Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments
Background: The drive to expedite patient access for diseases with high unmet treatment needs has come with an increasing use of single-arm trials (SATs), especially in oncology. However, the lack of control arms in such trials creates challenges to assess and demonstrate comparative efficacy. Ext...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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Becaris Publishing Limited
2024-01-01
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| Series: | Journal of Comparative Effectiveness Research |
| Subjects: |
| _version_ | 1827357669081481216 |
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| author | Katherine Appiah Maria Rizzo Grammati Sarri Luis Hernandez |
| author_facet | Katherine Appiah Maria Rizzo Grammati Sarri Luis Hernandez |
| author_sort | Katherine Appiah |
| collection | DOAJ |
| description | Background: The drive to expedite patient access for diseases with high unmet treatment needs has come
with an increasing use of single-arm trials (SATs), especially in oncology. However, the lack of control
arms in such trials creates challenges to assess and demonstrate comparative efficacy. External control
(EC) arms can be used to bridge this gap, with various types of sources available to obtain relevant
data. Objective: To examine the source of ECs in single-arm oncology health technology assessment
(HTA) submissions to the National Institute for Health and Care Excellence (NICE) and the Pharmaceutical
Benefits Advisory Committee (PBAC) and how this selection was justified by manufacturers and assessed
by the respective HTA body. Methods: Single-arm oncology HTA submission reports published by NICE
(England) and PBAC (Australia) from January 2011 to August 2021 were reviewed, with data qualitatively
synthesized to identify themes. Results: Forty-eight oncology submissions using EC arms between 2011
and 2021 were identified, with most submissions encompassing blood and bone marrow cancers (52%).
In HTA submissions to NICE and PBAC, the EC arm was typically constructed from a combination of data
sources, with the company’s justification in data source selection infrequently provided (PBAC [2 out of
19]; NICE [6 out of 29]), although this lack of justification was not heavily criticized by either HTA body.
Conclusion: Although HTA bodies such as NICE and PBAC encourage that EC source justification should be
provided in submissions, this review found that this is not typically implemented in practice. Guidance is
needed to establish best practices as to how EC selection should be documented in HTA submissions. |
| first_indexed | 2024-03-08T05:37:13Z |
| format | Article |
| id | doaj.art-c86344d2e3e44db1a7d628ef2b1ae55d |
| institution | Directory Open Access Journal |
| issn | 2042-6313 |
| language | English |
| last_indexed | 2024-03-08T05:37:13Z |
| publishDate | 2024-01-01 |
| publisher | Becaris Publishing Limited |
| record_format | Article |
| series | Journal of Comparative Effectiveness Research |
| spelling | doaj.art-c86344d2e3e44db1a7d628ef2b1ae55d2024-02-05T15:58:54ZengBecaris Publishing LimitedJournal of Comparative Effectiveness Research2042-63132024-01-0113210.57264/cer-2023-0140Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessmentsKatherine Appiah0Maria Rizzo1Grammati Sarri2Luis Hernandez3Cytel, Rotterdam, NetherlandsCytel, London, UKCytel, London, UKTakeda Pharmaceuticals America, Inc., Lexington, MA, USABackground: The drive to expedite patient access for diseases with high unmet treatment needs has come with an increasing use of single-arm trials (SATs), especially in oncology. However, the lack of control arms in such trials creates challenges to assess and demonstrate comparative efficacy. External control (EC) arms can be used to bridge this gap, with various types of sources available to obtain relevant data. Objective: To examine the source of ECs in single-arm oncology health technology assessment (HTA) submissions to the National Institute for Health and Care Excellence (NICE) and the Pharmaceutical Benefits Advisory Committee (PBAC) and how this selection was justified by manufacturers and assessed by the respective HTA body. Methods: Single-arm oncology HTA submission reports published by NICE (England) and PBAC (Australia) from January 2011 to August 2021 were reviewed, with data qualitatively synthesized to identify themes. Results: Forty-eight oncology submissions using EC arms between 2011 and 2021 were identified, with most submissions encompassing blood and bone marrow cancers (52%). In HTA submissions to NICE and PBAC, the EC arm was typically constructed from a combination of data sources, with the company’s justification in data source selection infrequently provided (PBAC [2 out of 19]; NICE [6 out of 29]), although this lack of justification was not heavily criticized by either HTA body. Conclusion: Although HTA bodies such as NICE and PBAC encourage that EC source justification should be provided in submissions, this review found that this is not typically implemented in practice. Guidance is needed to establish best practices as to how EC selection should be documented in HTA submissions.external controlshealth technology assessmentoncologyreal-world datareal-world evidencesingle-arm trial |
| spellingShingle | Katherine Appiah Maria Rizzo Grammati Sarri Luis Hernandez Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments Journal of Comparative Effectiveness Research external controls health technology assessment oncology real-world data real-world evidence single-arm trial |
| title | Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments |
| title_full | Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments |
| title_fullStr | Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments |
| title_full_unstemmed | Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments |
| title_short | Justifying the source of external comparators in single-arm oncology health technology submissions: a review of NICE and PBAC assessments |
| title_sort | justifying the source of external comparators in single arm oncology health technology submissions a review of nice and pbac assessments |
| topic | external controls health technology assessment oncology real-world data real-world evidence single-arm trial |
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