Industry urges policy on CGTs as PwC Pathfinder Study shows potential to tackle unmet medical needs

  • Breakthrough cell and gene therapies need White Paper that guides towards policy for adoption by health services
  • Cross-stakeholder dialogue can tie together assessment, access and reimbursement
  • New health information infrastructure can enable real-world evidence collection, linking therapeutic interventions and patient outcomes
  • CGTs already approved by the EMA for diseases that would treat thousands of patients in Ireland if they were available locally 
  • State’s spend of €54m on Treatment Abroad Scheme could be used to help fund access for patients here

The originator biopharmaceutical industry has called for a policy on the adoption of cell and gene therapies (CGTs) by the health services as a new study shows the breakthrough treatments have the potential to tackle unmet medical needs.

The policy would emerge from a White Paper, led by the Department of Health. The industry is keen to engage in structured cross-stakeholder dialogue on a pathway for the urgent adoption of cell and gene therapies by the health services.

The call comes as the industry, through the Irish Pharmaceutical Healthcare Association, today [Thursday] published the ‘Pathfinder Study on the Adoption of Cell and Gene Therapies in Ireland’ which was carried out by the global professional services firm, PwC. The study was supported by Pfizer, Janssen, Novartis, Gilead, Bayer, Takeda and Astellas. It urges a cross-stakeholder approach to realising the clinical potential of CGTs, bringing together government, industry, clinical and patient leaders to create a policy on adoption.

Cell therapy replaces diseased, faulty or missing cells with healthy versions. Gene therapy replaces faulty DNA to cure genetic diseases. These breakthrough treatments, aiming to treat, prevent and potentially cure genetic and acquired diseases, are not yet available in Ireland.

Across the world, cell and gene therapies are either in use or in research for the treatment of a range of disease areas, including cancer, blood disorders, ophthalmology, neurology, and musculoskeletal, metabolic and endocrinological conditions. In the US, the medicines regulator, the Food and Drug Administration, says it expects to approve between 10 and 20 new cell and gene therapies between now and 2025. Europe’s medicines regulator, the European Medicines Agency, has approved cell and gene therapies for diseases affecting the eyes, for gastroenterology, for spinal muscular atrophy, for metachromatic leukodystrophy and for cancer.

PwC examined globally available efficacy data on CGTs and interviewed a range of stakeholders in Ireland, including patient, clinical and industry leaders, in compiling the study. It makes several recommendations that would help patients to access the potentially life-changing therapies, including the development of novel payment models. The key recommendations are:

  • The development of a CGT adoption policy, guided by a structured dialogue led by the Department of Health, which draws together proposals for tackling the related strands of assessment, access and reimbursement;
  • Introduce novel reimbursement models for CGTs that ensure broad access and value for money for patients;
  • Improve the information infrastructure and implement new policy initiatives to enable real-world evidence collection for key disease areas likely to benefit from CGTs in the short term and the start of planning for a broader rollout of CGTs in other areas in the medium term; and,
  • Continue to invest in facilities and staff to ensure a smooth national rollout of CGTs, exploring the creation of centres of excellence at certain hospital sites and allied investment in training and engagement for clinicians and patients.

The study shows that CGTs have achieved ground-breaking results in several therapeutic areas, including in cancer, degenerative diseases (eye disease), neurological diseases (spinal muscular atrophy) and haemophilia. CAR-T, a form of cell therapy, is used to treat leukaemia, lymphoma and multiple myeloma. Two CAR-Ts for three indications in cancer are available to patients in more than 15 EU countries – but not in Ireland. Irish patients are travelling to the UK to receive CAR-T treatment on the Treatment Abroad Scheme. The Treatment Abroad Scheme covers treatments not available in Ireland or where there is delay in getting a treatment. In 2019, the scheme cost the State €54 million, according to the Health Service Executive.

CGTs are usually one-time treatments that can add months, sometimes years, to a patient’s life, replacing a lifetime of treatment. The study argues that, although the upfront cost of CGTs is  significant, they could reduce the direct and indirect costs of chronic treatment for certain illnesses, as well as improving patient outcomes. The study cites cancer, haemophilia and SMA Type 1 as diseases where CGTs could yield economic and clinical upsides.

Oliver O’Connor, Chief Executive of IPHA, said the study was aimed at moving Ireland closer to having a policy on the adoption of CGTs by the health services.

“That will take dialogue and careful planning – but, on the basis of clinical evidence so far, it is the right thing to do for better patient care, aligned with the goals of Sláintecare. We must be open to adopting new innovations affordably and at pace in our health services. This study is an industry contribution to an urgent public policy need. We were keen to gather experts’ perspectives, including from doctors, patients and our own industry. We hope the study can prompt structured dialogue in the form of a cross-stakeholder forum, led by the Department of Health, which would ultimately yield a national policy on the adoption of CGTs in Ireland. This is important for patients and for their clinicians. It recognises, too, that current reimbursement models will not work for CGTs. We need to explore how we can deliver both clinical benefit and value for money from CGT adoption, ” said Mr O’Connor.

Professor David Keegan, a consultant at the Mater Misericordiae University Hospital, Dublin, and a specialist in ophthalmology, said Ireland should move with urgency in seizing the clinical potential of CGTs.

“CGTs are revolutionising medicine. They often offer better treatment options for life-threatening illnesses. In some cases, they have the potential to cure diseases altogether. Gene therapy for ocular diseases is a significant area of research, especially since many rare, blinding retinal diseases do not have treatments now. We can work on the eye more easily because it is an enclosed organ with elements of immune privilege and several identified genetic mutations that could be targeted. The way forward is to have a policy on CGTs that makes it possible for doctors to prescribe approved CGTs for their patients locally,” said Professor Keegan.

Haemophilia is an inherited bleeding disorder where a missing factor means that the blood does not clot properly. The most common type is Haemophilia A, followed by Haemophilia B. Brian O’Mahony, Chief Executive of the Irish Haemophilia Society, said CGTs offer hope for patients with blood disorders.

“Standard treatment since the 1970s has been intravenous infusions of the missing clotting factor. Now, science is offering potentially breakthrough treatments for haemophilia. Last March, the first Irish person with haemophilia B was treated with gene therapy as part of a clinical trial. It was a landmark moment for the haemophilia community in Ireland. We are moving closer to making gene therapy a possible functional cure for haemophilia. This study is an important signpost on a journey towards the adoption of approved CGTs in the health services. The impact on patients’ lives could be transformational,” said Mr O’Mahony.

PwC’s Shane Gannon, the report’s lead author, said he hoped it would lead to healthcare policy change in the near term.

“The new therapies bring clear clinical benefits but they can be expensive. How the health system pays for them, and how their value is measured in the community, are areas for further exploration. We hope this study can prompt a debate among all stakeholders for how Ireland can realise the potential of new therapeutic interventions in a way that makes sense for the healthcare system. Ultimately, we will need a pathway everyone can support,” said Dr Gannon.

The study is available here.

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