Pioneering large-scale outcomes trials in cardiovascular disease

Cardiovascular disease (CVD) is the number one killer worldwide, representing approximately 30% of all global deaths.1 At AstraZeneca we have been fighting CVD for over a hundred years and remain dedicated to improving understanding and revolutionising treatment options for the millions of patients impacted around the globe.

For the last century AstraZeneca has led the way in scientific discoveries in the field of cardiology, with a number of significant advances including the introduction of the first beta-blocker and one of the most potent statins to date.2,3

In many therapeutic areas, either appropriate surrogates can serve as a substitute for the efficacy assessment as they correlate with clinical relevant outcomes, or given the specific characteristics of the disease, all patients recruited can contribute to the efficacy assessment. For outcomes trials in the CVD arena, this is not generally the case, which presents specific challenges for trial sponsors.

Given the pathophysiology of CVD and the incidence of the outcome of interest in different patient populations, these trials need to include substantial numbers of study participants to be able to assess the efficacy of our products.

Consequently, large outcomes trials are the biggest research and development (R&D) investment a pharmaceutical company will make. That’s why we have over 12,000 scientists, and nine research centres across three different continents, committed to pioneering new therapeutic options across the entire spectrum of CVD. Trials of this scale provide us with a wealth of data regarding disease characteristics and safety as well as information of critical subgroups to support adequate labelling.

Our strong experience means that we at AstraZeneca understand the challenges involved in planning and managing these extensive trials, and achieving the best results. However in light of the investments associated, AstraZeneca is looking into new avenues to execute these trials while maintaining scientific rigor. This would allow us to significantly change the scope of these studies as well as broadening the horizon of research.

In addition, work is ongoing to identify meaningful surrogates to substitute clinical outcomes which in itself could revolutionise the field. An intriguing example for this approach was the GALAXY programme, a ground-breaking initiative which explored a wide variety of patient populations using the surrogate endpoint of LDL-C and a few targeted outcomes studies to reconfirm clinical relevance. The total programme encompassed 28 studies; with a population of more than 67,000 patients in 57 countries.

To effectively manage these vast trials, our collaboration with partners in the CVD field is of crucial importance. By sharing new ideas and enabling scientific innovation to cross boundaries between companies, academia, government and non-profit organisations, the results of our trials can have an even greater impact.

Our learnings from GALAXY and the initiatives referred to above have enabled us to progress to more streamlined efficiently run outcomes trials in the ongoing PARTHENON programme – a comprehensive, long-term evolving global research initiative, designed to address unanswered questions in atherothrombotic disease, and involving more than 80,000 patients in more than 30 countries.

Through our significant investment in pharmaceutical research and development, coupled with our heritage of innovation and ability to develop and bring to market new medicines, we have demonstrated a long-standing commitment to conducting vital research that answers the most important questions to improve the identification, prevention and management of all aspects of CVD.

Our goal is to help the millions of patients affected by CVD across the globe who remain at the heart of all our work and continue to serve as the driving force behind our dedication to researching and developing first-class therapies that revolutionise the treatment of cardiovascular diseases.

At AstraZeneca we envision a future in which people with CVD can trust they will be able to lead long, productive lives, and our continued investment into more targeted efficient delivery models including amongst others large-scale clinical outcomes trials brings us ever closer to achieving that goal.

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1. World Health Organisation. Cardiovascular diseases (CVDs), fact sheet N°317, updated January 2015. Available at: Last accessed February 2015.

2. Royal Society of Chemistry. Sir James Black, OM (1924-2010), updated March 2010. Available at Last accessed February 2015.

3. Kapur N, Musunuru K. Clinical efficacy and safety of statins in managing cardiovascular risk. Vascular Health and Risk Management. 2008. 4, 341–353