Use of a web-based portal to return normal individual research results in Early Check: Exploring user behaviors and attitudes.

Early Check is a voluntary, large-scale expanded newborn screening study in North Carolina that uses a self-directed web-based portal for return of normal individual research results (IRR). Little is known about participant perspectives in using web-based portals to receive IRR. This study explored user attitudes and behaviors within the Early Check portal using three methods: (1) a feedback survey available to the consenting parent of participating infants (typically mothers), (2) semi-structured interviews conducted with a subset of parents, and (3) Google Analytics. During an approximate 3-year period, 17 936 newborns received normal IRR and there were 27 812 visits to the portal. Most surveyed parents reported viewing their baby's results (86%, 1410/1639). Parents largely found the portal easy to use to get results, and helpful in understanding the results. However, 10% of parents said it was difficult to find enough information to understand their baby's results. In Early Check, providing normal IRR via the portal made a large-scale study practical, and was highly rated by most users. Return of normal IRR may be particularly amenable to web-based portals, as the consequences to participants from not viewing results are modest, and the interpretation of a normal result is relatively straightforward.

Assessing Assessments: Substance use disorder treatment providers' perceptions of intake assessments.

Background: Assessments to determine patients' treatment needs and preferences when they begin substance use disorder (SUD) treatment are essential. The objectives of this paper are to identify the perspectives of providers who conduct assessments on (1) assessments' utility in determining the level of care where patients will receive treatment, (2) strategies to engage patients in treatment during assessments, and (3) assessment strengths and shortcomings. Methods: Semi-structured interviews were conducted with 30 California treatment providers who routinely perform SUD assessments for Medicaid beneficiaries. Interviews asked about the utility of assessment tools in determining appropriate levels of care, patient engagement during assessments, and strengths and shortcomings of intake assessment processes. Interviews were audio-recorded, transcribed, and analyzed by multiple researchers using template analysis. Results: Providers reported that assessments linked to level-of-care decision rules sometimes generate recommendations inconsistent with their clinical judgment, and that the timing of assessments can influence the quality of the information collected. Providers described engagement strategies that help patients feel more comfortable during assessments and that encourage more thoughtful and accurate responses. Providers valued assessments that helped ensure comprehensive collection of patient information, that allowed flexibility to probe for additional information and context, and that facilitated treatment planning. Providers did not like assessments that were long and repetitive or those that did not collect detailed information about patients' mental health and recovery environments. Conclusions: Assessments can be improved if providers conduct them in a manner that makes patients feel comfortable while building trust and rapport. Ensuring that assessments are not long or repetitive and giving comprehensive assessments once patients have developed trusting relationships with treatment programs can improve assessment processes. Further research is needed to optimize SUD assessments.

"The assessment really helps you with the first step in recovery." What do clients think substance use disorder treatment intake assessments should look like?

Background: In general, research has found that patient-centered substance use disorder treatment is positively correlated with improved patient outcomes. However, little research has examined what factors make intake assessments-the first step in addiction treatment-patient-centered. Methods: We conducted interviews with 30 Medicaid-enrolled individuals who received addiction treatment in California about their experiences with the intake assessment process. Results: Participants reported that the intake assessment process evoked strong feelings, both positive and negative. Some participants said that answering detailed questions about their substance use, mental health, and social relationships, for example, was cathartic and gave them helpful insights. Other participants found the questions invasive, exhausting, and anxiety provoking. Participants also emphasized how critical it is for the person conducting the assessment to be supportive, nonjudgmental, and attentive. Participants recommended delaying the comprehensive assessment because they did not feel physically or emotionally ready to complete the intake. Conclusions and recommendations: Patients' introduction to addiction treatment is typically the intake assessment. By understanding how patients experience intake assessments, providers can make the process more patient-centered, which may lead to improved patient outcomes.

Patients' Understanding of Oncology Clinical Endpoints: Environmental Scan and Focus Groups.

