Accelerating access to medicines for rare diseases: A case study of the use of the NICE Advice service as part of the Innovative Licensing and Access
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 NICE published draft guidance on a new treatment option for late-onset Pompe disease (LOPD) ahead of it gaining marketing authorisation by the MHRA.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,...
