Imagine you’re in a foreign country. You don’t speak the language. The symbols on the signs look familiar, but you don’t understand what they mean. You need to get out of this place and quickly. Finding an escape could be the difference between life and death.
Unfortunately, this is how many patients with rare diseases feel when reading clinical trial summaries. Without a scientific or medical background, clinical trial data can come across as a confusing mix of letters and numbers. This is frustrating for patients, because they don’t know if the drug has shown promise or if it’s back to the drawing board.
In 2007, President George W. Bush signed the U.S. Food and Drug Administration Amendments Act into law. A provision of this bill mentioned the possibility of disseminating a “summary of the clinical trial and its results that is written in non-technical, understandable language for patients.”1 While not a mandatory regulation, the biopharma industry certainly noticed this strong suggestion.
The EU followed suit in 2014, with the introduction of the more enforceable “EU Clinical Trials Regulation 536/2014/Article 37”. This regulation required plain language summaries of clinical trial data to be accessible in a new database known as the “EU Clinical Trials Register,” which is expected to be available in late 2018.2
While European companies are obligated by law to provide easily understood summaries of clinical trial data, it would be wise for biopharma companies based in the U.S. to take the same action. Offering plain language summaries would be a major boost for any company claiming to be “patient-centric” in the eyes of patients, critics, and other stakeholders.
Plain language summaries may give patients a better sense of control and understanding, in turn reducing anxiety and alleviating fears of the unknown, even if the data are not quite what they were hoping for.
Patient organizations can also benefit from plain language summaries. By allowing these groups the opportunity to fully digest the results of a clinical study, they are then better able to design their advocacy strategies.
Data transparency has also proven to be an especially high concern of a very specific subset of the patient population: the trial participants. In a study published by the Center for Information & Study on Clinical Research Participation (CISCRP), survey results showed that a whopping 91% of respondents felt that receiving a study summary after participation would be beneficial. Despite this desire, however, only 47% had ever received such a summary.3 Correcting this discrepancy would be an incredibly meaningful method for any pharmaceutical company to openly demonstrate a commitment to embodying a patient-centric worldview.
It may require some extra work for a biopharma company to produce a plain language summary, but the benefits vastly exceed the cost; it involves patients in the drug development process and builds a better understanding of rare diseases and their treatment across all stakeholders.
1NIH U.S. National Library of Medicine. FDAAA 801 and the Final Rule. https://clinicaltrials.gov/ct2/manage-recs/fdaaa. Accessed May 11, 2018.
2 European Medicines Agency. About the EU Clinical Trials Register. https://www.clinicaltrialsregister.eu/about.html. Accessed May 11, 2018
3 The Center for Information & Study on Clinical Research Participation. Public and Patient Perceptions & Insights Study. http://www.ciscrp.org/services/research-services/public-and-patient-perceptions-insights-study/. Accessed May 11, 2018.