Incorporating the patient voice and patient engagement in GOAL‐Hēm: Advancing patient‐centric hemophilia care

Roberts, Jonathan C.; Recht, Michael; Gonzales, Sarah; Stanley, Justin; Denne, Michael; Caicedo, Jorge; Rockwood, Kenneth

doi:10.1002/rth2.12655

Cited by 5 publications

(17 citation statements)

References 14 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The final goal menu, which includes these patient‐centred outcomes, is customized by age and has patient‐centred language derived from qualitative feedback from key stakeholders in several settings. The final goals are in three domains: haemophilia management, haemophilia complications, and impact on life 9 . PwH endorsed GOAL‐Hēm for the following reasons: level of individualization, patient‐centred language, and empowerment through the goal‐setting process 4,9 .…”

Section: Discussionmentioning

confidence: 99%

“…Participants reviewed GOAL‐Hēm content and implementation methods via three separate methods: online survey, individual interviews, or focus group. While the feasibility of the GOAL‐Hēm menu was previously demonstrated, the Patient Voice Study aimed to evaluate how comprehensible the language used to describe the goals and their descriptors was to PwH and their caregivers, and the relevance and usefulness of each goal; it also obtained direct feedback on how to revise the GOAL‐Hēm menu 9 …”

Section: Methodsmentioning

confidence: 99%

“…The focus group was 3 h long and involved PwH from the Bleeding and Clotting Disorders Institute, all of whom were involved in the feasibility study. The format of the focus group was semistructured and included three topics: (1) current tools for PwH and their caregivers in everyday life (i.e., haemophilia care and management); (2) the best presentation of GOAL‐Hēm to PwH and their caregivers, including most important features; and (3) the best use of the tool in routine clinic visits 9 …”

Section: Methodsmentioning

confidence: 99%

“…hensible the language used to describe the goals and their descriptors was to PwH and their caregivers, and the relevance and usefulness of each goal; it also obtained direct feedback on how to revise the GOAL-H ēm menu. 9 The online survey reviewed titles of goal areas and descriptors with regards to language and practical relevance, and the number of participants rating each item as 'clear' and 'relevant' was captured.…”

Section: Feasibility Study: Quantitative Interviews At Haemophilia Tr...mentioning

confidence: 99%

“… GOAL‐Hēm is sensitive to small but important changes, 4 and requires significant input from PwH and healthcare providers, in both development and implementation. Methodological considerations from the Patient Voice Study revealed that individual interviews and focus groups provided more robust feedback (e.g., quantity, personal comments, details) than online surveys 9 The experiences of PwH during the research and development of GOAL‐Hēm generated enthusiasm for the tool; they reported having more control over disease management by focusing on what is most important to them.…”

Section: Lessons Learnedmentioning

confidence: 99%

See 4 more Smart Citations

The GOAL‐Hēm journey: Shared decision making and patient‐centred outcomes

et al. 2022

Self Cite

View full text Add to dashboard Cite

Introduction: GOAL-H ēm is a novel, haemophilia-specific, patient-centred outcome measure (PCOM) based on goal attainment scaling, allowing people with haemophilia (PwH) to set and monitor the attainment of individualized goals for treatment. Aim:To provide a thorough overview of the creation, validation, and development of GOAL-H ēm.Methods: Clinician workshops were held to develop a haemophilia-specific goal menu.Qualitative data from semistructured interviews with PwH and their caregivers guided further revisions to the goal menu (i.e., goal domains and descriptors). A feasibility study was performed including a 12-week, prospective, noninterventional evaluation involving clinicians and PwH at four US haemophilia treatment centres. Finally, the Patient Voice Study gathered feedback from PwH and their caregivers via an online survey, interviews, and a focus group. Results:The feasibility study validated GOAL-H ēm with successful outcomes in construct/content validity and responsiveness, including a large effect in patient-and clinician-rated goal attainments. The Patient Voice Study led to significant refinement of GOAL-H ēm goals and descriptors, resulting in a more straightforward and relatable menu for PwH and their caregivers. Overall, GOAL-H ēm captured qualitative data in areas important to PwH and employed quantitative methods to evaluate meaningful changes in those areas. The individualized tool was well equipped to handle the complex and chronic nature of haemophilia and was endorsed by PwH, their caregivers, and clinicians. Conclusion:The GOAL-H ēm development journey may serve as a roadmap for other PCOMs in a variety of settings, including clinical studies, haemophilia treatment centres for care planning, and as a tool to gather real-world evidence.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Methodsmentioning

