The ARA has called for improved access to medicines for people with rare autoimmune diseases such as Bechet’s syndrome or neurosarcoidosis.
In a submission to the Parliamentary Inquiry into approval processes for new drugs and novel medical technologies in Australia, the ARA said autoimmune diseases such as rheumatoid arthritis and vasculitis were among many conditions that have benefited from PBS approval of breakthrough treatments such as biologics.
However rare diseases such as Bechet’s or neurosarcoidosis will likely never have a phase 3 trial to determine the benefit of an anti-TNF so approvals could not proceed under the current system.
Similarly, other trials unlikely to occur were those of anti-IL-1 drugs for indications such as adult onset Still’s disease or familial Mediterranean fever (FMF).
“Where a phase 3 trial program has shown efficacy and acceptable safety, this is an entirely appropriate mechanism to provide reimbursement approval,” the submission said. “Both TGA and PBS approval are given in terms of a specific medicine for a specific condition.”
“An unintended consequence of this is that patients with uncommon or rare diseases, in which traditional major trials are basically impossible, have no mechanism through which to obtain PBS-funded access to specialised medicine, and never will.”
“As a result, patients who have a clear opportunity to benefit from a given medicine, supported by evidence such as appropriate molecular mechanisms and peer-reviewed case series, cannot gain this benefit.”
“For our members, and their patients, this creates a scenario wherein a rheumatologist might see consecutive patients on a given day whose disease has the potential to be successfully treated with a medicine, one of whom can access this medicine and one of whom cannot.”
The submission, signed on behalf of the ARA by vice president Dr Claire Barrett and its therapeutics committee chair Associate Professor Sean O’Neill, called for a review of access mechanisms to medicines in diseases of high unmet need where the traditional pathway for PBS listing was unfeasible.
“In some cases, this difference is simply the result of human-determined disease nomenclature which classifies patients in, or out, of PBS-funded diagnostic groups for otherwise similar diseases. In other cases, a disease is simply too rare for large scale trials to ever be done, and so under the current regimen PBS-funded access to life saving and/or life changing therapy will never occur.”
“An outcome of this review could include a highly specialised drug approval mechanism, potentially limited to particular specialists and a list of medicines paired to rare diseases with an appropriate evidence base.”
“Under such a regime, subject to approval, patients with these diseases could access specialised medicines subsidised by the PBS. The potential examples of this scenario are many in terms of individual diseases, but few in terms of total patient numbers.”
Dr Barrett told the limbic she was optimistic that there was a way forward.
“The ARA would be delighted to enter dialogue with Government and other stakeholders,” she said.
On behalf of the ARA, Dr Barrett acknowledged Professor Eric Morand for his pivotal contribution to the submission.
Disclosure Statement: Dr Barrett declared UCB had provided an unrestricted education grant for research on bDMARDs in pregnancy, however no personal funds were received.