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What is the probability of discovering breakthrough interventions in industry versus publicly sponsored randomized controlled trials

Date and Location




Friday 20 September 2013 - 10:30 - 12:00


Presenting author and contact person

Presenting author

Benjamin Djulbegovic

Contact person

Benjamin Djulbegovic
Branko Miladinovic
Abstract text
Background: Research efforts tested in randomized controlled trials (RCTs) have played a vital role in the development of new treatments in medicine. Treatment research in medicine is primarily performed by publicly funded entities or privately funded drug companies. Objective: To study whether new interventions tested in publicly versus industry funded RCTs are highly successful or "breakthrough." Methods: All completed phase 3 RCTs conducted by 5 publicly funded groups (820 trials, 1,064 comparisons, 331,004 patients) and GlaxoSmithKline (40 trials, 55 comparisons, 19,889 patients) were reviewed. Published and unpublished data were used. Breakthroughs were defined two ways: semi-quantitatively (score 1–6): the intervention was so successful according to the original researchers that it should be adopted immediately as a new therapeutic standard (=6), and quantitatively: the intervention effect resulted in a log odds ratio < -0.5 for the primary outcome. Results: Quantitatively, 14% (n=149) of comparisons resulted in breakthrough interventions in publicly funded studies versus 34% (n=19) in industry sponsored RCTs. According to the original investigators, 15% (n=124) of experimental testing were breakthrough in the cohort of publicly funded versus 35% (n=14) in industry sponsored studies. Semi-quantitatively 12% (n = 15) of breakthroughs were for breast cancer in publicly funded studies versus 78% (n = 11) in privately funded studies for chemo/radiation induced emesis. Induction and curative therapies jointly resulted in 62% and 73% of breakthroughs by quantitative and semi-quantitative assessment, in publicly funded RCTs versus 2% quantitatively and 0% semi-qualitatively in industry sponsored RCTs. Conclusions: This is the first comprehensive assessment of success of innovative therapies in medicine tested in a cohort of public versus industry sponsored RCTs. Majority of breakthroughs by publicly funded RCTs addressed curative therapy strategies compared with supportive care in industry funded RCTs. All stakeholders should understand what it takes to produce a successful breakthrough intervention in medicine.