Making gene therapy a reality

The Cell and Gene Therapy Catapult, bluebird bio and the New Statesman brought together a group of experts to discuss the evolving challenges in access to gene therapy.

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Thanks to incredible developments in medical science and technology, treatments once considered theoretical could now become reality. But progress can be slow, and access to these potentially game-changing therapies depends on effective trials, as well as a responsive regulatory and access landscape. Earlier this month, the New Statesman, the Cell and Gene Therapy Catapult, and bluebird bio gathered a group of industry experts and policymakers to take part in a round table discussion, examining the pathways that new gene therapies and cellular interventions take from production through to adoption in the NHS.

Sir John Bell, Regius Professor of Medicine at the University of Oxford and the author of the government’s life sciences industrial strategy, said in his opening address that the United Kingdom should put “infrastructure” in place to ensure that “the National Health Service became a good adopter of new products”. He noted that the Life Sciences Industrial Strategy mentioned cell and gene therapies as an area of excitement. However, he also flagged that in the past the UK had “not made the most” of its leading role in the development of technologies, such as monoclonal antibodies. Therefore, Sir John called for more to be done to realise the commercial opportunities attached to innovation in genetic and cellular medicine. He described the Accelerated Access Collaborative (AAC) initiative – additional government support specifically for “breakthrough technologies” – as a means of encouraging “rapid uptake” of innovative new products by the NHS. But the incentive for uptake depended, he said, on whether the NHS could assess “value” in its decision to commission a certain therapy or treatment.

Innovation, the group heard, is often accompanied by scepticism. This isn’t to say, clarified Matthew Durdy, Chief Business Officer of The Cell and Gene Therapy Catapult, a private sector research and innovation organisation specialising in the advanced therapeutic industry, that healthcare professionals and decision-makers are negatively pre-disposed. Yet, “qualifying and quantifying value” is a “crucial consideration” for the health service. Durdy said he appreciated that “the NHS only has so much money… so of course it has to be rigourous when it comes to what it invests in”.

But Durdy added that while “new technologies could be seen as a risk compared to pre-existing ones”, the life science industry needs to get better at articulating the potential “return” at an earlier stage. Durdy asked: “What is the value of 20 or 30 years of extra life? What does a patient stand to gain from a gene or cell treatment, if it is a cure for the condition they have? That could save the NHS millions in treatment costs over time.” According to Durdy, a lot of discussion about value is stalled by the inability of UK system to adapt to multi-year budgeting in health and social care.

Anne Marie Morris MP, chair of the all-party parliamentary group on access to medicines and medical devices, called for “co-ordinated communication [about gene and cell therapies]” with UK business. Morris stated that political will drives political action and politicians are often moved by their constituents.  “Patient stories are powerful, but we have to think about this more broadly. What does that mean for the UK internationally?” She said that while “most members of the general public” would likely be enthusiastic about the UK being a “world leader in science and technology”, it is important to build further on that and argue the case for “health economics”. If we can communicate how gene therapies will affect everybody, not just rare and ultra-rare disease patients, people may be more likely to be enthusiastic about [taxpayers’] money being spent on new gene therapies and treatments, concluded Anne Marie Morris.

Any chance at effective collaboration between academia, industry and politics, said, Dr Jacqueline Barry, the Chief Clinical Officer at Cell and Gene Therapy Catapult, would be improved by the further development of Advanced Therapy Treatment Centres (ATTCs), joint government-funded ventures, bringing together Industrial, NHS and academic partners to blend experience, expertise and insight. “These dedicated specialist centres are something really unique. This is very much an open collaboration with all parties working together to develop systems to accelerate patients’ access to advanced therapies through the establishment of best practices” for manufacturing, supply and safe and effective delivery [of therapies]. “The idea is to have an integrated supply chain and data capturing mechanisms which will ensure good patient follow up and data capture.” The power of data collection and collation, Barry concluded, should not be underestimated in informing better decision around cell and gene therapy.

