In theory there is no difference between theory and practice. In practice there is.Yogi Berra
This quotation from baseball icon and American cult philosopher Yogi Berra addresses a problem we face regularly in clinical medicine: interventions that should work in theory and appear to work in controlled research settings do not work nearly as well, or not at all, during “real-world” applications. Although we think of translational research as translating basic research into clinical application, the most difficult translation may be in taking treatments that are efficacious in clinical trials, applying them in the real world of clinical practice, and getting the same results. This dichotomy describes the difference between efficacy and effectiveness. In order for an efficacious treatment to be effective, the treatment must be readily available, practitioners must apply the treatment to the recommended target population, and patients must be willing to accept and adhere to treatment recommendations.
- Singal A.G.
- Higgins P.D.
- Waljee A.K.
A primer on effectiveness and efficacy trials.
Treatment for tobacco dependence using behavioral therapy and pharmacotherapy has been proven efficacious in numerous clinical trials and less rigorous uncontrolled studies.
Combined pharmacotherapy and behavioural interventions for smoking cessation.
In this issue of Mayo Clinic Proceedings
, Kotz et al
Prospective cohort study of the effectiveness of smoking cessation treatments used in the “real world.”.
report on the nationwide effectiveness of combined behavioral therapy and pharmacotherapy for tobacco dependence treatment in England. In a previous effectiveness study by the same authors, a cross-sectional survey was used to confirm that evidence-based treatment can be translated to the real world, and they were able to validate a measure that accounted for one of the most important confounding factors for any study of tobacco dependence treatment: the degree of tobacco dependence assessed by strength of urges to smoke.
‘Real-world’ effectiveness of smoking cessation treatments: a population study.
In the current research, the investigative team applied this new measure to a prospective cohort of smokers with follow-up for up to 6 months.
In the United Kingdom, the National Health Service has invested heavily in providing tobacco dependence treatment as a key component of the tobacco control efforts. Specialized clinics for the treatment of tobacco dependence have been established widely, and most general practitioners have been trained to provide brief intervention services for tobacco cessation. Additionally, all of the first-line medications (ie, medications proven efficacious and having a labeled indication for smoking cessation) for tobacco dependence are available for use. The current study used a survey method to obtain information regarding tobacco quit attempts among smokers, and categorized smokers as those who (1) had been seen in a specialized clinic for tobacco cessation treatment, (2) received brief intervention from their general practitioner, (3) used over-the-counter nicotine replacement therapy without behavioral intervention, or (4) simply tried to quit unassisted. Prescription pharmacotherapy was provided to patients seen in specialized clinics or by their general practitioners.
One of the important methodological features of the study by Kotz et al is the adjustment of results for important confounders, including adjustment for the degree of nicotine dependence before the quit attempt. Their results were striking. Smokers who attempted to quit smoking using specialist clinics plus prescription medication had an odds ratio (OR) for successful abstinence of 2.58 [95% CI, 1.48-4.52] compared with unassisted quit attempts. Similarly, prescription medication plus brief advice by a general practitioner resulted in an increased odds of successful abstinence compared with unassisted quitting (OR, 1.55; 95% CI, 1.11-2.16). Finally, an unexpected result was the finding that the use of over-the-counter nicotine replacement therapy without any behavioral support resulted in a lower chance of successful abstinence compared with unassisted quitting (OR, 0.68; 95% CI, 0.49-0.94).
One key question to ask is whether these investigators truly approximated the real world in which most practitioners and smokers live. Positive elements of the research design are that the authors studied a heterogeneous population that was unselected, and practitioners delivered the intervention in routine clinical settings. Kotz et al were able to make adjustments for potential confounders, although unaccounted for confounders cannot be ruled out. Factors that may be different in the clinical setting found in England compared with most other countries include the availability of specialized clinics that provide a standardized approach to tobacco dependence treatment and a corps of general practitioners who are largely trained and given incentives for providing intervention for smokers. For example, in the United States there are tobacco treatment specialists sprinkled across the country among medical practices, clinics, and hospitals, but there are very few specialized clinics to which smokers can be referred for evidence-based behavioral treatment and pharmacotherapy for tobacco dependence. In low- and middle-income countries, there is virtually no capacity for tobacco dependence treatment, and the availability of effective pharmacotherapy is quite limited. The reality for most health care professionals outside the United Kingdom is that the most effective treatment as described by Kotz et al is either unavailable altogether or inaccessible to most patients.
