Journal Article > ResearchFull Text
PLOS Glob Public Health. 23 April 2025; Volume 5 (Issue 4); e0003759.; DOI:10.1371/journal.pgph.0003759
Gotham D, Martin M, Barber MJ, Kazounis E, Batts C, et al.
PLOS Glob Public Health. 23 April 2025; Volume 5 (Issue 4); e0003759.; DOI:10.1371/journal.pgph.0003759
Clinical trials are considered to be the largest contributor to pharmaceutical development costs. However, public disclosure of the costs of individual clinical trials is rare. Médecins Sans Frontières (MSF) sponsored a phase 2b-3 randomised controlled trial (TB-PRACTECAL), which identified a new treatment regimen for drug-resistant TB. We aimed to analyse the costs of undertaking a pivotal clinical trial conducted in relatively low-resource health settings and to demonstrate the feasibility of reporting clinical trial costs. TB-PRACTECAL trial costs were analysed using MSF accounting documents. Costs were broken down by cost category, year, and trial site. Total costs for TB-PRACTECAL were €33.9 million and the average cost per patient was €61,460. Twenty-six percent of total costs represented central activities (e.g. trial planning, trial management) and 72% represented trial site activities, with 2% uncategorizable. Within trial site costs, personnel costs were the largest cost (43%) followed by external diagnostic services (11%), medicines (9%), and other medical consumables (7%). Cost variation across trial sites was driven by different varying levels of pre-existing trial infrastructure. A review of previous studies yielded a wide range of cost estimates for clinical trials (ranging US$7–221 million/trial for pharmaceutical phase 2 and 3 trials). Nearly all previous estimates derive from industry reporting that is neither standardized nor auditable; to our knowledge, this is the first published comprehensive analysis of direct expenditures of a specific clinical trial including detailed cost breakdowns. The €34 million cost of TB-PRACTECAL included investments in developing clinical trial infrastructure, the complexity of managing six sites across three health systems, and medical expenditures that are not typical of standard clinical trials. Greater transparency in drug development costs can inform medicine pricing negotiations and is a key element in the design and implementation of more equitable systems of biomedical research and development.
Conference Material > Abstract
Gotham D, Martin M, Barber M, Kazounis E, Batts C, et al.
MSF Scientific Day International 2024. 16 May 2024; DOI:10.57740/aMphKRQ
INTRODUCTION
Clinical trials are a cornerstone of medical innovation. Nonetheless, little information on the cost of conducting clinical trials is available, especially for clinical trials in the global south. This lack of data and transparency hinders the creation of reliable cost estimates and adequate funding of clinical trials in resource- limited settings. Following the recent adoption of the Médecins Sans Frontières (MSF) Clinical Trial Transparency Policy, we present a detailed cost report for TB-PRACTECAL.
METHODS
TB-PRACTECAL was an open-label, phase 2–3, multicentre randomised trial of all-oral regimens for the treatment of drug- resistant tuberculosis. Trial planning began in 2013 and work on publications continued into 2023. The trial took place in six sites across Belarus, South Africa, and Uzbekistan, and enrolled 552 patients. We analysed accounting data for the TB-PRACTECAL project, comprehensively including different costs, presented into 27 categories, by site, and by year, and at the per-patient level.
RESULTS
Total costs for TB-PRACTECAL were €33.9 million, of which 26% were at central level (costs incurred by the UK clinical trial team including trial planning, management, quality assurance, and analysis of results), while 72% were at the trial site level (across all six sites) and 2% were uncategorisable. At trial sites, the largest cost category was staff (43%), followed by external diagnostic services (11%), medicines (9%), other medical consumables (7%), external non-medical services (6%), and transport and travel (6%). Among medicines, the costliest were bedaquiline (46% of medicine costs), linezolid (16%), imipenem/ cilastatin (10%), and delamanid (9%). The mean cost per patient enrolled was €61,460 across the whole trial (including trial management overhead). When only site-level costs were considered, per-patient costs ranged between €19,998 and €45,942 across the six sites.
CONCLUSION
The costs of TB-PRACTECAL were similar to previously reported estimates for comparable clinical trials. However, TB- PRACTECAL included additional costs that would not typically be incurred in a commercial trial, such as investments in clinical research infrastructure and purchase of investigative medical products. To our knowledge, this is the first time MSF, or any other entity, published and analysed the disaggregated costs of a specific clinical trial. These data could help generate reliable predictions for future clinical trials and support planning and involvement, particularly in low-resource settings. Additionally, this study highlights the role of clinical trial cost disclosure in supporting both practical and policy discussions around the development of a more equitable system of biomedical R&D and fairer medicine pricing. Additionally, we developed a financial reporting template to facilitate future reporting of clinical trial cost by MSF and other entities investing in research.
