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Today, Wednesday 22 November, the government announced its support for the Rare Therapies Launch Pad, a new programme that will develop a pathway for children with rare conditions to access individualized therapies.

The programme’s first project will explore the use of individualized therapies known as antisense oligonucleotides (ASOs) to treat children with ultra-rare and life-threatening brain conditions.

The pilot is developed by a consortium including Genomics England, the Medicines and Healthcare products Regulatory Agency (MHRA), Oxford-Harrington Rare Disease Centre, Mila’s Miracle Foundation and the Association of the British Pharmaceutical Industry (ABPI). It will help identify a sustainable and scalable approach to delivering individualized therapies for children across a wider range of rare conditions, including establishing a proportionate regulatory pathway. This end-to-end pathway would cover diagnosis, the design and rapid manufacturing of these therapies, and treatment. The project also aims to generate evidence to help establish potential reimbursement for individualized therapies beyond the pilot.

Tens of millions of children globally and hundreds of thousands in the UK have rare and potentially life-threatening genetic conditions but less than 10% of rare conditions have a treatment. New therapies can now be created for use by a single patient but currently there is no route to access these individualized medicines for most patients.

Julia Vitarello’s daughter Mila, hydrocodone 100 mg was diagnosed with a fatal, rare condition called Batten disease at the age of 6 and became the first person in the world to receive an individualised therapy. She first set up the charity Mila’s Miracle Foundation to drive and fund the creation of Mila’s drug ‘Milasen’.

Julia Vitarello, founder of Mila's Miracle Foundation and the N=1 Collaborative, said: "We're living in an incredible time where science is no longer the limiting factor. We now have the technology to find the underlying genetic cause of disease and rapidly develop a medicine to target it, even if unique to just one person. Through the Rare Therapies Launch Pad, the UK is leading the world in building the pathway from Mila to Millions." 

Over the last decade, continued investment has placed the country at the forefront of genomics, enabling us to offer the best clinical care for patients and provide more diagnoses to those who need them. Recent scientific advances mean we’re now in a position for genomics to move beyond diagnosis and support the development of innovative therapies. In the UK, we are uniquely placed to do that.

The Rare Therapies Launch Pad aims to do more for the hundreds of thousands of children in the UK with rare and potentially life-threatening genetic conditions by helping to identify a route for them to access individualized therapies, tailored specifically for them, quickly and safely. Today’s announcement is an important step in that direction.”   

Dr Rich Scott, Interim CEO, Genomics England

Dr June Raine, MHRA Chief Executive, said: “We are excited to be part of this new initiative which has the potential to make so much difference to children suffering from rare genetic diseases, and their families.

“The MHRA’s role will be to develop a proportionate regulatory pathway for the UK market that delivers rapid access to potentially life-saving treatments; and to independently assess products coming through this pilot to determine if they are safe and effective for patients. 

“As an enabling regulator, we prioritize patient safety while encouraging innovation at pace within the market, to make the UK the best place in the world to bring life-changing healthcare products to patients, safely.”

Professor Matthew Wood, Director, Oxford-Harrington Rare Disease Centre, said: “The Rare Therapies Launch Pad is a very welcome major advance towards delivering therapies to people with rare genetic diseases. It will put the UK at the forefront of therapeutic and regulatory innovation in this area, building on its global leadership in genomics. The Oxford-Harrington Rare Disease Centre has a mission dedicated to the development of medicines for rare diseases and will play a central role in the Rare Therapies Launch Pad in the discovery and delivery to patients of these new, individualized drugs.”

Dr Dan O'Connor, Director of Regulatory and Early Access Policy, ABPI, said: “Developing medicines in small populations is challenging and is particularly so in those patients who have very rare conditions. That is why this opportunity is so exciting for clinicians, industry, and patients, as it will support the development of new treatment pathways, giving better access to long-needed therapies for patients with high unmet medical needs.”