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Large Simple Trials and Knowledge Generation in a Learning Health System Workshop Summary (2013) / Chapter Skim
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2 Large Simple Trials Now and Looking Forward
Pages 11-18

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From page 11...
... Horwitz • LSTs pose a series of challenges and opportunities for the clinical research enterprise. These include solidification of their external validity, a better understanding of the implications of LSTs for the detection of treatment heterogeneity and patient safety, and exploration of opportunities for greater integration of patient-reported outcomes.
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From page 12...
... He pointed to the year 1976, when Steven Sasson, a Kodak engineer, invented the digital camera, which has subsequently proceeded to eclipse the photographic film and film camera business almost completely. Although the technology was developed by Kodak, the company decided not to exploit its advantage and stuck to its film-based business model.
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From page 13...
... Most RCTs involve a small number of subjects able to meet a narrow set of criteria and collect large amounts of very specific data on each subject. RCT recruitment and data collection, monitoring, and auditing processes are very costly; and often, RCTs can be supported only in academic medical centers.
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From page 14...
... It was possible to conduct a trial for a disease that affects only 5 in 1 million women because a significant number of LAM patients were willing to participate in clinical trials through their organization, the LAM Foundation, to which many had already contributed clinical profiles. Lauer proposed a new model that integrates trials into routine clinical care and that would involve the simultaneous conduct of many LSTs.
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From page 15...
... Horwitz asked if it is possible that a very small benefit shown by an LST might have a negative benefit in the intended target population. He noted that LSTs, like all clinical trials, have selection criteria, and therefore, it is possible that patients meeting those criteria may have a response different from that of the overall target population.
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From page 16...
... When adverse events occur with any new medicine, detailed data on the clinical context in which the adverse event occurred are required for regulatory review and approval. Horwitz noted that it can be frustrating to rely on data collected for other purposes to identify the antecedent events associated with an adverse event when the relevant data were not systematically
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From page 17...
... They can then be conducted without requiring patients to travel to brick-and-mortar research facilities by using electronic information technology systems for recruitment, informed consent, medication orders, and follow-up. Horwitz mentioned that a group called Mytrus is trying to pioneer such approaches and that social media like PatientsLikeMe could be used to obtain PROs.
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Key Terms

  • treatment heterogeneity
  • clinical trials
  • treatment effects
  • learning health
  • clinical trial

  • business model
  • target population
  • myocardial infarction
  • clinical decision
  • clinical encounter

  • coronary care
  • digital camera
  • gissi trial
  • care units
  • gruppo italiano

  • studio della
  • della sopravvivenza
  • horwitz asked
  • acute myocardial
  • adverse event

  • thrombus aspiration
  • relevant clinical
  • simple clinical
  • standard treatment
  • relevant data

  • thrombolytic treatment
  • standard business
  • contributed clinical
  • data routinely
  • moderate treatment


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