BACKGROUND: Understanding treatment options is important for patients with cancer and their caregivers. This may be difficult, however, because oncology treatments are often approved based on complex clinical endpoints. The study aimed to explore lay understanding of oncology clinical endpoints by assessing the definitions of clinical endpoints available online and gathering qualitative focus group data on cancer survivors' and the general public's understanding of clinical endpoints. METHODS: We conducted an environmental scan to find Web sites accessible by a general audience that defined three clinical endpoints: overall survival, progression-free survival, and response rate. Next, we conducted a series of eight focus groups across the U.S. with cancer survivors (n = 36) and general population adults (n = 36). RESULTS: We found several online resources defining each endpoint; however, many of the definitions we identified used technical language that may not be easily understood by patients and caregivers. Few focus group participants were familiar with the technical terms for these endpoints. When presented with the endpoint terms and definitions, participants had misconceptions about treatment efficacy. Specifically, they tended to expect that all endpoints were a variation on living longer. CONCLUSION: The results point to the need for more patient-friendly definitions of clinical endpoints developed with input from the general public and from patients with cancer. IMPLICATIONS FOR PRACTICE: As the number of oncology prescription drug approvals and the advertising of those drugs to consumers increase, it is timely and critical to understand how to discuss treatment benefits with patients. Patient-friendly definitions of common clinical endpoints, such as overall survival and progression-free survival, would help health care providers describe treatment benefits to patients. This research provides evidence regarding patients' understanding of these endpoints and suggests definitions for additional research. This represents a first step in creating evidence-based patient-friendly language to describe clinical endpoints.

Taking Repeated Exposure into Account: An Experimental Study of Direct-To-Consumer Prescription Drug Television Ad Effects.

Introduction: Little is known about how repeated exposure to direct-to-consumer prescription drug promotion can impact consumers' retention and perceptions of drug information. The study described here tested the effects of varied ad exposure frequency on these outcomes. Methods: In an in-person experiment, participants with seasonal allergies (n = 616) were randomized to view a mock prescription drug television ad either once, twice, or four times within 1 h of television programming, embedded with six commercial breaks. Respondents then answered a 20-min survey administered via computer. Results: Those who viewed the ad more frequently were better able to recall both risk (X2 = 20.93, p < .001) and benefit information (X2 = 9.34, p = .009) and to recognize risk (F(2,597) = 11.89, p = .001) and benefit information (F(2,597) = 3.17, p = .043) than those who viewed the ad one time. Ad exposure frequency was not associated with perceptions about the magnitude or likelihood of risks or benefits. In general, risk information seemed to require more repetitions than benefit information to be accurately remembered. The recall was mediated by elaborate processing. Discussion: Effects on memory were small; retention of both risks and benefits remained low overall even after four exposures.

Estimating Costs of Implementing Stroke Systems of Care and Data-Driven Improvements in the Paul Coverdell National Acute Stroke Program.

PURPOSE AND OBJECTIVES: We evaluated the costs of implementing coordinated systems of stroke care by state health departments from 2012 through 2015 to help policy makers and planners gain a sense of the potential return on investments in establishing a stroke care quality improvement (QI) program. INTERVENTION APPROACH: State health departments funded by the Paul Coverdell National Acute Stroke Program (PCNASP) implemented activities to support the start and proficient use of hospital stroke registries statewide and coordinate data-driven QI efforts. These efforts were aimed at improving the treatment and transition of stroke patients from prehospital emergency medical services (EMS) to in-hospital care and postacute care facilities. Health departments provided technical assistance and data to support hospitals, EMS agencies, and posthospital care agencies to carry out small, rapid, incremental QI efforts to produce more effective and efficient stroke care practices. EVALUATION METHODS: Six of the 11 PCNASP-funded state health departments in the United States volunteered to collect and report programmatic costs associated with implementing the components of stroke systems of care. Six health departments reported costs paid directly by Centers for Disease Control and Prevention-provided funds, 5 also reported their own in-kind contributions, and 4 compiled data from a sample of their partners' estimated costs of resources, such as staff time, involved in program implementation. Costs were analyzed separately for PCNASP-funded expenditures and in-kind contributions by the health department by resource category and program activity. In-kind contributions by partners were also analyzed separately. RESULTS: PCNASP-funded expenditures ranged from $790,123 to $1,298,160 across the 6 health departments for the 3-year funding period. In-kind contributions ranged from $5,805 to $1,394,097. Partner contributions (n = 22) ranged from $3,912 to $362,868. IMPLICATIONS FOR PUBLIC HEALTH: Our evaluation reports costs for multiple state health departments and their partners for implementing components of stroke systems of care in the United States. Although there are limitations, our findings represent key estimates that can guide future program planning and efforts to achieve sustainability.

