The role of patient and healthcare professionals in the era of new hemophilia treatments in developed and developing countries

Nossair, Fadi; Thornburg, Courtney D.

doi:10.1177/2040620718784830

Cited by 30 publications

(44 citation statements)

References 52 publications

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“…Given the gaps in the evidence base, it will be important to embrace a shared decision‐making approach . Physicians should give patients a clear understanding of the benefits and risks of GT based on the best available evidence at the time to enable a collaborative decision on the best treatment choice based on an individual's clinical history, preferences and treatment goals . As discussed below, patient organisations are also likely to be an important source of information.…”

Section: How To Discuss Gene Therapy: Physician and Patient Perspectimentioning

confidence: 99%

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How to discuss gene therapy for haemophilia? A patient and physician perspective

et al. 2019

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Gene therapy has the potential to revolutionise treatment for patients with haemophilia and is close to entering clinical practice. While factor concentrates have improved outcomes, individuals still face a lifetime of injections, pain, progressive joint damage, the potential for inhibitor development and impaired quality of life. Recently published studies in adeno‐associated viral (AAV) vector‐mediated gene therapy have demonstrated improvement in endogenous factor levels over sustained periods, significant reduction in annualised bleed rates, lower exogenous factor usage and thus far a positive safety profile. In making the shared decision to proceed with gene therapy for haemophilia, physicians should make it clear that research is ongoing and that there are remaining evidence gaps, such as long‐term safety profiles and duration of treatment effect. The eligibility criteria for gene therapy trials mean that key patient groups may be excluded, eg children/adolescents, those with liver or kidney dysfunction and those with a prior history of factor inhibitors or pre‐existing neutralising AAV antibodies. Gene therapy offers a life‐changing opportunity for patients to reduce their bleeding risk while also reducing or abrogating the need for exogenous factor administration. Given the expanding evidence base, both physicians and patients will need sources of clear and reliable information to be able to discuss and judge the risks and benefits of treatment.

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Section: How To Discuss Gene Therapy: Physician and Patient Perspectimentioning

confidence: 99%

“…Given the gaps in the evidence base, it will be important to embrace a shared decision-making approach. 49 Physicians should…”

Section: Sources Of Information On Gene Therapymentioning

confidence: 99%

How to discuss gene therapy for haemophilia? A patient and physician perspective

et al. 2019

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“…[37][38][39] Concepts from Kahneman and Thaler may be useful to better understand the factors that influence clinical decision-making, and these could be used to further improve haemophilia decision-making models. [1][2][3][4][5][6] The results of the study may also be of interest from a policy perspective in understanding the influences and barriers that physicians experience when making treatment decisions for the management of haemophilia and may be applicable to other disease states with high treatment costs. Findings may also be a relevant in physician self-assessment of decision-making styles and biases and to offer insights into how chronic care can be improved.…”

Section: Discussionmentioning

confidence: 99%

“…Recently published haemophilia decision‐making models of care promote ‘shared’, ‘deliberative’ or ‘tailored’ techniques to optimize treatment . These models reflect a shift from a paternalistic model of medicine to a process whereby healthcare providers and their patients make treatment decisions jointly.…”

Section: Introductionmentioning

confidence: 99%

UK vs US physician decision‐making in the treatment of haemophilia

2019

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IntroductionPatient–physician shared decision‐making (SDM) has become increasingly seen as having a positive effect on management of chronic diseases. However, little is known of the factors that encourage SDM or how effective it may be at improving health outcomes or how cost‐effective it is.AimTo investigate the uses and applications of patient physician–SDM in the management of haemophilia and the influence of healthcare systems in the United States and the United Kingdom.MethodsThis was a qualitative study based on interviews with treatment experts in the United States and United Kingdom. A grounded theory approach was used to analyse the data from the transcribed interviews and themes that emerged as related to the decision influencers. Twelve physicians from each country were interviewed by the author.ResultsTreatment guidelines were viewed as having only limited applicability because of the lack of universal best options in haemophilia. The US physicians in the sample appeared to be more influenced by patient preferences than physicians in the UK, who instead tended to follow policies and standards of care more closely. Physicians in both countries commented that many of their patents had become highly knowledgeable of their bleeding disorder. US physicians were sometimes limited by insurance company policies but also reported that they were often successful in appealing insurance decisions.ConclusionThe research suggests that there are different influences on decision‐making between healthcare systems; patients and overarching healthcare systems play a major role in how physicians treat haemophilia.

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“…The need for additional research and the lack of an integrated SDM theory are an identified gap in the literature and if developed will contribute to enhancing the use and impact of SDM in medical decision making . Recently proposed SDM models could benefit from an understanding of these factors …”

Section: Introductionmentioning

confidence: 99%

Shared decision making: Does a physician's decision‐making style affect patient participation in treatment choices for primary immunodeficiency?

Lamb

Wang

Lyytinen

2019

Evaluation Clinical Practice

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Overall health care spending in the United States is equivalent to more than 15% of GDP, yet outcomes rank below the top 25 in most quality categories when compared with other Organization for Economic Cooperation and Development (OECD) countries. The majority of spending is consumed by small patient populations with chronic diseases. Experts believe increased patient‐physician shared decision making (SDM) should result in better overall longitudinal care but understanding the physician's role in facilitating SDM is limited. Structural equation modelling was applied to results of a 2016 questionnaire‐based survey of 330 US physicians who treat approximately 55% of primary immune deficiency requiring immune globulin therapy; it tested the relationship between slow/rational vs fast/intuitive decision‐making styles and SDM as mediated by patient‐centric care and moderated by physician's trust in the patient. The results showed a statistically significant relationship between slow/rational decision making and SDM. The results also suggest differences related to age, gender, education, and race but no differences related to trust.

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The role of patient and healthcare professionals in the era of new hemophilia treatments in developed and developing countries

Cited by 30 publications

References 52 publications

How to discuss gene therapy for haemophilia? A patient and physician perspective

How to discuss gene therapy for haemophilia? A patient and physician perspective

UK vs US physician decision‐making in the treatment of haemophilia

Shared decision making: Does a physician's decision‐making style affect patient participation in treatment choices for primary immunodeficiency?

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