'Divesting' older technologies will be required

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The rapid emergence of precision medicine will have significant implications for patients and means the government will need to consider divesting existing interventions and technologies, according to a new whitepaper.

The whitepaper developed following a stakeholder roundtable on precision medicine convened by MTPConnect has focused significant attention on the need to ensure 'fit-for-purpose' regulatory and reimbursement systems.

MTPConnect is the federal government funded Growth Centre for Australia’s medical technology, biotechnology and pharmaceutical sector.

Its roundtable involved participants from the pharmaceutical, biotechnology and medical technology sectors. It also involved researchers, government agencies and members of key advisory bodies.

Precision medicines like gene and cell therapies face significant hurdles in current regulatory and reimbursement processes, according to the whitepaper.

One challenge is the high upfront cost. Another is the need for decision-makers to have "robust evidence" they will deliver improved patient outcomes compared to existing medicines, treatments or other interventions.

Evidence collection is a challenge because trials of precision medicines like gene and cell therapies often involve a very small number of patients suffering from rare disorders. 

The high upfront cost, combined with limited early evidence in small patient groups but the potential to be curative, creates uncertainty. This is a challenge when it comes to navigating current regulatory and reimbursement processes.

The whitepaper has identified other "uncertainties" related to the "lack of clarity of requirements and standards across these varied and rapidly evolving technologies."

"Regulatory and reimbursement process needs to be fit for purpose," it says.

"Given the complexity and pace of evolution of technologies, Precision Medicines will challenge current legislative and regulatory structures. A central issue was the generation of evidence to support regulatory and reimbursement submissions – as these would be challenged by changes in clinical trial design, the availability of real-world data and increased integration of artificial intelligence and algorithms into analysis and interpretation of data and therefore clinical practice."

Unlike the existing paradigm of many pharmaceutical interventions, which treat symptoms, gene therapies have the potential to be curative by correcting the underlying cause of a disease. They could restore the health of an infant with a severe and life-threatening genetic disorder, effectively giving them a normal healthy lifespan. This will have implications for existing treatments and interventions, including medicines.

"The importance of disinvesting in technologies that are replaced by precision medicine, otherwise the claimed efficiencies/benefits will not be realised," says the whitepaper.

It says there must be action to ensure Australia keeps pace with "current with international practices" and remains an "active and valued player in the medical technology industries".