PERSPECTIVE HE RANDOMIZED REGISTRY TRIAL prominent role Will registry data Scandinavia, in places where be possible to design and conduct (or data coming from other digi- health care and clinical data are megatrials with what we have al sources, such as electronic fragmented and of lower quality? bigger data and smaller budgets health records) be of high enough Some American investigators are Yet we must also recognize and quality? Will too many data fields already using the approach (e. g, acknowledge the daunting chal- be missing? How will we balance the Study of Access Site for En- lenges that diverse groups of re- we transition single registries nary Intervention for Women; overcome to get there ders must efficacy versus effectiveness? Can hancement of Percutaneous Coro- searchers and stakehe from efficacy to effectiveness, NCTo1406236) But even if we can The view ssed in this article are making it possible to assess ex- perform many more randomized those of the authors and do not necessarily ternal validity much more expedi- registry trials in the United States, tional Heart, Lung, and Blood Institute. Dr tiously than we do now? What are we must recognize that the ap- Lauer is the National Institutes of Health lations to study? How will we ap- lems we have with trials. For tee ot the tate pcortered ne of ome iwe roach concerns about privacy certain kinds of trials, such as expressed here represent those of PCORI or and informed consent (particu- metabolic efficacy studies that its Methodology Committee larly in the context of trials that focus on complex physiologic and closure forms provided by the authors are available with the full text of this article compare acceptable standards of metabolic pathways hypothesized at NEJM.org care and use cluster-randomiza- to respond to changes in diet or ble?Will researchers be able to agents, current organizational Cardiovascular Sciences, the Natons o tion methods)? Is blinding possi- to experimental pharmacologic From the Office of the Director, Division of Heart, Lung, and Blood Institute, Bethesda obtain long-term follow-up or structures would probably work MD(MSL); and the Mathematics and Sta- measure composite outcomes? much better with only minor tistics Department, Boston University, and How will we standardize and ad- modifications judicate certain outcomes? Can The randomized registry trial that even within a registry there a technology that transforms ex- 2013, at NE/M.org. hed on September I we assure representativeness, given represents a disruptive technology, This article was published on September may be systematic differences be- isting standards, procedures, and tween patients who are and are cost structures. Will it be given 1. Antman EM, Harrington RA Transform not eligible for randomization or serious consideration as a way to mission critical for health and economic between those who do or do not resolve the recognized limitations well-being. JAMA 2012:308: 1743.4 2. Pocock S), Elbourne DR Randomized tri- consent of current clinical-trial design? als or observational tribulations? N Engl J These are only some of the Theodore Roosevelt once said, Med 2000:342: 1907-9 problems we will have to address. "Do what you can, with what you 3. Frobert o, Lagergvist B, Gudnason The TASTe trial was performed have, where you are. " Today we myocardial infarction in Scandinavia(TASTE Scand afford care and information technology randomized effectiveness trials ized, controlled clinical registry trial based environments are markedly dif. that cost tens or hundreds of mil- ty Registry(SCAAR)platform: study design ferent from those elsewhere in lions of dollars. But today we alse the world. Can randomized regis- have registries and other power- Dol: 10.1056/NEJMp1310102 try trials be undertaken outside ful digital platforms. Today it may Copyright o 2013 Massachusetts Medical. Smoothing the Way to High Quality, Safety, and Economy Eugene Litvak, Ph. D, and Harvey V. Fineberg, M. D, Ph D n recent years, health care in- aid Services is implementing fections associated with ventila- have awakened to multiple incentives and penalties tors or central venous catheters the need to provide safe, high- intended to help realize this have had demonstrated success quality care at lower cost. The goal. For example, innovations We believe that greater attention Centers for Medicare and Medic- designed to reduce the rate of in- to a frequently overlooked param- N ENGLJ MED 369: 17 NEJM.ORG OCTOBER 24, 2013
