World Sickle Cell Day 2025
Groundbreaking gene editing therapy for sickle cell disorder (SCD) became available on the NHS in England earlier this year.1 Although SCD is the fastest growing genetic disorder in the UK,2 with nearly 300 babies born with it each year,3 long-term underinvestment in research has led to a dearth of treatment options. The new gene therapy, exagamglogene autotemcel (exa-cel), is a welcome alternative to stem cell transplants, which is particularly vital given the shortage of matched donors.4 While this is potentially life-changing for eligible patients, who are among those more severely affected by SCD, everyone living with this disorder deserves better treatment options and holistic care.
For underserved conditions such as SCD, the barriers to reimbursement of novel therapies are considerable. Poor disease awareness leads to a lack of understanding of the unmet need, while a lack of high-quality data means that quantifying the natural history, burden of disease and potential therapeutic benefits is fraught with uncertainty.
What can we learn from the recent NICE appraisal of exa-cel, and how can we use these insights to better shape reimbursement and market access for patients with SCD and other underserved or rare conditions?
Market Access Insights: Patient Voice
In their recent appraisal of exa-cel, NICE highlighted the
large number of comments they received from the public, healthcare
professionals, carers and people with sickle cell disorder in response to the
consultation. Patient experts and patient testimonials were cited more than 25
times in the committee discussion, with NICE incorporating patient perspectives
into conclusions regarding the burden of the disease, limitations of current
therapies, approach to modelling utilities, and when accounting for both carer
quality of life and health inequalities by accepting a higher degree of
uncertainty in their decision making.4
At Visible Analytics, we have the expertise required to
bring the patient voice into reimbursement decision making. We can provide
training for patient organisations on the reimbursement process, conduct
patient interviews and burden of illness studies, compile value stories that
effectively bridge between the lived experience of patients and payer
requirements, and manage the HTA submission process to ensure that the value to
patients is communicated end-to-end.
Market Access Insights: Managed Access
Exa-cel is one of the first treatments to be made available
through the Innovative Medicines Fund managed access scheme.4 The
Innovative Medicines Fund was introduced in June 2022 in recognition of the
challenges facing novel therapies, particularly in rare diseases or underserved
populations, where the data are not always suitable for NICE to make decisions
on long-term reimbursement. The aim of the Innovative Medicines Fund is to
provide faster access to promising new non-cancer therapies on the condition
that additional data are collected during the managed access period to address
the outstanding uncertainties in the clinical or health economic evidence base.5
Since its launch, twelve drugs have appeared on the
Innovative Medicines Fund list, but ten of these were recommended for routine
commissioning, with interim funding provided via the fund. Only two—both gene
therapies for blood disorders—have been recommended for ongoing managed access
via the Innovative Medicines Fund: exa-cel for SCD and for
transfusion-dependent beta thalassaemia, and etranacogene dezaparvovec for
moderately severe or severe haemophilia B.6
One solution to the strains on healthcare resources is to implement a learning health system, a systematic approach to continuous, data-driven improvements in care.7 Conditional reimbursement agreements such as managed access through the Innovative Medicines Fund, where access is time-limited and conditional on additional data collection to inform longer-term decision making, form part of such a system. But when might other forms of value-based pricing and reimbursement, such as performance-based discounts or rebates, be more appropriate? At Visible Analytics, we can guide you through the most appropriate access route, providing strategic advice and actionable recommendations to secure reimbursement.
