Enrico Ferri Grazzi, HCD Health Economist attended the EAHAD 2021 Virtual Event, and offers his personal selection and summary of the key sessions and landmark developments presented over the three day meeting. To discuss with Enrico please e-mail: firstname.lastname@example.org.
Novel therapies in haemophilia were presented on Day 1 of EAHAD, David Lillicrap, MD, FRCPC, Professor in the Department of Pathology and Molecular Medicine at Queen’s University, Kingston, Canada, explored the future role for FVIII concentrate in a very detailed review, comparing the pros and cons of current FVIII and placing these conclusions in the context of the pros and cons of newer therapeutic alternatives including FVIII mimetic antibodies, haemostasis rebalancing agents and gene therapy.
David is the recipient of a Senior Canada Research Chair in Molecular Hemostasis. In 2013, he was elected to the Fellowship of the Royal Society of Canada. He is a member of the World Federation of Haemophilia’s (WFH) Medical Advisory Board.
The comparative safety of standard and novel therapies (inhibitors, pegylation and thrombosis) was thoroughly reviewed by Dan Hart. His review included questions around optimal adverse event reporting and selection of comparator choice. Within his conclusions he observed ‘thrombosis risk has become a priority safety outcome consideration in haemophilia therapeutic studies, long term follow up and translation to real world setting.
Dan Hart’s clinical interest in haemostasis and thrombosis with Prof Machin, Dr Marie Scully and Dr Hannah Cohen, led to a Senior clinical fellowship under the directorship of Prof Ted Tuddenham at the Royal Free Haemophilia & Thrombosis Centre after completion of his FRCPath in January 2009. He was then appointed as Senior Lecturer in Haematology at Barts and The London School of Medicine & Dentistry, Queen Mary University London, in Sep 2009 to join the clinical team in the Haemophilia centre and thrombosis service, whilst setting up a research programme in immunohaemostasis in the Blizard institute.
Glenn Pierce, MD, PhD, currently serves on the World Federation of Haemophilia (WFH) Vice President Medical and WFH USA Board of Directors and National Haemophilia Foundation (NHF) U.S. Medical and Scientific Advisory Council, delivered his overview of gene therapy, exploring the topic ‘where are we now’. The lecture covered scientific development in gene therapy for Haem A and B, the observed efficacy and comparative safety of AAV FVIII and FIX. Glenn called for long term follow up to assess efficacy and safety, and concluded,
- Therapeutic levels are achieved most of the time
- There are variable and unpredictable responses
- There is no evidence of gene immune responses
- There is no industry wide consensus on manufacturing
Day 1 was launched by Brian O’Mahony, Chief Executive of the Irish Haemophilia Society. He represents the Society on the statutory National Haemophilia Council and he is the Vice Chair of the Haemophilia Product Selection and Monitoring Advisory Board. Brian leads the organisation, prepares policies and plans for the organisation and oversees the implementation of the strategic plan. In his opening presentation, Brian examined the patient perspective of electronic health records, drawing upon experience with EHR since 2006 in the Republic of Ireland. The new system of HER, launched in 2019 was used to show the empowering patient centric opportunity offered by patient access to a 24 hour secure on-line website and app that delivers real time clinical management (bleed alerts), improved physician/patient decision making and a higher level of security than the earlier paper based systems.
The diagnosis of bleeding disorders in women and girls was addressed on Day 2, in Session 2. Roseline D’Oiron identified disparities between genders in their access to treatment and diagnosis, supported by data from US and French registry data. Early diagnosis and its importance were advocated, along with recommendations for improvement in clinical approach and the tools that support early diagnosis by clinicians. Roseline is a Clinician Investigator and Associate Director at the Reference Centre for Haemophilia and Other Congenital Rare Bleeding Disorders, Congenital Platelets Disorders and von Willebrand Disease at Bicêtre Hospital AP-HP – University Paris XI, Le Kremlin–Bicêtre, France.
Other topics selected by Enrico , include paediatrics and platelet disorders and better understanding of ankle pain in haemophilia patients.
Please e-mail email@example.com for further discussions.
For more information on the current and planned real world evidence research undertaken by HCD Economics, please contact firstname.lastname@example.org.