scholarly journals Factors of feasibility: an interview study of physicians’ experiences of expanded access to investigational drugs in three countries

2021 ◽  
Vol 8 (1) ◽  
Author(s):  
Stefan F. Vermeulen ◽  
Marjolijn Hordijk ◽  
Nikkie Aarts ◽  
Eline M. Bunnik
Keyword(s):  
Clinical Practice ◽  
The United States ◽  
Pharmaceutical Companies ◽  
Structured Interviews ◽  
Application Process ◽  
Expanded Access ◽  
Treatment Regimens ◽  
Investigational Drugs ◽  
Hospital Policies ◽  
Seriously Ill Patients

AbstractSeriously ill patients who have exhausted all approved treatment regimens and who cannot be enrolled in clinical trials may resort to expanded access programmes in order to gain access to unapproved, investigational drugs. It seems that in some countries, expanded access to investigational drugs is offered in clinical practice on a more routine basis than in other countries. This study is the first to investigate the experiences of physicians with expanded access to investigational drugs in different healthcare systems, with a focus on factors that facilitate or hinder expanded access. Semi-structured interviews (n = 36) were carried out with medical specialists in the Netherlands (n = 14), Turkey (n = 9) and the United States of America (n = 13), and analysed thematically. This study identifies five sets of factors pointed out by physicians that determine the degree to which expanded access to investigational drugs is deemed feasible in clinical practice: the suitability of investigational treatments, the application process, hospital policies, support by pharmaceutical companies, and funding and reimbursement arrangements. Based on the interviews conducted, we conclude that, while legally allowed and technically possible, expanded access is not always feasible for—and not always considered an option by—treating physicians. This is mainly due to lack of familiarity with expanded access, the extensive time and effort required for the application process, willingness or ability of pharmaceutical companies to supply the drugs, and funding issues.

scholarly journals “I Think It’s Been Met With a Shrug:” Oncologists’ Views Toward and Experiences With Right-to-Try

2020 ◽  
Author(s):  
Cambray Smith ◽  
Jeremiah Stout ◽  
Alex A Adjei ◽  
Jan Buckner ◽  
Mark Wentworth ◽  
...  
Keyword(s):  
The United States ◽  
Cancer Center ◽  
Structured Interviews ◽  
Expanded Access ◽  
Medical Centers ◽  
Investigational Drugs ◽  
Regulatory Bodies ◽  
Improve Access ◽  
Access Programs

Abstract Background The federal Right-to-Try (RTT) Act created an alternate regulatory pathway for preapproval access to investigational drugs. A few studies have examined the experiences of physicians with the Food and Drug Administration’s Expanded Access Programs, but to our knowledge, no study has yet to examine their attitudes and experiences toward RTT. Methods This study explored the views of 21 oncologists at a major cancer center with 3 main sites across the United States using semi-structured interviews and qualitative analysis. Participants were selected to have experience with Expanded Access Programs. Results Most oncologists had limited familiarity with RTT, and several reported confusion about the legislation, including whether patients have a right to investigational drugs and an obligation for companies to provide them. Although oncologists were interested in decreased regulatory burdens, 3 areas of concern were articulated: lack of safety and oversight, unclear structure and no provision for data collection, and potential heightening of patient expectations. Only 4 oncologists had experience discussing RTT, and none formally attempted to obtain the drug through this mechanism. Participants questioned the practicality of RTT legislation and suggested alternative ways to improve access. Conclusions The study provides foundational empirical data underlying challenging ambiguities by experienced oncologists familiar with off-trial use of investigational therapeutics and reaffirms the role of physicians and regulatory bodies in mitigating the risks of investigational drugs. Our findings highlight the need for medical centers to inform oncologists about RTT and other preapproval pathways so that they are able to address questions from patients interested in nontrial investigational drugs.

scholarly journals COVID-19 Pandemic Adversely Affects the Provision of Desired Newborn Circumcision: Perinatal Physician Perspectives

2022 ◽  
Vol 1 ◽  
Author(s):  
Lauren E. Corona ◽  
Ilina Rosoklija ◽  
Ryan F. Walton ◽  
Derek J. Matoka ◽  
Catherine M. Seager ◽  
...  
Keyword(s):  
United States ◽  
Qualitative Study ◽  
The United States ◽  
Structured Interviews ◽  
Newborn Period ◽  
Policy Makers ◽  
Urban City ◽  
Physician Perspectives ◽  
Interview Guide ◽  
Hospital Policies

