How safe are our drugs?

Clinical trials are key to modern medicine: these studies evaluate both how effective and safe new medical treatments are. By conducting trials and publishing results, scientists test new treatments and allow others to learn from and build upon their research.

However, clinical trials are only useful to science if their results are published – that includes trials where results reflect badly on the drug or medical device that is being produced by the company paying for the trial.

In the EU, regulations require the results of drug trials to be published – but worryingly, these regulations aren’t being enforced. This is slowing down the development of new treatments and cures, and contributing to the mounting costs of healthcare.

Every year, $85 billion of the money spent globally on health and medical research is being wasted because of this failure to publicly report findings. In fact, a recent study showed that 89% of applicable trials run by European universities violate the EU’s own regulations because they are missing results on the public registry.

While there is widespread non-reporting of clinical trials, those with positive results – when a drug appears to work — are more likely to be published. In contrast, the results of trials that show drugs to be ineffective or to have dangerous side effects, are far less likely to be published.

Worryingly, this means that new, expensive drugs can appear to be far more effective and safe than they really are. As a result, public health systems, private insurers and individual patients are often paying for expensive drugs when cheaper, generic alternatives would be a better treatment option.

For example, a 2008 study of antidepressant drugs highlighted the large gap between the overall evidence generated by clinical trials, and the evidence that made its way to doctors and patients. Many of the trials showing the drugs to be ineffective never saw the light of day, while others were presented as having been successful. 

The human cost of this lack of effective regulation is staggering. One clinical trial identified the deadly side effects of Lorcainide – a drug for heart disease already on the market – but the results were not made public. Tens of thousands of people using the drug died from unrecognised side effects.

In the UK, the NHS wasted £424 million on the drug Tamiflu, unaware that unpublished trial results had found it to be totally ineffective. Even today, the NHS remains unable to accurately assess the benefits and harms of drugs and medical devices because trial reporting regulations remain unenforced.

This is the kind of deregulation by stealth that we should be challenging, to ensure that the protections we need to put our safety, health and wellbeing are actually put into place.

This disregard for public health regulations has not gone unnoticed. At EU level, health groups have called to ensure that Horizon Europe — the €100 billion research funding programme — incorporates best practices in clinical trial transparency. Similar calls have been made in the UK too. In October 2018, the UK Science and Technology Committee of the House of Commons called on the government to ensure that all UK clinical trials fully report their results.

Here, the Committee proposed a clear and simple solution. The Health Research Authority — the NHS body overseeing health research — should monitor whether clinical trial results are published within the 12 month deadline outlined by the World Health Organisation, imposing fines on those that fail to do so.

In this respect, the US is already miles ahead. Pharmaceutical companies and universities already face fines of over $11,000 for each day that results are not made public, and it seems the US government is beginning to take its duty to enforce its own rules seriously.

So, how can the problem be fixed? A new piece of EU clinical trials regulation, designed to protect patients and promote public health, comes into force in 2019 – 2020. It requires that the results of drug trials are reported within 12 months. However, unless action is taken at both the European and national levels, little, if anything, will improve.

At the EU level, the European Medicines Agency currently does not even send out reminder emails to those who break its rules, despite regulation requiring it to do so. Still, many completed trials are incorrectly listed as ​‘ongoing’ on the European registry, making it hard to determine who is breaking the rules, undermining attempts to improve compliance. 

Furthermore, under the regulation, it is individual member states who are responsible for enforcing the rules. However, none of the national governments have adopted plans to do this. Unless national governments start imposing fines as the UK is now considering doing, this eminently common-sensical rule will remain a paper tiger – and patients and taxpayers across Europe will continue to pay a steep price.

Proponents of broader processes of de-regulation often claim that deregulation will automatically improve efficiency and save money. In the case of clinical trial reporting, this is most certainly not the case. An important side effect of the deregulatory culture sweeping across Europe is the development of a culture where existing regulations are not enforced or are simply ignored. This is the kind of deregulation by stealth that we should be challenging, to ensure that the protections we need to put our safety, health and wellbeing are actually put into place. 

NEF are working with partners across Europe to halt the EU’s deregulation drive, and ensure that effective regulations are enforced in protection of the public interest. To find out more about the project see here.

TranspariMED is a global initiative that develops and promotes policy​solutions to the problem of evidence distortion in medical research.