Evidence-Based Medicine and the Changing Nature of Health Care 2007 IOM Annual Meeting Summary (2008) / Chapter Skim
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6 Transforming the Speed and Reliability of New Evidence
6 Transforming the Speed and Reliability of New Evidence
Pages 127-154
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From page 127... ...
In the area of drug development, a combination of regulatory and market pressures is making new sources of information even more critical. This chapter examines how electronic medical records and clinical data registries could be used to expand the evidence base in many areas, as well as the unique problems facing pharmaceutical companies as they begin to develop individually tailored medicines.
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From page 128... ...
The data also could be used to generate new evidence and drive it into clinical practice more quickly. Professional societies such as the Society of Thoracic Surgeons, the American Heart Asso ciation, and the American College of Cardiology National Cardiovascular Data Registry all have rich data sets on patients with coronary disease, heart failure, and stroke.
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From page 129... ...
It will be ideal for highly structured clinical trials. In particular, classic clinical trials can be far better supported if the data are electronic.
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From page 130... ...
Anyone who is going down the EMR pathway should begin with the end in mind and design data sets to support clinical trials. As EMRs are designed, medical research must be identified as one of the outcomes of the process so that the data fields and data sets necessary are included for that purpose.
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From page 131... ...
That model is also currently not well organized or focused enough to work. What is likely to actually work to achieve real reform would be if the nation took a hard look at the fact that five medical conditions drive more than half of our healthcare costs.
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From page 132... ...
A provider-led clinical registry can be defined as a clinicianorganized network for collecting detailed patient information in a uniform fashion for a given population, often defined by a particular disease or medical treatment, and used for addressing research, quality assessment, and/or policy purposes. The concept for these registries can be traced back
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From page 133... ...
. Briefly, an ideal clinical registry should enroll representative patients, providers, and settings; collect information using standardized data elements and definitions; contain patient identifiers that allow linking of encounter records within and among data registries; have data quality and auditing systems in place to promote the accuracy and completeness of data entered; be flexible enough to allow rapid addition or deletion of variables to meet ever-changing clinical and research needs;
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From page 134... ...
. Certain states have begun requiring registry participation as part of state-based certificate of need and quality assurance programs (Massachusetts Data Analysis Center, TABLE 6-1 Selected Provider-Led Cardiovascular Clinical Data Registries Years of Data No.
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From page 135... ...
These include the standardization of data elements and definitions; the clarification of patient privacy rules; the development of new data harvesting technologies; the creation of longitudinally linked hybrid databases; and the growing collaboration among professional societies, insurers, and government regulators. Data Standards Efforts While the development of standards for medicine terminology has traditionally been elusive, cardiovascular clinical registries are now making great progress toward this goal.
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From page 136... ...
To capitalize on this, novel data harvesting and warehouse systems are now being developed that will permit providers to seamlessly map any existing stored patient information into a given clinical registry, thereby "pre-populating" the registry case report form and limiting redundant data entry. Additionally, data warehouses are moving toward the development of Web-based modular augmentation tools that will allow registries to rapidly collect new clinical information when needed.
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From page 137... ...
– Study rare events, late outcomes, and "off-label indications." Comparative Effectiveness Studies • Support more efficient randomized clinical trials. – Identify patients and investigators; streamline data collection.
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From page 138... ...
As a result, there are several recent examples where marketed therapies are subsequently found to be ineffective or unsafe. Clinical registries can be used to track the acute and long-term outcomes of therapies used in diverse patient populations, in on- and off-label indications, and under routine community clinical conditions and settings (O'Shea et al., 2004; Peterson et al., 2004)
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From page 139... ...
If interested, the patient would simply be randomized to one therapy or another, with all data collection and outcome assessment needed for the trial being conducted as part of routine registry operations. Comparative Effectiveness In situations where randomized treatment comparisons are not ethical or practical, or simply have not been conducted, observational comparative effectiveness studies of registry data provide a secondary source for evidence development.
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From page 140... ...
Quality Assessment and Quality Improvement This later point is indicative of a final important role that clinical registries can play, namely to ensure that evidence is fully and appropriately translated into clinical practice. The provider-led clinical registries were developed first and foremost as tools to support quality assessment and improvement.
