New report says time to prepare for cell and gene therapies

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A new report commissioned by Novartis says change is required to ensure Australian patients are able to access the more than 2,000 cell and gene therapies currently in development with the US FDA expecting to evaluate 10 to 20 each year by 2025.

The report - 'Cell and Gene Therapies: Rising to the Challenge' - was developed by consultancy Evohealth.

It says the potential for improved outcomes delivered by cell and gene therapies (CGT) is "indisputable" but that change is required to ensure Australia's health system is prepared for and able to accommodate these innovations that often target small patient populations with a severe and unmet need.

"They can treat not only inherited genetic disorders but also some cancers and viral infections. Many CGTs target rare diseases that have a high unmet need. These diseases often have limited or no treatment options, such as cystic fibrosis, some ocular and a range of neuromuscular conditions," says the report.

The report points to research that shows around 80 per cent of rare diseases are genetic in origin and highlights the need for early diagnosis through enhancements to programs like newborn screening.

"Standard Australian newborn screening covers only a fraction of the estimated 5,000 inherited rare diseases," says the report.

The Department of Health recently announced a complex and lengthy multi-staged new process for the consideration of adding conditions to Australia's newborn screening program. The new process does not end with any commitment to funding the addition of the new conditions.

The Evohealth report highlights the complexity and limitations of Australia's current health and technology processes with the path for cell and gene therapies essentially determined by their administration.

The Pharmaceutical Benefits Advisory Committee (PBAC) is limited to evaluating and providing advice on technologies if they are suitable for the PBS or National Immunisation Program. The Medical Services Advisory Committee (MSAC) evaluates technologies that involve the provision of a health service, such as the processing of human cells outside the body and their administration to the patient.

MSAC is currently evaluating CAR-T cell therapies and a gene therapy for a genetic eye condition while PBAC is evaluating a gene therapy for Spinal Muscular Atrophy. The different pathways and approaches to evaluation do raise the risk of the inequitable treatment of patient need. The report says this pathway complexity is compounded by existing system inequalities for patients living with severe and rare diseases.

"CGTs provide an important and urgent opportunity for our Government to address health inequity, consistent with what society expects. Rising to the challenge of evaluating and funding safe and effective CGTs, allows the Government to level the playing field for those Australians that need more support," says the report, comparing the current situation with that in the UK where the National Institute for Health and Care Excellence (NICE) has proposed the inclusion of severity of disease and broader health inequalities into its evaluation methods.

A senior representative of NICE recently told the current Australian parliamentary inquiry into approval processes for new drugs and novel medical technologies that the agency has an explicit focus on promoting innovation and early patient access to new technologies. Australian decision-making processes do not have a similar mandate.

The UK has also established the Accelerated Access Collaborative and a Cell and Gene Therapy Catapult to provide clinical, technical, regulatory, and market access expertise for CGTs. The Evohealth report recommends the creation of an Office of Rare Diseases as an agency in the Australian Department of Health.

When it comes to the health technology assessment of CGT therapies, it says their potential to deliver significant long-term benefits or even be curative by fixing a disease 'at its source' represents a paradigm shift in patient outcomes but that it is currently confounded by their newness, a lack of long-term data and an evaluation process that will significantly discount those long-term benefits. 

Discounting penalises interventions like vaccines and CGT therapies that deliver long-term benefits. Australia's annual 5 per cent discount rate of health gains compares to 1.5 per cent in Belgium and The Netherlands. "Belgium, justifies this differential discounting (1.5 per cent for effects) to avoid penalising interventions that deliver most of their benefits in the future," says the report, adding some systems also apply different discount rates based on the severity of a disease.

It says evaluation processes should also consider the wider societal benefits of CGT therapies for patients with rare and severe diseases, including productivity gains and a reduction in direct and indirect costs.

The authors and contributors highlight the need for change to ensure Australia's health system, which is based on a complex mix of jurisdictions and payers, is prepared for the wave of these therapies that are starting to emerge from company pipelines. It also recommends formal horizon scanning, the creation of an expert advisory committee to support PBAC and MSAC in the consideration of CGT therapies and a national cross-jurisdictional approach to consider the evaluation and funding of CGT therapies.