scholarly journals Stakeholder perspectives on clinical research related to therapies for rare diseases: therapeutic misconception and the value of research

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Kylie Tingley ◽  
◽  
Doug Coyle ◽  
Ian D. Graham ◽  
Pranesh Chakraborty ◽  
...  
Keyword(s):  
Focus Group ◽  
Clinical Research ◽  
Health Care Providers ◽  
Rare Diseases ◽  
Metabolic Diseases ◽  
Population Level ◽  
Qualitative Description ◽  
Therapeutic Misconception ◽  
Care Providers ◽  
Access To Treatment

Abstract Background For many rare diseases, few treatments are supported by strong evidence. Patients, family members, health care providers, and policy-makers thus have to consider whether to accept, recommend, or fund treatments with uncertain clinical effectiveness. They must also consider whether and how to contribute to clinical research that may involve receiving or providing the therapy being evaluated. Objective To understand why and how patients and families with rare metabolic diseases, specialist metabolic physicians, and health policy advisors choose whether to participate in studies and how they use and value research. Methods We conducted separate focus group interviews with each stakeholder group (three groups in total); two groups were conducted by telephone and the third was held in-person. Participants were recruited using purposive sampling. We analyzed each interview transcript sequentially using a qualitative description approach to inductively identify key themes. Several strategies to ensure credibility and trustworthiness were used including debriefing sessions after each focus group and having multiple team members review transcripts. Results Four patients/caregivers, six physicians, and three policy advisors participated. Our findings did not support conventional perspectives that therapeutic misconception (gaining access to treatment) is the main motivating factor for patients/caregivers to participate in clinical research. Rather, patients’/caregivers’ expressed reasons for participating in research included advancing science for the next generation and having an opportunity to share their experiences. Patients/caregivers and physicians described the difficulties in weighing risks versus benefits of accepting treatments not well-supported by evidence. Physicians also reported feeling conflicted in their dual role as patient advisor/advocate and evaluator of the evidence. Policy advisors were primarily focused on critically appraising the evidence to make recommendations for the health system. Conclusions Stakeholders differ in their perspectives on rare disease research but share concerns about the risks versus benefits of therapies when making individual- and population-level decisions.

scholarly journals Access to information supporting availability of medicines for patients suffering from rare diseases looking for possible treatments: the EuOrphan Service

Medicina ◽  
2007 ◽  
Vol 43 (6) ◽  
pp. 441 ◽  
Author(s):  
Donatas Stakišaitis ◽  
Indrė Špokienė ◽  
Jonas Juškevičius ◽  
Konstantinas Valuckas ◽  
Paola Baiardi
Keyword(s):  
Health Care ◽  
Health Care Providers ◽  
Rare Diseases ◽  
Health Care Professionals ◽  
Large Population ◽  
New Drugs ◽  
Public Health Care ◽  
Quality Information ◽  
Care Providers ◽  
Pharmaceutical Industries

Currently in Europe, approximately 30 million people suffer from rare diseases, and a major problem is that many patients do not have access to quality healthcare for their disorders. Moreover, there is also a lack of quality information and a networking system aimed at supporting interaction among patients, clinicians, researchers, pharmaceutical industries, and governmental bodies. The purpose of this article is to inform physicians, public health care professionals, and other health care providers about EuOrphan service, the aim of which is to ensure easier access to quality information on rare diseases and their treatment. A set of web-based services is available at www.euorphan.com where information for target-users on treatments and products available worldwide for rare disease care as well as indications about healthcare centers are provided. Moreover, the service aims at providing consultancies for pharmaceutical companies to ultimately support the European legislation in bringing new drugs of a high ethical standard to the market and to exert a positive impact on the large population of patients suffering from rare diseases in Europe. The services provided by EuOrphan can facilitate concrete networking among patients, patient associations, doctors, and companies and also support the organization of clinical trials. In this perspective, EuOrphan could become a very valuable tool for globalizing the information about the availability of treatment (authorized or under development) of orphan patients.