n engl j med 369;17 nejm.org october 24, 2013 PERSPECTIVE 1581 The Randomized Registry Trial prominent role. Will registry data (or data coming from other digital sources, such as electronic health records) be of high enough quality? Will too many data fields be missing? How will we balance efficacy versus effectiveness? Can we transition single registries from efficacy to effectiveness, making it possible to assess external validity much more expeditiously than we do now? What are the best populations or subpopulations to study? How will we approach concerns about privacy and informed consent (particularly in the context of trials that compare acceptable standards of care and use cluster-randomization methods)? Is blinding possible? Will researchers be able to obtain long-term follow-up or measure composite outcomes? How will we standardize and adjudicate certain outcomes? Can we assure representativeness, given that even within a registry there may be systematic differences between patients who are and are not eligible for randomization or between those who do or do not consent? These are only some of the problems we will have to address. The TASTE trial was performed in Scandinavia, where the health care and information technology environments are markedly different from those elsewhere in the world. Can randomized registry trials be undertaken outside Scandinavia, in places where health care and clinical data are fragmented and of lower quality? Some American investigators are already using the approach (e.g., the Study of Access Site for Enhancement of Percutaneous Coronary Intervention for Women; NCT01406236). But even if we can perform many more randomized registry trials in the United States, we must recognize that the approach cannot solve all the problems we have with trials. For certain kinds of trials, such as metabolic efficacy studies that focus on complex physiologic and metabolic pathways hypothesized to respond to changes in diet or to experimental pharmacologic agents, current organizational structures would probably work much better with only minor modifications. The randomized registry trial represents a disruptive technology, a technology that transforms existing standards, procedures, and cost structures. Will it be given serious consideration as a way to resolve the recognized limitations of current clinical-trial design? Theodore Roosevelt once said, “Do what you can, with what you have, where you are.” Today we can no longer afford to undertake randomized effectiveness trials that cost tens or hundreds of millions of dollars. But today we also have registries and other powerful digital platforms. Today it may be possible to design and conduct megatrials with what we have: bigger data and smaller budgets. Yet we must also recognize and acknowledge the daunting challenges that diverse groups of researchers and stakeholders must overcome to get there. The views expressed in this article are those of the authors and do not necessarily represent the official positions of the National Heart, Lung, and Blood Institute. Dr. Lauer is the National Institutes of Health representative on the Methodology Committee of the Patient-Centered Outcomes Research Institute (PCORI); none of the views expressed here represent those of PCORI or its Methodology Committee. Disclosure forms provided by the authors are available with the full text of this article at NEJM.org. From the Office of the Director, Division of Cardiovascular Sciences, the National Heart, Lung, and Blood Institute, Bethesda, MD (M.S.L.); and the Mathematics and Statistics Department, Boston University, and the Harvard Clinical Research Institute — both in Boston (R.B.D.). This article was published on September 1, 2013, at NEJM.org. 1. Antman EM, Harrington RA. Transforming clinical trials in cardiovascular disease: mission critical for health and economic well-being. JAMA 2012;308:1743-4. 2. Pocock SJ, Elbourne DR. Randomized trials or observational tribulations? N Engl J Med 2000;342:1907-9. 3. Fröbert O, Lagerqvist B, Gudnason T, et al. Thrombus Aspiration in ST-Elevation myocardial infarction in Scandinavia (TASTE trial): a multicenter, prospective, randomized, controlled clinical registry trial based on the Swedish Angiography and Angioplasty Registry (SCAAR) platform: study design and rationale. Am Heart J 2010;160:1042-8. DOI: 10.1056/NEJMp1310102 Copyright © 2013 Massachusetts Medical Society. Smoothing the Way to High Quality, Safety, and Economy Eugene Litvak, Ph.D., and Harvey V. Fineberg, M.D., Ph.D. I n recent years, health care institutions have awakened to the need to provide safe, highquality care at lower cost. The Centers for Medicare and Medicaid Services is implementing multiple incentives and penalties intended to help realize this goal. For example, innovations designed to reduce the rate of infections associated with ventilators or central venous catheters have had demonstrated success. We believe that greater attention to a frequently overlooked param-
PERSPECTIVE SMOOTHING THE WAY TO QUALITY, SAFETY, AND ECONOMY eter in health service design standing tradition of cost-plus if they re admitted during a valley patient load and flow would reimbursement, the readiest phys- in patient demand and unfortu- accelerate progress toward reli- ical accommodation for a peri- nate if they arrive during a peak, able. safe. efficient care odic imbalance between patient when all hospital resources(beds, A reliable health system is one demand and delivery-system ca- nurses, physicians, radiology equip- that functions properly in diffi- pacity has been to provide addi- ment, etc )are stretched thin cult and unanticipated circum- tional capacity(e.g, build more Excess flow of patients is not stances as well as in normal or hospital beds at a capital cost simply a feature of natural disas easy ones -indeed, difficult alone of more than $1 million ters, pandemics, or tragic events and unanticipated circumstances each), so that peaks in demand At every