Evaluating Patient-Centered Outcomes In Clinical Trials Of Procedural Sedation
Part 1 Efficacy: Sedation consortium on endpoints and procedures for treatment, education, and research recommendations.
Authors: Williams MR, Ward DS, Carlson D, Cravero J, Dexter F, Lightdale JR, Mason KP, Miner J, Vargo JJ, Berkenbosch JW, Clark RM, Constant I, Dionne R, Dworkin RH, Gozal D, Grayzel D, Irwin MG, Lerman J, O’Connor RE, Pandharipande P, Rappaport BA, Riker RR, Tobin JR, Turk DC, Twersky RS, Sessler DI. Anesth Analg 2017; 124:821-30.
Reviewer: Patricia D. Scherrer MD
The goal of this consensus-driven paper was to provide a core set of outcome domains and measures for evaluating clinical trials of drugs, delivery systems, or protocols for procedural sedation in terms of efficacy and patient/family centered measures. This SCEPTER consortium group was appointed by the leadership of ACTTION, which is a public-private partnership with the Critical Path Initiative of the FDA. The goal of ACTTION is to “identify, prioritize, sponsor, coordinate, and promote innovative activities, with a special interest in optimizing clinical trials, that will expedite the discovery and development of improved analgesic, anesthetic, addiction, and peripheral neuropathy treatments for the benefit of public health.” The SCEPTER group specifically focuses on procedural sedation, and it will address defining a similar core set of outcomes measures in the domain of safety in a subsequent publication.
The SCEPTER consortium group, comprised of 27 experts from anesthesiology, procedural sedation, dentistry, emergency medicine, and gastroenterology as well as several individuals from the FDA, met in April 2014 to deliberate and reach consensus. All participants were sent a variety of articles to review that illustrated the diversity of available outcomes measures in terms of efficacy and patient/family satisfaction. Of note, these articles included the Dartmouth Operative Conditions Scale paper and the original PSRC publication regarding overall adverse events from 2006. This publication represents a summary of notes, presentations, and revisions based on the consensus meeting.
In defining the initial domains of sedation outcomes that should be investigated, the consortium chose to follow the construct of the Institute of Medicine’s “Crossing the Quality Chasm.” It was decided that only four of the six aims were relevant to procedural sedation randomized controlled trials: safety, efficacy, patient/family centered care, and efficiency. The group recommended that all procedural sedation clinical trials include these four outcome domains in study planning and design. The SCEPTER participants defined each of these four IOM aims in terms of their concept of interest for procedural sedation clinical trials:
Safe – Avoid physical or psychological harm.
Effective – Proceduralist is satisfied with sedation, including patient cooperation, no more movement than is appropriate for a given procedure, and sedation allowed for successful completion of the procedure.
Patient and family centered – Patient, family, or surrogate is satisfied with sedation, including adequate analgesia, amnesia as appropriate, lack of nausea or vomiting, and absence of adverse psychological sequelae.
Efficient – Provision of sedation is economical from the patient, institutional, and societal perspectives.
Again, this Part 1 evaluation focused on sedation efficacy, utilizing proceduralist satisfaction, validated sedation effectiveness measures, and measures of patient/family satisfaction as primary measures of efficacy. Proceduralist and patient/family satisfaction measures were felt to be advantageous because they are universally applicable to permit comparisons across populations (including children and adults) and across a variety of procedures. For children, the group recommended use of the University of Michigan Sedation Scale to assess the level of sedation for ongoing comparisons. The Observer’s Assessment of Alertness and Sedation scale was recommended for adults. However, the authors acknowledged that such scales are less useful for non-painful procedures such as radiographic imaging since they are predicated upon response to patient stimulation and that, for these types of procedures, observational scales are likely more appropriate. The need for additional sedative/analgesic or rescue medications was also noted to be important in determining overall efficacy.
