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Restoring invisible and abandoned trials: a call for people to publish the findings

BMJ 2013; 346 doi: https://doi.org/10.1136/bmj.f2865 (Published 13 June 2013) Cite this as: BMJ 2013;346:f2865

Rapid Response:

Call to action: RIAT restoration of a previously unpublished methodology in Cervarix vaccine trials

We write to issue a call to action to restore the reporting of thirteen trial publications or published follow-up studies in GSK’s clinical development program for bivalent human papillomavirus (HPV) vaccine (Cervarix). These publications are:

NCT00689741 Harper DM, Franco EL, Wheeler C, Ferris DG, Jenkins D, Schuind A, Zahaf T, Innis B, Naud P, De Carvalho NS, Roteli-Martins CM, Teixeira J, Blatter MM, Korn AP, Quint W, Dubin G; GlaxoSmithKline HPV Vaccine Study Group. Efficacy of a bivalent L1 virus-like particle vaccine in prevention of infection with human papillomavirus types 16 and 18 in young women: a randomised controlled trial. Lancet. 2004 Nov 13-19;364(9447):1757-1765.

NCT00120848 Harper DM, Franco EL, Wheeler CM, Moscicki AB, Romanowski B, Roteli-Martins CM, Jenkins D, Schuind A, Costa Clemens SA, Dubin G; HPV Vaccine Study group. Sustained efficacy up to 4.5 years of a bivalent L1 virus-like particle vaccine against human papillomavirus types 16 and 18: follow-up from a randomised control trial. Lancet. 2006 Apr 15;367(9518):1247-1255.

NCT00294047 Skinner SR, Szarewski A, Romanowski B, Garland SM, Lazcano-Ponce E, Salmerón J, Del Rosario-Raymundo MR, Verheijen RH, Quek SC, da Silva DP, Kitchener H, Fong KL, Bouchard C, Money DM, Ilancheran A, Cruickshank ME, Levin MJ, Chatterjee A, Stapleton JT, Martens M, Quint W, David MP, Meric D, Hardt K, Descamps D, Geeraerts B, Struyf F, Dubin G; VIVIANE Study Group. Efficacy, safety, and immunogenicity of the human papillomavirus 16/18 AS04-adjuvanted vaccine in women older than 25 years: 4-year interim follow-up of the phase 3, double-blind, randomised controlled VIVIANE study. Lancet. 2014 Dec 20;384(9961):2213-27.

NCT00586339 Denny L, Hendricks B, Gordon C, Thomas F, Hezareh M, Dobbelaere K, Durand C, Hervé C, Descamps D. Safety and immunogenicity of the HPV-16/18 AS04-adjuvanted vaccine in HIV-positive women in South Africa: a partially-blind randomised placebo-controlled study. Vaccine. 2013 Nov 19;31(48):5745-53. doi: 10.1016/j.vaccine.2013.09.032.

NCT00481767 Sow PS, Watson-Jones D, Kiviat N, Changalucha J, Mbaye KD, Brown J, Bousso K, Kavishe B, Andreasen A, Toure M, Kapiga S, Mayaud P, Hayes R, Lebacq M, Herazeh M, Thomas F, Descamps D. Safety and immunogenicity of human papillomavirus-16/18 AS04-adjuvanted vaccine: a randomized trial in 10-25-year-old HIV-Seronegative African girls and young women. J Infect Dis. 2013 Jun 1;207(11):1753-63.

NCT00518336 (Publication 1) De Carvalho N, Teixeira J, Roteli-Martins CM, Naud P, De Borba P, Zahaf T, Sanchez N, Schuind A. Sustained efficacy and immunogenicity of the HPV-16/18 AS04-adjuvanted vaccine up to 7.3 years in young adult women. Vaccine. 2010 Aug 31;28(38):6247-6255.

