Dialog Box


Timely access not so simple for gene and cell therapy

14 May 2019 at 3:27 PM

A bi-partisan political commitment to a review of the National Medicines Policy is confirmation we have embarked on a journey of technology-driven change.

The National Medicines Policy (NMP) is currently applicable to prescription and non-prescription medicines, including complementary healthcare products.

Its four central pillars remain incredibly important - timely access to the medicines that Australians need, at a cost individuals and the community can afford; medicines meeting appropriate standards of quality, safety and efficacy; quality use of medicines; and, maintaining a responsible and viable medicines industry.

PBAC chair Professor Andrew Wilson, who first called for the review in 2017, said it would provide an opportunity to 're-endorse' the two-decade-old policy.

A review could result in the appearance of a minor change such as broadening the policy's reach to include 'therapy'.

Yet even a name change would have significant implications because of the specific challenges associated with the evaluation and affordability of gene and cell therapies - the detail underpinning the NMP would also need to change.

An example might be applying the objective of 'timely access'.

A re-endorsement of this objective sounds simple, completely obvious, straightforward and uncontroversial.

However, it would almost certainly require a compromise in Australia's current approach to evidence and cost, and maybe even a change to the PBAC's current legislative framework that mandates cost-effectiveness.

The NMP currently says funding decisions must consider 'effectiveness' and 'cost-effectiveness'.

Cost-effectiveness has been a very useful tool to inform payers about value for money for small molecules and biologics - but it is not always clear.

The key advisory bodies - PBAC and MSAC - often highlight 'uncertainty' even when presented with evidence based on late-stage clinical trials involving hundreds and even thousands of patients.

They may not see anything like this kind of evidence with gene and cell therapies - a high level of uncertainty is inevitable to the extent it makes the traditional cost-effectiveness analysis used in Australia completely unreliable and arguably worthless as any guide to the worth of a gene or cell therapy.

Other countries are granting regulatory and funding approval to gene and cell therapies based on early and mid-phase single arm non-comparative studies involving tiny patient populations - and the evidence suggests Australia is already falling behind.

The reality is that a more pragmatic and flexible approach to cost-effectiveness will be needed for gene and cell therapies if the government decides to recommit to the NMP's objective of timely access and ensuring the "quality, safety and efficacy of medicines available in Australia should be equal to that of comparable countries".

Why is this an issue for cell and gene therapies and not conventional medicines?

These therapies are targeted, potentially restorative and even life-saving - waiting for evidence to reduce or even eliminate uncertainty will just mean delays for patients and undermine a potentially re-endorsed NMP objective of 'timely access'.

This is a new challenge for our system - so used to small molecules and biologics - given it has rarely dealt with curative therapies designed to correct the underlying cause of a disease.

MSAC's recently gave very limited approval to Novartis' CAR-T therapy KYRMIAH.

It appears to have made a value and risk-based judgment because the evidence is highly promising but very early - it has limited approval to a very small subset of paediatric patients, but not the larger group of patients with diffuse large B-cell lymphoma. This should probably be seen as evidence of the problem and why a review is so timely.

Dr Amanda Ruth

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