Cure SMA Launches New Surveys to Share Our Community’s Experience

By Cure SMA | Published On June 24, 2016

Cure SMA Launches New Surveys to Share Our Community’s Experience

Cure SMA is committed to addressing the needs of individuals and families affected by SMA. In order to accomplish this goal, we’ve been working for the past several years to collect data and information on the experiences of living with SMA, as well as our community’s goals, hopes and challenges.

This information can be collected via quantitative means such as data, surveys, and questionnaires, or qualitative means such as patient and family stories.

The Importance of Comprehensive Data from Our Community

Data collected from our community can be used in many ways. For example:

  • Shaping the direction of clinical trials for SMA, guiding our pharmaceutical partners to recruit participants efficiently, and designing trials with endpoints that are clinically meaningful to quickly determine whether the treatments are effective.
  • Communicating our community’s goals and priorities to the FDA and other regulators, such as our community’s viewpoints on benefit/risk and meaningful change in SMA patients’ lives.
  • Providing insight into daily life with SMA and the ongoing medical challenges. This information can be used by healthcare providers to better define the standard of care, and target interventions for the greatest impact.
  • Revealing aspects of SMA that are poorly understood, leading researchers to develop new tools, measurements and screening methods to better understand and measure these aspects.

Exploring Patient and Family Experience

Earlier this year, we expanded our ongoing data collection efforts by launching a survey tool for those newly diagnosed with SMA. The goal of this survey is to increase our understanding of the SMA at the time of diagnosis.

Though participation in this survey is voluntary, we encourage all families to complete it within six months of diagnosis. In the next several months, we will announce a second survey targeted toward individuals and families not newly diagnosed. Participants will be asked to update their information on a yearly basis. Our goal is to create a database that will demonstrate the impact of SMA over time.

By sharing your story and how SMA has impacted your lives, you will help the scientific and research communities create answers that address these real-world concerns, and accelerate therapy development for SMA.

Past Data Collection Efforts to Share our Community’s Experience

Particularly over the past three years, Cure SMA has been working on a number of fronts to ensure our community’s experiences are properly represented. These include:

We completed a survey project consisting of focus groups and interviews with 96 participants including: 21 with individuals with SMA; 64 parents of individuals affected by SMA; and 11 clinicians who specialize in the care of SMA patients. The goal of this project was to document the perspective of individuals and families living with SMA and to assess whether the currently used trial outcomes are meaningful to patients.

Cure SMA, along with collaborators from Biogen and the SMA Foundation, used this survey information to publish a paper in the journal BMC Neurology, reporting on patient and family experiences. In addition, a second paper from these focus groups is currently under review for journal publication. The second paper will focus on defining meaningful change in drug development, and determine critical parameters for measuring change from the patient perspective.

Parent Project Muscular Dystrophy (PPMD) and Cure SMA collaborated in two separate survey projects on clinical trials. One project focused on individuals whose children have participated in a clinical trial, including their motivations, hopes, and communication. The second focused on individuals whose children have not participated in a clinical trial, and the reasons for this lack of participation.

We brought the Voices of SMA project before key FDA leaders. Last year, we called upon our community to submit their experiences of living with SMA. Guided by themes identified through various data collection projects, we created The Voices of SMA, a booklet summarizing and highlighting these key themes through selected excerpts from your stories. This booklet, along with every submitted story, was distributed to all the top decision-makers at the FDA. As part of the PFDD (patient-focused drug development) initiative, these stories will influence the review process when an SMA drug is brought forward for approval.

Privacy Notice

Participation in any survey, focus group, or other patient voice project is entirely voluntary. You may complete all, some or none of questions asked in each exercise. Before deciding to participate, please take a moment to review this privacy notice. This explains how the information in the survey might be used, and also explains the steps that we take to safeguard your information:

This information may be shared with Cure SMA grant recipients, content providers, sponsors, service providers or other third parties to advance the goals of research, education, dissemination of information and otherwise in fulfillment of the goals of Cure SMA. Cure SMA uses Personal Information to assist in recruiting individuals into clinical studies and trials, providing aggregate disease statistics to third parties, providing de-identified individual patient disease information to third parties, contributing data to third party journal publications, and to refer patients to specialized SMA clinical centers. Such third parties are obligated to maintain the confidentiality of Personal Information and are not authorized to use such information for any purpose other than its intended purpose. Unless we have your consent or as required by law, Cure SMA will not share your Personal Information with any person or entity other than those affiliated with Cure SMA, entities acting on behalf of Cure SMA and relevant third parties such as Cure SMA research grant recipients. By submitting Personal Information through the Sites or to a Cure SMA contact, you authorize Cure SMA to share this Personal Information for the purposes identified above.

Topics: Support & Care, Our Impact, Advocacy, Front Page News

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