Concept of ‘benefit to patients’ offers a unique set of challenges
Every day we manage patients based on what we have learned and experienced in clinical practice. We are obligated to provide the best care available based on our knowledge as well as consider many clinical factors, including the shared decision-making process with patients.
We develop a personal relationship with every patient and our efforts are frequently framed by the question, “If this was you or your loved one, what would you recommend?” However, as we move into the era of value-based orthopedic care, third parties interested in the elimination of expensive interventions that do not demonstrate clear benefit will scrutinize all aspects of care without consideration of the personal physician-patient relationship. Understanding that value is the benefit divided by the cost, it is easy to understand why cost is readily measured and managed. This can challenge our patient care model if we believe more expensive care is in the best interest of the patient, but its access is restricted based on cost. We can argue on behalf of the patient based on our belief of the benefits of the care even though strong evidence is absent or limited.
Unique set of challenges
The concept of “benefit to patients” offers a unique set of challenges. In many areas, we have not agreed on the best way to collect data that helps define benefit. Also, we have not agreed on the best way to analyze the data. However, there is a greater value on studies that provide higher levels of evidence, such as rare orthopedic randomized (level 1) or nonrandomized (level 2) clinical trials or case-controlled (level 3) prospective studies. These expensive methods, which are most commonly performed at major medical centers, are intended to avoid bias that can be inherent in many other methods to determine the best care for patients.
Anthony A. Romeo
The nature of conducting these studies at academic institutions may introduce bias in ways we do not presently understand. These studies, and the academic orthopedic surgeons who frequently have reduced clinical and surgical volumes to provide time for research and writing, may not reflect the outcomes seen in the private sector, which represents more than two-thirds of the membership of the American Academy of Orthopaedic Surgeons (AAOS). If the research was conducted in the private sector, the results may be better, or may be worse, but we do not know because research and outcomes resources are not generally available.
Historically, if study results are statistically significant, then it is assumed one treatment is more beneficial than another treatment. The importance of statistical significance has been questioned when analyzing the impact of a medical intervention on patient populations. Study results may indicate that the result of one treatment is statistically significantly better than another. Therefore, when faced with the decision on what option to provide, we choose the option that demonstrated better outcomes based on statistical analysis, which shows the better result did not happen randomly or by chance.
The concept of “meaningful clinically important improvement” (MCII) has now entered into the analysis. Results of a study may demonstrate statistical significance over the control group or another treatment group, but these results may not reflect a meaningful clinical difference for the population studied. The mathematical estimates of benefit often do not help when we are in front of patients and know a treatment can be helpful for some patients, but we are prohibited from providing that care because studies have failed to demonstrate high levels of evidence or positive MCII results.
Clinical practice guidelines
As you will read in this month’s Cover Story, the AAOS has sponsored and supported the development of clinical practice guidelines (CPGs). The purpose of establishing CPGs is to formally establish treatment recommendations based on the best evidence available at the time of the process. A CPG provides physicians with the confidence that they are following a “road map” to the best outcome for their patients. The current method used by for CPG development incorporates the concept of MCII and higher levels of evidence. When this data is absent, the CPG development process does not default to using lower levels of evidence.
The use of CPGs inherently decides what treatments deserve a share of limited economic resources. Insurance companies, government and others financially responsible for health care, are scrutinizing the CPG conclusions and are using the information in the consideration of their coverage of orthopedic care. This process was predicted before the development of CPGs and now is confirmed after their development. Recommendations and guidelines have led to the loss of therapeutic options. The AAOS leadership has publicly stated that CPGs were not intended to affect coverage decisions, but when the goal is to control cost, either because of limited resources or for insurance companies to continue seeing record-setting profits, the public statement from the AAOS brings into question the responsibility of the AAOS toward its constituency. When I see these efforts for CPG development, I cannot help but call into question if the AAOS is truly serving its mission statement at its highest level.
When provided with gaps in practice guidelines, one evaluates information available in scientific studies and expert opinions. When we process any information, we have three levels of response. A reflex or level 1 response requires little mention or criticism. A level 2 response means one has some understanding of the information, but can only say what is wrong with it. In a level 3 response, one understands the communication, can recognize lapses in the communication and has the ability to respond with constructive comments and thoughts that lead to higher level of communication and better information. As the CPG process continues forward, I see level 2 thinking and wonder if there will be leadership for a level 3 thinking.
The process of CPG development is controversial, and in some cases, may inaccurately reflect what is truly in the best interest of patients. The evidence for the treatment of a torn rotator cuff is weak, but the patient who has not slept for months, has tried every possible nonoperative intervention, and is now sincerely pleading to have their pain managed will not want to know about CPGs or evidence-based medicine. Based on many level 4 and level 5 studies, you could offer patients a strong and confident recommendation for surgical intervention. Lower levels of evidence may provide practice guidelines that are appropriate to help patients, even if the science lacks at this time.
Medical interventions are valuable to the patient-physician relationship on many levels. Hands-on care, sense of empathy, positive response to intervention even for a limited period of time, and the development of trust are essential aspects of successful medical care. We are not able to measure, analyze and manage this critical part of medical care, which means we cannot define its benefit. If we cannot demonstrate a benefit, then the decision about value will be based entirely on economics.
The research performed to establish a CPG should be transformed into a proposal and project that, in fact, supports the AAOS membership in trying to provide the missing information or strengthen the information available. Many years ago the Society of French Arthroscopy established a tradition of setting up at least two prospective level 1 or level 2 multicenter studies every year sponsored by the membership. Each study aims to answer an important question about patient care. The studies are presented at the annual meeting of the society and are later published. This type of initiative for the AAOS would be a welcome addition to their many activities.
As the impact of the CPG development continues to affect our abilities to provide the best care we believe is appropriate for patients, we should be motivated to become more involved in the debate about what members of our profession best represent orthopedic surgeons on the CPG development panels. We also need to be involved in the methods used to select the data to be analyzed, the proper use of mathematical estimates and manipulations to understand the data, and then the proper words to describe appropriate use of the current information. Other parties can prioritize the economics while we focus on providing the best musculoskeletal care available.