It was great to attend the Terrapinn Advanced Therapies Congress in London last week.
I had the opportunity to speak to leaders driving innovation in cell and gene therapies; a large portion of discussion was around the challenges we face in ensuring patients can benefit from these advancements in a timely manner and what we, as industry, together in collaboration with our key governmental and advocacy stakeholders, can do to help overcome barriers to access.
In my role as a Therapeutic Area Market Access Leader, my team and I work through these challenges on a daily basis – driving patient access to innovative medicines is our passion and it is what drives us.
Right now, most healthcare systems are centred around treating disease, rather than preventing it. In the case of some inherited retinal diseases (IRDs), an area I am working in, intercepting the disease before a patient displays symptoms may show to be more advantageous than halting disease progression when patients have already developed visual impairment.[1] For some IRDs, symptoms can begin anytime, and individuals can experience differences in severity or type of vision loss.[2]
It can be overwhelming and frightening for those experiencing these symptoms.[3],[4] Many of those affected experience hopelessness and question if they will be able to maintain their independence, pay for needed medical care, retain employment, and provide for themselves and their families.[4] But this doesn’t need to be the case. If we’re able to identify and intercept the disease earlier, we could vastly improve the quality of life of those living with IRDs.[5]
This underpins the sense of urgency that my team and I share with so many others in the IRD community, so it was refreshing to hear the word ‘urgent’ being referred to throughout the meeting. Whilst I’m still digesting everything I heard, discussed and saw, I wanted to share some of my take-aways:
- It was clear that a paradigm shift is needed when it comes to understanding the value of gene therapies, and collaborative approaches will be key to achieving this.
- It was re-emphasised that current health technology assessment processes do not consider the holistic value of gene therapies on patients and society at large.
- We need to continue collaborating with all relevant stakeholders to come up with a comprehensive plan to enhance patient access and address the current high unmet needs.
Focusing our efforts on shifting treatment to earlier disease interception through advancement and investment of cell and gene therapies is critical to identifying and intercepting IRDs earlier. It’s a mission we can all get behind.
#adtherapies2023 #CellAndGeneTherapy
[1] Hu, M. L., Edwards, T. L., O’Hare, F., Hickey, D. G., Wang, J. H., Liu, Z., & Ayton, L. N. (2021) Gene therapy for inherited retinal diseases: progress and possibilities. Clinical & experimental optometry, 104(4), 444–454.
[2] Prevent Blindness. Eye Diseases & Conditions: Inherited Retinal Diseases. Available at: https://preventblindness.org/inherited-retinal-diseases/ Last accessed: March 2023.
[3] Chivers, M., Li, N., Pan, F., Wieffer, H., Slowik, R., & Leartsakulpanitch, J. (2021). The Burden of X-Linked Retinitis Pigmentosa on Patients and Society: A Narrative Literature Review. ClinicoEconomics and outcomes research : CEOR, 13, 565–572.
[4] Welp, A., Woodbury, RB., McCoy, M.A., et al., editors. (2016) Making Eye Health a Population Health Imperative: Vision for Tomorrow. Washington (DC): National Academies Press (US): 3, The Impact of Vision Loss. Available from: https://www.ncbi.nlm.nih.gov/books/NBK402367/ Accessed 13 March 2023
[5] Salzman, R., Cook, F., Hunt, T., Malech, H. L., Reilly, P., Foss-Campbell, B., & Barrett, D. (2018). Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments. Molecular therapy. The journal of the American Society of Gene Therapy, 26(12), 2717–2726.
