Patients with myasthenia gravis may be unable to access new therapies for the condition because Australia’s PBS reimbursement system makes it difficult to list treatments for rare diseases, industry experts have told a Parliamentary inquiry.
Treatments such as rozanolixizumab and zilucoplan are now in late-stage trials for the treatment of myasthenia gravis but manufacturers say they face major challenges and costs in obtaining sufficient data from small numbers of patients to gain approval from reimbursement bodies such as the Pharmaceutical Advisory Committee (PBAC).
Speaking at a public hearing for the “Approval processes for new drugs and novel medical technologies in Australia” inquiry at the House of Representative on 26 April, Selina Clifford of UCB Australia said there was a need to create a different approval pathway for novel medicines for conditions such as myasthenia gravis that affected 3000 people in Australia.
“The gold standard for evaluation is randomised control trials, but this is not generally possible for rare diseases,” she told the inquiry.
“For example, it is difficult to account for the variation of symptoms with rare diseases, the lack of established diagnostic criteria and the difficulty in conducting large-scale clinical trials.”
Furthermore, the costs of undertaking the regulatory and reimbursement processes were considerable for sponsors, since not all rare disease therapies would meet the eligibility criteria for orphan drug designation and the corresponding fee waivers.
“ In Australia, the alternative response for rare diseases is not to seek registration and reimbursement but rather then to supply on a patient-named basis under a special access scheme. This is a not a long-term solution, due to the costs borne by the patients and the considerable administrative burden on the healthcare system and the doctors who have to apply continuously for access to these medicines,” she said.
Industry representatives suggested a separate approval pathway that could include greater input from clinicians and carers and assessments in areas such as quality-of-life for patients.
“While the Pharmaceutical Benefits Advisory Committee processes welcome clinician and patient input to the submissions, the weight given to these important insights is limited in that they are not part of the PBAC decision-making process,” Ms Clifford noted..
“In addition, the assessment could consider the broader impacts of a medicine on the economy, including how a medicine enables increased workforce participation, which would provide an outcome that is more indicative of the true value of these medicines. This could be achieved through the establishment of an evaluation body which would include specialised expertise familiar with the complexities of rare diseases and their therapies,” she suggested..
Agents in phase 3 trials for MG include rozanolixizumab, a monoclonal antibody that acts by selectively binding neonatal Fc receptor (FcRn) protein, leading to reduced levels of circulating autoantibodies. Another investigational drug, zilucoplan, is a complement C5 inhibitor for the treatment of acetylcholine receptor autoantibody–positive generalised myasthenia gravis.