The UK Medicines and Healthcare products Regulatory Agency (MHRA) has launched a public consultation on proposed new guidance for rare disease therapies.
In simple terms, this is about exploring whether there should be a more flexible way to assess and approve treatments for very rare conditions, especially where traditional clinical trials can be difficult or sometimes impossible to run.
The aim is to help promising treatments reach people sooner, while still making sure they are safe and effective.
Key proposals include:
A framework specifically designed for rare diseases with high unmet need
Recognition that randomised clinical trials are not always practical for very small patient populations
Acknowledgement that there may be limited natural history data for some rare conditions
A stronger focus on patient involvement in decision-making and communication throughout the process
Greater alignment between clinical development, regulatory approval, patient access and reimbursement
Potential use of alternative forms of evidence, including biomarker-based outcomes, video evidence, wearable data, real-world evidence and adaptive approaches where appropriate
The MPS Society welcomes the consultation and supports regulatory approaches that enable timely access to innovative therapies for rare and ultra-rare diseases, while maintaining patient safety.
Engaging with our community
We are currently gathering insights from patients, families and carers on the questions raised in this consultation. Their experiences and perspectives are informing our response and helping to ensure that the patient voice is clearly represented.
At the same time, we encourage clinicians, researchers, pharmaceutical companies and other stakeholders to review the consultation and submit their own response directly to the MHRA. We are supporting this process by raising awareness of the consultation through our channels, helping to ensure a wide range of informed perspectives are included.
The consultation closes on 30 July 2026 and represents an important opportunity to influence the future of rare disease therapies and patient access in the UK.
