Methodology for Production of Best Practice Guidelines for Rare Diseases, by T. Sejersen, C. Del Giovane, G. Filippini, C. Giacomo Leo, J. J. Meerpohl, P. Mincarone, S. Minozzi, S. Sabina, H. Schunemann, J. Senecat, D. Taruscio, and the RARE-Bestpractices Consortium. Rare Diseases and Orphan Drugs. 2014;1:10.
Background and Purpose: Patients and providers benefit from best practice guidelines for clinical management of conditions. These guidelines allow providers to make recommendations based on best available evidence, help patients ensure that their treatment is provided according to evidence-based recommendations, and provide a common foundation for diagnosis and management of conditions, thereby facilitating multicenter clinical trials. Best practice guidelines may be particularly important for rare diseases, whether expertise is scarce, misdiagnosis is common, and clinical trials require multicenter recruitment to obtain adequate sample sizes. However, best evidence for rare disease is often based on small trials with variable quality and/or on expert opinion. To move forward with development of best practice guidelines for rare conditions, there is a need for better understanding of what guidelines already exist, development of a platform for storage and dissemination of guidelines, and agreement as to the methodological principles that are universal and should be employed when developing new guidelines. This article, which is part of the RARE-Bestpractices EU project, outlines plans for achievement of 4 aims: (1) collect and describe currently available best practice guidelines for rare diseases; (2) describe existing methodology for development of best practice guidelines and come to consensus on quality standards to use for future guideline development in rare diseases; (3) use agreed-upon methods to produce pilot best practice guidelines for 1 or 1 rare conditions, including development of a patient version of the guideline; and (4) devise a method for graphically representing best practice guideline decision logic algorithms to facilitate implementation of guidelines into practice.
Method: Each of the 4 aims has its own methodology. (1) The current state of guidelines for rare diseases will be determined via an online survey sent to health ministries, as well as local and national agencies responsible for developing best practice guidelines throughout Europe. Results of the survey will be qualitatively and quantitatively evaluated. (2) Consensus on methodology for development of guidelines will be achieved through application of the GRADE Working Group approach to evaluate several examples of health care questions related to rare diseases. For each question, profiles of available evidence and recommendation frameworks will be developed through a series of workshops and online exchanges. The group will then assess the processes employed in addressing these exemplar questions and make final recommendations of methodology to use for future development of best practice guidelines for rare disease. (3) One or 2 pilot guidelines will be developed using the methodology and quality guidelines set forth in aim 2. The group will ensure that methodology places the patients' needs at the center of the process and promotes a multidisciplinary approach. The process will include a group of patients to ensure that patients' perspectives are represented throughout, and a patient version of the pilot guideline will be developed using language adapted for patients and families. (4) A graphical representation method for illustrating sequencing and decision algorithms for implementation of guidelines in practice will be developed using formal modeling of workflow and care pathways. The method will be employed to develop a graphical representation of the pilot guideline(s) developed in aim 3.
Results: To be determined.
Conclusions: Evidence to inform treatment of rare disease is limited, and best practice guidelines are few and far between. The work proposed herein could greatly facilitate future development of best practice guidelines that are based on a common methodology, useful to providers and patients alike, and designed in a manner that facilitates implementation. Ultimately, these guidelines could enhance diagnosis and care for individuals with rare diseases.
Gammon M. Earhart, PT, PhD
Washington University in St. Louis
St. Louis, Missouri
Canadian Stroke Best Practice Recommendations: Stroke Rehabilitation Practice Guidelines, Update 2015, by D. Hebert, M. P. Lindsay, A. McIntyre, A. Kirton, P. G. Rumney, S. Bagg, M. Bayley, D. Dowlatshahi, S. Dukelow, M. Garnhum, E. Glasser. International Journal of Stroke. 2015;11(4):459-484.
Background and Purpose: Each year an estimated 62 000 individuals experience a stroke or transient ischemic attack in Canada. As a result, patients often require rehabilitation to address persistent deficits. Rehabilitation may begin in the acute care setting and can continue in a range of settings including inpatient, home care, outpatient, and community settings. Reports of variability in practice exist. These practice guidelines are the fifth update of the Canadian Stroke Best Practice Guidelines, developed to provide comprehensive, evidence-based recommendations regarding stroke rehabilitation. They are directed toward health care professionals across various practice settings, with the intent that these recommendations will reduce practice variations, increased standardization of care, and improve outcomes for individuals following stroke.
Method: Rehabilitation experts were recruited to participate in systematically identifying and reviewing literature, as well as writing and revising recommendations. Individuals who had experienced a stroke or their family members were also included as group members or external reviewers. The systematic literature searches and analysis were conducted by experienced staff of the Evidence Based Review of Stroke Rehabilitation research group and were not part of the guideline writing group. Literature searches were built upon prior guideline searches and included additional time frames from 2012 to 2015. The writing groups discussed the literature and by consensus came to draft best practice recommendations. All recommendations were assigned a level of evidence ranging from A to C. The writing group's draft of recommendations underwent an internal review by the Canadian Stroke Best Practices Advisory Committee and an external review by 20 experts in stroke rehabilitation not involved in the guideline development prior to finalization.