NCT00518336 (Publication 2) Roteli-Martins CM, Naud P, De Borba P, Teixeira JC, De Carvalho NS, Zahaf T, Sanchez N, Geeraerts B, Descamps D. Sustained immunogenicity and efficacy of the HPV-16/18 AS04-adjuvanted vaccine: up to 8.4 years of follow-up. Hum Vaccin Immunother. 2012 Mar;8(3):390-7.

NCT00518336 (Publication 3) Naud PS, Roteli-Martins CM, De Carvalho NS, Teixeira JC, de Borba PC, Sanchez N, Zahaf T, Catteau G, Geeraerts B, Descamps D. Sustained efficacy, immunogenicity, and safety of the HPV-16/18 AS04-adjuvanted vaccine: final analysis of a long-term follow-up study up to 9.4 years post-vaccination. Hum Vaccin Immunother. 2014;10(8):2147-2162.

NCT00344032 Bhatla N, Suri V, Basu P, Shastri S, Datta SK, Bi D, Descamps DJ, Bock HL; Indian HPV Vaccine Study Group. Immunogenicity and safety of human papillomavirus-16/18 AS04-adjuvanted cervical cancer vaccine in healthy Indian women. J Obstet Gynaecol Res. 2010 Feb;36(1):123-32. doi: 10.1111/j.1447-0756.2009.01167.x. Erratum in: J Obstet Gynaecol Res. 2010 Apr;36(2):466.

NCT00306241 Ngan HYS, Cheung AN, Tam KF, Chan KK, Tang HW, Bi D, Descamps D, Bock HL. Human papillomavirus-16/18 AS04-adjuvanted cervical cancer vaccine: immunogenicity and safety in healthy Chinese women from Hong Kong. Hong Kong Med J. 2010 Jun;16(3):171-179.

NCT00485732 Kim SC, Song YS, Kim YT, Kim YT, Ryu KS, Gunapalaiah B, Bi D, Bock HL, Park JS. Human papillomavirus 16/18 AS04-adjuvanted cervical cancer vaccine: immunogenicity and safety in 15-25 years old healthy Korean women. J Gynecol Oncol. 2011 Jun 30;22(2):67-75.

NCT00779766 Zhu FC, Chen W, Hu YM, Hong Y, Li J, Zhang X, Zhang YJ, Pan QJ, Zhao FH, Yu JX, Zhang YS, Yang X, Zhang CF, Tang H, Zhang H, Lebacq M, David MP, Datta SK, Struyf F, Bi D, Descamps D; HPV-039 study group. Efficacy, immunogenicity and safety of the HPV-16/18 AS04-adjuvanted vaccine in healthy Chinese women aged 18-25 years: results from a randomized controlled trial. Int J Cancer. 2014 Dec 1;135(11):2612-2622.

NCT00996125 Zhu F, Li J, Hu Y, Zhang X, Yang X, Zhao H, Wang J, Yang J, Xia G, Dai Q, Tang H, Suryakiran P, Datta SK, Descamps D, Bi D, Struyf F. Immunogenicity and safety of the HPV-16/18 AS04-adjuvanted vaccine in healthy Chinese girls and women aged 9 to 45 years. Hum Vaccin Immunother. 2014;10(7):1795-806.

These are pivotal publications that underpin the approval of the vaccine. [22]

It is our belief that these publications [1–13] incompletely reported important methodological details and that twelve [1–12] of thirteen inaccurately described the formulation that the control arm received, necessitating correction of the record.

We intend to restore the written record for these trials in accordance with the principles of the Restoring Invisible and Abandoned Trials (RIAT) initiative, of which we are founders. [14] This restoration will be similar to the restoration of Gardasil trials for which we issued two Calls to Action (11 Jan 2019 and 21 Feb 2019) and for which we recently published our focussed RIAT restoration. [15–17]

Our rationale for correcting the record

By obtaining the available unredacted CSRs for Cervarix, we have determined that the comparator in trials and follow-up studies of the Cervarix vaccine appears to have been an injection containing aluminum hydroxide. It is our assessment that the primary publications of these trials may have misrepresented that an active comparator was used rather than an inert substance (such as saline injection) through the use of the term “placebo-controlled”. We plan to conduct a thorough investigation of the primary publication for each study listed above to determine the extent of this misrepresentation, and further, to determine why an active comparator was used.

