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Pharmacoepidemiology in Clinical Research
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Pharmacoepidemiology in Clinical Research

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Pharmacology".

Deadline for manuscript submissions: closed (30 September 2023) | Viewed by 11069

Special Issue Editors

Dr. Eva Montané

E-Mail Website
Guest Editor
1. Department of Clinical Pharmacology, Hospital Universitari Germans Trias i Pujol, 08916 Badalona, Spain
2. Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, 08193 Cerdanyola del Vallès, Spain
Interests: adverse drug reactions; drug safety; pharmacoepidemiology; effectiveness; pharmacovigilance; patient safety
Dr. Mònica Sabaté

E-Mail Website
Guest Editor
1. Clinical Pharmacology Service, Vall d’Hebron Barcelona Hospital Campus, 08035 Barcelona, Spain
2. Department of Pharmacology, Therapeutics and Toxicology, Universitat Autònoma de Barcelona, 08193 Cerdanyola del Vallès, Spain
Interests: pharmacoepidemiology; pharmacovigilance; drug utilization

Special Issue Information

Dear Colleagues,

Pharmacoepidemiology refers to the study under real conditions and in large populations of the use, efficacy, and risks of drugs. With the constant approval of new drugs, there is a need to know their beneficial effects and the magnitude of risks in routine clinical practice.  First, we must confirm and explore how efficacy demonstrated in clinical trials translates to the real world. Second, pharmacovigilance actions need safety studies when drugs are used in a larger number of patients, and in patients who have multiple comorbidities and are polymedicated. With the aim of improving therapeutic decision making and patient health outcomes, the use of real-world data is increasing, as is the use of electronic health databases at the primary care and hospital level. As a consequence, new pharmacoepidemiological and statistical methods are being developed and should be disseminated to the scientific community.

With this Special Issue, the Journal of Clinical Medicine offers researchers the opportunity to showcase the scope of pharmacoepidemiology to a wide audience and the relevance of this discipline in usual clinical practice. We invite researchers to submit both original research and review articles that explore all of these aspects. We are especially looking for manuscripts dealing with real-world data studies from electronic health databases, patient registries, pharmacy registries, etc. with a relevant impact on patient health.

Dr. Eva Montané
Dr. Mònica Sabaté
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Journal of Clinical Medicine is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • adverse drug reactions
  • drug safety
  • pharmacoepidemiology
  • effectiveness
  • pharmacovigilance
  • drug utilization
  • real-world evidence

Published Papers (5 papers)

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Research

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13 pages, 7653 KiB  
Article
Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS)
by Derralynn A. Hughes, Patrick Deegan, Pilar Giraldo, Özlem Göker-Alpan, Heather Lau, Elena Lukina, Shoshana Revel-Vilk, Maurizio Scarpa, Jaco Botha, Noga Gadir and Ari Zimran
J. Clin. Med. 2022, 11(17), 5158; https://doi.org/10.3390/jcm11175158 - 31 Aug 2022
Cited by 6 | Viewed by 2733
Abstract
Switching between enzyme replacement therapies (ERT) and substrate reduction therapies (SRT) in patients with type 1 Gaucher disease (GD1) is not uncommon; however, the reasons for switchng treatments have not been explored in detail. Data from the Gaucher Outcome Survey (GOS), an international [...] Read more.
Switching between enzyme replacement therapies (ERT) and substrate reduction therapies (SRT) in patients with type 1 Gaucher disease (GD1) is not uncommon; however, the reasons for switchng treatments have not been explored in detail. Data from the Gaucher Outcome Survey (GOS), an international registry for patients with confirmed GD, were used to evaluate the reasons for, and consequences of, switching between these treatment types. Of the 1843 patients enrolled in GOS on 25 February 2020, 245 had undergone a treatment switch: 222 from initial ERT to SRT (of whom 88 later switched back to ERT) and 23 from initial SRT to ERT. The most common reasons for ERT–SRT switching were duration of infusion (25.4%), drug shortage (22.0%), and adverse events (AEs; 11.9%), and for SRT–ERT switching, AEs (63.6%), lack of beneficial effect (16.4%), and participation in a clinical trial (9.1%). Bodyweight and hematologic parameters largely remained stable before and after switching between ERT and SRT, although with substantial variation between patients. These findings contribute to understanding why treatment switching occurs in patients with GD, and may help physicians recognize the real-world impact of treatment switching between ERT and SRT for patients with GD. Full article
(This article belongs to the Special Issue Pharmacoepidemiology in Clinical Research)
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Review

