Physician experiences with and perceptions of risk evaluation and mitigation strategy programs with elements to assure safe use.

PURPOSE: The US Food and Drug Administration (FDA) Amendments Act of 2007 authorized the FDA to require risk evaluation and mitigation strategy (REMS) programs for drugs with important safety concerns. REMS can have elements to assure safe use (ETASU), such as patient registries, dispensing restrictions, and physician training and certification requirements. We aimed to understand physician experiences with and perceptions of a selection of ETASU REMS. METHODS: Physicians prescribing 1 of 4 ETASU REMS-covered drugs: natalizumab, riociguat, sodium oxybate, and vigabatrin. STUDY DESIGN: Descriptive phenomenological study based on semi-structured phone interviews. DATA COLLECTION/EXTRACTION METHODS: Qualitative content analysis to summarize physician responses to open-ended questions. RESULTS: Of 31 physicians (14 female), 6 prescribed riociguat, 6 vigabatrin, 7 sodium oxybate, and 12 natalizumab (5 for Crohn's disease, 7 for multiple sclerosis), most demonstrated good understanding of the rationale for and requirements of the ETASU REMS but believed that the programs had limited effect on clinical practice. Some physicians reported that the ETASU REMS made them more comfortable with prescribing covered drugs due to heightened oversight, facilitated discussions about treatment, and were likely more beneficial for non-specialists. Concerns were raised about the administrative effort needed to comply with the programs and the potential misuse of patient health information transmitted to manufacturers. CONCLUSIONS: Physicians are generally aware of ETASU REMS and get reassurance from the additional oversight, but the programs can be better integrated into clinical workflows and can be designed to better protect patient health information.

Meaningful Improvements in Rett Syndrome: A Qualitative Study of Caregivers.

BACKGROUND: Rett syndrome is a rare neurodevelopmental disorder primarily affecting females. This syndrome is associated with many comorbidities and impairments related to motor function, breathing, sleep, expressive language, and repetitive hand movements. The Rett Syndrome Behaviour Questionnaire (RSBQ) is one measure used to assess changes in Rett syndrome-related manifestations or core symptoms. Little is known about how caregivers think about meaningful changes in the items that make up the RSBQ scale. METHODS: This qualitative study explored how caregivers of individuals with Rett syndrome viewed changes in the symptoms covered in the RSBQ. We conducted semistructured interviews with 40 caregivers and employed thematic analysis, identifying themes using an iterative process. RESULTS: Two factors characterized caregivers' thoughts about meaningful changes in Rett syndrome manifestations. First, general features of these symptoms rendered them bothersome: the extent of bother compared to other symptoms, if or how they prevented desirable behaviors and their temporal qualities. Second, caregivers evaluated the meaning of improvements by considering the decrease in bother and the potential benefits of change. Improvements had social and psychological consequences for individuals with Rett syndrome and caregivers. In addition, implications for health, fine and gross motor skills, and communication were also substantial.

Treatment Goals for Achondroplasia: A Qualitative Study with Parents and Adults.

INTRODUCTION: Achondroplasia is characterized by disproportionate short stature accompanied by other changes to the musculoskeletal system. Individuals with this condition typically experience a variety of medical complications. As pharmacologic treatments continue to be developed for the treatment of achondroplasia, it is important to understand treatment goals among those affected by achondroplasia and the factors that shape their goals. METHODS: This qualitative study is based on semi-structured interviews with 19 parents of children with achondroplasia and five adults with achondroplasia in the USA. We employed thematic analysis using an iterative process to identify themes across the interviews. RESULTS: Participants had two goals for pharmacologic treatment of achondroplasia: ameliorating complications associated with the condition and increasing stature to overcome functional limitations and psychosocial challenges. Complications of particular concern were chronic pain and surgeries to repair spinal, ear, nose, and throat (ENT) problems, and neurological sequelae. Increased height would enhance independence, help individuals to fit in socially, and avoid social stigma. Countervailing factors included the importance of stature to their identity and the concern that the condition would remain despite treatment. CONCLUSIONS: This study offers evidence about how individuals affected by achondroplasia think about the pharmacologic treatment of this condition, including both the benefits of ameliorating complications and increasing height. The findings can offer practical insights for parents of children considering treatment, treating physicians, and decision-makers evaluating coverage decisions for treatment of achondroplasia.