Critical Aspects of a Sustainable Clinical Research Program in the Community-Based Oncology Practice

2019 ◽  
pp. 176-184 ◽  
Author(s):  
Jennifer L. Ersek ◽  
Stephanie L. Graff ◽  
Francis P. Arena ◽  
Neelima Denduluri ◽  
Edward S. Kim
Keyword(s):  
Clinical Research ◽  
Patient Care ◽  
Health Care Providers ◽  
Research Program ◽  
Improve Patient Care ◽  
Care Providers ◽  
Community Based ◽  
Technological Infrastructure ◽  
Community Based Research ◽  
Research Programs

Increasing enrollment into clinical trials is a top priority across the field of oncology. Because the vast majority of those afflicted with cancer receive their care in the community, creating strong clinical research programs in the community-based setting is important. This article comprehensively outlines the most important elements of creating and sustaining a successful community-based research program. Establishing a clear mission and defining the scope of the research program in collaboration with key physicians and administrative leadership are critical to success. Standard operating procedures should detail operational processes. Ensuring sound financial planning and protected physician time are crucial for a healthy program. Providing mentorship opportunities to investigators and other team members will provide necessary guidance for junior investigators and long-term program stability. Prioritizing provider and patient volunteer engagement through education and awareness will potentially improve enrollment and research ownership. Incorporating administrative and clinical research staff and health care providers, including physicians, advanced practice providers, and pharmacists, will result in a multidisciplinary and unified approach and may also promote research as a routine part of patient care. Regular safety and scientific meetings will reduce regulatory complications and, most importantly, improve patient care. Other keys to a successful program include establishing a diverse trial portfolio, collaboration between different institutions, and ensuring appropriate technological infrastructure. Serial programmatic review provides opportunities to refine suboptimal practices and recognize successful strategies. Community-based research programs are critical to improve access to optimal cancer care. Implementation of successful programs is possible with a collaborative and multidisciplinary approach.

Clinical Research With Pregnant Women: Perspectives of Pregnant Women, Health Care Providers, and Researchers

2018 ◽  
Vol 28 (13) ◽  
pp. 2033-2047 ◽  
Author(s):  
Kyoko Wada ◽  
Marilyn K. Evans ◽  
Barbra de Vrijer ◽  
Jeff Nisker
Keyword(s):  
Health Care ◽  
Prenatal Care ◽  
Clinical Research ◽  
Pregnant Women ◽  
Health Care Providers ◽  
Research Participation ◽  
Well Being ◽  
Constructivist Grounded Theory ◽  
Care Providers ◽  
Women Health

Limited clinical research with pregnant women has resulted in insufficient data to promote evidence-informed prenatal care. Charmaz’s constructivist grounded theory methodology was used to explore how research with pregnant women would be determined ethically acceptable from the perspectives of pregnant women, health care providers, and researchers in reproductive sciences. Semistructured interviews were conducted with a purposive sample of 12 pregnant women, 10 health care providers, and nine reproductive science researchers. All three groups suggested the importance of informed consent and that permissible risk would be very limited and complex, being dependent on the personal benefits and risks of each particular study. Pregnant women, clinicians, and researchers shared concerns about the well-being of the woman and her fetus, and expressed a dilemma between promoting research for evidence-informed prenatal care while securing the safety in the course of research participation.

Barriers and Facilitators to Obtaining Informed Consent in a Critical Care Pediatric Research Ward in Southern Malawi

2019 ◽  
Vol 14 (2) ◽  
pp. 152-168
Author(s):  
Lucinda Manda-Taylor ◽  
Alice Liomba ◽  
Terrie E. Taylor ◽  
Kristan Elwell
Keyword(s):  
Critical Care ◽  
Informed Consent ◽  
Clinical Research ◽  
Critically Ill ◽  
Health Care Providers ◽  
Pediatric Intensive Care ◽  
Research Participant ◽  
Critically Ill Children ◽  
Parental Distress ◽  
Care Providers

Informed consent is an ethical requirement in clinical research. Obtaining informed consent is challenging in resource-constrained settings. We report results of a formative qualitative study that examined factors that facilitate and hinder informed consent for clinical research among critically ill children in Malawi. We argue that truly informed consent in a pediatric intensive care unit (PICU) is challenged by parental distress, time constraints when balancing care for critically ill patients with research-related tasks, and social hierarchies and community mistrust toward certain research procedures. We interviewed health care providers and parents of children attending a critical care unit to identify potential challenges and solicit strategies for addressing them. Providers and caregivers suggested practical solutions to enhance research participant understanding of clinical trial research, including the use of visual materials, community engagement strategies, and using patients as advocates in promoting understanding of research procedures.

scholarly journals Identifying and describing a model region to evaluate the impact of telemedicine

2019 ◽  
Vol 29 (Supplement_4) ◽  
Author(s):  
L Harst ◽  
S Oswald ◽  
P Timpel
Keyword(s):  
Health Care ◽  
Focus Group ◽  
Focus Groups ◽  
Health Care Providers ◽  
Rural Areas ◽  
Demographic Data ◽  
Care Providers ◽  
Age Related ◽  
Model Region ◽  
The Impact

