ArticleClinical and translational science2019
Precision Medicines' Impact on Orphan Drug Designation.
Article in Clinical and translational science, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
7 citing papers in PubMed, 1 synthesis or guideline pooled it, 15 citations in OpenAlex.
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled it
- Recent advances in interstitial fluid dynamics for diagnostic, targeted drug delivery and precision medicine applications.Drug delivery and translational research · 2026Review
- Six-Year Trends in Real-World Data Use for Post-Marketing Surveillance of New Medical Products in Japan.Clinical and translational science · 2026Review
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
- Pharmacogenomics: Driving Personalized Medicine.Pharmacological reviews · 2023Review
- The costs of developing treatments for Alzheimer's disease: A retrospective exploration.Alzheimer's & dementia : the journal of the Alzheimer's Association · 2022Article
- Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017.JAMA network open · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The incentives provided under the Orphan Drug Act (ODA) have been credited for catalyzing the marketing approval of drugs for the treatment of rare diseases by the US Food and Drug Administration. Orphan drug designation, the granting of special status to drugs or biologics ("drugs") for the treatment of rare diseases, one of the ODA's key incentive programs, has seen major increases in volume over recent years. The new era of precision medicine and the development of therapies directed toward smaller "orphan" subsets of common diseases have been suggested as being a major driver. We evaluated the basis for orphan drug designations and orphan subsets in relation to the impact of precision medicines. We found that the increasing numbers of orphan drug designation determinations were not driven by precision medicines separating common diseases into orphan subsets and that orphan subsets overall also represented a relatively small proportion of designations.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.