Over half of boys in the United States undergo circumcision, which has its greatest health benefits and lowest risks when performed during the newborn period under local anesthesia. The COVID-19 pandemic has affected delivery of patient care in many ways and likely also influenced the provision of newborn circumcisions. Prior to the pandemic, we planned to conduct a qualitative study to ascertain physician perspectives on providing newborn circumcision care. The interviews incidentally coincided with the onset of the pandemic and thus, pandemic-related changes emerged as a theme. We elected to analyze this theme in greater detail. Semi-structured interviews were conducted with perinatal physicians in a large urban city from 4/2020 to 7/2020. Physicians that perform or counsel regarding newborn circumcision and physicians with knowledge of or responsibility for hospital policies were eligible. Interviews were transcribed verbatim and qualitative coding was performed. Twenty-three physicians from 11 local hospitals participated. Despite no specific COVID-19 related questions in the interview guide, nearly half of physicians identified that the pandemic affected delivery of newborn circumcision care with 8 pandemic-related sub-themes. The commonest sub-themes included COVID-19 related changes in: (1) workflow processes, (2) staffing and availability of circumcision proceduralists, and (3) procedural settings. In summary, this qualitative study revealed unanticipated COVID-19 pandemic-related changes with primarily adverse effects on the provision of desired newborn circumcisions. Some of these changes may become permanent resulting in broad implications for policy makers that will likely need to adapt and redesign the processes and systems for the delivery of newborn circumcision care.

The Practice of Pharmaceutics and the Obligation to Expand Access to Investigational Drugs

2019 ◽  
Vol 45 (2) ◽  
pp. 193-211 ◽  
Author(s):  
Michael Buckley ◽  
Collin O’neil
Keyword(s):  
Clinical Trial ◽  
Moral Obligation ◽  
Future Research ◽  
Pharmaceutical Companies ◽  
Expanded Access ◽  
Moral Permissibility ◽  
Trial Process ◽  
Investigational Drugs ◽  
Potential Impact ◽  
Pharmaceutical Practice

Abstract Do pharmaceutical companies have a moral obligation to expand access to investigational drugs to patients outside the clinical trial? One reason for thinking they do not is that expanded access programs might negatively affect the clinical trial process. This potential impact creates dilemmas for practitioners who nevertheless acknowledge some moral reason for expanding access. Bioethicists have explained these reasons in terms of beneficence, compassion, or a principle of rescue, but their arguments have been limited to questions of moral permissibility, leaving for future research the question of whether expanded access is morally obligatory. We take up this further question and argue that pharmaceutical companies have a moral obligation to expand access. Our defense is not based on beneficence, compassion, or rescue, but instead on a reciprocal moral expectation resulting from existing social commitments that help ensure a robust pharmaceutical practice within the broader healthcare system. Our aim is to give this obligation, along with several others, a coherent and plausible structure within the wider clinical trial process so that one might better explain the sources of the dilemmas and their possible resolutions.

scholarly journals The Role of Physicians in Expanded Access to Investigational Drugs: A Mixed-Methods Study of Physicians’ Views and Experiences in The Netherlands

2021 ◽  
Author(s):  
Eline M. Bunnik ◽  
Nikkie Aarts
Keyword(s):  
Mixed Methods ◽  
The Netherlands ◽  
Treatment Options ◽  
Mixed Methods Study ◽  
Structured Interviews ◽  
Expanded Access ◽  
Safety Risks ◽  
Investigational Treatment ◽  
Investigational Drugs ◽  
Potential Benefits

