Dataset Information

Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.

ABSTRACT:

Introduction

Exploring patient perceptions regarding gene therapies may provide insights about their acceptability to patients.

Objective

To investigate opinions of people with haemophilia (PWH) regarding gene therapies. Moreover, this study aimed to identify patient-relevant attributes (treatment features) that influence PWH's treatment choices.

Methods

Semi-structured individual interviews were conducted with Belgian PWH, types A and B. A predefined interview guide included information sections and open, attribute ranking and case questions. Qualitative data were organized using NVivo 12 and analysed following framework analysis. Sum totals of scores obtained in the ranking exercise were calculated per attribute.

Results

In total, 20 PWH participated in the interviews. Most participants demonstrated a positive attitude towards gene therapy and were very willing (40%; n = 8) or willing (35%; n = 7) to receive this treatment. The following five attributes were identified as most important to PWH in making their choice: annual bleeding rate, factor level, uncertainty of long-term risks, impact on daily life, and probability that prophylaxis can be stopped. While participants were concerned about the uncertainty regarding long-term safety, most participants were less concerned about uncertainty regarding long-term efficacy.

Conclusions

This qualitative study showed that most PWH have a positive attitude towards gene therapy and that besides efficacy, safety and the related uncertainties, also impact on daily life is important to patients. The identified patient-relevant attributes may be used by regulators, health technology assessment bodies and payers in their evaluation of gene therapies for haemophilia. Moreover, they may inform clinical trial design, pay-for-performance schemes and real-world evidence studies.

SUBMITTER: van Overbeeke E

PROVIDER: S-EPMC7894464 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature

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Publications

Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.

van Overbeeke Eline E Michelsen Sissel S Hauber Brett B Peerlinck Kathelijne K Hermans Cedric C Lambert Catherine C Goldman Michel M Simoens Steven S Huys Isabelle I

Haemophilia : the official journal of the World Federation of Hemophilia 20201107 1

<h4>Introduction</h4>Exploring patient perceptions regarding gene therapies may provide insights about their acceptability to patients.<h4>Objective</h4>To investigate opinions of people with haemophilia (PWH) regarding gene therapies. Moreover, this study aimed to identify patient-relevant attributes (treatment features) that influence PWH's treatment choices.<h4>Methods</h4>Semi-structured individual interviews were conducted with Belgian PWH, types A and B. A predefined interview guide includ ...[more]

PMID: 33161616

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Project description:Background and objectiveNew treatments for haemophilia are under development or entering the market, including extended half-life products, designer drugs and gene therapy, thereby increasing treatment options for haemophilia. It is currently unknown how people with haemophilia decide whether to switch to a new treatment. Therefore, the objective of this study was to explore what factors may play a role when Dutch patients and parents of boys with moderate or severe haemophilia make decisions about whether to switch to a different treatment, and how disease and treatment characteristics may affect these decisions. This may aid clinical teams in tailored information provision and shared decision making.MethodsWe conducted interviews among adults with moderately severe or severe haemophilia and parents of young boys with severe haemophilia. We aimed to include participants from a variety of backgrounds in terms of involvement in the haemophilia community, age, treatment centre and treatments. Participants were recruited through the Netherlands Haemophilia Society and a haemophilia treatment centre. Semi-structured interviews were recorded and transcribed verbatim. Thematic content analysis was used to analyse the data.ResultsTwelve people with haemophilia and two mothers of boys with haemophilia were included. In general, participants reported to be satisfied with their current treatment. However, they considered ease of use of the medication (fewer injections, easier handling, alternative administration) an added value of new treatments. Participants were aware of the high cost of coagulation factor products and some expressed their concern about the Netherlands Haemophilia Society's long-term willingness to pay for current and novel treatments, especially for increased usage due to high-risk activities. Participants also expressed their concerns about the short- and long-term safety of new treatments and believed the effects of gene therapy were not yet fully understood. Participants expected their treatment team to inform them when a particular new treatment would be suitable for them.ConclusionsWith the number of treatment options set to increase, it is important for healthcare providers to be aware of how patient experiences shape patients' decisions about new therapies.

Project description:BackgroundUK government guidelines and initiatives emphasise equity in delivery of care, shared decision-making, and patient-centred care. This includes sharing information with patients as partners in health decisions and empowering them to manage their health effectively. In the UK, general practitioners (GPs) routinely receive hospital discharge letters; while patients receiving copies of such letters is seen as "good practice" and recommended, it is not standardised. The effects and consequences of whether or not this happens remains unclear. The aim of this study (one of three forming the Discharge Communication Study) was to explore patient perspectives on receiving discharge letters and their views on how this could be improved in order to optimise patient experience and outcomes.MethodsSemi-structured interviews were conducted with a diverse sample of 50 patients recruited from 17 GP surgeries within the West Midlands, UK. All participants were adults with a recent episode of general hospital inpatient or outpatient care. Data were audio recorded, transcribed and analysed using mixed methods corpus linguistics techniques.ResultsParticipants reported inconsistent access to discharge letters. Most wanted to receive a copy of their discharge letter although some expressed reservations. Perceived benefits included: increased understanding of their condition and treatment, reduced anxiety, and increased satisfaction. Consequences where participants had not received letters included: letter inaccuracies being overlooked, missed follow up actions, failure to fully remember diagnosis, treatment, or self-management or recommendations, and confusion and anxiety at what occurred and what will happen next. Participants felt the usefulness of receiving copies of letters could be increased by: including a patient information section, avoidance of acronyms, and jargon or technical terms explained with lay language.ConclusionsMost patients value receiving copies of hospital discharge letters, and should be consistently offered them. Patients' preferences for letter receipt could be logged in their health records. To enable positive outcomes letters should have a clear and accessible format that reflects the priorities and information needs of patients. Patients appear not to be receiving or being offered copies of letters consistently despite UK policies and guidelines supporting this practice; this suggests a need for greater standardisation of practice.

Dataset Information

Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.

Introduction

Objective

Methods

Results

Conclusions

Publications

Patient perspectives regarding gene therapy in haemophilia: Interviews from the PAVING study.

Similar Datasets

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