The use of a comparator that was neither an inert substance nor an efficacious vaccine against another disease demands explanation. The clinical rationale for such a decision is unclear, as the trial arms do not mimic the real-life choice of deciding whether or not to receive the HPV vaccine, and it is incompatible with established ethical principles regarding the use of placebo in vaccine trials. [18] Furthermore, because aluminum hydroxide is not inert, the choice of aluminum-containing control complicates the interpretation of efficacy and safety results in trials.

Therefore, we consider the omission in journal articles, of any rationale for the selection of aluminum-containing control, to be a form of incomplete reporting (of important methodological details) and believe that the rationale must be reported. We also consider that use of the term “placebo” to describe a non-inert comparator like aluminum hydroxide inaccurately describes the formulation that the control arm received and constitutes an important error that requires correction. If trial participants were told they could receive “placebo” (widely defined as referring to an “inactive” [19, 20] or “inert” [18] substance) without being informed of all non-inert contents of the control arm injection, this raises ethical questions about trial conduct as well.

Scope of our restoration

We will systematically correct the record for all Cervarix trial publications identified above.

Our sources

Our population of trials potentially eligible for restoration is defined as the primary publications of clinical trials and follow-up studies for which we have obtained CSRs from Health Canada and from the GSK Clinical Study Register. Following a ruling in Canadian Federal Court, we have all unredacted CSRs for Cervarix that are in the holdings of Health Canada in our possession. [21]

Questions our restoration aims to answer

1. Has the rationale for using a control arm formulation that contained aluminum hydroxide been documented? If so, where, and what was it?

2. What comprised the control arm formulation in controlled Cervarix trials?

3. How was the control arm formulation described across trial publications, registry entries, CSRs, and informed consent forms?

4. What are the consequences of such a choice for trial participants, and how might it affect the interpretation of the trial results?