hospital examined by the are par for the course in health can be met. 2 Institute for Healthcare Optimi- care. Among the most common Health systems have only three zation and others, the majority such conditions are periods of staffing choices for accommodat- of variability in patient flow is excess patient load that can over- ing peaks in patient flow: having attributable to scheduled admis whelm even the most conscien- enough staff available at all times sions. tious physician or nurse and im- to meet the peaks, an option that In many U.S. hospitals, it has pair the quality of care. a growing is neither affordable to the sys- become standard practice to body of evidence illuminates the tem as a whole nor widely feasi- form as many planned surgeries adverse consequences of exces- ble in the face of the insufficient each day as are requested by sur- sive patient load. Studies have number of nurses; intentionally geons with admitting privileges shown that increased patient vol- staffing for below-peak patient When many surgeries are sched ume increases the likelihood of levels and tolerating periods of uled for the same day, they create harm to patients, the rate of inadequate care; or establishing artificial peaks in "patient"de- health care-associated infections, dynamic nursing pools to fill in mand. These peaks are truly the average length of stay, and the during the peaks, a scheme that driven not by patient needs, de- odds of readmission. Nearly 40% fails if bed occupancy changes sire, or benefit but rather by a of hospitalists report experienc- every hour or two confluence of managerial inatten- ing an unsafe volume of patients All these unsatisfactory op- tion and deeply rooted profes- at least monthly. 1 tions are based on the premise sional prerogatives and institu angd s patient demand fluctuates that peaks in patient demand are tional practices. These artificial the number of patients per patient-driven - that is, primar- peaks and valleys continue to nurse increases, delivery of high- ily dependent on natural fluctua- foster health care delivery that quality, safe care becomes unre- tions in the occurrence of illness endangers patients, reduces ac liable. Even the best protocols and injury that are beyond the cess to care, puts artificial pres- cannot be consistently followed control of health care profession- sure on clinicians during peaks, when an organization is over- als and institutional leaders. That and results in underutilization of whelmed with patients and staff supposition, however, is highly health care resources during val- must take shortcuts or delay at- dubious. ys. tending to some patients Overall, the average occupancy ment to building a safe and effi- need care. In addition, periodic rate of U.S. hospital beds is 65 to cient delivery system stresses on the delivery system 67%- substantially lower than Artificial peaks and valleys in undermine clinicians' morale. that in many other industrialized patient flow can and should be Once providers and institutions countries, according to the Orga- analyzed and smoothed, by means adopt shortcuts and tolerate de- nization for Economic Coopera- of a practically proven method.3,4 lays during peak days and hours, tion and Development. Despite the This method requires first sepa- during valleys in patient load. at any given time, utilized hospi- tive and unscheduled procedures Periodic peaks in patient flot fluctuates daily d the pressure hospitals to spend more in individual hospitals(see graph), numbers of elective admissions on both physical and human and hospitals are often over- occur in different hospital wards sources. Because of the long- crowded. Patients are fortunate each day. 3 Hospitals such as Johns N ENGLJ MED 369: 17 NEJM.ORG OCTOBER 24, 2013
PERSPECTIVE 1582 n engl j med 369;17 nejm.org october 24, 2013 eter in health service design — patient load and flow — would accelerate progress toward reliable, safe, efficient care. A reliable health system is one that functions properly in difficult and unanticipated circumstances as well as in normal or easy ones — indeed, difficult and unanticipated circumstances are par for the course in health care. Among the most common such conditions are periods of excess patient load that can overwhelm even the most conscientious physician or nurse and impair the quality of care. A growing body of evidence illuminates the adverse consequences of excessive patient load. Studies have shown that increased patient volume increases the likelihood of harm to patients, the rate of health care–associated infections, the average length of stay, and the odds of readmission.1 Nearly 40% of hospitalists report experiencing an unsafe volume of patients at least monthly.1 As patient demand fluctuates and the number of patients per nurse increases, delivery of highquality, safe care becomes unreliable. Even the best protocols cannot be consistently followed when an organization is overwhelmed with patients and staff must take shortcuts or delay attending to some patients who need care. In addition, periodic stresses on the delivery system undermine clinicians’ morale. Once providers and institutions adopt shortcuts and tolerate delays during peak days and hours, they may come to accept