Drug–device combinations in rare diseases: Challenges and opportunities

E. A. Tataru; M. Dooms; C. Gonzaga-Jauregui; A. M.G. Pasmooij; D. J. O'Connor; A. H. Jonker

doi:10.1016/j.drudis.2025.104343

Drug–device combinations in rare diseases: Challenges and opportunities

E. A. Tataru, M. Dooms, C. Gonzaga-Jauregui, A. M.G. Pasmooij, D. J. O'Connor, A. H. Jonker^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › Academic › peer-review

Abstract

Drug–device combinations (DDCs) are therapeutic products that integrate drugs with medical devices to enhance treatment efficacy and/or safety. These combinations hold significant promise for rare diseases, which affect millions of patients globally, by improving drug delivery, targeting specific organs, and reducing side effects. However, the regulatory framework for DDCs remains complex and lacks specific incentives for rare diseases, unlike orphan drugs. This review examines regulatory approaches and case studies of DDCs in rare diseases, and highlights specific challenges and untapped opportunities. Moreover, the publication discusses recommendations to overcome these challenges through tailored policies and incentives to unlock the potential of DDCs in the context of rare diseases.

Original language	English
Article number	104343
Journal	Drug Discovery Today
Volume	30
Issue number	4
DOIs	https://doi.org/10.1016/j.drudis.2025.104343
Publication status	Published - Apr 2025

Bibliographical note

Publisher Copyright:
© 2025 The Author(s)

Funding

The work was performed in a cross-committee meeting of the Therapies Scientific Committee and the Interdisciplinary Scientific Committee from the International Rare Diseases Research Committee, which was supported by the European Joint Programme on Rare Diseases (No. <EM><STRONG> </STRONG></EM>825575) and the European contract European Rare Diseases Research Alliance (ERDERA) (No. 101156595).

Funders	Funder number
European contract European Rare Diseases Research Alliance (ERDERA)	825575
Not added	101156595

Keywords

combination products
drug–device combinations
orphan drugs
orphan medical devices
rare diseases

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Cite this

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AU - O'Connor, D. J.

AU - Jonker, A. H.

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Drug–device combinations in rare diseases: Challenges and opportunities

Abstract

Bibliographical note

Funding

Keywords

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