Clinical trials are a cornerstone of medical innovation. Nonetheless, little information on the cost of conducting clinical trials is available, especially for clinical trials in the global south. This lack of data and transparency hinders the creation of reliable cost estimates and adequate funding of clinical trials in resource- limited settings. Following the recent adoption of the Médecins Sans Frontières (MSF) Clinical Trial Transparency Policy, we present a detailed cost report for TB-PRACTECAL.
METHODS
TB-PRACTECAL was an open-label, phase 2–3, multicentre randomised trial of all-oral regimens for the treatment of drug- resistant tuberculosis. Trial planning began in 2013 and work on publications continued into 2023. The trial took place in six sites across Belarus, South Africa, and Uzbekistan, and enrolled 552 patients. We analysed accounting data for the TB-PRACTECAL project, comprehensively including different costs, presented into 27 categories, by site, and by year, and at the per-patient level.
RESULTS
Total costs for TB-PRACTECAL were €33.9 million, of which 26% were at central level (costs incurred by the UK clinical trial team including trial planning, management, quality assurance, and analysis of results), while 72% were at the trial site level (across all six sites) and 2% were uncategorisable. At trial sites, the largest cost category was staff (43%), followed by external diagnostic services (11%), medicines (9%), other medical consumables (7%), external non-medical services (6%), and transport and travel (6%). Among medicines, the costliest were bedaquiline (46% of medicine costs), linezolid (16%), imipenem/ cilastatin (10%), and delamanid (9%). The mean cost per patient enrolled was €61,460 across the whole trial (including trial management overhead). When only site-level costs were considered, per-patient costs ranged between €19,998 and €45,942 across the six sites.
CONCLUSION
The costs of TB-PRACTECAL were similar to previously reported estimates for comparable clinical trials. However, TB- PRACTECAL included additional costs that would not typically be incurred in a commercial trial, such as investments in clinical research infrastructure and purchase of investigative medical products. To our knowledge, this is the first time MSF, or any other entity, published and analysed the disaggregated costs of a specific clinical trial. These data could help generate reliable predictions for future clinical trials and support planning and involvement, particularly in low-resource settings. Additionally, this study highlights the role of clinical trial cost disclosure in supporting both practical and policy discussions around the development of a more equitable system of biomedical R&D and fairer medicine pricing. Additionally, we developed a financial reporting template to facilitate future reporting of clinical trial cost by MSF and other entities investing in research.
Conference Material > Slide Presentation
Gotham D, Martin M, Barber M, Kazounis E, Batts C, et al.
MSF Scientific Day International 2024. 16 May 2024; DOI:10.57740/asasBXQ
Journal Article > CommentaryFull Text
Bull World Health Organ. 28 October 2019; Volume 97 (Issue 12); 851-853.; DOI:10.2471/BLT.18.228585
Elder K, Saitta B, Ducomble T, Alia M, Close R, et al.
Bull World Health Organ. 28 October 2019; Volume 97 (Issue 12); 851-853.; DOI:10.2471/BLT.18.228585
Vaccination is an effective intervention to reduce disease, disability, death and health inequities worldwide. Over the last two decades, vaccines have become more accessible in low-income countries; however, significant gaps remain, particularly in humanitarian emergencies, where populations face increased risks of many diseases. In 2013, the World Health Organization (WHO) published "Vaccination in acute humanitarian emergencies: a framework for decision-making," to provide guidance on which vaccines to prioritize during emergencies. However, substantial obstacles, especially high prices for new vaccines, hinder implementation of this framework and of critical vaccination activities in emergency settings.
In response to these challenges, global health stakeholders held a series of consultations in 2016 and proposed a WHO-based mechanism, the Humanitarian Mechanism, for the rapid procurement of affordable vaccines during emergencies, to be used by nongovernmental organizations (NGOs), civil society organizations, United Nations (UN) agencies and governments. Here we present the background of the creation of the mechanism from the perspective of Médecins Sans Frontières (MSF), including a description of our past challenges in accessing affordable pneumococcal conjugate vaccine (PCV), a critical vaccine during many emergencies. We then describe how the mechanism has so far facilitated access to more affordable PCV and outline steps that could increase its potential for saving lives.
In response to these challenges, global health stakeholders held a series of consultations in 2016 and proposed a WHO-based mechanism, the Humanitarian Mechanism, for the rapid procurement of affordable vaccines during emergencies, to be used by nongovernmental organizations (NGOs), civil society organizations, United Nations (UN) agencies and governments. Here we present the background of the creation of the mechanism from the perspective of Médecins Sans Frontières (MSF), including a description of our past challenges in accessing affordable pneumococcal conjugate vaccine (PCV), a critical vaccine during many emergencies. We then describe how the mechanism has so far facilitated access to more affordable PCV and outline steps that could increase its potential for saving lives.
