How much do clinical trials cost? The answer is overdue.
Today MSF is publicly announcing its Clinical Trial Transparency Policy (CTTP). Transparency is one of MSF’s five working principles, alongside impartiality, independence, neutrality and bearing witness, which are all key to support the delivery of our medical humanitarian mission.
MSF is involved in a number of clinical trials, and as such, the MSF CTTP commits to publishing research protocols, registering clinical trials and publishing data in open access formats. In addition, in a landmark step, the MSF CTTP commits to publishing a minimum set of cost items for these trials. This demonstrates MSF’s recognition and commitment that cost transparency around clinical trials is a critically important step towards increasing equity in the biomedical research and development (R&D) system, and access to medical products for all who need them.
This comes at a time when there is increasing political momentum and discussion around clinical trials, presenting an opportunity for governments to prise open the black box of pharmaceutical R&D costs and to require the publication of the costs of publicly funded clinical trials.
To coincide with the policy announcement, and to capitalise on the growing interest in how transparency can support better access, MSF is hosting the second roundtable in a series titled, “How can disclosing clinical trial costs increase access to medical products?”
The fundamental necessity of ensuring high-quality, well-designed clinical trials in the biomedical R&D process was made painfully clear during the COVID pandemic. The pandemic saw a proliferation of clinical trials in a very short space of time, the vast majority of which delivered unusable data. The World Health Organization (WHO) recently estimated during a public consultation related to clinical trials that only a small proportion of clinical trials run during COVID produced data that went on to inform WHO policy. These trials, such as the RECOVERY trial, became the bedrock of the global response, without which the discovery of safe and effective treatments and vaccines would not have been possible.
Of course, clinical trials have played an essential role in the discovery of critical treatments and vaccines in modern medicine, and yet the question of how they could be done better in order to improve public health outcomes has not had the attention it deserves — until now.
The clinical trial failures during the COVID pandemic spurred many into action to reform clinical trial policies, both within the context of a pandemic but also more broadly. Central to these efforts is the WHO Clinical Trial Resolution, adopted at the World Health Assembly (WHA) this year, which aims to strengthen the coordination of clinical trials, to improve quality, and to encourage transparency and timely publication of data. The ongoing WHO Pandemic Treaty negotiations are also an opportunity to recognise the central importance of clinical trials to any pandemic response.
However, there is one critical piece of information that is still neglected in policy discussions: how much clinical trials cost. This has not featured in these discussions so far, despite calls to do so.
Critically, there is currently very little available data on the true costs of clinical trials. Estimates for full R&D costs for the development of new drugs (for which clinical trials are usually the most expensive part), range from US$43.4 million to US$4.2 billion, depending on the methodology used. This range varies by 100-fold, and only serves to demonstrate how important it is for us to be able to establish the true costs of R&D, including clinical trials.
Why does transparency around clinical trial costs matter?
Access to information about the costs of clinical trials is vitally important for five key interrelated reasons:
- Informing policy development and evidence-based decision-making: A lack of information about clinical trial costs hinders the creation of reliable cost estimates and thus allocation of proportionate funds for R&D, especially critical in low-resource settings. Transparency on these costs would therefore help inform decision-making around R&D investments.
- Promoting innovation, especially from non-traditional actors: The high-end estimates of R&D and clinical trial costs can act as a deterrent to some actors and governments considering conducting clinical trials, especially in low-resource settings. Access to data about how much they actually cost would therefore help incentivise and support the development of these initiatives — in turn, this would increase equity in the biomedical R&D ecosystem.
- Price negotiation: Sky high prices for medicines, vaccines and diagnostics are often justified by the pharmaceutical industry through claims that it costs them billions in R&D investment to bring them to market, and the subsequent “need” to “recoup” these costs. There is however clear evidence this is may not be the case, with research demonstrating that there is in fact little link between R&D costs and prices. Since clinical trials are widely accepted as the most expensive part of the R&D process, more balanced access to information around how much they cost would increase the ability of governments and treatment providers to challenge industry claims of the need to recoup high R&D costs, and thus aid them in regulating and negotiating medical-product prices more effectively.
- Accountability and maximising impact of public funding: Where public funding is invested in clinical trials, the costs of these trials should be made publicly available. There is increasing evidence that it is possible for clinical trials to be both high quality and low cost, thus maximising public return on public investment, when supported by accountability and transparency. Cost transparency would also support governments and other funders to set appropriate and proportional incentives and financing models for the delivery of clinical trials.
- Access to medical products: Overall, the impacts of increased transparency of clinical trial costs would promote more diverse involvement in clinical trials, as well as support better access to the final products. This would both expand research and knowledge development into different areas of public health, whilst also ensuring access is a central consideration in these initiatives.
MSF publishes first Clinical Trial Transparency Policy
MSF has been involved directly in a number of clinical trials, including the endTB and TB PRACTECAL clinical trials, both aiming to evaluate the safety and efficacy of new treatments and treatment durations for tuberculosis (TB), with groundbreaking results.
Now, MSF is taking steps to publish its clinical trial costs through committing to publishing a minimum number of key data points related to the cost of clinical trials, wherever pragmatic and possible. This commitment is captured in the MSF CTTP announced this week. Efforts are now underway to consider how best to collect and publish accurate information about the costs of these trials. As a first step towards implementing the CTTP, we hope to publish the costs of the TB PRACTECAL clinical trial, while also developing some best-practice guidelines to inform future publication of MSF clinical trial costs.
Transparency initiatives are multiplying
More broadly, since a large proportion of clinical trials receive public funding, all governments can and should take steps to ensure that the costs of any publicly funded clinical trials are made publicly available. The US government, for example, is the largest global funder of biomedical R&D. Concerned about high drug prices and spurred by evidence suggesting pharmaceutical companies exaggerate their R&D spending, US lawmakers have recently taken action towards mandating greater transparency of clinical trial costs. Members in both chambers of US Congress introduced legislation that would require pharmaceutical corporations and other drug developers to disclose the costs of future clinical trials in a public, searchable cost data repository linked to a website where trial results are currently published (ClinicalTrials.gov). The bill would also require drug manufacturers to include detailed clinical trial cost information in the publicly available financial reports that all publicly traded companies are required to file each year with the US government.
Legislation is also beginning to emerge in a number of other countries with a focus on disclosure of R&D costs. In South Africa, information about clinical trial costs are collected through the South African Clinical Trial Registry, but further steps are needed to ensure this information is made publicly available. As part of these efforts, it is critical to ensure that clinical trial cost transparency should include the sharing of information about the different costs incurred by different actors in different stages of the clinical trial process. This would help shed light on the relative financial contributions of each, expose the most costly aspects, inform the debate around fair pricing of products, and support efforts to ensure a reasonable return on public investments.
Disclosure of clinical trial costs is a critical piece of any attempt to strengthen the clinical trials ecosystem, both domestically and through WHO-led initiatives. Increased access to this information brings real opportunities to inform policy development and decision-making, strengthen the clinical trial landscape and participation, ensure accountability, and support efforts to increase access to affordable medical products. Given the fundamental importance of clinical trials in discovering safe and effective medical products, it is simply not justifiable that we don’t know how much they cost. Access to this information is long overdue.
The authors:
Roz Scourse, Policy Advisor, MSF Access Campaign
Ava Alkon, Policy Advisor, MSF USA
Candice Sehoma, Advocacy Advisor, MSF Access Campaign
Dr Bern-Thomas Nyang’wa, MSF Medical Director, and Chief Investigator, TB-PRACTECAL
For more information about our activities to increase access to medicines, visit our website msfaccess.org
