Adoption of Decentralization: Are Our Perceptions Holding Us Back?

Incorporating decentralized approaches into clinical trials is a critical innovation with potential implications for improved accessibility and diversity, as well as lower burden for participants and caregivers. As we move forward in a collective effort to modernize clinical trials, we consistently hear of hurdles that interfere with the adoption of decentralized approaches. But are these hurdles really the impediments we think they are? In this commentary, we offer three perceptions that are commonly heard as impediments to the adoption of digital and decentralized clinical trials. Leveraging the Clinical Trial Transformation Initiative's Digital Health Trial hub of work, interactions with members and regulators, and observations related to adoption, we address those perceptions and note some resources that exist to overcome them. In working through these barriers, we can instill confidence in sponsors and designers to leverage all the clinical trial design tools available to them to advance the use of decentralized approaches.

Commitments for Ethically Responsible Sourcing, Use, and Reuse of Patient Data in the Digital Age: Cocreation Process.

BACKGROUND: Personal information, including health-related data, may be used in ways we did not intend when it was originally shared. However, the organizations that collect these data do not always have the necessary social license to use and share it. Although some technology companies have published principles on the ethical use of artificial intelligence, the foundational issue of what is and is not acceptable to do with data, not just the analytical tools to manage it, has not been fully considered. Furthermore, it is unclear whether input from the public or patients has been included. In 2017, the leadership at a web-based patient research network began to envision a new kind of community compact that laid out what the company believed, how the company should behave, and what it promised both to the individuals who engaged with them and to the community at large. While having already earned a social license from patient members as a trusted data steward with strong privacy, transparency, and openness policies, the company sought to protect and strengthen that social license by creating a socially and ethically responsible data contract. Going beyond regulatory and legislative requirements, this contract considered the ethical use of multiomics and phenotypic data in addition to patient-reported and generated data. OBJECTIVE: A multistakeholder working group sought to develop easy-to-understand commitments that established expectations for data stewardship, governance, and accountability from those who seek to collect, use, and share personal data. The working group cocreated a framework that was radically patient-first in its thinking and collaborative in the process of its codevelopment; it reflected the values, ideas, opinions, and perspectives of the cocreators, inclusive of patients and the public. METHODS: Leveraging the conceptual frameworks of cocreation and participatory action research, a mixed methods approach was used that included a landscape analysis, listening sessions, and a 12-question survey. The methodological approaches used by the working group were guided by the combined principles of biomedical ethics and social license and shaped through a collaborative and reflective process with similarities to reflective equilibrium, a method well known in ethics. RESULTS: Commitments for the Digital Age are the output of this work. The six commitments in order of priority are (1) continuous and shared learning; (2) respect and empower individual choice; (3) informed and understood consent; (4) people-first governance; (5) open communication and accountable conduct; and (6) inclusivity, diversity, and equity. CONCLUSIONS: These 6 commitments-and the development process itself-have broad applicability as models for (1) other organizations that rely on digitized data sources from individuals and (2) patients who seek to strengthen operational policies for the ethical and responsible collection, use, and reuse of that data.

Patient perspectives on neuromyelitis optica spectrum disorders: Data from the PatientsLikeMe online community.

BACKGROUND: Few studies have evaluated patient perspectives on neuromyelitis optica (NMO) and NMO spectrum disorder (NMOSD). OBJECTIVE: Describe patient-reported clinical and treatment experience in NMOSD and compare disease characteristics of NMOSD with those of multiple sclerosis (MS). METHODS: This retrospective, observational study included 522 members with NMO or NMOSD (hereafter collectively referred to as NMOSD) from PatientsLikeMe (PLM), an online patient community. Data describing member demographics, symptoms, and treatments were collected, analysed descriptively, and compared with data from PLM members with MS. RESULTS: Fatigue, pain, and stiffness/spasticity were each rated as moderate to severe by more than half of NMOSD members, and 59% reported that their health limited the type of work or other activities they could perform all or most of the time. Overall, symptom severity and disability levels were comparable between NMOSD and MS members; however, NMOSD members were more likely than MS members to attribute disability to vision-related symptoms and were less likely to report moderate to severe cognitive and emotional symptoms, including brain fog, depressed or anxious mood, and emotional lability. CONCLUSION: This analysis underscores the challenges of living with fatigue, pain, stiffness/spasticity, and visual difficulties, prevalent NMOSD symptoms among members of the PLM community.

Comparative effectiveness research in the United States: a catalyst for innovation.

Recent calls for value in the US healthcare system have spurred an increase in comparative effectiveness research, which generates evidence on competing treatment options to inform healthcare stakeholders. As a large healthcare purchaser, the federal government has made several significant investments in comparative effectiveness research. Notably, in 2009 the American Recovery and Reinvestment Act allocated $1.1 billion for comparative effectiveness research, and in 2010 the Patient Protection and Affordable Care Act established a federal institute to organize the federal investment in comparative effectiveness research going forward. Over the past several years, comparative effectiveness research from the public and private sectors has begun to provide a foundation for innovation within the insurance and life sciences industries. Health plans and other payers are experimenting with nuanced coverage and reimbursement policies informed by comparative effectiveness evidence. Anticipating changes in payer, patient, and provider behaviors, drug manufacturers are refocusing their efforts on the development of novel and better-differentiated medications. As more comparative research becomes available in the future, continued innovation in payer and manufacturer strategies appears likely.

