Patient‐centred value framework for haemophilia

O’Mahony, Brian; Dolan, G.; Nugent, Diane J.; Goodman, Clifford

doi:10.1111/hae.13456

Cited by 21 publications

(39 citation statements)

References 25 publications

(63 reference statements)

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“…4,16 Presence of target joints has been associated with fatigue, loss of enjoyment, reduced leisure pursuits and particularly pain, with more than eight times as many patients reporting extreme pain or discomfort in the presence of a target joint. 18,19 Therefore, to properly evaluate the impact of joint impairment on patients with haemophilia, it is important to consider such qualitative outcomes that result directly from joint impairment, 20 for example, pain-an insidious outcome reported by the majority of PwH in the US and EU that impacts daily living by reducing range of motion, impairing mobility and limiting function and productivity. 18,19 Therefore, to properly evaluate the impact of joint impairment on patients with haemophilia, it is important to consider such qualitative outcomes that result directly from joint impairment, 20 for example, pain-an insidious outcome reported by the majority of PwH in the US and EU that impacts daily living by reducing range of motion, impairing mobility and limiting function and productivity.…”

Section: Milestone 2 Minimal Joint Impairment/ Improved Quality Ofmentioning

confidence: 99%

“…17 In the value equation, defined by Porter as outcomes relative to costs, outcomes are multidimensional and condition-specific and, for haemophilia, including objective measures of survival, ABR and joint impairment, as well as qualitative measures such as quality of life and participation in activities of daily living. 18,19 Therefore, to properly evaluate the impact of joint impairment on patients with haemophilia, it is important to consider such qualitative outcomes that result directly from joint impairment, 20 for example, pain-an insidious outcome reported by the majority of PwH in the US and EU that impacts daily living by reducing range of motion, impairing mobility and limiting function and productivity. 21 In the CHESS study, patients with target joints had worse pain/discomfort, in addition to accompanying lower EQ-5D measures of mobility, selfcare, usual activities and anxiety/depression.…”

Section: Milestone 2 Minimal Joint Impairment/ Improved Quality Ofmentioning

confidence: 99%

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Achieving the unimaginable: Health equity in haemophilia

et al. 2019

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Haemophilia. 2020;26:17-24.wileyonlinelibrary.com/journal/hae | 17 | INTRODUC TI ONCurrent prophylaxis protocols for haemophilia A are designed conservatively, with a minimal goal of achieving factor VIII (FVIII) trough levels of ≥1%. This threshold was originally selected based on generally favourable outcomes from early cryoprecipitate studies that targeted trough FVIII levels of 1%-3%, supportive evidence that moderate haemophilia was associated with fewer bleeds and better preserved joint function than severe haemophilia, and limitations in plasma resources at the time. 1-3 However, persons with haemophilia (PwH) who are maintained at 1% FVIII continue to experience spontaneous bleeding and remain at higher risk for injury with daily activities. 4 Furthermore, current prophylaxis regimens do not prevent haemophilic arthropathy over a PwH's lifetime. 5 With the growing availability of current therapies, the validity of the 1% threshold has come under scrutiny, accompanied by the proposal to target higher thresholds ≥15%. 6 Recent success in clinical development of haemophilia therapies opens an era of new possibilities and holds great promise for people Abstract Historically, treatment based on the availability of clotting factor replacement has resulted in an arcane guideline for the correction of factor deficiencies in people with haemophilia (PwH). While all other disease entities seek to restore function to a normal level, PwH are restricted to factor nadirs still equivalent to mild or moderate disease, resulting in continued risk of bleeding. A new treatment paradigm is needed based on the defined needs of PwH. A treatment model was developed by a panel of haemophilia providers, patient advocates and health economists to establish specific treatment milestones and targeted outcomes. The panel defined a series of treatment milestones to characterize the activity and outcomes linked to level of factor deficiency correction. All agreed that the ultimate goal should be 'functional cure' and 'health equity'. Seven levels to achieving a functional cure were identified, (a) Sustain life; (b) Minimal joint impairment; (c) Freedom from any spontaneous bleeds;(d) Attainment of 'normal' mobility; (e) Able to sustain minor trauma without additional intervention; (f) Ability to sustain major surgery or trauma; and (g) Normal haemostasis. A parallel set of patient-reported outcomes to achieve health equity was identified. These guidelines are now comparable with other disorders where the goal is to replace missing proteins to attain normal activity levels. As we are no longer limited by plasma supply due to the manufacture of recombinant factors, mimetics, and the early success of gene therapy, health equity is now achievable. K E Y W O R D Shaemophilia, outcomes, prophylaxis, replacement factor How to cite this article: Skinner MW, Nugent D, Wilton P, et al.Achieving the unimaginable: Health equity in haemophilia.

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Section: Milestone 2 Minimal Joint Impairment/ Improved Quality Ofmentioning

confidence: 99%

Section: Milestone 2 Minimal Joint Impairment/ Improved Quality Ofmentioning

confidence: 99%

Achieving the unimaginable: Health equity in haemophilia

et al. 2019

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“…Moving to the specific hemophilia field, O'Mahony and colleagues developed a patient‐centered framework with global applicability for assessing value in hemophilia care based on Porter's model for assessing value . They adapted that framework to hemophilia in the three tiers, Tier 1 addresses health status achieved or retained, including bleeding frequency, musculoskeletal complications and life‐threatening hemorrhage.…”

Section: The Evolution Of Outcome Measurement Theory Patient‐reportementioning

confidence: 99%

“…49 Moving to the specific hemophilia field, O'Mahony and colleagues developed a patient-centered framework with global applicability for assessing value in hemophilia care based on Porter's model for assessing value. 74 There is currently no standard approach to PIO and PROs in hemophilia trials. 53 Hemophilia-specific health quality of life tools have been developed and are used, although not in a uniform way across studies.…”

Section: The Evolution Of Outcome Measurement Theory Patient-repormentioning

confidence: 99%

Hemophilia trials in the twenty‐first century: Defining patient important outcomes

Konkle

Skinner

Iorio

2019

Research and Practice in Thrombosis and Haemostasis

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Treatment for hemophilia has advanced dramatically over the past 5 decades. Success of prophylactic therapy in preventing bleeding and decreasing associated complications has established a new standard of care. However, with the advent of gene therapy and treatments that effectively mimic sustained coagulation factor replacement, outcome measures that worked well for assessing factor replacement therapies in past clinical trials need to be reassessed. In addition, while therapies have advanced, so has the science of outcome assessment, including recognition of the importance of patient important and patient reported outcomes. This manuscript reviews strengths and limitations of outcome measures used in hemophilia from both a provider and patient perspective.

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“…A core set of combined clinical and patient‐reported outcomes does not yet exist for haemophilia. Brian O'Mahony, Gerard Dolan and colleagues set off to map value in haemophilia onto the three‐tiered framework of outcomes. They defined haemophilia outcomes in each tier and subsequently applied the framework to three clinical scenarios (eg the impact of receiving care at a haemophilia treatment centre versus not receiving care at a specialized centre; the superiority of prophylaxis over on‐demand therapy; and the utilization of extended half‐life products versus standard therapy).…”

mentioning

confidence: 99%