Expediting treatments in the 21st century: orphan drugs and accelerated approvals.

Background: In response to activated patient communities' catalyzation, two significant efforts by the FDA to expedite treatments have now been in place for multiple decades. In 1983, the United States Congress passed the Orphan Drug Act to provide financial incentives for development of drugs for rare diseases. In 1992, partly in response to the HIV epidemic, the FDA implemented Accelerated Approval (AA) to expedite access to promising new therapies to treat serious conditions with unmet medical need based on surrogate marker efficacy while additional clinical data is confirmed.

Family caregivers of service members in United States Department of Defense health care report impairment in longitudinal health outcomes.

Objective: To examine elevated symptoms on health-related quality of life (HRQOL) measures over 2 years in caregivers of service members with traumatic brain injury (TBI). To compare outcomes to caregivers of veterans.

Method: Caregivers ( = 315) were classified into two groups: (a) service member caregiver group ( = 55) and (b) veteran caregiver group ( = 260).

Low resilience is associated with worse health-related quality of life in caregivers of service members and veterans with traumatic brain injury: a longitudinal study.

Purpose: To examine [a] the association of caregiver health-related quality of life (HRQOL) and service member/veteran (SMV) neurobehavioral outcomes with caregiver resilience; [b] longitudinal change in resilience at the group and individual level; and [c] the magnitude of change at the individual level.

Methods: Caregivers (N = 232) of SMVs with traumatic brain injury completed a resilience measure, and 18 caregiver HRQOL and SMV neurobehavioral outcome measures at a baseline evaluation and follow-up evaluation three years later. Caregivers were divided into two resilience groups at baseline and follow-up: [1] Low Resilience (≤ 45 T, baseline n = 99, follow-up n = 93) and [2] High Resilience (> 45 T, baseline n = 133, follow-up n = 139).

The COPD Foundation on Its Twentieth Anniversary.

This article does not contain an abstract.

Longitudinal health-related quality of life in military caregivers no longer providing care.

Purpose/objective: To examine health-related quality of life (HRQOL) in caregivers when providing care and no longer providing care to service members/veterans with traumatic brain injury.

Research Method/design: Participants included 466 caregivers enrolled in a 15-year longitudinal study. During an annual follow-up evaluation, a subsample of caregivers self-identified as no longer providing care and were retained in the study as a No Longer Caregiving group ( = 48).

Health outcomes in caregivers of service members and veterans with traumatic brain injury enrolled in the U.S. Veterans Affairs Caregiver Support Program.

To explore health outcomes in caregivers of service members and veterans (SMV) with traumatic brain injury (TBI) enrolled in two programs within the U.S. Department of Veterans Affairs (VA) Caregiver Support Program (CSP) (General and Comprehensive Programs) and those not enrolled.

A Hybrid Effectiveness/Implementation Clinical Trial of Adherence to Long-Term Oxygen Therapy for Chronic Obstructive Pulmonary Disease.

Interventions to promote adherence to long-term oxygen therapy (LTOT) in chronic obstructive pulmonary disease (COPD) are needed. To examine the real-world effectiveness of phone-based peer coaching on LTOT adherence and other outcomes in a pragmatic trial of patients with COPD. In a hybrid effectiveness/implementation pragmatic trial, patients were randomized to receive phone-based proactive coaching (educational materials, five phone-based peer coaching sessions over 60 d), reactive coaching (educational materials, peer coaching when requested), or usual care.

Health outcomes before and during the COVID-19 pandemic in caregivers of service members and veterans with traumatic brain injury.

Purpose: To examine change in health-related quality of life (HRQOL) during the COVID-19 pandemic in caregivers of service members/veterans (SMVs) with traumatic brain injury (TBI), by comparing HRQOL during the first year of the pandemic to HRQOL 12 months pre-pandemic.

