scholarly journals Seeing the bigger picture: Qualitative research in the Zoom® age

2021 ◽  
Vol 8 (1) ◽  
pp. 141-144
Author(s):  
Simon Fletcher
Keyword(s):  
Clinical Trials ◽  
Focus Groups ◽  
Life Stories ◽  
Qualitative Interviews ◽  
Real Life ◽  
Know How ◽  
Life Impact ◽  
New Treatment ◽  
New Treatments

Abstract Participants in clinical trials for new haemophilia treatments are routinely asked to complete quality of life (QoL) questionnaires using validated and disease-specific instruments. Yet too often in clinical research we know very little about the life stories of individuals, making it difficult to know how they have been affected by a new therapy and what exactly has changed for the better – or for the worse. In my own research, I wanted to understand the differences that new treatments are really making to people's everyday lives. While traditional QoL instruments can be helpful, using a qualitative approach that involves speaking directly with people with haemophilia (PwH) and their family members has enabled me find out what has really been going on their lives, including impacts on the wider family. The Covid pandemic and the need to maintain social distancing changed the way in which my research has been carried out, but in fact provided an opportunity to see an even bigger picture. I believe that using videoconferencing platforms to conduct interviews and focus groups has both allowed me to see more of the world in which the participants live and has enabled participants to be more relaxed and open in their conversations, resulting in a potentially richer dataset. While this approach to qualitative QoL research should not replace interviews and focus groups, the use of videoconferencing should be considered as another methodology researchers can and should use to enable them to glean the richest data possible. Qualitative interviews offer an important complementary addition to the validated QoL measures used in clinical trials, enabling us to hear more about where improvements have occurred, where further improvements can be made, and the real-life impact of a new treatment for PwH and their families.

scholarly journals Current challenges in the development of new treatments for lupus

2019 ◽  
Vol 78 (6) ◽  
pp. 729-735 ◽  
Author(s):  
Maria Dall'Era ◽  
Ian N Bruce ◽  
Caroline Gordon ◽  
Susan Manzi ◽  
Janis McCaffrey ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Lupus Erythematosus ◽  
Turn Of The Millennium ◽  
Eligibility Criteria ◽  
Considerable Impact ◽  
New Treatment ◽  
New Treatments ◽  
Chronic Autoimmune Disease

Systemic lupus erythematosus (SLE) is a chronic autoimmune disease with a considerable impact on patients’ quality of life. Despite the plethora of clinical trials for SLE since the turn of the millennium, only one new treatment has been approved for the condition, and the overall pace of successful drug development remains slow. Nevertheless, the myriad of clinical studies has yielded insights that have informed and refined our understanding of eligibility criteria, outcome measures and trial design in SLE. In this review, we highlight the achievements of clinical trials as well as the major pitfalls that have been identified in drug development for SLE and, in doing so, identify areas where collaboration and consensus will be important to facilitate progress.

scholarly journals Approaches to Assessing the Quality of Observational Studies of Clinical Practice Based on the Big Data Analysis

2021 ◽  
Vol 17 (4) ◽  
pp. 584-593
Author(s):  
S. R. Gilyarevsky
Keyword(s):  
Clinical Trials ◽  
Big Data ◽  
Clinical Practice ◽  
Observational Studies ◽  
Randomized Clinical Trials ◽  
Oral Anticoagulants ◽  
Direct Oral Anticoagulants ◽  
Real Life ◽  
Hierarchy Of Evidence

The article is devoted to the discussion of the problems of assessing the quality of observational studies in real clinical practice and determining their place in the hierarchy of evidence-based information. The concept of “big data” and the acceptability of using such a term to refer to large observational studies is being discussed. Data on the limitations of administrative and claims databases when performing observational studies to assess the effects of interventions are presented. The concept of confounding factors influencing the results of observational studies is discussed. Modern approaches to reducing the severity of bias in real-life clinical practice studies are presented. The criteria for assessing the quality of observational pharmacoepidemiological studies and the fundamental differences between such studies and randomized clinical trials are presented. The results of systematic reviews of real-life clinical trials to assess the effects of direct oral anticoagulants are discussed. 

scholarly journals Brief Engagement and Acceptance Coaching for Hospice Settings (the BEACHeS study): results from a Phase I study of acceptability and initial effectiveness in people with non-curative cancer

2021 ◽  
Vol 20 (1) ◽  
Author(s):  
Nicholas J. Hulbert-Williams ◽  
Sabrina F. Norwood ◽  
David Gillanders ◽  
Anne M. Finucane ◽  
Juliet Spiller ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Palliative Care ◽  
Focus Groups ◽  
Advanced Cancer ◽  
Psychological Intervention ◽  
Qualitative Interviews ◽  
Future Research ◽  
Study Results ◽  
Palliative Care Setting

Abstract Objectives Transitioning into palliative care is psychologically demanding for people with advanced cancer, and there is a need for acceptable and effective interventions to support this. We aimed to develop and pilot test a brief Acceptance and Commitment Therapy (ACT) based intervention to improve quality of life and distress. Methods Our mixed-method design included: (i) quantitative effectiveness testing using Single Case Experimental Design (SCED), (ii) qualitative interviews with participants, and (iii) focus groups with hospice staff. The five-session, in-person intervention was delivered to 10 participants; five completed at least 80%. Results At baseline, participants reported poor quality of life but low distress. Most experienced substantial physical health deterioration during the study. SCED analysis methods did not show conclusively significant effects, but there was some indication that outcome improvement followed changes in expected intervention processes variables. Quantitative and qualitative data together demonstrates acceptability, perceived effectiveness and safety of the intervention. Qualitative interviews and focus groups were also used to gain feedback on intervention content and to make design recommendations to maximise success of later feasibility trials. Conclusions This study adds to the growing evidence base for ACT in people with advanced cancer. A number of potential intervention mechanisms, for example a distress-buffering hypothesis, are raised by our data and these should be addressed in future research using randomised controlled trial designs. Our methodological recommendations—including recruiting non-cancer diagnoses, and earlier in the treatment trajectory—likely apply more broadly to the delivery of psychological intervention in the palliative care setting. This study was pre-registered on the Open Science Framework (Ref: 46,033) and retrospectively registered on the ISRCTN registry (Ref: ISRCTN12084782).

