Conference Material > Abstract
Gotham D, Martin M, Barber M, Kazounis E, Batts C, et al.
MSF Scientific Day International 2024. 2024 May 16; 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.
Journal Article > CommentaryFull Text
BMJ Glob Health. 2022 April 19; Volume 7 (Issue 4); e007490.; DOI:10.1136/bmjgh-2021-007490
Perrin C, Athersuch K, Elder G, Martin M, Alsalhani A
BMJ Glob Health. 2022 April 19; Volume 7 (Issue 4); e007490.; DOI:10.1136/bmjgh-2021-007490
Two drugs with novel mechanisms of action, the diarylquinoline bedaquiline and the nitroimidazole delamanid—as well as pretomanid from the same class of drugs as delamanid—have recently become available to treat drug-resistant tuberculosis (DR-TB) after many decades of little innovation in the field of DR-TB treatment. Despite evidence of improved efficacy and reduced toxicity of multidrug regimens including the two agents, access to bedaquiline and delamanid has been limited in many settings with a high burden of DR-TB and consistently poor treatment outcomes. Aside from regulatory, logistic and cost barriers at country level, uptake of the novel agents was complicated by gaps in knowledge for optimal use in clinical practice after initial market approval. The main incentives of the current pharmaceutical research and development paradigm are structured around obtaining regulatory approval, which in turn requires efficacy and safety data generated by clinical trials. Recently completed and ongoing clinical trials did not answer critical questions of how to provide shorter, less toxic treatment DR-TB treatment regimens containing bedaquiline and delamanid and improve patient outcomes. Voluntary generation of evidence that is not part of this process—yet essential from a clinical or policy perspective—has been left to non-sponsor partners and researchers, often without collaborative efforts to improve post-regulatory approval access to life-saving drugs. Additionally, these efforts are currently not recognised in the value chain of the research and development process, and there are no incentives to make this critical research happen in a coordinated way.
Journal Article > CommentaryFull Text
BMJ Glob Health. 2022 July 15; Volume 7 (Issue 7); e009709.; DOI:10.1136/bmjgh-2022-009709
Perehudoff K, 't Hoen E, Mara K, Balasubramaniam T, Abbott F, et al.
BMJ Glob Health. 2022 July 15; Volume 7 (Issue 7); e009709.; DOI:10.1136/bmjgh-2022-009709
SUMMARY BOX
⇒ The COVID-19 pandemic highlighted how current international laws and practices fail to ensure medical countermeasures (i.e., vaccines, therapeutics, diagnostics and personal protective equipment) are
equitably distributed in a global health crisis.
⇒ In 2021, the 194 Member States of the World Health Organization agreed to begin negotiations towards an international instrument that would better position the world to prevent, respond and prepare for future pandemics (often called a ‘pandemic treaty’.)
⇒ A pandemic treaty presents an opportunity to address these challenges in international law, and craft a better system, based on solidarity, for the global development and distribution of medical countermeasures.
⇒ We recommend that a pandemic treaty ensure sufficient financing for biomedical research and development (R&D), creates conditions for licensing government-funded R&D, mandates technology transfer, shares intellectual property, data and knowledge needed for the production and supply of products, and streamlines regulatory standards and procedures to market medical countermeasures.
⇒ We also recommend that a pandemic treaty ensures greater transparency and inclusive governance of these systems.
⇒ The aim of these components in a pandemic treaty should be to craft a better collective response to global health threats, consistent with existing international law, political commitments and sound public health practice.
⇒ The COVID-19 pandemic highlighted how current international laws and practices fail to ensure medical countermeasures (i.e., vaccines, therapeutics, diagnostics and personal protective equipment) are
equitably distributed in a global health crisis.
⇒ In 2021, the 194 Member States of the World Health Organization agreed to begin negotiations towards an international instrument that would better position the world to prevent, respond and prepare for future pandemics (often called a ‘pandemic treaty’.)
⇒ A pandemic treaty presents an opportunity to address these challenges in international law, and craft a better system, based on solidarity, for the global development and distribution of medical countermeasures.
⇒ We recommend that a pandemic treaty ensure sufficient financing for biomedical research and development (R&D), creates conditions for licensing government-funded R&D, mandates technology transfer, shares intellectual property, data and knowledge needed for the production and supply of products, and streamlines regulatory standards and procedures to market medical countermeasures.
⇒ We also recommend that a pandemic treaty ensures greater transparency and inclusive governance of these systems.
⇒ The aim of these components in a pandemic treaty should be to craft a better collective response to global health threats, consistent with existing international law, political commitments and sound public health practice.
Conference Material > Slide Presentation
Gotham D, Martin M, Barber M, Kazounis E, Batts C, et al.
MSF Scientific Day International 2024. 2024 May 16; DOI:10.57740/asasBXQ