The government is consulting doctors and patients on a framework towards earlier licensing for therapies for millions of people in the UK.
The Medicines and Healthcare products Regulatory Agency has launched a public consultation on the Rare Disease Therapies Regulatory Framework. It aims to centre the development of rare disease treatment in the UK.
The framework sets out a new model for how individual rare disease therapies could be tested, licensed and monitored. The goal is to make it quicker and less expensive to safely develop therapies for rare diseases. In total, these affect 3.5 million people across the UK, yet fewer than 5% of rare diseases currently have an approved treatment.
Frameworks usually require developers to meet standards designed for large-population diseases. However, this one would introduce risk-proportionate flexibility, balancing the realities of rare disease development with patient safety.
Significant need
The new framework will apply to rare conditions affecting not more than one in 50,000 people where there are quantifiable barriers to ‘standard’ clinical trials. Entry will be guided by criteria such as the severity of the disease and unmet need.
“By helping innovative therapies reach patients faster, this framework has the potential to transform lives, strengthen the UK’s position as a global leader in life sciences and give renewed hope to families who have waited too long for progress,” said public health minister Sharon Hodgson.
The Rare Disease Therapies Framework itself was announced in November last year and is supported by patient groups, universities, industry organisations and regulators in the Rare Disease Consortium.
Doctors, researchers, patients and the public can respond to the consultation before 30 July.