Pathways to Program Success: A Qualitative Comparative Analysis (QCA) of Communities Putting Prevention to Work Case Study Programs.

OBJECTIVE: To examine the elements of capacity, a measure of organizational resources supporting program implementation that result in successful completion of public health program objectives in a public health initiative serving 50 communities. DESIGN: We used crisp set Qualitative Comparative Analysis (QCA) to analyze case study and quantitative data collected during the evaluation of the Communities Putting Prevention to Work (CPPW) program. SETTING: CPPW awardee program staff and partners implemented evidence-based public health improvements in counties, cities, and organizations (eg, worksites, schools). PARTICIPANTS: Data came from case studies of 22 CPPW awardee programs that implemented evidence-based, community- and organizational-level public health improvements. INTERVENTION: Program staff implemented a range of evidence-based public health improvements related to tobacco control and obesity prevention. MAIN OUTCOME MEASURE: The outcome measure was completion of approximately 60% of work plan objectives. RESULTS: Analysis of the capacity conditions revealed 2 combinations for completing most work plan objectives: (1) having experience implementing public health improvements in combination with having a history of collaboration with partners; and (2) not having experience implementing public health improvements in combination with having leadership support. CONCLUSION: Awardees have varying levels of capacity. The combinations identified in this analysis provide important insights into how awardees with different combinations of elements of capacity achieved most of their work plan objectives. Even when awardees lack some elements of capacity, they can build it through strategies such as hiring staff and engaging new partners with expertise. In some instances, lacking 1 or more elements of capacity did not prevent an awardee from successfully completing objectives. These findings can help funders and practitioners recognize and assemble different aspects of capacity to achieve more successful programs; awardees can draw on extant organizational strengths to compensate when other aspects of capacity are absent.

How accurate are Americans' perceptions of their own weight?

As obesity/overweight has increased in the United States (Centers for Disease Control and Prevention, 2009 ), studies have found that Americans' perceptions of their own weight often are not aligned with their actual body mass index (BMI; Brener et al., 2004 ; Christakis, 2003; Johnson-Taylor et al., 2008 ). Taylor, Funk, and Craighill ( 2006 ) found that half of Americans whose BMI indicated they were overweight perceived their weight to be just about right. The purpose of this study was to examine factors that influence the accuracy of weight self-perceptions and whether accuracy influences health behaviors. Using data from the 2007 Health Information National Trends Survey, the authors compared respondents' weight self-perceptions to their actual BMI to determine the accuracy of their weight self-perceptions. About 28%of respondents were obese, 35%were overweight, 35%were of normal weight, and 2%were underweight. About three quarters of the sample's self-perceptions of weight were aligned with their BMI. About 10%of the sample had a BMI that indicated they were overweight, but they perceived themselves to be of normal weight; about 10%were of normal weight but perceived themselves to be overweight; and about 5%of respondents were of normal weight but thought they were underweight. Gender, race, and education were associated with the accuracy of respondents' weight perceptions. Results suggest that asking patients about their weight self-perceptions could be useful in clinical settings and that weight perception accuracy could be used to segment audiences and tailor messages.

Preferences for patient medication information: what do patients want?