With regard to proceduralist satisfaction, patient cooperation, lack of unwanted/disruptive movement, and apparent analgesia were all considered important attributes for evaluation. The authors recommended the Clinician Satisfaction with Sedation Instrument as the only adequately validated scale to measure proceduralist satisfaction. However, especially in children, they suggested incorporating objective assessments of intra-procedure movement and distress, utilizing scoring systems such as the Dartmouth Operative Conditions Scale and the Children’s Hospital of East Ontario Pain Scale.
Finally, in the assessment of patient and family satisfaction, the consortium group advised evaluating perceived pain, unpleasant recall, resumption of normal activities, and other items important to patients and families. The authors found that there are no currently published, validated questionnaires or satisfaction scales specific to patient/family satisfaction in procedural sedation for children. The previously mentioned scoring systems for assessment of intra-procedure distress with regard to proceduralist satisfaction were also advised for assessment of perceived pain, in addition to more traditional pediatric pain rating metrics such as the revised Faces pain scale. The modified Brice score has been validated in adults for recall assessment after general anesthesia. Analogous to satisfaction scales for procedural sedation in children, the authors did not find any measures that have been evaluated specifically to address recall in children.
Procedural sedation clinical trials should incorporate assessment of four key outcome domains: efficacy and effectiveness, safety, patient/family outcomes, and efficiency. With regard to efficacy and patient/family experience, the consortium group recommended specific core outcome measures for level of sedation, proceduralist satisfaction, patient/family satisfaction, occurrence of recall, and adequacy of analgesia. They advised that investigators incorporate these outcome measures when appropriate for development of clinical trial objectives and testing of specific sedation regimens or procedures. And, due to the limited number of currently existing measures to assess pediatric procedural sedation efficacy in particular, they endorsed a clear and immediate need to pursue development and validation of such measures for children.
Comparing apples to oranges is one thing; comparing apples to apples requires an agreed upon set of vocabulary and definitions to ensure valid and reliable comparisons between key characteristics. Sedation outcomes studies have long been lacking in this standardized vocabulary, limiting the ability to compare outcomes and perform meta-analyses. The SCEPTER group has been convened to develop consensus definitions and guidelines that should be incorporated into clinical trials that evaluate sedation and procedure-specific sedation regimens - so that apples really can be compared to other apples. Utilization of the Institute of Medicine’s 6 aims to establish construct domains for evaluation of sedation quality is becoming more prevalent, and this group’s continuation of that use fits in well with previous sedation quality work, including that of the SPS.
This first report from the SCEPTER group addresses the domains of effectiveness and patient/family centered care. As is often the case, pediatric-specific metrics and guidelines are lacking in many of these areas. Fortunately, there is work currently in progress by a number of SPS members to fill in these gaps. The Dartmouth Operative Conditions Scale goes a long way in helping us to objectively compare the intra-procedure sedation experience. Work is underway on a robust patient and family satisfaction score for pediatric procedural sedation that should help this critically important domain to be taken more seriously in the consideration of overall quality – and in the value equation. Amnesia relative to procedural sedation is not separately assessed by any current pediatric metric; this might be a fruitful area for new research moving forward.
As this report represents a summary of consensus among experts in the procedural sedation world, the validity of the construct, i.e. utilization of the IOM six aims and development of associated metrics and definition recommendations, is not in and of itself evidence based. However, the metrics themselves have been largely well validated, and even the simple agreement as to which scales or scores should be utilized to report domains such as effectiveness will go a long way towards establishing congruity between studies. Similar consensus statements from this expert group should be available in the near future regarding the domains of safety and efficiency.
Interestingly, the SCEPTER group decided that timeliness and equity were not relevant outcomes domains for procedural sedation studies because they were felt to be “generally a function of clinical care environments rather than research per se.” As the SPS has already discussed in the “Great Expectations” consensus paper from 2015, reducing delays in and barriers to accessing high quality pediatric procedural sedation care are also meaningful areas for further study, especially in the ever-changing political climate surrounding the provision of health care to children.
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