From lab breakthrough to NHS treatment: Getting innovative therapies to patients
pharmafile | September 5, 2026 | Feature | Clinical trials supplies & documentation logistics, Due Diligence, NICE / SMC Submissions, Pharmacovigilance, Regulatory Affairs |Â Â OncologyÂ
By Chloe Williams and Sarah Ellson
Innovative therapies are transforming the treatment landscape for serious diseases, particularly cancer. Advances in areas such as immunotherapy, gene therapy and personalised medicine offer new hope for patients with conditions that have historically had limited treatment options. However, turning a promising scientific discovery into a treatment that is routinely available on the NHS involves far more than a successful laboratory breakthrough.
Before patients can benefit, new therapies must navigate a complex pathway involving clinical research, regulatory scrutiny, health technology assessment and NHS funding decisions. While there is significant pressure to make promising treatments available quickly, it is equally important to ensure that they are safe, effective and represent value for the healthcare system.
Building the clinical evidence
The foundation of any new therapy is robust clinical evidence. Before a potential cancer therapy can be used routinely in patients, it must usually be supported by good clinical evidence demonstrating that it is both safe and effective. This typically includes results from the various phases of clinical trials, including showing that the treatment improves meaningful patient outcomes, such as overall survival, progression-free survival, tumour response rates and, where possible, quality of life. Regulators will also look closely at the nature and seriousness of any side effects, particularly for novel therapies such as cell and gene therapies.
This process can be especially challenging. While early studies may show encouraging results, larger trials are often required to confirm that benefits are consistent across wider patient populations and that risks are understood and manageable.
Earlier access to promising medicines
In the UK, there are mechanisms that can provide patients with earlier access to particularly promising medicines. Through the MHRA’s Early Access to Medicines Scheme (EAMS), medicines intended to treat life-threatening or seriously debilitating conditions may receive a Promising Innovative Medicine (PIM) designation and, subsequently, an EAMS Scientific Opinion before formal marketing authorisation, where early data suggests a significant benefit and an unmet clinical need. This can allow eligible patients access to innovative treatments while additional real-world evidence is still being generated.
However, early access does not remove the need for detailed and rigorous testing. Rather, it reflects a careful balance between accelerating access and ensuring patient safety.
Orphan medicinal products
Given the very high costs of developing and the consequential price of medicines which treat rare diseases (address a life-threatening or chronically debilitating condition affecting ≤5 in 10,000 in the UK, or one with insufficient return on investment), there are incentives including full or partial fee refunds for market authorisation applications, scientific advice fee waivers for UK based SMEs, no fee for the orphan designation application itself and most significantly 10 years of market exclusivity (or 12 for certain paediatric medicines)
The role of NICE
A therapy’s journey does not end with regulatory approval. For a treatment to become routinely available on the NHS, it must usually undergo assessment by the National Institute for Health and Care Excellence (NICE).
NICE plays an important role alongside these early access pathways by working with manufacturers, NHS England and the MHRA to assess the evidence being collected and to prepare for potential NHS adoption.
NICE leaves the MHRA to address marketing authorisation criteria but its role is to assess clinical effectiveness and cost effectiveness, this includes consideration of the likely impact on patients’ quality of life when deciding whether a treatment should be routinely funded by the NHS.
For highly innovative therapies, these assessments can be complex. Clinical evidence may be based on relatively small patient populations, and long-term outcomes may still be emerging.
Translating research into real-world care
One of the greatest challenges in healthcare innovation is that encouraging results from laboratory studies or early-stage clinical trials do not always translate into meaningful benefits for larger and more diverse patient populations. Researchers must demonstrate that a treatment delivers consistent clinical benefits, has an acceptable safety profile and can be manufactured and delivered reliably at scale. This can be particularly challenging for advanced therapies such as personalised cell therapies, where manufacturing processes are often complex, expensive and highly specialised.
Another key challenge is generating the breadth of evidence required by regulators, health technology assessment bodies and healthcare systems. While early access schemes can help promising medicines reach patients sooner, long-term data on effectiveness, durability of response and safety are often still needed.
For breakthrough therapies in areas such as brain cancer, the balance between providing rapid access to innovative treatments and ensuring there is sufficient evidence to support their widespread use remains one of the most significant challenges. Early access pathways such as EAMS can help bridge that gap, but robust clinical evidence remains essential before a treatment can become part of routine NHS care.
Looking ahead
Bringing innovative therapies to NHS patients requires collaboration between researchers, manufacturers, regulators, NICE and NHS England. While schemes such as EAMS help accelerate access, strong clinical evidence remains the cornerstone of decision-making.
As medicine becomes increasingly personalised and advanced therapies become more common, the challenge will be to ensure that innovation reaches patients as quickly as possible without compromising the rigorous standards that protect patient safety and support sustainable NHS funding.
The treatments that succeed in navigating this pathway have the potential not only to improve outcomes for individual patients but also to reshape the future of healthcare.
The goal is to strike the right balance between innovation, evidence and access, ensuring that promising scientific advances translate into meaningful benefits for NHS patients.
Pic: Akram Huseyn
- Chloe Williams is Associate and Sarah Ellson is Co-Head of Regulatory at Fieldfisher. The law firm advises a broad spectrum of healthcare clients, including healthcare providers, pharmacies, charities, clinics and manufacturers, on regulatory and compliance matters and specialises in appeals against NICE decisions