AbstractTreating physicians have key roles to play in expanded access to investigational drugs, by identifying investigational treatment options, assessing the balance of risks and potential benefits, informing their patients, and applying to the regulatory authorities. This study is the first to explore physicians’ experiences and moral views, with the aim of understanding the conditions under which doctors decide to pursue expanded access for their patients and the obstacles and facilitators they encounter in the Netherlands. In this mixed-methods study, semi-structured interviews (n = 14) and a questionnaire (n = 90) were conducted with medical specialists across the country and analysed thematically. Typically, our respondents pursue expanded access in “back against the wall” situations and broadly support its classic requirements. They indicate practical hurdles related to reimbursement, the amount of time and effort required for the application, and unfamiliarity with the regulatory process. Some physicians are morally opposed to expanded access, with an appeal to safety risks, lack of evidence, and “false hope.” Some of these moral concerns and practical obstacles may be essential targets for change, if expanded access to unapproved drugs is to become available for wider groups of patients for whom standard treatment options are not—or no longer—available, on a more consistent and equal basis.

scholarly journals A survey of pediatric hematologists/oncologists’ perspectives on single patient Expanded Access and Right to Try

2021 ◽  
Vol 5 ◽  
pp. 239920262110059
Author(s):  
Carolyn Riley Chapman ◽  
Hayley M Belli ◽  
Danielle Leach ◽  
Lesha D Shah ◽  
Alison Bateman-House
Keyword(s):  
The United States ◽  
Recent Experience ◽  
Single Patient ◽  
Cross Sectional Survey ◽  
Eligibility Criteria ◽  
Cross Sectional ◽  
Expanded Access ◽  
Investigational Drugs ◽  
Survey Results ◽  
Physician Support

Introduction: Physicians in the United States play an essential role guiding patients through single patient pre-approval access (PAA) to investigational medical products via either the Food and Drug Administration (FDA)’s Expanded Access (EA) or the federal Right To Try (RTT) pathways. In this study, we sought to better understand pediatric hematologist/oncologists’ attitudes about seeking PAA, on behalf of single patients, to investigational drugs outside of clinical trials. Methods: A cross-sectional survey was developed and sent to pediatric hematologist/oncologists via St. Baldrick’s Foundation’s email distribution list. Results: Of 73 respondents (10.1% of those who received the survey), 56 met eligibility criteria and are included in the analysis. Over 80% ( n = 46) had prior experience with single patient PAA. Respondents were most concerned about the unknown risks and benefits of investigational drugs and financial implications of PAA for patients. One hundred percent and 91.1% of respondents indicated a willingness to support patients through EA and RTT pathways, respectively. When asked about their most recent experience with PAA, 40 out of 46 indicated that they used the FDA’s EA pathway to seek PAA and 4 out of 46 indicated that they used the RTT pathway. Of 44 respondents who had used the EA or RTT pathway, 43 indicated that the biotechnology or pharmaceutical company they solicited granted access to the requested product. Conclusion: Survey results support other findings suggesting a need for additional physician support and education about PAA and that physicians may have unequal access to information about investigational drugs and concerns about financial implications of PAA for their patients.

Overview of Current Approaches to Aural Rehabilitation for Adults With Cochlear Implants

2020 ◽  
Vol 5 (5) ◽  
pp. 1175-1187
Author(s):  
Rachel Glade ◽  
Erin Taylor ◽  
Deborah S. Culbertson ◽  
Christin Ray
Keyword(s):  
Health Care ◽  
Clinical Practice ◽  
Cochlear Implants ◽  
Practice Patterns ◽  
The United States ◽  
Care Providers ◽  
Aural Rehabilitation ◽  
Starting Point ◽  
Clinical Models ◽  
Hearing Health

Purpose This clinical focus article provides an overview of clinical models currently being used for the provision of comprehensive aural rehabilitation (AR) for adults with cochlear implants (CIs) in the Unites States. Method Clinical AR models utilized by hearing health care providers from nine clinics across the United States were discussed with regard to interprofessional AR practice patterns in the adult CI population. The clinical models were presented in the context of existing knowledge and gaps in the literature. Future directions were proposed for optimizing the provision of AR for the adult CI patient population. Findings/Conclusions There is a general agreement that AR is an integral part of hearing health care for adults with CIs. While the provision of AR is feasible in different clinical practice settings, service delivery models are variable across hearing health care professionals and settings. AR may include interprofessional collaboration among surgeons, audiologists, and speech-language pathologists with varying roles based on the characteristics of a particular setting. Despite various existing barriers, the clinical practice patterns identified here provide a starting point toward a more standard approach to comprehensive AR for adults with CIs.

