News and Insights
Rare disease treatment changes: communication is critical
December 2, 2025
Rare disease regulation in the UK is on the cusp of a change that will be felt far beyond Whitehall. It will reshape how patients, clinicians, innovators and communicators talk about what access really means. For health communicators, it is an opportunity to lead a narrative about innovation, equity and evidence, championing the voices of people living with rare conditions.
In this blog, you will learn:
- What the MHRA’s major changes for rare disease treatments mean
- How the changes sit within the wider context of the UK’s life sciences and rare diseases strategies
- Communicators’ role in explaining the changes clearly and honesty, championing the voices of people whose lives are at stake.
The MHRA’s decision to overhaul the rulebook for rare disease therapies – announced last month – is not just a technical regulatory update. It’s a response to sustained pressure from patients, clinicians, industry and advocacy groups. It’s a test of whether the UK is serious about being a life sciences superpower that works for people with the greatest unmet need.
For someone who has spent more than two decades in healthcare communications, much of it working with rare disease communities, this feels like an important moment. It’s an opportunity to match scientific possibility with regulatory realism and clearer, more honest conversations with patients and the public.
Reducing the treatment odyssey: what the MHRA’s changes mean for underserved communities
Around 3.5 million people in the UK live with a rare disease, that’s one in 17 of us. However, fewer than 5% have an approved treatment – a huge gap between need and what the system currently delivers. Families routinely spend years pursuing a diagnosis, often only to discover that there is little or nothing available in terms of licensed therapy, while the economic cost of delayed diagnosis and limited treatment options runs into tens of billions of pounds each year.
The MHRA’s position paper signals a shift toward more flexible, proportionate pathways, including the possibility of a single early approval covering both clinical trial authorization and marketing, underpinned by real‑world evidence and intensive safety monitoring. For people living with a rare disease, that could mean taking years off the journey from promising data to meaningful access, particularly in conditions where every month of delay has irreversible consequences.
Gene-based and highly individualized therapies, as well as mRNA platforms, have already transformed what is scientifically possible in rare conditions. The proposed framework recognizes that some of these treatments may be tailored to tiny subgroups, or even to a single individual, with adaptive and iterative evidence packages being considered, rather than the traditional one-and-done pivotal trial model.
Bringing stakeholders together
One of the most encouraging aspects of the MHRA’s approach is the creation of a Rare Disease Consortium that brings together regulators, NICE, DHSC, NHS England, patient groups, academics and industry. This suggests an acknowledgment that regulation does not happen in a vacuum. It’s a flexible pathway which only matters if health technology assessment processes, commissioning decisions and service design keep pace.
For NHS leaders and policy makers, that means thinking beyond the marketing authorization to questions of workforce, infrastructure, data quality and long‑term follow‑up, especially for therapies that may be given once but need tracking for life.
A strong signal for supporting innovation in rare disease therapies
The MHRA’s proposals sit within the government’s wider life sciences and rare diseases strategies, including the England Rare Diseases Action Plan 2025. For biopharma, biotech and MedTech companies – particularly those specializing in orphan and ultra‑orphan therapies – the signal is clear: the UK intends to be a more attractive, predictable and collaborative environment in which to develop and launch rare disease products.
The language of “flexible licensing”, “investigational marketing authorizations” and adaptive trial designs will quickly make its way into investor presentations and pipeline announcements. Done well, this can strengthen the UK’s positioning as a rare disease innovation hub. Done badly, it risks heightening public skepticism about “fast‑tracked” medicines, fueling fears that safety is being traded off against speed.
Clear communication will be critical
The proposed reforms represent an opportunity and a stress test all at once for the NHS. For faster approvals and more individualized therapies, robust infrastructure must be in place for genomics, digital records, consented data sharing and long-term outcome tracking, as well as clear pathways for how specialist centers and regional networks will work together.
Communicating these changes internally will be just as important as the messaging externally; clinicians and managers need to understand how new regulatory routes align with service priorities, budgets and workforce planning, particularly in a stretched system. This is where the communications function inside the NHS and its partner organizations can play a strategic role.
If the UK is going to realize its ambition of becoming a global leader in rare disease innovation, success will be measured not just in the licenses granted or investment attracted, but in how clearly and honestly these changes are communicated to the people whose lives are at stake.
Takeaways for communicators
From a communications perspective, these reforms create an opportunity to reframe rare disease from a rarely seen story, into a lens through which to talk about system-wide innovation, equity and the use of real‑world evidence. The voices that should lead that conversation are those of people living with rare conditions and the organizations that have advocated for change for years; the role of professional communicators is to amplify them, not to speak over them.
In this environment:
- Responsible communication must hold two truths at once. Firstly, that the science is genuinely groundbreaking and secondly, that speed of access does not remove uncertainty around long‑term safety
- Comms teams will need to translate technical concepts into real‑world value stories: how flexible pathways reduce development risk; how earlier engagement with regulators and payers improves launch readiness; and how working with UK patient organizations and the NHS can generate richer data and better-designed studies
- Treatments may reach patients earlier based on more limited traditional trial data, so explaining what that means for benefit–risk, ongoing evidence generation and patient choice will require nuance and trusted voices
- Equity of access does not disappear just because a faster route is created. We must continue to elevate the voices of those at risk of being left behind when innovation is pushed into action.
Comms teams working in rare diseases hold a unique role in ensuring that this new rulebook is not only written and implemented, but understood, turning regulatory reform into informed hope rather than hollow headline. This means resisting the urge to present regulatory innovation as an overnight revolution. Instead, the task is to co‑create narratives with patients, advocates and clinicians, which describe rolling evidence generation, conditional approvals and real‑world data in plain language, while transparently articulating what is known now, as well as what will only be understood with time.
How FINN Partners can help
FINN Partners works with the product innovation sector, alongside advocacy groups, governments, policy advisors and health providers to deliver award-winning brand and corporate communication campaigns that transform lives. Find out about our work in health.
Frequently Asked Questions
1. What are the MHRA’s major changes for rare disease treatments?
The MHRA has signaled a shift towards more flexible, proportionate pathways, including the possibility of a single early approval covering both clinical trial authorization and marketing, underpinned by real‑world evidence and intensive safety monitoring. It could take years off the journey from promising data to meaningful access. The proposal recognizes that gene-based and individualized therapies, as well as mRNA platforms, may be tailored to subgroups or even individuals.
2. What do the MHRA’s rare disease treatments mean for the UK’s life sciences and rare diseases strategies?
The signal to biopharma, biotech and MedTech companies – particularly those specializing in orphan and ultra‑orphan therapies – is clear: the UK intends to be a more attractive, predictable and collaborative environment in which to develop and launch rare disease products. It could strengthen the UK’s positioning as a rare disease innovation hub.
3. How should communicators explain the changes around rare disease treatments?
Success of this initiative will be measured not just in licenses granted or investment attracted, but in how clearly and honestly the changes are communicated to people whose lives are at stake. Communicators must translate technicalities into real-world stories, be honest and realistic and elevate the voices of those at risk of being left behind.
POSTED BY: Sophie Taylor-Roberts