References
1. Harper DM, Franco EL, Wheeler C, et al. Efficacy of a bivalent L1 virus-like particle vaccine in prevention of infection with human papillomavirus types 16 and 18 in young women: a randomised controlled trial. Lancet Lond Engl. 2004;364(9447):1757-1765. doi:10.1016/S0140-6736(04)17398-4
2. Harper DM, Franco EL, Wheeler CM, et al. Sustained efficacy up to 4.5 years of a bivalent L1 virus-like particle vaccine against human papillomavirus types 16 and 18: follow-up from a randomised control trial. Lancet Lond Engl. 2006;367(9518):1247-1255. doi:10.1016/S0140-6736(06)68439-0
3. Skinner SR, Szarewski A, Romanowski B, et al. Efficacy, safety, and immunogenicity of the human papillomavirus 16/18 AS04-adjuvanted vaccine in women older than 25 years: 4-year interim follow-up of the phase 3, double-blind, randomised controlled VIVIANE study. Lancet Lond Engl. 2014;384(9961):2213-2227. doi:10.1016/S0140-6736(14)60920-X
4. Denny L, Hendricks B, Gordon C, et al. Safety and immunogenicity of the HPV-16/18 AS04-adjuvanted vaccine in HIV-positive women in South Africa: a partially-blind randomised placebo-controlled study. Vaccine. 2013;31(48):5745-5753. doi:10.1016/j.vaccine.2013.09.032
5. Sow PS, Watson-Jones D, Kiviat N, et al. Safety and Immunogenicity of Human Papillomavirus-16/18 AS04-Adjuvanted Vaccine: A Randomized Trial in 10–25-Year-Old HIV-Seronegative African Girls and Young Women. J Infect Dis. 2013;207(11):1753-1763. doi:10.1093/infdis/jis619
6. De Carvalho N, Teixeira J, Roteli-Martins CM, et al. Sustained efficacy and immunogenicity of the HPV-16/18 AS04-adjuvanted vaccine up to 7.3 years in young adult women. Vaccine. 2010;28(38):6247-6255. doi:10.1016/j.vaccine.2010.07.007
7. Roteli-Martins CM, Naud P, De Borba P, et al. Sustained immunogenicity and efficacy of the HPV-16/18 AS04-adjuvanted vaccine: up to 8.4 years of follow-up. Hum Vaccines Immunother. 2012;8(3):390-397. doi:10.4161/hv.18865
8. Naud PS, Roteli-Martins CM, De Carvalho NS, et al. Sustained efficacy, immunogenicity, and safety of the HPV-16/18 AS04-adjuvanted vaccine: final analysis of a long-term follow-up study up to 9.4 years post-vaccination. Hum Vaccines Immunother. 2014;10(8):2147-2162. doi:10.4161/hv.29532
9. Bhatla N, Suri V, Basu P, et al. Immunogenicity and safety of human papillomavirus-16/18 AS04-adjuvanted cervical cancer vaccine in healthy Indian women. J Obstet Gynaecol Res. 2010;36(1):123-132. doi:10.1111/j.1447-0756.2009.01167.x
10. Ngan HYS, Cheung ANY, Tam KF, et al. Human papillomavirus-16/18 AS04-adjuvanted cervical cancer vaccine: immunogenicity and safety in healthy Chinese women from Hong Kong. Hong Kong Med J Xianggang Yi Xue Za Zhi. 2010;16(3):171-179.
11. Kim SC, Song YS, Kim Y-T, et al. Human papillomavirus 16/18 AS04-adjuvanted cervical cancer vaccine: immunogenicity and safety in 15-25 years old healthy Korean women. J Gynecol Oncol. 2011;22(2):67-75. doi:10.3802/jgo.2011.22.2.67
12. Zhu F-C, Chen W, Hu Y-M, et al. Efficacy, immunogenicity and safety of the HPV-16/18 AS04-adjuvanted vaccine in healthy Chinese women aged 18-25 years: results from a randomized controlled trial. Int J Cancer. 2014;135(11):2612-2622. doi:10.1002/ijc.28897
13. Zhu F, Li J, Hu Y, et al. Immunogenicity and safety of the HPV-16/18 AS04-adjuvanted vaccine in healthy Chinese girls and women aged 9 to 45 years. Hum Vaccines Immunother. 2014;10(7):1795-1806. doi:10.4161/hv.28702
14. Doshi P. RIAT Support Center. RIAT Support Center. Accessed June 3, 2020. https://restoringtrials.org/
15. Call to action: RIAT restoration of a previously unpublished methodology in Gardasil vaccine trials. Published online May 14, 2020. Accessed May 19, 2020. https://www.bmj.com/content/346/bmj.f2865/rr-7
16. Doshi P, Bourgeois F, Hong K, et al. Adjuvant-containing control arms in pivotal quadrivalent human papillomavirus vaccine trials: restoration of previously unpublished methodology. BMJ Evid-Based Med. Published online March 17, 2020. doi:10.1136/bmjebm-2019-111331
17. Additional trials within scope: a follow-up to our “Call to action: RIAT restoration of a previously unpublished methodology in Gardasil vaccine trials.” Published online August 10, 2020. Accessed August 10, 2020. http://www.bmj.com/content/346/bmj.f2865/rr-9
18. Rid A, Saxena A, Baqui AH, et al. Placebo use in vaccine trials: recommendations of a WHO expert panel. Vaccine. 2014;32(37):4708-4712. doi:10.1016/j.vaccine.2014.04.022
19. Merck Clinical Trials. Accessed August 4, 2020. https://www.merckclinicaltrials.com/
20. Glossary of Common Site Terms - ClinicalTrials.gov. Accessed August 4, 2020. https://clinicaltrials.gov/ct2/about-studies/glossary
21. Precedent pushing practice: Canadian court orders release of unpublished clinical trial data - The BMJ. Accessed August 4, 2020. https://blogs.bmj.com/bmj/2018/07/19/precedent-pushing-practice-canadian...
22. 2009 - FDA Approves New Vaccine for Prevention of Cervical Cancer. Accessed September 3, 2020. http://wayback.archive-it.org/7993/20170112212440/http://www.fda.gov/New...

