Gene therapy (GTx) offers a revolutionary treatment option for patients with haemophilia. It’s a one-time treatment that could potentially provide continuous protection for years. This significant treatment decision warrants a thorough understanding and careful consideration of all relevant information.

Prime HCD’s mission is:

  • To empower patients with haemophilia, their families, and their haematologists to make informed treatment decisions by providing them with a comprehensive roadmap of  GTx considerations.
  • Including key discussion points, milestones, and potential timelines, all designed to facilitate informed decision-making.

We conducted a quantitative survey across 16 UK haemophilia treatment centres as part of a multi-stakeholder and multi-disciplinary research project. The collated results informed an advisory board discussion on the most suitable model of care for implementing haemophilia GTx in the UK. The board comprised of haemophilia healthcare professionals, expert patients, and patient advocacy representatives.

To facilitate decision-making, we developed a milestone-based patient consultation process with a two-gateway model of informed consent.

The process comprised three main milestones and four main periods:

  • Initial Discussion Period: where risks, benefits, safety and efficacy are discussed, with the involvement of the home and dosing centre (where applicable), culminating in the “expression of interest” by the person with haemophilia (PwH) and eligibility testing

Gateway 1: Referral to the dosing centre and the first point of consent.

  • Validation Period: expected duration of at least 6 weeks, where liver and cardiovascular assessments take place and logistics of treatment are discussed, including potential for steroid use and an assessment of the PwH’s expected ability to comply with requirements

Gateway 2: The second point of consent.

  • Cooling Off Period: expected duration of at least 6 weeks, where final GTx delivery is discussed (including all practicalities and follow-up logistics), with discussions envisaged to take place with the involvement of both home and dosing centres, family/friends and the nursing staff of the dosing centre

Dosing Milestone: The final consent is collected prior to dosing and then GTx is administered.

  • Follow-up Period: Ongoing monitoring and support post-treatment.

At Prime we are proud to Engage. Educate. Empower. We hope the forming of a practical roadmap for GTx consideration, will add to the development of services and ensure relevance in service provision.

For the full poster showcasing the research please visit WFH 2024 World Congress ePoster gallery, Poster no.022 or contact Lesley.piltz@primeglobalpeople.com