Nick Meade, director of policy at Genetic Alliance UK, a national charity working to improve the lives of patients and families affected by all types of genetic conditions,  suggested that the “risk” associated with new drugs and technologies should be looked at differently. The current predilection for cautiousness, however well-meaning, he said, was a risk in itself. “We’re taking so long to decide [on whether a new treatment should be reimbursed] that people are actually dying. That is unacceptable.” Meade lamented the “fragmentation” of commissioning processes, “not least because of devolution”. Meade referred to a compromise recommended by the Scottish Medical Consortium and suggested that the best way to bring new gene therapies to market more quickly was to test them “within the NHS and monitor them closely”. This would, Meade said, provide “real-world evidence in real time… while patients would have the chance to access life-saving treatments” more quickly. Meade added: “The concept of releasing medicines into the NHS may seem a risky thing to do, and you’d worry about being a hostage to fortune. But we can come up with frameworks, collaboratively, between industry and patients and the NHS.” Meade said that patients should be consulted, and could play an active role in rolling out new treatments.

The theme of uncertainty was further pursued by Marc Turner and Mark Briggs, from the Midlands and Wales Advanced Therapy Treatment Centre, and Scottish National Blood Transfusion service respectively. Turner highlighted small patient populations and time-limitations in clinical trials which mean that any long-term benefits of gene therapies are uncertain. Briggs said that uncertainty in cost-benefit assessment is difficult for current HTA methodologies to deal with and collection of real-world evidence is challenging. “It is easier to offer a discount”, Briggs said. Norman Lamb MP asked whether there were any other countries that the UK could learn from in this respect. Sir John Bell, in turn, signalled that some gene therapies may not work, and life-long effects are uncertain, therefore “we have to share the risk”.

Meindert Boysen, the director of NICE’s Centre for Health Technology Evaluation, referring to a report on ATMPs [advanced therapy medicinal products] which focused on CAR-Ts, said that the current methodologies are relevant for assessment of ATMPs. He highlighted, however, the attitude to risk as a key element where NICE takes into account the guidance from NHSE. Blake Dark, the commercial medicines director at NHS England, responded that the recently negotiated Voluntary Pricing and Access Scheme addresses this by allowing the use of commercial flexibility for outcomes-based schemes. According to Dark, outcomes measures in such schemes need to be objective and “obvious”, and also need to be part of care and not to be add on to existing services. Dr Jacqueline Barry added that the development of such outcome measures can be facilitated by ATTCs. Meindert Boysen, went on to explain that decisions on which medicine and therapies to reimburse should also consider their proportionate impact on the wider NHS. While he agreed that game-changing gene therapy could benefit patients suffering from rare conditions, Boysen warned that investment had to have some accordance with rates of incidence. “You’ve got to take the societal benefits into account,” he explained. “It is risky if you start taking money from somewhere else.”

Blake Dark said that while the organisation “truly supports any transformative therapies”, it was important to acknowledge “affordability”. We know that cost-effectiveness comes with some caveats that can put huge strains on the overall health budget and create a cost displacement effect.”. He also highlighted the importance of transactability: “I’m looking downstream at the service provision… One of the CAR-T services in a hospital required the training or re-training of some 300 staff, so you’ve got to take those things into account.” Dark also highlighted that “pricing needs to be fair” when it comes to industry pitching products to the NHS. “We do have some ATMPs already in effect and they are being funded.” Dark stressed that if industry was keen to see swift rollout, then companies had to be “responsible” when modelling their prices.

Nicola Redfern, General Manager UK for bluebird bio, spoke about the fact that industry very much wanted to partner and work with the NHS and NICE to achieve access for patients, and this was supported by Dr Jacqueline Barry from Catapult. One solution to the access challenges offered by Redfern was to have flexibility in the assessment of gene therapies through STA, to go above the QALY thresholds, where large quality of life gains over time would be possible, as happens in HST currently.

Ultimately, the round table concluded, for the swifter but no less safe rollout of gene therapies, all stakeholders had to work together. Decision-makers and influencers would need to address the underlying issues of uncertainty in cost-benefit assessment, and develop innovative risk sharing schemes based on clear patient outcomes measures, such as payment for results over time as suggested by Matthew Durdy, or modifiers for severity or rarity, as Nicola Redfern suggested. Durdy’s vision would see a universal, global and future-proof scheme of patient access to cell and gene therapies. This scheme needs to be feasible and add value, for example by recognising value of therapies outside the healthcare system, such as industrial value. Durdy stated that failure to develop such scheme would mean that UK patients will be secondary patients on a global scale. The UK, it was agreed, has the potential to be a leader in gene therapy and export its expertise internationally.  It was clear at Portcullis House that the appetite to innovate in gene therapies in the UK is there.

This round table event was held in collaboration with the Cell and Gene Therapy Catapult and sponsored by Bluebird Bio.