Screening for lung cancer: ACCP evidence-based clinical practice guidelines (2nd edition).

BACKGROUND: Lung cancer typically exhibits symptoms only after the disease has spread, making cure unlikely. Because early-stage disease can be successfully treated, a screening technique that can detect lung cancer before it has spread might be useful in decreasing lung cancer mortality. OBJECTIVES: In this article, we review the evidence for and against screening for lung cancer with low-dose CT and offer recommendations regarding its usefulness for asymptomatic patients with no history of cancer. RESULTS: Studies of lung cancer screening with chest radiograph and sputum cytology have failed to demonstrate that screening lowers lung cancer mortality rates. Published studies of newer screening technologies such as low-dose CT and "biomarker" screening report primarily on lung cancer detection rates and do not present sufficient data to determine whether the newer technologies will benefit or harm. Although researchers are conducting randomized trials of low-dose CT, results will not be available for several years. In the meantime, cost-effectiveness analyses and studies of nodule growth are considering practical questions but producing inconsistent findings. CONCLUSIONS: For high-risk populations, no screening modality has been shown to alter mortality outcomes. We recommend that individuals undergo screening only when it is administered as a component of a well-designed clinical trial with appropriate human subjects' protections.

Medical oncologists' views on communicating with patients about chemotherapy costs: a pilot survey.

PURPOSE: As chemotherapy costs escalate, financial concerns have become relevant for more cancer patients. Yet, little is known about how oncologists communicate with patients about coping with chemotherapy treatment costs. METHODS: To understand how medical oncologists view these issues, we developed a survey eliciting their attitudes and behaviors surrounding discussing costs of therapy in the context of treatment decision making. The survey was piloted at a national meeting and then mailed to a random sample of actively practicing 2005 American Society of Clinical Oncology members in the 50 United States. Survey items used a 5-point Likert scale designed to elicit oncologists' practices and attitudes regarding discussion of chemotherapy costs with patients. The first survey wave also included two clinical vignettes about hypothetical patients contemplating treatment with high-cost regimens. RESULTS: Of 530 mailed surveys, 167 were returned for an overall response rate of 31.5%. Most oncologists reported knowledge of their patients' financial well-being: 15% all of the time, 39% most of the time, 33% some of the time, and 13% rarely/none of the time. There was substantial variation in oncologists' attitudes toward discussing chemotherapy costs with patients: 42% did so always or most of the time, 32% sometimes did, and 26% rarely or never did. Most oncologists (80%) felt that it was important to be explicit about the impact of treatment choices on patients' finances. However, 20% maintained that costs play no role in clinical encounters, and 31% reported a high degree of discomfort in discussing costs with patients. Referral to support staff for these discussions was common. Only a few oncologists (16%) acknowledged omitting treatment options on the basis of their perceptions of patients' ability to afford treatment. CONCLUSION: There is important heterogeneity in medical oncologists' attitudes toward discussing chemotherapy treatment costs with patients. Mismatch between physician and patient viewpoints has the potential to result in miscommunication and suboptimally informed treatment decisions.

Fragmentation of care for frequently hospitalized urban residents.

BACKGROUND: Fragmentation across sites of care may impede efficient healthcare delivery. OBJECTIVES: The objectives of this study were to evaluate fragmentation of hospital care for chronically ill New York City (NYC) residents and its association with enrollment in the New York State (NYS) Medicaid program. RESEARCH DESIGN: We conducted a cross-sectional study using the NYS Department of Health's Statewide Planning and Research Cooperative System discharge database. We identified 53,031 NYC residents admitted 3 or more times to acute care hospitals between 2000 and 2002 with the same principal diagnosis of a specific chronic illness (diabetes, sickle cell anemia, psychosis, substance abuse, cancer, gastrointestinal disease, chronic obstructive pulmonary disease/asthma, coronary artery disease, or congestive heart failure). We also evaluated a larger cohort of 225,421 patients with >or=3 admissions for a specific chronic illness coded as either the principal or a secondary diagnosis. A generalized logit model was used to examine the relationship between fragmentation and each patient's primary insurance adjusted for diagnosis and demographic characteristics. MEASURES: Fragmentation was characterized as high, moderate, or low based on the number of distinct hospitals a patient visited relative to the patient's total number of hospitalizations over the 3-year interval. RESULTS: Among frequently hospitalized NYC residents with select chronic conditions, 17.1% experienced highly fragmented care. This rate was 9.9% for patients with commercial insurance, 24.4% for those with Medicaid, and 9.7% for those with Medicare. The unadjusted odds ratio describing high fragmentation of Medicaid enrollees compared with commercially insured patients was 3.82 (95% confidence interval [CI], 3.50-4.18) and, although attenuated, remained significant after adjustment for demographic characteristics (odds ratio, 1.33; 95% CI, 1.20-1.47). The strongest predictor of fragmentation was a diagnosis of psychosis (OR, 2.81; 95% CI, 2.43-3.25) or substance abuse (OR, 7.58; 95% CI, 6.55-8.77). CONCLUSIONS: In NYC, Medicaid enrollment is associated with greater fragmentation of hospital care, but this is largely attributable to the preponderance of Medicaid enrollees with diagnoses of psychosis and substance abuse. Strategies to improve the efficiency of healthcare delivery should focus on patients with mental illness who are frequently admitted to general hospitals.