Identifying the Benefits and Risks of Emerging Treatments for Idiopathic Pulmonary Fibrosis: A Qualitative Study

Bridges, John F.P.; Paly, Victoria Federico; Barker, Elizabeth; Kervitsky, Dolly

doi:10.1007/s40271-014-0081-0

Cited by 22 publications

(19 citation statements)

References 29 publications

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“…They discussed issues of paucity of scientific data and the need to balance risks and benefits. Bridges et al [7] pick up this theme in their paper about patients with idiopathic pulmonary fibrosis. Analysis of interviews with patients, researchers and policy/industry informants showed that patients seemed to understand issues relating to emerging treatments.…”

Section: Attitudes To New Medicinesmentioning

confidence: 99%

The Imperative for Patient-Centred Research to Develop Better Quality Services in Rare Diseases

Facey¹,

Hansen

2015

Patient

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Section: Attitudes To New Medicinesmentioning

confidence: 99%

The Imperative for Patient-Centred Research to Develop Better Quality Services in Rare Diseases

Facey¹,

Hansen

2015

Patient

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“…Moreover, then the question arises of which patient organization to involve. A potential solution to address these issues might be, as suggested by some interviewees and several authors ( 10 , 17 , 27 , 31 , 33 , 34 , 36 , 38 – 42 ), the use of evidence from (quantitative) patient preference studies, by which patient preferences are being measured from a group of patients. To decide whether the input from disease-specific or umbrella patient organizations is needed, the authors propose to address this problem bottom-up, via looking at what type of patient information is needed per decision.…”

Section: Discussionmentioning

confidence: 99%

Patient Involvement in the Lifecycle of Medicines According to Belgian Stakeholders: The Gap Between Theory and Practice

et al. 2018

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Background: Patient involvement is often acknowledged as an important aspect of the lifecycle of medicines. Although different typologies exist, patient involvement has been described as the involvement of patients in decision-making regarding medicines. In view of the diversity of stakeholders and types of decisions in which patients might be involved, an in-depth understanding of these stakeholders' views toward involving patients in the lifecycle of medicines is essential.Methods: Interviews and surveys were used to gain insights into the perspectives and experiences of Belgian healthcare stakeholders. Interviews (n = 22) were conducted with academics, hospital pharmacists and representatives from health insurance funds, the Belgian reimbursement agency, pharmaceutical industry and patient organizations. Interviews underwent a framework analysis. Surveys (n = 108) were completed by hospital visitors and analyzed descriptively.Results: Despite an increasing amount of efforts to involve patients, interviewees labeled the level of actively involving patients as rather low and scattered across the different phases of the lifecycle of medicines. The main opportunities for patient involvement highlighted by interviewees were for: (i) informing early development decisions on which treatments to develop, (ii) clinical trial endpoint selection and (iii) clinical trial protocol design. However, remaining questions surrounding patient knowledge, and particularly how and which patients to involve represent important barriers toward implementing patient involvement in the lifecycle of medicines. Of survey participants, 77% indicated to be willing to participate in patient preference studies. Reasons for participating mentioned most frequently were “to improve development of treatments,” because “it is important to explore and listen to patient preferences” and “to have a voice as patients”.Conclusions: The barriers identified in this study hamper transitioning patient involvement from theory to practice. Bridging this gap requires addressing the identified barriers and unresolved questions surrounding the right methodology for involving patients, the “right patients” to involve and means to increase patient knowledge. In order to do so, further research should focus on assessing the value of methods that allow to indirectly capture patients' perspective both in the context of development as well as in the context of evaluation.

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“…As a consequence, potential side effects of these drugs need to be balanced with the treatment effect [ 33 , 34 ]. Evaluation of patient experiences can thus play a significant role when considering treatment approaches [ 35 , 36 ]. In The Netherlands, it is estimated that between 800 and 1600 people are diagnosed with IPF each year [ 37 ].…”

Section: Conceptual Model and Contextmentioning

confidence: 99%

Development and Pretesting of a Questionnaire to Assess Patient Experiences and Satisfaction with Medications (PESaM Questionnaire)

Kimman

Rotteveel

Wijsenbeek

et al. 2017

Patient

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BackgroundThe aim of this study was to develop, together with the Lung Foundation Netherlands and Dutch Kidney Patients Association, patients and clinicians, a measure to evaluate patient experiences with the orphan drugs pirfenidone (for idiopathic pulmonary fibrosis [IPF]) and eculizumab (for atypical haemolytic uraemic syndrome [aHUS]), as well as a generic measure of patient experiences and satisfaction with medications.MethodsDevelopment of the Patient Experiences and Satisfaction with Medications (PESaM) questionnaire consisted of four phases: literature review (phase I); focus groups and individual patient interviews (phase II); item generation (phase III); and face and content validity testing (phase IV). Literature review aimed to identify existing disease-specific and generic patient experience measures to provide guidance on the domains of medication use relevant to patients, the number of items and type of response categories, and to generate an initial pool of items. Subsequent focus groups and patient interviews were conducted to gain insight into the perceived effectiveness of the therapies, the burden of side effects, and how the medication impacted on a patient’s daily life. Focus groups and interviews were recorded and transcribed verbatim. Coding was carried out by highlighting passages in the text and assigning each passage a code representing the following predefined categories: (1) perceived effectiveness; (2) side effects; (3) ease of use; and (4) impact of medication. Using data from phase I and II, a panel of experts selected items relevant for inclusion in the questionnaire. Individual patient interviews with IPF and aHUS patients (n = 18), using a retrospective verbal probing technique, were conducted to assess face validity, time needed to fill out the questionnaire, and content validity.ResultsThe PESaM questionnaire that was developed consisted of two disease-specific modules that assessed patient experiences with pirfenidone for the treatment of IPF, and eculizumab for the treatment of aHUS, a generic module, applicable to any medication, and a module to assess patient expectations. Review of the literature identified multiple disease- or medication-specific questionnaires and two generic patient satisfaction questionnaires. Common domains across most questionnaires were effectiveness, side effects, ease of use and overall satisfaction. Patient interviews revealed the social impact (e.g. unable to go outside) of side effects such as photosensitivity associated with pirfenidone and the risk of infection associated with eculizumab. Each PESaM module focuses on patients’ perceived effectiveness of the medication, side effects, and ease of use, and the impact these aspects have on physical and emotional health and daily life. The generic module additionally includes items related to satisfaction with the medication. Individual interviews with patients in phase IV confirmed, in general, that questions and response options of the modules were clear and content validity was good. The mean ...

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Identifying the Benefits and Risks of Emerging Treatments for Idiopathic Pulmonary Fibrosis: A Qualitative Study

Cited by 22 publications

References 29 publications

The Imperative for Patient-Centred Research to Develop Better Quality Services in Rare Diseases

The Imperative for Patient-Centred Research to Develop Better Quality Services in Rare Diseases

Patient Involvement in the Lifecycle of Medicines According to Belgian Stakeholders: The Gap Between Theory and Practice

Development and Pretesting of a Questionnaire to Assess Patient Experiences and Satisfaction with Medications (PESaM Questionnaire)

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