Journal Article > Pre-PrintFull Text
bioRxiv. 1 February 2019; DOI:10.1101/533851
Saran K, Masini T, Chikwanha I, Paton G, Scourse R, et al.
bioRxiv. 1 February 2019; DOI:10.1101/533851
BACKGROUND
Tuberculosis (TB) poses a global health crisis requiring robust international and country-level action. Adopting and implementing TB policies from the World Health Organization (WHO) is essential to meeting global targets for reducing TB burden. However, many high TB burden countries lag in implementing WHO recommendations. Assessing the progress of implementation at national level can identify key gaps that must be addressed to expand and improve TB care.
METHODS
In 2016/2017, Médecins Sans Frontières and the Stop TB Partnership conducted a survey on adoption and implementation of 47 WHO TB policies in the national TB programs of 29 countries. Here we analyze a subset of 23 key policies in diagnosis, models of care, treatment, prevention, and drug regulation to provide a snapshot of national TB policy adoption and implementation. We examine progress since an analogous 2015 survey of 23 of the same countries.
RESULTS
At the time of the survey, many countries had not yet aligned their national guidelines with all WHO recommendations, although some progress was seen since 2015. For diagnosis, about half of surveyed countries had adopted the WHO-recommended initial rapid test (Xpert MTB/RIF). A majority of countries had adopted decentralized models of care, although one-third of them still required hospitalization for drug-resistant (DR-)TB. Recommended use of the newer drugs bedaquiline (registered in only 6 high-burden TB countries) and delamanid (not registered in any high-burden country) was adopted by 23 and 18 countries, respectively, but short-course (9-month) and newer pediatric regimens by only 13 and 14 countries, respectively. Guidelines in all countries included preventive treatment of latent TB infection for child TB contacts and people living with HIV/AIDS, but only four extended this to adult contacts.
CONCLUSION
To reach global TB targets, greater political will is needed to rapidly adopt and implement internationally recognized care guidelines.
Tuberculosis (TB) poses a global health crisis requiring robust international and country-level action. Adopting and implementing TB policies from the World Health Organization (WHO) is essential to meeting global targets for reducing TB burden. However, many high TB burden countries lag in implementing WHO recommendations. Assessing the progress of implementation at national level can identify key gaps that must be addressed to expand and improve TB care.
METHODS
In 2016/2017, Médecins Sans Frontières and the Stop TB Partnership conducted a survey on adoption and implementation of 47 WHO TB policies in the national TB programs of 29 countries. Here we analyze a subset of 23 key policies in diagnosis, models of care, treatment, prevention, and drug regulation to provide a snapshot of national TB policy adoption and implementation. We examine progress since an analogous 2015 survey of 23 of the same countries.
RESULTS
At the time of the survey, many countries had not yet aligned their national guidelines with all WHO recommendations, although some progress was seen since 2015. For diagnosis, about half of surveyed countries had adopted the WHO-recommended initial rapid test (Xpert MTB/RIF). A majority of countries had adopted decentralized models of care, although one-third of them still required hospitalization for drug-resistant (DR-)TB. Recommended use of the newer drugs bedaquiline (registered in only 6 high-burden TB countries) and delamanid (not registered in any high-burden country) was adopted by 23 and 18 countries, respectively, but short-course (9-month) and newer pediatric regimens by only 13 and 14 countries, respectively. Guidelines in all countries included preventive treatment of latent TB infection for child TB contacts and people living with HIV/AIDS, but only four extended this to adult contacts.
CONCLUSION
To reach global TB targets, greater political will is needed to rapidly adopt and implement internationally recognized care guidelines.
Journal Article > ResearchFull Text
Hepatol Commun. 1 October 2018; Volume 2 (Issue 10); 1160-1167.; DOI:10.1002/hep4.1247
Ford NP, Scourse R, Lemoine M, Hutin Y, Bulterys M, et al.
Hepatol Commun. 1 October 2018; Volume 2 (Issue 10); 1160-1167.; DOI:10.1002/hep4.1247
Successful treatment outcomes for chronic hepatitis B virus (HBV) infection requires high levels of adherence to treatment. We searched three databases and abstracts from two conferences up to January 2018 for studies reporting the proportion of patients who were adherent to HBV antiviral therapy and pooled data using random effects meta-analysis. We included 30 studies, providing data for 23,823 patients. Overall, adherence to treatment was 74.6% (95% confidence interval [CI] 67.1%-82.1%). Adherence was similar in high-income settings (75.1%; 95% CI, 65.4%-85.0%) and in low-income and middle-income settings (72.9%; 95% CI, 57.8%-88.0%). Reported barriers to adherence included forgetting, limited understanding of the importance of adherence, and change to routine.
CONCLUSION
There is a need to reinforce assessment and reporting of adherence as a routine part of HBV care and to assess the extent to which evidence-based interventions to improve adherence to medication for human immunodeficiency virus [HIV] and other chronic diseases are effective for HBV infection.
CONCLUSION
There is a need to reinforce assessment and reporting of adherence as a routine part of HBV care and to assess the extent to which evidence-based interventions to improve adherence to medication for human immunodeficiency virus [HIV] and other chronic diseases are effective for HBV infection.