Patient and Caregiver Experiences With and Perceptions of Risk Evaluation and Mitigation Strategy Programs With Elements to Assure Safe Use.

Importance: The US Food and Drug Administration (FDA) Amendments Act of 2007 authorized the FDA to impose safety requirements on drugs with important risks, such as prescriber certification or routine laboratory testing, to ensure that the benefits of use outweighed the risks. However, little is known about patient and caregiver experiences with these Risk Evaluation and Mitigation Strategy (REMS) programs with Elements to Assure Safe Use (ETASU). Objective: To understand patient and caregiver experiences with and perceptions of REMS programs with ETASU. Design, Setting, and Participants: This qualitative study included semistructured qualitative phone interviews conducted between 2016 and 2017, with initial analysis performed in 2017 and reanalysis performed in 2021. Adult patients prescribed natalizumab or sodium oxybate, adult patients or caregivers of adult patients prescribed vigabatrin, and adult female patients of reproductive age prescribed riociguat were included. Main Outcomes and Measures: Assessment of knowledge, decision-making, medication access, and perceptions of medical privacy. Results: Among 63 participants, 46 (73%) were female. Twenty-five participants (40%) had taken natalizumab, 10 (16%) riociguat, 15 (24%) sodium oxybate, and 10 (16%) vigabatrin. One participant had taken both natalizumab and vigabatrin; 4 (6%) were caregivers of patients using vigabatrin. Most participants expressed knowledge of REMS program requirements, but many lacked the insight that these requirements were part of an FDA-mandated special safety program and expressed difficulty understanding program education materials. REMS requirements made some participants more likely to initiate treatment. However, many reported burdens accessing medication, including the need to travel to certified prescribers or pharmacies. Manufacturer access to personal health information was also controversial, although some participants expressed an altruistic desire to assist others. Conclusions and Relevance: This qualitative study found that REMS programs with ETASU reassured patients and their caregivers about drug safety and helped support medication initiation. However, steps are needed to improve the quality of REMS educational materials, promote efficient medication access, and protect patient privacy.

The fuzzy world of precision medicine: deliberations of a precision medicine tumor board.

AIM: To understand how a cancer precision medicine tumor board (CPM-TB) made choices about return of results. MATERIALS & METHODS: Observed CPM-TB deliberations and completed in-depth interviews with committee members. RESULTS: Responding to complex evidence of ambiguous significance, deliberations of the CPM-TB were predicated on analytic validity and clinical utility. Members had concerns both about potential harms due to returning results based on weak evidence and about withholding potentially meaningful results. Group dynamics and the clinical experiences of individual committee members shaped their work. CONCLUSION: Both scientific evidence and the social context surrounding deliberations of a CPM-TB influenced decisions about return of results. Subjective elements, while present in any scientific endeavor, may carry more weight in the face of ambiguous findings.

Patient and Physician Perceptions of Drug Safety Information for Sleep Aids: A Qualitative Study.

INTRODUCTION: The US Food and Drug Administration uses drug safety communications (DSCs) to release emerging information regarding post-market safety issues, but it is unclear the extent of awareness by patients and providers of these communications and their specific recommendations. OBJECTIVE: We conducted semi-structured interviews with patients and physicians to evaluate their awareness and understanding of emerging drug safety information related to two sleep aids: zolpidem or eszopiclone. METHODS: We conducted interviews with 40 patients and ten physicians recruited from a combination of insurer claims databases and online sources. We evaluated (1) sources of drug safety information; (2) discussions between patients and physicians about the two medications; (3) their knowledge of the DSC; and (4) preferences for learning about future drug safety information. Interviews were transcribed and analyzed thematically. RESULTS: Patients cited their physicians, pharmacy inserts, and the Internet as sources of drug safety information. Physicians often referred to medical journals and online medical sources. Most patients reported being aware of information contained in the DSC summaries they were read. Almost all patients and physicians reported discussing side effects during patient-provider conversations, but almost no patients mentioned that physicians had communicated with them key messaging from the DSCs at issue: the risk of next-morning impairment with zolpidem and the lower recommended initial dose for women. CONCLUSIONS: Some risks of medications are effectively communicated to patients and physicians; however, there is still a noticeable gap between information issued by the Food and Drug Administration and patient and physician awareness of this knowledge, as well as patients' decisions to act on this information. Disseminators of emerging drug safety information should explore ways of providing user-friendly resources to patients and healthcare professionals that can update them on new risks in a timely manner.