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13 pages, 620 KiB  
Review
Pharmacoepidemiology: An Overview
by Mònica Sabaté and Eva Montané
J. Clin. Med. 2023, 12(22), 7033; https://doi.org/10.3390/jcm12227033 - 10 Nov 2023
Cited by 2 | Viewed by 3137
Abstract
The aims of this review are to provide a comprehensive overview of the definition and scope of pharmacoepidemiology, to summarize the study designs and methodologies used in the field, to discuss the future trends in the field and new methodologies to address bias [...] Read more.
The aims of this review are to provide a comprehensive overview of the definition and scope of pharmacoepidemiology, to summarize the study designs and methodologies used in the field, to discuss the future trends in the field and new methodologies to address bias and confounding, and finally to give some recommendations to clinicians interested in pharmacoepidemiologic research. Because drug efficacy and safety from randomized clinical trials do not reflect the real-world situation, pharmacoepidemiological studies on drug safety monitoring and drug effectiveness in large numbers of people are needed by healthcare professionals and regulatory institutions. We aim to highlight the importance of pharmacoepidemiologic research in informing evidence-based medicine and public health policy. The development of new designs and methodologies for the generation of valid evidence, as well as new initiatives to provide guidance and recommendations on how to incorporate real-world evidence into the drug development process, are reported on. In addition, we have touched on the implication of artificial intelligence in the management of real-world data. This overview aims to summarize all important aspects to consider when conducting or interpreting a pharmacoepidemiologic study. Full article
(This article belongs to the Special Issue Pharmacoepidemiology in Clinical Research)
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14 pages, 323 KiB  
Review
Making Sense of Composite Endpoints in Clinical Research
by Daniela Baracaldo-Santamaría, John Edwin Feliciano-Alfonso, Raul Ramirez-Grueso, Luis Carlos Rojas-Rodríguez, Camilo Alberto Dominguez-Dominguez and Carlos Alberto Calderon-Ospina
J. Clin. Med. 2023, 12(13), 4371; https://doi.org/10.3390/jcm12134371 - 29 Jun 2023
Cited by 4 | Viewed by 1660
Abstract
Multiple drugs currently used in clinical practice have been approved by regulatory agencies based on studies that utilize composite endpoints. Composite endpoints are appealing because they reduce sample size requirements, follow-up periods, and costs. However, interpreting composite endpoints can be challenging, and their [...] Read more.
Multiple drugs currently used in clinical practice have been approved by regulatory agencies based on studies that utilize composite endpoints. Composite endpoints are appealing because they reduce sample size requirements, follow-up periods, and costs. However, interpreting composite endpoints can be challenging, and their misuse is not uncommon. Incorrect interpretation of composite outcomes can lead to misleading conclusions that impact patient care. To correctly interpret composite outcomes, several important questions should be considered. Are the individual components of the composite outcome equally important to patients? Did the more and less important endpoints occur with similar frequency? Do the component endpoints exhibit similar relative risk reductions? If these questions receive affirmative answers, the use and interpretation of the composite endpoint would be appropriate. However, if any component of the composite endpoint fails to satisfy the aforementioned criteria, interpretation can become difficult, necessitating additional steps. Regulatory agencies acknowledge these challenges and have specific considerations when approving drugs based on studies employing composite endpoints. In conclusion, composite endpoints are valuable tools for evaluating the efficacy and net clinical benefit of interventions; however, cautious interpretation is advised. Full article
(This article belongs to the Special Issue Pharmacoepidemiology in Clinical Research)

Other

Jump to: Research, Review

3 pages, 176 KiB  
Reply
Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on “Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158”
by Derralynn A. Hughes, Patrick Deegan, Pilar Giraldo, Özlem Göker-Alpan, Heather Lau, Elena Lukina, Shoshana Revel-Vilk, Maurizio Scarpa, Jaco Botha, Noga Gadir and Ari Zimran
J. Clin. Med. 2023, 12(12), 4017; https://doi.org/10.3390/jcm12124017 - 13 Jun 2023
Viewed by 893
Abstract
Thank you for allowing us the opportunity to respond to the commentary from Mistry and colleagues (Comment: The two substrate reduction therapies for type 1 Gaucher disease are not equivalent) [...] Full article
(This article belongs to the Special Issue Pharmacoepidemiology in Clinical Research)
4 pages, 413 KiB  
Comment
The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11, 5158
by Pramod K. Mistry, Priya S. Kishnani, Manisha Balwani, Joel M. Charrow, Judy Hull, Neal J. Weinreb and Timothy M. Cox
J. Clin. Med. 2023, 12(9), 3269; https://doi.org/10.3390/jcm12093269 - 4 May 2023
Cited by 3 | Viewed by 1724
Abstract
In their paper, Hughes et al. [...] Full article
(This article belongs to the Special Issue Pharmacoepidemiology in Clinical Research)
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