Standards of Care for DMD - Assessing Efficacy
Guidelines for Standards of care (SoC) have several objectives. The primary one is to aid individuals with DMD get the most appropriate care, providing both individuals/families and professionals with recommended assessments and interventions on the individual level. SoC is also an important aid in improving multi-centre clinical trials by providing a joint basis for diagnosis and care, which in turn is an aid for analysing outcome of a trial. In order for these objectives to be relevant, we need to know both at an individual and collective level what the outcome of SoC recommendations are. Developing SoC is thus only the first step. The SoC then need to be made accessible, implemented, revised and updated over time, and importantly monitored and reviewed to ensure efficacy. Work is already under way to address some of the other essential elements in appraising SoC, such as assessing implementation. This discussion focuses on the complementary task of assessing the outcome and efficacy of the standards of care we implement. It begs the question; is outcome assessment something that should be addressed in clinical guidelines? If so, what specific issues do those guidelines need to address?
We have in TREAT-NMD worked in parallel with issues related to SoC (focus on patient care), and issues related to outcome measures (focus on clinical trials). The intention has been to integrate these two with each other, and this is a good time to discuss if and how this best be done. In order to best do this, a brief background on the TREAT-NMD work on developing a Registry of Outcome Measures (ROM) is appropriate. The ROM is an on-line tool (www.researchrom.com) that may help to give guidance, information and assistance when undertaking the crucial task of choosing the right outcome measures (OMs) for the job at hand.
Some initial questions come to mind related to tools to use to appraise the efficacy if SOC, each of which can be expanded with several follow-up questions once we know more “how the wind blows”:
1. Should we for SoC in general integrate knowledge and experience from parallel work on outcome measures, e.g. ROM?
2. Should we for individual assessments presently recommended in the SoC for DMD integrate information from ROM? (Could be done e.g. in a web version where SoC assessment recommendations are linked to ROM info on that specific assessment tool)
3. Should we in the SoC recommendations for individual assessment make an effort to find if there is a need to add tools from ROM presently lacking in the SoC (e.g. function tests etc)?
4. Should we make an effort in identifying (e.g. from ROM) assessments / outcome measures to recommend utilizing in order to follow efficacy of SoC at a group level (to be used e.g. as optional items in the DMD registry)?
We would be very interested in hearing your thoughts on this topic and relaying them to the community via this website to inform the discussion. While the focus of this discussion is DMD, we recognise synergies with other diseases and are therefore interested in comments from the broader community.
If you are interested in providing comment, please email Thomas Sejersen and Jo Auld.
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