As investigational hemophilia gene therapies move closer to regulatory authorization, community stakeholders have recognized the acute need for people with hemophilia (PWH) and healthcare professionals (HCPs) to be fully engaged in shared decision making (SDM). While the concept of SDM whereby patient and provider collaborate to reach informed treatment decisions has gained support in recent years, the arrival of such a paradigm-shifting will present unique challenges and opportunities.

Disparities in health literacy, the proliferation of inaccurate and contradictory content on social media, direct-to-patient marketing, plus the sheer complexity of gene therapy, may prevent a PWH from fully engaging in SDM. While some HCPs may also lack a thorough enough understanding of gene therapy, hindering their full participation in the SDM model. In addition, an HCP’s perception of their patient’s comprehension of this therapy may not align with their patient’s actual understanding, a discordance that further compromises SDM, increasing the potential for delayed treatment decisions and other negative outcomes.

In light of these anticipated obstacles, an international and multidisciplinary group known of as the Council of the Hemophilia Community (CHC) was convened. Composed of independent advisors, HCPs, industry and patient representatives, the goal of the CHC was to fill these information gaps through the development of a resource that would help generate an ongoing dialogue between PWH/HCP, with patient-centricity as it’s guiding principle.

The CHC held three roundtable meetings between November 2020 and May 2021 wherein they fleshed out a series of questions and answers that would best foster a genuine SDM process amongst PWHs/HCPs. The majority of the decided-upon questions fell under several over-arching categories including treatment regimen/adherence requirements, treatment predictability and variability, treatment durability, and the risk/benefit profile.

Each of the questions were subsequently assigned to the five stages of the patient “decision making journey.” These included 1) Pre-gene therapy information seeking 2) Pre-gene therapy decision making 3) Treat initiation 4) Short-term post-gene therapy follow up (less than one year since receiving gene therapy)  5) Long-term gene therapy follow up (more than one year after receiving gene therapy).

A recent paper published online in the journal Patient Preference and Adherence (PPA), describes in greater detail the process of developing the resource and context in which it was created. Read the open-access PPA article.

The authors highlight the value of this tool to enhance SDM relevant to hemophilia gene therapy, while also hinting at its potential utility in other disease groups.

“The educational and decision support resources described herein recognize that each patient’s decision journey will evolve throughout their lifetime with their individual preferences at different life stages, and with the emergence of new therapies and a growing evidence base,” explain the authors. “The Q&A resource provides HCPs and PWH with timely, relevant information, facilitates discussions, and empowers PWH to engage in shared decision-making. As gene therapy products enter the market, the themes and questions mapped here should stimulate discussion and aid interactions among HCPs, PWH, and family members, to ensure that they are fully informed and realize the clinical potential of this treatment. While the issues discussed here pertain to hemophilia, they could also be applied to other hereditary diseases with multiple treatment options.”

Access “Hemophilia Gene Therapy: Your Questions Answered” in PDF.

Wang M, Negrier C, Driessler F, Goodman C, Skinner MW. The Hemophilia Gene Therapy Patient Journey: Questions and Answers for Shared Decision-Making. Patient Prefer Adherence. 2022 Jun 9;16:1439-1447. doi: 10.2147/PPA.S355627. PMID: 35707346; PMCID: PMC9191577.

Disclaimer: NHF provides periodic synopses of articles published in peer reviewed journals, the purpose of which is to highlight papers that cover a wide range of topics and speak to a broad spectrum of the inherited blood disorders community. Topics include shared decision making, gene therapy, health equity, and more. NHF hopes you find this content to be informative and engaging.

Any questions about the articles featured here should be directed to the publishing journal and/or the study authors. This content is for general information only. NHF does not give medical advice or engage in the practice of medicine. NHF under no circumstances recommends particular treatment for specific individuals and in all cases recommends that you consult your physician or local treatment center before pursuing any course of treatment.