Recognizing the need for personalization of haemophilia patient‐reported outcomes in the prophylaxis era

Abstract

The safety and efficacy of treatment options for patients with haemophilia have significantly improved over the last two decades, particularly with greater utilization of prophylactic approaches. Consequently, it is becoming increasingly difficult to differentiate the treatment benefits of available choices based on standard endpoints such as annualized bleeding rates and joint health scores. Patient-reported outcomes (PROs) have shown limited ability to discriminate between treatment outcomes, in part because of their comprehensive nature; i.e. differences in specific outcomes meaningful to individual patients are masked by a global scoring system based on a fixed set of items, many of which may be unimportant for any given patient. There is a clear need for new outcome measures. Initiatives to develop patient-centric outcomes that capture clinically meaningful change are ongoing. One such approach, goal attainment scaling (GAS), allows patients, in collaboration with a trained clinician, to select goals from a medical condition-specific menu of options and subsequently facilitates quantitative assessment of goal realization. Thus, it is fully personalized and sensitive to small, often idiosyncratic, treatment benefits, such as improvements in functional capacity. In this paper, we present the underlying rationale for GAS and one other novel approach to PRO personalization, and discuss their potential to augment current outcome measures by reliably detecting and quantifying treatment effects in individuals with haemophilia on prophylaxis.

The behaviour and perception of illness: modulating variables of adherence in patients with haemophilia

“… and Recht et al . stress the importance of listening to the views of both the health professionals and the patient, it is essential to understand the role of prophylaxis and haemophilia in the life of the individual .…”

“…However, we have not yet established other fundamental aspects that cause whether the patient follows treatment. As shown in our work and in other studies , the patients’ perception of their illness is essential in knowing how they will cope with the disease, including treatment.…”

Goal Attainment Scaling for haemophilia (GAS‐Hēm): testing the feasibility of a new patient‐centric outcome measure in people with haemophilia

“…Consequently, traditional clinical outcome measures, such as annualized bleeding rate (ABR), and quality of life (QoL) measures (eg the Short Form (36) health survey [SF‐36]), are less able to discriminate clinically and personally meaningful change in PwH . There is growing need for a personalized patient‐reported outcome (PRO) measuring such change in clinical settings . Therefore, an approach based on Goal Attainment Scaling (GAS) was implemented to develop a goals‐based, personalized outcome measure for haemophilia: the GAS‐Hēm .…”

“…Therefore, an approach based on Goal Attainment Scaling (GAS) was implemented to develop a goals‐based, personalized outcome measure for haemophilia: the GAS‐Hēm . The key to this method is setting measurable, meaningful goals relating directly or indirectly to the challenges of having haemophilia and assessing goal attainment over specified intervals …”

Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes

“…Since the scope for HTA and coverage decision is ultimately comparing the return for investment among different treatments and choosing those treatments with the highest return (opportunity cost), 72 standardized generic measures (eg, SF-36, EQ-5D) have to be collected 77,78. There is urgent need for identifying and adopting outcome measures appropriate to assess gene therapy in terms of value propositions 83. In the more common situation of innovation coming at an incremental cost, a final layer of assessment is needed.…”