Earlier today, UK-based regulator the National Institute for Health and Care Excellence (NICE) approved Voxelotor for use across the NHS to treat over 4,000 Sickle Cell Disease (SCD) patients.
After years of setbacks in its approval, with support from novel data generation firm Sanius Health, the therapy will provide thousands of patients with SCD across England with the long-awaited opportunity to dramatically improve their quality of life and overall outcomes.
Sources close to the matter confirmed that, during the NICE Technology Appraisal meeting held on the 4th of April 2024, Sanius Health’s novel real-world evidence encompassing biometrics, quality of life, Patient Reported Outcomes (PROs), therapy impact insights, and research innovations was referenced at several times as a critical component towards final approval of the therapy.
The committee agreed that greater uncertainty in the estimates of clinical effectiveness to support the economic modelling for Voxelotor could be accepted, given the substantial and longstanding disadvantages experienced by people with SCD.
It was frequently quoted that Sanius Health’s extensive real-world data and evidence was critical in demonstrating their findings on the clinical effectiveness and potential impact of Voxelotor for patients with SCD.
Sanius’ data provided a compelling real-world complement to support the overall approval decision.
This included the provision of invaluable insights leveraging the company’s industry-leading real-world evidence, generated from the largest databank of SCD patient data.
Working alongside research colleagues, Sanius’ insights helped to demonstrate the significant unmet needs of the population and highlighted Voxelotor’s potential transformative impact.
Sanius Health’s work cuts across multiple domains in accelerating research and evidence generation for faster therapy approvals, with the patient voice and unmet need powering this new way of producing real-world insights.
As one of the largest cohorts of patients with SCD, Sanius leads the way in terms of innovative research and driving access to therapies.
Orlando Agrippa, Chief Executive Officer of Sanius Health, said that the company is “thrilled that Voxelotor has been approved as it promises to improve the quality of life for thousands living with sickle cell disease.”
“After years of working on the evidence-generation strategy around Oxbryta, today marks a truly emotional day for us,” Agrippa said.
“It was one of the first therapies we started to work on within SCD, with our mission being to support the many industry partners we work with in providing access to new and novel therapies.
“Despite multiple setbacks, Sanius was able to deliver a successful result for patients — choice for patients, clinicians, better access and better outcomes being at the core of our motivation.”
Sanius Health’s analyses demonstrated remarkable reductions in vaso-occlusive crises, acute chest syndrome episodes, and other life-disrupting complications experienced by patients initiating Voxelotor.
Within the Sanius Health patient ecosystem, Voxelotor users have shared extraordinary stories of improved quality of life, reduced pain, and substantial energy level increases.
“This ground-breaking approval means patients now have the opportunity to access this innovative tablet treatment that aims to improve energy levels and reduce pain from veno-occlusive crises”, added Dr. Sanne Lugthart, Clinical Director Haematology at Sanius Health.
“We would love to see how patients get on with Voxelotor and monitor how their outcomes improve through our supportive patient ecosystem at Sanius.”
Sanius will continue leveraging its leading data platform to support the development of breakthrough sickle cell disease treatments and ensure patient perspectives remain at the forefront of innovation.
To learn more about Sanius Health, visit: www.saniushealth.com