Perceptions of erectile dysfunction and phosphodiesterase type 5 inhibitor therapy in a qualitative study of men and women in affected relationships.

Erectile dysfunction negatively affects men and women in relationships; however, the subjective experience of erectile dysfunction and phosphodiesterase-type 5 inhibitor therapy remains poorly understood. The authors therefore characterized participants' subjective understanding of erectile dysfunction and phosphodiesterase-type 5 inhibitor therapy using individual interviews with affected heterosexual men (n = 58) and women (n = 65). Responses were characterized by 6 psychosocial domains: explanation of the experience, emotional responses, socially expected responses, value of sex, communication with the partner, and treatment expectations. The findings may aid clinicians in relating to men with erectile dysfunction and thus potentially improve effectiveness of therapy.

"I was able to still be her mom"--parenting at end of life in the pediatric intensive care unit.

OBJECTIVES: The death of a child in the pediatric intensive care unit is perhaps one of the most devastating and challenging experiences a parent can ever endure. This article examines how parents of children dying in the pediatric intensive care unit understood their role and discusses implications for clinical care and policy. DESIGN: Retrospective, qualitative study. SETTING: Two pediatric intensive care units located in children's hospitals within academic medical centers in the northeastern United States. SUBJECTS: Parents of 18 children who died in the pediatric intensive care unit. INTERVENTIONS: Semistructured telephone interviews, digitally recorded and transcribed. MEASUREMENTS AND MAIN RESULTS: Many of the factors deemed important by the parents related to their capacity to be a "good parent" to their child throughout his or her stay in the pediatric intensive care unit. Specifically, parents sought meaningful ways to express and assert their parenthood across three domains: 1) providing love, comfort, and care; 2) creating security and privacy for the family; and 3) exercising responsibility for what happens to one's child. CONCLUSIONS: Parents' ability to fulfill the essential features of their role as parents of children dying in the pediatric intensive care unit shapes how they perceive the quality of the experience. Pediatric intensive care unit clinical care and policies can and should uphold and protect these features enabling parents to feel that, despite the outcome, they had done their best on behalf of their children.

Supportive housing approaches in the Collaborative Initiative to Help End Chronic Homelessness (CICH).

The Federal Collaborative Initiative to Help End Chronic Homelessness funded 11 sites to expand permanent housing and offer supportive services to persons experiencing chronic homelessness and suffering from mental and substance use disorders. This study examines qualitative data on how the projects used US Department of Housing and Urban Development funding and three housing approaches (scattered units, congregate/clustered, or a combination) for rapid placement of clients. Each housing approach called for adaptations by the services teams and property personnel in order to support clients with independent living skills, prevent housing loss, and promote their overall health in line with Initiative goals. Property personnel reported taking on new roles with clients and forming new collaborative arrangements with services teams. The authors discuss the lessons reported by sites that were associated with housing configuration, type of lease, and role of property personnel.

Adopting best practices: lessons learned in the Collaborative Initiative to Help End Chronic Homelessness (CICH).

The Collaborative Initiative to Help End Chronic Homelessness (CICH) was established to provide housing and supportive services for individuals experiencing chronic homelessness. As part of this initiative, 11 projects across the country received funding to apply models of best practices to support their clients in housing. This paper reports on the experiences of the CICH projects in their use of Assertive Community Treatment (ACT) and Motivational Interviewing (MI), clinical practice models commonly used by CICH projects. A qualitative analysis identified five areas of challenge for the projects: (1) Incomplete and underdeveloped staff teams; (2) Incomplete understanding of the practice models; (3) Using the elements of the practice models; (4) Interagency teaming; and (5) Competing expectations of multiple federal agencies. The paper describes the specific challenges in each of the five areas as well as training approaches and gaps in training and the perceived benefits of the practice models as reported by project staff.

