Maria Luisa Giorello’s presentation focused on the complexities and strategies involved in implementing gene therapies, particularly for haemophilia. Giorello, a member of Pfizer's gene therapy global team, emphasised the significant challenges in hospital readiness, patient flow, product flow, and payment processes.
The presentation began by highlighting the need for education and infrastructure to support gene therapy. It was noted that the patient journey required additional workflow at the beginning and end, including education about gene therapy and post-treatment monitoring. The product flow dimension was discussed, emphasising the need for ultra-cold chain delivery and the preparation required in hospital pharmacies.
One of the most critical points addressed was the payment flow. Gene therapy's high cost posed significant challenges for hospitals, including complex payment and reimbursement processes. Giorello mentioned that in many markets, there were no DRG codes for gene therapy, and additional reimbursement settings needed to be discussed.
The hub and spoke model was identified as essential in haemophilia settings, requiring the creation of referral networks and standard operating procedures between different centres. The presentation also highlighted the importance of education at the hospital level, with successful examples from the European Society of Haemophilia and the National Haemophilia Foundation in the US.
Giorello then discussed how the industry plays an active role in complementing educational efforts, including producing product-specific educational material and providing services like mobile phlebotomy and neutralising antibody testing. Giorello finished the talk by calling for more proactive allocation of resources and standardisation of procedures before gene therapies were approved and on the market.
Overall, the presentation provided a comprehensive overview of the challenges and strategies in implementing gene therapy, highlighting the need for education, infrastructure, and payment solutions to ensure successful hospital readiness.
