Mila to Millions: A New Era of Individualized Medicines

3rd February 2026

Summary: Published on 2 February 2026, this heartfelt MHRA article begins with an MHRA foreword from Chief Executive Lawrence Tallon, emphasising the agency’s commitment to placing patients with rare and ultra-rare diseases at the centre of regulatory innovation. The guest blog by Julia Vitarello recounts her daughter Mila’s battle with Batten disease and the creation of milasen — the first ever medicine uniquely designed for one person — illustrating the promise of individualised therapies. Vitarello explains how scientific advances now make it possible to design highly targeted treatments for many rare conditions, but that current systems favour “one medicine for many” and are not fit for these hyper-personalised approaches. To bridge that gap, she highlights the Rare Therapies Launch Pad pilot in the UK — a collaborative initiative bringing together regulators, industry, clinicians and patient advocates to develop scalable pathways and regulatory frameworks that can deliver bespoke medicines more broadly and equitably. The blog describes early progress, including approval of a Master Protocol trial covering children with varied fatal neurological conditions, with ambitions to move from “Mila to millions” by enabling process-based approval mechanisms and robust access infrastructure so children with rare diseases can receive tailored treatments earlier.

Link: https://www.gov.uk/government/news/mila-to-millions-a-new-era-of-individualized-medicines

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