Written by Thea Collier- Lawlor, Analyst & Josh McLuckie, Analsyt
The well-known challenges faced by developers of advanced therapies for rare diseases have been under close examination in recent times. The cut-price sale of bluebird bio and withdrawal of Pfizer’s gene therapy Beqvez for haemophilia B are the latest events to send shock-waves throughout the industry.
However, in this backdrop, non-industry stakeholders in the wider UK rare disease sector (including non-profits, academic institutions and multi-stakeholder collaborations) are mobilising to ensure that patients do not lose out. Here, we showcase a selection of 3 examples of the exciting, creative approaches these organisations are using to navigate key pinch points in the journey to patient access.
De-risking the early stages of drug discovery and pre-clinical development: The Oxford-Harrington Rare Disease Centre (OHC)
An early hurdle faced in advanced therapy development is successfully making it out of the lab and into the clinic. This critical period between pre-clinical validation and clinical trials is referred to as the ‘valley of death’, and many promising technologies fail to escape it. Reasons for failure at this stage include:
- Lack of funding and resources
- High attrition rates
- Complex regulatory requirements
Enter the OHC’s ‘Therapeutics Accelerator’. This UK-US collaboration between the University of Oxford, Harrington Discovery Institute and venture capital firm Oxford Science Enterprises was established as a novel approach to avoid the valley of death.1 The Accelerator aims to identify, fund and advance innovative academic discoveries. It uses a non-profit/for-profit model that initially provides philanthropic funding and utilises Oxford University’s scientific leadership pedigree in rare disease research. Later, the Accelerator is able to bring in further investment and provides crucial drug development, commercial strategy and business development expertise to advance the promising therapies identified.
The approach may already be bearing fruit. The first Accelerator-funded company (AlveoGene, founded in 2023) recently received FDA orphan drug designation for an inhalable gene therapy to treat an ultra-rare paediatric respiratory disorder.2 Although there is still a long way to go, the news highlights how initiatives such as the OHC Accelerator can help to overcome challenges at this point in the journey to patient access.
Novel ways to clear manufacturing, regulatory and commercial hurdles preventing access to gene therapies for ultra-rare diseases: Great Ormond Street Hospital (GOSH)
There are growing numbers of potentially transformative therapies being abandoned by biopharma companies because of the limited commercial opportunity. One such example is a gene therapy for an ultra-rare, paediatric immune deficiency disorder called ADA-SCID, which was dropped by Orchard Therapeutics in 2021 for commercial reasons despite posting strong clinical data.3
GOSH NHS Trust has taken up the challenge with a unique approach for the UK that bypasses the need for industry involvement. In 2025, the organisation is planning to apply to the MHRA to become the first example of an NHS Trust holding the marketing authorisation for a gene therapy.4 GOSH is uniquely positioned for such a model as it also possesses its own licensed facilities for the manufacture of cell and gene therapies.
With GOSH taking similar steps to other academic/non-commercial organisations in Europe (for example, in Italy, Spain and Germany),5 are we already seeing broader adoption of a development model that involves academic institutions manufacturing and licensing advanced therapies themselves?
Developing an end-to-end pathway to ensure children with rare diseases have access to ‘individualised therapies’: The Rare Therapies Launchpad (RTLP)
Many rare diseases are caused by ‘private mutations’ and require individualised therapies, which are specific treatments developed for one or a few individuals.6 In 2018, a young girl Mila was given an unprecedented second chance with the treatment Milasen, the first fully personalised medicine, supplying hope to patients and families suffering from similar situations.7 Unfortunately, regulatory, commercial and economic barriers continue to limit access to similar treatments.
This is where the RTLP steps up to bat, a multi-stakeholder collaboration that includes the Association of the British Pharmaceutical Industry, GOSH Charity and University College London.8 Their aim is to develop an end-to-end pathway for patients to access individualised therapies by identifying infrastructure needs, mapping stakeholder responsibilities, and creating a sustainable reimbursement system for these medicines.9
In its key pilot, the RTLP will explore antisense oligonucleotide (ASO) therapies for children with rare neurological conditions. Over 3-5 years, real-world experience with this small patient cohort will allow the RTLP to generate evidence to identify the key ingredients for success in building such a pathway.10 The unique challenges inherent to individualised medicines highlight how system-level innovation is required. The RTLP has a crucial role to play in breaking down those market access barriers facilitate patient access.
What does the future hold?
It is clear that new, creative approaches are required to bridge the disconnect between the clinical potential of advanced therapies and limited patient access in some cases. However, the examples discussed above highlight how non-industry stakeholders in the UK are willing to take on the task, with patient access at the heart of a wide range of pioneering initiatives and approaches.
With many of these initiatives in pilot stages, it will be fascinating to follow their progress and see what it means for the wider sector. Could we be in the early stages of seeing radically different models for developing and commercialising advanced therapies? Endorsement of the RTLP in the UK Rare Disease plan demonstrates the commitment of the UK government to exploring such innovative approaches.11 Similarly, beyond the UK, the European Medicines Agency (EMA) will soon be reporting the results of a pilot designed to support academic and non-profit developers of advanced therapies,12 providing clear evidence of regulatory appetite to explore less conventional approaches.
Finally, it is interesting to consider what these developments mean for biopharma companies. In a future world where academic and non-profit organisations play a greater role in the development and commercialisation of advanced therapies, how might the biopharma industry respond?
At P4A, a key part of our mission is to maximise patient access to advanced therapies. We’re especially inspired by the ongoing initiatives in the UK to achieve this goal, and will closely follow developments as they evolve.
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