them as their usual standard of care even during valleys in patient load. Periodic peaks in patient flow pressure hospitals to spend more on both physical and human resources. Because of the longstanding tradition of cost-plus reimbursement, the readiest physical accommodation for a periodic imbalance between patient demand and delivery-system capacity has been to provide additional capacity (e.g., build more hospital beds at a capital cost alone of more than $1 million each), so that peaks in demand can be met.2 Health systems have only three staffing choices for accommodating peaks in patient flow: having enough staff available at all times to meet the peaks, an option that is neither affordable to the system as a whole nor widely feasible in the face of the insufficient number of nurses; intentionally staffing for below-peak patient levels and tolerating periods of inadequate care; or establishing dynamic nursing pools to fill in during the peaks, a scheme that fails if bed occupancy changes every hour or two. All these unsatisfactory options are based on the premise that peaks in patient demand are patient-driven — that is, primarily dependent on natural fluctuations in the occurrence of illness and injury that are beyond the control of health care professionals and institutional leaders. That supposition, however, is highly dubious. Overall, the average occupancy rate of U.S. hospital beds is 65 to 67% — substantially lower than that in many other industrialized countries, according to the Organization for Economic Cooperation and Development. Despite the fact that on average, one third of U.S. hospital-bed capacity is idle at any given time, utilized hospital capacity fluctuates daily even in individual hospitals (see graph), and hospitals are often overcrowded. Patients are fortunate if they’re admitted during a valley in patient demand and unfortunate if they arrive during a peak, when all hospital resources (beds, nurses, physicians, radiology equipment, etc.) are stretched thin. Excess flow of patients is not simply a feature of natural disasters, pandemics, or tragic events. At every hospital examined by the Institute for Healthcare Optimization and others,3 the majority of variability in patient flow is attributable to scheduled admissions. In many U.S. hospitals, it has become standard practice to perform as many planned surgeries each day as are requested by surgeons with admitting privileges. When many surgeries are scheduled for the same day, they create artificial peaks in “patient” demand. These peaks are truly driven not by patient needs, desire, or benefit but rather by a confluence of managerial inattention and deeply rooted professional prerogatives and institutional practices. These artificial peaks and valleys continue to foster health care delivery that endangers patients, reduces access to care, puts artificial pressure on clinicians during peaks, and results in underutilization of health care resources during valleys. They are a major impediment to building a safe and efficient delivery system. Artificial peaks and valleys in patient flow can and should be analyzed and smoothed, by means of a practically proven method.3,4 This method requires first separating the hospital resources (e.g., operating rooms) used for elective and unscheduled procedures and then ensuring that similar numbers of elective admissions occur in different hospital wards each day.3 Hospitals such as Johns Smoothing the Way to Quality, Safety, and Economy
PERSPECTIVE SMOOTHING THE WAY TO QUALITY, SAFETY, AND ECONOMY life. underwrite biomedical re- search. reduce the national debt support schools, and serve many other private and public purposes At the same time, properly man- ag medical errors and enhance the quality of care. We owe these im- provements to our patients, to the health care commun the next generation are available with the full text of this artic at NEJM.org. From the Institute for h Month Newton, MA(EL); and the Institute of medicine of the National Academies Washington, DC(HVF). Daily Fluctuations in One Hospitals Admissions for Emergency and Elective Surgery. Data are for weekdays only and are from the Institute for Healthcare Optimization 1. Studies of the effects of patient flow vari- Hopkins,ciNcinnatiChildren,s,incapitalcostsandincreaseditszation2013(http://www.ihoptimize.org/ and the Mayo Clinic in Florida margin by more than $100 mil- what-we-do-methodology-quality-and-safety have applied this method success- lion annually while improving 2. Litvak E, Bisognano M. More patients, fully, through leadership commit- the quality of care. 5 If each of less payment: increasing hospital efficiency ment, engagement of physicians the almost 6000 U. S. hospitals (millwood/ 201: 30: 6-30 alth reform Health aff irgeons, use achieved ese 3. Litvak E, ed Managing patient flow: strat local data, and reliance on the nancial results, it would mean egies and solutions. 