Health Research Authority response to MHRA rare disease therapies regulatory framework consultation

We have responded to the Medicines and Healthcare products Regulatory Agency's (MHRA) consultation on a proposed regulatory framework for rare disease therapies.

The proposals aim to support the development of treatments for very rare conditions by creating a regulated pathway that enables earlier patient access while continuing to generate evidence on safety and effectiveness.

In our response, we support the principle of bringing more rare disease treatments into a regulated, evidence-generating pathway rather than relying on off-label use. We highlight that a regulated route can provide stronger safeguards for patients through ethical review, safety monitoring, transparency and structured evidence generation.

We also emphasise that public confidence in the framework will depend on maintaining strong participant protections, transparent reporting of evidence, and meaningful public involvement.

Read our full response

Read our full response to the rare disease therapies regulatory framework consultation below.

Section 1 – Overall aim and patient views

Question 1. In your view, is it desirable for more pharmaceutical treatments for rare diseases to be brought into a regulated pathway rather than used 'off-label' as many are at present?

The HRA supports, in principle, bringing more rare disease treatments into a regulated, evidence-generating pathway rather than relying on off-label use. A regulated route allows treatments to be developed and overseen within a framework of ethical review, structured evidence generation, safety monitoring and transparency. This protects patients better, and builds public confidence more effectively, than ad hoc off-label prescribing, where evidence is rarely captured systematically or shared.

The benefit depends on the research and oversight elements being genuinely rigorous and proportionate, so that earlier access does not come at the cost of participant protection or the quality of the evidence generated.

Question 2. The consultation sets out challenges associated with the development and regulation of treatments for rare diseases. Please identify any other challenges you are aware of that have not been considered or elaborate on those already identified.

Beyond those identified, we would highlight a number of further challenges:

- the pathway needs to integrate with existing research approvals, research ethics review, combined review and the UK Clinical Trials Regulations, so that sponsors experience one consistent process rather than a parallel or duplicative pathway