Placebo comparator group selection and use in surgical trials : The aspire project including expert workshop
(2021) In Health Technology Assessment 25(53).- Abstract
Background: The use of placebo comparisons for randomised trials assessing the efficacy of surgical interventions is increasingly being considered. However, a placebo control is a complex type of comparison group in the surgical setting and, although powerful, presents many challenges. Objectives: To provide a summary of knowledge on placebo controls in surgical trials and to summarise any recommendations for designers, evaluators and funders of placebo-controlled surgical trials. Design: To carry out a state-of-the-art workshop and produce a corresponding report involving key stakeholders throughout. Setting: A workshop to discuss and summarise the existing knowledge and to develop the new guidelines. Results: To assess what a placebo... (More)
Background: The use of placebo comparisons for randomised trials assessing the efficacy of surgical interventions is increasingly being considered. However, a placebo control is a complex type of comparison group in the surgical setting and, although powerful, presents many challenges. Objectives: To provide a summary of knowledge on placebo controls in surgical trials and to summarise any recommendations for designers, evaluators and funders of placebo-controlled surgical trials. Design: To carry out a state-of-the-art workshop and produce a corresponding report involving key stakeholders throughout. Setting: A workshop to discuss and summarise the existing knowledge and to develop the new guidelines. Results: To assess what a placebo control entails and to assess the understanding of this tool in the context of surgery is considered, along with when placebo controls in surgery are acceptable (and when they are desirable). We have considered ethics arguments and regulatory requirements, how a placebo control should be designed, how to identify and mitigate risk for participants in these trials, and how such trials should be carried out and interpreted. The use of placebo controls is justified in randomised controlled trials of surgical interventions provided that there is a strong scientific and ethics rationale. Surgical placebos might be most appropriate when there is poor evidence for the efficacy of the procedure and a justified concern that results of a trial would be associated with a high risk of bias, particularly because of the placebo effect. Conclusions: The use of placebo controls is justified in randomised controlled trials of surgical interventions provided that there is a strong scientific and ethics rationale. Feasibility work is recommended to optimise the design and implementation of randomised controlled trials. An outline for best practice was produced in the form of the Applying Surgical Placebo in Randomised Evaluations (ASPIRE) guidelines for those considering the use of a placebo control in a surgical randomised controlled trial. Limitations: Although the workshop participants involved international members, the majority of participants were from the UK. Therefore, although every attempt was made to make the recommendations applicable to all health systems, the guidelines may, unconsciously, be particularly applicable to clinical practice in the UK NHS. Future work: Future work should evaluate the use of the ASPIRE guidelines in making decisions about the use of a placebo-controlled surgical trial. In addition, further work is required on the appropriate nomenclature to adopt in this space.
(Less)
- author
- Beard, David J.
; Campbell, Marion K.
; Blazeby, Jane M.
; Carr, Andrew J.
; Weijer, Charles
; Cuthbertson, Brian H.
; Buchbinder, Rachelle
; Pinkney, Thomas
; Bishop, Felicity L.
; Pugh, Jonathan
; Cousins, Sian
; Harris, Ian
; Lohmander, L. Stefan
LU
; Blencowe, Natalie
; Gillies, Katie
; Probst, Pascal
; Brennan, Carol
; Cook, Andrew
; Farrar-Hockley, Dair
; Savulescu, Julian
; Huxtable, Richard
; Rangan, Amar
; Tracey, Irene
; Brocklehurst, Peter
; Ferreira, Manuela L.
; Nicholl, Jon
; Reeves, Barnaby C.
; Hamdy, Freddie
; Rowley, Samuel C.S.
; Lee, Naomi
and Cook, Jonathan A.
(Less) - organization
- publishing date
- 2021
- type
- Contribution to journal
- publication status
- published
- subject
- in
- Health Technology Assessment
- volume
- 25
- issue
- 53
- publisher
- National Co-ordinating Centre for HTA
- external identifiers
-
- pmid:34505829
- scopus:85117375961
- ISSN
- 1366-5278
- DOI
- 10.3310/HTA25530
- language
- English
- LU publication?
- yes
- additional info
- Publisher Copyright: © 2021 Beard et al.
- id
- 285d31cc-78e3-43d6-ba15-3e1358e3c458
- date added to LUP
- 2022-01-13 11:22:31
- date last changed
- 2024-08-11 04:40:55
@article{285d31cc-78e3-43d6-ba15-3e1358e3c458,
abstract = {{<p>Background: The use of placebo comparisons for randomised trials assessing the efficacy of surgical interventions is increasingly being considered. However, a placebo control is a complex type of comparison group in the surgical setting and, although powerful, presents many challenges. Objectives: To provide a summary of knowledge on placebo controls in surgical trials and to summarise any recommendations for designers, evaluators and funders of placebo-controlled surgical trials. Design: To carry out a state-of-the-art workshop and produce a corresponding report involving key stakeholders throughout. Setting: A workshop to discuss and summarise the existing knowledge and to develop the new guidelines. Results: To assess what a placebo control entails and to assess the understanding of this tool in the context of surgery is considered, along with when placebo controls in surgery are acceptable (and when they are desirable). We have considered ethics arguments and regulatory requirements, how a placebo control should be designed, how to identify and mitigate risk for participants in these trials, and how such trials should be carried out and interpreted. The use of placebo controls is justified in randomised controlled trials of surgical interventions provided that there is a strong scientific and ethics rationale. Surgical placebos might be most appropriate when there is poor evidence for the efficacy of the procedure and a justified concern that results of a trial would be associated with a high risk of bias, particularly because of the placebo effect. Conclusions: The use of placebo controls is justified in randomised controlled trials of surgical interventions provided that there is a strong scientific and ethics rationale. Feasibility work is recommended to optimise the design and implementation of randomised controlled trials. An outline for best practice was produced in the form of the Applying Surgical Placebo in Randomised Evaluations (ASPIRE) guidelines for those considering the use of a placebo control in a surgical randomised controlled trial. Limitations: Although the workshop participants involved international members, the majority of participants were from the UK. Therefore, although every attempt was made to make the recommendations applicable to all health systems, the guidelines may, unconsciously, be particularly applicable to clinical practice in the UK NHS. Future work: Future work should evaluate the use of the ASPIRE guidelines in making decisions about the use of a placebo-controlled surgical trial. In addition, further work is required on the appropriate nomenclature to adopt in this space.</p>}},
author = {{Beard, David J. and Campbell, Marion K. and Blazeby, Jane M. and Carr, Andrew J. and Weijer, Charles and Cuthbertson, Brian H. and Buchbinder, Rachelle and Pinkney, Thomas and Bishop, Felicity L. and Pugh, Jonathan and Cousins, Sian and Harris, Ian and Lohmander, L. Stefan and Blencowe, Natalie and Gillies, Katie and Probst, Pascal and Brennan, Carol and Cook, Andrew and Farrar-Hockley, Dair and Savulescu, Julian and Huxtable, Richard and Rangan, Amar and Tracey, Irene and Brocklehurst, Peter and Ferreira, Manuela L. and Nicholl, Jon and Reeves, Barnaby C. and Hamdy, Freddie and Rowley, Samuel C.S. and Lee, Naomi and Cook, Jonathan A.}},
issn = {{1366-5278}},
language = {{eng}},
number = {{53}},
publisher = {{National Co-ordinating Centre for HTA}},
series = {{Health Technology Assessment}},
title = {{Placebo comparator group selection and use in surgical trials : The aspire project including expert workshop}},
url = {{http://dx.doi.org/10.3310/HTA25530}},
doi = {{10.3310/HTA25530}},
volume = {{25}},
year = {{2021}},
}