Competing interests: FB’s work is supported by a grant from the Burroughs Wellcome Fund and by the Harvard-MIT Center for Regulatory Science. The Laura and John Arnold Foundation funds the RIAT Support Center which supports the salaries of Peter Doshi (PD), Kyungwan Hong (KH), Mark Jones (MJ), Tom Jefferson (TJ), and Anisa Rowhani-Farid (ARF). In addition: PD has received travel funds from the European Respiratory Society (2012) and Uppsala Monitoring Center (2018); grants from the Laura and John Arnold Foundation (2017-20), American Association of Colleges of Pharmacy (2015), Patient-Centered Outcomes Research Institute (2014-16), Cochrane Methods Innovations Fund (2016-18), and UK National Institute for Health Research (2011-14); and is an editor at The BMJ and unpaid member of the Reagan-Udall Foundation for the FDA. KH received the Maryland CERSI Scholar award from the Food and Drug Administration (grant # 5U01FD005946-04). MJ reports research funds from the Cochrane Methods Innovation Fund to assist with providing interim guidance on the inclusion of clinical study reports and other regulatory documents in Cochrane Reviews. He is also deputy coordinating editor for the Cochrane Acute Respiratory Infections Group. TJ was in receipt of a Cochrane Methods Innovations Fund grant to develop guidance on the use of regulatory data in Cochrane reviews (2015-018). In 2014–2016, TJ was a member of three advisory boards for Boehringer Ingelheim. TJ was a member of an independent data monitoring committee for a Sanofi Pasteur clinical trial on an influenza vaccine. TJ is occasionally interviewed by market research companies about phase I or II pharmaceutical products for which he receives fees (current). TJ was a member of three advisory boards for Boehringer Ingelheim (2014-16). TJ was a member of an independent data monitoring committee for a Sanofi Pasteur clinical trial on an influenza vaccine (2015-2017). TJ is a relator in a False Claims Act lawsuit on behalf of the United States that involves sales of Tamiflu for pandemic stockpiling. If resolved in the United States’ favor, he would be entitled to a percentage of the recovery. TJ is co-holder of a Laura and John Arnold Foundation grant for development of a RIAT support centre (2017-2020) and Jean Monnet Network Grant, 2017-2020 for The Jean Monnet Health Law and Policy Network. TJ is an unpaid collaborator to the project Beyond Transparency in Pharmaceutical Research and Regulation led by Dalhousie University and funded by the Canadian Institutes of Health Research (2018-2022). TJ consulted for Illumina LLC on next generation gene sequencing (2019-2020). TJ was the consultant scientific coordinator for the HTA Medical Technology programme of the Agenzia per i Serivizi Sanitari Nazionali (AGENAS) of the Italian MoH (2007-2019). TJ is Director Medical Affairs for BC Solutions, a market access company for medical devices in Europe. TJ is funded by NIHR UK and the World Health Organization (WHO) to update Cochrane review A122, “Physical Interventions to interrupt the spread of respiratory viruses”. TJ is funded by Oxford University to carry out a living review on the transmission epidemiology of COVID-19. Since 2020, TJ receives fees for articles published by The Spectator and other media outlets. ARF and LS have no competing interests to declare.

05 October 2020
Peter Doshi
Associate professor
Florence Bourgeois, Anisa Rowhani-Farid, Kyungwan Hong, Mark Jones, Larissa Shamseer, Tom Jefferson
University of Maryland School of Pharmacy
Baltimore, MD, USA