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From page 141... ...
Many such reports provide clinicians with multiple comparative benchmarks, as well as highlighting for the clinician specific care processes that need attention within his or her practice. Finally, provider-led registries are now supplying clinicians with specific tools to help show them not only "what" they are doing wrong, but "how" to practice better care.
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From page 142... ...
Despite these advances, however, the way in which health care is currently delivered by a large proportion of healthcare providers, and experienced by most patients, remains largely empirical. Therapeutic interventions are frequently applied in a "one-size-fits-all" approach, and the means by which individual patients are matched to therapeutic interventions often occurs by "trial and error." While it is important not to underestimate the impact of this historical approach to treating and managing many diseases, it is also clear that this rather empirical approach must evolve to embrace the principles of comparative effectiveness and evidence-based medicine.
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From page 143... ...
As part of our commitment to this goal, we have recently developed and implemented a business model (for both R&D and commercialization) that we refer to as "tailored therapeutics." In short, tailored therapies give greater assurance that the "right drug" will be prescribed for the "right patient at the right dose and at the right time and with the right information and supporting tools." A critical success factor for delivering tailored therapeutics is our evolving and much greater understanding of the considerable heterogeneity that exists in the etiology and pathophysiology of disease and in the pharmacological response (both beneficial and adverse)
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From page 144... ...
Finally, it is widely expected that the use of generic drugs will dramatically increase over the next decade given the many scheduled near-term patent expirations. Demonstrating "comparative effectiveness" for a patent-protected drug versus a generic, in addition to monitoring a branded drug's safety profile in the post-marketing (generic)
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From page 145... ...
. Importantly, the substantial late-stage attrition that characterizes drug development at present also complicates and confounds the timing and initiation of health outcome and comparative effectiveness studies, an essential "component" of future drug development and evidence-based medicine.
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From page 146... ...
. Thus, it is possible, perhaps even likely, that comparative effectiveness studies of drugs if carried out in large heterogeneous patient populations may miss subgroups of patients in whom a given drug may actually prove to be superior with respect to either its efficacy or its safety, or both.
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From page 147... ...
Compelling health outcome data exist for only a handful of biomarkers that allow physicians (and patients) to know the likely and predictable benefits of a given drug for a given patient.
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From page 148... ...
For example, in discovery, disease state modeling is utilized as a tool to compare new drug candidates with existing medicines in the marketplace. In essence, these models enable the selection of drug candidates that will demonstrate improved health outcomes.
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From page 149... ...
Again, in the real world -- often in sharp contrast to the clinical trials required to establish safety and efficacy in the first place -- such factors will in good measure determine the effectiveness and ultimate health outcome for any biopharmaceutical. Impact of Tailored Therapies on Drug Development and Comparative Effectiveness Tailoring therapies to the patients who will most benefit from them could improve R&D productivity by having an impact on the three important productivity levers (i.e., cost, time lines, attrition)
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From page 150... ...
In parallel with efforts focused on identifying the "right patient, right dose, and right time" for therapeutic intervention, it is imperative to utilize the principles of tailored therapeutics to improve relevant patient outcomes to establish comparative effectiveness among all treatment options. An equal effort must be focused on understanding which outcomes are relevant and value-added for patients, either at an individual or at a population level.
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From page 151... ...
2005. Gender disparities in the diagnosis and treatment of non-st-segment elevation acute coronary syndromes: Large-scale observations from the crusade (can rapid risk stratification of unstable angina patients suppress adverse outcomes with early implemen tation of the American College of Cardiology/American Heart Association guidelines)
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From page 152... ...
2003. Use of continuous quality improvement to increase use of process measures in patients undergoing coronary artery bypass graft surgery: A randomized controlled trial.
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From page 153... ...
2005. Racial variations in treat ment and outcomes of black and white patients with high-risk non-ST elevation acute coronary syndromes: Insights from crusade (Can rapid risk stratification of unstable angina patients suppress adverse outcomes with early implementation of the ACC/AHA guidelines?
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