From ‘high conviction’ to patient impact: inside BIA’s UK Biotech Financing 2025 report – Part two
Interview with Dr Martin Turner, director of policy and external affairs, and Rosie Lindup, senior policy and public affairs manager, the UK BioIndustry Association (BIA)
Estimated reading time: 11 minutes
Catch up with part two of our conversation with experts Dr Martin Turner, director of policy and external affairs, and Rosie Lindup, senior policy and public affairs manager at BIA as we discuss the role of policy and regulatory bodies in driving investment in the UK
Policy, access and the patient pathway
If UK Biotech Financing 2025 suggests that the capital side of the ecosystem is finally turning a corner, BIA’s Innovation to Impact work is a reminder that finance alone cannot get treatments to patients. For rare disease innovators, the real test is whether regulatory and reimbursement systems evolve fast enough to match the science—and whether they do so in ways that keep patients, not just balance sheets, at the centre.
Bridging the gap from investment to real patient access
Rosie is clear: while the science is flourishing, too many rare disease products still fail at the access stage. “There have been some highly innovative rare diseases products that have managed to navigate the UK’s regulatory reimbursement processes. But there also are many that haven’t, and some of those have been made available in European countries. It’s not just in comparison to the US that we’re behind.”
The data are stark: “The current data1 shows us that around half of rare disease medicines are not being made available to patients in the UK on the NHS. Some of those are not even being submitted to the MHRA, because the company knows there’s just no likelihood of making it through that final piece.”
In other words, the access environment is now an investment risk factor. If companies (and their investors) believe a product is unlikely to clear NICE and NHS reimbursement hurdles, they may deprioritise the UK entirely, diverting trials, launches or even marketing authorisation submissions elsewhere.
Rosie argues that fixing this requires both regulatory innovation to make it feasible to generate and interpret evidence in small-scale populations, and reimbursement reform, especially for rare and ultra‑rare conditions: “On the whole, so far, the issues have been more on the reimbursement side of things, but we also definitely need to reform and see some change on the regulatory side, especially if we want to have more innovative therapies coming through and at an earlier stage.”
BIA see clear and concrete solutions2—from more adaptive payment models to better alignment between MHRA approvals and NICE assessments—aimed at ensuring that the “renewed investor confidence” highlighted in 2025 doesn’t simply collide with an unchanged appraisal system.
The new regulatory rulebook: MHRA as a signal to investors
The MHRA’s overhaul of its rare disease pathways is one of the most significant system changes in play, and one that is highly legible to global investors.
Rosie highlights that the agency is deliberately designing regulatory routes that are attractive to smaller, innovation‑led companies: “They’re considering certain designations that they could give at certain stages of the process, that is essentially a way of the MHRA saying that this is a promising medicinal product that hopefully would attract investors to know that that’s something worth investing in before perhaps getting to the final approval stage.”
This is important because such early designations function as credibility signals: a form of de‑risking that investors can price into their decisions long before full marketing authorisation.
Rosie notes that MHRA is also exploring earlier, staged access backed by continued data collection: “They’re looking at an investigational marketing authorisation that allows earlier approval, while still collecting data. Ultimately especially for severe rare diseases where timing is so important, allowing access at that earlier stage whilst data is collected.”
For investors, these kinds of pathways can:
- shorten time to revenue in ultra‑rare indications
- make real‑world data (RWD) an accepted, formal part of the evidence package
- reduce the binary risk of “all or nothing” decisions at the very end of development
Crucially, however, Rosie stresses that regulatory progress must be mirrored on the payer side: “If MHRA are going to be doing things differently and being more pragmatic and flexible, we’re going to need to see that on the payer side as well.”
Without NICE and other HTA bodies adapting their frameworks for small populations and non‑traditional evidence, regulatory innovation alone cannot fully de‑risk the UK for rare disease investors.
The life sciences sector plan: tackling “small population” economics
The UK Government’s Life Sciences Vision and the newer Life Sciences Sector Plan are increasingly being written with rare disease in mind—not only at the level of rhetoric, but in some of the specific structural commitments being proposed that affect small‑population economics.
Rosie points to the way the Sector Plan has been developed in lockstep with the NHS 10‑Year Health Plan, which is critical for ensuring that industrial policy and patient care move together:
“The Life Sciences Plan was developed alongside the NHS 10 Year Health Plan, there’s some bits that appear in both of those, which is great because the two are aligned much better. And we were part of those discussions at BIA as both documents were being developed.”
Two pillars are particularly relevant for rare diseases that struggle to fit a traditional VC model: universal newborn screening at scale, building on the Genomics England project, with an ambition toward 2035. This increases the identifiable patient base, enabling earlier intervention, more efficient trials and more predictable markets.
By explicitly investing in infrastructure that reduces uncertainty—earlier diagnosis, richer longitudinal data, more predictable care pathways—the Sector Plan aims to tackle one of the core reasons venture capital (VC) has historically shied away from the rarest conditions: opacity of patient numbers, trajectories and outcomes.