In addition to the main results in the report by Kotz et al, some of the secondary results are noteworthy. First, only 4.8% of patients received the most effective therapy (specialist support plus prescription medication) during their last quit attempt, and only 20.8% received brief advice and medication. Thus, almost 75% of smokers either used no medication or support or chose nicotine replacement therapy purchased on their own—ie, the most ineffective treatments (see subsequent discussion). This finding is consistent with the results of polls of smokers who believe that unassisted quitting is most effective, an approach supported by some experts as well.
Quitting smoking unassisted: the 50-year research neglect of a major public health phenomenon.
Perhaps surprising to many, the results of the study by Kotz et al suggest that nicotine replacement therapy purchased over the counter results in a worse outcome compared with simply quitting unassisted. Tobacco dependence treatment experts have long believed that any attempt to quit, using any means available, should be encouraged. However, the results of the current study raise the possibility that using nicotine replacement therapy without additional support may undermine abstinence and discourage future attempts to quit. Why should an efficacious treatment provide such a negative result? The authors speculate that poor adherence to treatment (incorrect dose, inappropriately brief treatment duration) may be the cause, a speculation supported by population-based survey studies.
- Kasza K.A.
- Hyland A.J.
- Borland R.
- et al.
Effectiveness of stop-smoking medications: findings from the International Tobacco Control (ITC) Four Country Survey.
How should we apply the results of this study to the real world in which each of us lives? First, behavioral therapy and pharmacotherapy work synergistically to improve abstinence outcomes. Every patient who uses tobacco should be offered at least brief behavioral intervention and first-line pharmacotherapy to aid in quitting. Years of productive life are added for every smoker who successfully quits. Health care professionals should discourage patients from simply buying nicotine replacement therapy off the shelf and trying it on their own. This strategy may be even less effective than unaided quitting. Not only is it ineffective, but it may also undermine future quit attempts by making smokers feel that pharmacotherapy will not work for them. Practitioners should learn simple techniques to provide brief behavioral support, advise patients that medications will help them in their attempt to quit, and provide medications to those who are motivated to quit. When access to a tobacco treatment specialist is available, practitioners should simply ask patients about their smoking status, advise them to quit, and refer them for specialist treatment.
Tobacco use continues to be prevalent and deadly in the United States and worldwide. Further, smoking cessation is one of the most important health behavior changes that we can encourage in our patients. Hundreds of clinical trials that included thousands of patients have documented the efficacy of combined behavioral therapy and pharmacotherapy for tobacco dependence treatment. The research by Kotz et al confirms that this approach can be translated to the real world and provide real benefit. This is a case in which there is happily no difference between “theory and practice.” Health systems, hospitals, clinics, and physicians now need to practice the well-established standard of care to save real lives in their real world.
- Singal A.G.
- Higgins P.D.
- Waljee A.K.
A primer on effectiveness and efficacy trials.Clin Transl Gastroenterol. 2014; 5: e45
Combined pharmacotherapy and behavioural interventions for smoking cessation.Cochrane Database Syst Rev. 2012; 10: CD008286
Prospective cohort study of the effectiveness of smoking cessation treatments used in the “real world.”.Mayo Clin Proc. 2014; 89: 1360-1367
‘Real-world’ effectiveness of smoking cessation treatments: a population study.Addiction. 2014; 109: 491-499
Quitting smoking unassisted: the 50-year research neglect of a major public health phenomenon.JAMA. 2014; 311: 137-138
- Kasza K.A.
- Hyland A.J.
- Borland R.
- et al.
Effectiveness of stop-smoking medications: findings from the International Tobacco Control (ITC) Four Country Survey.Addiction. 2013; 108: 193-202
© 2014 Mayo Foundation for Medical Education and Research. Published by Elsevier Inc. All rights reserved.