At first glance, it seems that clinical trials are the much-criticized pharmaceutical industry’s best contribution to the medicines process. The industry leans on governments to fund early-stage research, then claims the patent rights to the most promising fruits of that research. That arrangement frustrates many public health advocates, especially since it enables the industry to collect more in profits, and invest more in marketing and lobbying, than it spends on its own research.
But the industry does fund the majority of clinical trials of medicines. Clinical trials are research on human subjects—from Phase One testing on small numbers of people to Phase Three trials that involve large-number studies and are the final step before seeking regulatory approval. Economist Marianna Mazzucato notes that the private industry’s involvement at the late stages of the research process after public-sector funding during the less-promising early stages allows companies to “hop on for the easier ride down the way.” But clinical trials are an undeniably expensive stage of the process, and they do pose some financial risk for the companies.
So, with all the well-documented ways that the profit motive can exert a toxic influence on public health, are clinical drug trials actually an activity where the private sector makes a positive contribution? I argue the answer is no.
The most glaring problem with for-profit sponsorship of clinical trials is the inherent risk of bias when research is funded by a company that is highly motivated to sell the medicine being tested. It is well-documented that industry funding colors reported research results. One particularly disturbing example was the delayed reporting of the dangers of erythropoetin-stimulating agents (ESAs), aimed to treat anemia in cancer patients. Early industry-sponsored research in the 1990s showed benefits of ESAs, but later reports by independent researchers found the drugs significantly increased the risk of patient death. None of the industry-sponsored research reported these major problems, but 90% of studies not funded by pharmaceutical corporations did.
When reporting his 2015 analysis showing that private industry is sponsoring an increasing proportion of clinical trials, Stephen Ehrhardt of the Johns Hopkins Bloomberg School of Public Health explained why this trend is troublesome. “(W)hen I am a drug company testing my new product, my objectivity can be compromised by the company’s bottom line since it costs me millions of dollars to develop and test my product to get it on the market,” Ehrhardt said. “It might be difficult for me to be completely objective. The stakes are very high.”
There are other problems, too. Industry-sponsored clinical trials typically compare the company’s drug only to a placebo, rather than to a similar medicine. That approach lines up with the much-criticized “better than nothing” US FDA standard for drug approval. But setting such a low bar means the studies’ results do not reveal whether new medicines are an improvement over existing therapies, the information that would be most valuable to clinicians.
That placebo-comparison protocol for clinical trials also creates a platform for private companies to test and then market drugs that simply mimic existing treatments. Those so-called “me-too” drugs are developed not to improve care, but to enable companies to have a share of a lucrative market. Recent studies suggest that as many as 70% of approved drugs fall into this “me-too” category—wasting billions of dollars on unnecessary clinical trials.
To some physicians, including Ben Goldacre, author of Bad Pharma: How Drug Companies Mislead Doctors and Harm Patients, the most concerning problem with industry-funded clinical trials is the widespread failure to share trials results that are not positive for the company. “For me, missing data is the key to this whole story,” Goldacre writes. “(I)t poisons the well for everybody. If proper trials are never done, if trials with negative results are withheld, then we can simply not know the true effects of the treatments we use . . . With missing data, we are all in this together, and we are all misled.”
Finally, industry sponsorship of clinical trials can lead to extended price-spiking monopolies on essential medicines. In the debates over the Trans-Pacific Partnership Agreement and other international trade deals, public health advocates have heavily criticized proposed and existing terms for exclusivity, which ban the use of privately-obtained clinical test data to support approval of generic alternatives. This data exclusivity acts as a barrier that extends beyond patent terms to block patients in low-income countries from accessing affordable essential medicines.
Fortunately, there is an obvious and far better alternative to this broken model. The clinical trial stage of drug research should be fully publicly funded in the same way that governments already support basic science research. Public sector funding and management of clinical trials is essential because medicines research is a long-acknowledged public good, and access to the fruits of that research is a human right and a moral imperative. This is hardly a new idea: physicians, economists, attorneys, and public health experts like Tracy Lewis, Jerome Reichman, Anthony So, and Dean Baker have been calling for a publicly-funded clinical trials system for more than a decade.
An immediate benefit from such a system would be transparency of trial results. Full disclosure of the data will allow clinicians to make informed choices about the best therapy for their patients. That clinical benefit will be compounded by the fact that publicly-funded trials, freed from the distortions of profit-seeking, can compare new drugs versus existing ones—not just a placebo. Transparency also bypasses the negative influence of the private quest for patent monopolies, enabling the kind of follow-on research that has proven to be transformational in the open-source software movement. The prospects for enhanced follow-on research are a chief reason why some researchers and medical journal editors are already calling for greater disclosure of clinical trials data.
It is hard to argue against the many benefits of removing the pharmaceutical industry fox from its role guarding the clinical trials henhouse. But there is the question of how to replace the private dollars that fund the current model. The answer to that question can be found in the massive public subsidies that already undergird the current for-profit medicines system, albeit in a decidedly inefficient manner.
In an earlier blog post, I summarized the analysis of economist Dean Baker, who calculates that removing government-provided monopoly patent subsidies for medicines would generate enough savings to more than replace every dollar of private research funding. Baker and others have also argued that financing of a public clinical trials system can be obtained by removing the estimated 40% premium paid by the United States due to its voluntary ban on negotiating Medicare drug prices. Further savings could come if the United States finally exercised long-dormant rights to license generic manufacture of drugs discovered with US funding.
There are different ways to manage the mechanics of a publicly-funded clinical trials system. Existing or new public agencies could develop the capacity to directly conduct the trials themselves, or governments could contract out the research to independent, accountable entities. Whatever approach is used, there would be immediate and substantial savings flowing from the abandonment of multiple for-profit inefficiencies. A publicly-funded trials system would not be distracted by clinically irrelevant “me too” drug research, or phase IV clinical trials that are focused more on marketing than on uncovering useful information. The public system would embrace the sharing of data and coordination of research that brings significant cost savings as well. A 2016 report by the Wellcome Trust estimated that a clinical trial network for new antibiotics could reduce overall costs by as much as 60%.
Even among the notoriously market-oriented US public, there is increasing recognition that the pursuit of profits has a toxic influence on healthcare. Corporate sponsorship of clinical drug trials is no exception to that rule. The model needs to be replaced by a publicly-funded and managed system that will provide more impactful, transparent, and efficient results.
Fran Quigley is a clinical professor and director of the Health and Human Rights Clinic at Indiana University McKinney School of Law, and a coordinator of People of Faith for Access to Medicines. His book, Prescription for the People: An Activist’s Guide to Making Medicines Affordable to All will be published by Cornell University Press this fall.