Abstract Background Telemedicine solutions providing patient-centered care over distance need to be integrated into the regional setting. The acceptance by both providers and patients hat to be continuously evaluated using methods of participatory implementation research. In controlled trials, often taking place in laboratory settings, these methods cannot be applied. In the following, research in progress is presented. Methods Based on socio-demographic data, epidemiology prevalence of age-related chronic diseases and data on the value of health care provision in Saxony, Germany a model region was chosen. Then, a focus group (n = 6) was conducted to differentiate the results and analyze the health networks of patients. For this, network maps putting the individual in the middle and his/her sources of information and support in case of illness in concentric circles around it, were used. The focus group was audiotaped, transcribed and analyzed by two researchers using MaxQDA. Results With a mean age of 47.8 years (n = 17,431), high prevalence of diabetes (>15.85 %) and hypertension (>39.1%) and an expected shortage of primary physicians in 2030, the town of Kamenz is a mirror image of the current health care challenges in rural areas of Saxony. Participants of the focus groups also stated problems in finding a primary physician or a dentist. Compensatory behavior, such as traveling large distances, relying on self-researched online diagnoses and immediately going to the emergency room for medical support was described. According to the network maps, primary sources of support in case of illness are partners and relatives, yet there is little connection between those and health care providers, as well as between different medical specialists. Conclusions The results will lead to potential use cases of telemedicine to be included into a standardized questionnaire for the assessment of telemedicine readiness in the model region. Key messages Telemedicine implementation in a rural area can be studied using a participatory approach. Focus groups and network maps are useful qualitative methods for participatory research and can inform the design of quantitative measurements.

A focus group study of DES daughters: implications for health care providers

2000 ◽  
Vol 9 (5) ◽  
pp. 439-444 ◽  
Author(s):  
Suzanne S. Duke ◽  
Sarah A. Mcgraw ◽  
Nancy E. Avis ◽  
Amanda Sherman
Keyword(s):  
Health Care ◽  
Focus Group ◽  
Health Care Providers ◽  
Care Providers ◽  
Focus Group Study

scholarly journals Walk the Talk: The Transforming Journey of Facility-Based Death Review Committee from Stillbirths to Neonates

2021 ◽  
Vol 2021 ◽  
pp. 1-12
Author(s):  
Yousef S. Khader ◽  
Nihaya A. Al-Sheyab ◽  
Khulood K. Shattnawi ◽  
Mohammad S. Alyahya ◽  
Anwar Batieha
Keyword(s):  
Focus Group ◽  
Health Care Providers ◽  
Healthcare Services ◽  
Power Dynamics ◽  
Care Providers ◽  
Review Committee ◽  
Contributing Factors ◽  
Neonatal Deaths ◽  
Modifiable Factors

Background. Facility-based death review committee (DRC) of neonatal deaths and stillbirths can encourage stakeholders to enhance the quality of care during the antenatal period and labour to improve birth outcomes. To understand the benefits and impact of the DRCs, this study was aimed at exploring the DRC members’ perception about the role and benefits of the newly developed facility-based DRCs in five pilot hospitals in Jordan, to assess women empowerment, decision-making process, power dynamics, culture and genderism as contributing factors for deaths, and impact of COVID-19 lockdown on births. Methods. A descriptive study of a qualitative design—using focus group discussions—was conducted after one year of establishing DRCs in 5 pilot large hospitals. The number of participants in each focus group ranged from 8 to10, and the total number of participants was 45 HCPs (nurses and doctors). Questions were consecutively asked in each focus group. The moderator asked the main questions from the guide and then used probing as needed. A second researcher observed the conversation and took field notes. Results. Overall, there was an agreement among the majority of DRC members across all hospitals that the DRC was successful in identifying the exact cause of neonatal deaths and stillbirths as well as associated modifiable factors. There was also a consensus that the DRC contributed to an improvement in health services provided for pregnant women and newborns as well as protecting human rights and enabling women to be more interdependent in taking decisions related to family planning. Moreover, the DRC agreed that a proportion of the neonatal deaths and stillbirths occurring in the hospitals could have been prevented if adequate antenatal care was provided and some traditional harmful practices were avoided. Conclusions. Facility-based neonatal death review audit is practical and can be used to identify exact causes of maternal and neonatal deaths and is a valuable tool for hospital quality indicators. It can also change the perception and practice of health care providers, which may be reflected in improving the quality of provided healthcare services.

