Biologics for paediatric Crohn’s PBS listed to address ‘unnecessary suffering’

Children with severe Crohn’s disease will now have earlier access to first-line biologic therapies through the Pharmaceutical Benefits Scheme (PBS), following persistent advocacy by Crohn’s and Colitis Australia (CCA) and the Gastroenterological Society of Australia (GESA).

Effective 1 August, the change allows eligible paediatric patients to access biologics without first undergoing conventional therapies. This is a shift expected to benefit more than 400 children currently living with severe Crohn’s disease.

The Pharmaceutical Benefits Advisory Committee (PBAC) accepted evidence demonstrating improved clinical outcomes associated with earlier biologic treatment, recognising that delays in accessing effective therapies can have significant long-term consequences for children, including impacts on growth, development and education.

During the most recent Senate Estimates, Senator David Pocock, Independent Senator for the ACT, highlighted the broader regulatory barriers affecting access to paediatric biologic treatments. He noted the limited commercial incentive for pharmaceutical companies to undertake additional Australian Register of Therapeutic Goods (ARTG) registration processes and PBAC submissions for paediatric indications where biosimilars are already available in the market.

Senator Pocock scrutinised the government’s delay in funding effective treatments for paediatric patients, citing a Canberra family with two young brothers living with Crohn’s disease, where one child experienced significant health deterioration on older therapies before improving after accessing biologic treatment. He said the family was now facing the prospect of the younger sibling following a similar path because existing PBS rules were preventing timely access to biologics, exposing the child to “unnecessary suffering” and long-term consequences.

The PBAC’s evaluation of biologic use in paediatrics also supported amendments to address longstanding concerns raised by GESA regarding tightened PBS eligibility requirements.

Patients transitioning from non-PBS-funded treatment to PBS-funded biologics were required to “requalify” for continued access. This created challenges for some patients who had commenced treatment under certain circumstances, including children who began treatment before reaching adulthood and later transitioned into adult care, as well as patients who commenced biologics while living overseas and subsequently moved to Australia.

Meanwhile, AbbVie’s Rinvoq (upadacitinib) has been recommended by the the PBAC for patients with complex refractory fistulising Crohn’s disease represents progress in another area of significant unmet need.

The committee determined that Rinvoq was “likely to be as effective and safe as the other PBS-listed therapies” including infliximab, adalimumab and Stelara (ustekinumab). The PBAC recommended that the therapy be cost-minimised against the least costly comparable treatment, a decision that would make the listing cost neutral or potentially deliver a small saving to the PBS.

However, whether pricing negotiations between AbbVie and the government will result in PBS listing remains unresolved as pharmaceutical companies continue to push back against the undervaluing of medicines by the government.

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