methods for its assessment, b) exchanges of valuable and trustworthy information between relevant stakeholders from an early stage, c) innovative reimbursement approaches to balance the need for evidence- based decisions with timely access to innovative drugs for patients with rare diseases, d) societal participation in the risky entrepreneurship of producing orphan drugs. Considering the areas reported in the proposed framework, we identified a possible roadmap for its implementation via three critical phases: i) sharing available experiences, ii) integrating and systematizing methods for appropriate use, iii) identifying delegate agencies. CONCLUSIONS: Additional piloting of emerging experiences and sharing of implementations developed worldwide are needed, along with the identification of an internationally agreed-upon taxonomy and pan-national recognised subjects to be delegated part of the HTA activities currently spread over a multiplicity of subjects.

Autore Pugliese

• Leo Carlo Giacomo , Sabina Saverio

Tutti gli autori

• Mincarone P. ; Leo C.G. ; Sabina S. ; Tordrup D. ; Taruscio D. ; Kanavos P.

Titolo volume/Rivista

Value in health

Anno di pubblicazione

2015

ISSN

1098-3015

ISBN

Non Disponibile

Numero di citazioni Wos

Nessuna citazione

Ultimo Aggiornamento Citazioni

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Numero di citazioni Scopus

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Ultimo Aggiornamento Citazioni

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Settori ERC

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Codici ASJC

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