Key Summary
- MHRA is inviting public consultations on its new regulatory framework for rare disease therapy.
- Pharmaceutical and life sciences industries can respond by 30th July.
- Rare diseases affect more than 3.5 million people in the UK.
The Medicines and Healthcare products Regulatory Agency (MHRA) is seeking public consultations on a new regulatory framework to boost the UK’s rare disease therapy development.
It is inviting pharmaceutical and life sciences industries to respond to the proposed Rare Disease Therapies Framework.
Rare diseases affect more than 3.5 million people in the UK. However, there is only a limited number of approved medicinal treatments.
This programme is for developing treatments for incredibly rare diseases in the UK (affecting 1 in 50,000 people or fewer). It is specifically meant for cases where it is normally too difficult or impossible to run standard clinical trials and develop medicine the usual way.
According to MHRA, the new plan targets the main scientific, testing, and financial roadblocks that stop rare disease treatments from reaching patients through the normal development process.
The consultation runs until 30 July, and industry input is critical to enable earlier access to potentially life-saving therapies for rare diseases.
The framework has been developed with input from the Rare Disease Consortium - a cross-sector group including the Medicines and Healthcare products Regulatory Agency (MHRA), the Health Research Authority (HRA), the National Institute for Health and Care Excellence (NICE), the Department of Health and Social Care (DHSC), NHS England, patient advocacy organisations, academia, and industry partners. The consultation now opens the process for wider input.
Public Health minister Sharon Hodgson said that landmark proposals like these represent an important step towards a more agile and compassionate system. She urges patients, clinicians, researchers, and carers to take part in this consultation.
Julian Beach, executive director of Healthcare Quality and Access at the MHRA, said that the guidance provides a more streamlined and efficient process for developers.
“By working closely with patients, partners and industry, we’re building a framework that supports innovation while maintaining the high standards of safety that patients expect,” he added.