2nd edOakbrook principles of operations research. about $60 billion in savings, plus race, IL Joint Commission Resources, 2010 Applying this method over 1 to reduced overcrowding, complica- Re-engineering the operating room using 2 years, these hospitals have tions, readmissions, and mortal- variability methodology to achieved multimillion-dollar re- every hospital avoid 013:216559-68. ductions in annual costs while adding just one extra bed for ac- 5. Ryckman E, Adler E,Anneken A, et al. Cin imultaneously improving patient commodating artificial influxes cinnati Children's Hospital Medical Cente safety, quality of care, and satis- of patients, the health care system fees igning eeneopt trails to wm using apers ng patients, S6 bill to$12 nd hos anagers ke.2,4, 5 lion in capital costs alone For example, by properly manag. Direct and indirect savings ng patient flow, Cincinnati Chil- from smoother patient flow could sion Resources, 2010:97-111 dren's Hospital saved $100 million give Medicare a new lease on copyright e 2013 Massachusetts Medical Society The debt of life thai lessons on a process-Oriented Ethical logic Scott D. Stonington, M. D, Ph D Te love him so much, "said -or at least I want to, "she said case on rounds a failed Ms M, standing over her tearfully, acknowledging the dis- bone marrow trar nt, he'd had father as he lay on life support in agreement among her siblings a myocardial infarction, which a Boston ICU where I was an in- about how to proceed. Later that had led to heart failure, then tern. " We want to do everything morning, I presented her father's nal failure, then pneumonia and N ENGLJ MED 369: 17 NEJM.ORG OCTOBER 24, 2013 1583
n engl j med 369;17 nejm.org october 24, 2013 PERSPECTIVE 1583 Smoothing the Way to Quality, Safety, and Economy Hopkins, Cincinnati Children’s, and the Mayo Clinic in Florida have applied this method successfully, through leadership commitment, engagement of physicians and especially surgeons, use of local data, and reliance on the principles of operations research. Applying this method over 1 to 2 years, these hospitals have achieved multimillion-dollar reductions in annual costs while simultaneously improving patient safety, quality of care, and satisfaction among patients, clinicians, and hospital managers alike.2,4,5 For example, by properly managing patient flow, Cincinnati Children’s Hospital saved $100 million in capital costs and increased its margin by more than $100 million annually while improving the quality of care.5 If each of the almost 6000 U.S. hospitals achieved only 10% of these financial results, it would mean about $60 billion in savings, plus reduced overcrowding, complications, readmissions, and mortality.2 If every hospital avoided adding just one extra bed for accommodating artificial influxes of patients, the health care system would save $6 billion to $12 billion in capital costs alone. Direct and indirect savings from smoother patient flow could give Medicare a new lease on life, underwrite biomedical research, reduce the national debt, support schools, and serve many other private and public purposes. At the same time, properly managed patient flow can reduce medical errors and enhance the quality of care. We owe these improvements to our patients, to the health care community, and to the next generation. Disclosure forms provided by the authors are available with the full text of this article at NEJM.org. From the Institute for Healthcare Optimization, Newton, MA (E.L.); and the Institute of Medicine of the National Academies, Washington, DC (H.V.F.). 1. Studies of the effects of patient flow variability on healthcare quality and safety. Newton, MA: Institute for Healthcare Optimization, 2013 (http://www.ihoptimize.org/ what-we-do-methodology-quality-and-safety .htm). 2. Litvak E, Bisognano M. More patients, less payment: increasing hospital efficiency in the aftermath of health reform. Health Aff (Millwood) 2011;30:76-80. 3. Litvak E, ed. Managing patient flow: strategies and solutions. 2nd ed. Oakbrook Terrace, IL: Joint Commission Resources, 2010. 4. Smith CD, Spackman T, Brommer K, et al. Re-engineering the operating room using variability methodology to improve health care value. J Am Coll Surg 2013;216:559-68. [Erratum, J Am Coll Surg 2013;216:1230.] 5. Ryckman F, Adler E, Anneken A, et al. Cincinnati Children’s Hospital Medical Center: redesigning perioperative flow using operations management tools to improve access and safety. In: Litvak E, ed. Managing patient flow in hospitals: strategies and solutions. 2nd ed. Oakbrook Terrace, IL: Joint Commission Resources, 2010:97-111. DOI: 10.1056/NEJMp1307699 Copyright © 2013 Massachusetts Medical Society. No. of Daily Admissions 80 40 50 10 60 20 30 0 70 Month Elective Emergency 1 2 3 Daily Fluctuations in One Hospital’s Admissions for Emergency and Elective Surgery. Data are for weekdays only and are from the Institute for Healthcare Optimization. The Debt of Life — Thai Lessons on a Process-Oriented Ethical Logic Scott D. Stonington, M.D., Ph.D. “We love him so much,” said Ms. M., standing over her father as he lay on life support in a Boston ICU where I was an intern. “We want to do everything — or at least I want to,” she said tearfully, acknowledging the disagreement among her siblings about how to proceed. Later that morning, I presented her father’s case on rounds: after a failed bone marrow transplant, he’d had a myocardial infarction, which had led to heart failure, then renal failure, then pneumonia and
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