Objective: To update on and describe the role of Disease Specific Programmes (DSPs), a multi-perspective real-world data (RWD) source, in the context of the evolution of the value and acceptance of real-world evidence (RWE) in clinical, regulatory and guideline decision-making.Methods: DSPs are multinational, multi-subscriber, multi-therapy cross-sectional surveys incorporating retrospective data collection from patient, caregiver and physician perspectives. Information collected covers the patient journey, including treatment/prescribing patterns and rationale, patient-reported outcomes, impact on work and everyday activities, attitudes towards and perceptions of the condition, adherence to treatment and burden of illness. Published peer-reviewed DSP papers were aligned with current key RWE themes identified in the literature, alongside their contribution to RWE.Results: RWE themes examined were: using RWE to inform clinical practice, patient and caregiver engagement, RWE role in supporting health technology assessments and regulatory submissions, informing value-driven healthcare decisions, real-world patient subgroup differences and therapeutic inertia/unmet needs; highlighting patients' and caregivers' experience of living with a disease, disconnect from their physicians, unmet needs and educational gaps.Conclusions: DSPs provide a wealth of RWD in addition to evidence generated by registries, clinical trials and observational research, with wide use for the pharmaceutical industry, government, funding/regulatory bodies, clinical practice guideline insights and, most importantly, informing improvements in people's lives. The depth, breadth and heritage of information collected via DSPs since 1995 is unparalleled, extending understanding of how diseases are managed by physicians in routine clinical practice and why treatment choices are made, patients' perceptions of their disease management and caregiver burden.

Real-world evidence generation from patients, their caregivers and physicians supporting clinical, regulatory and guideline decisions: an update on Disease Specific Programmes.

Asthma and chronic obstructive pulmonary disease (COPD) are associated with substantial morbidity and mortality. This analysis assessed patient, caregiver, and healthcare provider (HCP) opinions regarding asthma and COPD diagnosis and treatment, including inhaler device preferences. 

Perspectives on Treatment Decisions, Preferences, and Adherence and Long-Term Management in Asthma and COPD: A Qualitative Analysis of Patient, Caregiver, and Healthcare Provider Insights

What Do Adults With Cystic Fibrosis Want From Their Doctors?

Patient preferences are qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions. In simpler terms, they are assessments of what patients want or would accept in their care. This PhD thesis presents methodological questions regarding the assessment of patient preferences along with research addressing these questions. The aim of this thesis is to help stakeholders who would potentially use this information to better understand and conduct patient preference studies and support the use of patient preferences in medical product decision-making. Chapter 1 reports on a survey conducted with members of the PREFER consortium. In this survey the stakeholders ranked a list of methodological questions regarding their priority for research to better understand and conduct patient preference studies and support their use in decision making. From this prioritized list, the following research topics are addressed in this thesis. First, the generalizability of preferences from one specific population in a disease to different populations in that or related diseases is addressed in Chapters 2 and 3. Chapter 2 compares the preferences of Diabetes patients in the Netherlands and Poland for glucose monitoring technology. Chapter 3 presents a study identifying common opinions about the most important areas of unmet medical needs in patients with two types of neuro-musculoskeletal diseases, myotonic dystrophy type 1 and mitochondrial disorders and their caregivers. Second, the impact of attribute framing on preferences is addressed in Chapter 4 using a case study in which participants were asked to complete a discrete choice experiment which varied in the way that the attributes were framed (positively, negatively, or both) when presented to participants. Third, how the results of simpler/cheaper preference elicitation methods differ from more complex/expensive methods is assessed in two case studies using Discrete Choice Experiments and Swing Weighting. In Chapter 5 these methods are compared in a case study assessing patient preferences for glucose monitoring for self-management of diabetes, and in Chapter 6 they are compared in a case study measuring treatment preferences of patients with non-small cell lung cancer. Fourth, how preferences results differ when participants are presented with information in a video-based educational tool versus traditional text-based education is explored in Chapter 7 using a case study of diabetes patients. Finally, the initial aim of Chapter 1 is revisited in Chapter 8 which reports on a second survey (conducted 4 years after the first study), asking stakeholders in the research area of patient preference assessment what methodological topics and questions for future studies they think are important to answer to increase acceptance of preference methods and the use of their results by decision makers. This thesis concludes with a summary of the findings, a general discussion of the research conducted, and the implications for future patient preference studies. 

Methodological Questions Regarding the Elicitation of Patient Preferences and Empirical Evidence to Help Address Them

Abstract Objectives While patient input to health technology assessment (HTA) has traditionally been of a qualitative nature, there is increasing interest to integrate quantitative evidence from patient preference studies into HTA decision making. Preference data can be used to generate disease-specific health utility data. We generated a health utility score for patients with chronic obstructive pulmonary disease (COPD) and consider its use within HTAs. Methods Based on qualitative research, six symptoms were identified as important to COPD patients: shortness of breath, exacerbations, chronic cough, mucus secretion, sleep disturbance, and urinary incontinence. We employed a discrete choice experiment (DCE) and the random parameter logistic regression technique to estimate utility scores for all COPD health states. The relationship between patients’ COPD health utility scores, self-perceived COPD severity, and EQ-5D-3L utility scores was analyzed, with data stratified according to disease severity and comorbidity subgroups. Results The COPD health utility score had face validity, with utility scores negatively correlated with patients’ self-perceived COPD severity. The correlation between the COPD health utility scores and EQ-5D-3L values was only moderate. While patient EQ-5D-3L scores were impacted by comorbidities, the COPD health utility score was less impacted by comorbid conditions. Conclusions Our COPD utility measure, derived from a DCE, provides a patient-centered health utility score and is more sensitive to the COPD health of the individual and less sensitive to other comorbidities. This disease-specific instrument should be considered alongside generic health-related quality of life instruments when valuing new COPD therapies in submissions to licensing and reimbursement agencies.

