- The newly announced program aims to develop a pathway for children with rare diseases to access therapies designed specifically for them.
- The first project will focus on the use of antisense oligonucleotides (ASO) in children with extremely rare and life-threatening brain diseases.
- In the long term, the Rare Therapies Launch Panel will support access to other types of personalized therapies across a broader range of rare conditions.
Today, Wednesday, November 22, the government announced support for the Rare Therapies Launch Panel, a new program that will develop pathways for children with rare diseases to access individualized therapies. impersonal.
The program’s first project will explore the use of personalized therapies called antisense oligonucleotides (ASO) to treat children with extremely rare and life-threatening brain diseases.
The pilot program is being developed by a consortium including Genomics England, the Medicines and Healthcare products Regulatory Agency (MHRA), the Oxford-Harrington Rare Diseases Centre, Mila’s Miracle Fund and the Industry Association British Pharmaceutical Industry (ABPI). It will help define a sustainable and scalable approach to delivering personalized therapies to children for a wider range of rare conditions, including establishing a corresponding regulatory pathway . This end-to-end roadmap will include rapid diagnosis, design and manufacturing of these therapies as well as treatment. The project also aims to generate evidence that will help establish reimbursement for personalized therapies beyond the pilot program.
After years of significant investment in life sciences, including genomics and innovative medicines regulation, the UK is uniquely positioned to provide a Launchpad for rare therapies.
Tens of millions of children globally and hundreds of thousands of children in the UK have rare and potentially life-threatening genetic diseases but less than 10% of rare diseases are treated. New therapies can now be created for a single patient to use, but there is currently no way to access these patient-specific medications.
Julia Vitarello’s daughter Mila was diagnosed with a rare, fatal disease called Batten disease at age 6 and became the first person in the world to receive personalized therapy. She first founded the charity Mila’s Miracle Foundation, the initiator of the pilot program, to promote and fund the creation of Mila’s drug ‘Milasen’.