Methods: Caregivers (N = 246) were classified into three COVID-19 Pandemic Impact groups based on impact ratings of the pandemic on HRQOL: No Impact (n = 50), Mild Impact (n = 117), and Moderate-Severe Impact (n = 79). Caregivers completed 19 measures across physical, social, caregiving, and economic HRQOL domains, and a measure of SMV Adjustment.

Caregiver sleep impairment and service member and veteran adjustment following traumatic brain injury is related to caregiver health-related quality of life.

Study Objectives: To examine the relationship between caregiver sleep impairment and/or service member/veteran (SMV) adjustment post-traumatic brain injury, with caregiver health-related quality of life (HRQOL).

Methods: Caregivers (n = 283) completed 18 measures of HRQOL, sleep impairment, and SMV adjustment. Caregivers were classified into 4 sleep impairment/SMV adjustment groups: 1) Good Sleep/Good Adjustment (n = 43), 2) Good Sleep/Poor Adjustment (n = 39), 3) Poor Sleep/Good Adjustment (n = 55), and 4) Poor Sleep/Poor Adjustment (n = 146).

Effect of Two Interventional Strategies on Improving Continuous Positive Airway Pressure Adherence in Existing COPD and Obstructive Sleep Apnea Patients: The O2VERLAP Study.

Background: Obstructive sleep apnea (OSA) is a sleep disorder prevalent in >10% of individuals diagnosed with chronic obstructive pulmonary disease (COPD). Continuous positive airway pressure (CPAP) is the first-line therapy for OSA, but many do not use it enough during sleep to effectively manage OSA. The OVERLAP study compared (PC)-structured web-based peer-coaching education and support intervention versus (RC)-education and support based on limited scheduled interactions and patient-initiated contacts.

The national economic burden of rare disease in the United States in 2019.

Background: To provide a comprehensive assessment of the total economic burden of rare diseases (RD) in the United States (U.S.) in 2019.

Common Challenges and Identified Solutions for State Newborn Screening Programs during COVID-19 Pandemic.

During the COVID-19 pandemic, state newborn screening programs faced challenges to ensure this essential public health program continued to function at a high level. In December 2020, the EveryLife Foundation for Rare Diseases held a workshop to discuss these common challenges and solutions. Newborn screening officials described challenges including short staffing across the entire program, collection and transport of specimens, interrupted follow-up activities, and pilot study recruitment.

Outcomes Important to Patients Diagnosed with Both COPD and Sleep Apnea: Findings from the O2VERLAP Study Focus Groups.

Introduction: Few studies have asked chronic obstructive pulmonary disease (COPD) -obstructive sleep apnea (OSA) overlap syndrome patients what outcomes of continuous positive airway pressure (CPAP) therapy are important to them, while also considering their self-reported CPAP adherence barriers and facilitators. This study conducted a series of focus groups to learn about those issues with the goal of applying these findings to the design of a larger Patient-Centered Outcomes Research Institute-funded scientific study, the O2VERLAP Study.

Methods: People previously diagnosed with both COPD and OSA, who were current or recent past users of the standard therapy for each condition (i.

Post-Traumatic Stress Disorder Symptoms Are Related to Cognition after Complicated Mild and Moderate Traumatic Brain Injury but Not Severe and Penetrating Traumatic Brain Injury.

Although post-traumatic stress disorder (PTSD) has been associated with worse cognitive outcomes after mild traumatic brain injury (TBI), its impact has not been evaluated after more severe TBI. This study aimed to determine whether PTSD symptoms are related to cognition after complicated mild, moderate, severe, and penetrating TBI. Service members ( = 137) with a history of complicated mild/moderate TBI ( = 64) or severe/penetrating TBI ( = 73) were prospectively enrolled from United States Military Treatment Facilities.

Unhealthy family functioning is associated with health-related quality of life among military spouse caregivers.

Objective: The current study examines health-related quality of life (HRQOL) and family functioning in a sample of spouse caregivers assisting post-9/11 service members and veterans (SMV) following traumatic brain injury (TBI).

