Evidence map›Paper›PMID 35652051›Full record

ArticleFrontiers in pharmacology2022

Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study.

Francesca Gorini, Michele Santoro, Anna Pierini, Lorena Mezzasalma, Silvia Baldacci, Elena Bargagli, Alessandra Boncristiano, Maurizia Rossana Brunetto, Paolo Cameli, Francesco Cappelli and 14 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Frontiers in pharmacology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
1.1field-weighted citation impact, top 21% of its field
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

5 citing papers in PubMed, 6 citations in OpenAlex.

  1. Article
  2. Review
  3. Review
  4. Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023
    Review
  5. Profile of Drug Utilization in Patients with Rare Diseases in Tuscany, Italy: A Population-Based Study.International journal of environmental research and public health · 2023
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

24 authors at 8 institutions in 1 country.

Francesca GoriniUnit of Epidemiology of Rare Diseases and Congenital Anomalies, Institute of Clinical Physiology, National Research Council, Pisa, Italy.
Michele SantoroUnit of Epidemiology of Rare Diseases and Congenital Anomalies, Institute of Clinical Physiology, National Research Council, Pisa, Italy.
Anna PieriniUnit of Epidemiology of Rare Diseases and Congenital Anomalies, Institute of Clinical Physiology, National Research Council, Pisa, Italy.
Lorena MezzasalmaUnit of Epidemiology of Rare Diseases and Congenital Anomalies, Institute of Clinical Physiology, National Research Council, Pisa, Italy.
Silvia BaldacciUnit of Epidemiology of Rare Diseases and Congenital Anomalies, Institute of Clinical Physiology, National Research Council, Pisa, Italy.
Elena BargagliRespiratory Diseases Unit, Department of Medical and Surgical Sciences and Neurosciences, University of Siena, Siena, Italy.
Alessandra BoncristianoNeuroscience Department, A. Meyer Children Hospital-University of Florence, Florence, Italy.
Maurizia Rossana BrunettoDepartment of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy.
Paolo CameliRespiratory Diseases Unit, Department of Medical and Surgical Sciences and Neurosciences, University of Siena, Siena, Italy.
Francesco CappelliCardiomyopathy Unit, Careggi University Hospital, University of Florence, Florence, Italy.
Giancarlo CastamanCenter for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy.
Barbara CocoHepatology Unit, University Hospital of Pisa, Pisa, Italy.
Maria Alice DonatiMetabolic and Muscular Unit, A. Meyer Children Hospital, Florence, Italy.
Renzo GuerriniNeuroscience Department, A. Meyer Children Hospital-University of Florence, Florence, Italy.
Silvia LinariCenter for Bleeding Disorders and Coagulation, Department of Oncology, Careggi University Hospital, Florence, Italy.
Vittoria MurroDepartment of Neuroscience, Psychology, Drug Research and Child Health, University of Florence, Careggi University Hospital, Florence, Italy.
Iacopo OlivottoCardiomyopathy Unit, Careggi University Hospital, University of Florence, Florence, Italy.
Paola ParronchiDepartment of Experimental and Clinical Medicine, SOD Immunologia e Terapie Cellulari, Careggi University Hospital, University of Florence, Florence, Italy.
Francesca PochieroMetabolic and Muscular Unit, A. Meyer Children Hospital, Florence, Italy.
Oliviero RossiImmunuallergology Unit, SOD Immunoallergologia, Careggi University Hospital, Florence, Italy.
Barbara ScappiniHematology Unit, Careggi University Hospital, Florence, Italy.
Andrea SodiDepartment of Neuroscience, Psychology, Drug Research and Child Health, University of Florence, Careggi University Hospital, Florence, Italy.
Alessandro Maria VannucchiCenter Research and Innovation of Myeloproliferative Neoplasms (CRIMM), Department of Experimental and Clinical Medicine, Careggi University Hospital, University of Florence, Florence, Italy.
Alessio CoiUnit of Epidemiology of Rare Diseases and Congenital Anomalies, Institute of Clinical Physiology, National Research Council, Pisa, Italy.
University of Florence · ITAzienda Ospedaliero-Universitaria Careggi · ITNational Research Council · ITIstituto di Fisiologia Clinica · ITMeyer Children's Hospital · ITUniversity of Siena · ITAzienda Ospedaliera Universitaria Pisana · ITUniversity of Pisa · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

orphan drugpopulation-basedprescriptionrare diseaseregistry

Identifiers

PMID35652051
PMCPMC9148958
OpenAlexW4280619932

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.