Development of a disease-specific health utility score for chronic obstructive pulmonary disease from a discrete choice experiment patient preference study.

Introduction This patient preference study sought to quantify the preferences of people living with COPD regarding symptom improvement in the UK, USA, France, Australia and Japan. Methods The inclusion criteria were people living with COPD aged 40 years or older who experienced ≥1 exacerbation in the previous year with daily symptoms of cough and excess mucus production. The study design included: 1) development of an attributes and levels grid through qualitative patient interviews; and 2) implementation of the main online quantitative survey, which included a discrete choice experiment (DCE) to allow assessment of attributes and levels using hypothetical health state profiles. Preference weights (utilities) were derived from the DCE using hierarchical Bayesian analysis. A preference simulator was developed that enabled different health state scenarios to be evaluated based on the predicted patient preferences. Results 1050 people living with moderate-to-severe COPD completed the survey. All attributes were considered important when patients determined their preferences in the DCE. In a health state preference simulation, two hypothetical health states (comprising attribute levels) with qualitatively equivalent improvements in A) cough and mucus and B) shortness of breath (SOB) resulted in a clear preference for cough and mucus improved profile. When comparing two profiles with C) daily symptoms improved and D) exacerbations improved, there was a clear preference for the daily symptoms improved profile. Conclusions People living with moderate-to-severe COPD prefer to reduce cough and mucus production together over improvement of SOB and would prefer to reduce combined daily symptoms over an improvement in exacerbations. There is a need for symptom-focused therapies to deliver meaningful outcomes for people living with COPD https://bit.ly/3KqcSur

/pdf/people-living-with-moderate-to-severe-copd-prefer-2nisxkx0.pdf

People living with moderate-to-severe COPD prefer improvement of daily symptoms over the improvement of exacerbations: a multicountry patient preference study

Ann McDonald and Margaret Taylor, Older People and the Law , Policy Press, Bristol, Avon, 2006, 184 pp., hbk £55.00, ISBN 978 1 86134 715 2, pbk £16.99, ISBN 13: 978 1 86134 714 5.

Impact of baseline lung function on future exacerbations in patients with moderate-to-severe asthma

Popular Tasks used by Researchers

How can I help with your research?

Get more enhanced response by uploading the PDFs you want me to reference.

No relevant PDFs in your library

SciSpace is the AI research assistant for academics. Run systematic literature reviews on 280M+ papers, and write papers with cited sources. Trusted by 1M+ students, PhDs & researchers.

SciSpace | AI for Research

We encountered an error while loading the panel. Please try refreshing the page.

Something went wrong

Analyze PDFs

Code & Manuscripts

Funding & Grants

Literature & Patents

Medical & Clinical Data

Systematic Review

Visualize & Present

Web & Data

Build a Google Scholar-like website for your research.

Build a website

Create charts and images for your research

Create a Chart

Write a paper for submission to a journal

Draft a manuscript

Patent Search

Design eye-catching scientific posters in minutes.

Scientific Poster Generation

Systematic Literature Review

Anyone with the link can view this chat. You can switch it back to private anytime.

One task is running at the moment. Your messages will be shown right after.

Drag and drop or click here to browse

Loved by <highlight>1 million+</highlight> researchers

Extract a list of specific topics and their sources from unstructured text

Topics

Compare and analyze relevant papers that matches with your search

Papers

Get insights from PDFs and bookmarked papers from your library

My library

Recent searches

Try searching for:

Catch AI-generated content in scholarly and non-scholarly content

{ai} Detector

Ai Writer

Get PDF Summaries, highlighted text explanations 

Chat with PDF

Effortlessly create in-text citations and bibliographies in APA and 2,500 other formats

Citation generator

Get explanations, summaries, and answers on academic papers

Ease up your research workflow with {scispace}'s cohort of exciting AI tools

Elevate your academic writing skills and convey your ideas the way you want

Paraphraser

Explore our range of reading and writing tools

Your file is being prepared and should be ready in a few minutes. If it's a large file, it might take a bit longer. You can close this window, and we'll email you the file when it's done.

You have reached a maximum limit of <strong>{limit}</strong> columns in the table. Remove at least <strong>1</strong> column to add or create another one.

Byron Jones

Author Tools

Chat about Author

Papers

People living with moderate-to-severe COPD prefer improvement of daily symptoms over the improvement of exacerbations: a multicountry patient preference study

Ann McDonald and Margaret Taylor, Older People and the Law , Policy Press, Bristol, Avon, 2006, 184 pp., hbk £55.00, ISBN 978 1 86134 715 2, pbk £16.99, ISBN 13: 978 1 86134 714 5.

Impact of baseline lung function on future exacerbations in patients with moderate-to-severe asthma