Method: Participants were 316 spouse (and partner) caregivers of SMVs following a mild, moderate, severe, or penetrating TBI. Caregivers completed the Family Assessment Device General Functioning subscale, 24 HRQOL questionnaires, and the Mayo-Portland Adaptability Inventory (4th ed.

Prioritization and Refinement of Patient-Informed Value Elements as Attributes for Chronic Obstructive Pulmonary Disease Treatment Preferences.

Background And Objective: Formative research studies can inform stated-preference instrument development to quantify the importance of various attributes of healthcare treatments. The objective of this study was to elicit from patients with chronic obstructive pulmonary disease the prioritization of an established set of patient-informed value elements.

Methods: Using an iterative mixed-methods study design, we engaged individuals living with chronic obstructive pulmonary disease in Phase 1 value element elicitation and Phase 2 language refinement.

Post-Traumatic Stress Disorder Is Associated with Neuropsychological Outcome but Not White Matter Integrity after Mild Traumatic Brain Injury.

The aim of this study was to examine neuropsychological functioning and white matter integrity, in service members and veterans (SMVs) after mild traumatic brain injury (MTBI), with versus without post-traumatic stress disorder (PTSD). Participants were 116 U.S.

New Adopters of Telemedicine During the Coronavirus-19 Pandemic in Respondents to an Online Community Survey: The Case for Access to Remote Management Tools for Individuals with Chronic Obstructive Pulmonary Disease.

Objectives: To investigate telemedicine adoption, emergency department avoidance, and related characteristics of patients with chronic obstructive pulmonary disease (COPD) with and without exacerbations since the coronavirus 2019 (COVID-19) pandemic began.

Methods: We conducted the second of a series of online surveys via SurveyMonkey.com of people with COPD between May 1, 2020 and May 31, 2020.

Resilience is associated with health-related quality of life in caregivers of service members and veterans following traumatic brain injury.

Purpose: To examine factors related to resilience in military caregivers across caregiver health-related quality of life (HRQOL), caregiver sociodemographic variables, and service member/veteran (SMV) injury and health status.

Methods: Caregivers (N = 346, Female = 96.2%; Spouse = 91.

Longitudinal trajectories and risk factors for persistent postconcussion symptom reporting following uncomplicated mild traumatic brain injury in U.S. Military service members.

Objective: The purpose of this study was to examine individual postconcussion symptom [PCSx] trajectories following mild traumatic brain injury (MTBI), and to examine risk factors for persistent PCSx reporting.

Method: Participants were 138 U.S.

Design of the patient navigator to Reduce Readmissions (PArTNER) study: A pragmatic clinical effectiveness trial.

Previous work indicates the potential for community health workers and peer coaches serving as patient navigators to improve processes of care and health outcomes during care transitions, but have not been sufficiently tested to determine if such programs improve measures of patient experience in minority serving institutions. The objectives of the Patient Navigator to Reduce Readmissions (PArTNER) study was to: 1) conduct a pragmatic clinical effectiveness trial comparing a multi-faceted, stakeholder-supported Navigator intervention (in-person CHW visits in the hospital and after hospital discharge, plus telephone-based peer coaching) versus usual care on the experience of hospital-to-home care transitions in patients hospitalized with heart failure, pneumonia, chronic obstructive pulmonary disease, myocardial infarction, or sickle cell disease; 2) examine the effectiveness of the Navigator intervention in patient subgroups; and 3) understand the barriers and facilitators of successfully implementing the Navigator intervention across patient populations. The co-primary outcomes are the 30-day changes in: 1) Patient Reported Outcomes Measurement Information System (PROMIS) emotional distress-anxiety, and 2) PROMIS informational support.

Long-term neurobehavioural symptom reporting following mild, moderate, severe, and penetrating traumatic brain injury in U.S. military service members.

The purpose of this study was to examine long-term neurobehavioural symptom reporting following mild, moderate, severe, or penetrating TBI in U.S. military service members and veterans (SMV).