Australia's Cell & Gene Catalyst (the Catalyst) has called for streamlined and equitable access for cell and gene (C&G) therapies in Australia in its response to the Department of Health and Aged Care's Health Technology Assessment (HTA) Policy and Methods Review - Consultation 2.
As part of the HTA Review, an Options Paper was developed to present options for reform being considered by the reference committee to improve Australia's HTA policies and methods and the funding and approval pathways.
The purpose of the HTA is to provide necessary information to understand the benefits and comparative value of health technologies and procedures. Commonly applied to pharmaceuticals including vaccines, diagnostic tests, medical devices and other public health interventions, the HTA assists government funding decisions.
In its submission, the Catalyst called for three options to be significantly strengthened to enable the growth, development, and translation of C&G therapies in Australia. These were:
- Option 1.4: The Catalyst supports a nationally cohesive approach to HTA for technologies that are currently jointly funded, however considers the ideal way to simplify and streamline access pathways for cell and gene therapies is to both assess and fully fund these advanced therapeutics at the Federal level. Regular consultation, communication and collaboration with States and Territories will continue to be important for these products. To enable holistic consideration of product costs (therapy plus delivery), States and Territories would need to articulate the total costs of product delivery for inclusion as inputs into the HTA process from the beginning.
- Option 3.3: Reducing the discount rate not only recognises the value of long-term future benefits – it also brings Australia in line with international best practice. Cell and gene therapies are designed to have a lifelong, potentially curative impact. Treatment involves an upfront cost followed by potentially lifelong benefits for the patient, their family and society in general. Australia's high discount rate means our HTA system is ill-equipped to adequately value the lifelong benefit these treatments can offer.
- Option 4.3: To ensure equal and equitable access to cell and gene therapies, a high-level principles-based framework for accepting and assessing real world evidence, non-randomised control trial evidence and surrogate health outcomes is required. Conditions treated by cell and gene therapies can be rare or extremely rare which means they can have small patient cohorts in Australia. This means recruiting participants for clinical trials is more challenging and generating randomised control trial evidence more difficult, emphasising the value of RWE. Real world evidence (RWE) and broader societal, health and economic benefits can be transparently considered and used in HTA decision-making – potentially leading to faster, more equitable patient access.
The submission stated, "Given Australia's current systems were not set up for these potentially revolutionary therapies, addressing this priority areawill enable health system readiness and industry growth, ensuring everyone in Australia will have access to these advanced therapeutics."
The Catalyst is a national joint venture of the industry peak bodies AusBiotech and Medicines Australia, and aims to accelerate Australia's cell and gene industry to ensure everyone in Australia has access to world-class advanced therapeutics.
The Catalyst partners and Expert Steering Group, comprised of Cell Therapies, CSL, Novartis, Pfizer, Roche, and Therapeutic Innovation Australia, endorse Medicines Australia's recommendations and responses to the HTA options paper in full.
The reference committee will consider all evidence and input received during Consultation 2 and throughout the review as it develops its final report and recommendations to the Australian Government. The review is due to be completed by 15 April 2024.
Read the Catalyst's full submission here.