More than victims: Resiliency of undocumented Latinas near Chicago, United States, and the strategic use of psychological suffering in obtaining legal status

2019 ◽  
Vol 16 (3) ◽  
pp. 361-368
Author(s):  
Stephanie Jean Kohl
Keyword(s):  
United States ◽  
Domestic Violence ◽  
Legal Status ◽  
The United States ◽  
Immigration Law ◽  
Application Process ◽  
Theoretical Concepts ◽  
Psychological Suffering ◽  
Visa Application ◽  
Biological Citizenship

Caught between abusive partners and restrictive immigration law, many undocumented Latina women are vulnerable to domestic violence in the United States. This article analyzes the U-Visa application process experienced by undocumented immigrant victims of domestic violence and their legal advisors in a suburb of Chicago, United States. Drawing on theoretical concepts of structural violence and biological citizenship, the article highlights the strategic use of psychological suffering related to domestic violence by applicants for such visas. It also investigates the complex intersection between immigration law and a humanitarian clause that creates a path towards legal status and eventual citizenship.

Review on the Clinical Pharmacology of Hydroxychloroquine Sulfate for the Treatment of COVID-19

2020 ◽  
Vol 21 (6) ◽  
pp. 427-435 ◽  
Author(s):  
Cheng Cui ◽  
Siqi Tu ◽  
Valerie Sia Jie En ◽  
Xiaobei Li ◽  
Xueting Yao ◽  
...  
Keyword(s):  
Drug Interactions ◽  
Clinical Efficacy ◽  
Relevant Literature ◽  
The United States ◽  
Efficacy And Safety ◽  
Open Label ◽  
Infected People ◽  
Treatment Regimens ◽  
Therapeutic Drugs

Background: As the number of severe acute respiratory syndrome coronavirus 2 (SARS-COV-2) infected people is greatly increasing worldwide, the international medical situation becomes very serious. Potential therapeutic drugs, vaccine and stem cell replacement methods are emerging, so it is urgent to find specific therapeutic drugs and the best treatment regimens. After the publications on hydroxychloroquine (HCQ) with anti- SARS-COV-2 activity in vitro, a small, non-randomized, open-label clinical trial showed that HCQ treatment was significantly associated with reduced viral load in patients with coronavirus disease-19 (COVID-19). Meanwhile, a large prophylaxis study of HCQ sulfate for COVID-19 has been initiated in the United States. HCQ offered a promising efficacy in the treatment of COVID-19, but the optimal administration is still being explored. Methods: We used the keyword "hydroxychloroquine" to conduct a literature search in PubMed to collect relevant literature on the mechanism of action of HCQ, its clinical efficacy and safety, pharmacokinetic characteristics, precautions for clinical use and drug interactions to extract and organize information. Results: This paper reviews the mechanism, clinical efficacy and safety, pharmacokinetic characteristics, exposureresponse relationship and precautions and drug interactions of HCQ, and summarizes dosage recommendations for HCQ sulfate. Conclusion: It has been proved that HCQ, which has an established safety profile, is effective against SARS-CoV-2 with sufficient pre-clinical rationale and evidence. Data from high-quality clinical trials are urgently needed worldwide.

Telemedicine for Patients with Inflammatory Bowel Disease (TELE-IBD) Clinical Trial: Qualitative Assessment of Participants’ Perceptions of the TELE-IBD Intervention (Preprint)

2019 ◽  
Author(s):  
Charlene C Quinn ◽  
Sarah Chard ◽  
Erin G Roth ◽  
J. Kevin Eckert ◽  
Katharine M Russman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Disease Activity ◽  
Remote Monitoring ◽  
Standard Care ◽  
Disease Process ◽  
The United States ◽  
Qualitative Assessment ◽  
Structured Interviews ◽  
Treatment Groups ◽  
Inflammatory Bowel

