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Gene Therapy Success in UK Likely Depends on Overcoming Education, Psychological Support Challenges


Education, psychological support, and implementation guidance are the top unmet needs identified by investigators from the United Kingdom concerning gene therapy use for hemophilia.

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Education, psychological support, and implementation guidance are needed for gene therapy.

To optimize gene therapy’s potential for use in hemophilia, investigators from the United Kingdom have identified top unmet needs in the space, and these are education, psychological support, and implementation guidance during the preinfusion, peri-infusion, and postinfusion phases of treatment for patients and health care professionals (HCPs) alike, according to their findings published in Orphanet Journal of Rare Diseases.1

Initial approvals for gene therapy’s use in hemophilia A came as recently as 2022 and early 2023 from the European Medicines Agency and the FDA, respectively—valoctocogene roxaparvovec (Roctavian; BioMarin Pharmaceutical)2,3—and in hemophilia B in 2022 and early 2023 from the European Commission and the UK Medicines and Healthcare products Regulatory Agency, respectively—etranacogene dezaparvovec (Hemgenix; CSL Behring).4,5

“The patient journey through gene therapy treatment pathways is new to many [HCPs],” the authors wrote. “Our conclusions are made with specific regard to gene therapy for haemophilia in the United Kingdom.”

The investigators highlight that education on gene therapy for hemophilia is predominantly provided in the process of a clinical trial or via verbal communication, and despite the European Haemophilia Consortium producing materials to help guide treatment decisions, patient understanding of how to even access these materials is lacking. Proposed solutions for patients include a centralized easily accessible education list and direction on how to access from patient advocacy groups.

For education for HCPs and patients’ multidisciplinary care team members, widespread knowledge of resources is limited overall, the investigators note, despite professional groups often sharing resources with members. The authors highlight the usefulness of the World Federation of Hemophilia Shared Decision Making Tool, contributions from the UK Haemophilia Centre Doctors’ Organisation and The Haemophilia Society for specialist education, a readily accessible resource inventory that covers a wide range of topics, and ensuring messaging on gene therapy use in hemophilia is consistent to prevent misinformation.

Further, patients need to be prepared for potential outcomes and challenges, they and their families will require support during immunosuppression, and the potential for changes to self-identity is great, the study authors emphasize. Therefore, cross-functional discussions should always include the health care team, patients, and patients’ families and cover whether gene therapy coincides with a patient’s treatment goals, alternative treatment options when available, and treatment preferences.

A primary goal of these discussions, which should begin in childhood or adolescence, is to avoid treatment remorse because gene therapy is irreversible, the study authors emphasize. This information should also address the concerns of all involved and manage their expectations. Clinicians may want to use the teach-back method, the authors added, to ensure patients understand the information they are receiving. It is also important to address treatment eligibility and to review the qualification criteria for gene therapy with patients when they are trying to make treatment decisions, so that their motivations, expectations, and commitments do not adversely affect their future ability to adhere to treatment follow-up, which could affect their safety.

There is also a need for equitable access to psychosocial professionals and hematologists experienced in caring for patients who have hemophilia. Fulfilling this need can ensure that patients and their families can properly process decisions, including the disappointment of being excluded from receiving gene therapy. A baseline psychological assessment, the authors add, could go a long way to help monitor patient reaction to the benefits and unwanted adverse effects of the gene therapy.

“Ultimately, the patient journey is unlikely to progress in a linear manner. The decision to undergo gene therapy should not be rushed,” the authors conclude. “The decision should be shared between people with haemophilia, and HCPs and should move at each individual patient’s pace, ensuring that they and their families feel informed and fully supported throughout the process.”


  1. Boyce S, Fletcher S, Jones A, et al. Educational needs of patients, families, and healthcare professionals to support the patient journey in haemophilia gene therapy in the UK. Orphanet J Rare Dis. 2023;18(1):366. doi:10.1186/s13023-023-02977-y
  2. First gene therapy to treat severe haemophilia A. News release. European Medicines Agency. June 24, 2022. Accessed November 27, 2023.https://www.ema.europa.eu/en/news/first-gene-therapy-treat-severe-haemophilia
  3. FDA approves first gene therapy for adults with severe hemophilia A. News release. FDA. June 29, 2023. Accessed November 27, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-frst-gene-therapy-adults-severehemophilia
  4. First gene therapy for hemophilia B, CSL’s Hemgenix, approved by the European Commission. News release. CSL Behring. February 20, 2023. Accessed November 27, 2023. https://newsroom.csl.com/2023-02-20-First-Gene-Therapy-for-Hemophilia-B,-CSLs-HEMGENIX-R-,-Approved-by-the-European-Commission
  5. CSL’s Hemgenix receives MHRA authorisation. News release. PharmaTimes. March 27, 2023. Accessed November 27, 2023. https://www.pharmatimes.com/news/csls_hemgenix_receives_mhra_authorisation_1490181
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