This study investigated respondent preferences on how best to display patient medication information (PMI) that accompanies prescription medications to promote comprehension and appropriate usage. The authors identified 30 individuals diagnosed with select immune disorders, 30 with other chronic diseases, and 30 from the general public and had them review one of two PMI handouts that varied by format, organization, and content. The authors explored preferences for the PMI handout using one-on-one interviews. The authors analyzed the qualitative data to identify relevant themes and patterns using NVivo9 qualitative software. The majority of respondents noted that the formats of the two PMI handouts were more informative than those they currently receive from the pharmacist, with a preference for the 2-column, segmented design. However, respondent PMI preferences varied by age, education, and health status. Patients need simpler and more concise drug information to make better decisions about their health. Current PMI handouts are dense and complex, which can be confusing and not reader friendly. To improve PMI understandability and usefulness, the U.S. Food and Drug Administration is working with stakeholders, consumer advocates, and academics. Findings from this study may help inform future development of more user-friendly PMI.

Parent perspectives following newborn screening resulting in diagnoses of fragile X syndrome or fragile X premutation.

BACKGROUND: Fragile X syndrome (FXS) is the most common inherited cause of intellectual disability. Early Check, a voluntary newborn screening study, screened 18,833 newborns for FXS over ∼3 years. Exploring parental attitudes and perspectives can provide insight to the potential future acceptability of public health screening. METHODS AND PROCEDURES: Mothers of infants who received a screen positive result for FXS (n = 6) or fragile X premutation (FXPM; n = 18) were interviewed about their perceptions and experiences. OUTCOMES AND RESULTS: Mothers of children with FXS described utility in receiving information about their child, particularly to monitor for potential developmental issues and intervene early; overall mothers did not regret participating. Mothers reported various reactions to receiving the FXS or FXPM results including (1) stress and worry; (2) guilt; (3) sadness and disappointment; (4) neutrality, relief, and acceptance; and (5) confusion and uncertainty. CONCLUSIONS AND IMPLICATIONS: Despite initial reactions such as sadness, stress, and worry, mothers found value in learning of their child's presymptomatic diagnosis of FXS, particularly the anticipated long-term benefits of early diagnosis to their child's health and wellbeing. Our results indicate that professionals returning positive newborn screening results should anticipate and prepare for reactions such as parental shock, guilt, sadness, and uncertainty. Genetic counseling and psychosocial support are critical to supporting families.

Addiction Treatment and Telehealth: Review of Efficacy and Provider Insights During the COVID-19 Pandemic.

OBJECTIVE: Addiction treatment via telehealth expanded to unprecedented levels during the COVID-19 pandemic. This study aimed to clarify whether the research evidence on the efficacy of telehealth-delivered substance use disorder treatment and the experience of providers using telehealth during the pandemic support continued use of telehealth after the pandemic and, if so, under what circumstances. METHODS: Data sources included a literature review on the efficacy of telehealth for substance use disorder treatment, responses to a 2020 online survey from 100 California addiction treatment providers, and interviews with 30 California treatment providers and other stakeholders. RESULTS: Eight published studies were identified that compared addiction treatment via telehealth with in-person treatment. Seven found telehealth treatment as effective but not more effective than in-person treatment in terms of retention, therapeutic alliance, and substance use. One Canadian study found that telehealth facilitated methadone prescribing and improved retention. In the survey results reported here, California addiction treatment providers said that more than 50% of their patients were being treated via telehealth for intensive outpatient treatment, individual counseling, group counseling, and intake assessment. They were most confident that individual counseling via telehealth was as effective as in-person individual counseling and less sure about the relative effectiveness of telehealth-delivered medication management, group counseling, and intake assessments. CONCLUSIONS: Telehealth may help engage patients in addiction treatment by improving access and convenience. Additional research is needed to confirm that benefit and to determine how best to tailor telehealth to each patient's circumstances and with what mix of in-person and telehealth services.

Effects of additional context information in prescription drug information sheets on comprehension and risk and efficacy perceptions.