BACKGROUND Inflammatory bowel diseases (IBD), comprising Crohn’s disease and ulcerative colitis, affects 1 to 3 million people in the United States. Telemedicine has shown promise in IBD. The objective of the parent study, TELE-IBD, was to compare disease activity and quality of life (QoL) in a one-year randomized clinical trial of IBD patients receiving telemedicine versus standard care. Treatment groups experienced improvements in disease activity and QoL but there was not significant differences between groups. Study adherence to the text-based intervention was less than the 80% of the targeted goal. OBJECTIVE To understand adherence to remote monitoring, the goal of this qualitative assessment was to obtain TELE-IBD trial participants’ perceptions of the TELE-IBD system, including their recommendations for future TELE-IBD monitoring. METHODS In the parent study, patients attending three tertiary referral centers with worsening IBD symptoms in the previous two years were eligible for randomization to remote monitoring via texts every other week (EOW), weekly (W) or standard care. Participants (n=348) were evenly enrolled in the treatment groups and 259 (74.4%) completed the study. For this study, a purposive sample of adherent (N=15) and non-adherent (N=14) patients was drawn from the TELE-IBD trial population. Adherence was defined as the completion of 80% or more of the W or EOW self-assessments. Semi-structured interviews conducted by phone surveyed 1) the strengths and benefits of TELE-IBD; 2) challenges associated with using TELE-IBD; and 3) how to improve the TELE-IBD intervention. Interviews were recorded, professionally transcribed, and coded based on a priori concepts and emergent themes with the aid of ATLAS.ti qualitative data analysis software. RESULTS Participants' discussions centered on three elements of the intervention: 1) self-assessment questions, 2) action plans, and 3) educational messages. Participants also commented on: text-based platform, depression and adherence, TELE-IBD system in place of office visit, and their recommendations for future TELE-IBD systems. Adherent and non-adherent participants prefer a flexible system that is personalized, including targeted education messages, and they perceive TELE-IBD as effective in facilitating IBD self-management. CONCLUSIONS Participants identified clear benefits to the TELE-IBD system, including obtaining a better understanding of the disease process, monitoring their symptoms, and feeling connected to their health care provider. Participants' perceptions obtained in this qualitative study will assist in improving the TELE-IBD system to be more responsive to patients with IBD. CLINICALTRIAL NCT01692743

scholarly journals Male autistic youth experiences of belonging in integrated physical education

Autism ◽  
2021 ◽  
pp. 136236132110186
Author(s):  
Justin A Haegele ◽  
Anthony J Maher
Keyword(s):  
Physical Education ◽  
Peer Relationships ◽  
Peer Interactions ◽  
Physical Space ◽  
The United States ◽  
Educational Needs ◽  
Subjective Experiences ◽  
Structured Interviews ◽  
Autistic People ◽  
Locker Room

The study examined the subjective experiences of autistic youth regarding the role of peer interactions and relationships in feelings of belonging in integrated physical education classes. The term integrated is used to describe a setting in which all students, regardless of educational needs, are educated in the same physical space. Eight autistic youth (all male, aged 13–18 years) who had received most of their physical education in integrated classes acted as participants. Semi-structured interviews were used to generate qualitative data, which were analyzed using a reflexive thematic approach. Findings are presented in three themes: (a) bullying can lead to self-harm and self-isolation, (b) peer interactions and relationships in the locker room, and (c) peer relationships are based on shared interests and take time to develop. Even though autistic students were educated in the same spaces as their non-autistic peers, feelings of belonging were largely unavailable to them. Lay abstract Recent years have seen calls to amplify the voices of autistic people in research about their subjective experiences. Despite this, we know little about how autistic youth experience integrated physical education, particularly in the United States. The term integrated is used to describe a setting in which all students, regardless of educational needs, are educated in the same physical space. In this study, we sought to explore the perspectives of autistic youth toward their experiences in integrated physical education, and the roles of social interactions and relationships with peers in those experiences. Findings noted that several factors influenced the ways and extent to which our participants interacted with their peers during physical education. Unfortunately, most of our participants recalled experiencing bullying, and that physical education offered an environment where bullying was most frequent and comparatively unique compared to other contexts throughout the school day. The locker room, a space linked to physical education, was of particular concern because of a lack of teacher presence. Despite the negative views of and experiences in physical education, there was evidence of participants actively pursuing to connect with peers in this context. However, most instances where participants recalled pursuing friendship were not welcomed from others, which stunted their sense of belonging in this space. Given the role that belonging plays in what it means “to be included,” our research supports emerging ideas that even though autistic students were educated in the same physical spaces as their non-autistic peers, feelings of inclusion were largely absent.

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