Big Pharma cannot justify the exorbitant prices charged for lifesaving medications, which are often attributed to research and development expenses, points out MSF, calling for honesty and disclosure within the industry.
In Nukus, Uzbekistan, 34-year-old surgical nurse Dilaram was diagnosed with drug-resistant tuberculosis (DR-TB). But instead of facing the standard treatment, including nearly 15 000 pills to be taken over two years and painful injections causing severe side effects, she could enrol in TB-PRACTECAL, the MSF-led clinical trial testing an all-oral, six-month regimen for DR-TB.
After completing treatment with virtually no side effects, she returned to work and to caring for her two young daughters. This trial transformed her TB treatment journey and recovery.
This profound revolution in her treatment journey is not a coincidence, write Farhat Mantoo & Bern-Thomas Nyang’wa for Health Policy Watch.
Rather, it is a result of years of dedicated clinical research, shaped by experiences of people like her and driven by significant contributions and efforts from public and non-profit organisations working closely with people affected by TB in low-resource settings.
TB-PRACTECAL, a landmark clinical trial led by Médecins Sans Frontières (MSF), not only identified an all-oral, six-month regimen for DR-TB, but it is also the first clinical trial for which the detailed costs, €33.9m, were published in the journal PLOS Global Public Health.
This stands in stark contrast to the opaque norms of pharmaceutical research and development (R&D), where there is no transparency about what it costs to develop new medicines although high drug prices are often justified based on high R&D costs.
This moment is more than a medical milestone. It marks a critical step toward accountability in medical innovation, and demonstrates to all stakeholders that transparency in R&D is both possible and essential.
When costs are hidden, governments – and organisations like MSF that purchase medical products – lose the leverage to negotiate affordable prices.
We therefore ask: what does the pharmaceutical industry have to hide? If their costs are truly high, why not publish them? They refuse because transparency would undermine their ability to charge whatever prices they like, even for lifesaving TB medicines.
Costs of TB-PRACTECAL clinical trial
TB-PRACTECAL was a phase 2b-3 adaptive trial, which means it was designed to test how well different treatments work and confirm those results in a larger group of people, while also adapting the study design over time based on early results.
The trial tested three new regimens for DR-TB against the standard of care. The total cost of €33.9m was further broken down into 27 cost categories, enabling a detailed analysis of the key cost drivers of the trial.
Current estimates for the full R&D costs of developing a new drug range from €40m to €3.9bn, depending on the methodology used. Estimates for phase 2 and phase 3 pharmaceutical clinical trials alone range between €4.7m and €133m.
While the overall cost of TB-PRACTECAL fits within this range, several factors pushed its cost higher. These include the fact that it was both a phase 2b and phase 3 trial combined; it included multiple sub-studies; ran for a long duration of five years, and required significant investment in health facilities and infrastructure to carry out the trial.
Medicine costs were also high. The cost of bedaquiline alone made up 46% of all medicine expenses.
Why transparency matters
The TB-PRACTECAL trial was conducted in resource-limited settings in South Africa, Uzbekistan, and Belarus – countries with a high prevalence of DR-TB. In 2022, the trial confirmed that a shorter, all-oral regimen using bedaquiline, pretomanid, linezolid, and moxifloxacin (BPaLM) could treat patients in six months or less, with significantly higher cure rates and fewer side effects, compared with the previous 18–24 month regimens that relied on painful injections and caused severe side effects.
After publication of the trial results, the World Health Organisation (WHO) recommended the regimen as the preferred treatment for rifampicin-resistant TB.
It has since been adopted for use in 40 countries. Sustained advocacy by TB activists and MSF played a crucial role in pushing for price reductions of key newer drugs, including bedaquiline and pretomanid, making broader rollout of this regimen possible.
Historically, pharmaceutical companies have justified sky-high drug prices under the pretext of recouping R&D expenses without ever disclosing the actual costs. For life-threatening diseases like TB, this secrecy costs lives.
MSF and TB activists witnessed this with bedaquiline, a core drug that is now an essential part of all DR-TB regimens. Although it was developed with substantial public funding, Johnson & Johnson charged an exorbitant price, citing the need to recoup high R&D costs and sustain future innovation.
Academic research showed that public investment in bedaquiline was up to five times greater than private investment. Civil society and health advocates used this information to successfully push for a price reduction.
This transparency marked a turning point in the fight for access to this medicine and demonstrated the critical role of R&D cost transparency in enabling affordable access. The justification of high prices due to high R&D costs is a recurring argument from the pharmaceutical industry that MSF has encountered repeatedly when addressing the high cost of lifesaving medicines.
This can no longer remain an evidence-free zone, which is why MSF chose to play its part by publishing the detailed cost breakdown of the TB-PRACTECAL trial.
We are calling on all funders and implementers of clinical trials – governments, product-development partnerships (PDPs), philanthropic organisations, academics, institutions, and pharmaceutical companies – to publicly disclose their R&D costs.
MSF has developed a Clinical Trial Cost Reporting Toolkit to support publishing clinical trial costs, building on our own experiences.
Medicines shouldn’t be a luxury
Every year, 1.3m people die of TB, making it the world’s deadliest infectious disease, disproportionately affecting people in low- and middle-income countries.
Countries like India, which bear the world’s largest burden of TB and DR-TB, need timely access to the latest WHO-recommended treatments at affordable prices. However, exorbitant pricing has long stood in the way of widespread implementation of these regimens.
Without generic competition, national TB programmes struggle to widely implement updated WHO guidance on treatments that rely on newer, and often more expensive drugs.
The global community cannot afford another delay like the one we saw with bedaquiline, where people with DR-TB in low- and middle-income countries were left behind for more than a decade. To truly end TB, we need to dismantle the barriers of cost, control, and corporate secrecy that continue to undermine access.
What needs to change
At MSF, we do not accept funding from pharmaceutical companies. From the front lines of conflict to work done with communities battling epidemics, our work is rooted in compassion, equity, and medical ethics.
We took a big step to openly share our trial costs because we believe that when public resources are used, the public deserves accountability.
We urge the global health community to support transparency in medical R&D. We ask all stakeholders to recognise that transparency is not a threat but a lifeline. Without it, access to care remains a privilege for the few; with it, it becomes a shared responsibility to protect the many.
Six years ago, the World Health Assembly adopted the transparency resolution that urges all member states to “take appropriate measures to publicly share information on the net prices of health products”.
All governments must take urgent steps to enact legislation mandating the disclosure of disaggregated R&D costs, including clinical trial costs, especially where the R&D has received public funding.
We will keep speaking out against systems that put profits ahead of people – because no one should be left to suffer, or die, from a disease that could be treated, simply due to hidden costs.
Secrets cost lives.
Farhat Mantoo is the Executive Director of Médecins Sans Frontières (MSF) South Asia, with more than two decades of leadership experience in humanitarian operations across Asia, Europe, and East Africa.
Bern-Thomas Nyang’wa is the Medical Director of Médecins Sans Frontières (MSF) Netherlands. He was the Chief Investigator of TB-PRACTECAL and has extensive TB clinical, programmatic and research experience.
See more from MedicalBrief archives:
Can Big Pharma justify hefty drug prices?
J&J price probe dropped after TB drug costs slashed
Health costs force TB patients deeper into poverty
J&J patent deal allows for cheaper generic TB drugs, but SA may lose out