BioDay Registry: Prospective, observational data collection regarding the use of new systemic treatment options in patients with atopic diseases in daily practice (Preprint)

2020 ◽  
Author(s):  
Jorien BioDay Registry ◽  
Lieneke Ariens ◽  
Lotte Spekhorst ◽  
Ischa Kummeling ◽  
Judith Thijs ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Medical Ethics ◽  
Outcome Measures ◽  
Systemic Treatment ◽  
Treatment Options ◽  
Real Life ◽  
Daily Practice ◽  
Ethics Committee ◽  
Atopic Diseases ◽  
New Treatment

BACKGROUND Dupilumab is the first antibody based treatment for atopic dermatitis (AD). It is expected that many new treatment options become available for the treatment of atopic diseases in the near future. Despite the high quality of clinical trials, results are not always generalizable to daily practice. OBJECTIVE Registry based collection of real life data can add information that is not gained in clinical trials. METHODS The BioDay Registry prospectively collects real life treatment results regarding effectiveness and safety of patients treated with new systemic treatment options for AD in a multicenter setting. Effectiveness is assessed based on both physician measured clinical eczema scores as well as patient-reported outcome measures. Long-term safety risks are monitored. The possibility for dose reduction in patients with disease control can be investigated. The treatment effect on other atopic co-morbidities will be monitored. The BioDay Registry can be easily adjusted, and new modules on new treatment options can be added. As outcome measures are in line with the core outcomes for eczema recommended by the global Harmonising Outcome Measures for Eczema (HOME) initiative, it is possible to merge data with other registries in future. RESULTS The BioDay Registry was considered as non-interventional by the local Medical Ethics Committee and the collection of data is performed according to the Helsinki Declaration. Protocol amendments will always be submitted for review to the Medical Ethics Committee. CONCLUSIONS The BioDay Registry will provide unique prospectively collected data on the use of targeted therapies in daily practice for AD patients. The results of this registry will contribute to the development of more personalized treatment strategies for patients with moderate to severe AD. CLINICALTRIAL ClinicalTrials.gov identifier: NCT03549416, retrospectively registered June 8, 2018.

scholarly journals Challenges and Opportunities for Real-World Evidence in Metastatic Luminal Breast Cancer

Breast Care ◽  
2021 ◽  
pp. 1-7
Author(s):  
Diana Lüftner ◽  
Andreas D. Hartkopf ◽  
Michael P. Lux ◽  
Friedrich Overkamp ◽  
Hans Tesch ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Clinical Trials ◽  
Metastatic Breast Cancer ◽  
Real World ◽  
Real Life ◽  
Metastatic Breast ◽  
Molecular Structures ◽  
Luminal Breast Cancer ◽  
The Real

Background: The therapeutic armamentarium for patients with metastatic breast cancer is becoming more and more specific. Recommendations from clinical trials are not available for all treatment situations and patient subgroups, and it is therefore important to collect real-world data. Summary: To develop recommendations for up-to-date treatments and participation in clinical trials for patients with metastatic breast cancer, the Prospective Academic Translational Research PRAEGNANT Network was established to optimize the quality of oncological care in the advanced therapeutic setting. The main aim of PRAEGNANT is to systematically record medical care for patients with metastatic breast cancer in the real-life setting, including the outcome and side effects of different treatment strategies, to monitor quality-of-life changes during therapy, to identify patients eligible for participation in clinical studies, and to allow targeted therapies based on the molecular structures of breast carcinomas. Key Messages: This article describes the PRAEGNANT network and sheds light on the question of whether the various end points from clinical trials can be transferred to the real-world treatment situation.

Adaptive Designs for Non-inferiority Trials with Multiple Experimental Treatments

2017 ◽  
Vol 27 (11) ◽  
pp. 3255-3270 ◽  
Author(s):  
Wenfu Xu ◽  
Feifang Hu ◽  
Siu Hung Cheung
Keyword(s):  
Clinical Trials ◽  
Adaptive Design ◽  
Treatment Allocation ◽  
Balanced Design ◽  
Adaptive Treatment ◽  
Biased Coin Design ◽  
New Treatment ◽  
Experimental Treatments ◽  
Treatment Comparisons ◽  
New Treatments

The increase in the popularity of non-inferiority clinical trials represents the increasing need to search for substitutes for some reference (standard) treatments. A new treatment would be preferred to the standard treatment if the benefits of adopting it outweigh a possible clinically insignificant reduction in treatment efficacy (non-inferiority margin). Statistical procedures have recently been developed for treatment comparisons in non-inferiority clinical trials that have multiple experimental (new) treatments. An ethical concern for non-inferiority trials is that some patients undergo the less effective treatments; this problem is more serious when multiple experimental treatments are included in a balanced trial in which the sample sizes are the same for all experimental treatments. With the aim of giving fewer patients the inferior treatments, we propose a response-adaptive treatment allocation scheme that is based on the doubly adaptive biased coin design. The proposed adaptive design is also shown to be superior to the balanced design in terms of testing power.

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