View in EDS HTML Full Text PDF Full Text

Patient-reported outcome measures in drugs for neurological conditions approved by European Medicines Agency 2017–2022.

Saved in:
Bibliographic Details
Title: Patient-reported outcome measures in drugs for neurological conditions approved by European Medicines Agency 2017–2022.
Authors: Ciani, Oriana (AUTHOR), Meregaglia, Michela (AUTHOR), Battaglia, Mario Alberto (AUTHOR), Brichetto, Gianpaolo (AUTHOR), Conte, Antonella (AUTHOR), Gasperini, Claudio (AUTHOR), Sansone, Valeria (AUTHOR)
Source: Neurological Sciences. Aug2023, Vol. 44 Issue 8, p2933-2937. 5p. 1 Diagram, 1 Graph.
Subjects: European Medicines Agency, Patient reported outcome measures, Neurological disorders, Drug approval, Patient experience, Government agencies
Abstract: Background: Regulatory agencies have been responsive to public demand for inclusion of the patient experience in evaluating and approving therapies. Over the years, patient-reported outcome measures (PROMs) have become increasingly prevalent in clinical trial protocols; however, their influence on regulators, payers, clinicians, and patients' decision-making is not always clear. We recently conducted a cross-sectional study aimed at investigating the use of PROMs in new regulatory approvals of drugs for neurological conditions between 2017 and 2022 in Europe. Methods: We reviewed European Public Assessment Reports (EPARs) and recorded on a predefined data extraction form whether they considered PROMs, their characteristics (e.g., primary/secondary endpoint, generic/specific instrument) and other relevant information (e.g., therapeutic area, generic/biosimilar, orphan status). Results were tabulated and summarized by means of descriptive statistics. Results: Of the 500 EPARs related to authorized medicines between January 2017 and December 2022, 42 (8%) concerned neurological indications. Among the EPARs of these products, 24 (57%) reported any use of PROMs, typically considered as secondary (38%) endpoints. In total, 100 PROMs were identified, of which the most common were the EQ-5D (9%), the SF-36 (6%), or its shorter adaptation SF-12, the PedsQL (4%). Conclusions: Compared to other disease areas, neurology is one where the use of patient-reported outcomes evidence is inherently part of the clinical evaluation and for which core outcome sets exist. Better harmonization of the instruments recommended for use would facilitate the consideration of PROMs at all stages in the drug development process. [ABSTRACT FROM AUTHOR]
Copyright of Neurological Sciences is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
Database: Psychology and Behavioral Sciences Collection
Full text is not displayed to guests.
FullText Links:
  – Type: pdflink
Text:
  Availability: 1
Header DbId: pbh
DbLabel: Psychology and Behavioral Sciences Collection
An: 164900300
AccessLevel: 6
PubType: Academic Journal
PubTypeId: academicJournal
PreciseRelevancyScore: 0
IllustrationInfo
Items – Name: Title
  Label: Title
  Group: Ti
  Data: Patient-reported outcome measures in drugs for neurological conditions approved by European Medicines Agency 2017–2022.
– Name: Author
  Label: Authors
  Group: Au
  Data: <searchLink fieldCode="AR" term="%22Ciani%2C+Oriana%22">Ciani, Oriana</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Meregaglia%2C+Michela%22">Meregaglia, Michela</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Battaglia%2C+Mario+Alberto%22">Battaglia, Mario Alberto</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Brichetto%2C+Gianpaolo%22">Brichetto, Gianpaolo</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Conte%2C+Antonella%22">Conte, Antonella</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Gasperini%2C+Claudio%22">Gasperini, Claudio</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Sansone%2C+Valeria%22">Sansone, Valeria</searchLink> (AUTHOR)
– Name: TitleSource
  Label: Source
  Group: Src
  Data: <searchLink fieldCode="JN" term="%22Neurological+Sciences%22">Neurological Sciences</searchLink>. Aug2023, Vol. 44 Issue 8, p2933-2937. 5p. 1 Diagram, 1 Graph.
