#Health: Clinical trials, transparency and getting the message across

Transparency is a buzz word these days. We expect to see it in declarations by our politicians, company financial results, in science, and in decisions made on medicines and treatments by health-care bodies, writes European Alliance for Personalised Medicine Executive Director Denis Horgan.

The latter are usually based on clinical trial results regarding safety and efficacy, as well as, inevitably, cost. The European Medicines Agency, or EMA, is quoted as saying just over a year ago: “We…have set new standards for clinical trial data transparency by adopting two landmark policies.”

These were a 2010 policy on access to document and a 2014 policy on publication of clinical data for medicinal products for human use. These, say the agency, “demonstrate the EMA’s commitment to continuing on its path toward transparency, within the boundaries of its mandate and in the interest of public health”.

Writing in The New England Journal of Medicine, the authors (Sergio Bonini, Hans-Georg Eichler and Guido Rasi) stated that the policy on document access: “Enables interested parties to request data from clinical trials that have been submitted for marketing authorization of medicinal products.”

They added that this “represents the first step in implementing the principle of allowing the widest possible access to data while respecting the privacy of personal data and confidential commercial information that might be contained in a marketing-authorization dossier”.

In the following three years, there were 750 requests for document access, with the number increasing substantially each year, with just-over one quarter referring to clinical trial data. The largest number of requests came from the pharmaceutical industry (marginally above one-third) ), followed by law firms (17.5%) and journalists (15.9%). A mere 10% came from academics or research institutes, although more-than 40% of those referred to clinical trial data.

Only 5.5%, 1.5%, and 0.5% of requests came from the general public, patient bodies, and non-profit organizations, in that order. Being transparent, the EMA tells us that, in 2013: “Three cases were brought to the General Court of the European Union by pharmaceutical companies seeking annulment of decisions to grant access to clinical reports underpinning EMA marketing authorization for their products, claiming that the EMA violated the protection of confidential commercial information.”

Subsequently, two of these cases were withdrawn. Between September 2013 and June 2014, say the authors, “the EMA and pharmaceutical companies had differences of opinion on suggested redactions in almost half the documents to be released”. At the time of these statements, in late 2014, only one court case was still pending and no new cases had been initiated. The authors described this as “an encouraging sign of the (pharmaceutical) industry’s changing attitude toward transparency”.

The Brussels-based European Alliance for Personalised Medicine (EAPM) and its multi-stakeholder membership warmly welcome these developments, although admits surprise that so few patient organisations requested information. The same holds true for the general public and this must surely change. Personalised medicine is all about the patient, and knowledge is empowerment.

It is true that ‘“the final publication policy was developed with public input that stressed the need to protect both patient confidentiality and confidential commercial information and to avoid inappropriate use — especially commercial use — of data”.

And the authors stated that the EMA “tried to consider the various, often conflicting, positions taken by many different parties (and was) was guided by the conviction that public health interests must outweigh any private intellectual or commercial interest”.

However, while the EMA emerges with considerable credit, perhaps more patients and patient advocates should know about just how transparent this information actually is. The agency has its own ideas: “The media also play a key role and need to provide accurate information gleaned from documents they receive from the EMA.

We believe that broader use of clinical trial data by academia and research institutes should also be promoted, since methodologically sound and unbiased reanalyses of data may advance science and help regulators review their decisions, as appropriate.”

Stanimir Hasurdjiev, board member of the European Patient Forum and the Patient Access Partnership, has previously emphasized that more and more patients’ groups and individual citizens are becoming aware of the potential of personalised medicine, with its ability to give them the right treatment at the right time. He said that patients want empowerment, they want to have their illnesses and the treatment options explained in a transparent, understandable manner to allow them to become involved in co-decision. This would give them greater access to treatments that could improve their lives and, in some cases, save them.

The EMA has played its part, now it’s time to get the message out.

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