The Case for the Use of Patient and Caregiver Perception of Change Assessments in Rare Disease Clinical Trials: A Methodologic Overview.

INTRODUCTION: The traditional model of evaluating treatments based primarily on primary outcome measures has stumbled in its application to rare disease. Rare disease clinical trials face the methodological challenges of small, heterogeneous patient populations and relatively few validated, disease-specific outcome measures. Incorporating qualitative research into rare disease clinical trials may help sponsors, regulators, payers, and prescribers to better understand the real-world and patient-specific impact of a potential therapy. This paper provides a methodologic overview of the use of Patient and Caregiver Perception of Change (PPC and CPC) Assessments utilizing patient and caregiver video interviews to complement the data captured by traditional endpoints in rare disease clinical trials. METHODS: Incorporating qualitative patient and caregiver video interviews into clinical trials allows for the rigorous capture of patient experiences and caregiver observations. Interview guides informed by input from key stakeholders provide the opportunity to solicit structured feedback on experiences before, during, and after the clinical trial. Patients and caregivers can complete their video interviews in a study mobile application, and interview transcripts are analyzed by independent coders. Themes are summarized by the treatment group and individual patient, which adds context to the clinical outcome measures of how patients feel and function, as well as elucidates the degree of change that is meaningful to patients and caregivers. The qualitative results can be compared to the data captured in clinical trials to assess data concordance. CONCLUSION: Capturing patient experience data with sufficient rigor allows it to contribute to the body of evidence utilized in regulatory, payer, and prescriber decision-making. Adding PPC and CPC Assessments to rare disease clinical trials offers an innovative and powerful way to tap into the unique insights of patients and their families to develop a fuller picture of the patient experience in the clinical trial. FUNDING: Stealth BioTherapeutics Inc.

Activity-specific aquatic therapy targeting gait for a patient with incomplete spinal cord injury.

INTRODUCTION: Aquatic therapy can lead to improved mobility and health in individuals with various conditions. This case report looks at an activity-specific aquatic therapy targeting gait for a patient with incomplete spinal cord injury (iSCI). CASE REPORT: The patient participated in an individualized aquatic therapy program two times a week for 6 weeks. Assessment occurred prior to the intervention. There were two follow-up assessments after the intervention. Follow-up assessment 1 was completed within the same week as the final intervention. Follow-up assessment 2 was completed 4 weeks after the first follow-up to assess for carryover. RESULTS: Improvements that met minimal detectable change and minimal clinically important difference were noted in The Walking for Spinal Cord Injury Index-II, Spinal Cord Injury Functional Ambulation Index gait parameters, and gait speed. DISCUSSION: An activity-specific aquatic therapy program improved gait in a patient with iSCI. The properties of water create a practical environment for safe practice of skills. Further studies are warranted in large samples.

Interprofessional educational experience to assist in student readiness toward neurorehabilitation.

Occupational and physical therapy professionals are natural partners in neurorehabilitation especially in the acute stages of recovery. To enhance student-learning experiences from both disciplines, we have developed an interprofessional (IP) learning unit that was embedded in neurorehabilitation tracks of each respective program. The primary goal of this project was to examine interprofessional attitudes and perception toward interprofessional learning and practice. A total of 117 occupational therapy (53) and physical therapy (64) students completed an IP case-based learning module with the goal of developing an IP care plan typically seen in neurorehabilitation. Using a sequential explanatory mixed methods design, we collected data pre- and postintervention using the Readiness for Interprofessional Learning Survey (RIPLS). We also utilized a postintervention questionnaire and focus groups intended to solicit information regarding IP competencies. At pretest, students had high ratings on the RIPLS that led to minimal changes at posttest (p = .157). However, common threads from the questionnaire and focus groups showed positive effects on student learning and attitudes toward interprofessionalism. Students reported positive experiences with the IP module. An IP case-based educational module has the potential to facilitate professional development and readiness for future IP practice. Students from different disciplines appreciate the value of IP education (IPE) in reinforcing their professional identity and understanding the professional contributions of others. Our findings are consistent with literature on best practices for IPE.

The effects of the Nintendo™ Wii Fit on gait, balance, and quality of life in individuals with incomplete spinal cord injury.

PURPOSE: To assess the effects of virtual reality using the NintendoTM Wii Fit on balance, gait, and quality of life in ambulatory individuals with incomplete spinal cord injury (iSCI). RELEVANCE: There is a need for continued research to support effective treatment techniques in individuals with iSCI to maximize each individual's potential functional performance. SUBJECTS: Five males with a mean age of 58.6 years who had an iSCI and were greater than one-year post injury. METHODS: An interrupted time series design with three pre-tests over three weeks, a post-test within one week of the intervention, and a four-week follow up. Outcome measures: gait speed, timed up and go (TUG), forward functional reach test (FFRT) and lateral functional reach test (LFRT), RAND SF-36. Intervention consisted of one-hour sessions with varied games using the Nintendo Wii Fit twice per week for seven weeks. Survey data was also collected at post-test. Results There were statistically significant changes found in gait speed and functional reach. The changes were also maintained at the four-week follow up post-test. Survey reports suggested improvements in balance, endurance, and mobility with daily tasks at home. CONCLUSION: All subjects who participated in training with the NintendoTM Wii Fit demonstrated statistically significant improvements in gait speed and functional reach after seven weeks of training. Given the potential positive impact that the NintendoTM Wii Fit has on functional reach and gait speed in patients with iSCI, physical therapists may want to incorporate these activities as part of a rehabilitation program.