– Name: Subject
  Label: Subjects
  Group: Su
  Data: <searchLink fieldCode="DE" term="%22European+Medicines+Agency%22">European Medicines Agency</searchLink><br /><searchLink fieldCode="DE" term="%22Patient+reported+outcome+measures%22">Patient reported outcome measures</searchLink><br /><searchLink fieldCode="DE" term="%22Neurological+disorders%22">Neurological disorders</searchLink><br /><searchLink fieldCode="DE" term="%22Drug+approval%22">Drug approval</searchLink><br /><searchLink fieldCode="DE" term="%22Patient+experience%22">Patient experience</searchLink><br /><searchLink fieldCode="DE" term="%22Government+agencies%22">Government agencies</searchLink>
– Name: Abstract
  Label: Abstract
  Group: Ab
  Data: Background: Regulatory agencies have been responsive to public demand for inclusion of the patient experience in evaluating and approving therapies. Over the years, patient-reported outcome measures (PROMs) have become increasingly prevalent in clinical trial protocols; however, their influence on regulators, payers, clinicians, and patients' decision-making is not always clear. We recently conducted a cross-sectional study aimed at investigating the use of PROMs in new regulatory approvals of drugs for neurological conditions between 2017 and 2022 in Europe. Methods: We reviewed European Public Assessment Reports (EPARs) and recorded on a predefined data extraction form whether they considered PROMs, their characteristics (e.g., primary/secondary endpoint, generic/specific instrument) and other relevant information (e.g., therapeutic area, generic/biosimilar, orphan status). Results were tabulated and summarized by means of descriptive statistics. Results: Of the 500 EPARs related to authorized medicines between January 2017 and December 2022, 42 (8%) concerned neurological indications. Among the EPARs of these products, 24 (57%) reported any use of PROMs, typically considered as secondary (38%) endpoints. In total, 100 PROMs were identified, of which the most common were the EQ-5D (9%), the SF-36 (6%), or its shorter adaptation SF-12, the PedsQL (4%). Conclusions: Compared to other disease areas, neurology is one where the use of patient-reported outcomes evidence is inherently part of the clinical evaluation and for which core outcome sets exist. Better harmonization of the instruments recommended for use would facilitate the consideration of PROMs at all stages in the drug development process. [ABSTRACT FROM AUTHOR]
– Name: AbstractSuppliedCopyright
  Label:
  Group: Ab
  Data: <i>Copyright of Neurological Sciences is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
PLink https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=pbh&AN=164900300
RecordInfo BibRecord:
  BibEntity:
    Identifiers:
      – Type: doi
        Value: 10.1007/s10072-023-06825-6
    Languages:
      – Code: eng
        Text: English
    PhysicalDescription:
      Pagination:
        PageCount: 5
        StartPage: 2933
    Subjects:
      – SubjectFull: European Medicines Agency
        Type: general
      – SubjectFull: Patient reported outcome measures
        Type: general
      – SubjectFull: Neurological disorders
        Type: general
      – SubjectFull: Drug approval
        Type: general
      – SubjectFull: Patient experience
        Type: general
      – SubjectFull: Government agencies
        Type: general
    Titles:
      – TitleFull: Patient-reported outcome measures in drugs for neurological conditions approved by European Medicines Agency 2017–2022.
        Type: main
  BibRelationships:
    HasContributorRelationships:
      – PersonEntity:
          Name:
            NameFull: Ciani, Oriana
      – PersonEntity:
          Name:
            NameFull: Meregaglia, Michela
      – PersonEntity:
          Name:
            NameFull: Battaglia, Mario Alberto
      – PersonEntity:
          Name:
            NameFull: Brichetto, Gianpaolo
      – PersonEntity:
          Name:
            NameFull: Conte, Antonella
      – PersonEntity:
          Name:
            NameFull: Gasperini, Claudio
      – PersonEntity:
          Name:
            NameFull: Sansone, Valeria
    IsPartOfRelationships:
      – BibEntity:
          Dates:
            – D: 01
              M: 08
              Text: Aug2023
              Type: published
              Y: 2023
          Identifiers:
            – Type: issn-print
              Value: 15901874
          Numbering:
            – Type: volume
              Value: 44
            – Type: issue
              Value: 8
          Titles:
            – TitleFull: Neurological Sciences
              Type: main
ResultId 1