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Drugs

, Volume 78, Issue 4, pp 399–410 | Cite as

Generic Substitution of Orphan Drugs for the Treatment of Rare Diseases: Exploring the Potential Challenges

  • Antonello Di Paolo
  • Elena Arrigoni
Current Opinion

Abstract

Generic drugs are important components of measures introduced by healthcare regulatory authorities to reduce treatment costs. In most patients and conditions the switch from a branded drug to its generic counterpart is performed with no major complications. However, evidence from complex diseases suggests that generic substitution requires careful evaluation in some settings and that current bioequivalence criteria may not always be adequate for establishing the interchangeability of branded and generic products. Rare diseases, also called orphan diseases, are a group of heterogeneous diseases that share important characteristics: in addition to their scarcity, most are severe, chronic, highly debilitating, and often present in early childhood. Finding a treatment for a rare disease is challenging. Thanks to incentives that encourage research and development programs in rare diseases, several orphan drugs are currently available. The elevated cost of orphan drugs is a highly debated issue and a cause of limited access to treatment for many patients. As patent protection and the exclusivity period of several orphan drugs will expire soon, generic versions of orphan drugs should reach the market shortly, with great expectations about their impact on the economic burden of rare diseases. However, consistent with other complex diseases, generic substitution may require thoughtful considerations and may be even contraindicated in some rare conditions. This article provides an overview of rare disease characteristics, reviews reports of problematic generic substitution, and discusses why generic substitution of orphan drugs may be challenging and should be undertaken carefully in rare disease patients.

Notes

Acknowledgements

The author thanks Lorenza Lanini, an independent medical writer acting on behalf of Springer Healthcare Communications, who drafted the outline and first draft of this manuscript, as well as Matt Weitz of Springer Healthcare Communications for English editing and formatting of the manuscript for submission. Mimi Chan, PhD, of Springer Healthcare Communications, assisted with English editing of post-submission revisions. This editorial support was funded by Orphan Europe – Recordati Group.

Compliance with ethical standards

Funding

Orphan Europe-Recordati Group funded the assistance for medical writing.

Conflict of interest

ADP acted as advisory board member for Novartis Farma SpA.

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Copyright information

© Springer International Publishing AG, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Section of Pharmacology, Department of Clinical and Experimental MedicineUniversity of PisaPisaItaly

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