Collaborative initiative to help end chronic homelessness: introduction.

The Collaborative Initiative to Help End Chronic Homelessness was a coordinated effort by the US Departments of Health and Human Services (HHS), Housing and Urban Development (HUD), and Veterans Affairs (VA), and the US Interagency Council on Homelessness to house and provide comprehensive supportive services to individuals with serious psychiatric, substance use, health, and related disabilities who were experiencing long-term chronic homelessness. Eleven communities received 3-year grants from HHS and VA (2003-2006) and up to 5-year grants from HUD (2003-2008) to implement the initiative. This article provides background on chronic homelessness, describes the federal collaboration to comprehensively address chronic homelessness, and introduces the seven articles in this special issue that describe the findings and lessons learned from the participating communities in addressing chronic homelessness. Collectively, these articles offer insight into the challenges and benefits of providing housing and services to individuals experiencing chronic homelessness.

Assessing symptoms of hypogonadism by self-administered questionnaire: qualitative findings in patients and controls.

BACKGROUND: Current screening instruments for hypogonadism lack adequate specificity and diagnostic accuracy. A new self-administered questionnaire of hypogonadism symptoms is being developed to address this need. The process for questionnaire development and results from the first (qualitative) phase are presented. METHODS: Qualitative interviews were conducted based on a new conceptual model of hypogonadism and according to standards for questionnaire development. An item pool was generated from focus groups and in-depth interviews with two groups of hypogonadal patients, treated (N = 26) and untreated (N = 26), and age-equivalent controls (N = 28). Standardized scoring of the qualitative interviews was used to confirm conceptual domains in the model and to generate questionnaire items for further validation. RESULTS: Key domains identified in both patients and controls included: (a) physical function; (b) bodily signs and symptoms; (c) sexual function and libido; (d) sleep function; (e) mood and affective function; (f) memory and cognitive function. The final domain is distress or bother associated with hypogonadism symptoms. This domain was only relevant to the patient groups. CONCLUSIONS: The first stage in the design of a new hypogonadism screener has been completed. Seven domains were identified and draft items were developed in each domain according to current standards of patient-reported outcomes.

A community capacity-enhancement approach to breast and cervical cancer screening among older women of color.

In the Screening Older Minority Women project, the authors applied a community capacity-enhancement approach to promoting breast and cervical cancer screening among older women of color. Members of informal support networks were recruited for this health promotion intervention to empower Latina and African American women to engage in positive health behaviors. The authors describe the phases of the intervention and the experiences from the community. Guidelines are identified to help researchers and practitioners in planning and implementing community health promotion intervention with women of color.

What matters to older African Americans facing end-of-life decisions? A focus group study.

BACKGROUND: To better understand what matters to African American elders who are faced with issues of death, dying, and end-of-life care, a qualitative study was conducted to elicit their perspective. METHODS: Focus groups were convened across the state of Connecticut. A total of 196 individuals participated in the 90-minute interview sessions. Using an interview guide, a trained moderator conducted the racially homogeneous discussion groups. Transcriptions of the group narratives with 22 older African Americans were coded to identify themes. Data were organized and analyzed using NUD-IST 4 and constant comparative method of qualitative data analysis. RESULTS: Five major themes emerged from the focus group data on older African Americans: (1) spirituality, (2) burden on family, (3) trust, (4) health insurance coverage, and (5) cultural concerns. CONCLUSION: Recommendations are made for outreach education, involvement of informal helpers, and a level of acceptability in practice for diverse care needs.

Intended use of informal long-term care: the role of race and ethnicity.

