Unveiling the reform options in HTA Review’s second consultation

Pharma News: The Health Technology Assessment (HTA) Policy and Methods Review has reached a crucial milestone with the unveiling of the Consultation 2 Options Paper. A key deliverable outlined in the Strategic Agreement between Medicines Australia and the government, the HTA Review seeks to minimise barriers for patients to access innovative health technologies.

Medicines and therapies have been a major contributor to improved healthy life expectancy over the past century. At the beginning of 2010, the most expensive medicine on the PBS was ~$24,000 each time a patient filled a prescription for the maximum quantity. By 2020, it was ~$110,000 whereas, today, the most expensive medicine on the PBS is ~$2.5 million, mostly due to gene therapies’ potential as one-time, curative treatments.

Navigating Submissions and Price Negotiations: A Complex Web

Australia finds itself positioned in the middle among Organisation for Economic Co-operation and Development (OECD) countries for the time it takes for a new therapy to be reimbursed. In 2021-2022, the median timeframe for PBS listing of a new drug from Australian Register of Therapeutic Goods (ARTG) registration was 21 months, while the maximum timeframe stretched to 84 months. Notably, vaccines, advanced therapies, and medicines funded through specialised programs experienced extended timelines.

“The main reason for this source of delay to funding new health technologies is that the economic evaluation in submissions is being used as a proxy for negotiation of the prices to be paid for health technologies,” stakeholders told the HTA Review Reference Committee.

An intriguing correlation has been identified between the number of submissions to the Pharmaceutical Benefits Advisory Committee (PBAC) and the extent of price reductions. The average price reduction between first and last submission for health technologies submitted 2 times was 30%. This increased to 70% for health technologies submitted 4 times.

Stakeholder Engagement: A Paradigm Shift in Transparency

Patients and clinicians grapple with unequal knowledge of the application, evidence submission, and HTA process. The Options Paper proposes the development of an engagement framework to ensure involvement of consumers and clinicians “earlier and more consistently throughout the HTA processes, including clear and transparent guidance about how input should be prepared and is used by committees.”

To address concerns related to First Nations people, the establishment of a First Nations Advisory Committee in addition to the appointment of a First Nations representative on the PBAC is suggested.

Revamping Funding and Assessment Pathways: A Staged Approach

The existing separation of pathways based on technology types is deemed historical and increasingly complex to navigate, especially for new health technologies such as co-dependent technologies and cell and gene therapies.

To address this, the Options Paper recommends a staged approach to developing a single-entry gateway for HTA submissions. In the short term, it suggests making the PBAC the sole committee for medicines of ultra-rare diseases, eliminating double handling. Additionally, it suggests streamlining the pathway for listing of a vaccine on the National Immunisation Program (NIP) by removing the requirement for the sponsor to get ATAGI advice prior to submission, and developing criteria to have the assessment of vaccines be proportionate to the level of risk of the product.

Looking to the medium term, the Options Paper envisions expanding the role of the PBAC for a broader range of health technologies, including co-dependent health technologies. In the long term, the vision is to develop a unified, national HTA pathway encompassing all health technology evaluations. The proposed committee would be appropriately resourced, drawing on specialist expertise as needed for diverse technologies seeking public funding.

Methods for HTA: A Holistic Approach to Government Subsidy

The Options Paper highlights stakeholder concerns about the choice of comparator, pricing implications, and the lack of stakeholder input into the PICO (population, intervention, comparator and outcomes) stage. The proposed reforms include increased early input from patients and clinicians to ensure a more nuanced consideration of relevant patient populations and outcomes.

Given the evolving evidence base for health technologies, particularly for rare diseases and biomarker-targeted therapies, the Options Paper also suggests the development of a qualitative value framework on the use of non-traditional evidence, including indirect comparisons, use of surrogate outcomes, and the use of ‘Real-World Data’ (RWD) and ‘Real-World Evidence’ (RWE).

Economic Evaluation: Recognising the Full Value

A critical aspect of the Options Paper addresses concerns about economic evaluation methods and the perceived undervaluation of novel health technologies. The pharmaceutical industry believes the full value of health technologies is not being recognised due to elements such as comparator selection, high discount rate, approaches to uncertainty, societal and equity principles, and distributional impacts.

To address these concerns, the Options Paper proposes clarifying the selection of comparators relevant for submissions claiming superiority as opposed to non-inferiority. In addition, it suggests developing models to assess the impact of HTA Review recommendations, considering different scenarios with varying discount rates for technologies like vaccines and gene therapies. Consulting stakeholders to gauge their opinions on accepting higher prices for therapies in specific circumstances is also being proposed.

Navigating Uncertainty: Innovative Approaches

Managing uncertainty in clinical outcomes, value for money and budget impact is a key concern for stakeholders. The Options Paper introduces alternative instruments and financing tools to address budget impact, time-limited funding for therapies in areas of high unmet clinical need, and post-listing reassessment of health technologies.

Next Steps

The HTA Review Consultation 2 commenced on 25 January and will close at on 23 February 2024, with online and in-person workshops planned to take place throughout February. Following this process, the HTA Review Reference Committee will prepare a final report with recommendations which will be provided to the PBAC and the government in April for consideration. 

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