IntroductionThe 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.MethodsIncorporating 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.ConclusionCapturing 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.FundingStealth BioTherapeutics Inc.

Introduction We have previously demonstrated that assisted partner services (aPS) increases HIV testing and case finding among partners of persons living with HIV (PLHIV) in a cluster randomized trial in Kenya. However, the efficacy of aPS may vary across populations. In this analysis, we explore differences in aPS efficacy by characteristics of index participants. Methods Eighteen HIV testing sites were randomized to immediate versus 6‐week delayed aPS. Participants were PLHIV (or index participants) and their sexual partners. Partners of index participants were contacted for HIV testing and linked to care if HIV positive. Primary outcomes were the number of partners per index participant who: 1) tested for HIV, 2) tested HIV positive and 3) enrolled in HIV care. We used generalized estimating equations to assess differences in aPS efficacy by region, testing location, gender, age and knowledge of HIV status. Results From 2013 to 2015, the study enrolled 1119 index participants, 625 of whom were in the immediate group. These index participants named 1286 sexual partners. Immediate aPS was more efficacious than delayed aPS in promoting HIV testing among partners in high compared to low HIV prevalence regions (Nyanza incidence rate ratio (IRR) 7.2; 95% confidence interval (CI) 5.4, 9.6 vs. Nairobi/Central IRR 3.4 95% CI 2.3, 4.8). Higher rates of partner HIV testing were also observed for index participants in rural/peri‐urban compared to urban sites (IRR 6.6; 95% CI 4.5, 9.6 vs. IRR 3.5 95% CI 2.5, 5.0 respectively), for female versus male index participants (IRR 5.8 95% CI 4.2, 7.9 vs. IRR 3.7; 95% CI 2.4, 5.8 respectively) and for newly diagnosed versus known HIV‐positive index participants (IRR 6.0 95% CI 4.2, 8.7 vs. IRR 3.3; 95% CI 2.0, 7.7 respectively). Providing aPS to female versus male index participants also had a significantly higher HIV case finding rate (IRR 9.1; 95% CI 4.0, 20.9 vs. IRR 3.2 95% CI 1.7, 6.0 respectively.) Conclusions While it is known that aPS promotes increases in HIV testing and case finding, this is the first study to demonstrate significant differences in aPS efficacy across characteristics of the index participant. Understanding these differences and their drivers will be critical as aPS is brought to scale in order to ensure all PLHIV have access to these services.

Background Geosocial networking (GSN) app use among men who have sex with men (MSM) has presented new opportunities for increasing the reach and efficiency of sexual health interventions but also poses challenges to HIV/sexually transmitted disease partner notification. Understanding MSM's attitudes toward app-based preventive sexual health services can help inform their development and delivery. Methods We recruited US MSM who had met a sex partner on GSN apps in the last year to participate in an online survey assessing acceptability and preferences regarding app-based partner notification, health department presence, and sexual health services. Three app-based notification strategies were presented: sending notification messages through participant's/partner's app profile, health department app profile, or in-app anonymous messaging. Results Of 791 respondents, a majority (70%) preferred to be notified by their partner directly; however, most would get tested if notified by health department profile (95%) or anonymous in-app message (85%). Given the options provided, 50% preferred notifying a partner using their own profile, 26% with health department assistance, and 24% via in-app anonymous message. A majority (71%) were comfortable notifying a partner through a health department profile, and 74% were comfortable using in-app anonymous messaging. Most participants (82%) were comfortable with health departments having app profiles to provide sexual health services. Conclusions Our results suggest that GSN app–based partner notification and sexual health services would be used by and are acceptable to US MSM. Partnering with app companies to integrate these services and increase access to public health programs has potential to improve MSM sexual health.

Meeting sex partners through geosocial networking (GSN) apps is common among men who have sex with men (MSM). MSM may choose not to exchange contact information with partners met through GSN apps, limiting their own and health departments' ability to notify partners of HIV/STD exposure through standard notification methods. Using online focus groups (four groups; N = 28), we explored the perspectives of U.S. MSM regarding offer of partner notification features through GSN apps. Most participants were comfortable with HIV/STD partner notification delivered via GSN apps, either by partner services staff using a health department profile or through an in-app anonymous messaging system. While most participants expressed a responsibility to notify partners on their own, app-based partner notification methods may be preferred for casual or hard-to-reach partners. However, participants indicated that health departments will need to build trust with MSM app users to ensure acceptable and effective app-based delivery of partner notification.

Introduction The Duchenne Video Assessment (DVA) assesses quality of movement as an indication of Duchenne muscular dystrophy (DMD) disease severity. Caregivers video record patients performing home‐based movement tasks using a mobile application, and physical therapists (PTs) rate the videos using scorecards with prespecified compensatory movement criteria. Reliability and construct validity of the DVA were tested using video and Pediatric Outcomes Data Collection Instrument (PODCI) data from patients with DMD and healthy controls from a separate study. Methods Fifteen PTs were trained and certified as DVA raters. All raters scored videos of five subjects performing each movement task; nine raters rescored the same videos four weeks later. Three raters scored videos from an average of 25 subjects for each movement task. Aggregate scores were used to test construct validity. An expert DMD clinician assigned each video to a severity group for known‐groups analyses. Differences between rater scores across severity groups were tested and correlations between DVA and PODCI scores were calculated. Results Inter‐rater reliability (intraclass correlation coefficient [ICC]) between all 15 raters ranged from 0.70 to 0.97 for all movement tasks. Mean intra‐rater reliability ICC for nine raters ranged from 0.82 to 0.98 for all movement tasks. There were statistically significant differences between known severity groups for all movement tasks. The DVA correlated strongly with related PODCI constructs of physical function and weakly with unrelated constructs. Discussion The DVA was found to be a reliable and valid tool for measuring quality of movement as an indication of disease severity.