Health data as an asset: Why 2026 matters for trials and launch decisions
The Health Data Research Service (HDRS) a new initiative designed to make UK health data easier to use for research and trials, including in very small subpopulations, also has a potentially vital role here. Both Martin and Rosie see the HDRS as potentially transformative, especially for rare disease, where data scarcity has long been a binding constraint.
Martin explains the ambition: “It’s going to be hugely important, particularly for rare diseases, because it’s, in theory, going to give access to all patients that are in the NHS. So that level of detail with all the electronic patient records, sometimes matched up with genomic sequencing will allow a much more granular, detailed understanding of these diseases that are within the population.”
For a rare disease patient—or an advocacy group weighing where to encourage trials—better UK data access changes the calculus in several concrete ways:
- Site selection: sponsors can identify clusters of eligible patients more accurately, making the UK a more attractive primary or lead trial location.
- Study design: RWD and linked genomic data enable more efficient designs (e.g. external controls, adaptive designs), which is vital where recruitment is inherently constrained.
- Launch sequencing: A country that can both run strong trials and generate post‑marketing evidence is more likely to be seen as a priority launch market.
Martin also highlights that HDRS must balance large pharma demand with access for small, innovative TechBio and rare disease companies: “If we can make sure that those small, innovative UK companies get access to HDRS as well, they’ll be able to do some incredible things with that data.” He is equally clear about the governance challenge: “It absolutely has to be done with public trust, making sure that the public understand why we’re doing this, how their data is going to be used, the benefits in terms of better medicines, better health care, more efficient NHS. This is really exciting and the public need to come on that same journey.”
For rare disease communities, 2026 and the early years of HDRS will be decisive in determining whether the UK becomes the default home for small‑population trials—or whether sponsors continue to favour the US and other geographies perceived to be more predictable on data access.
Patient voice in policy: Keeping the sector’s “strategic maturing” human
Rosie’s dual role at BIA and as a Trustee of Metabolic Support UK—gives her a vantage point across both the policy machinery and the lived experience of rare disease. “It really does help bring home the purpose of what we’re all trying to do in terms of developing new treatments. Sometimes you can get a bit lost in all the detail and forget that what we ultimately are trying to do is support people living with rare diseases to live happy, long lives.”
She is emphatic that current policy still under‑weights the non‑clinical reality of living with a rare condition: “For most rare disease patients, there’s not a treatment immediately on the horizon, and we want to change that. But making things better also means more joined‑up care, overcoming diagnosis challenges, increasing awareness among healthcare professionals and crucially outside of the healthcare setting as well, supporting people in education and work.”
Rosie argues that the UK Rare Disease Framework, led predominantly by health departments, is no longer sufficient by itself: “Rare disease policy has been largely driven by health departments and that does make sense. But clearly, the impact of rare diseases is not limited to healthcare. As we look to the future of the rare disease framework, we’re really keen to think about how we can bring in that broader piece to ultimately focus on what matters to patients most, and their families.”
Within BIA, several mechanisms aim to keep patient voice embedded rather than peripheral:
A dedicated charity partnership each year—currently Muscular Dystrophy UK—which brings patient speakers and lived experience into major BIA events and conversations, as Rosie expands: “The speakers are always incredibly inspiring and remind us of what it’s all about that we’re working on.”
More systematic patient and charity input into policy development, particularly around evidence expectations and trial design is also key as Rosie explains: “There have been some instances in the past of clinical programmes failing not because the science doesn’t work, but because there’s not been enough engagement with the community on—is this actually what they want, and is there the trust there? And that’s even more of a shame when that happens, because it should be avoidable.”
For Rosie, ensuring that the strategic maturing of the UK biotech sector remains patient‑centric means measuring success not only in IPOs, M&A multiples, or pension fund allocations, but in earlier diagnosis, fairer access, better everyday lives and meaningful involvement of patients in decisions that affect their futures.
For teams at the earliest stages of rare disease research or biotech, the current moment in the UK is demanding but unusually promising. In a high‑conviction market, only rigorously designed, clearly differentiated science that anticipates regulatory and access hurdles will attract sustained backing. Yet the UK is now reshaping its rare disease rulebook through more flexible MHRA pathways, beginning to unlock domestic capital via the Mansion House reforms, and investing in health data and genomics infrastructure that can make even ultra‑rare populations visible and trial‑ready.
For early‑stage innovators, the opportunity lies in building programmes from day one that integrate robust data, TechBio capabilities and UK clinical expertise, while working closely with patient organisations to ensure that what is developed is both investable and truly aligned with community needs.
If those strands are woven together, rare disease ventures that start in the UK have a genuine chance not only to stay and scale here, but to deliver world‑leading treatments from this ecosystem.
You can read BIA’s full analysis and report here:https://biotechfinance.org
To learn more about BIA please visit:https://www.bioindustry.org
Connect with Martin
Connect with Rosie
References:
[1] https://efpia.eu/media/oeganukm/efpia-patients-wait-indicator-2024-final-110425.pdf
[2] https://www.bioindustry.org/resource-report/from-innovation-to-impact-unlocking-patient-access-to-innovative-rare-disease-medicines.html
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