Are You Ready for the Next Advances in Haemophilia Care? [Supported Content]
Special feature brought to you by CSL Behring.
The healthcare industry has faced unprecedented disruption in the last 10 years, via pioneering therapies, modern technologies and global crises. Haemophilia care is no exception, and with a wave of innovation and novel therapies on the horizon, we need to ask: are we ready?
An event, therapy or technology is considered disruptive to healthcare when it alters the current care model (1). The COVID-19 pandemic introduced unprecedented challenges within the healthcare system (2), but haemophilia treatment centres have been able to adapt rapidly and introduce new methods for communicating with their patients, including remote consultations and alternative ways of providing treatment services or medications, ensuring continued access to care (3,4). The introduction of extended half-life products for prophylaxis established a new standard of care and initiated a wave of new treatment options (5). In haemophilia A, the first subcutaneous non-factor therapy was approved in 2018. Each change to the treatment paradigm has been adopted globally despite the changes required within haemophilia treatment centres to dosing, laboratory monitoring, patient education and safety monitoring (6,7). All of these are examples of how the haemophilia treaters and their institutions could rapidly adapt to disruptions to the current care models.
The next generation of therapies in development may change haemophilia care even more, and the challenges that need to be overcome by healthcare providers and haemophilia treatment centres to integrate these therapies into clinical practice are currently under debate. To better understand the potential challenges these innovations may bring, we can look at new interventions in other therapy areas and use them as models to see how healthcare communities addressed dramatic shifts in the treatment paradigm and brought new medicines into clinical practice. Adeno-associated virus vector-based gene therapies have now been approved for the treatment of spinal muscular atrophy (SMA) and retinal dystrophy, completely changing treatment and care for many individuals with these diseases. For example, in SMA, the presence of an effective gene therapy has led to increased new-born screening for SMA globally. Without treatment, most individuals with SMA would die or require permanent respiratory support by the age of 2. Registries have also been set up to gather real-world data on treatment for this rare disease (9,10). Immunotherapies and CAR-T cell therapies have given cancer patients new therapeutic options. Biomarker analysis has now become standard in most cancers to identify who would benefit from these new therapeutic options (11). Combination oral therapies for hepatitis C shifted the focus for care beyond treatment, toward global eradication of the disease. This has led to increased screening for hepatitis C and a significant push to increase education among healthcare professionals and the population (12,13). The successful implementation of these treatments in clinical practice has paved the path and laid the foundation for the next generation in haemophilia.
During this non-promotional symposium organised and supported by CSL Behring, we want to ask the question, ‘How Do We Keep Up with the Times?’ Experts in haemophilia will provide their perspectives on how to overcome barriers, take advantage of facilitators and manage shifts in care models within clinical practice.
We invite you to attend our symposium: Advancing Care for Haemophilia: How Do We Keep Up with the Times? Attend our symposium in person, watch live Sunday 10 July, 13:15–14:30 BST or stream on-demand at https://www.isth2022.org/.
1. Shaw et al., Front Pharmacol 2020, 11, 818. 2. Hermans et al., Haemophilia 2020, 26 (3), 371-2. 3. Zhang et al., Haemophilia 2020, 26 (6), 1031-7. 4. Valentino et al., Haemophilia 2020, 26 (5), e230-1. 5. Lambert et al., Ther Adv Hematol 2018, 9 (9), 295-308. 6. Krumb et al., Haemophilia 2021, 27 (5), 736-43. 7. Bowyer et al., Haemophilia 2021, 27 (S3), 142-7. 8. Talib et al., Asia Pac J Ophthalmol 2020, 9 (3), 159-79. 9. Schorling et al., J Neuromuscul Dis 2020, 7 (1), 1-13. 10. Stevens et al., Ann Pharmacother 2020, 54 (10), 1001-9. 11. Yap et al., Cancer Discov 2021, 11 (6), 1368-97. 12. Applegate et al., Infect Dis Clin North Am 2018, 32 (2), 425-45. 12. Medlinskiene et al., BMC Health Serv Res 2021, 21 (1), 1198. 13. Bakhai et al., BMJ Open Qual 2019;8:e000577