OBJECTIVE: To determine how additional explanatory text (context) about drug side effects in a patient medication information handout affected comprehension and perceptions of risk and efficacy. METHODS: We conducted an online experiment with a national sample of 1,119 U.S. adults with rheumatoid arthritis and related conditions, sampled through random-digit dialing, address-based sampling, and online ads. We randomized participants to receive one of several versions of a patient information handout for a fictitious drug, either with or without additional context, then measured comprehension and other outcomes. RESULTS: Additional qualitative context about warnings and side effects resulted in lower comprehension of side effect information, but not information about uses of the drug or warnings. The effect of additional context on risk perceptions depended on whether the medication handout was delivered online or through the mail. Those who received a hardcopy of the handout with additional context had higher perceived risk of side effects than those who saw the version without additional context. CONCLUSION: More clarifying information is not always better and may lead to cognitive overload, inhibiting comprehension. PRACTICE IMPLICATIONS: Additional research should further explore effects of context in online vs. hard-copy formats before practice implications can be determined.

Physicians' use of and preferences for FDA-approved prescribing information.

BACKGROUND: The Prescribing Information (PI) is the US Food and Drug Administration (FDA)'s primary tool for communicating a summary of the essential scientific information needed for the safe and effective use of a prescription drug to healthcare providers.[1] One challenge with this type of communication is balancing the need to be thorough with the need to be concise. OBJECTIVES: This study aimed to explore physicians' preferences for and understanding of specific content and formatting in the PI. This study also explored physicians' use of and perceptions of the PI. METHODS: Seventy semi-structured qualitative interviews were conducted with primary care physicians (n = 35) and physicians from a wide range of specialties (n = 35) using web conferencing technology. Using fictitious PI examples, the guide assessed physicians' interpretation of language and preferences for how certain information is organized and communicated in select sections of the PI. The interview guide also included questions about the resources physicians use to find information about prescription drugs, when and how physicians access the PI, and their perceptions of the PI. RESULTS: The findings suggest that of the content and formatting items surveyed, physicians had the greatest preference for: (1) uniformly specifying the age group for which the drug is indicated in the INDICATIONS AND USAGE section, even for medical conditions that are highly associated with only one particular age group (e.g., adult patients), and (2) uniformly including administration information in relation to food (e.g., "with or without food") in the DOSAGE AND ADMINISTRATION section for drugs with oral dosing. The findings also suggest that including a long list of interacting drug examples in the DRUG INTERACTIONS section may be misinterpreted to be a comprehensive list. CONCLUSION: This qualitative research suggests physicians may prefer more clarity in some sections of the PI.

The impact of interactive advertising on consumer engagement, recall, and understanding: A scoping systematic review for informing regulatory science.

We conducted a scoping systematic review with respect to how consumer engagement with interactive advertising is evaluated and if interactive features influence consumer recall, awareness, or comprehension of product claims and risk disclosures for informing regulatory science. MEDLINE, PsycINFO, Business Source Corporate, and SCOPUS were searched for original research published from 1997 through February 2021. Two reviewers independently screened titles/abstracts and full-text articles for inclusion. Outcomes were abstracted into a structured abstraction form. We included 32 studies overall. The types of interactive ads evaluated included website banner and pop up ads, search engine ads, interactive TV ads, advergames, product websites, digital magazine ads, and ads on social network sites. Twenty-three studies reported objective measures of engagement using observational analyses or laboratory-based experiments. In nine studies evaluating the association between different interactivity features and outcomes, the evidence was mixed on whether more interactivity improves or worsens recall and comprehension. Studies vary with respect to populations, designs, ads evaluated, and outcomes assessed.

Patient Perceptions of Switching to a Generic Dry Powder Inhaler - Increased Understanding Through Journey Mapping.

