A National Spinal Muscular Atrophy Registry for Real-World Evidence.

BACKGROUND: Spinal muscular atrophy (SMA) is a devastating rare disease that affects individuals regardless of ethnicity, gender, and age. The first-approved disease-modifying therapy for SMA, nusinursen, was approved by Health Canada, as well as by American and European regulatory agencies following positive clinical trial outcomes. The trials were conducted in a narrow pediatric population defined by age, severity, and genotype. Broad approval of therapy necessitates close follow-up of potential rare adverse events and effectiveness in the larger real-world population. METHODS: The Canadian Neuromuscular Disease Registry (CNDR) undertook an iterative multi-stakeholder process to expand the existing SMA dataset to capture items relevant to patient outcomes in a post-marketing environment. The CNDR SMA expanded registry is a longitudinal, prospective, observational study of patients with SMA in Canada designed to evaluate the safety and effectiveness of novel therapies and provide practical information unattainable in trials. RESULTS: The consensus expanded dataset includes items that address therapy effectiveness and safety and is collected in a multicenter, prospective, observational study, including SMA patients regardless of therapeutic status. The expanded dataset is aligned with global datasets to facilitate collaboration. Additionally, consensus dataset development aimed to standardize appropriate outcome measures across the network and broader Canadian community. Prospective outcome studies, data use, and analyses are independent of the funding partner. CONCLUSION: Prospective outcome data collected will provide results on safety and effectiveness in a post-therapy approval era. These data are essential to inform improvements in care and access to therapy for all SMA patients

Ohio Federal Military Jobs Commission Report

APRI staff provided research support to the Ohio Federal Military Jobs Commission as they met and developed their final report on strengthening the state\u27s federal military jobs sector. APRI primarily provided research and authorship for Chapter 3 of the report, titled Expand Small Business Federal Contracting. Chapter 3 discusses the strategy to enhance communication and collaboration among State entities in order to create statewide response to the federal initiatives that make contracts available to small business and veteran-owned businesses. Placing Small Business Development Centers and Procurement Technical Assistance Centers on a common information technology netowrk and educating small businesses on how to do work for the federal government can help accomplish the Commission\u27s strategy. Chapter 3 also includes a recommendation for a state-level matching opportunity for Small Business Innovation Research.https://corescholar.libraries.wright.edu/apri_defpol/1004/thumbnail.jp

From Adherence to Self-Determination: Evolution of a Treatment Paradigm for People with Serious Mental Illnesses

Treatment adherence and nonadherence is the current paradigm for understanding why people with serious mental illnesses have low rates of participation in many evidence-based practices. The authors propose the concept of self-determination as an evolution in this explanatory paradigm. A review of the research literature led them to the conclusion that notions of adherence are significantly limited, promoting a value-based perspective suggesting people who do not opt for prescribed treatments are somehow flawed or otherwise symptomatic. Consistent with a trend in public health and health psychology, ideas of decisions and behavior related to health and wellness are promoted. Self-determination frames these decisions as choices and is described herein via the evolution of ideas from resistance and compliance to collaboration and engagement. Developments in recovery and hope-based mental health systems have shepherded interest in self-determination. Two ways to promote self-determination are proffered: aiding the rational actor through approaches such as shared decision making and addressing environmental forces that are barriers to choice. Although significant progress has been made toward self-determination, important hurdles remain. (Psychiatric Services 63:169–173, 2012; doi: 10.1176/appi.ps.201100065) Many people with serious mental illnesses do not seem to adhere to treatments as prescribed. In this Open Forum we propose that the concepts of self-determination and choice make greater sense of this phenomenon than the concept of treatment adherence and nonadherence. In 1990, one of us (PWC) coauthored an article published in this journal titled “From Noncompliance to Collaboration in the Treatment of Schizophrenia” (1). The article noted that many people with serious mental illnesses did not benefit from recommended practices, in part because they did not fully participate in them. The 1990 article sought to expand on outdated notions of resistance and compliance by framing treatment decisions about evidence-based practices as a collaborative partnership. Although the model described in the 1990 article was a substantial improvement over ideas of the time, it was nevertheless limited, and further shifts in conceptualizing this phenomenon were required. To support such shifts, we formed the Center on Adherence and Self-Determination (www.casd1.org), which is funded by the National Institute of Mental Health. The first five authors of this paper are co-principal investigators of the center. Self-determination is the crux of the new model, and choice is at the heart of self-determination. We begin this Open Forum by briefly recapping what research has shown—that many people with serious mental illnesses do not fully benefit from available evidence-based care. To make sense of this shortfall, we then consider the evolution of ideas in psychiatric practice, from resistance through collaboration to self-determination. The evolution paralleled a significant change in the mental health system, with themes of recovery, hope, and empowerment becoming more salient. The evolution also informs strategies for helping people decide which services will benefit them, and this Open Forum ends by describing decision-making processes

A Delphi-Based Consensus Statement on the Management of Anticoagulated Patients With Botulinum Toxin for Limb Spasticity.

Objective To create a consensus statement on the considerations for treatment of anticoagulated patients with botulinum toxin A (BoNTA) intramuscular injections for limb spasticity. Design We used the Delphi method. Setting A multiquestion electronic survey. Participants Canadian physicians (N=39) who use BoNTA injections for spasticity management in their practice. Interventions After the survey was sent, there were e-mail discussions to facilitate an understanding of the issues underlying the responses. Consensus for each question was reached when agreement level was ≥75%. Main Outcome Measures Not applicable. Results When injecting BoNTA in anticoagulated patients: (1) BoNTA injections should not be withheld regardless of muscles injected; (2) a 25G or smaller size needle should be used when injecting into the deep leg compartment muscles; (3) international normalized ratio (INR) level should be ≤3.5 when injecting the deep leg compartment muscles; (4) if there are clinical concerns such as history of a fluctuating INR, recent bleeding, excessive or new bruising, then an INR value on the day of injection with point-of-care testing or within the preceding 2-3 days should be taken into consideration when injecting deep compartment muscles; (5) the concern regarding bleeding when using direct oral anticoagulants (DOACs) should be the same as with warfarin (when INR is in the therapeutic range); (6) the dose and scheduling of DOACs should not be altered for the purpose of minimizing the risk of bleeding prior to BoNTA injections. Conclusions These consensus statements provide a framework for physicians to consider when injecting BoNTA for spasticity in anticoagulated patients. These consensus statements are not strict guidelines or decision-making steps, but rather an effort to generate common understanding in the absence of evidence in the literature