Owen Marks
Head of Rare Disease, Pfizer UK
There must be a renewed effort to evolve medicine access challenges for gene therapy in the UK in order to help those living with rare genetic conditions.
The last 18 months have been challenging for so many people, and I imagine for the rare disease community it has been particularly tough. As we adjust to living a new type of normal, we must place a renewed focus on improving the outlook for people with rare diseases. Over the next couple of years through scientific breakthroughs, we have a great opportunity to do just that.
Introduction of gene therapy
Following decades of research and development, gene therapy, a treatment which fixes the ‘faulty’ genes that cause illnesses, could become a new standard of care for people with rare genetic conditions.
Unlike other treatments, gene therapy can offer long-term transformative benefits, either eliminating the need for ongoing therapies or reducing the burden of daily disease management and potentially extending the lives of those with rare genetic conditions. It could also help reduce strain on the healthcare system by limiting the need for ongoing interactions with the healthcare system.
Science has really delivered with gene therapy, but with any new advancement it will bring challenges that will change the status quo. In particular, the way in which new medicines are assessed and valued needs to evolve if we’re to embrace this new wave of innovation.
Following decades of research and development, gene therapy, a treatment which fixes the ‘faulty’ genes that cause illnesses, could become a new standard of care for people with rare genetic conditions.
The rare disease gap
For too many years the rare disease community has fallen victim to the rare disease gap, resulting in slow diagnosis and limited access to treatment.1 The National Institute of Health and Care Excellence (NICE) has typically been more geared to assessing medicines that treat tens of thousands of patients or those for ultra-rare conditions, which treat very small numbers of patients. Therefore, many rare disease medicines fall between the gap, not reaching people living with rare diseases.
Evolving assessment of medicines
As we prepare for the introduction of gene therapy, that needs to change. In its Life Sciences Vision, the Government has committed to delivering a high ambition NICE Methods Review. Through the recently launched public consultation on NICE Methods, we have an opportunity to ensure the proposals go further to meet the Government ambition and deliver meaningful change for patients.
Now is the time for the pharmaceutical industry, Government, NHS England, NICE and patient groups to work together to deliver on that promise and ensure that the many years of hard earnt science, results in people with genetic rare diseases being able to access the treatment they so rightly deserve.
This content has been developed and paid for by Pfizer UK
PP-PFE-GBR-3881 | September 2021
[1] Rare Disease UK (2019). Illuminating the Rare Reality. [online] London: Genetic Alliance UK. Available at: https://www.raredisease.org.uk/wp-content/uploads/sites/7/2019/02/Illuminating-the-rare-reality-2019.pdf [Accessed 17 August. 2021].
