{"doi":"10.1016/j.drudis.2025.104343","title":"Drug–device combinations in rare diseases: Challenges and opportunities","abstract":"• DDCs can allow for more effective patient outcomes through improved drug delivery, localized drug release, the cumulative action of the device, and enhanced diagnostic capabilities. • There are no specific incentives for the development and approval of DDCs in the rare disease space, which is potentially a missed opportunity. • The development and deployment of DDCs for rare diseases poses several challenges, many of which are intertwined with ethical, financial, regulatory, and practical considerations, making it a complex landscape to navigate. • To realize the full potential of DDCs in the rare disease space, a coordinated policy approach is needed. This could involve tailored regulatory pathways, enhanced collaboration between pharmaceutical and device manufacturing sectors, and the introduction of incentives specific to DDCs for rare diseases. 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.","journal":"Drug Discovery Today","year":2025,"id":522935,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":4,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9511,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2025-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1395426,"name":"Marc Dooms","orcid":null,"position":1,"is_corresponding":false},{"id":271386,"name":"Claudia Gonzaga‐Jauregui","orcid":"0000-0002-4667-3679","position":2,"is_corresponding":false},{"id":1119078,"name":"Anna M.G. Pasmooij","orcid":"0000-0003-0641-3829","position":3,"is_corresponding":false},{"id":234767,"name":"Daniel O’Connor","orcid":"0000-0001-7378-1125","position":4,"is_corresponding":false},{"id":1395427,"name":"Anneke Jonker","orcid":null,"position":5,"is_corresponding":false},{"id":1395425,"name":"Elena-Alexandra Tãtaru","orcid":null,"position":0,"is_corresponding":true}],"reference_count":47,"raw_metadata":null,"created_at":"2026-07-19T02:49:58.707747Z","pmid":"40122448","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}