OBJECTIVE: To examine the role of race/ethnicity in the use of informal long-term care among African American and white elders, using an expanded Andersen model of health services use (Bradley et al., Health Services Research, vol. 37, pp. 1221-1242, 2002). DESIGN: Four hundred respondents (n = 200 African American; n=200 white) aged 65 and older, who had been hospitalized within the last year. Data were collected using a cross-sectional survey analyzed with ordered logistic regression. Independent variables included individuals' predisposing factors, enabling factors, need, and psychosocial factors. Intended use of informal long-term care was defined based on responses to a hypothetical scenario of future use of unpaid services by family members, relatives, friends, or neighbors for help with daily needs. RESULTS: African American respondents were more likely than white respondents to intend to use informal long-term care. This effect persisted (p < 0.05) after controlling for predisposing, enabling, and need factors. However, race/ethnicity-related differences in intended use were attenuated substantially (14-18%) after controlling for psychosocial differences. Further, in the fully adjusted models, race/ethnicity was no longer significantly associated with intended long-term care use. CONCLUSIONS: Psychosocial factors, particularly social norms concerning family caregiving, mediated the relationship between race/ethnicity and intended use of informal long-term care. A fuller appreciation of the multiple influences on healthcare decision making of older adults has the potential to inform policy efforts to appropriately meet the respective long-term care needs of an ethnically diverse frail older population.

What is a good death? Minority and non-minority perspectives.

While much attention has been directed at improving the quality of care at the end of life, few studies have examined what determines a good death in different individuals. We sought to identify common domains that characterize a good death in a diverse range of community-dwelling individuals, and to describe differences that might exist between minority and non-minority community-dwelling individuals' views. Using data from 13 focus groups, we identified 10 domains that characterize the quality of the death experience: 1) physical comfort, 2) burdens on family, 3) location and environment, 4) presence of others, 5) concerns regarding prolongation of life, 6) communication, 7) completion and emotional health, 8) spiritual care, 9) cultural concerns, 10) individualization. Differences in minority compared to non-minority views were apparent within the domains of spiritual concerns, cultural concerns, and individualization. The findings may help in efforts to encourage more culturally sensitive and humane end-of-life care for both minority and non-minority individuals.

How can we improve care at the end of life in Connecticut? Recommendations from focus groups.

A qualitative study to elicit views among residents of Connecticut about death, dying, preferences for care, and gaps in available care was conducted for the Connecticut Coalition to Improve End-of-Life Care. Twenty-eight focus groups were convened across the state between May 1999 and March 2001. The focus groups included adolescents and adults from diverse religious, ethnic, and professional backgrounds. A total of 196 individuals participated in the 90-minute sessions. Trained moderators conducted the discussion groups following a guide. Transcriptions of the groups were coded to identify themes. Thirty-two recommendations to improve care based on the focus group discussions are presented. These are grouped under the following actions: 1) enhance public education; 2) offer better professional provider education and support; and 3) augment services. The recommendations are illustrated with quotations from the focus groups.

Expanding the Andersen model: the role of psychosocial factors in long-term care use.

OBJECTIVE: To examine a prevailing conceptual model of health services use (Andersen 1995) and to suggest modifications that may enhance its explanatory power when applied to empirical studies of race/ethnicity and long-term care. STUDY SETTING: Twelve focus groups of African-American (five groups) and white (seven groups) individuals, aged 65 and older, residing in Connecticut during 2000. STUDY DESIGN: Using qualitative analysis, data were coded and analyzed in NUD-IST 4 software to facilitate the reporting of recurrent themes, supporting quotations, and links among the themes for developing the conceptual framework. Specific analysis was conducted to assess distinctions in common themes between African-American and white focus groups. DATA COLLECTION: Data were collected using a standardized discussion guide, augmented by prompts for clarification. Audio taped sessions were transcribed and independently coded by investigators and crosschecked to enhance coding validity. An audit trail was maintained to document analytic decisions during data analysis and interpretation. PRINCIPAL FINDINGS: Psychosocial factors (e.g., attitudes and knowledge, social norms, and perceived control) are identified as determinants of service use, thereby expanding the Andersen model (1995). African-American and white focus group members differed in their reported accessibility of information about long-term care, social norms concerning caregiving expectations and burden, and concerns of privacy and self-determination. CONCLUSIONS: More comprehensive identification of psychosocial factors may enhance our understanding of the complex role of race/ethnicity in long-term care use as well as the effectiveness of policies and programs designed to address disparities in long-term care service use among minority and nonminority groups.