Drug trials: publish and be damned

The headline in the British Medical Journal in October 2010 could barely have been starker: "Patients and doctors are being misled by published data on medicines".

The study it referred to, by a group of researchers at the German Institute for Quality and Efficiency in Health Care, argued that the drug reboxetine was, overall, an ineffective and potentially harmful antidepressant.

Even more worrying for the pharmaceutical industry, it concluded that nearly three-quarters of the data on patients who took part in trials of reboxetine had not been published until then, and that published data on the drug had overestimated the benefits and underestimated the harms of treatment, "all underlining the urgent need for mandatory publication of all clinical trial results".

So, was this a clear-cut case of 'nasty Big Pharma' trying to pull the wool over the eyes of an unwitting public and, in the process, making a fast buck? Absolutely not, says Christine-Lise Julou, director, scientific and regulatory affairs at the European Federation of Pharmaceutical Industries and Associations (EFPIA): "It's not so much about people withholding information, it's about an increased appetite for information. This has to be recognised and acknowledged."

Nevertheless, for the EFPIA and other organisations, such as the International Federation of Pharmaceutical Manufacturers & Associations, the Japan Pharmaceutical Manufacturers Association and the Pharmaceutical Research and Manufacturers of America, this creates a potential headache.

Clearly, although pharmaceutical companies want and need to respond to this new climate of transparency as best they can, an information free-for-all would have serious ramifications, not only for issues around commercial confidentiality, but also in terms of ensuring the public get accurate and robust information about trial results and the efficacy of the drugs coming through R&D pipelines.

Between them, the four organisations have published a number of joint position papers, and continue to monitor how the landscape is evolving in regards to this area.

Great expectations: industry and regulators

Legislation in Europe and the US has been in place for some time, but the environment has evolved in the past 20 years or so, as have the expectations of patients and consumers, according to Julou.

"People used to be quite happy for the benefit and risk assessment of drugs to be left to the regulators," she says. "The industry and regulators were, as a result, not very transparent and did not particularly need to be. They were not required to give a huge amount of detail on everything. But expectations have changed, and people want to be better informed."

Another issue for industry sponsors is the complexity of seeking to publish results of clinical trials in peer-reviewed scientific literature, while needing to place information on multiple databases worldwide. Finding the appropriate information on these multiple databases and literature sources can be complex. Yet Julou feels that the regulations already in place are both robust and adequate; for example, in Europe there are already public databases and procedures in place to populate them.

"The regulations are already very broad and do not need to be tougher," she says. "It is important to respect commercial confidentiality and to not have a negative impact on the pharmaceutical R&D that delivers new medicines for patients. We would have some concerns if there was a proposal to make all information on trials available before a product has been authorised for public use - there is no reason for that.

"While we support enhanced transparency within clinical trials information, it is important to maintain privacy for personal data and protection of commercial and contractual interests so as not to undermine the incentive for research," she adds.

Support and accuracy for clinical trials

There are important clinical benefits associated with making clinical trial information more widely available, says Julou, but information shouldn't just be available on public websites; it also needs to appear in peer-reviewed journals in order to facilitate proper scientific discussion.

The information that the public can access needs to provide an accurate report of the trial findings, including adverse events. However, there are concerns that if this information isn't put into context, or is viewed by individuals without the appropriate knowledge and experience to understand it, it could be misinterpreted.