Results: The guidelines present comprehensive recommendations related to the organization and delivery of stroke rehabilitation (Initial Rehab Assessment; Stroke Rehab Units; Stroke Rehab Teams; Delivery of Inpatient Stroke Rehab; Delivery of Outpatient and Community-Based Rehab; Early Supported Discharge) as well as specific clinical interventions (Upper Extremity Dysfunction; Lower Extremity Dysfunction including mobility, balance, transfers, aerobic training, gait aids, spasticity management and falls prevention; Dysphagia and Nutrition; Visual-Perceptual Deficits; Central Pain; Language and Communication; Resumption of Life Roles and Activities). A major addition includes recommendations specific to children from newborn to the age of 18 years.
Conclusions: The guidelines are presented in a concise and easy-to-read format that allows clinicians to efficiently locate a topic and associated recommendations. In daily practice, clinicians can easily access information to guide an evidence-based plan of care for a patient (eg, What interventions are appropriate to address lower extremity spasticity in my patient?). Within a department or institution, these guidelines could assist with implementing best practice across various disciplines (eg, Are there ways in which we, as an interdisciplinary team, can prevent hemiplegic shoulder pain and subluxation?). Clinicians outside of Canada should take into account potential differences in health care systems when applying recommendations related to the organization of stroke rehabilitation systems and delivery of care. For example, with a median length of stay of 4 days for acute ischemic stroke in the United States as compared with 12 days in Canada, one should consider the potential differences in patients identified for Early Supported Discharge and develop appropriate plans to ensure safe and effective transitions. Given the heterogeneity of stroke rehabilitation in the United States, there are often many factors that can influence access to rehabilitation, and rehabilitation services can vary widely in terms of intensity, duration, and interventions provided. Stroke rehabilitation guidelines are important resources in increasing standardization of care and improving outcomes.
Kristin Parlman, PT, DPT, NCS
Massachusetts General Hospital
Updating Contextualized Clinical Practice Guidelines on Stroke Rehabilitation and Low Back Pain Management Using a Novel Assessment Framework That Standardized Decisions, by E. J. Fambito, C. B. Gonzalez-Suarez, K. A. Grimmer, C. M. Valdecanas, J. M. Dizon, M. E. Beredo, M. T. Zamora. BMC Research Notes. 2015; 8:643.
Background and Purpose: Clinical practice guidelines are being developed in an attempt to improve care and, in some cases, to effect payment for rehabilitation services. Developing and updating clinical practice guidelines (CPGs) is costly, and there is little information to guide CPG updates in developing countries. The purpose of the study was to develop a tool that could be used to update CPGs.
Method: Thirty-five physical medicine and rehabilitation physicians (25% of those who had previously participated in development of a CPG) were taught about CPGs, participated in discussions about how to update CPGs, and then developed a tool to update guidelines. The authors chose to evaluate and update the low back pain and stroke guidelines previously developed by Philippine Academy of Rehabilitation Medicine. The participants developed a method to consistently categorize recommendations so that comparisons could be made between different CPGs in the literature. A search was conducted via various databases to identify low back pain or stroke CPGs written after 2011. Search criteria for inclusion and exclusion of CPGs were described. The International Centre for Allied Health Evidence (iCAHE) criteria were used to evaluate and compare the quality of the included CPGs. All guidelines had to have a score of 71% or better on the iCAHE criteria to be included.
Results: Eleven low back pain and 10 stroke guidelines were reviewed with the iCAHE criteria. Of the 11 low back pain guidelines, 1 was not in English, 2 were not available, and 1 was not a “new” guideline. Of the remaining 7 guidelines, 3 did not meet the iCAHE criteria score of 71% or better, leaving the authors with 4 low back pain CPGs. Of the 10 stroke guidelines initially included, 3 had no mention of rehabilitation, 1 did not rank the quality of the evidence, and another had a score on the iCAHE of less than 71% or better, resulting in 5 included stroke CPGs in the final review.
After critically appraising the included guidelines, the authors utilized their updated evidence to keep their previous recommendations, to strengthen the evidence within their recommendations, to identify that there is inconsistent evidence or a lower level of evidence based on the previous recommendations, or to report that the new evidence is not consistent with what was previously reported in their low back pain and stroke guidelines.
Conclusions: The authors developed a standardized and well-defined framework to assist authors in the revision of published guidelines. The framework was designed to decrease the cost of guideline revision and to ensure that only quality guidelines were utilized in guideline revisions. The methods described to update CPGs have value in developing countries, as they minimize costs and help standardize decision making about the value of recent guidelines.
Susan L. Whitney, DPT, PhD, NCS
University of Pittsburgh