A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint
Abstract Background It has been argued that true endpoints (or ‘hard’ endpoints) for clinical trials, which are meaningful to clinicians, researchers and patients alike, are limited to those that measure health status, survival and cost. Other endpoints are termed 'surrogate' endpoints and...
| Main Authors: | , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2018-07-01
|
| Series: | Pilot and Feasibility Studies |
| Subjects: | |
| Online Access: | http://link.springer.com/article/10.1186/s40814-018-0324-2 |
| _version_ | 1819084186932215808 |
|---|---|
| author | M. J. Campbell G. A. Lancaster S. M. Eldridge |
| author_facet | M. J. Campbell G. A. Lancaster S. M. Eldridge |
| author_sort | M. J. Campbell |
| collection | DOAJ |
| description | Abstract Background It has been argued that true endpoints (or ‘hard’ endpoints) for clinical trials, which are meaningful to clinicians, researchers and patients alike, are limited to those that measure health status, survival and cost. Other endpoints are termed 'surrogate' endpoints and are intended to substitute and predict the true endpoint. A number of trials that describe using surrogate endpoints use the term ‘pilot’ in the title of the paper but the reason for this, as related by the authors, is the use of these surrogate endpoints in the trial. The conduct and reporting of such a trial may follow the traditional pattern for a conventional randomised controlled trial (RCT) as defined by the original CONSORT statement, with power-based sample size calculations, and significance tests of the results. However, this is contrary to the guidelines of the CONSORT extension for the reporting of pilot trials. Main body We review the definition of a surrogate endpoint and the use of surrogate endpoints in clinical trials. We consider to what extent a trial could be considered a pilot trial if it uses a surrogate endpoint and discuss two examples that illustrate current practice. Conclusion Trials which use surrogate endpoints should only be described as ‘pilot’ when a definitive trial is a distinct possibility and the authors consider conditions which would indicate whether the definitive main trial was worthwhile and feasible. Simply because a trial uses a surrogate endpoint is not justification for calling it a pilot trial. |
| first_indexed | 2024-12-21T20:44:28Z |
| format | Article |
| id | doaj.art-5c385f7b5df04b1488dcb3e6ea885a7c |
| institution | Directory Open Access Journal |
| issn | 2055-5784 |
| language | English |
| last_indexed | 2024-12-21T20:44:28Z |
| publishDate | 2018-07-01 |
| publisher | BMC |
| record_format | Article |
| series | Pilot and Feasibility Studies |
| spelling | doaj.art-5c385f7b5df04b1488dcb3e6ea885a7c2022-12-21T18:50:52ZengBMCPilot and Feasibility Studies2055-57842018-07-01411410.1186/s40814-018-0324-2A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpointM. J. Campbell0G. A. Lancaster1S. M. Eldridge2ScHARR, University of SheffieldResearch Institute for Primary Care and Health Sciences, Keele UniversityCentre for Primary Care and Public Health, Queen Mary University of LondonAbstract Background It has been argued that true endpoints (or ‘hard’ endpoints) for clinical trials, which are meaningful to clinicians, researchers and patients alike, are limited to those that measure health status, survival and cost. Other endpoints are termed 'surrogate' endpoints and are intended to substitute and predict the true endpoint. A number of trials that describe using surrogate endpoints use the term ‘pilot’ in the title of the paper but the reason for this, as related by the authors, is the use of these surrogate endpoints in the trial. The conduct and reporting of such a trial may follow the traditional pattern for a conventional randomised controlled trial (RCT) as defined by the original CONSORT statement, with power-based sample size calculations, and significance tests of the results. However, this is contrary to the guidelines of the CONSORT extension for the reporting of pilot trials. Main body We review the definition of a surrogate endpoint and the use of surrogate endpoints in clinical trials. We consider to what extent a trial could be considered a pilot trial if it uses a surrogate endpoint and discuss two examples that illustrate current practice. Conclusion Trials which use surrogate endpoints should only be described as ‘pilot’ when a definitive trial is a distinct possibility and the authors consider conditions which would indicate whether the definitive main trial was worthwhile and feasible. Simply because a trial uses a surrogate endpoint is not justification for calling it a pilot trial.http://link.springer.com/article/10.1186/s40814-018-0324-2Pilot trialsSurrogate endpointsRandomised controlled trialsCONSORT |
| spellingShingle | M. J. Campbell G. A. Lancaster S. M. Eldridge A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint Pilot and Feasibility Studies Pilot trials Surrogate endpoints Randomised controlled trials CONSORT |
| title | A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint |
| title_full | A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint |
| title_fullStr | A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint |
| title_full_unstemmed | A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint |
| title_short | A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint |
| title_sort | randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint |
| topic | Pilot trials Surrogate endpoints Randomised controlled trials CONSORT |
| url | http://link.springer.com/article/10.1186/s40814-018-0324-2 |
| work_keys_str_mv | AT mjcampbell arandomisedcontrolledtrialisnotapilottrialsimplybecauseitusesasurrogateendpoint AT galancaster arandomisedcontrolledtrialisnotapilottrialsimplybecauseitusesasurrogateendpoint AT smeldridge arandomisedcontrolledtrialisnotapilottrialsimplybecauseitusesasurrogateendpoint AT mjcampbell randomisedcontrolledtrialisnotapilottrialsimplybecauseitusesasurrogateendpoint AT galancaster randomisedcontrolledtrialisnotapilottrialsimplybecauseitusesasurrogateendpoint AT smeldridge randomisedcontrolledtrialisnotapilottrialsimplybecauseitusesasurrogateendpoint |