Purpose: This qualitative study explored patients' attitudes about and perceptions of generic dry powder inhaler (DPI) substitution for the brand product and patients' views of generic product quality, efficacy, design, and usability. Methods: Forty COPD and asthma patients (36 adults, four adolescents), who were actively using a brand DPI product, participated in one of six focus groups. Participants completed a journey mapping exercise to assess attitudes and opinions about a scenario where they refill their prescription and unexpectedly receive a generic DPI instead of their brand DPI. The focus groups were audio recorded, transcribed, and analyzed thematically. Results: The hypothetical scenario of unexpectedly receiving a generic DPI elicited mixed feelings including: happiness and relief about potential cost savings, confusion, disappointment, anger, and/or frustration with the unexpected switch. Participants in most groups anticipated anxiety or hesitation in using the generic DPI due to concerns about potential differences in usability, uncertainty about correct use, and questions about efficacy. Participants across all groups said they would ask a pharmacist or healthcare provider for information or answers to their questions, and some participants said they would use online resources. When participants held the brand and generic DPI devices, most preferred the brand DPI device and found it easier, less cumbersome, or more convenient to use (due to size and weight). However, many participants reiterated that the potential reduced cost of the generic DPI would be a primary factor in their decision-making related to generic DPI substitution for their brand DPI. Conclusion: Patients experienced a mixture of positive and negative feelings when faced with an unexpected generic DPI substitution. Some patients have doubts about their ability to successfully navigate differences in generic device design, and most expressed the desire to participate in discussions and decision-making with their HCP about generic DPI sameness and substitution.

Knowledge of genomic testing among early-stage breast cancer patients.

BACKGROUND: Genomic recurrence risk test results now inform clinical decisions about adjuvant treatment for women with early-stage breast cancer. We sought to understand patients' knowledge of these tests and correlates of their knowledge. METHODS: Participants in this cross-sectional study were 78 women, treated for early-stage, estrogen receptor-positive breast cancer with 0-3 positive lymph nodes, whose medical records indicated they received Oncotype DX testing earlier. We mailed a questionnaire that assessed knowledge of genomic recurrence risk testing (13 item scale, alpha=0.83) and reviewed medical charts of consenting patients. RESULTS: Knowledge about genomic recurrence risk testing was low (mean knowledge score=67%, SD=0.23). Low knowledge scores were more commonly due to responses of 'don't know' than incorrect answers. Most women (91%) clearly understood that test results can aid decisions about chemotherapy, and few (22%) understood that the test's estimate of the chance of metastasis assumes the patient is receiving hormone therapy. Higher knowledge about genomic recurrence risk testing was associated with higher education, reading ability, and numeracy. Knowledge was higher among women who recalled receiving both verbal and printed information about the test and among women who had active roles in deciding about their treatments. Higher knowledge was also associated with having fewer concerns about genomic testing. DISCUSSION: Among early-stage breast cancer patients who received Oncotype DX, we found low knowledge about many aspects of genomic recurrence risk testing. Research is needed to understand testing information provided to patients and best practices for patient education.

When genomic and standard test results diverge: implications for breast cancer patients' preference for chemotherapy.

PURPOSE: We examined how women incorporate potentially differing genomic and standard assessments of breast cancer recurrence risk into chemotherapy decisions. METHODS: 165 women previously treated for early-stage breast cancer indicated their interest in chemotherapy regimens to prevent recurrence of breast cancer in response to six hypothetical vignettes that presented breast cancer recurrence risk estimates from standard criteria and a genomic test, some of which were discordant. RESULTS: Standard and genomic test results each elicited greater interest in chemotherapy when they indicated high rather than low risk for recurrence (89% vs. 26%, and 87% vs. 22%, respectively, Ps < 0.001). Genomic test results had a larger impact on chemotherapy preferences than standard measures to predict recurrence. CONCLUSIONS: Some women may be reluctant to forgo chemotherapy when genomic tests indicate low recurrence risk but standard criteria suggest high risk. Additional research including replication of the findings of this small, vignette-based study is needed.

Health literacy and cancer risk perception: implications for genomic risk communication.