confidence: 99%

Section: Feasibility Study: Quantitative Interviews At Haemophilia Tr...mentioning

confidence: 99%

Section: Lessons Learnedmentioning

confidence: 99%

See 3 more Smart Citations

The GOAL‐Hēm journey: Shared decision making and patient‐centred outcomes

et al. 2022

Self Cite

View full text Add to dashboard Cite

show abstract

Embedding Patient-Centricity by Collaborating with Patients to Transform the Rare Disease Ecosystem

Sharma

Ahmed²,

Campagnari

et al. 2023

Pharm Med

View full text Add to dashboard Cite

What is patient-centricity? In some contexts, it has been associated with targeting therapies based on biomarkers or enabling healthcare access. There has been a surge in patient-centricity publications, and in many cases for the biopharmaceutical industry, patient engagement is used to endorse pre-held assumptions at a specific moment in time. Rarely is patient engagement used to drive business decisions. Here we describe an innovative partnership between Alexion, AstraZeneca Rare Disease and patients that allowed a deeper understanding of the biopharmaceutical stakeholder ecosystem and an empathic understanding of each patient’s and caregiver’s lived experience. Alexion’s decision to build patient-centricity frameworks resulted in the formation of two unique organisation design platforms: STAR ( S olutions T o A ccelerate R esults for patients) and LEAP ( L earn, E volve, A ctivate and deliver for P atients) Immersive Simulations. These interconnected programmes required cultural, global, and organisational shifts. STAR generates global patient insights that are embedded in drug candidate and product strategies while helping to establish enterprise foundational alignment and external stakeholder engagement plans. LEAP Immersive Simulations produce detailed country-level patient and stakeholder insights that contribute to an empathetic understanding of each patient’s lived experience, support country medicine launches and provide ideas to have a positive impact along the patient journey. Combined, they deliver integrated, cross-functional insights, patient-centric decision making, an aligned patient journey, and 360° stakeholder activation. Throughout these processes, the patient is empowered to dictate their needs and validate the proposed solutions. This is not a patient engagement survey. This is a partnership where the patient co-authors strategies and solutions.

show abstract

Development of a haemophilia A gene therapy shared decision‐making tool for clinicians

Limjoco

Thornburg

2023

Haemophilia

View full text Add to dashboard Cite

IntroductionAs gene therapies are incorporated into clinical practice, shared decision‐making (SDM) is recommended for implementation.AimTo inform development of a clinician SDM tool for haemophilia A gene therapy.MethodsClinicians at US Hemophilia Treatment Centers completed semi‐structured interviews about their experience with SDM and provided feedback on a clinician SDM tool prototype. Interviews were transcribed verbatim for coding and thematic content analysis.ResultsTen participants enrolled, eight physicians and two haemophilia nurses. All participants care for adults with haemophilia (1‐27 years of experience) and 7 have gene therapy trials open at their institution. Confidence in having a clinical discussion about gene therapy included none (N = 1), slight (N = 3), moderate (N = 5) and high (N = 1). All participants reported familiarity with SDM and agreed that the tool would be useful for their clinical practice. Key themes in participant feedback for the tool were (1) language and presentation; (2) content; and (3) implementation. Participants highlighted the importance of providing unbiased information and having companion tools with patient‐centric language.ConclusionThese data highlight the need for SDM tools for haemophilia A gene therapy. Key information to include in the tool are safety, efficacy, cost and detailed information on the gene therapy process. Data should be provided in an unbiased format and allow comparison to other treatments. The tool will be evaluated in clinical practice and refined as clinical trial data and real‐world experience mature.

show abstract

Incorporating the patient voice and patient engagement in GOAL‐Hēm: Advancing patient‐centric hemophilia care

Cited by 5 publications

References 14 publications

The GOAL‐Hēm journey: Shared decision making and patient‐centred outcomes

The GOAL‐Hēm journey: Shared decision making and patient‐centred outcomes

Embedding Patient-Centricity by Collaborating with Patients to Transform the Rare Disease Ecosystem

Development of a haemophilia A gene therapy shared decision‐making tool for clinicians

Contact Info

Product

Resources

About