Reflecting on rare diseases and the importance of the ongoing work to improve access to specialist medicines, it is worth spotlighting a case study published by NICE showcasing how the ILAP can speed up patient access to new medicines. NICE published draft guidance on a new treatment option for late-onset Pompe disease (LOPD) ahead of it gaining marketing authorisation by the MHRA. This marks the first time in NICE’s history that such an event has happened.
Pompe disease is a rare, genetic, chronic and progressive metabolic disorder. LOPD can occur any time from 12 months up to late adulthood. It is characterised by progressive muscle weakness and respiratory problems, having a severe effect on quality and length of life. Amicus Therapeutics’ cipaglucosidase alfa in combination with miglustat target an unmet need for adults with LOPD. It is one of the first medicines that used the ILAP.
The ILAP encourages manufacturers to engage with key players in the UK healthcare systems very early, from the pre-clinical stage, and often in the journey from development to patient access. It aims to accelerate the time to market by providing companies with strategic advice and anticipating potential challenges in achieving market access.
In their preparations for submitting evidence as part of the appraisal process, the company chose to use the NICE Advice health economic model peer review service early in the process as part of ILAP discussions. NICE Advice service is one of the institute’s established programmes providing sound foundations for the delivery of the ILAP. The NICE Advice service helped identify areas of uncertainty around health states that could be topics of discussion during the appraisal process. This helped minimise potential delays that could have arisen later in the process. The ILAP enabled regular, constructive and open dialogue with NICE paving the way for accelerated patient access to this medicine, underscoring the importance of innovative access pathways in advancing rare disease care.
Building better system alignment of processes and partners throughout the access journey can save time and bring substantial benefit to patients. It will be interesting to watch how the ILAP pathway and other schemes aiming to enable innovation - such as the Early Access to Medicines Scheme and the Innovative Medicines Fund - evolve over the next few years, and particularly the benefit they bring to patients living with rare diseases in terms of timely access to promising treatments. As noted in the England Rare Diseases Action Plan 2024, ILAP partners are currently working together to refresh the existing operating model to ensure that ILAP is delivering on its ambition.
Photo credit: Eugene A (iStock)
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