BACKGROUND: As new genomic technology expands the number of medical tests available to physicians and patients, identifying gaps in our understanding of how best to communicate risk is increasingly important. We examined how health literacy informs breast cancer survivors' understanding of and meaning assigned to recurrence risks yielded by genomic tests. METHODS: Study participants were posttreatment female breast cancer survivors (N =163) recruited at a university breast cancer clinic. We assessed their health literacy (using REALM) and their interpretation of hypothetical recurrence risk results from a genomic test, presented in several verbal and numerical formats. Analyses controlled for women's objective recurrence risk, age, income, and race. RESULTS: Women with lower health literacy gave higher mean estimates of recurrence risk for a hypothetical group of women with early-stage breast cancer than did women with higher health literacy (52% v. 30%, P < 0:001). Women with lower health literacy also gave more variable estimates in this and several other tasks. When making chemotherapy decisions using risks presented in verbal formats, decisions by women with lower health literacy were less sensitive to the difference between low and high recurrence risk. Ease of understanding of risk formats differed by health literacy. CONCLUSIONS: Health literacy affected the meanings women assigned to recurrence risk when presented in certain formats. The greater variability in responding by women with lower health literacy supports the hypothesis that they have less precise mental representations of risk, but more research is needed to rule out other possible explanations.

Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.

OBJECTIVES: Duchenne muscular dystrophy (DMD) is a rare neuromuscular disorder that causes progressive weakness and early death. Gene therapy is an area of new therapeutic development. This qualitative study explored factors influencing parents' and adult patients' preferences about gene therapy. METHODS: We report qualitative data from 17 parents of children with DMD and 6 adult patients. Participants responded to a hypothetical gene therapy vignette with features including non-curative stabilizing benefits to muscle, cardiac and pulmonary function; a treatment-related risk of death; and one-time dosing with time-limited benefit of 8-10 years. We used NVivo 11 to code responses and conduct thematic analyses. RESULTS: All participants placed high value on benefits to skeletal muscle, cardiac, and pulmonary functioning, with the relative importance of cardiac and pulmonary function increasing with disease progression. More than half tolerated a hypothetical 1% risk of death when balanced against Duchenne progression and limited treatment options. Risk tolerance increased at later stages. Participants perceived a 'right time' to initiate gene therapy. Most preferred to wait until a highly-valued function was about to be lost. CONCLUSION: Participants demonstrated a complex weighing of potential benefits against harms and the inevitable decline of untreated Duchenne. Disease progression increased risk tolerance as participants perceived fewer treatment options and placed greater value on maintaining remaining function. In the context of a one-time treatment like gene therapy, our finding that preferences about timing of initiation are influenced by disease state suggest the importance of assessing 'lifetime' preferences across the full spectrum of disease progression.

Expectations of benefit in early-phase clinical trials: implications for assessing the adequacy of informed consent.

BACKGROUND: Participants in early-phase clinical trials have reported high expectations of benefit from their participation. There is concern that participants misunderstand the trials to which they have consented, which is based on assumptions about what patients mean when responding to questions about likelihood of benefit. METHODS: Participants were 27 women and 18 men in early-phase oncology trials at 2 academic medical centers in the United States. To determine whether expectations of benefit differ depending on how patients are queried, the authors randomly assigned participants to 1 of 3 interviews corresponding to 3 questions about likelihood of benefit: frequency type, belief type, and vague. In semistructured interviews, participants were queried about how they understood and answered the question. Participants then answered and discussed 1 of the other questions. RESULTS: Expectations of benefit in response to the belief-type question were significantly greater than expectations in response to the frequency-type and vague questions (P=0:02). The most common justifications involved positive attitude (n=27 [60%]) and references to physical health (n=23 [51%]). References to positive attitude were most common among participants with higher (> 70%) expectations (n = 11 [85%]) and least common among those with lower ( < 50%) expectations (n = 3 [27%]). CONCLUSIONS: The wording of questions about likelihood of benefit shapes the expectations that patients express. Patients who express high expectations may not do so to communicate understanding but rather to register optimism. Ongoing research will clarify the meaning of high expectations and examine methods for assessing understanding.