scholarly journals A design and focus group evaluation of dietary choices tools for an underserved population

2016 ◽  
Vol 6 (2) ◽  
pp. 101-108
Author(s):  
Tressa McMorris ◽  
Gary Sweet ◽  
Christopher J. Sullivan ◽  
Nicole B. Washington ◽  
Nancy Brahm
Keyword(s):  
Focus Group ◽  
Health Care Providers ◽  
Pilot Project ◽  
Mental Illnesses ◽  
Community Treatment ◽  
Specific Information ◽  
Care Providers ◽  
Visual Aids ◽  
Group Feedback ◽  
Dietary Choices

Abstract Introduction: Our objective was to develop and evaluate dietary teaching tools for a select population diagnosed with a severe mental illness and limited financial ability. Patients with severe mental illnesses face many challenges, including common health comorbidities of diabetes, high blood pressure, high cholesterol, and obesity. Cognitive deficits may limit educational programming; financial resources can affect access to a healthy diet. The Integrated Multidisciplinary Program of Assertive Community Treatment (IMPACT) program, a university-based program, provides individualized services to this population. One focus is healthy nutritional choices. Methods: In Phase One, a clinical pharmacist and a first-year pharmacy resident created visual aids. These cards were given to health care providers (HCPs) to be used with IMPACT members. HCPs were asked to participate in a focus group and provide feedback. Phase Two: Based on specific focus group feedback, additional resources were created to address identified nutritional needs. Results: Phase One: Ten cards were created and distributed to the HCPs. A focus group was conducted. HCPs reported the cards were useful in opening dietary choices dialogues and were able to give more specific information on alternative choices. Phase Two: From focus group feedback, specific cards for disease states, calorie guidelines, and budget limitations were developed. HCPs immediately utilized them. Discussion: This pilot project was used to design and create educational cards to facilitate discussions on healthy or healthier dietary choices. Feedback from the HCPs participating in the focus group was positive, and they were enthusiastic about both sets of cards, particularly those pertaining to budget choices.

scholarly journals se-atlas: Mapping of Health Care Providers for People With Rare Diseases - a Cross-sectional Survey From the User Perspective

2021 ◽  
Author(s):  
Jannik Schaaf ◽  
Michaela Neff ◽  
Manuela Till ◽  
Niels Tegtbauer ◽  
Holger Storf
Keyword(s):  
Health Care ◽  
Health Care Providers ◽  
Rare Diseases ◽  
User Satisfaction ◽  
Online Survey ◽  
Care Providers ◽  
Cross Sectional Survey ◽  
Total Period ◽  
Patient Organisations ◽  
System Usability Scale

Abstract BackgroundIn rare diseases, only a low number of regionally distributed experts are available in medical care. The health service platform for rare diseases (www.se-atlas.de) provides a search for health care providers and patient organisations in Germany for specific rare diseases and presents the results to patients or physicians. The objective of this study was to examine the background and purpose, user satisfaction and usability when using se-atlas and to receive suggestions on improvements for implementation in the next release of se-atlas. MethodsWe conducted an online survey over a total period of five weeks between December 2020 and January 2021. Participants were members of patient organisations of rare diseases and experts of rare diseases centres in Germany. The questionnaire addressed the objectives of this study in 10 questions. We used Likert scales (4 to 6 points), as well as the System Usability Scale to measure usability (range: 0 to 100). The data obtained from the survey was analysed descriptively. ResultsIn total, 55 participants completed the survey (16 experts and 39 members of patient organisations). The results demonstrate that users know se-atlas mainly through patient organisations and the German National Action League for People with Rare Diseases. Furthermore, the experts use se-atlas more frequently than members of patient organisations do. Regarding to user satisfaction, participants were satisfied when using se-atlas (scale 1-6, mean = 4.31, SD = 1.18). They rated se-atlas functions with an average between 3.82 and 4.4 (scale 1-6). Additionally, se-atlas functions were considered as important with an average between 3.11 and 3.75 (scale 1-4). With regard to usability, the website was rated with an overall SUS score of 67.1, whereas the results differ between the participants group (experts = 76.1, patient organisations = 63.1). Moreover, participants made suggestions, e.g. that more disease entries should be available and usability can be improved. ConclusionsThis study involved experts and members of patient organisations to assess the background and purpose, user satisfaction and usability when using se-atlas. Despite the promising results and first new implementations, further optimisations of the platform in terms of usability and various functionalities are necessary.

Sign in / Sign up

Export Citation Format

Share Document