Rebecca Middleton, Vice Chair for Rare Conditions on the Genomics England Participant Panel, shares her reflections from the Cell and Gene Therapy Conference in April.
With both my patient and advocates hats firmly on, representing Hereditary Brain Aneurysm Support and the Participant Panel at Genomics England, I had the incredible opportunity of attending and presenting at the inaugural Cell and Gene Therapy Conference in Brussels last month.
It was a privilege to be invited to talk about how to build engagement and trust with patient groups. My panel session on day 2 of the conference focused on how to ensure patient experience and insight is reflected in trial design and through the cell and gene therapy ecosystem that promises in a paradigm shift in global healthcare and rare disease treatment.
Our panel was expertly led by Jo Pisani, a member of the board of trustees at Beacon Rare Diseases and included Henny Braund, Chief Executive at Anthony Nolan, William Cole, Life Sciences Partnership Manager at Kidney Research UK and Christos Tsagkaris from the European Health Parliament: a powerful array of voices with a huge amount of experience in this space.
The session included lots of great questions and discussion, with all speakers agreeing that only by including patients early in the development phase and building meaningful relationships that impact culture and strategy will trust be built in the cell and gene therapies that will shift and evolve the standard ‘lifetime of care’ to a ‘one and done’ system.
During my presentation, I highlighted the challenge faced by small patient groups, often referred to as the 'David’s' in the ecosystem, when approaching large pharmaceutical companies or healthcare providers, the 'Goliaths'. Conversely, we also discussed how the 'Goliaths' sometimes struggled or were uncertain about establishing connections with the 'David’s’. It is through dialogue and an understanding of patient insights and patient-relevant outcomes that the practicalities of trial design and therapy development can be improved and made sustainable in the long term.
Around 300 healthcare experts and participants took part over the 2 days. They heard panel discussions on subjects ranging from democratising healthcare access to therapies, bringing policy up to speed, investor insights and the potential of transformative therapies in rare diseases. Through all of these sessions, the patient experience was reflected, though through the industry lens rather than hearing from patients or their groups directly. Our session, in particular, brought the patient voice firmly in, as we reflected on case studies, best practices and the power of the patient to support generation of real world evidence, new patient-driven therapies and their valuable and powerful influence in policy and framework.
Right now, 27 cell and gene therapies are licensed, with around 2,000 more in various stages of development. The cell and gene wave is coming. To prepare, the market, society, regulators, healthcare systems, public and patients must get ready to unlock innovation and the many benefits and risks of cell and gene therapy.
Other key topics of the conference included:
Frameworks and regulation
More innovation and incentives to drive the market in the EU, with an added challenge of how to support the UK when it’s out of the EU but so important to the whole market. The challenge of regulation across global borders and the EU, ensuring it’s supporting an equitable market while realising it can’t be based on ‘standard healthcare’ systems.
Cost versus value
How do payers, globally, decide on a framework that is socially acceptable and respects the full value for patients, families and healthcare systems. Who should get the economic value? Therapy developers, healthcare providers, patients?
Cell and gene therapies are receiving the lion's share of life-science funding, according to investors. So how can we balance interests and conflicts in areas where there are little to no treatment options for rare disease patients, and how do their valuable ‘needs’ fit in?
The importance of data
There is a need for more real-world data for different agencies' approvals and research. Global regulators need different levels and types of data; what is available and requested by regulators today is not fully appropriate for novel cell and gene therapy development. How can patients play an active role in generating data and working in partnership to bring forward approvals?
‘Science is there to serve’ and ‘therapies are not developed on an island’ – were 2 stand-out quotes from the conference. It’s clear that we need an approach that brings all stakeholder voices in and balances the differing priorities, benefits, and barriers to access. Strong collaboration matters – cell and gene therapy is a team sport.
The novelty of cell and gene therapies can’t be ignored
There is a fear of ‘miracle cures’ and a societal angst about therapies that change our cells and our bodies for our lifetime. As we move from treatment of symptoms to ‘one and done’ treatments, a different patient pathway is needed, and long-term relationships are formed as ongoing surveillance and evaluation continues. We need a new cell and gene therapy patient engagement playbook.
Equity of access
Ethical challenges were addressed across both of the 2 days, including much discussion around public attitudes about advanced therapies in an emotionally and politically charged environment. Equity of access came up as a key concern, raised by a number of speakers, including the chair of Sickle Cell Society.
There is a mountain to climb to ensure equitable access to all rare disease areas and to all corners of the globe, including those with challenging healthcare and payment structures.
Some final thoughts…
Genomics England is a ‘Goliath’ compared with many in the ecosystem and its National Genomic Research Library holds the largest global research collection of whole genome sequences from patients with cancer and rare diseases. The organisation is already playing a critical role in the development of innovative treatments. Academic, clinical and industry researchers work together on the data in the NGRL, looking for patterns in the de-identified data of thousands of participants and patients, to help make breakthroughs in genomic science that could lead to more therapeutic discoveries and opportunities for clinical trials, which includes cell and gene therapies.
Hereditary Brain Aneurysm Support is very much a ‘David’. We’re young, we’re growing and we’re learning. Our disease is yet to have its genetic foundation confirmed but we remain hopeful for answers in the years ahead. Identifying the genetic cause will, in theory, unlock potential exploration for future cell and gene therapies. This is still far down the track for our condition, but learning as we go and taking part in these valuable discussions means we’re ready to start conversations when the time is right.